Dr. William Gahl and NIH Undiagnosed Diseases Program

There are over 6,000 Rare Diseases and these affect up to 25 million Americans. They are defined by law as disorders that affect fewer than 200,000 each...there are only a few hundred treatments for these [because]...it's not profitable for pharmaceutical companies to invest in a disorder that involves only 20 people, 50 people, or even 500 people...
We have potential treatments that make sense, that have presumably very few side-effects, and yet, we are not allowed to use them...The Food and Drug Administration ensures the safety of our foods and of the drugs that come to us...but there is one issue, for rare diseases, they apply the same criteria for the use of an investigational drug as they do for common diseases.

FDA: Ensures Drug Safety
People with rare diseases deserve
the same protection as othe human subjects.

Dr. Gahl proposes a change to this FDA's policy,

FDA: Ensures Drug Safety
People with rare diseases deserve
the same protection as othe human subjects.
Risk vs. Benefits?

Some diseases are so rare that there is no financial incentive for pharmaceutical or biotech companies to seek cures or treatments. That leaves physicians like Dr. Gahl without the assistance of research normally performed by profit-seeking pharmaceutical companies. Since FDA does not approve the use of medication on any patient without first having gone through a very expensive research protocol, these potentially effective treatments cannot be administered to rare disease patients, who then go untreated and at times face certain death.

"People in the Rare Disease Community want to be protected, but don't want to be protected to death."
Dr. Gohl
http://www.cbsnews.com/video/watch/?