and Human Services (HHS)
EXPIRED
Participating Organization(s) |
National Institutes of Health (NIH) |
National Center for Advancing Translational Sciences (NCATS) |
|
Funding Opportunity Title |
Data Management and Coordinating Center (DMCC) for Rare Diseases Clinical Research Network (RDCRN) (U01) |
Activity Code |
U01 Research Project Cooperative Agreements |
Announcement Type |
Reissue of RFA-OD-08-002 |
Related Notices |
|
Funding Opportunity Announcement (FOA) Number |
RFA-TR-13-003 |
Companion Funding Opportunity |
RFA-TR-13-002, U54 Specialized Center- Cooperative Agreements |
Catalog of Federal Domestic Assistance (CFDA) Number(s) |
93.121, 93.853 |
Funding Opportunity Purpose |
The purpose of this Funding Opportunity Announcement (FOA) is to invite new and renewal cooperative agreement applications for the DMCC, which supports the RDCRN. The Network facilitates clinical research in rare diseases through support for 1) collaborative clinical research in rare diseases, including longitudinal studies of individuals with rare diseases, clinical studies and/or phase I , II and II/III trials; 2) training of clinical investigators in rare diseases research; 3) pilot and demonstration projects; 4) clinical data management that incorporates novel approaches and technologies for data management, data mining, and data sharing across rare diseases, data types, and platforms; and 5) access to information related to rare diseases for basic and clinical researchers, academic and practicing physicians, patients, and the lay public. The DMCC (U01) cooperative agreement application will focus on innovative ways to provide the infrastructure and support to the individual sites in their activities relevant to items 1-3 as well as a focus on issues relevant to items 4 and 5. See companion FOA RFA-TR-13-002 |
Posted Date |
October 21, 2013 |
Open Date (Earliest Submission Date) |
November 19, 2013 |
Letter of Intent Due Date(s) |
November 19, 2013 |
Application Due Date(s) |
December 19, 2013), by 5:00 PM local time of applicant organization. Applicants are encouraged to apply early to allow adequate time to make any corrections to errors found in the application during the submission process by the due date. |
AIDS Application Due Date(s) |
Not Applicable |
Scientific Merit Review |
April/May 2014 |
Advisory Council Review |
October 2014 |
Earliest Start Date |
September 2014 |
Expiration Date |
December 20, 2013 |
Due Dates for E.O. 12372 |
Not Applicable |
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). 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 Office of Rare Diseases Research (ORDR)-NCATS and NINDS invite new and renewal applications (U01) for the RDCRN Data Management and Coordinating Center (DMCC). In a companion FOA, the ORDR-NCATS, along with 10 Institutes, Centers, and Offices, invite new and renewal applications (U54) for the Rare Diseases Clinical Research Consortium (RDCRCs) component of the Rare Diseases Research Network (RDCRN). The purpose of the Network is to facilitate clinical research in rare diseases through support for 1) collaborative clinical research in rare diseases, including longitudinal studies of individuals with rare diseases, clinical studies, and/or phase I, II and II/III trials; 2) training of clinical investigators in rare diseases research; 3) pilot and demonstration projects; 4) clinical data management that incorporates novel approaches and technologies for data management, data mining, and data sharing across rare diseases, data types, and platforms; and 5) access to information related to rare diseases for basic and clinical researchers, academic and practicing physicians, patients, and the lay public. The purpose of the DMCC is to focus on innovative ways to provide the infrastructure and support to the individual sites in their activities relevant to items 1-3 as well as a focus on issues relevant to items 4 and 5.
Each RDCRC will perform collaborative multi-site clinical research in rare diseases, train new investigators in rare diseases research, and provide content for an internet resource site on rare diseases. Each RDCRC will consist of a consortium of clinical investigators, institutions, and relevant organizations, including patient advocacy group organizations, and will focus on at least three related rare diseases, disorders or syndromes. Previous experience with the RDCRN has demonstrated that RDCRCs that both engage and integrate patient advocacy groups into their research program have achieved greater success in enrollment in studies. The focus of each RDCRC can be on particular defects, e.g., lysosomal storage diseases, amino acid metabolism defects; particular organ systems, e.g., primary immune deficiencies, neurodevelopmental delay and intellectual disability syndromes; or other groupings. Since rare diseases are diverse, the nature of clinical research that is feasible varies. The individual RDCRCs will be responsible for the design and implementation of their clinical studies. The biostatistician from RDCRC will provide statistical support for protocol development and assist in study design and analysis. The DMCC will provide the data management infrastructure and support necessary for the RDCRCs to function optimally. The DMCC will work with each Consortium to integrate protocols, forms, and research tools into the Network.
