This refers to the practice of giving terminally ill patients who are otherwise ineligible for early phase clinical trials access to investigational agents. At the Brugge ESGCT meeting, Finnish researcher Akseli Hemminki described providing 125 patients compassionate use access to a novel oncolytic gene transfer vector– Ad5/3-Cox2L-D24. Hemminki was not reassuring when asked by an audience member whether a concurrent clinical trial was actually testing the approach, and rumors swirled that he had charged patients for the agent.
Compassionate Use is highly controversial. Patient advocates view it as a lifeline, and over the years, drug regulators like FDA and EMEA have eased restrictions on patient access to untested agents. In late 2006, for example, the FDA proposed new rules that would make it easier to provide untested agents to groups of patients (rather than individuals); the rules would allow companies to recover manufacturing costs from patients seeking access. A related set of proposed new rules would allow companies to charge patients for entering early phase clinical trials.
Compassionate Use raises troubling questions for ethics and policy. With respect to the former, is it really an act of compassion to offer terminal patients a completely untested composition of matter? If the answer is "yes," well, that makes any future clinical trial that will randomize some patients to standard of care (which, for terminal patients, is nothing) diabolical. That leads me to the policy concerns: if you can get access to a drug outside a study, why enter the trial at all? Compassionate use potentially complicates the collection of rigorous data about the safety and efficacy of new interventions, and thus has public health implications. As for the idea of actually charging patients for access– whether on or off a trial: that's plain wrong.
According to the American Cancer Society, no records are available on the number of agents provided through compassionate use, the volume of patients who receive drugs through compassionate use, or their outcomes. Regardless of where one stands, the practice would seem a policy black box. (photo credit: fgm878, 2007).