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
Is this exactly what my doctor prescribed?
How often should I take this drug?
What should I do if I miss a dose?
Does it matter what time of day I take this drug?
Is there anything I should avoid while taking this drug (such as driving or alcohol)?
How will it interact with other prescription or over-the-counter drugs I am taking?
How will it interact with vitamins, herbal supplements, or foods?
What side effects should I watch for?
What should I do if I have a bad re...
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
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.