Background
Approximately 25 million people in the United States are affected by an estimated 7,000 rare diseases or conditions leading to significant morbidity and mortality. 'Rare disease' is defined through an Amendment to the Orphan Drug Act of 1983 (Orphan Drug Act, P.L. 97-414; Health Promotion and Disease Prevention Amendments, P.L. 98-551) as a condition affecting fewer than 200,000 Americans or a disease with a greater prevalence but for which no reasonable expectation exists that the costs of developing or distributing a drug can be recovered from the sale of the drug in the United States.
In 1999 the NIH Office of Rare Diseases, now referred to as Office of Rare Diseases Research (ORDR) - NCATS, convened a Special Emphasis Panel comprised of academic scientists, representatives of voluntary patient support groups, pharmaceutical, biotechnology and device industries, and other Federal agencies. This panel made recommendations regarding the special research opportunities and health care issues posed by rare diseases. These recommendations encompassed four major areas: 1) Stimulating Research on Rare Diseases and Conditions with specific emphasis on clinical research and training of clinical research scientists, establishing diagnostic and treatment centers with informatics support, and promoting the collaboration of the voluntary patient support groups, health care systems, and industry; 2) Utilizing Research Resources with the NIH-supported GCRCs (now supported by Clinical and Translational Science Awards, CTSAs), the development of a centralized information database containing research resources, made available to research investigators, physicians, and patients for their use; 3) Coordination of Rare Diseases Research and Development Activities, with a primary responsibility of ORDR to coordinate activities and act as a liaison between the rare diseases community and the NIH, including the public, and intramural and extramural investigators at the NIH ICs and other Federal agencies, manufacturers, and voluntary organizations; and 4) Identifying Emerging Opportunities in Rare Diseases Research, specifically through the establishment of specialized research and diagnostic centers to attract the interests of industry to promote advances and products for the prevention, diagnosis, and treatment of rare diseases. These recommendations are contained within the Department of Health and Human Services National Institutes of Health Report on Steps to Coordinate Rare Diseases Research Programs," January 2001 http://rarediseases.info.nih.gov/html/reports/fy1999/SEP.html
In November 2002, the Rare Diseases Act of 2002 (Public Law 107-280) directed ORDR at NIH to support regional centers of excellence for clinical research into, training in, and demonstration of diagnostic, prevention, control, and treatment methods for rare diseases. This law provides the legislated mandate for this FOA to address the needs of rare disease clinical research.
Investigations into rare diseases offer promising leads for scientific advancement. Many rare diseases represent single gene defects whose abnormalities in specific genes or proteins offer insight into normal biologic function. Other rare diseases are complex, resulting from the interaction of two or more genes. Understanding the pathogenesis of rare diseases may advance our understanding of more common medical disorders.
Despite the advances and opportunities for research in rare diseases, difficulties remain in clinical diagnosis, clinical trials methodology, and clinical management. Diagnoses may be straightforward as a result of well-described phenotypes or due to the availability of diagnostics tests, or conversely, they may be challenged by a lack of well-defined diagnostic criteria. Furthermore, there are insufficient characterizations of the natural history of many rare diseases. Treatment can be equally challenging with many questions concerning clinical management, and a lack of therapeutic options. Rare diseases pose unique challenges to identification and coordination of resources and expertise for small populations dispersed over wide geographic areas. Rare diseases research often requires collaboration of scientists from multiple disciplines sharing research resources and patient populations. Rigorous characterization and longitudinal assessment is needed to facilitate discovery of biomarkers of disease risk, disease activity, and response to therapy. In addition, systematic assessment could help to improve and develop and evidence base for current treatment strategies. Well described patient populations will be important to bring promising therapies to the clinic.
