FDA touts approval speed as year ends

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Pushing back against complaints that the drug approvals process moves too slowly in the US, FDA issued a report saying it kept pace with its strong 2011 showing, and noted that it approved drugs faster than its international peers.

Globally, the agency said 75% of the drugs it approved in 2012 got the domestic green light before getting the go-ahead abroad. In the US, the FDA noted that it took an average of 19 months for a drug to get agency approval two decades ago. That figure was down to 10 months, on average, as of last year.

FDA also said that of the 35 New Molecular Entities (NMEs)—which refers to “novel chemical structures as well as biological products that have never been approved in the U.S.,” all but one met the target PDUFA date.

The agency attributed its agility in part to programs that allow the regulator to up the review pace, such as Fast Track, Priority Review and Accelerated Approval. Half of this year's green-lighted NMEs were approved using one of these three programs.

Fast Track launched in 2007, and includes frequent back-and-forth between the regulator and drugmakers during development, as a way to frontload the review process with insight so that fewer gaps need to be accounted for later. The agency said nine of the 12 fast-tracked drugs got through the approval process with just one review this year. Fast Track is for treatments that focus on serious or life-threatening diseases, such as AIDS, cancer and Alzheimer's.

Priority Review shortens the drug review time from the typical 10 months to six. FDA said 11 of the 12 Priority medications were approved with one review cycle, including Kalydeco, which targets a specific gene defect that causes cystic fibrosis. This approval pathway is for conditions which lack adequate therapy.

Accelerated Approval is also for therapies that treat life-threatening diseases, fill an unmet need and look like they could hit a clinical result, but lack the proof. This track comes with a caveat in that approval means a post-marketing review study.

Among this year's new drugs were Genentech's Erivedge for late-stage basal cell cancer, Medivation's Xtandi for late-stage prostate cancer, Jakafi for bone marrow cancer and Jazz Pharma's Erwinaze for acute leukemia.

The approvals process was the topic of heated debate at yesterday's Forbes Healthcare Summit, with Cleveland Clinic's Dr. Steven Nissen pronouncing Europe's less rigorous approvals system "an unmitigated disaster." "You often hear the mantra that FDA is the problem," said Nissen. "We do have an innovation problem, but not because of regulation. It's because the easy targets have been found." He added that "Innovation is good, but only when it makes people better, and the only way to make sure it makes people better is to have adequate testing."

Dr. Don Berry of the Anderson Cancer Center at the University of Texas countered that the American way of approvals is outmoded and that it's time to "rethink the drug approvals pyramid." "Randomized control trials changed the world, but they haven't changed in 60 years," said Berry, arguing that the process is ill-suited to testing ever more narrowly-targeted treatments. "Rare diseases are the canary in the coal mine," said Berry. "FDA doesn't know what they're going to do when everybody with cancer can be said to have an orphan disease."

Matthew Arnold contributed reporting from the Forbes Healthcare Summit

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