MOU 225-24-021

MEMORANDUM OF UNDERSTANDING
Between the
FOOD AND DRUG ADMINISTRATION,
CENTER FOR BIOLOGICS EVALUATION AND RESEARCH
and the
NATIONAL INSTITUTES OF HEALTH,
DIVISION OF PROGRAM COORDINATION, PLANNING AND STRATEGIC INITIATIVES
OFFICE OF STRATEGIC COORDINATION

I. PURPOSE

This Memorandum of Understanding (MOU) between the Food and Drug Administration/Center for Biologics Evaluation and Research (FDA/CBER) and the National Institutes of Health Division of Program Coordination, Planning and Strategic Initiatives, Office of Strategic Coordination (NIH/DPCPSI/OSC), each referred to as a “Party” and collectively referred to as “the Parties,” provides a framework for coordination and collaborative efforts between these two entities, which are both components of the Department of Health and Human Services. This MOU provides the principles and procedures for information sharing between FDA/CBER and NIH/DPCPSI/OSC.

II. BACKGROUND

FDA and NIH are sister agencies within the Department of Health and Human Services. Both FDA and NIH exist and work to protect the public health but have different statutory mandates and responsibilities.

FDA is a science-based regulatory agency authorized to enforce the Federal Food, Drug, and Cosmetic Act (the Act). In fulfilling its responsibilities under the Act, FDA, among other things, directs its activities toward promoting and protecting the public health by assuring the safety, efficacy, and security of drugs, veterinary products, biological products, medical devices and radiological products and the safety and security of foods, dietary supplements, and cosmetics.
FDA also has responsibility for regulating the manufacturing, marketing, and distribution of tobacco products to protect the public health and to reduce tobacco use by minors. To accomplish its mission, FDA must stay abreast of the latest developments in research and communicate with stakeholders about complex scientific and public health issues. Within FDA, CBER's mission is to ensure the safety, purity, potency, and effectiveness of biological products including vaccines, allergenics, blood and blood products, and cells, tissues, and gene therapies for the prevention, diagnosis, and treatment of human diseases or conditions. The regulation of these products is founded on science and law to ensure their purity, potency, safety, and efficacy.

NIH is the Federal focal point for biomedical research in the United States. The NIH mission is to uncover new knowledge that will lead to better health for everyone. NIH works toward that mission by conducting research in its own laboratories; supporting the research of non-Federal scientists in universities, medical schools, hospitals, and research institutions throughout the country and abroad; helping in the training of research investigators; and fostering communication of medical information.

The NIH/DPCPSI/OSC’s Somatic Cell Genome Editing (SCGE) program aims to reduce the burden of diseases caused by genetic changes. Genome editing technologies present an exciting prospect for treatments and possibly even cure for these diseases. The program is implemented as a Common Fund program and supported by a working group of NIH SCGE Program staff.
The goal of SGCE Phase I was to improve the efficacy and specificity of genome editing approaches. The SCGE Consortium of funded investigators developed new methods and improved systems that delivered genome editing machinery into various tissues with greater specificity, including tissues that present an unmet need and that are harder to reach such as the brain, ear, heart, and lung. The goal of SCGE Phase II is to accelerate the translation of genome editing therapies into the clinic by developing targeted delivery technologies; advancing clinical development and evaluation of novel genome editing therapeutics; establishing new regulatory pathways to lay the groundwork for clinical trials that assess the safety and efficacy of promising genome editing therapies to treat multiple diseases; and disseminating successful strategies for starting clinical trials through a publicly accessible platform. The NIH SCGE Consortium will consist of four initiatives, as overseen by NIH SCGE Program staff:

• Developing technologies and assays for safety and efficacy studies to aid in developing genome editing treatments.
• Optimizing genome editing-based therapeutic leads to support advancement of a platform approach towards IND submissions.
• Supporting novel genome editing clinical trials approaches for multiple diseases.
• Establishing a Translational Coordination and Development Center to foster collaboration and share new technologies and protocols with the public and research community.

NIH’s and FDA’s respective missions to protect the public health are complementary and may overlap depending upon the subject matter. The agencies work collaboratively to protect and improve public health. Sometimes FDA/CBER or NIH/DPCPSI/OSC may have information that could be useful to the other unit in that unit's performance of its responsibilities. Timely sharing of information between NIH/DPCPSI/OSC and FDA/CBER is therefore critical to protect and improve the public health.

