NIH’s new Application Submission System & Interface for Submission Tracking (ASSIST) is available for the electronic preparation and submission of multi-project applications through Grants.gov to NIH. Applications to this FOA must be submitted electronically; paper applications will not be accepted. ASSIST replaces the Grants.gov downloadable forms currently used with most NIH opportunities and provides many features to enable electronic multi-project application submission and improve data quality, including: pre-population of organization and PD/PI data, pre-submission validation of many agency business rules and the generation of data summaries in the application image used for review.

Required Application Instructions

It is critical that applicants follow the instructions in the SF424 (R&R) Application Guide, except where instructed to do otherwise (in this FOA or in a Notice from the NIH Guide for Grants and Contracts) and where instructions in the Application Guide are directly related to the Grants.gov downloadable forms currently used with most NIH opportunities. Conformance to all requirements (both in the Application Guide and the FOA) is required and strictly enforced. Applicants must read and follow all application instructions in the Application Guide as well as any program-specific instructions noted in Section IV. When the program-specific instructions deviate from those in the Application Guide, follow the program-specific instructions. Applications that do not comply with these instructions may be delayed or not accepted for review.

Part 1. Overview Information
Part 2. Full Text of the Announcement
Section I. Funding Opportunity Description
Section II. Award Information
Section III. Eligibility Information
Section IV. Application and Submission Information
Section V. Application Review Information
Section VI. Award Administration Information
Section VII. Agency Contacts
Section VIII. Other Information

The purpose of this Funding Opportunity Announcement (FOA) is to solicit applications for Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers (MDCRCs). These Centers promote collaborative basic, translational and clinical research and provide important resources that can be used by the national muscular dystrophy research community. The Centers also provide outstanding environments for the training of new scientists electing to pursue careers conducting research in high priority areas of muscular dystrophy. Finally, Center investigators are expected to participate in community outreach efforts to increase awareness and convey the importance and implications of their research activities to the patient and advocacy communities.

The Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2001 (the MD-CARE Act, Public Law 107-84) specified a number of provisions for expanding and intensifying research on muscular dystrophy. One provision of the MD-CARE Act was that the NIH establish centers of excellence for research on muscular dystrophy. The MDCRCs program was subsequently developed in honor of Senator Paul D. Wellstone, a champion of muscular dystrophy research. Through open competitions and peer review of applications for awards, the participating NIH institutes established three Centers in 2003, three more in 2005 and have since maintained six active MDCRCs.

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Neurological Disorders and Stroke (NINDS) are committed to continuing and enhancing the tradition of scientific excellence that has been fostered in the MDCRC program.

Muscular dystrophy refers to a group of hereditary, progressive degenerative disorders causing weakness of the skeletal or voluntary muscles. There are many different forms of muscular dystrophy, which differ in their age of onset, severity, and pattern of muscles affected. Most types of muscular dystrophy are not simply muscle disorders, but rather multi-system disorders with manifestations in a variety of body systems, including the heart, gastrointestinal system, endocrine glands, skin, eyes, brain, and other organ systems. The major forms of muscular dystrophy include congenital, distal, Duchenne/Becker, Emery-Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal. Although some forms first become apparent in early childhood, others may not appear until middle age or later, but all have a significant clinical, economic, and psychosocial burden of disease.

Currently available therapies are very limited in their ability to change the clinical course of the diseases. But, there have been significant advances in recent years in early-stage clinical trials and preclinical development of a wide range of candidate therapeutics that address specific disease mechanisms. Furthermore, recent advances in genetics, pathogenic mechanisms, therapeutic technology, and patient diagnostics provide additional, compelling opportunities for advancing translational and clinical science in the muscular dystrophies. The availability of effective, disease-modifying treatments for all dystrophy patients is likely quite some time away. Symptomatic treatment, though not able to stop disease progression, is also an important goal of research, and may improve patient quality of life. In addition to the need for new disease- or symptom-modifying therapies, there are gaps in understanding how the muscular dystrophies affect the lives of patients and their families and interfere with social interactions, education and career plans. There may also be missed opportunities for optimizing health care and improving accesses to care and services. Advances in therapy development and improvements in understanding the factors affecting patients' lives may lead to novel strategies to lessen the burden of these diseases.

The Action Plan for the Muscular Dystrophies ( http://www.ninds.nih.gov/about_ninds/groups/mdcc/MDCC_Action_Plan.pdf ), approved and released in January 2006 by the interagency Muscular Dystrophy Coordinating Committee (MDCC) (http://www.ninds.nih.gov/about_ninds/groups/mdcc/ ), is a consensus document with input from patients, advocacy groups, basic scientists, clinicians, and Federal agencies that highlights many of these scientific opportunities and the need for broad cooperation in seeking effective treatments for the muscular dystrophies and decreasing disease burden. The Action Plan is being updated, based on the recommendations of panels of experts on disease mechanisms, translational research, diagnosis and screening and disease burden. The MDCC will take up approval of the updated plan later this year.

NIAMS, NICHD, NHLBI and NINDS seek to further develop the MDCRC program and continue to advance research in the muscular dystrophies leading to effective treatments and other strategies for reducing disease burden. Under this FOA, each Center application should propose clinical research and may also include synergistic basic and/or preclinical translational research addressing important goals and hypotheses. Applications should contribute to the long-term goals of developing therapies and reducing the burden of one or more form of muscular dystrophy, as well as the training, research resource sharing and patient/community outreach goals described below. Projects should be focused only on muscular dystrophy research and may include studies of the impact of these diseases on skeletal muscle, the heart, pulmonary system, smooth muscle, the central nervous system, gut or other organ systems as well as neuropsychological or neurobehavioral studies. Areas of research can include, but are not limited to the following:

• Identification, characterization and validation of therapeutic targets
• Development and validation of useful pharmacodynamic, predictive or prognostic biomarkers (molecular, biochemical, physiological, imaging, etc.)
• Development and validation of outcome measures or surrogate markers for use in clinical trials
• Development and characterization of new cell or animal models of disease
• Screening, optimization and preclinical efficacy testing of candidate therapeutics
• Activities leading to an investigational new drug (IND) or investigational device exemption (IDE) application to the FDA
• Clinical studies aimed at clinical trial preparation including natural history studies
• Epidemiological, behavioral or health outcomes studies with the eventual goal of reducing the burden of disease
• Early-stage clinical trials (proof of concept trials, safety/tolerability, dose ranging studies, etc. However, inclusion of phase 3/registration trials is not permitted.)

MDCRC applicants are encouraged to consider projects that address the high priority areas described in the Action Plan for the Muscular Dystrophies ( http://www.ninds.nih.gov/about_ninds/groups/mdcc/MDCC_Action_Plan.pdf).

The research problems proposed should require substantial collaborative efforts to solve, and thus are best carried out in a center setting. Collectively and in cooperation with the NIH, the MDCRCs form part of a coordinated national program. Applicants are expected to emphasize new ideas, novel approaches, and state-of-the-art technologies to address the needs for effective treatments and other strategies to improve the lives of muscular dystrophy patients. Applications should include multidisciplinary collaborative efforts, in particular those involving basic scientists and clinicians with appropriate expertise. MDCRC applicants must also propose resource core facilities, training and outreach activities that will enhance muscular dystrophy research on a national or international level.

Applicants seeking support for projects that are stand-alone, single-component basic, translational, clinical studies or trials in muscular dystrophy should contact the Scientific/Research Contact listed in Section VII. Agency Contacts below for guidance on other, more appropriate funding opportunities.

The organizational structure of the proposed MDCRC should facilitate the flow of new scientific findings and technologies into translational and clinical research. Each center must include clinical research as defined in the PHS398 Supplemental Instructions Part II (http://grants1.nih.gov/grants/funding/phs398/phs398.html).

Clinical research is research with human subjects that is:

1. Patient-oriented research. Research conducted with human subjects (or on material of human origin such as tissues, specimens, and cognitive phenomena) for which an investigator (or colleague) directly interacts with human subjects. Excluded from this definition are in vitro studies that utilize human tissues that cannot be linked to a living individual. It includes:

• mechanisms of human disease
• therapeutic interventions
• clinical trials
• development of new technologies

2. Epidemiological and behavioral studies.

3. Outcomes research and health services research.

http://grants.nih.gov/grants/glossary.htm#ClinicalResearch

Although guidance is provided in this FOA for interventional clinical trials in MDCRCs, clinical trials using investigational drugs or biologics are NOT a required element of an MDCRC. Each center may also contain basic and/or translational studies research with an emphasis placed upon moving the research field forward toward novel or improved therapies or other strategies for reducing disease burden for the muscular dystrophies.

The minimal structural requirements of a Wellstone MDCRC under this FOA are:

• Two or more projects involving collaborations of investigators. At least one of these projects should address clinical research aims and should involve the direct interaction of Center investigators with MD patients;
• A Center Director and Co-Director, responsible for scientific and administrative oversight of the Center;
• An Administrative Core that promotes a Center environment through enhanced communication and collaboration of investigators within and outside the Center. The Administrative Core should also promote awareness in the patient and advocacy communities regarding research objectives and strategies, and promote enrollment in clinical studies. The Administrative Core will establish a Center Advisory Committee including external scientific and lay members (members of the Advisory Committee should be identified in renewal applications only; applications for new Centers should not identify candidate Advisory Committee members or contact them prior to award);
• A Scientific Research Resource Core that is to be shared with the national muscular dystrophy research community and meets the needs of the community. A Center can have other cores that are justified by the level of use within the Center, but are not necessarily shared with researchers outside the Center; and
• A Training Core to enhance the Center’s training environment and provide stipend and research support for predoctoral, postdoctoral and clinical fellow trainees.

Projects

Each of the proposed research projects should address problems that require a substantial collaborative research effort to solve, and are best suited for a center environment rather than a stand-alone grant. Collectively, the projects should involve synergistic teams of researchers with complementary expertise such as basic and clinical, skeletal muscle and other organ systems, primary data collection and bioinformatics, etc. Collaborations should be arranged to bring the best expertise to bear on a problem, whether the proposed collaborations are all on-site or utilize consortium agreements with off-site investigators at existing MDCRCs or off-site investigators not affiliated with an MDCRC. Although a clinical project is required, this need not be a clinical intervention trial. See section IV.2 for additional guidance on projects that propose a clinical trial. Epidemiological, behavioral and health outcomes research studies for the muscular dystrophies are also encouraged.

Leadership

The Center Director and Co-Director should develop and maintain a center environment that fosters traditional and novel approaches to multi-disciplinary research collaborations and training. The Center Director and Co-Director cannot serve as the Program Director/Principal Investigator of a project in another active MDCRC award. But, other than this restriction, collaborations among Centers are encouraged.

