Today from Revio we’re happy to announce a new pilot program developed by the U.S. Food and Drug Administration (FDA) 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.

This new program is denominated “Support for clinical Trials Advancing Rare disease Therapeutics (START)”, selected participant of the 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.

Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research states that, “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.”

Pilot Program

• The FDA will be accepting applications to the START (Support for clinical Trials Advancing Rare Disease Therapeutics) program between January 2, 2024, and March 1, 2024.
• Program will be open to sponsors of products currently in clinical trials under an active Investigational New Drug application (IND).
• 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 a maximum of three participants for each center: Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER).
• 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 condition, including those of rare genetic metabolic type.
• After assessing the outcomes of this initial phase and gathering input from the chosen 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.

Additional efforts on the treatment of rare disease

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.
• Additionally, FDA has 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.
• FDA has also 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.

The official FDA website contains more information if you want to know more or to consult the complete updated information related to the Pilot Program launched to Help Further Accelerate Development of Rare Disease Therapies, you can check it by clicking here!

We have launched an specialised course on development of Advanced Therapies, where we talk about CMC Challenges including Comparability, feel free to consult it here! If you are interested.

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