III. SUBSTANCE OF AGREEMENT AND RESPONSIBILITIES OF EACH AGENCY

A. Coordination and Collaboration Relative to Public Health Activities

It is mutually agreed that, on an as needed basis and as resources permit:

1. FDA/CBER and NIH/DPCPSI/OSC will coordinate and collaborate with each other to protect and improve the public health pertaining to genome editing based therapeutics development. To achieve this, each Party will capitalize on the expertise, resources, and relationships of the other to increase its own capability and readiness to respond to evolving research and regulatory science needs and opportunities. In addition, each Party will designate central contact points to coordinate communications from the other, dealing with matters covered by this agreement.

2. Each Party will participate in periodic meetings to promote better communication and understanding of regulations, policies, and statutory responsibilities, and to serve as a forum for questions and challenges that may arise.

3. Each Party may notify the other when issues of mutual concern become evident to the extent such notification does not interfere with the public health, oversight, enforcement, or compliance responsibilities of the notifying agency.

4. Parties will present reciprocal in-house presentations to their corresponding staff on topics of common interest such as FDA’s managed regulatory review process and NIH/DPCPSI/OSC’s extramural, federally funded translational and clinical research initiatives and programs, specifically those applicable to development of genome- editing based therapies for treatment of different human genetic diseases.

5. Where appropriate, FDA/CBER will provide relevant reference documents that describe the investigational product review process and marketing approval processes for use by NIH SCGE program staff involved in conferring with prospective translational and clinical research grantees to assist in the design and implementation of nonclinical and clinical studies that follow FDA/CBER guidance and regulations.

6. FDA/CBER and NIH/DPCPSI/OSC’s MOU Parties will seek opportunities to work collaboratively to improve efficiency of the submission and review process for nonclinical and clinical investigator applications that are expected to require submission of an Investigational New Drug (IND) application to FDA/CBER. For example, potential areas of collaboration could include the identification of criteria and conditions to harness the “sameness” of one Genome Editing product to inform the development of another similar product for the same or different disease indication; or the determination of prerequisites to designate a platform technology with a reasonable likelihood to bring significant efficiencies to the development, and/or manufacturing process and/or to the review process of SCGE programs.

7. Parties will promote communication and consultation on select policy issues and guidance documents of particular interest and relevance to researchers, consumers and/or health care professionals pertaining to genome editing based therapies. This cooperative interaction will target possible health risk posed to patients as well as address research and regulatory processes affecting the pace of translation of genome editing therapeutics from bench to bedside. As an example, during drafting of policy documents such as FDA Guidance for Industry or NIH Points-to-Consider that apply to genome editing based therapies, each Party is encouraged to seek input from its counterpart in order that appropriate modifications to draft documents may be made prior to initiation of a formal clearance process.

8. Conduct periodic NIH/CBER meetings to facilitate ongoing exchange of information and to identify joint future actions.

9. As appropriate and in compliance with all applicable statutes and regulations, FDA/CBER will invite participation of NIH/SCGE Program staff experts in pre-decisional evaluation of selected INDs for clinical studies involving novel genome editing based products whose scientific and clinical aspects may be complex and non-conventional.

10. NIH SCGE Program staff will provide opportunity for FDA/CBER staff to participate in NIH/SCGE Consortium-sponsored conferences that pertain to development of genome editing-based products. FDA/CBER contributions may include: (1) participation in workshops, (2) individual presentations, and (3) use of existing recorded FDA conferences/workshops on selected regulatory policy and process issues.

11. This MOU does not preclude NIH/DPCPSI/OSC or FDA/CBER from entering into other agreements that may enable special programs to be handled more efficiently and expertly.

B. Principles and Procedures for the Sharing of Non-Public Information

FDA/CBER and NIH/DPCPSI/OSC agree that the following principles and procedures will govern the sharing of non-public information, as resources permit, between the two parties.

