Theme 2: Health technology assessment (HTA) and evidence development

Theme 2: Addressing: 1) how should reimbursement decision-makers assess the evidence for a PM technology and identify the best value reimbursement strategy for their population; and 2) how should developers of PM technologies evaluate alternative research investment options to maximize the Expected Net Present Value of the investment from a commercial perspective, in a value-based reimbursement environment.

Part 1: Evidence development
Theme Leader: Jonathan Kimmelman, Faculty of Medicine, McGill University

Many lament the fact that the vast majority of drugs introduced into clinical testing later prove unsafe and/or ineffective. PM is often heralded as a fix for this problem, because researchers can use genetic markers to match patients to particular drugs. But there are several wrinkles. One is the fact that, by accelerating drug development, PM technologies may lead to drugs being approved before their properties are well understood. Another is that translating drugs by PM requires validating molecular diagnostics, and like the road to validating drugs, the road to validating diagnostics is long, narrow, and overgrown. Dr. Kimmelman’s team sets out to trace the way evidence accumulates about PM in cancer, exposing some of the strengths and weaknesses of current practice. The ultimate aim is to develop tools to help physicians and policy-makers better evaluate the clinical utility and uncertainties surrounding cancer PM’s.

Interested and Engaged Stakeholders: Clinicians, clinical trialists, healthcare decision-makers, HTA agencies.

Part 2: Heath technology assessment (HTA)
Theme Leader: Christopher McCabe, Faculty of Medicine and Dentistry, University of Alberta

Dr. McCabe’s research team develops methods for the design of efficient research and development programs for PM technologies, specifically for diagnostic, prognostic and monitoring test technologies using genomic, proteomic or molecular information and for co-dependent or combined technologies also known test-guided therapies. Working within the Value of Information Framework, the team is developing methods to establish the return on investment in research from both commercial and health system perspectives. It tests these methods on one or more of the PACEOMICS case study technologies.

Dr. McCabe’s and Dr. Kimmelmann’s teams work together to develop evidence appraisal frameworks for PM technologies that can be used by reimbursement authorities to specify reference case health technology appraisal evidence submissions. The teams link to Theme 1 by examining the value that Canadians attach to PM compared to conventional types of intervention. The PACEOMICS program complements Dr. McCabe’s ongoing research the commercialization of stem cell technologies in the presence of HTA reimbursement hurdles, funded by the Canadian Stem Cell Network and the development and reimbursement of treatments for rare diseases, funded by CIHR.

Interested and Engaged Stakeholders: PM technology developers, investors, HTA agencies, healthcare system decision-makers.