Redressing the Harm of Accelerated Approval

<p><i><span>The accelerated approval pathway of the United States Food and Drug Administration (FDA) enables drugs to come to market more quickly than would be possible under a traditional FDA approval pathway. Accelerated approval is based upon the agency’s determination that changes in a surrogate or intermediate clinical endpoint are “reasonably likely” to predict a clinical benefit meaningful for patients. In essence, the pathway affords sick patients earlier access to potentially beneficial drugs while trials to confirm clinical benefit continue. Accelerated approval has been likened to a social compromise in which promising drugs enter the market sooner in exchange for a sponsor’s promise to undertake so-called confirmatory trials—that is, postmarketing trials to “verify and describe” the predicted clinical benefit. This Article argues that patients, too, are expected to engage in a compromise when they take drugs approved under the pathway: patients must accept the risk that a drug will ultimately confer no meaningful benefit in exchange for the chance of treatment. But must the compromise end there?</span></i><span></span></p> <p><i><span>This Article deconstructs the harms that result from the accelerated approval pathway and explores how those harms should be remedied. For purposes here, the focus is on therapies later withdrawn from the market due to a sponsor’s inability to verify clinical benefit or its decision not to pursue confirmatory trials to completion. Patients and payers incur great expense during what the FDA has termed the “period of uncertainty,” the span of time between a therapy’s approval under the pathway and verification of clinical benefit or lack thereof. Moreover, therapies approved under accelerated approval, the bulk of which are immunotherapies to treat various types of cancer, often have serious side effect profiles. This means that patients who consume later-withdrawn therapies may suffer serious adverse effects or even a hastened death from a drug that ultimately yields them little to no benefit—in essence, a “toxic placebo.”</span></i><span></span></p> <p><i><span>This Article proposes a tort alternative modeled on the National Vaccine Injury Compensation Program to “make whole” patients harmed by accelerated approval’s “toxic placebos.” The proposed administrative compensation scheme, funded through a combination of prescription drug user fees and mandatory contributions from sponsors of later-withdrawn accelerated approval drugs, would allow patients to recover medical expenses, out-of-pocket costs for the accelerated therapy itself, compensation for side effects causally linked to a later-withdrawn drug, lost wages, pain and suffering, and wrongful death. This Article does not stand as a criticism of the accelerated approval pathway, which has successfully yielded a plethora of new cancer therapies, among others. Rather, it addresses why an administrative compensation scheme is a necessary adjunct to the pathway and preferable to the status quo in which patients harmed by accelerated approval typically remain without redress through the civil tort system. Finally, it counters the notion that the defense of assumption of risk should bar recovery for patients who incur harm from therapies approved under the pathway.</span></i><span></span></p>