Purpose
The Rare Diseases Clinical Research Network (RDCRN) will consist of all funded Rare Diseases Research Consortia (RDCRC) and a single Data Management and Coordinating Center (DMCC). This initiative will support the continuation of a collaborative and coordinated network of RDCRCs comprised of investigators at multiple institutions/sites and patient advocacy groups committed to investigation of rare diseases working in partnership to enhance communication and sharing of resources in a multidisciplinary approach. The reissuance of the DMCC RFA is open to new data management centers as well as the existing RDCRN data management center. The RDCRN (Network) focuses on the collection of clinical information to develop biomarkers and new approaches to diagnosis, prevention, and treatment and promote the training of new clinical investigators in rare diseases research. The Network supports a comprehensive and integrated approach to data collection, storage, and management, and the integration of clinical data with other unique data, including genetic, imaging, pathologic, and laboratory data. This DMCC will continue to serve the needs of the Network while striving to incorporate new approaches and technologies to increase efficiencies while lowering costs.
Each RDCRC within the Network (established under FOA TR-13-002) will consist of a consortium of clinical investigators, multiple institutions, and relevant organizations, including patient advocacy groups and will focus on at least three related rare diseases, disorders, conditions or syndromes that are relevant to the interests of the participating NIH Institutes and Centers (ICs). The DMCC will serve as a Network resource, working with and providing expertise for the RDCRCs. It will provide a scalable coordinated clinical data integration of developed and publicly available datasets for data mining at RDCRCs, web-based recruitment and referral, and a user-friendly resource site for the public. The DMCC will provide a management system for collection, storage, as well as a portal and tools for research scientists and clinicians. In addition, the DMCC, in conjunction with the NIH, will provide logistical and administrative assistance for Network activities; produce and/or maintain Network Operating Policy and Procedures, documents, worksheets, and data collection forms; and monitor Network compliance while addressing privacy and confidentiality issues related to database management, and multi-level data sharing. In order to participate in this Network each RDCRC is expected to use the DMCC supported by RDCRN. All RDCRCs will be expected to collaborate with the DMCC throughout the course of their study in order to assure compatibility and standardization of data management approaches.
Organization of the Rare Diseases Clinical Research Network
The Rare Diseases Clinical Research Network (RDCRN) is a cooperative network composed of multiple Rare Diseases Clinical Research Consortia (RDCRC) and a single DMCC to facilitate clinical research in rare diseases carried out by the RDCRCs. A Steering Committee, composed at a minimum of the Principal Investigator (Director) of each RDCRC, the Principal Investigator (Director) of the DMCC, the chairperson of the Coalition of Patient Advocacy Groups (CPAG), and the ORDR-NCATS Program Coordinator for RDCRN will establish the procedures for the function of the RDCRN, as outlined in section "Steering Committee." Membership of CPAG will include all participating patient advocacy groups.
The RDCRN will require cooperation among the ORDR-NCATS Program Coordinator for RDCRN, the participating IC Project Scientists, Directors of the RDCRC and their collaborators, and the Director of the DMCC to maximize their effectiveness. A number of issues need to be addressed in the cooperative agreement applications including those highlighted in Organization of the Rare Diseases Clinical Research Network and below under Cooperative Agreement Terms and Conditions.
The DMCC will develop and make available a secure, customizable coordinated clinical data management system for collection, storage, and analysis of diverse data types from multiple diseases and geographically disparate locations. The DMCC should develop and provide (as needed) a user friendly system for web-based recruitment and referral, tools for cross disease data mining, and a portal for access and integration of publicly available data resources. The DMCC should have computational sophistication for scaling the systems and tools to allow incorporation into a distributed, national clinical information network. The DMCC must address privacy and confidentiality issues related to database management and distributed computing and allow multiple levels of data sharing. The DMCC must also be able to work with consortia that have preexisting infrastructure (registries, patient databases, etc.) as well as those that are starting de novo. The Steering Committee will provide scientific and technical assistance and guidelines with respect to quality control, uniformity of data collection, management of the collective rare diseases database, and data analysis.
The DMCC will work collaboratively with the consortia to develop RCDRC-specific websites. The actual design and implementation of the site will be a collaborative activity of the DMCC and all of the RDCRC through the Steering Committee (see below). Each RDCRC and the DMCC must agree to work cooperatively to develop the web site resource and provide content related to the RDCRC’s specific rare diseases. After receiving funding, an RDCRC will work cooperatively with DMCC to develop the web site resource and provide content related to the RDCRC specific rare diseases. The DMCC will support the websites for communicating disease information. During the 5-year funded period, if an RDCRC proposes to collect biospecimens, the RDCRC must register contact information regarding its Biorepository with the Biospecimen/Biorepositories Website RD-HuB (http://biospecimens.ordr.info.nih.gov/). RDCRCs are also encouraged to upload information about their biospecimen into the RD-HuB specimen locator on this website to help researchers utilize available specimens.
