FDA launches program for rare diseases targeting ‘significant’ unmet need

The US Food and Drug Administration (FDA) has launched a program called Rare Disease Evidence Principles (RDEP) to expedite the approval of treatments for rare diseases that meet a significant unmet medical need and target a known genetic defect.

FDA said that through the program, sponsors will receive “clearer guidance on the types of evidence that can be used to demonstrate substantial evidence of effectiveness.”

The program will be implemented jointly by FDA’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).

To participate, products must address a specific known genetic defect related to a very small and rare disease population of fewer than 1,000 individuals, and the disease is characterized by a progressive decline in function, which can lead to significant disability or death within a relatively short timeframe. Additionally, there must not be any sufficient alternative treatments available.

“Drug developers – and the patients they hope to treat – deserve clear, consistent information from the FDA,” announced FDA Commissioner Marty Makary. “These principles ensure that FDA and sponsors are aligned on a flexible, common-sense approach within our existing authorities, and that we incorporate confirmatory evidence to give sponsors a clear, rigorous path to bring safe and effective treatments to those who need them most.”

FDA noted that drug development for rare diseases faces unique challenges not encountered with larger disease populations. The smaller the population or subpopulations involved in studying a rare disease, the more difficult it becomes to generate adequate evidence to fulfill statutory requirements for demonstrating efficacy using traditional trial designs, such as placebo-controlled studies, and the number of clinical studies needed.

Sponsors may apply for the RDEP process prior to the launch of a pivotal trial, and requests can be submitted as part of a formal meeting request.

FDA may approve the product based on a single adequate and well-controlled study, along with “strong” confirmatory evidence. This evidence may include the drug's treatment effects on the disease's direct pathophysiology, data from relevant non-clinical models, clinically significant pharmacodynamic data, or other clinical information such as case reports and data from expanded access programs.

Sponsors selected to participate will have an initial meeting with the appropriate FDA review team to determine what data will be used to substantiate safety and effectiveness.

Before applying, sponsors developing drugs for rare cancers should consult with the Oncology Center of Excellence to determine if the RDEP process applies.

FDA emphasizes that the RDEP program is distinct from its orphan drug designation program. A drug reviewed under the RDEP process does not automatically qualify for orphan drug designation, and participation in the RDEP has no impact on whether a drug may be eligible for that designation. A sponsor seeking orphan drug designation for their drug must adhere to the procedures outlined in section 526 of the federal Food, Drug and Cosmetic Act and 21 CFR Part 316.

This program builds on and complements other FDA initiatives to expedite treatment for rare diseases, including the Accelerating Rare Disease Cures Program in 2022 and the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program launched in 2023. (RELATED: Convergence: CBER chief discusses priorities, touts gene therapy approvals, Regulatory Focus 5 October 2023)

It also complements the agency’s rare disease endpoint advancement pilot program (RDEA). (RELATED: FDA officials say high priority will be given to applications with endpoints that can be leveraged for other rare diseases, Regulatory Focus 9 June 2023)

Announcement

FDA launches program for rare diseases targeting ‘significant’ unmet need

Related topics

Recommended content

Welcome to the new RAPS Digital Experience