We are excited to present our research at ISPOR 2025 in Montreal, where our posters will contribute to the global dialogue on health economics and outcomes research (HEOR). In alignment with this year’s theme, “Collaborating to Improve Healthcare Decision Making for All: Expanding HEOR Horizons,” our work explores innovative methodologies and real-world data applications aimed at enhancing healthcare decision-making processes.
Developing a Checklist for Patient Engagement in Rare Disease Value Research
This poster presents the development of a checklist to guide meaningful patient engagement in rare disease value research, focusing on comparative effectiveness research (CER) and health technology assessment (HTA). Initiated by the Center for Innovation & Value Research, the project involved extensive stakeholder engagement, including patients, caregivers, payers, and others, alongside a literature review to identify key gaps in current practices. The multi-stakeholder Advisory Board was convened to provide input on key aspects of the checklist, including formatting, level of detail, and criteria for selecting rare diseases for case studies. The checklist is organized into four research phases: Initiation & Planning, Execution, Monitoring, and Dissemination & Assessment. The checklist provides structured guidance, including rating-scale questions and examples, to help researchers systematically integrate and assess patient engagement. Planned case studies in three distinct rare diseases will further validate and refine the tool.
Integrating Dynamic Pricing into Cost-Effectiveness Models: Implications for US-Based Drug Evaluations
This study investigates the impact of dynamic pricing on a cost-effectiveness assessment (CEA) of a hypothetical new therapy for Major Depressive Disorder (MDD). Traditional CEAs usually assume static pricing, failing to account for real-world price reductions over time. By modifying our existing open-source individual-patient simulation model to incorporate dynamic pricing, this analysis compared a standard treatment pathway to a new treatment over 5- and 10-year horizons. Results showed that dynamic pricing significantly improves the cost-effectiveness of the new therapy, reducing total costs and improving the incremental cost-effectiveness ratio (ICER). These results highlight the importance of accounting for evolving drug prices in long-term health economic models.
