FDA pilot programme to boost rare disease therapies

FDA headquarters

The US Food and Drug Administration (FDA) has announced a pilot programme with the aim of accelerating the development of novel drug and biological products for rare diseases.

The agency is inviting a limited number of sponsors to participate in the programme, which allows for more frequent communication with FDA staff to address 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, Director of the FDA’s Center for Biologics Evaluation and Research. “These are complex products and we recognise the importance of sponsor communication with the FDA to facilitate development of products for patients with unmet medical needs.”

Participants in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot programme will be able to obtain frequent advice and regular ad-hoc communication with FDA staff to address product development issues.

The programme 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).

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.

The FDA will be accepting applications to the START programme between 2 January 2024 and 1 March 2024.

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