September 03, 2008

But You Promised: Court Opens New Strategy for Access to Experimental Therapies

There is an old saying that bad facts make bad law. In Gunvalson v. PTC Therapeutics, Inc., tough and emotional facts have made for increasingly complex law.

Gunvalson represents the new front in the battle over access to unapproved drugs for terminally ill patients. Remember that only last year, in Abigail Alliance v. von Eschenbach, 495 F.3d 695 (D.C. Cir. 2007), the D.C. Circuit Court of Appeals found that there is no constitutional right of access to experimental drugs (reversing the lower court finding to the contrary). So everyone thought that the legal battles over access to experimental therapies were over. Gunvalson proves that prediction wrong.

Just like Abigail Alliance, this case starts with a human tragedy. The plaintiff, a teenage boy, suffers from Duchenne Muscular Dystrophy (DMD), a disease that is inevitably fatal, usually by age 25. The defendant, PTC Therapeutics, Inc., is investigating a new drug called PTC124 for DMD. The factual twist in this case is that the plaintiff's mother had gotten heavily involved with PTC, patient advocacy, fund raising and, of course, her son's care. She had become good friends with various senior executives of PTC and even stayed at one of their homes.

Mrs. Gunvalson wanted her son enrolled in an early Phase II clinical trial of PTC124. She claims that PTC promised that while he didn't qualify for an initial Phase 2 study, he would be part of a future clinical trial. When subsequent trials commenced, he was denied access as he failed to meet the study criteria. PTC refused to pursue a compassionate use exemption.

Gunvalson sued asserting a right of access to PTC124 based on the alleged oral statements and contract/promissory estoppel theories. The plaintiff claimed that PTC had promised future access to PTC124 and that he had relied on these promises. Gunvalson sought and won a preliminary injunction requiring PTC to supply him with PTC124. The federal court also denied PTC's request for a stay pending appeal. The court went out of its way to try to limit the case to this "unique situation."

So, is Gunvalson an aberration triggered by the human tragedy of a child with DMD or does the case represent a new strategy for the terminally ill? We believe that it could be the latter. Gunvalson establishes the argument that statements by the company or its agents may create a right of access. Given this, any clinical trial sponsor or CRO should:

  • Ensure that no employee or agent makes any actual or implied promises,
  • Ensure that all written materials refrain from any access assurances,
  • Establish and follow internal procedures for the objective and independent review of compassionate use requests,
  • Consider adding integration and no oral representation clauses to clinical trial documents, and
  • Train employees, executives, investigators, CROs and other agents involved in the clinical development or advocacy not to make any implied promises or commitments.

Gunvalson raises many unanswered questions, including how PTC can provide the drug as ordered and still comply with FDA regulations, when can agents or investigators bind a company, pre-emption, and how explicit the "promises" must be to create a cause of action. What is clear is that terminally ill patients are becoming more aggressive and innovative in their quest for experimental drugs. These efforts can have far reaching effects on the patient, company, clinical research, advocacy, and the drug or device approval process.

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