Notice Number: NOT-HL-17-507

Key Dates
Release Date: April 7, 2017

Estimated Publication Date of Announcement: April/May 2017
First Estimated Application Due Date: June 2017
Earliest Estimated Award Date: September 2017
Earliest Estimated Start Date: October 2017

NOT-HL-17-496
NOT-HL-17-497
NOT-HL-17-498
NOT-HL-17-499
NOT-HL-17-500
NOT-HL-17-501
NOT-HL-17-502
NOT-HL-17-503
NOT-HL-17-504
NOT-HL-17-505
NOT-HL-17-506
RFA-HL-17-033

Issued by
National Heart, Lung, and Blood Institute (NHLBI)
National Eye Institute (NEI)
National Institute on Aging (NIA)
National Institute of Allergy and Infectious Diseases (NIAID)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
National Institute of Biomedical Imaging and Bioengineering (NIBIB)
National Institute on Deafness and Other Communication Disorders (NIDCD)
National Institute on Dental and Craniofacial Research (NIDCR)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
National Institute of Mental Health (NIMH)
National Institute of Neurological Disorders and Stroke (NINDS)
National Center for Advancing Translational Sciences (NCATS)
U.S. Food and Drug Administration (FDA)

Purpose

The National Institutes of Health (NIH) and participating NIH Institutes and Centers (ICs) and the U.S. Food and Drug Administration (FDA) intend to publish a Funding Opportunity Announcement (FOA) for competitive revisions that will support clinical research studies aimed at furthering the field of regenerative medicine (RM) using adult stem cells. A competing revision is a request for an increase in support in a current budget period for expansion of the project's approved scope or research protocol. Applicants must apply and undergo peer review.

The planned FOA, known as the Regenerative Medicine Innovation Project (RMIP), will support revision projects that utilize rigorous science and reproducible methods to establish proof of concept and a robust evidence base for clinical applications. In order to fulfill the mandate of advancing the field of RM more broadly, applicants will propose solutions to widely recognized issues in the development of safe and effective regenerative medicine therapies. Emphasis will be given to projects that address critical issues in product development relevant for regulatory submissions. Areas of focus may include improved tools, methods, standards, or applied science that support a better understanding and improved evaluation of product manufacturing, quality, safety, or effectiveness.

Toward these ends, the NIH will consider applications for clinical research involving adult stem cells in the context of generating or supplementing the necessary evidence for clinical development, including the submission of a pre-Investigational New Drug (IND) or pre-Investigational New Device Exemption (IDE) package; the submission of an IND/IDE application; or to support such research conducted under an authorized IND or IDE.

The FOA will utilize the P41 activity code and is expected to be published in April/May 2017 with an application due date in June 2017.

This Notice is being provided to allow potential applicants sufficient time to develop responsive proposals, consider the requirements that are integral to this initiative, and obtain necessary matching funds. Of note, this and 10 other notices are being issued for $2 million available for FY 2017 in order to solicit revision projects for the mechanisms listed herein under Related Announcements.

Research Initiative Details

The RMIP FOA represents one step in fulfilling a new statutory provision set forth in the 21st Century Cures Act. Given the potential of RM to enhance human health and treat disease, Congress included this provision for an Innovation Project in the Act authorizing a total of $30 million, with $2 million available for FY 2017 for the funding of clinical research to further the field of regenerative medicine using adult stem cells, including autologous cells. The Act stipulates that the NIH, in coordination with FDA, award funds contingent upon the recipient making available non-Federal contributions in an amount not less than $1 for each $1 of Federal funds provided in the award (i.e., a matching funds requirement).

The RMIP FOA is intended to support only research that involves: (1) human subjects or material of human origin, such as cells, tissues, and specimens; and (2) human stem cells that are not of embryonic or fetal origin. Applicable research on adult human stem cells may encompass, for example, research on biologics (e.g., growth factors, cytokines) and biomaterials (e.g., ECM, scaffolds) that stimulate host adult stem cell growth, differentiation, and function or otherwise directly act upon adult stems cells to support innate host healing mechanisms, treat disease, and/or restore function. Funding could be used, for example, for the appropriate chemistry, manufacturing, and controls development to support the production of such products for clinical trials using good manufacturing practices (GMP). Funds may not be used for research involving human cells of embryonic or fetal origin.

This FOA will support highly meritorious clinical research projects proposing to explore and enable the development of safe and effective RM interventions. Specifically, for FY 2017 funds, in addition to being subject to the standard NIH review criteria, clinical research projects will also be assessed according to the following criteria:

• Represents unfunded opportunities (can put funds to new and immediate use);
• Underlying project is well along/already underway (e.g., in the pre-IND/IDE stage or under an IND/IDE application);
• Matching funds available at time of award;
• Contributes to breadth/diversity of RM science;
• Addresses critical issues relevant to clinical research and regulatory submissions including those related to product development. Areas of focus may include improved tools, methods, standards, or applied science that support a better understanding and improved evaluation of product manufacturing, quality, safety, or effectiveness; and
• Will help to significantly build or advance the field of RM by contributing to fundamental knowledge as well as addressing a well-recognized challenge in clinical development including the development and evaluation of safe and effective RM products.

Applications that demonstrate potential to catalyze sustained and accelerated development of the RM field through contributing to the foundational knowledge critical for product development, clinical testing, and data standards and sharing, are strongly encouraged. For example, such projects may:

• Further development of standards and GMP for adult stem cell-based RM products;
• Leverage extant cell production facilities for product preparation and qualification;
• Promote and enhance mechanisms for data standardization, curation, integration, and sharing;
• Utilize clinical trial network(s) to leverage infrastructure and facilitate subject recruitment and follow up as well as data sharing; and/or
• Contribute to a better and shared understanding of current technical and operational barriers as well as the regulatory science issues.

Matching Requirement

The Government requires at least a 1:1 recipient cost matching for this program, as required by the 21st Century Cures Act. The Government will consider any non-federal funds, to include state and local funding (not originating from Federal funds), as well as private-sector investment and donations from foundations provided in support of the application as qualifying for the cost matching ratio requirement.

APPLICATIONS ARE NOT BEING SOLICITED AT THIS TIME.

Inquiries

Please direct all inquiries to:

Megan Mitchell, MPH
National Heart, Lung, and Blood Institute (NHLBI)
Telephone: 301-827-7950
Email: [email protected]