The Continued Use of Placebo-Controlled Clinical Trials in the Study of Peptic Ulcer Disease: A Sponsor Perspective

Abstract

Investigators and institutional reviews boards who/which have participated in recent controlled gastroenterology clinical research studies assessing new therapies for peptic ulcer disease have questioned the continued use of a placebo-control when efficacious therapies for peptic ulcer disease are currently available. As the sponsor of such studies, Glaxo is of the opinion that the use of placebo-controlled clinical research studies is appropriate in certain situations. In choosing between the use of an active comparator or placebo-controlled trial Glaxo recommends an assessment of the severity of the disease and the morbidity associated with use of placebo treatment. It further recommends the need to assess the efficacy of placebo therapy to establish the baseline remission rate for the disease. Glaxo suggests an assessment of the safety data for the new chemical entity under investigation, and if such data are lacking, minimize patient risk employing suggested study designs that maximize patient utilization. The statistical implications of sample size and the risks associated with Type I and II errors by comparing the number of patients required for a typical study using a placebo and active control are discussed. Finally, for all placebo-controlled studies performed with patients with peptic ulcer disease, the elimination of high risk patients, the use of supplemental antacids, and proper obtainment of informed patient consent is recommended.