Although there is no legal requirement that FDA/CBER and NIH/DPCPSI/OSC exchange information in all areas, the parties agree that there should be a presumption in favor of full and free sharing of information between FDA/CBER and NIH/DPCPSI/OSC. As public health agencies within the Department of Health and Human Services, there are no legal prohibitions that preclude FDA or the NIH from sharing with each other most information in the possession of either agency. Both parties recognize and acknowledge, however, that it is essential that any non-public information that is shared between FDA/CBER and NIH/DPCPSI/OSC whether written or oral must be protected from any disclosure that is not authorized by law or regulation. See e.g., 21 U.S.C. § 331(j), 18 U.S.C. § 1905, 21 CFR Parts 20 and 21, 45 CFR Parts 5 and 5b, and 42 U.S.C. § 241(d). Safeguards are needed to protect non- public information shared, both written and oral, such as trade secrets and confidential commercial information; identities of study participants and other personal privacy information; privileged and/or pre-decisional agency information; research proposals, progress reports, and/or unpublished data. The sharing of national security information is not contemplated by this MOU. Such safeguards also help ensure FDA/CBER’s and NIH/DPCPSI/OSC’s compliance with all applicable laws and regulations.

To facilitate the sharing of non-public information, written or oral, FDA/CBER and NIH/DPCPSI/OSC will implement procedures to ensure that such sharing is appropriate and that the recipient party will guard the confidentiality of all information received. Both parties are committed to responding to requests for information in a complete and timely manner, consistent with budgetary and resource constraints, and to the extent permitted by law, regulation or agency policy and practice. The party receiving shared non-public information (requesting party), whether written or oral, will be responsible for protecting that information from any unauthorized disclosure. Provisions for sharing of non-public information, both written and oral, in accordance with applicable statutes or regulations are set out below:

1. The requesting party must specify, in writing, the information requested (to facilitate identification of relevant information), provide a brief statement of why the information is needed, and include the following requesting party template language: “This request is made pursuant to the Memorandum of Understanding for Sharing of Non-Public Information between FDA/CBER and NIH/DPCPSI/OSC, dated [insert date agreement was signed]. [Requesting party] agrees not to disclose any non- public information shared between FDA/CBER and NIH/DPCPSI/OSC whether orally or in writing, in any manner.” This request shall state which internal unit offices and/or individuals are requesting the information.

2. The party receiving the request (sharing party) will determine, based upon the request described in section III.B.1 above, whether it is appropriate and practicable to share the requested non-public information.

3. The requesting party will comply with the following conditions:

a. The requesting party will limit the dissemination of shared non-public information it receives to internal unit offices and/or employees that have been identified in its written request and/or have a need to know. The unit official who signs the request letter shall be responsible for ensuring information is not distributed to inappropriate recipients.

b. The requesting party will agree in writing not to disclose any shared non-public information in any manner not authorized by law or regulation, including disclosure in publications and public meetings, or in the context of other agency collaborations. If the requesting party wishes to disclose shared information that the sharing unit has designated as non-public, the requesting party will ask the sharing party whether the information's non-public status has changed, and if so, will first obtain written confirmation and permission form the sharing party before disclosing that information. If the requesting party receives a Freedom of Information Act (FOIA) request for the shared information, it shall: (a) refer the FOIA request to the information-sharing contact person or designee for the sharing party to respond directly to the FOIA requester regarding the releasability of the information, and (b) notify the FOIA requester of the referral and that a response will issue directly from the sharing party. The requesting party will leave all final disclosure decisions up to the sharing party, including decisions on whether the records are responsive and whether they must be disclosed. According, the requesting will not indicate to the FOIA requester whether the sharing party has responsive records or releasable records.

c. The requesting party will promptly notify the contact person or designee of the sharing party of any attempt by a third party to obtain shared non-public information by compulsory process, including, but not limited to, a FOIA request, subpoena, discovery request, or litigation complaint or motion.

d. The requesting party will notify the sharing party before complying with any judicial order that compels the release of shared non-public information, so that the parties may determine the appropriate measures to take, including, where appropriate, legal action.

4. The sharing party will include a response in writing along with any agency information shared. The response will indicate the type of information (e.g., confidential commercial information, personal privacy, pre-decisional, etc.), and will include the following sharing party template language: “Pursuant to the Memorandum of Understanding for Sharing of Non-Public Information between the FDA/CBER and NIH/DPCPSI/OSC, dated [insert date agreement was signed], the non-public information provided in this communication may not be disclosed or shared in any manner.” Any shared documents containing non-public information should be stamped “Do not disclose or further distribute.”

