EMA’s reflection on placebo does not reflect patients’ interests

Abstract

The European Medicine Agency (EMA) is preparing a reflection paper on the need for an active control in pivotal trials supporting marketing authorization applications [1]. The paper takes for granted the presence of a placebo arm, while “the need for an active control must be considered on a case-by-case basis”. The EMA’s reflection stems from the consideration that “the most common primary objectives for pivotal clinical trials are to demonstrate superiority to placebo control, or to demonstrate non-inferiority or equivalence to an active control”. Though preferred by prospective marketing authorization holders developing new drugs, these approaches are not in the best interest of patients or those paying for the public healthcare system. Both the criteria of superiority over placebo and non-inferiority to active comparators may allow drugs onto the market that are in fact less effective or less safe than those already available, and do not help define the place in therapy and respective values of new and available treatments [2]. Extended use of placebo overlooks possible added value