FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies

FDA News Release

• For Immediate Release: September 29, 2023

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Today, the U.S. Food and Drug Administration is taking steps to help further accelerate the development of novel drug and biological products for rare diseases. The agency is announcing the opportunity for a limited number of sponsors to participate in a pilot program allowing for more frequent communication with FDA staff to provide a mechanism for addressing clinical development issues. 

“We hope the insight gained from this pilot will provide information on how best to facilitate more efficient development of potentially life-saving therapies with rare disease indications and help sponsors generate high-quality, compelling data to support a future marketing application,” said Peter Marks, M.D., Ph.D, director of the FDA’s Center for Biologics Evaluation and Research. “These are complex products and we recognize the importance of sponsor communication with the FDA to facilitate development of products for patients with unmet medical needs.”

Selected participants of the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program will be able to obtain frequent advice and regular ad-hoc communication with FDA staff to address product-specific development issues, including, but not limited to, clinical study design, choice of control group and fine-tuning the choice of patient population. 

The program will be open to sponsors of products currently in clinical trials under an active Investigational New Drug application (IND), regulated by the Center for Biologics Evaluation and Research (CBER) and/or the Center for Drug Evaluation and Research (CDER). Eligibility criteria for the pilot differs between CBER and CDER-regulated products. 

In addition to having an active IND, eligible CBER-regulated products must be a gene or cellular therapy intended to address an unmet medical need as a treatment for a rare disease or serious condition, which is likely to lead to significant disability or death within the first decade of life. Under CDER’s eligibility criteria, the product must be intended to treat rare neurodegenerative conditions, including those of rare genetic metabolic type. More information on the program’s eligibility requirements can be found in the Federal Register Notice.  

"We look forward to increased engagement with sponsors developing these important products for the rare disease community," said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research. "We share the goal of delivering potentially life-saving products to patients, and are committed to helping sponsors achieve regulatory milestones, while ensuring the safety, effectiveness and quality of these products."

The FDA will be accepting applications to the START program between Jan. 2, 2024, and March 1, 2024. Pilot participants will be selected based on application readiness (e.g., sponsors who demonstrate having the ability to move their development program forward towards a marketing application). The agency will select up to three participants for each center. Following an evaluation of this pilot and feedback from selected sponsors, the agency may consider a second iteration, which would be announced in the Federal Register at a later date.

Sponsor participation in the pilot will be considered concluded when the development program has reached a significant regulatory milestone, such as initiation of the pivotal clinical study stage or reaching the stage prior to submitting a marketing application (pre-Biologics License Application or pre-New Drug Application meeting stage), as agreed upon with the sponsor.

The FDA has undertaken additional efforts to further enhance and expedite the availability of therapies intended to treat rare diseases. The agency recently published a Request for Information for stakeholders to provide feedback regarding critical scientific challenges and opportunities to advance the development of cellular and gene therapies designed for an individual or very small number of patients diagnosed with a rare disease. The agency plans to use this information to inform the planning of possible future meetings, workshops, educational programs or discussion papers to ultimately facilitate the development of additional regulatory tools and/or framework.

The agency additionally published a docket for stakeholder feedback as part of the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) program under the CDER Accelerating Rare disease Cures (ARC) program. Feedback will be used for the identification of knowledge gaps in rare disease drug development and the development of publicly available resources to inform stakeholders who design and conduct rare disease drug development programs on regulatory considerations surrounding clinical trial design. 

The agency will continue to enhance its evidence-based regulatory framework and recommendations for sponsors of rare disease products, as demonstrated by its active guidance pipeline. The FDA recently published the draft guidance, Demonstrating Substantial Evidence of Effectiveness Based on One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence, and in the coming months, plans to publish a number of cell and gene therapy guidance documents, further exemplifying the agency’s commitment to help deliver important products to patients in need.