IV. NAME AND ADDRESS OF PARTICIPATING PARTIES

Food and Drug Administration
Center for Biologics Evaluation and Research
Office of Therapeutic Products
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002
Telephone: 1-800-835-4709 or 240 402-8010

National Institutes of Health
Division of Program Coordination, Planning and Strategic Initiatives
Office of Strategic Coordination
6001 Executive Blvd, Suite 8090
Rockville, MD 20852
Telephone: 301-827-6007

V. LIAISON OFFICERS

Liaison Officers will participate in the management, coordination and oversight of this agreement. The Liaison Officers will constitute a Steering Committee comprised of an equal number of member representatives from the FDA/CBER and the NIH SCGE Program staff. Two Liaison Officers, one designate from each participating agency, will serve as co-chairs of the Committee.

Member appointments shall be authorized by the signatories to this agreement. The Liaison Officer Steering Committee shall meet at least once every six months for the first year of this agreement and then once annually thereafter to review the progress of this agreement, resolve any issues and disputes that may arise and oversee necessary modifications to the agreement.

A. For FDA/CBER

Anna Kwilas, Ph.D. (Co-Chair)
Chief, Gene Therapy Branch 4
Division of Gene Therapy 2
Office of Gene Therapy CMC
Office of Therapeutic Products
Center for Biologics Evaluation & Research
U.S. Food and Drug Administration
10903 New Hampshire Ave, Bldg. 71 Room 6066
Silver Spring, MD 20993
Telephone: 240-402-6000
anna.kwilas@fda.hhs.gov

Denise Gavin, Ph.D.
Director, Office of Gene Therapy CMC
Office of Therapeutic Products
Center for Biologics Evaluation & Research
U.S. Food and Drug Administration
10903 New Hampshire Ave, Bldg. 71 Room 6054
Silver Spring, MD 20993
Telephone: 240-402-8291
denise.gavin@fda.hhs.gov

Sandhya Sanduja, Ph.D.
Chief, Pharmacology/Toxicology Branch 2
Division of Pharmacology/Toxicology 2
Office of Pharmacology/Toxicology
Office of Therapeutic Products
Center for Biologics Evaluation & Research
U.S. Food and Drug Administration
10903 New Hampshire Ave, Bldg. 71 Room 6270
Silver Spring, MD 20993
Telephone: 240-402-7358
sandhya.sanduja@fda.hhs.gov

Rondine Allen, Ph.D.
Biologist, Pharmacology/Toxicology Branch 1
Division of Pharmacology/Toxicology 1
Office of Pharmacology/Toxicology
Office of Therapeutic Products
Center for Biologics Evaluation & Research
U.S. Food and Drug Administration
10903 New Hampshire Ave, Bldg. 71 Room 6118
Silver Spring, MD 20993
Telephone: 240-402-0144
rondine.allen@fda.hhs.gov

B. For NIH/DPCPSI/OSC’s SCGE Program Staff

Chris H. Boshoff, Ph.D. (Co-Chair)
Program Director, Division of Translational Research
National Institute of Neurological Disorders & Stroke
National Institutes of Health
Neuroscience Center 6001 Executive Blvd., Room 5107
Bethesda, MD 20892
Telephone: 301-496-0664
chris.boshoff@nih.gov

Philip J. Brooks, Ph.D.
Deputy Director
Division of Rare Diseases Research Innovation
National Center for Advancing Translational Sciences (NCATS)
National Institutes of Health
6701 Democracy Blvd., Room 206
Bethesda, MD 20892
For courier use (Bethesda, MD 20817)
Telephone: 240-460-8422
pjbrooks@mail.nih.gov

Timothy LaVaute, Ph.D.
Program Director, Division of Neuroscience
National Institute of Neurological Disorders & Stroke
National Institutes of Health
Neuroscience Center, 6001 Executive Blvd., Room 5225
Bethesda, MD 20892
Telephone: 240-472-7590
lavautetm@ninds.nih.gov

Felicia Qashu, PhD
Program Leader
Office of Strategic Coordination
Division of Program Coordination, Planning, and Strategic Initiatives
Office of the Director
National Institutes of Health
6001 Executive Blvd, Suite 8180
Rockville MD 20852
Telephone: 301-451-7222
felicia.qashu@nih.gov