See Section VIII, Other Information - Required Federal Citations, for policies related to this announcement.
Funding Instrument |
Cooperative Agreement: A support mechanism used when there will be substantial Federal scientific or programmatic involvement. Substantial involvement means that, after award, NIH scientific or program staff will assist, guide, coordinate, or participate in project activities. |
Application Types Allowed |
New |
Funds Available and Anticipated Number of Awards |
NCATS and NINDS intend to commit an estimated total of $3 million in FY 2014 to fund one (1) cooperative agreement application in response to this FOA for the support a Data Management and Coordinating Center (DMCC) that will provide necessary functions for the support of up to seven new and/or renewal grant applications in response to companion RFA TR-13-002. If additional Institute funds become available, more consortia may be funded. Should this happen, the ORDR-NCATS will increase support of the DMCC up to $4 million per year. Any award issued in response to this FOA is contingent upon funds availability, the quality of applications received, and programmatic priorities. |
Award Budget |
Application budgets are limited to $3 million total costs per year |
Award Project Period |
Applicants must request 5 years of support. |
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
The following types of Higher Education Institutions are always encouraged to apply for NIH support as Public or Private Institutions of Higher Education:
Nonprofits Other Than Institutions of Higher Education
For-Profit Organizations
Governments
Other
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.
Program Directors/Principal Investigators (PD(s)/PI(s))
All PD(s)/PI(s) must have an eRA Commons account and should work with their organizational officials to either create a new account or to affiliate an existing account with the applicant organization’s eRA Commons account. 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.
Applications with multiple PDs/PIs are not allowed.
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.
NIH will not accept any application that is essentially the same as one already reviewed within the past thirty-seven months (as described in the NIH Grants Policy Statement), except for submission:
Applicants must download 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.
It is critical that applicants follow the instructions in the SF424 (R&R) Application Guide, except where instructed in this funding opportunity announcement to do otherwise. 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:
The letter of intent should be sent to:
David J. Eckstein, Ph.D.
Office of Rare Diseases Research
National Center for Advancing Translational Science
(NCATS)
Telephone: 301-402- 4336
Fax: 301-480-9655
Email: [email protected]
All page limitations described in the SF424 Application Guide and the Table of Page Limits must be followed
The forms package associated with this FOA includes all applicable components, required and optional. Please note that some components marked optional in the application package are required for submission of applications for this FOA. Follow all instructions in the SF424 (R&R) Application Guide to ensure you complete all appropriate optional components.
The following section supplements the instructions found in the SF424 (R&R) Application Guide and should be used for preparing an application to this FOA.
All instructions in the SF424 (R&R) Application Guide must be followed.
All instructions in the SF424 (R&R) Application Guide must be followed.
All instructions in the SF424 (R&R) Application Guide must be followed, with the following additional instructions:
The applicant must address the resources available in order to develop and make available a secure, customizable coordinated clinical data management system for collection, storage, and analysis of diverse data types from multiple diseases and geographically disparate locations; to develop and provide (as needed) a user friendly system for web-based recruitment and referral, tools for cross disease data mining, and a portal for access and integration of publicly available data resources; to have computational sophistication for scaling the systems and tools to allow incorporation into a distributed, national clinical information network; and to address privacy and confidentiality issues related to database management and distributed computing and allow multiple levels of data sharing. This should include a brief description of the features of the institutional environment that are or would be relevant to the effective implementation of the proposed program. As appropriate, describe available resources, geographic distribution of space and personnel, and consultative resources.
In addition, each DMCC applicant must provide a statement that addresses how the institutional commitment will be established and sustained, and how the DMCC efforts will be given a high priority within the institution. The institutional commitment may be in the form of support for recruitment of scientific talent, provision of discretionary resources to the DMCC Director, assignment of space, cost sharing of resources, and/or other ways proposed by the applicant institution. Letters from a high-level institution official(s) (e.g., Dean of the School of Medicine, President, and Vice President for Research) should be attached confirming this commitment. The institution that submits the U01 application must receive a formal written agreement(s) from the other participant organization(s), if any. This agreement should clearly delineate the institutional commitment of the participating organization(s) (in the ways outlined above) to the DMCC Program.
All instructions in the SF424 (R&R) Application Guide must be followed.
