FDA Makes Rules For Early Access To Experimental Drugs

Of all the criticism leveled at the Food and Drug Administration in recent years, some of the most emotional has focused on patient access to experimental drugs for serious illnesses.


It's a tough spot for the regulators. On one side are desperate and dying patients who feel they have little to lose by trying an unproven drug that could help, even if it might be toxic or eventually turn out to be a dud. If the patients aren't part of drugmakers' clinical tests, though, they're usually out of luck.

On the other side? Well, there's the problem of false hope and the needs of the rest of us, who might like a medicine some day that's been thoroughly tested to determine its effectiveness and shortcomings. If people can get experimental drugs on their own, they may not sign up for clinical trials. And that makes it tough to figure out whether the medications actually work.

Until now, the FDA's rules about so-called "compassionate use" of experimental drugs for patients not in clinical trials have been murky. Now the agency has clarified them, and added a few new conditions.

Find the consumer-friendly version here. And the lawyer-friendly version is here, if you want every regulation spelled out (it runs 99 pages).

The rules make it clear that companies can, if they want to, apply to the FDA for permission to provide drugs to patients in need. The agency will consider whether the company is doing adequate formal testing, and that the drug offers at least some reasonable hope.

For the first time, companies will be allowed to charge individual patients, but only enough to recoup the cost of manufacturing and distributing the drug, not the cost of entire research and development process.

The new rules are part of the agency's avowed push for greater transparency. "The goal is to make clear to patients, medical professionals and the pharmaceutical industry how investigational drugs can be used prior to approval," FDA chief Margaret Hamburg said at a press conference.



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