Obesity pharmacotherapy from a regulatory perspective: overview and key challenges.

Obesity is an epidemic with tremendous impact on both patients and health-care systems globally. This paper explores some of the questions related to the clinical development of new pharmacotherapies in the context of an evolving regulatory perspective. These include patient entry criteria, clinical database size, study designs, weight loss end points (including those for maintenance of weight loss and prevention of weight regain), clinically important patient-reported outcomes, comorbidity/risk factor end points, and challenges in establishing safety and efficacy in adolescent/pediatric patients, and approaches to the development of combination pharmacotherapies. Ultimately, patients, physicians, academia, industry, payers, and governments must continue to partner with regulators to help establish the appropriate balance between the known adverse consequences associated with inadequate treatment of the growing obesity epidemic and the concern for potential unknown risks that may be associated with the long-term use of new pharmacotherapies.