All instructions in the SF424 (R&R) Application Guide must be followed. The DMCC Director (PD/PI of the application) is expected to make a commitment of at least 2.0 months to the overall administration of the program.
Note that this cooperative agreement award mechanism is not intended for the acquisition of costly equipment, which should be funded through other sources. Under unusual circumstances, where costly items of equipment are requested, the application must document available equipment within the institution and provide clear justification.
All instructions in the SF424 (R&R) Application Guide must be followed.
All instructions in the SF424 (R&R) Application Guide must be followed, with the following additional instructions:
Specific Aims:
Research Strategy:
Describe the proposed program, including any plans for improving, augmenting, or changing the current RDCRN DMCC program. This description must include a detailed plan for addressing the DMCC responsibilities outlined below in the Research Plan. The ability to carry out the proposed program must also be addressed. Describe the proposed data management and coordination program for the RDCRN, indicating the experience in serving as a data management center for a large multi-institution research network. Describe the nature of the multidisciplinary team necessary to provide the internet, technical, and statistical resources required to maintain the DMCC. Include plans and approaches for interacting with a consortium of clinical investigators, institutions, and relevant organizations, including patient support organizations, focused on multiple subgroups of rare diseases.
To accomplish the program goals of the RDCRN, the DMCC must serve as a core resource to the Network and be able to provide a number of services and activities. DMCC applications must address the ability to and experience in providing the following services:
1) Provide a secure, customizable, scalable coordinated clinical data management system for the integration of clinical data with other unique data, including genetic, imaging, pathologic, and laboratory. This support should include developing and/or adapting new technologies and technological advances to facilitate data collection, storage, and management from clinical researchers working on many different types of rare diseases in geographically disparate locations;
2) Provide a portal and tools for integration of developed and publicly available datasets for data mining at RDCRCs;
3) Provide for web-based recruitment and referral;
4) Provide a user-friendly resource site for the public, research scientists, and clinicians;
5) Incorporate new approaches to distributed computing and federated databases;
6) Incorporate new approaches to database support and clinical study management to reduce the burden of rising costs of software as well as reducing the time and financial burdens of clinical site audits.
7) Address privacy and confidentiality issues related to database management and distributed computing and allow multiple levels of data sharing;
8) Provide logistical and administrative assistance in arranging meetings of the Steering Committee as well as the CPAG and provide other operational support for the Network (e.g., communications, subcommittee meetings). The DMCC will prepare, distribute, and maintain minutes of the meetings;
9) While the consortia will be responsible for the training necessary at the onset of new clinical protocols, this training must be done in conjunction with DMCC personnel so that all new forms and documents can appropriately be incorporated into the Network data management system;
10) Produce and maintain all Network documents, including Network Operating Policy and Procedures manuals;
11) Develop and maintain a "listserv" interactive email system for communication within the Network;
12) Develop worksheets and study data forms, as needed, for the collection of data in multi-center studies, verify all data, develop tests, and maintain software for within-form edit checks at data entry. For worksheets, forms, or other tools developed within a consortium, the DMCC will work to integrate the developed products into the Network data management system;
13) Develop uniform investigative clinical research protocols for data (and specimen) collection in collaboration with the Steering Committee;
14) Provide support services for the production of reports, graphics, and other materials as required;
15) Monitor Network protocol adherence, data collection and data submission, and report violations to the Steering Committee as well as other required entities;
16) Work with the individual Data and Safety Monitoring Boards to establish protocols for Adverse Events notification and reporting;
17) Coordinate site visits for auditing individual consortia sites;
18) Provide scalable infrastructure. The RDCRN is a pilot project and its size and scope may expand in the future. A successful applicant must have the ability and resources to expand its operations to meet such future needs.
Renewal Applications: Additional Material Required
All applications for renewal must provide information documenting the progress of the DMCC in the last funding period. Applicants should outline the DMCC accomplishments, including any pilot programs or newly developed tools, and discuss their potential impact on individual rare disease consortia as well as the RDCRN. Any difficulties in achieving the previously proposed specific aims should be addressed. See Section V. Application Review Information, Additional Review Criteria - Overall, for the renewals review criteria to be addressed.
Letters 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.
When conducting clinical research, follow all instructions for completing Planned Enrollment Reports as described in the SF424 (R&R) Application Guide.
