Overview

There is a well-recognized long-standing decline in R&D productivity in the pharmaceutical industry which has driven exploration of new approaches to the clinical development of pharmaceuticals.  These include innovative randomized controlled trial designs, including adaptive, umbrella, and basket studies. Adaptive trials, perhaps the best established of these, have already been the subject of guidance documents by both the European Medicines Agency and Food and Drug Administration. However, in many markets it is the reimbursement hurdle (rather than the regulatory hurdle) that is proving the major barrier to overcome in order to enable patient access. Therefore, payer perceptions on such innovative trial designs will be key to informing pharmaceutical company utilization of such approaches. This presentation will provide an overview of these innovative new designs and present payer perspectives on the benefits and challenges posed by conducting reimbursement assessments on data coming from these trial designs, based on discussions with a German and UK payer.