DIA 2023: FDA Rare Disease Town Hall


CDER and CBER leaders update initiatives in the rare disease space within the past year.

In the FDA Rare Disease Town Hall at DIA 2023, Chair James Valentine, Director for Hyman, Phelps & McNamara, PC, laid the foundation for FDA rare disease therapeutic approvals and then introduced the FDA panelists for the session-- Celia M. Witten, MD, PhD, Deputy Director, Office of the Center Director, CBER, FDA, and Janet Maynard, MD, MHS, Director, Office of Rare Diseases, Pediatrics, Urologic & Reproductive Medicine.

Maynard began stating that of the 37 NME approvals for 2022, 20 were rare diseases. She updated the audience on the CDER-wide effort to connect the new drugs office, office of the Center Director and translational science. That is the Accelerating Rare disease Cures (ARC) Program, which is now a year old. Maynard encouraged the audience to sign-up for its CDER Rare Disease enewsletter, which is now up to 10K subscribers.

Three notable initiatives in the rare disease space are:

Accelerating Access to Critical Therapies for ALS Act – ACT for ALS. This is a public-private partnership that requires, according to the website, “FDA to publish and implement a five-year action plan to foster drug development and facilitate access to investigational drugs for ALS and other rare neurodegenerative diseases.”

RDEA Rare Disease Endpoint Advancement (RDEA) Pilot Program. As part of PDUFA VII, the joint CBER, CDER pilot will develop novel endpoint efficacy development for drugs that treat rare diseases. Maynard encouraged sponsors to apply to the pilot, which will allow them to collaborate with the FDA in this effort.

The Learning and Education to Advance and Empower Rare Disease Drug Developers or LEADER 3D, is a collaboration of stakeholders that will seek to understand challenges in developing these drugs, including the design of clinical trials, and where knowledge gaps. exist. The goal is to then create or expand educational resources for stakeholders.

Moving to CBER, Witten noted the general trend is cell and gene therapy development, away from the plasma-derived products, which were the primary therapeutics reviewed by CBER in prior years.

She said that the agency and industry has growing experience in CAR-T cells, and “although quite novel, the length of time for clinical development has decreased over the years because the [scientific] community has learned.” While the science has increased, she did note that the CMC processes have yet to catch up.

To address these issues, the CMC Development Readiness Pilot (CDRP) has been launched to keep pace with clinical development. CBER will select nine proposals from sponsors to work with in this pilot with CBER.

Witten said that a meeting of the Clinical Trials Patient Experience and Patient Engagement and Regenerative Medicine meeting was held in April 2023 and was very interesting. The FDA will issue a transcript of the recording soon.

REFERENCE: FDA Rare Disease Town Hall, DIA Boston, June 28, 2023.

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