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). 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. NIH and Grants.gov systems check the application against many of the application instructions upon submission. Errors must be corrected and a changed/corrected application must be submitted to Grants.gov on or before the application due date. If a Changed/Corrected application is submitted after the deadline, the application will be considered late.
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.
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) 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. See Section III of this FOA for information on
registration requirements.
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. 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.
In order to expedite review, applicants are requested to notify the NCATS Referral Office by email at [email protected] when the application has been submitted. Please include the FOA number and title, PD/PI name, and title of the application.
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.
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).
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 project that by its nature is not innovative may be essential to advance a field.
Significance
Does the application address an important problem or a critical barrier to progress in the field? If the aims of the application 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?
Does this application provide a significant contribution to the support of the RDCRN? If the aims of the application are achieved, how will the activities of the Network be advanced? What will be the effect of these efforts on the methods, technologies, or services provided to the Network?
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? Is the DMCC Director (and DMCC Co-Director, if any) appropriately trained and well suited to carry out this work? Does the DMCC Director have experience in leading a core facility such as the DMCC? Is the work proposed appropriate to the experience level of the principal investigator and other significant contributors? Does the DMCC team bring complementary and integrated expertise and experience to the project? Are the significant contributors committed to the collaborative and cooperative nature of this program? Are the scientific qualifications and involvement of the DMCC Director and DMCC Co-Director (if any) as well as his/her scientific and administrative leadership capabilities and time commitment presented and sufficient for the requirements of the proposed DMCC? Does the DMCC Director have the ability to expand operations to meet the needs of a growing RDCRC Network?
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? Is the application original and innovative? For example: Does the project challenge existing paradigms or methodologies; employ an innovative approach or overcome a critical barrier to progress in the field? Does the application develop or employ novel concepts, approaches, methodologies, tools, or technologies to facilitate data collection, storage, and management from clinical researchers working on many different types of rare diseases in geographically disparate locations, including those that result in significant cost savings for clinical site audits and reduce the burden of rising costs of software? Are there novel methods for recruitment and outreach to health professionals, researchers, and community or patient organizations?
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 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? Are the designs, methods, and analyses adequately developed, well
integrated, well reasoned, and appropriate to support the RDCRN? Does the
proposed approach provide for a secure, customizable, scalable coordinated
clinical data management system for the integration of clinical data with other
unique data, including genetic, imaging, pathologic, and laboratory? Does the
proposed plan adequately address the informational tools necessary to support a
cooperative research network such as the RDCRN such as: the development of a
portal and tools for data mining; plans for web-based recruitment and referral;
the development of a user-friendly resource site for the public, research
scientists, and clinicians? Will this approach include developing and/or
adapting new technologies and technological advances for distributed computing
and federated databases? Does the applicant adequately address privacy and
confidentiality issues? Does the applicant acknowledge potential problem areas
and consider alternative tactics? Is the proposed plan appropriate for the
state of knowledge, current capabilities and resources for the rare diseases
research community? Is the structure of the program sufficiently
multidisciplinary in order to meet the needs of the RDCRN as well as the needs
of the rare disease community? Is the approach adequate to support the
logistical, administrative, and operational support needs as described in
Subsection 2 of Section IV Application and Submission Information ?
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? Does the proposed plan provide unique features to support the RDCRN? Do the plans for integrating the activities of DMCC into the RDCRN give confidence and sufficient evidence that such efforts are likely to be effective? Does the applicant have adequate resources at the institution to provide logistical and administrative assistance as well as operational support, including but not limited to website maintenance, meeting support, maintenance of Network documents and email listservs, and coordination of site visits for audits? Are resources available from the institution to allow expansion of DMCC activities to accommodate growth of the RDCRC Network?
As applicable for the project 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.
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 project 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.
Are the progress and achievements specific to the mission and relevant to the support of the RDCRN? Are the justifications for adding any new activities or programs appropriate and acceptable?
Has adequate progress been demonstrated on the development of a secure, customizable, scalable coordinated clinical data management system?
Are any difficulties in achieving the previously proposed specific aims addressed? Do the new specific aims constitute logical extensions of the previous funding period?
Is it evident that the DMCC team established productive working relationships among the various Network participants during the past performance period?
Have manuscripts describing previous findings been published or submitted for publication?
Revisions
Not Applicable
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.
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 the National Center for Advancing Translational Sciences,, 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:
Appeals of initial peer review will not be accepted for applications submitted in response to this FOA.
