Breakthrough Year for New Drugs

Feb 01, 2013

Jill Wechsler
The Food and Drug Administration hit a 16-year high in approving 39 new drugs in 2012, building on a continued rise in commercial INDs (investigational new drug applications) filed with the agency and provisions of the FDA Safety & Innovation Act (FDASIA) that encourage innovation and improve review operations. FDA had already approved more than 30 new molecular entities (NMEs) by early December, and eight more positive decisions emerged before year-end, including treatments for cancer, a rare leukemia, chicken pox, and drug-resistant tuberculosis.

Moreover, there's no more drug lag with Europe, according to John Jenkins, director of the Office of New Drugs in the Center for Drug Evaluation and Research (CDER), noting that more than 80 percent of last year's NMEs were first approved in the United States. And a continuing influx of INDs promises more new drugs in the future.

Targeted treatments

Prescription drug user fees authorized by FDASIA provide the agency with more resources to assist sponsors in proposing appropriate research programs and in filing more complete applications, a key metric in achieving more first-cycle approvals, Jenkins noted at Elsevier's FDA/CMS Summit in December 2012. FDA processed more than 70 percent of priority applications in one cycle in 2012, and 60 percent of standard NMEs. It helps that many of the applications were for targeted drugs with large effect size and for rare disease therapies with clear benefit/risk ratios.

A further stimulus for the development of more medicines for critical diseases may come from guidance in the works that will clarify the opportunities and requirements created by FDA's various expedited approval pathways—fast track, breakthrough, priority review, and accelerated approval. And new liaison staffers can provide assistance in navigating the FDA review system, which should be particularly helpful for the growing number of "emerging sponsors" filing their first new drug applications.

Bob Temple, CDER deputy director for clinical science, noted at the Elsevier conference that it's not new for FDA to approve highly innovative products based on limited clinical evidence, but that FDASIA is helpful by clearly instructing CDER to facilitate approval of "drugs with impact." The rise in new drug approvals, he observed, reflects an increase in very well-targeted cancer drugs with "huge effects" in specific populations, an approach that he hopes can be applied to other conditions, such as asthma.

Temple also is looking to wider use of enrichment strategies in clinical trials to accelerate drug development, as described in a December guidance outlining methods for decreasing heterogeneity and for selecting patients likely to respond to treatment. Temple has supported such study designs for years and anticipates that the guidance will encourage smaller, faster clinical trials that can speed critical drugs to patients. "While enrichment won't save a drug that doesn't work," Temple notes in an FDA blog, "it will help find one that will."

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