US court supports use of unapproved drugs in terminally ill patients

Terminally ill patients in the US who are seeking access to experimental, unapproved drugs have won a legal reprieve in US Court of Appeals for the District of Columbia Circuit.

A three-judge panel in the court voted two to one in favour of reinstating a lawsuit brought by the Abigail Alliance for Better Access to Developmental Drugs in 2003 against the US Food & Drug Administration (FDA), in which it was claimed that the FDA was enforcing a policy "that violates the constitutional privacy and liberty rights of terminally ill patients..[prohibiting] mentally competent patients with no other treatment options from purchasing investigational drugs - medicines showing initial evidence of safety and efficacy in clinical trials, but not yet approved - even though their physicians recommend these drugs as their best hope of surviving or of prolonging their lives".

The Court of Appeals stated that dying patients have a basic "right of self-preservation" and that drugs that have passed the Phase I FDA review if they have the potential to be life-saving. "Barring a terminally ill patient from the use of a potentially life saving treatment impinges on this right of self-preservation," it added. The case will be returned to the district court that dismissed it in 2004 for a full hearing and possibly a trial.

Paul Kamenar, senior executive counsel for the Washington Legal Foundation, the law firm that argued the case on behalf of the Abigail Alliance, described the ruling as "a tremendous victory for patient groups and those who are in need of these kinds of medicines" and a big defeat for the FDA.

"We remain sympathetic to the desire of terminally ill patients to gain access to experimental treatments when they have exhausted other therapeutic options, and are exploring a number of new efforts to improve how we make investigational drugs available through expanded access programs," said Scott Gottlieb, deputy commissioner for medical and scientific affairs at the FDA. The Agency has previously said that it already has programmes in place that make potentially life-saving drugs available before final approval, and that allowing large numbers of patients to take unapproved drugs could put many at unacceptable risk.

The Arlington, Virginia-based Abigail Alliance was founded in 2001 by Frank Burroughs, whose 21-year-old daughter died of cancer, to help cancer patients and others with life threatening illnesses gain increased access to developmental drugs.