Matthew Arnegard, PhD
Senior Scientific Advisor
Office of Strategic Coordination
Division of Program Coordination, Planning, and Strategic Initiatives
Office of the Director
National Institutes of Health
Telephone: 301-451-3201
matthew.arnegard@nih.gov

VI. PERIOD OF AGREEMENT

This agreement became effective on the date of latest signature, and shall hereinafter continue in effect for an indefinite period of time, as modified herein and pursuant to the attached summary of all modifications, and may be modified by mutual consent of both Parties or terminated by either Party upon a ninety (90) day advance written notice to the other party.
Parties will review the terms of this agreement every five (5) years to consider whether updates are needed or whether the agreement should remain in effect.

APPROVED AND ACCEPTED FOR THE NATIONAL INSTITUTES OF HEALTH
Division of Program Coordination, Planning and Strategic Initiatives, Office of Strategic Coordination

By: /s/
Carolyn Hutter, Ph.D.
Director, Office of Strategic Coordination National Institutes of Health
Date: 11/04/2024

By: /s/
Tara Schwetz, PhD
National Institutes of Health Deputy Director for Program Coordination, Planning, and Strategic Initiatives
Date: 11/04/2024

APPROVED AND ACCEPTED FOR THE FOOD AND DRUG ADMINISTRATION
Center for Biologics Evaluation and Research

By: /s/ 
Julia Tierney, J.D.
Deputy Director
Center for Biologics Evaluation and Research
Food and Drug Administration
Date: 10/23/2024
 

ATTACHMENTS:
Model Request Letter for FDA/CBER
Model Request Letter for NIH/DPCPSI/OSC
Model Transmittal Letter: NIH/DPCPSI/OSC to FDA/CBER
Model Transmittal Letter: FDA/CBER to NIH/DPCPSI/OSC

 

ATTACHMENTS

Model Language for Request from FDA/CBER

The Food and Drug Administration/Center for Biologics Evaluation and Research (FDA/CBER) requests the following information from the National Institutes of Health, (NIH/DPCPSI/OSC) for the following purposes: [Identify information and purpose]

FDA/CBER agrees that it will not disclose any information that NIH/DPCPSI/OSC shares with it and designates non-public without prior written permission from NIH/DPCPSI/OSC and that FDA/CBER will comply with the principles and procedures set forth in the Memorandum of Understanding on information sharing between FDA/CBER and NIH/DPCPSI/OSC dated [Insert date MOU between FDA/CBER and NIH/DPCPSI/OSC initiated]. FDA/CBER acknowledges that applicable laws and regulations may govern the disclosure of such information. See e.g., 21 U.S.C. § 331(j), 18 U.S.C. § 1905, 21 CFR Parts 20 and 21, 45 CFR Parts 5 and 5b, and 42 U.S.C. § 241(d).

FDA/CBER will limit dissemination of any shared information to the following FDA/CBER offices and/or employees, unless it identifies additional FDA/CBER employees who have a need to know the non-public information: [Identify office(s) and/or employee(s)]

Name _
Date _

[Signature and Date by FDA/CBER official with requisite responsibility and authority.]

Model Language for Request from NIH/DPCPSI/OSC

The National Institutes of Health (NIH/DPCPSI/OSC) requests the following information from the Food and Drug Administration/Center for Biologics Research and Review (FDA/CBER) that for the following purposes: [Identify information and purpose]

NIH/DPCPSI/OSC agrees that it will not disclose any information that FDA/CBER shares with it and designates nonpublic without prior written permission from FDA/CBER and that NIH/DPCPSI/OSC will comply with the principles and procedures set forth in the Memorandum of Understanding on information sharing between NIH/DPCPSI/OSC and FDA/CBER dated _.

NIH/DPCPSI/OSC acknowledges that applicable laws and regulations may govern the disclosure of such information. See e.g., 21 U.S.C. § 33l(j), 18 U.S.C. § 1905, 21 CFR Parts 20 and 21, 45 CFR Parts 5 and 5b, and 42 U.S.C. § 241(d).

