Failure to adequately address the issue of risk/benefit assessment during drug development can result in delays in the approval of new drugs. The concept of risk/benefit is elusive to many pharmaceutical sponsors and little information concerning this concept is delineated in the Code of Federal Regulations (CFR). The purpose of this paper is to outline key risk/benefit variables which influence the approval of new drugs and to present case histories to illustrate the impact of these variables on the approval process. Finally, the concept of the “ideal” drug which involves the analysis of risk factors associated with a given disease and its treatment is outlined.
MillerL. Pitfalls in the drug approval process: dose effect, experimental design, and risk benefit issues. Drug Info J.1992;26: 251–260.
2.
CocchettoDMNardiRV. Benefit-risk assessment of investigational drugs: Current methodology, limitations, and alternative approaches. Pharmacotherapy. 1986;6: 286–303.
3.
CFR §312.80–312.88.
4.
FrickMHEloOHaapaKHelsinki Heart Study: primary prevention trial with gemfibrozil in middle-aged men with dyslipidemia. N Engl J Med.1987;317: 1237–1245.
5.
Meeting of the Endocrine-Metabolism Drugs Advisory Committee. October 17, 1988.
6.
GreenKG. Interpretation of the clofibrate trial. Lancet. 1984;1: 1095–1096.
7.
Meeting of the Cardio-Renal Drug Advisory Committee. December 16–17, 1985.
8.
BorerJ. tPA and the principles of drug approval. N Engl J Med.1987;317: 1659–1661.
9.
KoweyPRThe tPA controversy and the drug approval process. JAMA. 1988;260: 2250–2252.
10.
Meeting of the Antiviral Drugs Advisory Committee. January 16, 1987.
11.
Meeting of the Antiviral Drugs Advisory Committee. August 6–7, 1991.
12.
HenryJAMartinAJ. The risk/benefit assessment of antidepressant drugs. Med Toxicology. 1987;2: 445–462.
