Citing a serious slowdown in the creation of new drugs, the Food and Drug Administration plans to give medical companies better guidelines on how to prove a new product works.
The main issue isn't how quickly agency scientists review treatments, which has sped up greatly in recent years, but the overall time it takes to research and develop new medications and medical devices, said new FDA Commissioner Mark McClellan.
That can take over a decade and cost hundreds of millions of dollars. McClellan hopes that by making the FDA's requirements for approval clearer up front, companies could do better research faster and might not be as reluctant to take chances on truly novel treatments instead of financially safer copycats.
That doesn't mean the FDA is relaxing its requirements, he stressed.
"If the drugs don't meet the standards for approval, they're not going to get through," McClellan told reporters. "But it's in everybody's interest to figure out more quickly and at a lower cost."
Sparking McClellan's concern: Drug companies are sending the FDA fewer groundbreaking medications. The FDA approved just 17 never-before-seen chemicals last year, down from 24 in 2001, said drug chief Dr. Janet Woodcock. It received 23 applications for innovative drugs last year, down from 30 the year before and a high of 60 in 1995.
Similarly, the FDA approved 32 novel medical devices last year, down from 56 in 2001.
The FDA is largely meeting congressional deadlines to review each application within six or 12 months, McClellan said.
But here's the rub: Many of those applications are rejected, largely because FDA scientists have questions the data can't answer. That happens to half of all novel medications and a stunning 93 percent of cost-saving generic drugs.
The clock ticks while companies struggle to answer the FDA's questions and refile the applications. Partly as a result, total approval time for the most important new drugs hit a median of 16 months last year, a 10-month increase from 2001.
Among the FDA's plans, to be formally issued Friday:
Help companies avoid sloppy or incomplete applications so more approvable treatments clear the FDA on the first try instead of the second or third.
Develop special guidelines for brand-new technology, such as gene therapy, bioengineered tissue or drug-and-device combinations so companies can design the right studies from the beginning.
Provide more training of FDA reviewers and consistency in requirements for each application.
McClellan's plans are more a repackaging than a completely new approach. For years, the FDA has encouraged companies to seek its advice early in a product's development.
But too few do, said veteran FDA watcher Ira Loss of Washington Analysis. He cited ImClone, a biotechnology company that imploded after the FDA rejected its potential cancer treatment Erbitux in December 2001, citing shoddy science that failed to tell if the drug had any effect. The company's top executive later was charged in an insider-trading scandal.
"At some point, you have to hold the industries accountable for not taking advantage of the opportunities presented to them," Loss said.
Drug discovery is cyclical and dependent on far more than the FDA — such as scientific serendipity and financial investments, Loss noted. But McClellan's plan "can't hurt," he said. "If it will help (with speed), we'll have to wait and see."
The medical device industry welcomed the news.
"The more innovators can know what the requirements are at the beginning of the process, the more quickly we can get those kinds of breakthroughs to patients," said Randy Burkholder of the Advanced Medical Technology Association.
The drug industry's Pharmaceutical Research and Manufacturers of America declined comment on the plan's specifics, but noted that over 1,000 experimental medications are in development, and almost 4,000 studies of those potential drugs are under way in patients.