Administrative Core

The Administrative Core should provide for the integration and management of activities within the MDCRC. The Administrative Core should also promote interactions and communications between the research and patient/advocacy communities. Funded MDCRCs are expected to utilize the Administrative Core to establish and maintain a website to communicate the Center mission and the availability of training opportunities and Scientific Research Resource Core services. Each Center should expect to form an external Center Advisory Committee (CAC) with scientific, clinical and patient advocate representation, composed of at least five members. The CAC should meet in-person or electronically approximately once a year, beginning in the first or second year of the Center award. Applicants are encouraged to form the strongest teams to address the research questions, regardless of geography, and the Administrative Core should be responsible for coordinating communication among the center sites and integrating participating researchers into a cohesive center environment, even if geographically dispersed.

In order to promote awareness of muscular dystrophy research and the Wellstone Centers program in the patient/advocacy communities, the Administrative Core should develop activities or materials, such as seminars, web-based information, or lab tours involving patients and their families interacting with junior and senior investigators. Participation of patient advocacy groups in the planning and conduct of outreach activities is encouraged.

Shared Resource Core

The shared Scientific Research Resource Core should be designed and managed to support the research of the MDCRC, as well as serving as a resource for the national and perhaps international muscular dystrophy research community. Applicants may wish to consult the Action Plan for the Muscular Dystrophies (http://www.ninds.nih.gov/about_ninds/groups/mdcc/MDCC_Action_Plan.pdf) for consensus statements on infrastructure needs of the muscular dystrophy research community. Applicants are encouraged to propose a core that will provide services, specimens or other resources that are rate limiting to the progress of muscular dystrophy research, so that the core will help to accelerate research conducted by users within and outside the MDCRC.

Training Core

As nationally recognized centers of excellence in muscular dystrophy research, the MDCRCs are expected to play a leadership role in training new researchers for the muscular dystrophy field, contributing to the development of future research leaders. Each center should include a Training Core to provide support for stipends and research costs for predoctoral, postdoctoral and/or clinical fellow trainees as well as support for activities that enhance the institution's environment for training of students, fellows and early-stage investigators in muscular dystrophy research. Leveraging existing training programs is encouraged, and support from the MDCRC should add value to existing programs by enhancing the focus on the muscular dystrophies and increasing the number of trainees. Mentored researchers pursuing their own research aims will become better prepared for careers as future independent investigators and leaders in the muscular dystrophy research community. This core may propose activities that enhance the training environment through specialized coursework, a seminar program, retreats for presentation of trainee research, journal clubs or other activities that contribute to the preparation of junior investigators for careers in muscular dystrophy research. Trainees should learn about the broad range of research conducted by investigators at their own and other centers including preclinical translational research and clinical studies. Exposure to research at other Wellstone Centers is also encouraged through exchange programs, short-term training opportunities or visits to learn new research approaches.

Recipients of MDCRC awards will become part of a national program in muscular dystrophy and will be expected to participate in MDCRC activities, including meetings of the Steering Committee (composed of MDCRC Directors and Co-Directors, and NIH staff) and an annual MDCRC meeting that rotates among the MDCRC sites.

NIH grants policies as described in the NIH Grants Policy Statement will apply to the applications submitted and awards made in response to this FOA.

Higher Education Institutions

• Public/State Controlled Institutions of Higher Education
• Private Institutions of Higher Education

The following types of Higher Education Institutions are always encouraged to apply for NIH support as Public or Private Institutions of Higher Education:

• Hispanic-serving Institutions
• Historically Black Colleges and Universities (HBCUs)
• Tribally Controlled Colleges and Universities (TCCUs)
• Alaska Native and Native Hawaiian Serving Institutions
• Asian American Native American Pacific Islander Serving Institutions (AANAPISIs)

Nonprofits Other Than Institutions of Higher Education

• Nonprofits with 501(c)(3) IRS Status (Other than Institutions of Higher Education)
• Nonprofits without 501(c)(3) IRS Status (Other than Institutions of Higher Education)

For-Profit Organizations

• Small Businesses
• For-Profit Organizations (Other than Small Businesses)

Governments

• State Governments
• County Governments
• City or Township Governments
• Special District Governments
• Indian/Native American Tribal Governments (Federally Recognized)
• Indian/Native American Tribal Governments (Other than Federally Recognized)
• Eligible Agencies of the Federal Government
• U.S. Territory or Possession

Other

• Independent School Districts
• Public Housing Authorities/Indian Housing Authorities
• Native American Tribal Organizations (other than Federally recognized tribal governments)
• Faith-based or Community-based Organizations
• Regional Organizations

Non-domestic (non-U.S.) Entities (Foreign Institutions) are not eligible to apply.
Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply.
Foreign components, as defined in the NIH Grants Policy Statement, are allowed.

Applicant Organizations

Applicant organizations must complete and maintain the following registrations as described in the SF 424 (R&R) Application Guide to be eligible to apply for or receive an award. All registrations must be completed prior to the application being submitted. Registration can take 6 weeks or more, so applicants should begin the registration process as soon as possible. The NIH Policy on Late Submission of Grant Applications states that failure to complete registrations in advance of a due date is not a valid reason for a late submission.

• Dun and Bradstreet Universal Numbering System (DUNS) - All registrations require that applicants be issued a DUNS number. After obtaining a DUNS number, applicants can begin both SAM and eRA Commons registrations. The same DUNS number must be used for all registrations, as well as on the grant application.
• System for Award Management (SAM) (formerly CCR) Applicants must complete and maintain an active registration, which requires renewal at least annually. The renewal process may require as much time as the initial registration. SAM registration includes the assignment of a Commercial and Government Entity (CAGE) Code for domestic organizations which have not already been assigned a CAGE Code.
  • NATO Commercial and Government Entity (NCAGE) Code Foreign organizations must obtain an NCAGE code (in lieu of a CAGE code) in order to register in SAM.
• eRA Commons - Applicants must have an active DUNS number and SAM registration in order to complete the eRA Commons registration. Organizations can register with the eRA Commons as they are working through their SAM or Grants.gov registration. eRA Commons requires organizations to identify at least one Signing Official (SO) and at least one Program Director/Principal Investigator (PD/PI) account in order to submit an application.
• Grants.gov Applicants must have an active DUNS number and SAM registration in order to complete the Grants.gov registration.

Program Directors/Principal Investigators (PD(s)/PI(s))

All PD(s)/PI(s) must have an eRA Commons account. PD(s)/PI(s) should work with their organizational officials to either create a new account or to affiliate their existing account with the applicant organization in eRA Commons. If the PD/PI is also the organizational Signing Official, they must have two distinct eRA Commons accounts, one for each role. Obtaining an eRA Commons account can take up to 2 weeks.

Any individual(s) with the skills, knowledge, and resources necessary to carry out the proposed research as the Program Director(s)/Principal Investigator(s) (PD(s)/PI(s)) is invited to work with his/her organization to develop an application for support. Individuals from underrepresented racial and ethnic groups as well as individuals with disabilities are always encouraged to apply for NIH support.

For institutions/organizations proposing multiple PDs/PIs, visit the Multiple Program Director/Principal Investigator Policy and submission details in the Senior/Key Person Profile (Expanded) Component of the SF424 (R&R) Application Guide.

This FOA does not require cost sharing as defined in the NIH Grants Policy Statement.

Applicant organizations may submit more than one application, provided that each application is scientifically distinct.

The NIH will not accept duplicate or highly overlapping applications under review at the same time. This means that the NIH will not accept:

• A new (A0) application that is submitted before issuance of the summary statement from the review of an overlapping new (A0) or resubmission (A1) application.
• A resubmission (A1) application that is submitted before issuance of the summary statement from the review of the previous new (A0) application.
• An application that has substantial overlap with another application pending appeal of initial peer review (see NOT-OD-11-101).

In addition, the NIH will not accept a resubmission (A1) application that is submitted later than 37 months after submission of the new (A0) application that it follows. The NIH will accept submission:

• To an RFA of an application that was submitted previously as an investigator-initiated application but not paid;
• Of an investigator-initiated application that was originally submitted to an RFA but not paid; or
• Of an application with a changed grant activity code.

Applicants can access the SF424 (R&R) application package associated with this funding opportunity using the Apply for Grant Electronically button in this FOA or following the directions provided at Grants.gov.

Most applicants will use NIH’s ASSIST system to prepare and submit applications through Grants.gov to NIH. Applications prepared and submitted using applicant systems capable of submitting electronic multi-project applications to Grants.gov will also be accepted.

It is critical that applicants follow the instructions in the SF424 (R&R) Application Guide, including Supplemental Grant Application Instructions except where instructed in this funding opportunity announcement to do otherwise and where instructions in the Application Guide are directly related to the Grants.gov downloadable forms currently used with most NIH opportunities. Conformance to the requirements in the Application Guide is required and strictly enforced. Applications that are out of compliance with these instructions may be delayed or not accepted for review.

For information on Application Submission and Receipt, visit Frequently Asked Questions Application Guide, Electronic Submission of Grant Applications.

Although a letter of intent is not required, is not binding, and does not enter into the review of a subsequent application, the information that it contains allows IC staff to estimate the potential review workload and plan the review.

By the date listed in Part 1. Overview Information, prospective applicants are asked to submit a letter of intent that includes the following information:

• Descriptive title of proposed activity
• Name(s), address(es), and telephone number(s) of the PD(s)/PI(s)
• Names of other key personnel
• Participating institution(s)
• Number and title of this funding opportunity

The letter of intent should be sent to:

Amanda Boyce, Ph.D.
Division of Musculoskeletal Diseases
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
One Democracy Plaza
6701 Democracy Boulevard, Suite 800
Bethesda, MD 20892-4872
Telephone: 301-594-5585
Email: boycea@mail.nih.gov

Component Types Available in ASSIST	Research Strategy/Program Plan Page Limits
Overall	12
Admin Core	12
NRSA Training (use for Training Core)	25*
Shared Resource (use for Shared Scientific Resource Core)	12
Project	12

Additional page limits described in the SF424 Application Guide and the Table of Page Limits must be followed.

* The 'NRSA Training' component type will follow the page limits for the Institutional Training activity code as defined in the Table of Page Limits. Note that the limit of 25 pages refers to the combination of Background, Program Plan, and Recruitment and Retention Plan to Enhance Diversity (attachments 2-4 of Research Training Program Plan form).

The following section supplements the instructions found in the SF424 (R&R) Application Guide, and should be used for preparing a multi-component application.

The application should consist of the following components:

• Overall: required
• Administrative Core: required
• Training Core : required
• Shared Scientific Resource Core : one or more required
• Project : two or more required

When preparing your application in ASSIST, use Component Type Overall .

All instructions in the SF424 (R&R) Application Guide must be followed, with the following additional instructions, as noted.

SF424 (R&R) Cover (Overall)

Complete entire form.

