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Edward NarkeEdward Narke is regulatory managing director and
cofounder, Design Space InPharmatics. Connect with Ed on linkedin at http://www.linkedin.com/in/edwardnarke.

The FDA’s Breakthrough Therapy Program was created by the Food and Drug Administration Safety and Innovation Act (“FDASIA”) that was signed into law, on July 9, 2012. This bill expanded many of the existing governing powers of the FDA but also added one intriguing new provision: the breakthrough therapy (BT) designation, intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. FDA’s guidance was highly anticipated, and offers the agency’s interpretation of the breakthrough therapy designation program.

To determine whether the improvement over available therapy is “substantial,” FDA weights the magnitude of the treatment effect and the importance of the observed clinical outcome. Preliminary clinical evidence should show a clear advantage over available therapy.

FDA is stressing that the ability to meet the expedited review goals of the BT pathway will hinge on the sponsor intensifying its upfront chemistry, manufacturing, and control (CMC) coordination and communication internally; with outside contractors involved with drug substance and product manufacturing, testing, and packaging; and with the agency. The new 33-page guidance explains how the BT pathway fits in with and combines elements of three other programs—fast track, accelerated approval, and priority review—that FDA already had in place to facilitate and expedite the development and review of new drugs and biologics that target unmet medical needs.

The February issue of the AAPS Newsmagazine reviews some challenges and opportunities for commercial manufacturing readiness in BT programs and the impact of accelerated development for these kinds of products on CMC and good manufacturing practices issues that need to be considered in developing both large- and small-molecule drugs. Read What It Means to Be “Breakthrough”: Expediting Drug Development—The New FDA Breakthrough Therapy Designation, from the Regulatory Sciences section of AAPS. Then participate in the discussion question below.

If a drug is designated a breakthrough therapy, will the timeline for bringing a revolutionary drug to market be reduced?