New white paper reviews the regulations and case law on compassionate-use access to experimental drugs

“Compassionate use” access to unapproved, experimental drugs often shows up in stories of desperate families seeking to exhaust every last resort to obtain a treatment for an ill relative. New therapies, and growing awareness of trials, is putting the demand for compassionate use access “on the rise,” says the author, Justine Cuccia, an underwriter/attorney at ACE Medical Risk & Life Sciences (Jersey City, NJ), a division of the international insurer ACE Group. (And, like a properly risk-aware insurance company would, ACE labels the “opinions and positions expressed in this paper [as] the author’s own.”) This demand “is putting drug companies at odds with the very people they are developing treatments to save,” she says.

According to Cuccia, FDA can grant an exception to drug trial sponsors to offer access to the experimental drug to those who do not qualify for participation in the trial, but whose lives are at risk from their disease. Another situation can arise when a trial is shut down or completed, and the sponsor cancels further access to the drug—a likely scenario when the trial is deemed unsuccessful. A seldom-recognized element of this process is that for ongoing trials, the drug sponsor is permitted to charge the test subject for access to the drug (this ability was granted under the FDA Amendments Act of 2007; the obvious challenge could be where a patient is granted compassionate access, but then can’t afford the treatment).

“When companies turn down compassionate use requests, the patients may turn to the courts for relief,” warns Cuccia. Litigation can start up over who is or is not allowed into a trial; what expectations patients entering the trial have; and what communications occur not just with the drug sponsor, but also the independent principal investigator (PI). The specifics of the “clinical trial agreement” (CTA) between the sponsor and the PI need to clearly delineate what authority the sponsor and the PI have. At the end of the day, “As part of a thorough risk management program, pharmaceutical and medical device companies should seek to partner with an insurer that has significant experience in human clinical trials and compassionate use situations,” she concludes.

The paper is available here.