Friday, August 04, 2006

Playing games with people's lives

The New England Journal of Medicine explores:
the ACCESS Act, a bill introduced this past November by Senator Sam Brownback (R-Kans.). Under the proposal, a drug could obtain tier 1 approval on the basis of phase 1 testing [usually consisting of only a few hundred patients and providing little if any guidance on side effects] and preclinical evidence — from testing in animals, case histories, pharmacologic studies, or computer modeling — that it "may be effective against a life-threatening condition." A drug with tier 1 approval could be marketed for seriously ill patients who had exhausted other treatment options, if they waived the right to sue the manufacturer and permitted collection of their clinical data. Tier 2 approval would correspond to today's accelerated approval, and tier 3 approval to full approval.

The bill has alarmed the clinical research community and large health-advocacy groups. Only 11 percent of drugs — and only 6 percent of cancer drugs — that enter clinical testing are ultimately approved; the rest either prove to be too toxic or do not work. If the bill became law, "the market would be flooded with drugs and we wouldn't have any idea which ones were effective. It would ultimately be very bad for patients," says Colin Begg, a biostatistician at Memorial Sloan-Kettering Cancer Center and a member of the board of the Society for Clinical Trials, which opposes the bill. Although the bill theoretically covers only "seriously ill or dying patients who have run out of options," warns Begg, "it's not at all clear that things would stop there."

Cancer-related clinical trials cannot accommodate all the patients who want experimental medications, but instead of permitting access after phase 1 testing, a better solution would be to expand treatment IND programs for later-phase drugs, says Bruce Chabner, clinical director of the Cancer Center at Massachusetts General Hospital, Boston. "Even safety is not resolved" by the end of phase 1, Chabner says. "I don't have a right to fly somebody's experimental airplane, so why should I have the right to some drug that a company has dreamed up?"

Rather than allowing untested medications to be marketed, "we should be promoting and making clinical cancer research normative and part of how we treat cancer," suggests Ellen Stovall, president of the National Coalition for Cancer Survivorship. Only about 5 percent of adults with cancer enroll in clinical trials, Stovall notes, partly because many community oncologists do not encourage participation and because databases of trials are confusing and incomplete. Yet stories like that of Abigail Burroughs resonate with the public. And "public opinion could pass a bad bill," notes Stovall.
Another thing we could do to improve the chances of curing cancer would be to allow broader funding of research on stem cells. But, of course, Brownback has done all he can to block such an expansion. I presume that this bill is his attempt at penance for hanging sick people out to dry, but it does so poorly. Giving patients unapproved drugs would complicate their existing treatments, expose them to unknown side effects, and undermine existing tests for efficacy.

The existing system of drug testing has worked quite well for us, keeping dangerous treatments off the market and making safe drugs available. While a few high profile cases exist where a drug slipped through based on faulty reporting (Vioxx), the infrequency of that result is a testament to the hard work the FDA does. People I know who have worked with drug companies preparing for an FDA hearing are always impressed with the level of preparation and care they have to go through to get approval.

That protects us all. Undermining the system hurts everyone. If Senator Brownback wants to improve access to medicine, he should start by giving Medicare the power to negotiate drug prices, then work on improving production of and access to generic drugs. If he wants to improve the clinical testing system (and there are plenty of ways it could be improved) why not offer to help fund clinical trials of drugs in exchange for shorter patents or other price breaks for consumers. That would improve the quality of the testing process by taking the testers off of the manufacturers' payrolls and also make quality drugs available at less cost.