Session (CTI)

PURPOSE: This session seeks to critically engage an international audience for operationalizing life-cycle health technology assessment (LC-HTA) and real-world evidence (RWE) to manage precision medicine uncertainties during the regulatory approval process. Our panel will: introduce a novel framework for life cycle assessment for highly uncertain precision medicine technologies; present learnings from the implementation of this framework within a Canadian cancer care system; and discuss international relevance and transportability of RWE generated through LC-HTA. DESCRIPTION: Advances in precision medicine challenge conventional regulatory and reimbursement processes. These advances are rapid, involve small patient groups, and are frequently evaluated without a randomized comparison group. Life-cycle assessment alongside patient engagement and continuous RWE generation can resolve evidentiary uncertainty on comparative effectiveness and value. This session will begin by introducing an LC-HTA framework operationalized within British Columbia, Canada’s cancer control system through the PRecision oncology Evidence Development in Cancer Treatment (PREDiCT) program (10’ Regier). Centred on a case study of entrectinib, a conditionally authorized treatment targeting advanced NTRK-gene fusion-positive cancers, presenters will then: establish comparative effectiveness by concatenating single-arm Phase I/II entrectinib trial data with RWD from British Columbia and the United States (13’ Weymann); determine real-world cost-effectiveness of entrectinib versus standard care, demonstrating how value for money evolves with new evidence in relation to a national index economic evaluation (13’ Krebs); and discuss RWE transportability for informing international regulatory and reimbursement decisions (13’ Adamson). The session will conclude with a facilitated discussion on key challenges for embedding LC-HTA within learning healthcare systems, engaging with the audience through a real-time prioritization polling exercise (10’).