Health Policy Briefs -Breakthrough Therapy Designation
The FDA has new authority to expedite the approval process for drugs intended to treat serious or life-threatening conditions.
What’s the issue?
The Food and Drug Administration Safety and Innovation Act (FDASIA), signed into law on July 9, 2012, introduced several reforms that are intended to streamline the FDA’s premarketing approval process for drugs and devices. Among these reforms was the creation of a new expedited development pathway for drugs that are intended to treat serious or life-threatening conditions. Under this pathway, a drug candidate may be designated by the FDA as a “breakthrough therapy” if early clinical evidence indicates that the drug may demonstrate a substantial improvement over existing therapies. Once a drug has been granted this status, the FDA commits to working particularly closely with the drug’s sponsor to create an efficient development plan and facilitate its approval.
The FDA released draft guidance on this new pathway in June 2013, and the pharmaceutical industry has responded enthusiastically to the opportunity for a drug to be designated a breakthrough therapy. As of April 2014 the agency had received 178 requests for breakthrough therapy designation. Of those requests, forty-four have been granted, and six drugs have been subsequently approved for use. It will likely be several years before the full impact of this designation is clear, and the degree of impact will ultimately depend on the successful implementation of other reforms currently under way, such as those aimed at expediting the development and review of co-diagnostics. However, it carries significant implications for approaches to clinical development, advancement of regulatory science, patient access to new drugs, and public health.
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