Wednesday, February 4, 2009
You or a loved one is terminally ill. There are no FDA-approved drugs available to treat the disease.
There is ongoing research on a drug that could dramatically increase hope of recovery. There are risks with this potential reward, as there has not yet been enough time to fully explore the drug's long term potential for benefit and/or adverse side effects.
Do you or that loved one take the risk?
Right now the answer is a flat no because of current U.S. regulations.
In his booklet "More Choices, Better Health," Bartley J. Madden advocates for patients and their families to have that choice available to them.
Madden questions the FDA's effectiveness in overseeing drug approval, and charges that the agency is more concerned in "playing it safe" and avoiding mistakes in approving drugs too soon, rather than considering all those who are dying because it takes a drug too long to reach the market.
Madden calls for passage of legislation that would give seriously ill patients the right to access promising drugs before the FDA completes all of its clinical trials.
Passage of the bill, called the ACCESS Act, would be a "genuine step forward in helping some patients with life-threatening illnesses and chipping away at the FDA's absolute control of access to unapproved drugs."
In making his argument, Madden points to the fact that before 1962 the U.S. had a much more streamlined process for getting new drugs to market.
While that's true, Madden fails to mention one of the reasons why the FDA's rules changed in 1962. The rules changed in part because of two words: thalidomide babies.
Thalidomide first rose to prominence in Europe and on a limited basis was provided for use by pregnant women in the U.S. as a treatment for morning sickness.
The drug was removed from the market in 1962 because its use led to severe birth defects involving arms and legs.
The number of children affected in the U.S. was minimized by FDA action, but thousands of children elsewhere in the world were born with the defects.
Personally, I would be against Madden's discussion in turning back the clock and pulling back on the regulatory process new drugs have to go through.
But if the discussion is limited to patients who are diagnosed with life-threatening diseases, then I agree there should be an avenue for them to pursue alternatives with potential reward that are not yet on the market.
The key, and here I again agree with Madden, is to make sure patients and their families have informed consent - that is full knowledge in advance of the potential damaging side effects from taking the drug.
Speaking of informed consent, a troubling thing about "More Choices, Better Health" is the argument for loosening FDA rules comes not from a medical doctor, but from a research economist funded by The Heartland Institute, a think-tank that says its goal is "developing and promoting free-market solutions to social and economic problems."
In other words, there may be a nugget of a good idea here, but it would be nice to see Madden's publication have some backing from the medical community before fully endorsing his booklet.
Otherwise, we risk duplicating the situation we have now in health care: economists and bean counters telling medical professionals who does and does not qualify for medical procedures.