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).

– See more at: http://healthblog.ncpa.org/social-media-shakes-fdas-power-a-case-study-of-compassionate-use/#sthash.aSszCrZx.dpuf

Source: healthblog.ncpa.org