Ontario’s new Minister of Health Eric Hoskin argued in a recent Toronto Star op-ed article for a national pharma plan as an essential component of equitable health care. Federal Health Minister Rosa Ambrone appears also supportive of exploring the development of such a plan. The arguments in favour, based on equity and cost containment, are strong. The development of such a plan will only increase the need to ensure the production of reliable drug safety and effectiveness information. Shannon Gibson and I discuss in a recent paper in the OUP journal Medical Law Review (Shannon Gibson & Trudo Lemmens, "Niche Markets and Evidence Assessment in Transition: A Critical Review of Proposed Drug Reforms" (2014) 22(2) Med. L. Rev. 200-220 available online here for people with UofT library access; and here in a pre-print version on SSRN), how the development of niche pharmaceutical products for which often exorbitant prices are being charged, creates additional pressure to adjust existing drug regulatory and funding systems. As pharmaceutical development strategies evolve and drug products become more complex, regulatory and policy responses must be able to evolve along with them. In our MLR paper, we explore 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 and more independent evidence gathering, must be incorporated into any reform efforts towards adaptive licensing and PBRSA.
A related paper, focusing more on the North-American market, will be coming out in 2015 as chapter in a book on the FDA in the 21st Century: The Challenges of Regulating Drugs and New Technologies (New York: Columbia University Press, forthcoming 2015).
