Food and Drug Administration's Obesity Drug Guidance Document

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An estimated 70% of adult men and 60% of adult women in the United States are overweight or obese.1 Excess body fat increases the likelihood of developing hypertension, dyslipidemia, and type 2 diabetes mellitus and is an independent risk factor for cardiovascular disease.2–4 Obesity is linked to an increased risk for certain cancers, osteoarthritis, and sleep apnea.5–8 Obese people are stigmatized.9 Medical costs attributable to obesity are enormous.10 The healthcare community and patients would thus welcome the development and approval of new obesity drugs with favorable benefit-risk profiles. To facilitate drug development, the US Food and Drug Administration's (FDA's) Center for Drug Evaluation and Research issues guidance documents for the pharmaceutical industry. These documents provide the agency's current thinking on therapeutic indications, target populations, clinical trial designs, and data analyses. This article examines the origins and evolution of the FDA's guidance document for the development of drugs to treat obesity. In 1947, the FDA approved the first prescription obesity drug, desoxyephedrine or methamphetamine.11 Approval of amphetamine congeners (eg, phentermine), fenfluramine, and other appetite suppressants followed over the next 2½ decades. Then, in 1973, with the country struggling with a long-running epidemic of amphetamine abuse, the FDA, concerned about the abuse potential of the amphetamine congeners and their transient efficacy, limited the indication of all obesity drugs to short-term use (ie, a few weeks).11,12 This restriction did little to counter opinions that vanity was the only reason to lose weight and that obesity drugs had no role in long-term weight loss. This mindset began to change in subsequent years. “[While] most public attention and economic activity related to obesity has been devoted to cosmetic and esthetic concerns about body weight, it has become increasingly obvious that obesity is …