Experimental Treatments? Unapproved But Not Always Unavailable

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.