PHS 398 Cover Page Supplement (Overall)

Note: Human Embryonic Stem Cell lines from other components should be repeated in cell line table in Overall component.

Research & Related Other Project Information (Overall)

Follow standard instructions.

Facilities and Other Resources: Describe any existing resources of the institution(s) that will be leveraged by the Center.

Project/Performance Site Location(s) (Overall)

Enter primary site only.

A summary of Project/Performance Sites in the Overall section of the assembled application image in eRA Commons compiled from data collected in the other components will be generated upon submission.

Research & Related Senior/Key Person Profile (Overall)

Include only the Project Director/Principal Investigator (PD/PI) and any multi-PDs/PIs (if applicable to this FOA) for the entire application.

Each applicant institution will name an MDCRC Center Director (Program Director/Principal Investigator) who will be the key figure in the administration, management, and coordination of the Center grant.

A summary of Senior/Key Persons followed by their Biographical Sketches in the Overall section of the assembled application image in eRA Commons will be generated upon submission.

Budget (Overall)

The only budget information included in the Overall component is the Estimated Project Funding section of the SF424 (R&R) Cover.

A budget summary in the Overall section of the assembled application image in eRA Commons compiled from detailed budget data collected in the other components will be generated upon submission.

PHS 398 Research Plan (Overall)

Specific Aims: Describe the overall goals of the center for the time frame of the grant. Describe the research objectives of the center for advancing understanding of disease mechanisms, developing therapies, advancing patient care or reducing disease burden. Also describe the center's objectives for developing and sharing research resources, training junior researchers, educating and engaging the patient/advocacy communities, and promoting enrollment in clinical studies.

Research Strategy: The Overall Research Strategy should describe the major theme of the Center, its goals and objectives, background information, the overall importance of the research in developing therapies or reducing the burden of disease, and the expected near- and long-term influence on the overall field of muscular dystrophy research if the goals and objectives are achieved. Describe the rationale for the total proposed program. Explain the strategy for achieving the goals defined for the overall program and how each research project and core relates to that strategy. A successful Center grant application will include a well-integrated research strategy that clearly shows how the proposed projects and cores will foster preclinical and or clinical development of novel therapeutics or other interventions to reduce the burden of the muscular dystrophies. The program should be viewed as interrelated research projects, each of which is not only individually scientifically meritorious but is also complementary to the other projects and related to the overall theme developed for the Center. Provide justification in the application that: (a) the proposed projects are such that they require an intensive collaborative effort to succeed and (b) that key personnel will collaborate effectively.

Describe the organizational structure of the MDCRC including the components of the Center. Also describe any connections between the proposed Center and other organizations such as patient advocacy groups or industry partners. Explain how different components of the organization, including key personnel, will interact, why they are essential to accomplishing the overall goal of the research, and how combined resources create capabilities that are more than the sum of the parts.

For renewal applications, document achievement of the goals of the prior funding period.

Resource Sharing Plan: Individuals are required to comply with the instructions for the Resource Sharing Plans (Data Sharing Plan, Sharing Model Organisms, and Genome Wide Association Studies (GWAS)) as provided in the SF424 (R&R) Application Guide,.

Appendix: Do not use the Appendix to circumvent page limits. Follow all instructions for the Appendix as described in the SF424 (R&R) Application Guide.

When preparing your application in ASSIST, use Component Type Admin Core.

All instructions in the SF424 (R&R) Application Guide must be followed, with the following additional instructions, as noted.

SF424 (R&R) Cover (Administrative Core)

Complete only the following fields:

• Applicant Information
• Type of Applicant (optional)
• Descriptive Title of Applicant’s Project
• Proposed Project Start/Ending Dates

PHS 398 Cover Page Supplement (Administrative Core)

Enter Human Embryonic Stem Cells in each relevant component.

Research & Related Other Project Information (Administrative Core)

Human Subjects: Answer only the Are Human Subjects Involved? and 'Is the Project Exempt from Federal regulations? questions.

Vertebrate Animals: Answer only the Are Vertebrate Animals Used? question.

Project Narrative: Do not complete. Note: ASSIST screens will show an asterisk for this attachment indicating it is required. However, eRA systems only enforce this requirement in the Overall component and applications will not receive an error if omitted in other components.

Project /Performance Site Location(s) (Administrative Core)

List all performance sites that apply to the specific component.

Note: The Project Performance Site form allows up to 300 sites, prior to using additional attachment for additional entries.

Research & Related Senior/Key Person Profile (Administrative Core)

• In the Project Director/Principal Investigator section of the form, use Project Role of Other with Category of Center Director and provide a valid eRA Commons ID in the Credential field. The Director will be responsible for the organization and operation of the center. The Director should be a recognized scientific leader experienced in the field of muscular dystrophy research and must be able to coordinate, integrate, and provide guidance in the establishment of research programs. In the additional Senior/Key Profiles section, list Senior/Key persons that are working in the component. A Co-Director should be named who will be involved in the administrative and scientific efforts of the Center. The Co-Director should be qualified to become the Director, should that need arise.
• Include a single Biographical Sketch for each Senior/Key person listed in the application regardless of the number of components in which they participate. When a Senior/Key person is listed in multiple components, the Biographical Sketch can be included in any one component.
• If more than 100 Senior/Key persons are included in a component, the Additional Senior Key Person attachments should be used.

Budget (Administrative Core)

Budget forms appropriate for the specific component will be included in the application package.

The Administrative Core of the MDCRC application should include up to $5,000 per year direct costs for travel of the Director, Co-Director, and other Center investigators to the annual meeting of the Wellstone Network, and for visits of Center investigators or trainees to other MDCRCs or other collaborative sites to exchange scientific ideas, to plan multi-Center research projects, or to receive training in specialized techniques.

The Center Director and Co-Director should each have a minimum commitment of 20% (2.4 calendar months) effort to the MDCRC, which should include appropriate effort to the Administrative Core as well as other cores and/or projects.

Note: The R&R Budget form included in many of the component types allows for up to 100 Senior/Key Persons in section A and 100 Equipment Items in section C prior to using attachments for additional entries. All other SF424 (R&R) instructions apply.

PHS 398 Research Plan (Administrative Core)

Specific Aims: Describe the goals of the Administrative Core and how these goals will contribute to a substantial and sustained influence of the center on the overall field of muscular dystrophy research. The specific aims should address plans for communication and coordination of efforts within the center, facilitating interactions with researchers outside the center, seeking advice from an external advisory committee, promoting outreach to the patient/advocacy communities, promoting enrollment in clinical studies, and any other goals at the discretion of the applicants.

Research Strategy: The Administrative Core will be responsible for the management and administration of the overall Center. This section of the application should describe the strategies and processes that will be used to manage the Center and achieve the goals. This Core, led by the Center Director, will provide oversight for the projects and cores, promote coordination and collaboration within the Center and with investigators and organizations outside the Center. A narrative description should be provided that includes the planning and coordination of research activities; the integration of cross-disciplinary research; the oversight of fiscal and resource management; and the maintenance of ongoing communication. Indicate who will be responsible for each of these activities. Applicants should specify appropriate administrative/business management staff and oversight mechanisms by the Center Director, Center Co-Director, and a local Executive Committee.

The Administrative Core should propose the development and maintenance of a website for the center. This website should provide information on the research projects conducted by the center, promote patient recruitment for clinical studies, publicize the availability of shared scientific resources or specialized services, publicize training opportunities and other information of value to the research or patient/advocacy communities or the general public. To promote the reproducibility of research results, detailed research protocols and standard operating procedures for research conducted by the center should be made available through the center website.

The Administrative Core will also be responsible for outreach activities to the patient and advocacy communities. Applicants are encouraged to propose activities such as presentations, lab tours or other face-to-face interactions between researchers and members of the patient and advocacy communities. Applicants are encouraged to educate the patient/advocacy communities about muscular dystrophy research, include the perspectives of patients and their families when making decisions regarding research directions and promote patient enrollment in clinical studies conducted by the center and at other institutions.

When multiple performance sites are planned, the Administrative Core should include leadership and communication plans adequate to manage the multiple sites.

The Administrative Core should establish an Executive Committee, composed of members of the Center and a Center Advisory Committee, composed of people outside the Center. Describe how the Center Advisory Committee will contribute to oversight of the research projects, core facilities and training environment of the Center. The Center Advisory Committee should meet approximately once a year and brief reports of the proceedings of the meeting and recommendations of the committee should be included in the progress reports of the Center. Describe plans for working with the Center Advisory Committee, but only name the members of the Advisory Committee if applying for the renewal of a Center.

In order to assure active collaboration with other Centers, the MDCRC Director, Co-Director, and other staff should attend annual meetings of the MDCRC Network, contribute to the coordination of effort, and/or help to refine and standardize operating procedures among the Centers.

Resource Sharing Plan: Individuals are required to comply with the instructions for the Resource Sharing Plans (Data Sharing Plan, Sharing Model Organisms, and Genome Wide Association Studies (GWAS)) as provided in the SF424 (R&R) Application Guide, with the following modification:

The Administrative Core should provide oversight of the resource sharing plans of the Center. Applications are expected to provide a plan regarding the timely sharing of specimens, cells, animal models and redacted data generated with support from this award with other qualified research scientists, both within and outside the MDCRC network, and ensuring that such data are HIPPA compliant. Applicants should consider describing plans for tracking requests for resources and monitoring the timely accomplishment of the activities described in the resource sharing plans..

Appendix: Do not use the Appendix to circumvent page limits. Follow all instructions for the Appendix as described in the SF424 (R&R) Application Guide.

Planned Enrollment Report (Administrative Core)

Not Applicable

PHS 398 Cumulative Inclusion Enrollment Report (Administrative Core)

Not Applicable

When preparing your application in ASSIST, use Component Type NRSA Training.

Follow all instructions provided in the SF424 (R&R) Application Guide for Preparing Institutional Ruth L. Kirschstein National Research Service Award (NRSA) except where indicated below:

SF424 (R&R) Cover (Training Core)

Complete only the following fields:

• Applicant Information
• Type of Applicant (optional)
• Descriptive Title of Applicant’s Project
• Proposed Project Start/Ending Dates

Project/Performance Site Location(s) (Training Core)

Follow "Special Instructions for 4.3 Research & Related Project/Performance Site Locations" provided in the SF424 (R&R) Supplemental Instructions for Preparing Institutional Ruth L. Kirschstein National Research Service Award (NRSA) Application.

Research & Related Other Project Information (Training Core)

Follow "Special Instructions for 4.4 Research & Related Other Project Information Form" provided in the SF424 (R&R) Supplemental Instructions for Preparing Institutional Ruth L. Kirschstein National Research Service Award (NRSA) Application, with the following additional modifications:

Human Subjects: Answer only the Are Human Subjects Involved? and 'Is the Project Exempt from Federal regulations? questions.

