Summary: GAO compiled information on the federal government's involvement in drug development programs to assist Congress in its consideration of the federal role in developing orphan drugs. GAO reported on federal controls over development and marketing of drugs and described federal drug development, highlighting the basis of government involvement, the scope of their activities, the status of drug development in these programs, and some factors to consider in establishing new drug development programs.
The Food, Drug, and Cosmetic Act provides that a new drug may not be introduced into interstate commerce unless the Food and Drug Administration (FDA) has approved a new drug application (NDA) for it. The process from research to marketing approval of a new drug takes from 7 to 13 years and costs $30 to $50 million. The process involves preclinical research aimed at discovering and identifying a new drug that is sufficiently promising to study in humans, clinical research to determine human efficacy and side effects, and FDA evaluation and approval. Most preclinical research takes place in industry and then is submitted to FDA. If FDA approves, clinical studies can begin. The NDA must contain all information on the safety and effectiveness of the new drug and information on the proposed manufacturing process and quality control. However, several federal agencies are involved in 12 drug development programs because of perceived needs for attention to a specific disease and a belief that industry could not be expected to fill these needs. Industry's reluctance is attributed to the uncertain profitability of developing and marketing the new drugs. The preclinical and clinical stages of drug development under the 12 federal programs were designed to satisfy the requirements of FDA regulations. The evidence obtained is later turned over to a private company to obtain an approved NDA from FDA. Upon FDA approval, the company may market the drug. Thirty-five new drugs have been developed under the program.
