Reconsidering the FDA

Jerry Avorn is one of the most important researchers of pharmaceuticals, their appropriate use and how to influence that use. He clearly understands the FDA, drug marketing and the problems of drug side effects.

This weeks’ NEJM features a perspective which he wrote on the FDA. This perspective is available without subscription – and is a must read for anyone considering how the FDA works and how it should work.

FDA Standards — Good Enough for Government Work?

He finishes – and please read the entire piece –

The agency is also required to determine whether a drug is safe enough for use, a decision that can be reasonably made only in relation to the drug’s actual clinical usefulness. Since all drugs have side effects, it is not a stretch to expect that the approvability of a drug should take into account whether its risks are acceptable in light of its real-world effectiveness. This would require evaluating clinical benefit by means of a more relevant measure than short trials with surrogate outcomes. It would also require consideration of a drug’s efficacy and safety as compared with alternative therapies. If such studies are not required as part of the approval process, it seems that we don’t have any way to ensure that they are ever conducted; as a result, they usually are not.4

Manufacturers have claimed that such evaluation requirements would make preapproval testing too lengthy and expensive, but that is not a compelling argument. The sums spent by the large pharmaceutical companies on meaningful research and development are less than a third of what they spend on marketing, promotion, and administration.5 A rebalancing of this relationship would be quite feasible and could generate more clinically useful content for all those promotional activities. The better prescribing that such improved data would make possible would surely save the country more than the new approach would cost, since it would allow doctors, patients, and payers to understand the true value of a costly new product. Important new drugs that meet urgent and serious health needs could still be provisionally approved on the basis of less demanding studies but with a required reassessment a few years later to evaluate more relevant outcomes. Such a reassessment could be required of the manufacturer as a condition of keeping the drug on the market, or it could be undertaken by independent drug-evaluation units, with the results disseminated broadly to inform decisions on prescribing and purchasing drugs.

Some in the industry would argue that the lowest possible standard of efficacy is good enough and that an act of Congress would be required to change the current rules. But such an act is not inconceivable. Increasing public concern about efficacy–risk–cost trade-offs may move this agenda forward in Washington, especially if Medicare becomes the nation’s largest drug purchaser in 2006. The ballooning cost of that program may bring together clinical scientists, advocates of prudent federal spending, and even free-market aficionados, all demanding more useful standards. The idea that government approval should be based on what a new drug really does for patients may soon come into its own.

This article is, in my opinion, a tour de force. He expresses his ideas clearly, and documents his ideas carefully. His article speaks to many ideas that I have tried to state in the past. His perspective puts flesh on the skeleton that others have considered.

Bravo!