Faster Access to Drugs for Serious or Life-Threatening Illnesses Through use of the Accelerated Approval Regulation in the United States

Abstract

The Food and Drug Administration issued a final regulation on accelerated approval for human drugs, antibiotics, and biologics on December 11, 1992. This regulation provided a formal regulatory mechanism for a sponsor to seek accelerated approval by submitting a New Drug Application for a drug intended to treat a serious or life-threatening illness. In order to qualify for accelerated approval, a drug must seek an indication for an illness having either a surrogate endpoint which is reasonably likely to predict clinical benefit or a reliable clinical endpoint other than survival or irreversible morbidity. This important and much needed regulation has been used to achieve accelerated approval of several antiretroviral and oncology drugs, as well as several other drugs and biologics. Since this regulation has been in place for almost five years, the purpose of this paper is to summarize all of the drugs that have utilized this regulatory mechanism to attain approval in the United States. From 1992 through the end of July 1997, a total of 20 drugs have received accelerated approval. This summary of these drugs shows that this regulation has been a successful means of providing more rapid regulatory review and approval for drugs intended to treat a serious or life-threatening illness. Regulatory affairs professionals should be familiar with the precedents for approval of drugs under this regulation, both to enable them to follow well-trodden paths and to consider extending the experience to drugs for other indications under appropriate circumstances.