Wim Goettsch, PhD, is Special Advisor in Health Technology Assessment (HTA) at the Dutch National Health Care Institute (ZIN). In his position at ZIN, he is a Member of the EU HTAR Coordination Group and its subgroup on Joint Scientific Consultation (JSC). Between 2010 and 2013, he was the Deputy Secretary of the Medicinal Products Reimbursement Committee at Dutch National Health Care Institute. He was the Director of the EUnetHTA JA3 (2016-2020) Directorate and Chair of the Executive Board of EUnetHTA between June 2016 and March 2018. Since December 2022, he also has a position as a Professor and Chair on HTA of pharmaceuticals at Utrecht University (NL), where he has been leading an H2020 consortium with fifteen partners around Europe called HTx, new methods for Health Technology Assessment (2019-2024). Since 2024, he is also the coordinator of a new Horizon Europe Project called SUSTAIN-HTA, an EU-wide initiative to build a supporting infrastructure to ensure ongoing implementation of the latest and fit-for-purpose HTA methodologies and tools in practice. He has more than 150 publications in peer-reviewed international journals.
This workshop will explore innovative approaches to cross-sector partnerships in the EU rare disease landscape. Participants will examine successful case studies that highlight innovative collaborations in rare disease research and diagnostics, showcasing how diverse stakeholders can work together to overcome challenges. The session will highlight partnerships that have demonstrated significant impact across the European rare disease ecosystem. By fostering knowledge exchange and promoting innovative strategies, this workshop aims to inspire and equip attendees with actionable insights to enhance multi-sector collaboration and ultimately improve outcomes for rare disease patients across Europe.
-What advancements have been made towards AI, RWE and scientific breakthroughs in rare diseases? e.g. Patient behaviour and trial recruitment, data collection, digital health, mRNA & other platforms, wearables devices etc.
-Are these technologies being used and do they add significant value to the healthcare ecosystem. E.g., to improve the diagnosis pathway, access to care, clinician workload and impact on healthcare system for rare diseases
-Are regulators, HTAs and payers supportive of these advancements, does it help with their decision-making process?