FDA Issues Final Guidance on Expedited Drug Approvals

Four programs speed innovative treatments to market

Published: Wednesday, June 11, 2014 - 13:23

In recent years, there have been important advances to ensure that therapies for serious conditions are approved and available to patients as soon as sufficient data can show that the therapies’ benefits outweigh their risks. Despite the progress, there is much more work to be done. Many scientific discoveries still need to be translated into treatments while patients are urgently waiting for new life-saving therapies.

In this context, the Food and Drug Administration (FDA) has 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. The vast majority of the time, the United States is the world’s first country to approve novel medicines. Just last year, the FDA approved three-quarters of the new drugs available before any other country did so.

Four programs that facilitate and expedite development and review of new drugs that address unmet medical needs in treating serious or life-threatening conditions have been especially noteworthy. A look at recent drug approvals suggests that these programs have played an important role in bringing innovative drugs to market. Nearly half of the 27 novel drugs approved by FDA last year took advantage of at least one of these expedited drug development and review approaches. Review times were as short as 4.5 months.

After incorporating input we received from stakeholders based on a draft version, we are finalizing our guidance to industry to provide a more detailed explanation of these programs and help drug innovators determine whether their products are likely candidates.

These expedited programs include:

Fast track designation: This provides for more frequent meetings and communications with the FDA to discuss the drug’s development plan and ensure the collection of appropriate data needed to support drug approval, including such things as the design of the proposed clinical trials and use of biomarkers.

Accelerated approval: This bases approval not on a clinical endpoint but on an agreed-upon surrogate marker, i.e., a measure such as a blood test or urine marker, that is believed to be indicative of a disease state and treatment effect, but not demonstrative of a direct health gain to the patient.

Priority review: Acting on drug applications within six months instead of 10 months for standard review.

Breakthrough therapy designation: This provides all the benefits of the fast track designation plus intensive guidance on an efficient drug development program, beginning as early as Phase 1, along with the commitment from the FDA’s review staff, including senior managers, to work closely together throughout the drug development and review process.

Certainly our new breakthrough therapy designation, created as part of the 2012 FDA Safety and Innovation Act (FDASIA), has been a virtual overnight success. As of May 5, 2014, we have received 186 requests for the designation, and granted 48. Six drugs have been approved, including a late-stage lung cancer drug that was approved four months ahead of its goal date, using evidence from a trial with 163 patients.

Since the accelerated approval’s inception in 1992, more than 80 new products have been approved under this pathway. It has been successful in driving innovation in cancer and HIV therapies, but we’re encouraging its broader application in other areas. The FDASIA has helped by clarifying that the FDA has the authority to consider epidemiologic, pharmacologic, or other evidence developed using biomarkers or other scientific methods or tools to determine whether an endpoint can support accelerated approval. We’re also exploring whether reviewer training programs and other measures might encourage greater use of this program.

It’s important to note that our own regulatory flexibility is likely reducing the number of sponsors that avail themselves of the accelerated approval program. Sponsors of most of the recent new drug approvals for rare diseases—products that might otherwise qualify for the accelerated approval program—aren’t opting for that development pathway simply because they don’t need to do so. Although all are being approved based on surrogate or intermediate clinical endpoints, most of these products are receiving “traditional” approvals―meaning that no additional trials will be needed to verify clinical benefit. That’s because we decided that the results were already strong enough.

We urge drug developers and others interested in this movement to take a close look at the FDA’s final guidance on expedited drug approvals. For those drugs that qualify, participating in one of these expedited programs can reduce the time and possibly the cost of developing new therapies that can save lives. That’s a win for drug innovation and for patients.

About The Author

Janet Woodcock

Janet Woodcock, M.D., is the director of the Center for Drug Evaluation and Research at the Food and Drug Administration. A prominent FDA scientist and executive, Woodcock has received numerous awards, including a Presidential Rank Meritorious Executive Award, the American Medical Association's Nathan Davis Award, and Special Citations from FDA Commissioners. Woodcock received her M.D. from Northwestern Medical School.  She completed further training, and held teaching appointments at the Pennsylvania State University and the University of California in San Francisco. She joined FDA in 1986.