Patricia is Director of the Cost-Effectiveness Analysis (CEA) Registry within the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center. Her research advances health economics and outcomes research to improve health equity and fair access to medicines. Her work spans health technology assessment, systematic review, and economic evaluation, with an emphasis on generating evidence to inform value assessment, pricing, coverage, and reimbursement decisions. Dr. Synnott has led and managed a substantial portfolio of grant- and industry-funded research and is actively engaged in training and mentorship through the CEA Registry fellowship and Tufts educational programs in systematic review, epidemiology, and biostatistics. She also serves on the Board of Directors for the ISPOR Boston Chapter. Prior to Tufts, she held senior leadership roles at the Institute for Clinical and Economic Review (ICER), where she led evidence review activities supporting U.S.-focused value assessments, and worked internationally in development and public health through the U.S. Peace Corps and global nutrition and food security organizations. Dr. Synnott received her PhD in Clinical and Translational Science from Tufts University and holds an MS in nutrition and a MALD in international development and humanitarian assistance.
Despite regulatory incentives and strong scientific innovation, orphan medicines are still frequently launched late, or not at all, in the UK, Ireland and parts of the EU. Smaller populations, complex HTA requirements and uncertainty around evidence expectations can push these markets down the launch sequence. This panel will examine how developers can de‑risk access decisions earlier, by aligning evidence generation across regulators, HTA bodies, payers and patient organisations. Drawing on perspectives from patient advocacy, policy and industry, the discussion will focus on what really moves the needle, from early scientific advice and patient‑relevant endpoints through to managed access agreements and reimbursement decisions.