FDA Issues Guidance on Expedited Programs for Serious Conditions

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FDA finalizes guidance on expedited programs for new drug approvals for treatment of serious and life-threatening conditions.

FDA has finalized guidance on expedited programs for drugs and biologics to treat serious conditions.  The guidance, released May 29, 2014, details four programs that the agency has designed to facilitate and expedite development and review of new drugs for the treatment of life-threatening conditions. FDA states that the guidance provides information on the four programs, presents criteria for determining if a drug is a candidate for the programs, and is “intended to help ensure that therapies for serious conditions are approved and available to patients as soon as it can be concluded that the therapies’ benefits justify the risks.”

The four programs are Fast-Track Designation, Breakthrough Therapy Designation, Accelerated Approval, and Priority Review Designation.

  • Fast-Track Designation provides frequent discussions about a drug’s development program with FDA to ensure the collection of appropriate data to support approval.

  • Accelerated Approval bases approval on an agreed surrogate marker instead of a clinical endpoint.

  • Priority Review Designation reduces the drug application review time from 10 months to 6 months.

  • Breakthrough Therapy Designation provides fast-track designation as well as FDA staff involvement in the drug development and review process.

In an FDA Voice blog post, Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, details the programs and says that while progress has been made to ensure critical therapies reach patients, “many scientific discoveries still need to be translated into treatments, while patients are urgently waiting for new life-saving therapies.”

According to Woodcock, these expedited drug approval programs have impacted new drug approvals and were used to expedite novel drugs in 2013 with review times as short as 4.5 months. The Breakthrough Therapy Designation has resulted in 48 out of 186 designation requests being granted as of May 5, 2014. And, Woodcock states that 80 new products have been approved through the Accelerated Approval pathway since 1992, resulting in innovative cancer and HIV treatments.

“The Food and Drug Administration (FDA) is committed to doing our part to help bridge this gap. In this context, we have been actively scrutinizing, strengthening and streamlining our regulatory processes at various steps along the path from drug discovery to delivery—including the clinical development phase, the longest and most expensive period of drug development. As part of this effort, we have developed and successfully used a number of flexible and innovative approaches to expedite the development and review of drugs—to the benefit of millions of American patients,” says Woodcock in the blog post.

Source: FDA.gov