Court's Friends Appeal in Gunvalson v PTC Therapeutics

The federal court ruling that requires PTC Therapeutics to give its experimental drug to a teenager with muscular dystrophy "could easily destroy" the drug's clinical-trial program. This argument is found in one of 2 amici curiae (friends-of-the-court) briefs, which were recently filed in the appeal of the really bad decision in Gunvalson v. PTC Therapeutics. (Background here.)

One amicus brief is on behalf of a 13-year-old boy with Duchenne muscular dystrophy, and the other is from the disease-advocacy groups Parent Projects for Muscular Dystrophy Research, Inc, and United Parent Projects Muscular Dystrophy. Both are in support of the defendant-appellant, PTC Therapeutics, and are now available at the Drug and Device Law blog, which is operated by 2 lawyers involved in the appeal proceedings.

Among the briefs' arguments:

The court's ruling, which allows one person to access to PTC124 (currently in the middle of phase 2 development), is unfair to others with DMD who must wait for FDA approval of the drug.

The ruling creates a precedent for more drug-access litigation, which will unfairly tax the resources of PTC Therapeutics specifically and those of drug-development companies generally. According to the PPMDR/UPPMD brief, 3 parents contacted the PPMDR founder and president to ask whether litigation is an effective means to obtain PTC124. The close relationship between the Gunvalsons and PTC Therapeutics, cited by the ruling judge as a "unique" factor that should mitigate the risk of similar lawsuits, is actually common in the DMD community.

The ruling impedes the clinical-trial process by 1) undermining communication between companies and families that eases or enables clinical-trial enrollment and 2) creating a disincentive for subjects to enroll in trials in which they may receive placebo. (It should be noted that the subpopulation of DMD patients with a nonsense mutation—the pool of potential enrollees for PTC124 trials—is small, approximately 1700.

The ruling undermines the public interest and informed decisions by Congress and the FDA, which state that publicly available drugs must be proven safe and effective (by means of well-controlled clinical trials). The FDA has declared that expanded access to drugs before approval must not compromise enrollment in clinical trials. One brief goes so far as to indicate that the ruling "could easily destroy the PTC124 clinical trial program and render impossible FDA approval that would permit access to the drug for all patients."

And in a curious side note...

The ruling prompted a recent educational program, "Compassionate Use: Changes You Should Make Following Gunvalson et al v. PTC Therapeutics," which indicates that companies should make changes to their compassionate-use policies on the basis of the decision. (The CD, featuring lawyer Clint Hermes, is available for the bargain [cough] price of $199.00.)