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

Medical Law Review, 2014, Forthcoming

22 Pages Posted: 12 Apr 2014  

Shannon Gibson

University of Toronto Faculty of Law

Trudo Lemmens

University of Toronto - Faculty of Law

Date Written: 2013

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

Suggested Citation

Gibson, Shannon and Lemmens, Trudo, Niche Markets and Evidence Assessment in Transition: A Critical Review of Proposed Drug Reforms (2013). Medical Law Review, 2014, Forthcoming. Available at SSRN: https://ssrn.com/abstract=2423569

Shannon Gayle Gibson

University of Toronto Faculty of Law ( email )

Department of Statistical Sciences
Toronto, Ontario M5S 3G8
Canada

HOME PAGE: http://www.linkedin.com/in/shannongibson

Trudo Lemmens (Contact Author)

University of Toronto - Faculty of Law ( email )

78 and 84 Queen's Park
Toronto, Ontario M5S 2C5
Canada

Paper statistics

Downloads
71
Rank
264,911
Abstract Views
456