Vertebrate Animals: Answer only the Are Vertebrate Animals Used? question.

Project Summary/Abstract: Summarize the objectives of the Training Core. Provide information regarding the types of muscular dystrophies and types of research (i.e. basic, translational, clinical) in which the mentors of the center are prepared to provide training. The trainees of the Training Core include those few trainees that will be supported by stipends from the Core and the larger pool of predoctoral, postdoctoral or clinical fellow researchers that are engaged in muscular dystrophy research in the labs of researchers supported through the projects and scientific core(s) of the MDCRC. Therefore, the Training Core should address the enhancements to the training environment that would affect all of the trainees associated with the MDCRC, and not just those directly supported through the Training Core. Include a brief description of the level(s) (i.e., predoctoral, postdoctoral) and duration of the proposed training, the projected number of participating trainees and their anticipated levels of experience, also specifying the number and levels of experience that would receive stipend support from the Core.

Research & Related Senior/Key Person Profile (Training Core)

Follow "Special Instructions for 4.5 Senior/Key Person Profile (Expanded) Form" provided in the SF424 (R&R) Supplemental Instructions for Preparing Institutional Ruth L. Kirschstein National Research Service Award (NRSA) Application, with the following additional modifications:

The Training Core Leader and any other individuals whose contributions are critical to the development, management and execution of the Training Core in a substantive, measurable way (whether or not salaries are reimbursed) should be identified as senior/key persons. These would include co-Leader(s), if applicable, and other Training Core staff.

• In the Project Director/Principal Investigator section of the form, use Project Role of Other with Category of Project Lead and provide a valid eRA Commons ID in the Credential field.
• In the additional Senior/Key Profiles section, list Senior/Key persons that are working in the component.

Do not include proposed mentors and training faculty members (other than senior/key persons) in this section. Biographical Sketches for mentors and participating faculty will be included in the PHS 398 Research Training Program Plan Form, Participating Faculty Biosketches attachment.

PHS 398 Cover Page Supplement (Training Core)

Follow "Special Instructions for 4.6 PHS 398 Cover Page Supplement" provided in the SF424 (R&R) Supplemental Instructions for Preparing Institutional Ruth L. Kirschstein National Research Service Award (NRSA) Application.

PHS 398 Training Budget Component (Training Core)

For the Training Core, follow "8.5. PHS 398 Training Budget" provided in the SF424 (R&R) Supplemental Instructions for Preparing Institutional Ruth L. Kirschstein National Research Service Award (NRSA) Application, with the following additional modifications:

The direct cost budget for the training core should not exceed $100,000, with most of the funds supporting the stipends of trainees.

Part A: The application should request stipends for trainees. Note: while NRSA stipend levels do not apply to this FOA, applications may use the current NRSA stipend levels as a guide to determining appropriate compensation for the trainees.

Part B: Enter the total costs for Trainee Travel. Include expenses associated with the training core (such as salary support for Training Core Leader and other staff, research supplies, etc.) in the "Training Related Expenses." Provide details on level of effort and salary of core leader/staff, description other expenses, etc. in the justification section.

PHS 398 Research Training Program Plan (Training Core)

Follow "8.7. Research Training Program Plan Form" provided in the SF424 (R&R) Supplemental Instructions for Preparing Institutional Ruth L. Kirschstein National Research Service Award (NRSA) Application, with the following modifications:

(Note: NRSA Tables are not applicable to this FOA.)

Background: Provide the rationale for the Training Core in muscular dystrophy research. Indicate how the Training Core will relate to current training activities at the applicant institution.

Program Plan: The Program Plan should be saved as a single pdf file and should consist of the following elements:

Program Administration: Describe the acknowledged strengths, leadership and administrative skills, training experience, scientific expertise, and active research of the Training Core Leader. Relate these strengths to the proposed management of the training core. Describe the planned strategy and administrative structure to be used to oversee and monitor the core.

Program Faculty: The application must include information about the Center faculty who will be available to serve as preceptors/mentors and provide guidance and expertise appropriate to the level of trainees proposed in the application. Describe the complementary expertise and experiences of the proposed Center Faculty, including active research and other scholarly activities in which the faculty are engaged, as well as experience mentoring and training individuals at the proposed career stage(s). For any proposed Center Faculty lacking research training experience, describe a plan to ensure successful trainee guidance by these individuals.

Proposed Training: Provide an overview of the proposed program, which would enhance training in muscular dystrophy research for all those trainees associated with the projects and cores of the MDCRC. Training activities that go beyond the MDCRC to enhance the experiences of trainees in other labs or at other institutions are also welcome. Outline the objectives of the program and the program activities that will be used to meet these objectives. Describe for whom the training program is intended, including the training level(s) of the trainees, the academic and research background needed to pursue the proposed training, and, as appropriate, plans to accommodate differences in preparation among trainees. Include information about planned courses, mentored research experiences, and any activities designed to develop specific technical skills or other skills essential for the proposed research training. Describe how trainees will be educated in the conduct of basic, preclinical translational and/or clinical research for the muscular dystrophies.

Program Evaluation: Describe a plan to review and determine the quality and effectiveness of the training program. Plans should be described for discussing with the Center Advisory Committee the data from the evaluation of the training program and suggestions for program improvements.

Trainee Candidates: Describe the nomination and selection process to be used to select candidates who would be supported by stipends from the Training Core. Applicants are encouraged to include the Center Advisory Committee in the process for selecting candidates. Applicants are encouraged to place a high priority on the recruitment of trainees with clinical expertise due to the significant need for well-trained clinical researchers in the muscular dystrophies. Do not name prospective Trainees in the application.

Institutional Environment and Commitment to the Program: The sponsoring institution must assure support for the training environment of the MDCRC as proposed in the Training Core including assurance that sufficient time will be allowed for the Training Core Leader and other Center Faculty to contribute to the proposed training. Institutions with ongoing research training, student development, or career development programs in which the MDCRC faculty are eligible to participate as mentors should explain what distinguishes the proposed Training Core from existing training programs at the same trainee level, how the programs will synergize, if applicable, whether trainees are expected to transition from one support program to another, and how the training faculty, pool of potential trainees, and resources are sufficiently robust to support the proposed training in addition to existing ones.

Recruitment and Retention Plan to Enhance Diversity: Individuals are required to comply with the instructions for Recruitment and Retention Plan to Enhance Diversity as provided in Chapter 8 of the SF424 (R&R) Application Guide. Applications must include a description of plans to recruit a diverse trainee pool and may wish to include data in support of past accomplishments. Information should be included on both successful and unsuccessful recruitment strategies.

Plan for Instruction in the Responsible Conduct of Research: Individuals are required to comply with the instructions for Plan for Instruction in the Responsible Conduct of Research as provided in Chapter 8 of the SF424 (R&R) Application Guide.

Progress Report (Renewal Applications Only): Renewal applications should report on the progress of their Training Core in supporting successful trainees, enhancing the training environment of the center, and engaging the patient/advocacy communities in education/outreach activities. Identify trainees that were supported by the core, describe their scientific productivity and how the support has contributed to the advance of their careers. Describe activities or events that have enhanced the training environment for muscular dystrophy research for the center and how this has impacted the scientific productivity and career advance of the trainee in the overall center. Describe activities or events that have increased awareness of muscular dystrophy research in the patient and advocacy communities. Note that applications for this FOA will now propose patient/advocacy outreach activities in the Administrative Core, instead of in the Training Core.

Participating Faculty Biosketches: Faculty Biosketches for participating faculty (excluding Senior/Key Personnel) should follow the Additional NIH and other PHS Agencies Instructions for a Biographical Sketch, except that a personal statement is not required for participating faculty. These should be attached as a single pdf document.

Data Tables: Not Applicable

Appendix: Do not use the Appendix to circumvent page limits. Follow all instructions for the Appendix as described in the SF424 (R&R) Application Guide.

When preparing your application in ASSIST, use Component Type Shared Resource.

All instructions in the SF424 (R&R) Application Guide must be followed, with the following additional instructions, as noted.

SF424 (R&R) Cover (Shared Scientific Resource Core )

Complete only the following fields:

• Applicant Information
• Type of Applicant (optional)
• Descriptive Title of Applicant’s Project
• Proposed Project Start/Ending Dates

PHS 398 Cover Page Supplement (Shared Scientific Resource Core)

Enter Human Embryonic Stem Cells in each relevant component.

Research & Related Other Project Information (Shared Scientific Resource Core)

Human Subjects: Answer only the Are Human Subjects Involved? and 'Is the Project Exempt from Federal regulations? questions.

Vertebrate Animals: Answer only the Are Vertebrate Animals Used? question.

Project Narrative: Do not complete. Note: ASSIST screens will show an asterisk for this attachment indicating it is required. However, eRA systems only enforce this requirement in the Overall component and applications will not receive an error if omitted in other components.

Project /Performance Site Location(s) (Shared Scientific Resource Core)

List all performance sites that apply to the specific component.

Note: The Project Performance Site form allows up to 300 sites, prior to using additional attachment for additional entries.

Research & Related Senior/Key Person Profile (Shared Scientific Resource Core)

• In the Project Director/Principal Investigator section of the form, use Project Role of Other with Category of Core Lead and provide a valid eRA Commons ID in the Credential field.
• In the additional Senior/Key Profiles section, list Senior/Key persons that are working in the component.
• Include a single Biographical Sketch for each Senior/Key person listed in the application regardless of the number of components in which they participate. When a Senior/Key person is listed in multiple components, the Biographical Sketch can be included in any one component.
• If more than 100 Senior/Key persons are included in a component, the Additional Senior Key Person attachments should be used.

Budget (Shared Scientific Resource Core)

Budget forms appropriate for the specific component will be included in the application package.

Note: The R&R Budget form included in many of the component types allows for up to 100 Senior/Key Persons in section A and 100 Equipment Items in section C prior to using attachments for additional entries. All other SF424 (R&R) instructions apply.

PHS 398 Research Plan (Shared Scientific Resource Core)

Specific Aims: Describe the goals of the Shared Scientific Resource Core. The aims should address not only the delivery of services or materials, but also the processes for ensuring the consistent quality of the services or materials, fair access by users and efficient use of the resources in important projects in muscular dystrophy research.

Research Strategy: Describe the function of the core as a resource to the program. This section must clearly present the facilities, techniques, and professional skills that the core will provide. A core is principally designed as a service or resource component; it would be highly unusual to include research in a core (a possible exception would be methodology development). Please contact the Institute staff if you require guidance on this issue.

Describe the role of the core as a resource to the program as a whole. Discuss ways in which these centralized services will produce an economy of effort and/or savings in overall costs compared to their inclusion as part of each project in the program. To aid in the review of the application it is recommended that applicants prepare in tabular form information concerning the research projects that each facility core unit would serve and the proportion of the cost of the facility core unit associated with each research project involved.

