FDA seeks feedback on new framework for rare disease drug development

The US Food and Drug Administration (FDA) plans to create a new framework focused on innovating rare disease drug development.

Building on earlier draft guidance intended to aid approval of treatments for ultra-rare diseases, a new white paper by the agency sets out an optimised framework that considers the personalisation requirements of drugs for the small patient populations inherent in rare diseases. This includes specific patient needs and product type.

FDA highlighted they have only approved treatments in the US for approximately five percent of rare conditions.

As such, the organisation is seeking industry feedback to support development of the new framework, which may include potential new or revised guidance, revisions to regulations and internal processes and resources, including staff training.

“Under President Trump, FDA is rethinking rare disease drug development by putting patients at the centre and matching regulatory requirements to the realities of small patient populations”

HHS Secretary Robert F. Kennedy, Jr

HHS Secretary Robert F. Kennedy, Jr. said: “Americans with rare diseases cannot afford a regulatory system that treats every condition the same. Under President Trump, FDA is rethinking rare disease drug development by putting patients at the centre and matching regulatory requirements to the realities of small patient populations.

“We will remove unnecessary barriers, embrace cutting-edge science, and accelerate promising treatments while upholding FDA’s rigorous standards.”

The FDA aims that the framework will:

• Provide greater clarity, consistency, and predictability in regulatory decision-making through tailored guidance and transparent communication
• Embedd the patient’s voice both early, and at clearly defined milestones, in the drug development and review process
• Further leveraging external expertise to support customisation of regulatory review
• Build shared resources, robust training, and institutional knowledge on rare disease drug development and review
• Apply risk-based approaches, particularly to chemistry, manufacturing, and controls review and pre-approval inspections
• Champion the use of cutting-edge science, novel methods and innovative statistical approaches in rare disease drug development.

The Hub’s Director, Amy Comstock Rick, J.D. added: “Patients with rare diseases and their families deserve a regulatory process that addresses the uniqueness of each of their conditions while maintaining the FDA’s rigorous standards.

“A bold vision to reimagine the framework for rare disease drug development that builds on key learnings and milestones is necessary to meet this moment. This new framework will enhance consistency and predictability for both sponsors and for patients.”

In May, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) also proposed a novel framework for rare disease therapy development.