MOU 225-22-011

MEMORANDUM OF UNDERSTANDING 
BETWEEN
THE NATIONAL INSTITUTES of HEALTH
NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES 
AND
THE FOOD AND DRUG ADMINISTRATION
CENTER FOR DRUG EVALUATION AND RESEARCH 

I. PURPOSE

This Memorandum of Understanding (MOU) provides a framework for collaboration  between the National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS) and the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER), each a “Party” and collectively the "Parties," to engage in collaborative activities that promote discussion within the scientific, medical and regulatory communities regarding rare disease drug and biological product (collectively referred to as “drug”) development. In support of this goal and under the terms of this MOU, the Parties intend to co-sponsor meetings and seminars, such as a    workshop on "Regulatory Fitness in Rare Disease Trials".

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) as amended (21 U.S.C. § 301, et seq.). 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, 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, CDER's mission is to ensure that safe and effective drugs are available to improve the health of the people in the United States. FDA has the authority to enter into this MOU pursuant to section 1003 of the Federal Food, Drug, and Cosmetic Act (21 USC § 393).

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.  NIH has the authority to enter into this MOU pursuant to Section 479 of the Public Health Service (PHS) Act (42 U.S.C. § 287).

Within NIH, NCATS houses the Office of Rare Diseases Research, whose mission is to accelerate the development of more therapies for rare diseases and get them to more patients more quickly. The development of translational science in this area may accelerate progress across the entire rare disease drug development field and stands to benefit rare disease patients and investigators.  

NCATS Office of Rare Diseases Research’s mission aligns with CDER’s Division of Rare Diseases and Medical Genetics, which provides regulation and oversight of a broad array of rare disease products intended for the treatment of Inborn Errors of Metabolism and other genetic or acquired disorders and houses the Rare Diseases Team which focuses on rare disease policy and educational, research, and stakeholder outreach functions.  

Thus, the collaboration envisioned by this MOU, including co-sponsorship of meetings, is consistent with the primary public health missions of both agencies, and provides a forum for bi-agency input and support for finding solutions for rare disease drug development and regulatory considerations.

There are many challenges in rare disease drug development that are broadly recognized by the rare disease drug development community.  Due to the large number of different rare diseases (approximately 7,000-10,000), small numbers of patients with the individual disorders, lack of drug development precedent for most rare diseases leading to a lack of known or accepted biomarkers and clinical outcomes measures for use in clinical trials, and because many rare diseases affect children as the primary patient population, among other considerations, designing and conducting good quality clinical trials able to support regulatory submissions (i.e., INDs) and eventually, marketing applications (i.e., NDAs, BLAs) can be difficult.  There are also rapid advances in science, particularly in the field of genetics, for rare diseases resulting in an active, highly innovative and rapidly evolving area of science and drug development.  Rare diseases represent an area of high unmet medical need.  Additionally, academic investigators, patient groups, and small or emerging pharmaceutical and biotechnology companies, who generally lack regulatory experience, are often the sponsors for rare disease drug development applications.  

Given these considerations, regulatory and rare disease drug development guidelines and advice have been identified by NCATS and CDER as areas where a meeting and public discussion would likely result in benefit to the rare disease drug development community. Such meetings will provide examples of successful drug development programs, share experience and expertise from multi-disciplinary stakeholders, discuss regulatory considerations for human subject research, and explore areas of drug development that would benefit from additional public discourse, such as endpoint identification and clinical trial design, among others. Training and discussion through a meeting in these research areas should improve the field of rare disease drug development and result in improved familiarity and understanding of Federal regulations, guidelines, and approaches for good quality rare disease clinical trials. 

