A Cure? "Compassionate Use" and Drug Regulation


Are government bureaucrats keeping dying patients from getting access to possibly life saving drugs?  That’s one way to read Margaret Talbot’s story in the Sunday New York Times (“Fighting for a Last Chance at Life.”  May 17, 2009).  Talbot describes how mother Kathy Thompson sought access to an unlicensed therapy Iplex for her Amyotrophic Lateral Sclerosis (ALS)- afflicted son, Joshua.  The drug, Iplex, was not licensed by FDA for use against ALS, and an almost identical drug had failed two randomized controlled trials in patients with ALS.

Buoyed by anecdotal patient reports on web forums, Thompson sought compassionate use access from the FDA and was rebuffed.  She ultimately engaged the Washington Legal Foundation to appeal FDA’s rejection of Thompson’s request for the drug. FDA relented, allowing the drug maker to run a trial of Iplex– recouping all costs from enrolled patients (about $100k/year).

There are a number of troubling features in this story.  First, the article (or, at least Thomspson) makes FDA out to be a bunch of heartless bastards. Some, like the Washington Legal Foundation, view FDA’s stringency as an affront to the “civil liberties of American Business” (to paraphrase Haley Barbour). But let’s remember that, for over a half a century, FDA has played a critical role in public health by keeping unproven drugs out of the pharmacy. Grants of compassionate use seriously weaken the ability of the state to collect evidence of a drug’s safety and efficacy, because they make enrollment in clinical trials much more difficult. So there need to be some pretty compelling policy reasons- backed by persuasive evidence- to grant exemptions.

Second, the article states “Many are Campaigning for the chance to be treated with drugs whose safety and effectiveness is not yet known.”  While Iplex itself hasn’t been tested against ALS, its active ingredient has been tested in two large randomized controlled trials. Both failed to show any advantage over placebo.

Third, there’s the business of allowing desperate patients to “buy” their slots in the trial approved by FDA. For starters, it’s hard to imagine that a trial that included only “several dozen patients” is going to be of any use in measuring safety or efficacy (the two failed studies involved about 180 and 300 patients). As well, there is something unseemly about a “compassionate use” exemption that is priced at $100K.

ALS is an awful disease, and I feel for people like Thompson and her son. Surely, more and better care and research are needed for ALS sufferers. But where’s the compassion in a program that ministers only all to the most wealthy and politically connected? More importantly, where’s the compassion in undermining a system that ultimately protects us all from clinics that prey on the desperation of patients, and companies that sell unsafe, ineffective, and extremely expensive drugs? (photo credit: Monster 2007)


    title = {A Cure? "Compassionate Use" and Drug Regulation},
    journal = {STREAM research},
    author = {Jonathan Kimmelman},
    address = {Montreal, Canada},
    date = 2009,
    month = may,
    day = 17,
    url = {http://www.translationalethics.com/2009/05/17/a-cure-compassionate-use-and-drug-regulation/}


Jonathan Kimmelman. "A Cure? "Compassionate Use" and Drug Regulation" Web blog post. STREAM research. 17 May 2009. Web. 25 Feb 2021. <http://www.translationalethics.com/2009/05/17/a-cure-compassionate-use-and-drug-regulation/>


Jonathan Kimmelman. (2009, May 17). A Cure? "Compassionate Use" and Drug Regulation [Web log post]. Retrieved from http://www.translationalethics.com/2009/05/17/a-cure-compassionate-use-and-drug-regulation/


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