Cores are seldom created specifically for the MDCRC and more often already exist in some form prior to the application. When proposing support for an already existing core, describe how the MDCRC award would enhance the resources or services already available through new innovation and technology development, expanded availability, increased throughput, etc.

Each Center should contain a scientific core that provides services and/or resources that are shared with other investigators nationally and perhaps internationally. These resources could be reagents, specimens, services or technical expertise that will help to accelerate progress of multiple projects toward the development of therapies or other strategies for improving the lives of patients with muscular dystrophy. Describe how this shared core will meet the needs of the national/international muscular dystrophy research community. If cores providing similar resources are already available, explain the need for this additional core.

Additional cores may be proposed if they are needed to advance the local research effort and if they fit within the budget limits described elsewhere in this FOA. For each scientific core, the applicant should identify projects that will depend on the services and/or resources proposed. Projects outside the Center that would use the core should be described in general terms and investigators outside the Center should not be contacted, as this would lead to conflicts of interest during peer review.

Resource Sharing Plan: Individuals are required to comply with the instructions for the Resource Sharing Plans (Data Sharing Plan, Sharing Model Organisms, and Genome Wide

Appendix: Do not use the Appendix to circumvent page limits. Follow all instructions for the Appendix as described in the SF424 (R&R) Application Guide.

Planned Enrollment Report (Shared Scientific Resource Core)

If the Core proposes to enroll participants in clinical research, follow all instructions for completing Planned Enrollment Reports as described in the SF424 (R&R) Application Guide.

PHS 398 Cumulative Inclusion Enrollment Report (Shared Scientific Resource Core)

If the Core proposes to enroll participants in clinical research, follow all instructions for completing Cumulative Inclusion Enrollment Report as described in the SF424 (R&R) Application Guide.

When preparing your application in ASSIST, use Component Type Project.

All instructions in the SF424 (R&R) Application Guide must be followed, with the following additional instructions, as noted.

SF424 (R&R) Cover (Project)

Complete only the following fields:

• Applicant Information
• Type of Applicant (optional)
• Descriptive Title of Applicant’s Project
• Proposed Project Start/Ending Dates

PHS 398 Cover Page Supplement (Project)

Enter Human Embryonic Stem Cells in each relevant component.

Research & Related Other Project Information (Project)

Human Subjects: Answer only the Are Human Subjects Involved? and 'Is the Project Exempt from Federal regulations? questions.

Vertebrate Animals: Answer only the Are Vertebrate Animals Used? question.

Project Narrative: Do not complete. Note: ASSIST screens will show an asterisk for this attachment indicating it is required. However, eRA systems only enforce this requirement in the Overall component and applications will not receive an error if omitted in other components.

Project /Performance Site Location(s) (Project)

List all performance sites that apply to the specific component.

Note: The Project Performance Site form allows up to 300 sites, prior to using additional attachment for additional entries.

Research & Related Senior/Key Person Profile (Project)

• In the Project Director/Principal Investigator section of the form, use Project Role of Other with Category of Project Lead and provide a valid eRA Commons ID in the Credential field.
• In the additional Senior/Key Profiles section, list Senior/Key persons that are working in the component.
• Include a single Biographical Sketch for each Senior/Key person listed in the application regardless of the number of components in which they participate. When a Senior/Key person is listed in multiple components, the Biographical Sketch can be included in any one component.
• If more than 100 Senior/Key persons are included in a component, the Additional Senior Key Person attachments should be used.

Budget (Project)

Budget forms appropriate for the specific component will be included in the application package.

Note: The R&R Budget form included in many of the component types allows for up to 100 Senior/Key Persons in section A and 100 Equipment Items in section C prior to using attachments for additional entries. All other SF424 (R&R) instructions apply.

PHS 398 Research Plan (Project)

Specific Aims: Describes the specific goals and objectives of the project.

Research Strategy: Clearly state the project's overall objective and explain its relevance to the central theme of the Center. In addition, an explanation should be included describing how the project relates to and both complements and enhances the other research projects and cores of the program. Why the project is best suited to be carried out in the Center environment should be highlighted. Specify the overall biomedical significance of the work proposed. Specify the niches filled by each project in advancing treatments for muscular dystrophy.

Each MDCRC should contain at least one clinical study, but this study is not required to be an interventional clinical trial. Options for clinical studies include but are not restricted to evaluation of natural history, development of biomarkers, or development/validation of endpoint measures. Knowledge from such clinical studies is essential to direct subsequent clinical trials and can be invaluable for the muscular dystrophy field.

If the project includes an interventional clinical trial, the description of the significance should support the potential value and feasibility of successfully completing the study within the term of the grant, including preclinical rationale. The preclinical rationale should provide evidence that the rigor of preclinical efficacy studies and the level of effect of the agent are both sufficient to warrant clinical testing of the agent (for guidance, see http://grants.nih.gov/grants/guide/notice-files/NOT-NS-11-023.html). The approach section should describe evidence that regulatory requirements will be met in a timely manner, evidence of drug/biologic availability for use in a trial, and agreement of all participating clinical/corporate partners. Clinical trials involving the testing of new investigational therapeutics, new indications for FDA-approved drugs, or other medical interventions under a research protocol should be performed under an IND, unless otherwise agreed upon by the FDA. If not exempt, the applicant must provide the NIH with the name and organization of the IND/IDE holder, the date the IND/IDE was filed with the FDA, the FDA IND/IDE number, and any comments from the FDA regarding this protocol. Studies will not be funded unless necessary regulatory approval has first been obtained; regulatory approval at the time of application is preferred. It is strongly encouraged that clinical studies utilize established Common Data Elements (CDEs; see http://www.commondataelements.ninds.nih.gov to ensure comparability with other clinical trials in muscular dystrophy.

Because of the budget limitation of the overall Center and requirements for other components, any interventional clinical trials proposed as part of the MDCRCs are likely to be phase 0/exploratory IND, phase 1, or early stage proof of concept trials. Any clinical trial proposed within MDCRCs should be designed to validate the therapeutic target or candidate therapeutic (phase 0 trial) or to provide specific data that will be necessary to design a subsequent definitive efficacy trial (phase 1 or early stage proof of concept trial). The proposed study must address questions that, when answered, will optimize the design of the eventual definitive clinical trial rather than simply address the clinical question with lower power. Underpowered efficacy trials that are unlikely to advance the development of a candidate therapeutic should be avoided. Examples of relevant clinical research include, but are not limited to, the following:

• Studies to assess whether the candidate therapeutic engages and modulates the presumptive molecular target (i.e., exploratory IND or phase 0 trials);
• Studies to optimize the intervention strategy: for example, studies designed to investigate dose-concentration, dose-response, or concentration-response relationships may contribute to optimal dosage selection for definitive trials;
• Studies to assess the appropriate delivery system or parameter settings of an electronic device or surgical technique;
• Studies to assess the safety and tolerability at various doses or concentrations of a specific intervention;
• Studies designed to evaluate whether an intervention produces sufficient evidence of short-term activity (e.g., biomarker activity) in humans as to justify an efficacy trial;
• Studies designed to select the best of two or more potential interventions or dosing regimens to evaluate in a subsequent definitive trial, based on tolerability or evidence of biological activity;
• Studies to identify inclusion and exclusion criteria to be applied in the phase III clinical trial.
• As noted previously, when clinical trials are proposed, there should be strong evidence provided in support of the potential value and feasibility of the study, including clear preclinical rationale, unimpaired regulatory status, evidence of drug/biologic availability for use in a trial, and agreement of all participating clinical/corporate partners.

Protection of Human Subjects: Subjects who participate in MDCRC clinical research projects should be fully informed, using appropriate consent procedures. The consent form for funded projects should specifically address the following: (1) disclosure that biological materials and clinical data will be distributed to other researchers; (2) assurance that such data will be de-identified and stored and maintained without personal identifiers; (3) disclosure that analyses of these data will be conducted by other scientists currently not included within the current research team, potentially including those with commercial interests; (4) that the data collected by the researchers may be used to study their specific disorder as well as other disorders.

Letters of Support: In studies where a pharmaceutical/biotechnology company is providing the study agent, a written agreement by a company official affirming this arrangement must be provided with the application as a letter of support.

Resource Sharing Plan: Individuals are required to comply with the instructions for the Resource Sharing Plans (Data Sharing Plan, Sharing Model Organisms, and Genome Wide Association Studies (GWAS)) as provided in the SF424 (R&R) Application Guide.

Appendix: Do not use the Appendix to circumvent page limits. Follow all instructions for the Appendix as described in the SF424 (R&R) Application Guide.

Planned Enrollment Report (Project)

When conducting clinical research, follow all instructions for completing Planned Enrollment Reports as described in the SF424 (R&R) Application Guide.

PHS 398 Cumulative Inclusion Enrollment Report (Project)

When conducting clinical research, follow all instructions for completing Cumulative Inclusion Enrollment Report as described in the SF424 (R&R) Application Guide.

Part I. Overview Information contains information about Key Dates. Applicants are encouraged to submit applications before the due date to ensure they have time to make any application corrections that might be necessary for successful submission.

Organizations must submit applications to Grants.gov (the online portal to find and apply for grants across all Federal agencies) using ASSIST or other electronic submission systems. Applicants must then complete the submission process by tracking the status of the application in the eRA Commons, NIH’s electronic system for grants administration.

Applicants are responsible for viewing their application before the due date in the eRA Commons to ensure accurate and successful submission.

Information on the submission process and a definition of on-time submission are provided in the SF424 (R&R) Application Guide.

This initiative is not subject to intergovernmental review.

All NIH awards are subject to the terms and conditions, cost principles, and other considerations described in the NIH Grants Policy Statement.

Pre-award costs are allowable only as described in the NIH Grants Policy Statement.

Applications must be submitted electronically following the instructions described in the SF424 (R&R) Application Guide. Paper applications will not be accepted.

For information on how your application will be automatically assembled for review and funding consideration after submission go to: http://grants.nih.gov/grants/ElectronicReceipt/files/Electronic_Multi-project_Application_Image_Assembly.pdf.

Applicants must complete all required registrations before the application due date. Section III. Eligibility Information contains information about registration.

For assistance with your electronic application or for more information on the electronic submission process, visit Applying Electronically.

Important reminders:
All PD(s)/PI(s) and component Project Leads must include their eRA Commons ID in the Credential field of the Senior/Key Person Profile Component of the SF424(R&R) Application Package. Failure to register in the Commons and to include a valid PD/PI Commons ID in the credential field will prevent the successful submission of an electronic application to NIH.