III.  RESPONSIBILITIES OF EACH PARTY

The parties agree to establish a Planning Committee to develop and operationalize workshops, seminars, and other events pursuant to the goals set forth in this MOU. The Planning Committee would be responsible for planning and implementing all strategic, operational, and logistical tasks associated with such events. In addition:   

•  Each Party will appoint one or more representatives to the Planning Committee. Representatives are expected to accurately represent their organization’s position and report back to their respective organizations on any proposals put forth by the Committee. Differences of opinion on strategic or policy direction should be referred out of the Planning Committee to the respective organizational management of the Parties.   
• Each Party agrees that the use of their agency’s logos can be used to promote the event to the public.
• Both Parties agree to coordinate publicity and promotion of planned events in furtherance of the goals established by this MOU.  
• Each Party may utilize the expertise and relationships of the other in order to maximize the benefits of this collaboration. 

IV.    GENERAL PROVISIONS

1.  Effective Date. The collaborative activities described herein will be implemented under terms agreed to by the Parties.  This MOU shall become effective upon the signature of all Parties and will continue in effect unless modified by mutual consent or terminated by either Party upon a 30-day advance notice to the other Party.
2.  Information exchange. All information exchanged under this MOU will be publicly available information. 
3.  Resource Obligations. This MOU represents the broad outline of the Parties intent to enter into collaborative efforts in intellectual areas of mutual interest. All activities undertaken pursuant to the MOU are subject to the availability of personnel and resources. This MOU does not affect or supersede any existing or future agreements or arrangements among the Parties. This MOU does not create binding, enforceable obligations against either NIH or FDA. This MOU and all associated agreements will be subject to the applicable policies, rules, regulations, and statutes under which FDA and NIH operate.
4.  Governing Law. This MOU shall be governed by U.S. Federal Law as applied in the Federal Courts of the District of Columbia.
5.  Notices and Meetings. All notices pertaining to or required by this MOU will be in writing, signed by an authorized representative of the notifying Party, and delivered by registered or certified mail, email or facsimile, or express/overnight delivery service and sent to the other Party at the designated address in Paragraph 6.
6.  Points of Contact. The following individuals are designated points of contact for the MOU: 

   The names of NCATS and CDER staff listed below represent the current persons in these assigned roles at the date of signing of this MOU. Additional NIH and FDA staff may be drawn to provide scientific expertise as needed.

       Scientific Contact for NCATS: 

       Philip John (P.J.) Brooks, PhD
       Acting Director
       Office of Rare Diseases Research
       National Center for Advancing Translational Sciences (NCATS)
       National Institutes of Health (NIH)
       6701 Democracy Boulevard, Suite 206, MSC 4874f
       Bethesda, MD 20892 (for mailings)
       Bethesda, MD 20817 (for deliveries/GPS)
       pj.brooks@nih.gov
       301-443-0513

       Science Contact for CDER: 

       Kerry Jo Lee, MD
       Associate Director for Rare Diseases
       Division of Rare Diseases and Medical Genetics (DRDMG) 
       Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine 
       (ORPURM) 
       Office of New Drugs (OND)
       Center for Drug Evaluation and Research (CDER)
       Food and Drug Administration
       10903 New Hampshire Ave Silver Spring, MD 
       20993-0002 
       240-402-4290
       Kerry.Lee@fda.hhs.gov

7.  Press Releases. Each Party agrees to provide proposed press releases that reference or rely upon the work under this MOU to the other Party for review and comment at least seven (7) days prior to publication.
8.  Miscellaneous Provisions. This MOU in no way restricts either Party from participating in any activity with other Public or Private agencies, organizations or individuals, as authorized by law.
   
   This MOU is strictly for internal management purposes for each of the Parties. This MOU shall not be construed to provide a private right or cause of action for or by any person or entity.

APPROVED AND ACCEPTED FOR            
THE NATIONAL CENTER FOR ADVANCING    
TRANSLATIONAL SCIENCES, NIH            

/s/    
Joni L. Rutter, PhD                    
Acting Director                   
Date: 03/07/2022                                  

APPROVED AND ACCEPTED FOR
CENTER FOR DRUG EVALUATION AND RESEARCH, FDA

/s/
Patrizia Cavazzoni, MD
Director
Date: 02/24/2022