Ethics in medical trials

[steuard]

There's a thought-provoking article on medical ethics in today's New York Times: New Drugs Stir Debate on Basic Rules of Clinical Trials. The crux of the article is this: controlled clinical trials are the gold standard of medical evidence because they give the clearest evidence about whether one treatment is better than the alternatives. If we have no clear expectation for which treatment is more effective, then there's no ethical dilemma: the control group might well be better off. But the more compelling the evidence that one treatment will turn out to be superior, the greater the moral difficulty in refusing that treatment to the study's controls.

As the article points out, there have been times in the past when we've short-circuited the trial/approval process for treatments that seemed clearly better, only to learn later that they actually hurt survival rates. Careful testing really is important! And if you release a promising new treatment before formal testing, you may never be able to convince people to participate in a trial that could deny it to them. But none of that makes it any easier to look someone in the eye and tell them, "You have to die so we can make sure this new drug works as well as we think it does."

So there's the question: Is there any scientifically acceptable alternative to the usual 50/50 controlled trial that would reduce the ethical dilemma in cases like this?

Or to frame that in a concrete (if extreme) scenario: Imagine that every participant in a small Phase 1 trial took a single pill, and their advanced cancer immediately vanished and showed no sign of recurring after six months. What sort of full-scale trial would you demand as hard evidence that the pill was effective? How far from this extreme would the results have to be before you could accept a standard trial?

Comments

[feuervogel]

The control group for new treatments for HIV and cancer doesn't get placebo. They get current best practices/standard of care. A lot of studies for diseases where the ethics of giving no treatment are less clear-cut, like hypertension, are designed as non-inferiority, as well (ie, "is our new ACE inhibitor as good as this other one?" which results in way too many me-too drugs that don't have any clear advantages over the other ones.)

[feuervogel]

This blog post may be relevant to your interests: http://scienceblogs.com/insolence/2010/09/balancing_scientific_rigor_versus_patien.php