Draft Guidance on Expedited Programs demonstrates FDA has broad regulatory flexibility to establish the safety and efficacy of innovative medicines to address unmet medical needs

Enhanced Communication Encouraged to Optimize Expedited Drug Development and Review

09.09.13 | By Stephanie Fischer

PhRMA has long supported the FDA’s appropriate use of innovative approaches and regulatory flexibility to establish the safety and efficacy of innovative medicines to address unmet medical needs.  We strongly supported the passage last year of the Food and Drug Administration Safety and Innovation Act (FDASIA) which enhanced the authority of FDA to consider appropriate scientific data, methods, and tools, and to expedite development and access to novel treatments for patients with a broad range of serious or life-threatening diseases or conditions. 

The Draft Guidance for Industry on Expedited Programs for Serious Conditions is a critical, single source tool for communicating to industry the processes and procedures for fast track designation, breakthrough therapy designation, accelerated approval and priority review designation.  If you’re new to the topic, you may want to check out the chart on pages 11-12 which provides a helpful overview of the criteria, timeline and features of each of the four programs.

The Draft Guidance demonstrates that the current system provides FDA with broad regulatory flexibility to establish the safety and efficacy of innovative medicines to address unmet medical needs (as noted in previous Catalyst posts on antibacterials). 

In our comments on the Draft Guidance, we provide suggestions to make the guidance even more helpful to the biopharmaceutical companies working on innovative medicines to address patients’ unmet medical needs.   For example, we recommend that FDA fully utilize enhanced communication as a tool to truly facilitate expedited development. Frequent and consistent interaction between FDA and sponsors is important for optimized drug development and timely review.

We also encourage the FDA to look at ways to apply existing regulatory flexibility to help accelerate the development of therapies for rare diseases.  In the last ten years, the number of medicines in development with an orphan drug designation averaged 140 per year compared to just 64 in the previous decade. While this is good news, we need to continually look for ways to encourage and accelerate the development of new treatments for rare disease, many of which currently have no treatment options. According to Global Genes, of the approximately 7000 rare diseases that have been identified, 95% do not have a single approved treatment.

We look forward to continuing to work with FDA, patients and other stakeholders to achieve the goal we all share - getting safe and effective medicines to patients with serious conditions more quickly.

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