Operationalizing Life-Cycle Health Technology Assessment for Precision Medicine

Purpose This session seeks to critically engage an international audience on operationalizing life-cycle health technology assessment (LC-HTA) and producing decision-grade real-world evidence (RWE) for precision medicine. Our panel will: introduce a novel LC-HTA framework for highly uncertain precision medicine technologies; present learnings from framework implementation within a Canadian cancer care system; and discuss transportability of RWE generated for informing international regulatory and reimbursement decisions. 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 HTA 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 care 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 via target trial emulation and fusing single-arm Phase I/II entrectinib trial data with real-world data from British Columbia and the United States (13’ Weymann); determine life-cycle cost-effectiveness of entrectinib versus standard care, demonstrating how value-for-money evolves as RWE emerges (13’ Krebs); and discuss evidence transportability across borders for international decision-making (13’ Adamson). The session will conclude with a facilitated discussion on advancing data fusion, LC-HTA operationalization, and RWE reliability across healthcare systems, engaging with the audience through a real-time prioritization polling exercise (10’).