Experimental Treatments: Not Always Unavailable

by Food and Drug Administration (FDA)

The scalpel has failed. The IV tubing stands abandoned on the side of the room. Friends and nurses visit less often. The doctors say the limits of medical knowledge have been reached and there is nothing left for you to do but go home and put your affairs in order.

This is a crushing moment. It's even more frightening than the day the doctors announced that you had a serious and life-threatening disease, such as AIDS, cancer or Alzheimer's disease. For many people, however, the limitations of medical knowledge do not define the limits of human hope. As long as life lingers, many patients will fight on, refusing to give up even when biology is against them. So, they set out on a search for treatment options.

And many alternatives exist. In today's medical bazaar, options range from alternative and complementary therapies like acupuncture and homeopathic and naturopathic medicine to nutritional supplements and macrobiotic diets, home-brewed remedies, and even outright frauds like laetrile. But greater promise resides in the drug development pipeline where tomorrow's therapeutics await proof that they work. Unlike alternative therapies, billions of dollars and decades of scientific study often have been invested in the research that leads to promising new therapies. Might there be, somewhere in that high-priced gauntlet, just the right molecule that cures a patient who is running out of time?

The answer is possibly, but finding it isn't easy. Short of randomly hearing about a promising study through the media, most people know relatively little about what drugs are in development. Even if experimental drugs existed in a database, "it is hard to know which drugs are truly promising," says David Banks of the Food and Drug Administration's Office of Special Health Issues. But on average, about 80 percent of the drugs in testing will ultimately be approved.

Getting your hands on a novel medicine can be even more difficult. Usually, only the company has any supply of the new medicine, which is extremely limited to begin with, and most of what is made will be used in clinical studies.

As if these hurdles are not enough, there is the long-held, but incorrect, public perception that FDA erects regulatory barriers that block patients from getting investigational new drugs (INDs). These are drugs that pharmaceutical companies have in clinical trials to demonstrate their safety and effectiveness, but which have yet to be approved by FDA for marketing. For those with a serious illness, the agency rarely blocks access to unproven medications. But FDA does strive to protect all patients, even those who may be dying, from undue risks associated with investigational new drugs. At the same time, FDA believes that the best way to benefit all patients is to speed promising new therapies through the development and approval process so safety, effectiveness and proper use can be established.

"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.

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."

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.