Rare Diseases Team

Mission Statement

To facilitate, support and accelerate the development of drug and biologic products for the benefit of patients with rare disorders.

Overview

• Coordinate the development of CDER policy, procedures and training for the review of treatments for rare diseases.
• Assist in outside development and maintenance of good science as the basis for the development of treatments for rare diseases.
• Work collaboratively with external and internal rare disease stakeholders to promote the development of treatments for rare disorders.
• Maintain collaborative relationships with CDER's review divisions to promote consistency and innovation in the review of treatments for rare disorders.
• Work collaboratively with international regulatory agencies to discuss and exchange scientific and regulatory information related to rare diseases.

CDER’s Rare Diseases Team PDUFA VI and VII Commitments

• Advancing Development of Drugs for Rare Diseases

Selected Conferences

• FDA & Duke-Margolis Endpoint Considerations to Facilitate Drug Development for NPC Workshop – January 24-25, 2022
• FDA CDER & NIH NCATS Regulatory Fitness in Rare Disease Clinical Trials Workshop – May 16-17, 2022
• FDA & Duke-Margolis Webinar on Endpoint Considerations to Facilitate Drug Development for NPC: Key Themes and Future Directions from the January 2022 Public Workshop – August 4, 2022
• FDA CDER & JHU CERSI Workshop | Addressing Challenges in the Design and Analysis of Rare Disease Clinical Trials: Considerations and Tools –  May 2 - 3, 2023

Rare Disease Resources

1. Definition of Rare Disease and Orphan Drug

a. What is a rare disease?

The Orphan Drug Act defines a rare disease as a disease or condition that affects less than 200,000 people in the United States.

b. What is the Orphan Drug Act?

The Orphan Drug Act is a law passed by Congress in 1983 that incentivizes the development of drugs to treat rare diseases. Companies and other drug developers can request orphan drug designation and FDA will grant such designation if the drug meets specific criteria. Orphan designation qualifies sponsors for various incentives, including:

• Tax credits for qualified clinical (in humans) testing
• Waiver of the Prescription Drug User Fee (currently at almost $3 million for a new drug)
• Potential 7 years of market exclusivity after approval

In addition, the Orphan Drug Act established the Orphan Product Grants Program to provide funding for developing products for rare diseases or conditions.

c. What is an orphan drug?

An orphan drug is a drug for a rare disease or condition. Some rare disease treatments have been “orphaned” or discontinued because there was not enough financial incentive to continue development or production. The Orphan Drug Act incentivizes drug development for rare diseases.

2. Selected FDA Programs and Initiatives to Facilitate Rare Disease Drug Development

1. CDER's Accelerating Rare disease Cures (ARC) Program
2. CDER Patient-Focused Drug Development
   1. Guidance Series to Enhance the Incorporation of the Patient's Voice in Medical Product Development and Regulatory Decision Making
   2. Patient-Focused Drug Development Meetings: FDA-led Patient-Focused Drug Development Public Meetings and Externally-led Patient-Focused Drug Development Meetings
3. Patient Listening Sessions
4. Orphan Drug Designation
5. Orphan Products Grants Program
6. Rare Pediatric Disease Designation and Voucher Programs
        i.  Rare Pediatric Disease Priority Review Vouchers Draft Guidance for Industry

3. Selected Ways that Patients Can be Involved in FDA’s Work on Rare Diseases

1. How can patients become involved in FDA's work on rare diseases?
2. How can patients participate in the development of Patient-Focused Drug Development meetings?
3. Where can patients get information about previously conducted Patient-Focused Drug Development meetings and Patient Listening Sessions?
4. Who can patients contact in CDER regarding stakeholder engagement, drug development, drug review, and drug safety questions?
5. Where can patients learn more about FDA patient engagement?

Please check back for more anticipated updates.