The applicant organization must ensure that the DUNS number it provides on the application is the same number used in the organization’s profile in the eRA Commons and for the System for Award Management (SAM). Additional information may be found in the SF424 (R&R) Application Guide.

See more tips for avoiding common errors.

Upon receipt, applications will be evaluated for completeness by the Center for Scientific Review and responsiveness by components of participating organizations, NIH. Applications that are incomplete and/or nonresponsive will not be reviewed.

Applicants are required to follow the instructions for post-submission materials, as described in NOT-OD-13-030.

Only the review criteria described below will be considered in the review process. As part of the NIH mission, all applications submitted to the NIH in support of biomedical and behavioral research are evaluated for scientific and technical merit through the NIH peer review system.

For this particular announcement, note the following:

Centers should be designed to include the following components: two or more scientific project(s), an Administrative Core, a Shared Scientific Research Resource Core with national impact, and a Training Core. Applications may include additional core facilities within the overall budget cap. After the review of the individual components, an overall impact score will be assigned to the center application. The overall score will reflect a) the scientific merits of the research project(s), b) the overall effectiveness and adequacy of core resources and facilities, c) the qualifications of the Center Director and Co-Director, d) the quality of the plans for management and oversight of the Center, e) the institutional commitment, and f) the synergy among the components and overall impact of the Center. The overall score for the center application may be higher or lower than the average of the individual components based on the assessment of whether the whole is greater than the sum of its parts.

Reviewers will provide an overall impact score to reflect their assessment of the likelihood for the Center to exert a sustained, powerful influence on the research field(s) involved, in consideration of the following review criteria and additional review criteria (as applicable for the Center proposed).

Reviewers will consider each of the review criteria below in the determination of scientific merit, and give a separate score for each. An application does not need to be strong in all categories to be judged likely to have major scientific impact. For example, a Center that by its nature is not innovative may be essential to advance a field.

Significance

Does the Center address an important problem or a critical barrier to progress in the field? If the aims of the Center are achieved, how will scientific knowledge, technical capability, and/or clinical practice be improved? How will successful completion of the aims change the concepts, methods, technologies, treatments, services, or preventative interventions that drive this field?

Investigator(s)

Are the PD(s)/PI(s), collaborators, and other researchers well suited to the Center? If Early Stage Investigators or New Investigators, or in the early stages of independent careers, do they have appropriate experience and training? If established, have they demonstrated an ongoing record of accomplishments that have advanced their field(s)? If the project is collaborative or multi-PD/PI, do the investigators have complementary and integrated expertise; are their leadership approach, governance and organizational structure appropriate for the project?

Innovation

Does the application challenge and seek to shift current research or clinical practice paradigms by utilizing novel theoretical concepts, approaches or methodologies, instrumentation, or interventions? Are the concepts, approaches or methodologies, instrumentation, or interventions novel to one field of research or novel in a broad sense? Is a refinement, improvement, or new application of theoretical concepts, approaches or methodologies, instrumentation, or interventions proposed?

Approach

Are the overall strategy, methodology, and analyses well-reasoned and appropriate to accomplish the specific aims of the Center? Are potential problems, alternative strategies, and benchmarks for success presented? If the project is in the early stages of development, will the strategy establish feasibility and will particularly risky aspects be managed?

If the Center involves human subjects and/or NIH-defined clinical research, are the plans to address 1) the protection of human subjects from research risks, and 2) inclusion (or exclusion) of individuals on the basis of sex/gender, race, and ethnicity, as well as the inclusion or exclusion of children, justified in terms of the scientific goals and research strategy proposed?

Environment

Will the scientific environment in which the work will be done contribute to the probability of success? Are the institutional support, equipment and other physical resources available to the investigators adequate for the project proposed? Will the project benefit from unique features of the scientific environment, subject populations, or collaborative arrangements?

Is there tangible institutional commitment to the establishment and growth of the MDCRC? Will the institution provide incentives and rewards to promote the mission of team-based research? Is there substantial institutional commitment to tenured faculty positions, dedicated space and other resources, and sufficient time release to allow the investigators to pursue the goals of the MDCRC? Is the physical distribution of Center investigators and core resources conducive to the synergy necessary for a successful MDCRC? Will existing NIH-supported core facilities be shared with the MDCRC? Do the institutional administration and environment provide opportunities for Center growth? If applicable, are there sufficient commitment and support on the part of institutions associated with the MDCRC through consortium agreements?

As applicable for the Center proposed, reviewers will evaluate the following additional items while determining scientific and technical merit, and in providing an overall impact score, but will not give separate scores for these items.

Center Justification

Is there strong justification that this will be a successful Center, with each of the projects and cores not only individually scientifically meritorious but also complementary to the other components, and related to the overall theme developed for the Center? Is there justification in the application that: (a) the proposed projects are such that they require an intensive collaborative effort to succeed and (b) that key personnel will collaborate effectively? Are there appropriate plans for the Center to collaborate and otherwise contribute to the national MDCRC program, through participating in the annual meeting, workshops, training, collaborative efforts, or other MDCRC-wide activities?

Therapy Development

Will the Center be effective in significantly accelerating progress toward effective treatments or other improvements in the lives of muscular dystrophy patients through coordinated and synergistic research and infrastructure activities? Are the aggregate quality and level of innovation of the Center’s research base sufficient and are the proposed research projects highly relevant to the overall goal of advancing therapeutic development in muscular dystrophy?

Reviewers will provide an overall impact score to reflect their assessment of the likelihood for the Project to exert a sustained, powerful influence on the research field(s) involved, in consideration of the following review criteria and additional review criteria (as applicable for the Project proposed). An application does not need to be strong in all categories to be judged likely to have major scientific impact. For example, a Center that by its nature is not especially innovative may be essential to advance a field.

Reviewers will consider each of the review criteria below in the determination of scientific merit, and give a separate score for each.

Significance

Does the Project address an important problem or a critical barrier to progress in the field? If the aims of the Project are achieved, how will scientific knowledge, technical capability, and/or clinical practice be improved? How will successful completion of the aims change the concepts, methods, technologies, treatments, services, or preventative interventions that drive this field?

Is the proposed project sufficiently novel and meritorious and the research plan feasible, in addressing one or more stages in the development of therapies or other strategies to improve the lives of muscular dystrophy patients? For disease mechanism/therapeutic target identification and validation projects, is a plan provided as to how these efforts help to support the therapeutic development pipeline?

For clinical studies or trials: Is there compelling evidence in support of the therapeutic target, candidate therapeutic(s) or other intervention? Is there a clear need for the data or resource for future clinical trials or is there clear justification for moving the selected candidate therapeutic(s) or other intervention forward with proof of concept or safety trials? If the aims of the application are achieved, how will scientific knowledge or clinical practice be advanced? What will be the effect of these studies on the concepts, methods, technologies, treatments, services, or preventative interventions that drive this field? Has the applicant addressed both the significance of the eventual definitive clinical trial AND the significance of this study in providing knowledge needed to proceed to the definitive clinical trial? Is there a sufficient body of high quality preclinical or clinical research that supports the rationale for the proposed study? What is the state of equipoise in the medical and patient communities with respect to the proposed intervention? What is the potential for the proposed intervention to have a powerful influence on patient care and quality of life? If the aims of the study are achieved, how will these results contribute to the design and implementation of the definitive clinical trial?

Investigator(s)

Are the PD(s)/PI(s), collaborators, and other researchers well suited to the Project? If Early Stage Investigators or New Investigators, or in the early stages of independent careers, do they have appropriate experience and training? If established, have they demonstrated an ongoing record of accomplishments that have advanced their field(s)? If the project is collaborative or multi-PD/PI, do the investigators have complementary and integrated expertise; are their leadership approach, governance and organizational structure appropriate for the project?

Do the collaborative efforts require substantial, and not token, contributions from the partners for successful completion? For studies aimed at developing therapeutics, are there plans for involving industry partners that would enhance the research, accelerate progress or increase the likelihood of developing a product that improves the lives of dystrophy patients?

Innovation

Does the application challenge and seek to shift current research or clinical practice paradigms by utilizing novel theoretical concepts, approaches or methodologies, instrumentation, or interventions? Are the concepts, approaches or methodologies, instrumentation, or interventions novel to one field of research or novel in a broad sense? Is a refinement, improvement, or new application of theoretical concepts, approaches or methodologies, instrumentation, or interventions proposed?

Approach

Are the overall strategy, methodology, and analyses well-reasoned and appropriate to accomplish the specific aims of the Project? Are potential problems, alternative strategies, and benchmarks for success presented? If the project is in the early stages of development, will the strategy establish feasibility and will particularly risky aspects be managed?

If the Project involves clinical research, are the plans for 1) protection of human subjects from research risks, and 2) inclusion of minorities and members of both sexes/genders, as well as the inclusion of children, justified in terms of the scientific goals and research strategy proposed?

For preclinical translational research projects, is there a clear step-by-step plan, including adequate milestones, to track and evaluate the therapeutic development effort?

Environment

Will the scientific environment in which the work will be done contribute to the probability of success? Are the institutional support, equipment and other physical resources available to the investigators adequate for the project proposed? Will the project benefit from unique features of the scientific environment, subject populations, or collaborative arrangements?

Do the problems to be addressed require both a center atmosphere and an intensive collaborative effort for successful completion?

The Administrative Core provides for leadership and management of all Center activities. Reviewers will consider each of the review criteria below in determining the scientific merit of this core. Each assigned reviewer will provide one score for the Administrative Core. The scientific merit of the Administrative Core should be considered by reviewers in determining the overall score for the Center.

Purpose

Is the proposed Administrative Core well matched to the needs of the overall center and the goal of educating and engaging the patient/advocacy community?

Management

Is the management proposed appropriate for scientific administration as well as fiscal administration, procurement, property and personnel management, planning, budgeting, etc.? Is the Center scientific and administrative structure sufficient, including its internal and external procedures for monitoring and evaluating the proposed research projects and core facilities/resources? Are there appropriate plans for establishing the Center Advisory Committee, and will this Committee contribute to the oversight of Center research projects, the Shared Resource Core, the Training Core and other components?

Leadership

Do the Director and Co-Director have the leadership and research qualifications to lead a Wellstone Center? Do they have the collective expertise to identify and focus research projects on clinically relevant issues? Are there plans for establishing an Executive Committee and interacting with this committee in a way that will promote the success of the Center?

Communication

Is there an appropriate plan for establishing and maintaining effective communications and cooperation among Center investigators and with investigators outside the Center? Are there appropriate plans for establishing and maintaining a website for the Center that provides useful information to the research community about shared resources, research protocols and training opportunities? Does the application include well-developed plans to inform the patient/advocacy communities about the research and other activities of the Center through web-based communications and direct interactions with patients (and their families when appropriate)? Does the application include strategies to encourage patient awareness and participation in clinical studies at the Center or at other institutions?