Applications will be assigned 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 NCATS Advisory Council. The following will be considered in making funding decisions:
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 the DUNS, SAM
Registration, and Transparency Act requirements as noted on the Award
Conditions and Information for NIH Grants 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 U.S. Office of Management and Budget (OMB) administrative guidelines, U.S. Department of Health and Human Services (DHHS) 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 (U54), 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/PI of the DMCC will have the primary responsibility for defining the details of the project within the guidelines of the RFA-TR-13-003 and for performing the scientific activity, and agrees to accept close coordination, cooperation, and participation of the NIH staff in those aspects of the scientific and technical management of the project described below. Specifically, awardees have primary responsibility as described below.
RDCRC Director and the DMCC Director
The Rare Diseases Clinical Research Consortium Directors (RDCRC Directors) with the assistance of the Data Management and Coordinating Center Director (DMCC Director) are responsible for the overall management of their RDCRC (Consortium) and coordination with the other Consortia. The relationship between the Consortia and the Data Management and Coordinating Center (DMCC) should be one of equal partners in the Rare Disease Clinical Research Network (Network).
Collaboration and Coordination
The collaboration of investigators between Consortia is highly encouraged based on shared interests and complementary talents and will be facilitated, when possible, by the DMCC.
Steering Committee Membership and Meeting Attendance
Each Consortium Principal Investigator, including the Principal Investigator of the Data Management and Coordinating Center, will be designated the Consortium Director. Each Consortium Director will be a voting member of the Network Steering Committee and participate in all Committee activities and decisions including, but not limited to, conference calls and special subcommittees as may be necessary. The Steering Committee shall be responsible for determining the frequency of meetings and scheduling the time and location. The Steering committee will establish the procedures for the function of the Consortia network, as outlined in section "Steering Committee."
Data Coordination and Management and Sharing
The RDCRC awardees will have primary rights to all data developed under those awards, subject to Government rights of access consistent with HHS and NIH policies. The DMCC will develop a data management system with the input of the Steering Committee. All Consortia will place their data at the DMCC. The intention of the NIH is that the data collected within this Network will become a resource for the Rare Disease Community and be made available to the scientific community through an ORDR-governed data repository. Criteria and mechanisms for data sharing among investigators within the Network and with the scientific community will be developed by the Steering Committee. The DMCC will also coordinate with NIH program staff including registration with and data uploading of appropriate RDCRN studies to ORDR-governed data repository (through dbGaP, a database for genotypes and phenotypes, National Library of Medicine). Data transfer to dbGaP is expected to occur on regular basis according to the data sharing policy for RDCRN established by the Steering Committee and as approved by NIH staff.
Publication and Presentation of Study Findings
Timely publication of major findings is encouraged. Publications and oral presentations of work performed under this agreement will require appropriate acknowledgment of the Rare Disease Clinical Research Network, ORDR-NCATS, and NIH ICs support. The Steering Committee will establish unifying procedures and criteria for presentation and publication of data developed within the Rare Disease Clinical Research Network so that these procedures and criteria are consistent across the Network.
Federally Mandated Regulatory Requirements
Each institution participating in the Rare Diseases Clinical Research Network is required to meet DHHS regulations for the protection of human subjects and FDA requirements for the conduct of research using investigational agents. At a minimum, these include:
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 from NINDS will have substantial programmatic
involvement that is above and beyond the normal stewardship role in awards, as
described below.
One representative from ORDR-NCATS will be designated to serve as the Program
Coordinator for this cooperative agreement. The ORDR-NCATS Program Coordinator
for DMCC and one Project Scientist from NINDS will have substantial
scientific/programmatic involvement during the conduct of this activity through
technical assistance, advice and coordination above and beyond normal program
stewardship for awards, as described below.
Steering Committee Membership and Meeting Attendance
The NIH Program Coordinator from ORDR-NCATS and the Project Scientists from NINDS will serve on the Steering Committee (see below) and will participate in all Committee activities, including, but not limited to, meetings, conference calls, subcommittees, and special committees. They will assist in development of operating policies, quality control procedures, and policies that require cooperative action. While the ORDR Program Coordinator and NINDS Project Scientist will attend Steering Committee Meetings along with the Project Scientists from the RDCRN consortia, their cumulative votes may never exceed 1/3 of the total committee votes.
