Preapproval Access and Right-to-Try Initiatives: What Are We Willing to Give Up?

There is a growing concern within the US Food and Drug Administration (FDA) and the pharmaceutical/biotechnology communities about state-based initiatives to provide access to drugs that are in the early stages of development. These legislative bills allow a patient to circumvent the protections afforded by regulated procedures as embodied in FDA regulation and guidance. Over the course of more than 80 years, the latter have served to best ensure the safety and therapeutic efficacy of new medicines and have evolved into an effective set of protections of human privacy, dignity, and fairness. To undermine these longstanding principles may have consequences which we, as society, must be prepared to address. This article examines the dynamic tensions that exist between the perceived best interests of the individual patient, the patient community, legislators, regulators, physicians, and the society at large. Decisions have consequences and, too often, these fail to be considered in the process of choosing the pathway that, in a context fraught with uncertainty, best meets a desperate need.