Choice of Control Group in Therapeutic Device Trials

• Activity Number: 35
• Type: Topic Contributed
• Date/Time: Sunday, August 11, 2002 : 4:00 PM to 5:50 PM
• Sponsor: Biopharmaceutical Section*
• Author(s): Sarah Kogut*+
• Affiliation(s): Medtronic Neurological
• Keywords: : therapeutic medical device ; choice of control group ; ICH E10 ; placebo control ; sham surgery ; ethics

Abstract

Recent advances in technology have given medical devices the potential to restore function and quality of life in patients where medication is not completely effective in controlling symptoms or causes severe side effects. This talk will focus on modifications of customary designs that allow for ethical, practical and scientifically valid placebo-controlled device trials. The FDA requires at least one pivotal controlled trial to demonstrate safety and efficacy for a device in support of a premarket approval (PMA) application. However, choice of a control in medical device trials can be problematic. The ethics of withholding treatment from patients or performing sham surgeries has been a topic of debate. The controls considered in the E10 guidance document by the International Conference on Harmonization (ICH) include: placebo, no-treatment, dose-response, active, and external. Pilot studies can help choose an appropriate control group. Some design options in device trials are cross-over study; three-arm study with new treatment, standard treatment, and placebo control; or having subject serve as own control. Examples from the therapeutic device industry will be discussed.