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Commentary

Access Before Approval A Right to Take Experimental Drugs?
Susan Okie, M.D., New England Journal of Medicine, 8-3-06

Editor's Notes:

Dr. Okie examines a recent D.C. appeals court decision allowing terminally ill patients access to experimental drugs and argues that the court's decision may severely impair the FDA's ability to evaluate new medicines. Still, she is also acutely aware of the frustration facing terminally ill patients who are literally dying for the lack of new treatments.

Accepting the existence of such a right would fundamentally challenge the government's system for evaluating drugs. In mid-June, federal officials filed an appeal seeking to have the case reheard by the full nine-judge panel of the appeals court. If the ruling is upheld, "it's a huge, huge, devastating decision," says William Schultz, a former deputy commissioner for policy at the FDA. "The more you offer early access, the harder it is to get the data" on safety and efficacy, because many patients will seek treatments directly rather than enrolling in trials in which they might be randomly assigned to receive placebo or another treatment. "It would be very hard to figure out which drugs work," says Schultz; the incentive for conducting clinical trials "would seriously diminish"; and permitting companies to market drugs without evidence of efficacy would create "massive opportunity for fraud, involving people who are very sick and very desperate."

But some observers applaud the Abigail Alliance for highlighting the struggle to balance the desire of sick people for cutting-edge treatments with society's need for scientific evidence of safety and efficacy. Many people with life-threatening diseases cannot find appropriate clinical trials, live far from research centers, or do not meet eligibility criteria, and many cannot obtain experimental drugs from manufacturers through "compassionate-use" programs. Dale O'Brien, medical director of the California-based Lorenzen Cancer Foundation, one of several patient-advocacy groups that have expressed qualified support for the Abigail Alliance's efforts, argues that "dying people ought to have special latitude to work with the growing edge of science."

Hopefully, policymakers can better ways to expedite access to experimental treatments to this group of patients.



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