In recent years, there have been important advances to ensure therapies for serious conditions are approved and available to patients as soon as there is sufficient data to 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.


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. The vast majority of the time, the United States is the world’s first country to approve novel medicines. Just last year, three-quarters of the new drugs approved by FDA were approved in the United States before any other country.


Four programs that facilitate and expedite development and review of new drugs that address unmet medical needs in the treatment of 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. And review times were as short as 4.5 months.


After incorporating input we received from stakeholders to a draft version, we are finalizing our guidance to industry today in order 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: Providing for more frequent meetings and communications with FDA to discuss the drug’s development plan and ensure 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: Basing approval not on a clinical endpoint but on an agreed upon surrogate marker, that is a measure such as  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 6 months instead of 10 months for standard review, and;


Breakthrough Therapy Designation: Providing all of the benefits of Fast Track designation plus intensive guidance on an efficient drug development program, beginning as early as Phase 1, and the commitment from 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 its inception in 1992, more than 80 new products have been approved under the Accelerated Approval pathway. It has long been successful in driving innovation in cancer and HIV therapies, but we’re encouraging its broader application in other areas, helped by FDASIA which clarified that FDA has the authority to consider epidemiologic, pharmacologic or other evidence developed using biomarkers or other scientific methods or tools in determining 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. While 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 today’s final guidance. 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.


Janet Woodcock, M.D., is the Director of FDA’s Center for Drug Evaluation and Research



Posted on the FDA website on 29 May 2014