How will payers receive innovative trial designs?
As costs continue to rise and downward reimbursement price pressure increases, the industry is exploring new drug development approaches, including innovative clinical trial designs such as adaptive, umbrella, and basket studies. Adaptive trials, the most established of these, have already been the subject of guidance documents by both the European Medicines Agency (EMA) and Food and Drug Administration (FDA). Further, in 2017 the FDA provided its first site-agnostic approval of an oncology drug (pembrolizumab) based on data from basket trials. 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.