Alex Philippidis Senior News Editor Genetic Engineering & Biotechnology News

Agency’s recent efforts to shake off industry criticism are not supported by the numbers.

For the second time in less than a year, FDA is patting itself on the back for its performance in approving new drugs, though the numbers show a more mixed picture.

During the first half of 2012, FDA approved a combined 14 new molecular entities and new biologic entities, compared with 20 in January–June 2011. New drug filings by industry also fell, to 15 compared with 22 a year ago. Despite those declines, FDA remains on track to roughly replicate its performance for all last year, when 30 new drugs won approvals.

FDA disclosed first-half numbers through June 18 in “New Drug Review: An Update and a Look Ahead,” a presentation by John K. Jenkins, M.D., director of the Office of New Drugs in FDA’s Center for Drug Evaluation and Research, during a June 26 investor conference featuring agency officials and hosted by Morningstar.

Within two days, FDA approved two more drugs—weight loss treatment Belviq, manufactured by Swiss-owned Arena Pharmaceuticals and distributed by Eisai; and Astellas Pharma’s Myrbetriq for adults with overactive bladder.

Last November, FDA issued a press release trumpeting its approval of 35 new drugs in the 2011 federal fiscal year (versus 21 in FY 2010) as its second-highest number of approvals of any year in the past decade. Eager to shake off industry criticism that it was too bureaucratic and slow in reviewing new drugs, the agency said its accomplishment represented “a very strong performance, both by industry and by the FDA”—a theme the agency repeated last month despite the approval and filing declines.

“Our track record on our performance is much better than what you might hear in the conventional media or even in the analyst community,” Dr. Jenkins said. “We continue to hear assertions that Europe is much faster, but that doesn’t match up with the data.”

In May, The New England Journal of Medicine published a study concluding that between 2001 and 2010, FDA approved more new medicines more quickly than the EMA or Health Canada. However, FDA approved the lowest percentage, just 61.8%, of new drugs (139) after one cycle, compared with 68.7% (68) by Health Canada and 96.2% (179) by EMA. Last year, FDA’s percentage of first-cycle approvals for standard-review drugs stood at over 50% but surpassed 90% for priority-review drugs.

In addition to Belviq and Myrbetriq, FDA approved Amyvid (Alzheimer disease diagnosis), Erivedge (advanced basal cell carcinoma), Inlyta (advanced kidney cancer), Omontys (anemia), Perjeta (late-stage breast cancer), Picato (topical actinic keratosis), Stendra (erectile dysfunction), and Zioptan (elevated eye pressure).

Also approved were four rare disease drugs—Elelyso (Gaucher disease), Kalydeco (rare form of cystic fibrosis), Surfaxin (respiratory distress syndrome), and Voraxaze (toxic methotrexate levels due to kidney failure). Rare-disease drugs accounted for 29% of total approvals, which in recent years have hovered around one-third of all drugs okayed by FDA.

Smaller drug developers or “emerging sponsors” accounted for half of the 14 new drugs approved from January–June 2012, compared with 10 or one-third in all of 2011. That seemingly reflects how startups are picking up the research slack as big pharma sheds R&D. But FDA didn’t examine emerging sponsors’ relationships with biopharma giants. Elelyso sponsor Protalix partnered with Pfizer, which also sponsored Inlyta, while Omontys sponsors Affymax partnered with Takeda.

“You have to look at the big picture and say, how much money does the FDA have? How difficult is it to assess the safety and efficacy of the drugs that are coming through now? It’s actually much more difficult to do,” Richard (Erik) M. Gordon, clinical assistant professor at the University of Michigan’s Stephen M. Ross School of Business, told GEN.

Under PDUFA V, FDA will continue collecting user fees toward new drug reviews. The agency is set to collect $720 million in FY 2013 to fund 2,599 full-time equivalent staffers.

As for assessing safety and efficacy, Gordon said that task has been complicated by the nation’s aging population, which has multiple illnesses and thus more existing drugs with which new treatments must interact; a shift toward drugs for chronic diseases; and stricter review standards that include risk-benefit analysis, enacted after deaths and other side effects linked to blockbusters like Celebrex and Vioxx emerged.

“When drug companies talk about how much more difficult it is, all you can say is, no kidding. It sure is more difficult,” Gordon said. “The FDA are not the deal killers. They don’t see themselves as the goalie trying to keep the puck out of the goal.”

Maybe not, but investors still do, judging from a survey released Monday by the National Venture Capital Association citing concerns about government support for investment—read FDA—as a reason why biopharma placed 8th of 11 industry sectors in investor confidence. PDUFA V and better industry knowledge about FDA’s risk-benefit approach should help FDA improve its performance further, but investors and others will wait and see what happens.

Alex Philippidis is senior news editor at Genetic Engineering & Biotechnology News.

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