Lidewij Vat, PhD, works at Synergist, where she works for the Patient-Focused Medicines Development (PFMD) initiative and the From Testing to Targeted Treatments (FT3) consortium, leading the Patient Engagement and Patient Experience Data (PE&PED) workstream. With over 15 years of experience in the healthcare sector, she contributed to a range of national and international multi-stakeholder initiatives focused on advancing patient engagement in health research and medicines development, including Canada’s Strategy for Patient-Oriented Research (SPOR) initiative and IMI-PARADIGM. She holds a PhD from Vrije Universiteit Amsterdam, focusing on embedding patient engagement in health research and decision-making and a Master’s in Management, Policy Analysis and Entrepreneurship in Health and Life Sciences.
-Europe’s first JCAs, which examine how well new medicines work and how well they’re tolerated, compared with existing treatment options, have now been published. This provides an initial opportunity to examine how rare disease patient and caregiver perspectives are incorporated into the assessments. However, while patient voices are present in the reports, is there evidence that they influence the assessment itself?
In this working session, participants will:-
-Examine what the published rare disease JCAs reveal about the transparency and impact of patient and caregiver involvement
-Assess what "meaningful impact" looks like for patients involved in JCA decision-making during a roundtable discussion with our expert clinician and patient representative speakers
-Co-create recommendations to support meaningful representation of patient and caregiver perspectives in Europe’s JCA process for rare diseases and paediatric conditions