In remarks made at an industry conference the other day, the controversial Cleveland Clinic cardiologist took the agency to task for repeatedly missing deadlines for approving drugs, BioPharma Today writes. He was referring to PDUFA, or the Pharmaceutical Drug User Fee Act, in which the FDA accepts industry fees to review drugs and commits to completing those reviews by specified deadlines.

But Nissen says the FDA is not keeping up its end of the bargain and a first order of business for the next FDA commissioner should be to fix the problem. “If you take the money, and you agree to perform based upon the money, you’ve got to perform,” he told the crowd, while acknowleding he is not a big fan of the PDUFA concept. “The agency is not living up to its part of PDUFA legislation.”

Nissen, you may recall, regularly conducts research for drugmakers and is known as an industry skeptic thanks to his high-profile challenges to the safety of a number of products. He is also viewed as a leading candidate to succeed Andy von Eschenbach as FDA commish. For him to declare that FDA needs to move faster, at least in some circumstances, could be seen as a peace offering of sorts to drugmakers as political jockeying for the commissioner post gets underway, the newsletter points out.

Citing fewer resources and an emphasis on implementing safety legislation passed last year, the FDA decided to allow reviews to slip beyond the six-and 10-month targets it pledged to follow as part of the funding arrangement with pharma. The agency failed to meet PDUFA dates for at least 15 drugs during 2008, including several that could be considered major innovations, Nissen noted (back story).

“We’ve got to clear this backlog of drugs that have blown past their PDUFA dates without review, without action, leaving innovative drugs in limbo,” he told the crowd, adding that New Drug Applications should be given a thumbs up or thumbs down within statutory timeframes.

But he conceded the need to boost agency resources. “If you ask someone to do more than they have the resources to do, they’re not going to succeed,” he said, citing the FDA’s IT system. “To ask FDA work with 10-year-old computers and then be speedy in how it does its work doesn’t make sense. (You can’t expect FDA to) “do outstanding work on a poverty budget,” which he described as a “national priority.”

He also worried aloud that there may be few new drugs in a few years and contended genomics will not solve the problem. A solution may be “limited-term approval with requirements for large Phase IV outcome trials,” which would allow innovative but risky drugs to enter the market with a clear understanding that certain things must be proven or the approval could not be renewed, he explained.

To lower development costs, Nissen supports large, simple trials that can be more efficient and less expensive, and would be designed to answer the question: “at the end of the day, are more people who got the drug alive, healthy and free from strokes, myocardial infarctions or cancer, or whatever?”

To do this, FDA has to buy into the fact that these trials do not provide as much info as those conducted today, Nissen pointed out. The Critical Path Initiative was created to improve drug development, but “has largely been a disappointment,” he concluded. “It was well-intentioned, but it has not yielded any real serious difference.”

Source: BioPharmaToday