FDA’s Expedited Development Programs for Compassionate Use

FDA has four programs that are intended to facilitate and expedite development and review of new drugs and biologics that address unmet medical needs in the treatment of serious or life-threatening conditions. Serious conditions include diseases or conditions associated with morbidity that has a substantial impact on day-to-day functioning. The programs are described in FDA’s draft Guidance to Industry: Expedited Programs for serious Conditions – Drugs and Biologics, issued in June 2013. They include Breakthrough Therapy designation, Fast Track designation, Accelerated Approval, and Priority Review.

Breakthrough Therapy Designation: Breakthrough Therapy designation is intended to expedite the development and review of products that treat a serious or life-threatening condition and where preliminary clinical evidence indicates that the product may demonstrate substantial improvement in a clinically significant endpoint(s), over available therapies.

For Breakthrough Therapies, FDA intends to engage senior managers and experienced review staff in a proactive, collaborative, cross-disciplinary review. The sponsor will receive more intensive and timely guidance, will be able to engage in more frequent development meetings with FDA and will be able to make a rolling NDA/BLA submission. The sponsor must request specific designation for each indication desired.

Fast Track Designation: Fast Track designation is intended to expedite the development and review of products that treat a serious condition and where nonclinical or clinical data demonstrates the potential to address an unmet medical need (or the product is a qualified infectious disease candidate). A sponsor that receives fast track designation will be eligible for more frequent interactions with FDA during the development cycle and the product will be eligible for a rolling NDA/BLA review. The sponsor must request specific designation for each desired indication.

Accelerated Approval: Accelerated Approval is intended to expedite the development and approval of promising therapies that treat a serious condition and provide meaningful therapeutic benefit over available therapies. Accelerated Approval allows approval of a product based upon a reasonably predictive “surrogate endpoint” rather than on irreversible morbidity or mortality. After the product enters the market, the sponsor may be required to conduct post-marketing trials to verify the product’s clinical benefit with long-term studies. If the post-marketing trials fail to verify the predicted clinical benefit, FDA may withdraw approval. A product that has received a Breakthrough Therapy or a Fast Track designation can be eligible for Accelerated Approval, if the relevant criteria are met.

Priority Review: Priority Review shortens the FDA’s PDUFA review goal date to six months from the standard review timeline of 10 months. It is reserved for products that are intended to treat a serious condition and, if approved, would provide a significant improvement in safety or efficacy. This review designation is determined at the time of a BLA, NDA, or efficacy supplement submission. Any product, including those that have received Breakthrough Therapy designation, Fast Track designation, or being evaluated for Accelerated Approval can be granted Priority Review, if the relevant criteria are met.

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