Environment

Is the environment for the Administrative Core adequate and appropriate to support the overall Center as proposed? Is there evidence of institutional support for the management of the Center?

Reviewers will provide an overall impact score to reflect their assessment of the likelihood that the proposed training program will prepare individuals for successful, productive scientific research careers and thereby exert a sustained influence on the muscular dystrophy field, in consideration of the following review criteria and additional review criteria (as applicable for the core proposed).

Reviewers will consider each of the review criteria below in the determination of the merit of the training program, and give a separate score for each. When applicable, the reviewers will consider relevant questions in the context of proposed short-term training. An application does not need to be strong in all categories to be judged likely to have major scientific impact.

Training Program and Environment

• Are the research facilities and research environment conducive to preparing trainees for successful careers as muscular dystrophy research scientists?
• Are the objectives, design and direction of the proposed research training program likely to ensure effective training?
• Do the courses, where relevant, and research experiences provide opportunities for trainees to acquire state-of-the-art scientific knowledge, methods, and tools that are relevant to the goals of the training program?
• Does the program provide appropriate inter- or multidisciplinary research training opportunities in the muscular dystrophies?
• Is the proposed training program likely to ensure trainees will be well prepared for research-intensive and research-related careers?
• Is the level of institutional commitment to the training program, including administrative and research training support, sufficient to ensure the success of the program?
• Is it clear how the proposed training program is distinguished from other externally funded training programs at the institution?

Training Program Director(s)/Principal Investigator(s) (PD(s)/PI(s))

• Does the PD/PI have the scientific background, expertise, and administrative and training experience to provide strong leadership, direction, management, and administration of the proposed research training program?
• Does the PD/PI plan to commit sufficient effort to ensure the program’s success?
• For applications designating multiple PDs/PIs:
  • Is a strong justification provided that the multiple PD/PI leadership approach will benefit the training program and the trainees?
  • Is a strong and compelling leadership approach evident, including the designated roles and responsibilities, governance, and organizational structure consistent with and justified by the aims of the training program and the complementary expertise of the PDs/PIs?

Preceptors/Mentors

• Are sufficient numbers of experienced preceptors/mentors with appropriate expertise and funding available to support the number and level of trainees (including short-term trainees, if applicable) proposed in the application?
• Do the preceptors/mentors have strong records as researchers, including recent publications and successful competition for research support in areas directly related to the proposed research training program?
• Do the preceptors/mentors have strong records of training individuals at the level of trainees (including short-term trainees, if applicable) proposed in the program? Are appropriate plans in place to ensure that preceptors lacking sufficient research training experience are likely to provide strong and successful mentoring?

Trainees

• Are there well-defined and justified selection criteria for the trainees that would be supported by stipends from the Training Core?
• Does the application include a well-designed process for selecting the highest quality trainees for the award of stipends from the Training Core, which includes review by the Center Advisory Committee?

Training Record

• How successful are past students/postdoctorates that have been trained in muscular dystrophy research by the preceptors/mentors of this center, considering their research productivity and career advance?
• Has the training environment in muscular dystrophy research at the applicant institution(s) contributed to the success and productivity of past trainees in terms of research accomplishments, career positions, leadership roles, etc?
• Does the program propose a rigorous evaluation plan to assess the quality and effectiveness of the training?
• For applications that request short-term research training positions, is there a record of retaining health professional trainees in research training or other research activities for at least two years?

As applicable for the project proposed, reviewers will consider each of the following items, but will not give scores for these items, and should not consider them in providing an overall impact score.

Recruitment & Retention Plan to Enhance Diversity

Peer reviewers will separately evaluate the recruitment and retention plan to enhance diversity after the overall score has been determined. Reviewers will examine the strategies to be used in the recruitment and retention of individuals from underrepresented groups. The plan will be rated as ACCEPTABLE or UNACCEPTABLE, and the consensus of the review committee will be included in an administrative note in the summary statement.

Training in the Responsible Conduct of Research

All applications for support under this FOA must include a plan to fulfill NIH requirements for instruction in the Responsible Conduct of Research (RCR). Taking into account the specific characteristics of the training program, the level of trainee experience, and the particular circumstances of the trainees, the reviewers will evaluate the adequacy of the proposed RCR training in relation to the following five required components: 1) Format - Does the plan satisfactorily address the format of instruction, e.g. lectures, coursework and/or real-time discussion groups, including face-to-face interaction? (A plan involving only on-line instruction is not acceptable.); 2) Subject Matter Does the plan include a sufficiently broad selection of subject matter, such as conflict of interest, authorship, data management, human subjects and animal use, laboratory safety, research misconduct, research ethics? 3) Faculty Participation - Does the plan adequately describe how faculty will participate in the instruction? For renewal applications, are all training faculty who served as course directors, speakers, lecturers, and/or discussion leaders during the past project period named in the application? 4) Duration of Instruction - Does the plan meet the minimum requirements for RCR, i.e., at least eight contact hours of instruction? 5) Frequency of Instruction Does the plan meet the minimum requirements for RCR, i.e., at least once during each career stage (undergraduate, post-baccalaureate, predoctoral, postdoctoral, and faculty levels) and at a frequency of no less than once every four years?

Reviewers will consider each of the review criteria below in determining the scientific merit of this core. Each assigned reviewer will provide one score for the Scientific Research Resource Core. The scientific merit of the Scientific Research Resource Core should be considered by reviewers in determining the Overall Impact score for the Center.

Purpose

Are the core activities capable of effectively and efficiently supporting research productivity and collaborations and are they essential to the mission of the Center? Is there adequate scientific and technical merit to justify the core? If other, similar cores are already available to the research community, is there still a significant need for this core?

Quality and Quality Control

Is there a strong commitment to provide services to the national muscular dystrophy community and are plans for oversight and prioritization of user requests for core services adequate and fair?

Utilization Potential

Is there strong evidence via the core description that at least one Scientific Research Resource Core will serve as an important national/international resource for the muscular dystrophy research community? Is there sufficient likelihood that this core facility will be used by researchers within and outside the Center, and that the resources provided will significantly accelerate progress on the projects that make use of this core?

Leadership

Does the core provide adequate leadership and technical expertise to ensure that it meets its stated goals?

Cost Effectiveness

If the core facility is already established and supported by funding other than an MDCRC, how will the MDCRC award enhance the resources available? If designed as a fee-for-service facility, are the projected fees appropriate for recovery of only the variable costs (supplies, service contracts, etc.) and not the costs of personnel and equipment?

Environment

Are the staffing, allocated space, equipment, and other resources that are available to the core sufficient to meet the anticipated demand on its services?

Protections for Human Subjects

For research that involves human subjects but does not involve one of the six categories of research that are exempt under 45 CFR Part 46, the committee will evaluate the justification for involvement of human subjects and the proposed protections from research risk relating to their participation according to the following five review criteria: 1) risk to subjects, 2) adequacy of protection against risks, 3) potential benefits to the subjects and others, 4) importance of the knowledge to be gained, and 5) data and safety monitoring for clinical trials.

For research that involves human subjects and meets the criteria for one or more of the six categories of research that are exempt under 45 CFR Part 46, the committee will evaluate: 1) the justification for the exemption, 2) human subjects involvement and characteristics, and 3) sources of materials. For additional information on review of the Human Subjects section, please refer to the Guidelines for the Review of Human Subjects.

Inclusion of Women, Minorities, and Children

When the proposed Center involves human subjects and/or NIH-defined clinical research, the committee will evaluate the proposed plans for the inclusion (or exclusion) of individuals on the basis of sex/gender, race, and ethnicity, as well as the inclusion (or exclusion) of children to determine if it is justified in terms of the scientific goals and research strategy proposed. For additional information on review of the Inclusion section, please refer to the Guidelines for the Review of Inclusion in Clinical Research.

Vertebrate Animals

The committee will evaluate the involvement of live vertebrate animals as part of the scientific assessment according to the following five points: 1) proposed use of the animals, and species, strains, ages, sex, and numbers to be used; 2) justifications for the use of animals and for the appropriateness of the species and numbers proposed; 3) adequacy of veterinary care; 4) procedures for limiting discomfort, distress, pain and injury to that which is unavoidable in the conduct of scientifically sound research including the use of analgesic, anesthetic, and tranquilizing drugs and/or comfortable restraining devices; and 5) methods of euthanasia and reason for selection if not consistent with the AVMA Guidelines on Euthanasia. For additional information on review of the Vertebrate Animals section, please refer to the Worksheet for Review of the Vertebrate Animal Section.

Biohazards

Reviewers will assess whether materials or procedures proposed are potentially hazardous to research personnel and/or the environment, and if needed, determine whether adequate protection is proposed.

Resubmissions

Not Applicable

Renewals

For Renewals, the committee will consider the progress made in the last funding period.

Revisions

Not Applicable

As applicable for the Center or Project proposed, reviewers will consider each of the following items, but will not give scores for these items, and should not consider them in providing an overall impact score.

Applications from Foreign Organizations

Not Applicable

Select Agent Research

Reviewers will assess the information provided in this section of the application, including 1) the Select Agent(s) to be used in the proposed research, 2) the registration status of all entities where Select Agent(s) will be used, 3) the procedures that will be used to monitor possession use and transfer of Select Agent(s), and 4) plans for appropriate biosafety, biocontainment, and security of the Select Agent(s).

Resource Sharing Plans

Reviewers will comment on whether the following Resource Sharing Plans, or the rationale for not sharing the following types of resources, are reasonable: 1) Data Sharing Plan; 2) Sharing Model Organisms; and 3) Genome Wide Association Studies (GWAS).

Budget and Period of Support

Reviewers will consider whether the budget and the requested period of support are fully justified and reasonable in relation to the proposed research.

Applications will be evaluated for scientific and technical merit by (an) appropriate Scientific Review Group(s), convened by NICHD in accordance with NIH peer review policy and procedures, using the stated review criteria. Assignment to a Scientific Review Group will be shown in the eRA Commons.

As part of the scientific peer review, all applications:

• May undergo a selection process in which only those applications deemed to have the highest scientific and technical merit (generally the top half of applications under review) will be discussed and assigned an overall impact score.
• Will receive a written critique.

Appeals of initial peer review will not be accepted for applications submitted in response to this FOA.

Applications will be assigned on the basis of established PHS referral guidelines to the appropriate NIH Institute or Center. Applications will compete for available funds with all other recommended applications submitted in response to this FOA. Following initial peer review, recommended applications will receive a second level of review by the appropriate national Advisory Council or Board. The following will be considered in making funding decisions:

• Scientific and technical merit of the proposed project as determined by scientific peer review.
• Availability of funds.
• Relevance of the proposed project to program priorities.

After the peer review of the application is completed, the PD/PI will be able to access his or her Summary Statement (written critique) via the eRA Commons.

