Six Strategies For Accelerating Approval For Cell And Gene Therapies

Cell and gene therapies are experiencing important breakthroughs, demonstrating potential to slow disease progression, improve outcomes and, in some cases, potentially cure an array of illnesses. However, developers of these cutting-edge therapies are encountering new challenges as they navigate complex clinical and regulatory hurdles, particularly when they’re seeking accelerated approval.

For traditional pharmaceutical products, the FDA’s review process centers around clinical efficacy and safety rather than product manufacturing and delivery. However, for cell and gene therapies where manufacturing is more complex, and for autologous cell therapies, where “the product is the process,” quality aspects related to the manufacturing process drive the development program. Consequently, the FDA review focuses primarily on the therapy’s critical quality attributes and controlling these earlier in development, typically prior clinical studies. Data on potency, product manufacture, how it’s delivered and how it’s administered to the patient are required earlier in development.

As you advance through the continuously evolving regulatory landscape for cell and gene therapies, review these six proven strategies that can help sponsors reduce their risk of failure and expedite speed to market.