NIH/DPCPSI/OSC will limit dissemination of any shared information to the following NIH/DPCPSI/OSC offices and/or employees, unless it identifies additional NIH/DPCPSI/OSC employees who have a need to know the non-public information: [Identify office(s) and/or employee(s)]

Name _
Date _

[Signature and Date by NIH/DPCPSI/OSC official with requisite responsibility and authority.]

Model Transmittal letter from NIH/DPCPSI/OSC to FDA/CBER

   This letter accompanies information that the National Institutes of Health (NIH/DPCPSI/OSC) is sharing with the Food and Drug Administration/Center for Biologics Evaluation and Research (FDA/CBER) in response to FDA/CBER's request, dated _. This information contains one or more of the following categories of non-public information, including information the disclosure of which may be prohibited by law:

   [NIH/DPCPSI/OSC checks applicable numbers below]

_ confidential research proposals, progress reports, and/or unpublished data;
_ privileged or pre-decisional agency information;
_ trade secrets;
_ confidential commercial or financial information;
_ information the disclosure of which would constitute a clearly unwarranted invasion of personal privacy;
_ information contained in records subject to the Privacy Act;
_ information contained in the inter-agency or intra-agency memoranda;
_ records or information compiled for law enforcement purposes; or
_ other (explain).

   FDA/CBER shall notify the contact person or designee of NIH/DPCPSI/OSC if there are any attempts to obtain such shared non-public information by compulsory process, including, but not limited to, Freedom of Information Act requests, subpoenas, discovery requests, and litigation complaints or motions.

   FDA/CBER shall notify NIH/DPCPSI/OSC before complying with any judicial order that compels the release of such shared non-public information so that FDA/CBER and/or NIH/DPCPSI/OSC may take appropriate measures, including filing a motion with the court or an appeal.

   By a signed request letter dated _, FDA/CBER has agreed not to disclose the above-described shared non-public information without prior written permission of NIH/DPCPSI/OSC. FDA/CBER has acknowledged that applicable laws and regulations may govern the disclosure of such information. See e.g., 21 U.S.C. § 33l(j), 18 U.S.C. § 1905, 21 CFR Parts 20 and 21, 45 CFR Parts 5 and 5b, and 42 U.S.C. § 241(d).

   FDA/CBER has also agreed to comply with the principles and procedures set forth in the Memorandum of Understanding on information sharing between FDA/CBER and NIH/DPCPSI/OSC, dated _.

Model Transmittal letter from FDA/CBER to NIH/DPCPSI/OSC

   This letter accompanies information that the Food and Drug Administration/Center for Biologics Evaluation and Research (FDA/CBER) is sharing with the National Institutes of Health (NIH/DPCPSI/OSC) in response to NIH/DPCPSI/OSC’s request, dated _.This information contains one or more of the following categories of non-public information, including information the disclosure of which may be prohibited by law:

   [FDA/CBER checks applicable numbers below]

_ trade secrets;
_ confidential commercial or financial information;
_ information the disclosure of which would constitute a clearly unwarranted invasion of personal privacy
_ information contained in records subject to the Privacy Act;
_ information contained in inter-agency or intra-agency memoranda;
_ records or information compiled for law enforcement purposes; or
_ other (explain).

   NIH/DPCPSI/OSC shall notify the contact person or designee of FDA/CBER if there are any attempts to obtain such shared non-public information by compulsory process, including, but not limited to, Freedom of Information Act requests, subpoenas, discovery requests, and litigation complaints or motions.

   NIH/DPCPSI/OSC shall notify FDA/CBER before complying with any judicial order that compels the release of such shared non-public information, so that FDA/CBER and/or NIH/DPCPSI/OSC may take appropriate measures, including filing a motion with the court or an appeal.

By a signed request letter dated _, NIH/DPCPSI/OSC has agreed not to disclose the above-described shared non-public information without prior written permission of FDA/CBER. NIH/DPCPSI/OSC has acknowledged that applicable laws and regulations may govern the disclosure of such information. See e.g., 21 U.S.C. § 331(j), 18 U.S.C. § 1905, 21 CFR Parts 20 and 21, 45 CFR Parts 5 and 5b, and 42 U.S.C. § 241(d). NIH/DPCPSI/OSC has also agreed to comply with the principles and procedures set forth in the Memorandum of Understanding on information between FDA/CBER and NIH/DPCPSI/OSC, dated _.