Drugs, Devices, and the FDA: Part 1: An Overview of Approval Processes for Drugs

Abstract

Over the last 150 years, the U.S. Food and Drug Administration (FDA) has evolved from a small division of the U.S. Patent Office to 1 of the largest consumer protection agencies in the world. Its mission includes ensuring that new medical treatments reach the public as quickly as possible while simultaneously ensuring that new treatments are both safe and effective. In the face of urgent consumer need, the FDA has faced criticism that its processes are too lengthy and costly and that the time to new drug release is significantly longer in the United States than in other Western countries. Calls from the public to loosen FDA regulations to facilitate more rapid approval of drugs and devices have been countered by the occurrence of patient harm and deaths after some approved drugs have reached the marketplace. New drug and device approval in the United States take an average of 12 and 7 years, respectively, from pre-clinical testing to approval. Costs for development of medical devices run into millions of dollars, and a recent study suggests that the entire cost for a new drug is in excess of $1 billion. For investigators seeking approval for new drugs and devices, FDA processes can be formidable. This 2-part series is intended to provide an overview of the steps involved in bringing new drugs and devices through the FDA process. Part 1 concerns the process of new drug approvals. Part 2 continues with approval of medical devices.

Keywords: CDER, Center for Drug Evaluation and Research; EIND, emergency investigational new drug; FDA; FDA, U.S. Food and Drug Administration; IND, investigational new drug; NDA, new drug application; drug approval; investigational new drug.

Figure 1

Time, Money, and Success: Stages in Drug Development The highest failure rates occurs in Phase II testing, which is the first stage in which doses of drug in humans are escalated to reach levels expected to be clinically active (i.e., the first doses at which efficacy may fail and less common side effects appear). Cumulative probability of a drug reaching U.S. Food and Drug Administration approval declines with each stage. The overall probability of a drug passing all stages is approximately 11% as of 2014 .