Monitoring Performance
The ORDR-NCATS Program Coordinator and IC Project Scientists will assist the Steering Committee in the development of procedures for monitoring the performance of the clinical studies. This includes participation in periodic on-site monitoring with respect to compliance with protocol specifications, quality control and accuracy of data recording, and accrual.
Publication and Presentation of Clinical Studies Findings
The NIH staff may contribute, through review, comment, analysis, and/or co-authorship, to reporting results of the clinical studies to the investigator community and other interested scientific and lay organizations. Co-authorship by the NIH staff will be subject to approval in accordance with the NIH policies regarding staff authorship of publications resulting from extramural awards.
The Government, via the ORDR-NCATS Program Coordinator, IC Project Scientists and Program Officials, will have access to data generated under this Cooperative Agreement and may periodically review the data and progress reports. Information obtained from the data may be used by NIH staff for the preparation of internal reports on the activities of the clinical studies. However, awardees will retain custody of and have primary rights to all data developed under these awards.
Program Stewardship
The assigned Program Official from NINDS will be responsible for normal programmatic stewardship and monitoring of this award. The Program Official will not serve as the substantively involved IC Project Scientist. They may receive input and recommendations from other NIH staff in monitoring the awards.
Areas
of Joint Responsibility include:
All investigators within each Consortium and the DMCC must be willing to work
cooperatively and collaboratively both within their Consortium and with other
Consortia. Each Consortium is expected to send two Consortium participants to
two 2-day meetings in Washington, D.C. area each year.
Steering Committee
A Steering Committee will be established to serve as the main governing body of the cooperative network. At a minimum, the Steering Committee will be composed of one representative from each of the Consortium, one representative from the DMCC, the NIH Program Coordinator from ORDR-NCATS, and other participating IC Project Scientists. All members are expected to actively participate in all Steering Committee activities. The combined vote of NIH membership may never exceed 40 percent.
The Chairperson of the Steering Committee will be selected by the Steering Committee from among the non-Federal members during one of the early meetings of the Committee to be convened by the NIH Program Coordinator from ORDR-NCATS. All major decisions will be made by the Steering Committee. The Committee will meet at least semi-annually. As needed, the Steering Committee may establish subcommittees for special purposes. It is expected that most of the work of the Steering Committee will be performed in these subcommittees. All Consortia and DMCC must abide by decisions of the Steering Committee.
The Steering Committee will have responsibility of facilitating
the conduct of the clinical studies, promoting trans-Consortium collaboration,
establishing and updating the content of the web resource site, and
establishing procedures for reporting results of Consortium studies. 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 regulations 42 CFR Part 50, Subpart D and HHS regulations 45 CFR Part 16.
When multiple years are involved, awardees will be required to submit the annual Non-Competing Progress Report (PHS 2590 or RPPR) 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)
Web ticketing system: https://public.era.nih.gov/commonshelp
TTY: 301-451-5939
Email: [email protected]
Grants.gov Customer Support (Questions
regarding Grants.gov registration and submission, downloading forms and
application packages)
Contact CenterTelephone: 800-518-4726
Web ticketing system: https://grants-portal.psc.gov/ContactUs.aspx
Email: [email protected]
GrantsInfo (Questions regarding application instructions and
process, finding NIH grant resources)
Telephone: 301-710-0267
TTY 301-451-5936
Email: [email protected]
David J. Eckstein, Ph.D.
Senior Health Scientist Administrator, Office of Rare Diseases Research
National Center for Advancing Translational Sciences (NCATS)
Telephone: (301) 402 4336
Fax: (301) 480 9655
Email: [email protected]
Randall R. Stewart, Ph.D.
Program Director for Channels, Synapses and Circuits
National Institute of Neurological Disorders and Stroke (NINDS)
Telephone 301-496-1917
Fax 301-402-1501
E-mail: [email protected]
Carol Lambert, Ph.D.
Office of Scientific Review
National Center for Advancing Translational Sciences
(NCATS)
Telephone: 301-435-0814
Fax: 301-480-3660
Email: [email protected]
Tijuanna Decoster
Chief, Grants Management Branch
National Institute of Neurological Disorders and Stroke
(NINDS)
Telephone: (301) 496-9231
Fax: (301) 402-0219
Email: [email protected]
Vicki Maurer
Grants Management Specialist, Office of Grants Management
National Center for Advancing Translational Sciences (NCATS)
Telephone: (301) 435-0837
Fax: (301)-480-3777
Email: [email protected]
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.
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