Niche Markets and Evidence Assessment in Transition: A Critical Review of Proposed Drug Reforms

Abstract

As pharmaceutical development strategies evolve and drug products become more complex, regulatory and policy responses must be able to evolve along with them. We explore, with particular attention to UK developments, how proposals for “adaptive licensing” on the regulatory side and “performance-based risk sharing agreements” on the funding side are shifting the focus of drug regulation and reimbursement towards more incremental access to new therapies and more post-market evidence generation. At first glance, both reform proposals may appear as an effective response to managing the evidentiary challenges associated with niche markets, which have particularly been developed also in the context of pharmacogenomics, since they provide regulators and funders with mechanisms for controlling the risks and cost-implications of approving or reimbursing niche market products that may have been tested in smaller clinical trials and have a reduced evidence profile. However, a primary concern with both adaptive licensing and PBRSA proposals is that they may contribute to, respectively, a lowering of regulatory standards at the approval stage and premature market entry, and subsequently, mounting pressure to fund costly new pharmaceutical products with uncertain evidence profiles. We discuss the challenges associated with these developments, and argue that given the widely recognized problems associated with the evidence in existing regulatory and funding processes, measures to improve the reliability and independence of this evidence, such as improved transparency, must be incorporated into any reform efforts towards adaptive licensing and PBRSA, including consideration of how to implement such measures in the post-market phase.

Keywords: adaptive licensing, healthcare reform, pharmacogenetics, pharmaceutical regulation, drug reimbursement, risk-sharing agreements, data integrity, transparency, clinical trials