Information regarding the disposition of applications is available in the NIH Grants Policy Statement.

If the application is under consideration for funding, NIH will request "just-in-time" information from the applicant as described in the NIH Grants Policy Statement.

A formal notification in the form of a Notice of Award (NoA) will be provided to the applicant organization for successful applications. The NoA signed by the grants management officer is the authorizing document and will be sent via email to the grantee’s business official.

Awardees must comply with any funding restrictions described in Section IV.5. Funding Restrictions. Selection of an application for award is not an authorization to begin performance. Any costs incurred before receipt of the NoA are at the recipient's risk. These costs may be reimbursed only to the extent considered allowable pre-award costs.

Any application awarded in response to this FOA will be subject to terms and conditions found on the Award Conditions and Information for NIH Grants website. This includes any recent legislation and policy applicable to awards that is highlighted on this website.

All NIH grant and cooperative agreement awards include the NIH Grants Policy Statement as part of the NoA. For these terms of award, see the NIH Grants Policy Statement Part II: Terms and Conditions of NIH Grant Awards, Subpart A: General and Part II: Terms and Conditions of NIH Grant Awards, Subpart B: Terms and Conditions for Specific Types of Grants, Grantees, and Activities. More information is provided at Award Conditions and Information for NIH Grants.

Cooperative Agreement Terms and Conditions of Award

The following special terms of award are in addition to, and not in lieu of, otherwise applicable OMB administrative guidelines, HHS grant administration regulations at 45 CFR Parts 74 and 92 (Part 92 is applicable when State and local Governments are eligible to apply), and other HHS, PHS, and NIH grant administration policies.

The administrative and funding instrument used for this program will be the cooperative agreement an "assistance" mechanism (rather than an "acquisition" mechanism), in which substantial NIH programmatic involvement with the awardees is anticipated during the performance of the activities. Under the cooperative agreement, the NIH purpose is to support and stimulate the recipients' activities by involvement in and otherwise working jointly with the award recipients in a partnership role; it is not to assume direction, prime responsibility, or a dominant role in the activities. Consistent with this concept, the dominant role and prime responsibility resides with the awardees for the project as a whole, although specific tasks and activities may be shared among the awardees and the NIH as defined below.

The PD(s)/PI(s) will have the primary responsibility for:

The PD(s)/PI(s) will have the primary responsibility for: defining objectives and approaches, and to plan, conduct, analyze, and publish results, interpretations, and conclusions of their studies. The Principal Investigator will serve as Center Director and together with a Center Co-Director, will be responsible for the integration and management of activities within the MDCRC.

The Center Director shall be responsible for organizing a local Executive Committee for day-to-day management of the MDCRC, and an external Center Advisory Committee, with scientific, clinical and patient advocate representation (final membership to be approved by the NIH). The role of these Committees will include the solicitation, review, and selection of proposals for predoctoral and postdoctoral research positions and collaborative projects, and the selection and prioritization of projects that will use resources and services that are provided for through the MDCRC.

The Center Director and Co-Director of each MDCRC also serve as members of the MDCRC Steering Committee (see below) and are required to participate in its activities, to include regular conference calls and an annual MDCRC face-to-face meeting.

Awardees agree to participate in the overall coordination of NIH research efforts in muscular dystrophy. This participation may include collaboration and consultation with other NIH awardees, the sharing of information, data, and research materials, and participation in NIH efforts to standardize and harmonize pre-clinical and clinical data collection.

Awardees with a clinical trial component in their MDCRC agree to review of associated data, abstracts, and other publications by the DSMB and the NIH prior to their release.

Awardees will retain custody of and have primary rights to the data and software developed under these awards, subject to Government rights of access consistent with current HHS, PHS, and NIH policies.

NIH staff have substantial programmatic involvement that is above and beyond the normal stewardship role in awards, as described below:

An NIH Project Scientist will have substantial programmatic involvement that is above and beyond the normal stewardship role in awards, as described below.

Each MDCRC will have the support of Project Scientists, representing each participating Institutes, and a Program Official from NIH staff who are assigned administrative roles for the muscular dystrophies being studied and have expertise in the implementation of the MDCRC Program.

The NIH Project Scientists will have substantial scientific-programmatic involvement during conduct of this activity, through technical assistance, advice, and coordination above and beyond normal program stewardship for grants. The NIH Project Scientists also will assist in the interaction between the awardee and investigators of other institutions, as well as between the awardee and other Federal agencies and/or potential commercial sponsors. The NIH Project Scientists will be members of the MDCRC Steering Committee. The NIH Project Scientists retain the option to recommend additional research endeavors within the constraints of the approved research and negotiated budget.

An important part of the NIH MDCRC Program is the coordination of research efforts across different funding mechanisms and research structures, and coordination among efforts aimed at different muscular dystrophies. The NIH Project Scientists will have the primary responsibility for this overall coordination.

The NIH Project Scientists representing NINDS, NIAMS, NICHD, and NHLBI will, collectively, have a single NIH vote on the MDCRC Steering Committee (see below). NIH Project Scientists will abstain from voting on any issue where they are unable to reach a consensus.

The NIH will establish one or more Data Safety Monitoring Boards to provide oversight and to advise the NIH on any clinical trials that are supported by the MDCRC awards.

Additionally, an NIH Program Official will be primarily responsible for program oversight. The Program Official assigned to each MDCRC will exercise the normal stewardship responsibilities of an NIH Program Official, including assessment of the progress of the projects toward the accomplishment of specified objectives. NIH Program Officials also retain the option of recommending termination of studies if technical performance falls below acceptable standards, or when specific lines of research cannot be effectively pursued in a timely manner. This Program Official will be responsible for the normal scientific and programmatic stewardship of the award and will be named in the award notice.

Areas of Joint Responsibility include:

Overall coordination of the MDCRC Program will be done by a Steering Committee. The MDCRC Steering Committee will make strategic decisions with regard to goals and research implementation, including the establishment and development of collaborations.

The Steering Committee will consist of the Center Directors and Co-Directors of each MDCRC, NIH Project Scientists and a public member. The Steering Committee will be chaired and co-chaired by MDCRC Center Directors, who are elected by vote of the Steering Committee for staggered two-year terms. The Chair and Co-Chair will be responsible for conduct of regular conference calls. The Steering Committee will hold a face-to-face meeting at least annually. Each MDCRC and the public member will have one vote on the Steering Committee and the NIH Project Scientists, collectively, will have a single NIH vote.

Each full member will have one vote. Awardee members of the Steering Committee will be required to accept and implement policies approved by the Steering Committee.

Dispute Resolution:

Any disagreements that may arise in scientific or programmatic matters (within the scope of the award) between award recipients and the NIH may be brought to Dispute Resolution. A Dispute Resolution Panel composed of three members will be convened. It will have three members: a designee of the Steering Committee chosen without NIH staff voting, one NIH designee, and a third designee with expertise in the relevant area who is chosen by the other two; in the case of individual disagreement, the first member may be chosen by the individual awardee. This special dispute resolution procedure does not alter the awardee's right to appeal an adverse action that is otherwise appealable in accordance with PHS regulation 42 CFR Part 50, Subpart D and DHHS regulation 45 CFR Part 16.

When multiple years are involved, awardees will be required to submit the Non-Competing Continuation Grant Progress Report (PHS 2590 or RPPR) annually and financial statements as required in the NIH Grants Policy Statement.

A final progress report, invention statement, and the expenditure data portion of the Federal Financial Report are required for closeout of an award, as described in the NIH Grants Policy Statement.

The Federal Funding Accountability and Transparency Act of 2006 (Transparency Act), includes a requirement for awardees of Federal grants to report information about first-tier subawards and executive compensation under Federal assistance awards issued in FY2011 or later. All awardees of applicable NIH grants and cooperative agreements are required to report to the Federal Subaward Reporting System (FSRS) available at www.fsrs.gov on all subawards over $25,000. See the NIH Grants Policy Statement for additional information on this reporting requirement.

We encourage inquiries concerning this funding opportunity and welcome the opportunity to answer questions from potential applicants.

eRA Commons Help Desk (Questions regarding eRA Commons registration, submitting and tracking an application, documenting system problems that threaten submission by the due date, post submission issues)
Telephone: 301-402-7469 or 866-504-9552 (Toll Free)
Finding Help Online: http://grants.nih.gov/support/index.html
Email: commons@od.nih.gov

Grants.gov Customer Support (Questions regarding Grants.gov registration and submission, downloading forms and application packages)
Contact Center Telephone: 800-518-4726
Web ticketing system: https://grants-portal.psc.gov/ContactUs.aspx
Email: support@grants.gov

GrantsInfo (Questions regarding application instructions and process, finding NIH grant resources)
Telephone: 301-710-0267
Email: GrantsInfo@nih.gov

Glen H. Nuckolls, Ph.D.
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Telephone: 301-594-4974
Email: nuckollg@mail.nih.gov

Tiina Urv, Ph.D.
Eunice Kennedy Shriver National Institute for Child Health and Human Development (NICHD)
Telephone: 301-402-7015
Email: tu36j@nih.gov

John D. Porter, Ph.D.
National Institute of Neurological Disorders and Stroke (NINDS)
Telephone: 301-496-5739
Email: porterjo@ninds.nih.gov

Jonathan R. Kaltman, M.D.
National Heart, Lung, Blood Institute (NHLBI)
Telephone: 301-435-0510
Email: kaltmanj@nhlbi.nih.gov

Sherry Dupere, Ph.D.
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Telephone: 301-451-3415
Email: duperes@mail.nih.gov

Sheila Simmons
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Telephone: 301-594-9812
Email: simmonsS@mail.nih.gov

Bryan S. Clark, M.B.A.
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Telephone: 301-435-6975
Email: clarkb1@mail.nih.gov

Tijuanna DeCoster, MPA
National Institutes of Neurological Disorders and Stroke (NINDS)
Telephone: 301-496-9231
Email: decostert@ninds.nih.gov

Anthony Agresti
National Heart, Lung, and Blood Institute (NHLBI)
Telephone: 301-435-0186
Email: tony.agresti@nih.gov

Recently issued trans-NIH policy notices may affect your application submission. A full list of policy notices published by NIH is provided in the NIH Guide for Grants and Contracts. All awards are subject to the terms and conditions, cost principles, and other considerations described in the NIH Grants Policy Statement.

Awards are made under the authorization of Sections 301 and 405 of the Public Health Service Act as amended (42 USC 241 and 284) and under Federal Regulations 42 CFR Part 52 and 45 CFR Parts 74 and 92.

Additionally, awards are made under the authorization of 42 U.S. Code 283g - Muscular dystrophy; initiative through Director of National Institutes of Health, and 42 U.S. Code 285d 6 - Multipurpose arthritis and musculoskeletal diseases centers.