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Experimental Treatments? Unapproved But Not Always Unavailable

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"FDA has worked diligently to balance two compelling, and sometimes competing, factors," says FDA Commissioner Jane E. Henney, M.D. "On one hand, there is the need for the disciplined, systematic, scientifically controlled studies necessary to identify treatments that may improve patient health and that lead to the approval of new drugs. At the same time, there is the desire of seriously ill persons, with no effective options available, to have the earliest access to unapproved products that could be the best therapy for them."

Over the last decade, FDA's institutional philosophy has evolved to be more supportive of thoughtful risk-taking by patients who have run out of options. As a result, the agency has put in place a number of regulatory mechanisms and worked with manufacturers to ensure that seriously ill patients can get access to promising, but not fully evaluated, products. At the same time, FDA has protected the critical scientific studies that must be carried out so that patients, physicians and the agency can determine which drugs are truly safe and effective, and how they can best be used.

"We believe that the best means of providing access to useful medical treatments for all Americans is to continue to shorten the review times," Henney says, "and to continue to work with the industry to shorten development times for drugs, biologics and medical devices."

The Intervention of AIDS

Before the 1980s, a more paternalistic medical community argued that it was the government's job to protect patients from possible harm by withholding experimental drugs until there is proof that they work and are safe.

AIDS helped alter that view. Not only did that lethal disease spread with terrifying speed, but it struck a patient population capable of mounting a political response that grabbed the nation's attention and galvanized public health policymakers to reconsider long-held beliefs.

Experimental treatments should be available, The Washington Post quoted one activist at the time, "so people would be able to choose for themselves, working with their doctors, whether they want to risk taking a drug because of the possible benefits."

Critics accused FDA of denying dying patients access to possibly lifesaving drugs. To drive home the point, in October 1988, more than 1,000 gay activists staged a protest outside FDA's Rockville, MD, headquarters, trapping the agency's staff inside.

"FDA is the nexus between the government, the private sector and the consumer," the spokeswoman for one of the protest organizers told the Post. "That's why we're targeting (the agency)."

The protest had an effect. The agency, already focused on the issue by the urgency of AIDS, accelerated its reexamination of the way people with serious and life-threatening diseases could gain access to unproven remedies. Although the treatment IND regulations were finalized in 1987, FDA put in place additional mechanisms to make experimental drugs available to seriously ill patients earlier in the drug development process.

WebMD Public Information from the FDA

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