Gimme: The "Right" to Access Experimental Therapies

While sympathies for anyone with an untreatable and ultimately fatal condition—like Duchenne muscular dystrophy (DMD)—are undeniable, the right for such a person to access an experimental, proprietary therapy is not clear. There's the issue of unproven safety (not to mention efficacy) and the violation of numerous clinical-trial protocols (on which evidence-based medicine rests) that control important variables—like an enrollee's health status, the double-blind process, and randomization of treatment.

Nevertheless, a New Jersey federal judge ruled yesterday that a 16-year-old boy with DMD, Jacob Gunvalson, should have access to PTC124, a drug in clinical-phase development by PTC Therapeutics. According to PTC's web site, a phase 2b trial of the drug is being conducted in ambulatory DMD patients with a nonsense mutation. PTC124 is an orally administered, small molecule that targets this particular mutation type.

It has not been reported if Gunvalson even harbors a DMD nonsense mutation (if not, the use of PTC124 would be, well, nonsense). Also photographs in news reports suggest that the teenager, shown in a wheelchair, is not sufficiently ambulatory at his age to qualify for the PTC trial. Confirmed particulars of the case, expected to be available in the judge's forthcoming written order, should answer these and other issues* that may have implications for anyone seeking access to experimental treatments.

According to the NYT, PTC Therapeutics plans to appeal the decision, and federal regulators must still approve Gunvalson's application to use PTC124.

* For instance, will Gunvalson even be considered an enrollee in the phase 2b trial, should he receive PTC124?