A History of Accelerated Approval: Overcoming the FDA's Bureaucratic Barriers in order to Expedite Desperately Needed Drugs to Critically Ill Patients

Abstract

After the passage in 1962 of the Kefauver-Harris Drug Amendments that mandated that the FDA grant premarket approval for all drugs and added a requirement that drug manufacturers demonstrate the efficacy of their products, the drug approval process dramatically slowed for the next two decades. Only after a combination of sustained criticism by free market advocates and dramatic lobbying efforts and protests by AIDS activists desperate for any drug that might prolong their lives did the FDA relent and implement the accelerated approval program. The FDA can grant accelerated approval for drugs designed to treat life-threatening diseases for which no approved treatments currently exist based upon data from surrogate endpoints (laboratory markers), so long as the endpoints are reasonably believed to predict clinical benefit. This paper examines the history of accelerated approval, and in particular its application to AIDS and cancer drugs. Additionally, it explores critiques of the program from clinical academicians and consumer advocates, and replies from its defenders including the pharmaceutical industry, patient advocates and free market economists. It concludes with recommendations on how to reinvigorate the process in order to expedite the development of cancer drugs.