Experimental Treatments? Unapproved But Not Always Unavailable
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
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.
With the activism around AIDS and the demands of people with
other serious illnesses for access to unproven treatments, the medical
community, including FDA, began to appreciate that the traditional risk/benefit
models may have been inappropriate for people with serious and life-threatening
diseases. Dying patients were willing to take bigger risks for even the
slenderest hope of benefit.
"The hope part of it is that it might work and keep them
alive a little longer," says Theresa Toigo, Associate Commissioner for the
Office of Special Health Issues. "Even if it is only two months, by then
there might be a cure. It is a wonderful survival instinct."