Top 10 Medical Stories of 2008: No. 10

No. 10: Gunvalson v. PTC Therapeutics

A teenage boy with an X-linked muscular dystrophy, Jacob Gunvalson, and a small biopharmaceutical company, PTC Therapeutics, locked legal horns over access to the company's investigational agent, PTC124. Jacob and his parents claimed that key PTC employees had promised access to the agent, while discouraging clinical-trial enrollment, and PTC denied the charges.

The case highlighted a number of important considerations in the development of pharmaceutical products, particularly by small companies. Namely...

• The advisability of close communications between company representatives and patients;
• The nontrivial nature of a company's application for a single-patient (ie, compassionate-use) investigational new drug application (IND) to the FDA; and
• The maintenance of the integrity of the clinical-trial process (on which evidence-based medicine rests).

With respect to this last and vital issue, it is paramount that investigators maintain the general uniformity of trial enrollees and the fairness of drug access in the form of trial randomization, if at all possible. Moreover, they should not create a disincentive for individuals to enroll in clinical trials by providing medication to a particular individual outside of trial enrollment,* especially when trial subjects may be assigned to placebo, and others must wait for FDA approval to access the drug.

In August, a NJ federal judge ordered PTC to provide its drug to Jacob,** but that decision was reversed by a US appeals court last week. The appeals court determined that Jacob and his parents had failed to plead the requirements of an alleged promissory estoppel. Specifically PTC's "clear and definite promise" of access to PTC124 was not demonstrated and, perhaps most important, Jacob did not forgo enrollment in a key PTC124 trial (which would have provided PTC124 to Jacob in an extension phase) on the advice of a PTC employee. He was denied enrollment, because he did not meet the trial requirements.

For more details on Gunvalson v PTC Therapeutics, go here, here, here, here, and here and check out relevant posts of the more-than-passively interested lawyers at the Drug and Device Law blog.

* A prime example of privileged, and arguable unfair, drug access is provided by prominent personal injury lawyer Fred Baron's treatment with Tysabri (natalizumab; Biogen Idec/Elan) through the lobbying efforts of none other than Lance Armstrong and Hillary Clinton.

** In the form of submitting a single-patient IND to the FDA on behalf of Jacob, and, if the FDA denied the IND, by asking for a protocol exception so that he could enroll in a phase 2a extension trial of PTC124.