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Access to Experimental Drugs: Hard Cases Make Bad Law

I oppose open access to early-stage therapies. However, hard cases make bad law. This was amply demonstrated by the riveting front-page article in Sunday’s New York Times, “Months to Live: Fighting for a Last Chance at Life.” This is a must-read for anyone concerned about terminal illness or early access. Definitive rules are hard to develop, especially while heart-strings are pulled hard.

The reporter, Amy Harmon, follows the agony of a family whose son/husband/father has ALS (amyotrophic lateral sclerosis). There is a product previously on the market for a different use that allegedly provides relief from progression of the disease. Unfortunately, the firm that was developing it was restrained by a patent settlement. The company that won the patent dispute did not want to acquire responsibility for a population/use it had no intention of serving.

As is usually the case, FDA was torn between compassion today and assuring that clinical trials can still be undertaken in the future. The two companies and FDA also know how rare it is for internet-hyped medicine to prove useful.

To the reporter’s credit, she doesn’t blame either of the companies or FDA. The story is impactful and honest in chronicling the family’s frustration–without choosing good guys and bad guys.

I have had my own lessons in this. It was a crisp New England morning almost 20 years ago. I was helping a company prepare for a “shut the plant down” demonstration by AIDS activists determined to gain access to an experimental therapy. The drug had never been tested in humans. Nor, as I recollect, did it have significant animal testing to back it up.

Given these factors, it seemed like a particularly poor choice for early-access. Understandably, the activists saw this as a dire situation where taking on extraordinary risks was a choice they should be able to make for themselves. There are no good guys or bad guys in this type of situation.

The ALS situation is more nuanced than the one I faced 20 years ago. There appears to be safety data, but probably not at the dose levels that ALS patients would use. There is anecdotal support for the drug’s use, although such reports are notoriously unreliable. The ALS case does not necessarily raise what I consider a fundamental issue: should totally untried drugs be available on open access.

Generations of law students have been taught: hard cases make bad law. This certainly applies here. And FDA can never win. One recommendation would be to upgrade the review function and include some outside ethicists and scientific advisors in the process. However, the publicity that would come from doing so might have the effect of doubling or tripling the number of requests, many totally baseless.

The NY Times logged 165 comments in less than 2 days. Clearly the story (and the underlying issues) touched people. What advice would you give FDA in handling demands for early access where there is some data, but not enough to be sure of anything?


The article and the comments posted to NYT website: http://www.nytimes.com/2009/05/17/health/policy/17untested.html

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