Presentation (CTI)

OBJECTIVES: Outcomes-based agreements (OBAs) are an emerging option to address clinical uncertainties and financial risk with innovative medicines while enabling timely market access for patients. This research sought to determine if real-world data (RWD) from the Canadian Neuromuscular Disease Registry (CNDR) could be used to operationalize an OBA in Canada, focusing on spinal muscular atrophy (SMA).
METHODS: Using the Focus Group Discussion method, five individuals with expertise in the areas of SMA, the CNDR, health technology assessment and OBAs were convened seventeen times, from February to September 2024. Each meeting focused on a specific topic, including the CNDR data collection processes, SMA health outcomes, current available SMA therapies, international OBAs, and public payer processes, among others. An iterative process was used to summarize the data collected and develop study results, until consensus was achieved by the group.
RESULTS: The group identified eight health outcomes for SMA, one of which was suitable for an OBA (motor scales), and three of which were suitable in combination with others (survival; permanent invasive ventilation; feeding tube). Three key gaps and their solutions were identified when using the CNDR for an OBA: not all SMA patients are registered in the CNDR (~ 30%); patient consent does not allow individual-level data to be shared outside of the CNDR; reporting timelines require three months for data quality control processes. An operational process using the CNDR as the data source for an OBA was developed accordingly.
CONCLUSIONS: Using the CNDR to operationalize an OBA for SMA is feasible with the caveat that the solutions for the three identified gaps are deemed sufficient and implementable. Additional feedback from stakeholders is required, particularly from Canadian public payers and the pan-Canadian Pharmaceutical Alliance, to determine if further refinement to the proposed CNDR OBA process is needed.