FDA signals openness to faster drug approval pathways and greater transparency

The U.S. Food and Drug Administration held a CEO listening forum asking pharmaceutical and biotech companies how the FDA could work better and faster, while still maintaining standards and safety.

In the first of five planned CEO listening forums, approximately 60–70 executives from both large and small pharmaceutical companies in the DC/NY region attended. Each attendee had the opportunity to share one or two suggestions for how the FDA could improve. A summary is shown below of the key themes discussed:

Communication:

• There is a strong need to improve communication between FDA reviewers and companies.
• A brief 5-minute call could prevent months of back-and-forth via formal letters.
• Companies deserve a timely responses to their inquiries.
• Commissioner Makary noted: “Increased communication could help innovation".

Standards:

• Current Chemistry, Manufacturing, and Controls (CMC) standards are often burdensome, expensive and time-consuming. There’s a need to strike a balance between ensuring high-quality standards and reducing bureaucracy.
• Applications can span hundreds of thousands of pages. There is a need for balance between information that is submitted and information that can be available on request.
• There was support for the FDA’s use of AI to assist with scientific reviews.
• Regarding clinical endpoints, the FDA stated it will continue to show flexibility based on factors such as unmet need, population size, and validated efficacy measures (including surrogate endpoints). The recent approval of a CRISPR-based treatment for Baby KJ was cited as an example.
• The FDA confirmed it will continue to accept validated surrogate endpoints.
• There was a request about changing the requirement from two clinical trials to one; the FDA is thinking about this.
• The FDA will not change priority review and accelerated pathways currently in place and will look to build on these.

Transparency

• Predictability is important for companies and innovation.
• The FDA wants companies to know what they are thinking.
• There was a call for the FDA to make use of data from drugs that were never brought to market, in order to inform future innovation.

‍

Source: FDA Direct Video