Social Media Shakes FDA’s Power: A Case Study of Compassionate Use

We’ve written about states’ “right to try” laws, which expand desperate patients’ power to use drugs before the FDA approves them. These laws, gaining bipartisan approval in a growing number of states, open up the question of whether FDA powers that limit the availability of new medicines can be challenged by states.

The problem that “right to try” laws address is that patients who have no alternative treatment, except an experimental medicine, are often willing to take more risk than the FDA appreciates. They want the FDA out of the way and are not satisfied with federal policies that expand access to experimental medicines.

Pharmaceutical executives whom I have questioned assert that “right to try” laws are naive. In one sense they are: I have yet to identify a drug-maker that is supplying medicines under the auspices of a “right to try” law. The problem for a drug-maker with a unique new medicine for a deadly illness that strikes a small population is that every patient who takes the drug on an experimental basis is one less patient who can be enrolled in a randomized, double-blind clinical trial — the “gold standard” demanded by the FDA for approval.

A couple of pharmaceutical executives have written a very honest article about their experience dealing with a request for compassionate use of an experimental drug, while one of them was CEO of the company that was developing it.

Brincidofovir is an anti-viral drug designed for patients who receive stem-cell tranfusions for bone marrow or immune system failure. For a short time, the company (Chimerix) provided the unapproved medicine under a government arrangement that investigated its use against smallpox. However, when that ended, the company stopped approving requests for compassionate use, anxious that it would not be able to complete its necessary trials.

What is interesting is that the company changed its mind as a result of one family’s social-media campaign — not a change in law or regulation. The company was on the receiving end of such vilification that it pressured the FDA to find a way to allow compassionate use.

Also interesting is that the company’s stock jumped 50 percent once it started distributing the drug on a compassionate basis. The stock market, being a market, has a more accurate view of the value of the new medicine than either the company or the FDA (as discussed in an earlier blog entry).

Comments (6)

Trackback URL | Comments RSS Feed

  1. Devon Herrick says:

    Several obstacles come to mind. One is that an adverse reaction to a compassionate-use drug could derail or slow approval — even if the drug is good, but needs more evaluation. Another worry is that a compassionate-use patients’ family could later sue asserting harm.

    I agree with John that compassionate-use is hardly a way to speed approval. A drug would have to be nearly ready for clinical trials before most drug companies would willingly provide drugs for compassionate use. At best, the process would be sped up only by months. What we need is an alternative to traditional clinical trials using close monitoring and data analysis for patients willing to participate.

    • Shane Myers says:

      Is there nothing a pharmaceutical company can do to minimize liability? If not, this practice certainly does leave the door open for patients to exploit potential acts of kindness.

      • Ashley Dawson says:

        Forget exploiting acts of kindness. How compassionate is it to provide untested drugs to sick people?

        • John R. Graham says:

          Nobody is forcing anybody to take an experimental drug. As long as accurate information is disclosed, people should be free to choose.

  2. Perry says:

    Funny, Americans want access to untested drugs, but they’ll be quick to call an attorney if there are major side effect issues.

  3. John Thacker says:

    Also note that the CEO ended up leaving the company, and most people connect it with this entire incident (whether the initial bad press, or the stress, or what).