Stelios Kympouropoulos is a psychiatrist, a politician and a social activist. He has been a Member of the European Parliament and a Member of the Group of the European People's Party since 2019. He is a Member of the Committee on Employment and Social Affairs, the Delegation for relations with the Mashreq countries and the Delegation to the Parliamentary Assembly of the Union for the Mediterranean. In addition, he is a Substitute Member of the Committee on Regional Development and of the Subcommittee on Human Rights. He is also a Member of the Disability Intergroup of the European Parliament. He is also a Member and co-Founder of the Independent Living Organisation of Greece - i-living, the first organisation that promotes independent living in Greece.
Over the past decade, newborn screening programs throughout Europe have seen significant advancements, both in the number of countries adopting them and the variety of disorders they screen for. Despite this progress, considerable disparities remain between countries, with the number of screened conditions varying widely. This session will explore the reasons behind these inequalities and discuss the advantages of achieving more equitable access to newborn screening across Europe for both patients and society.
-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?
This session will explore the evolving landscape of Joint Clinical Assessments (JCAs) and their potential impact on equitable access to orphan drugs in Europe. Participants will delve into key lessons learned from ongoing JCAs, examining how evidence frameworks, PICO development, and expert involvement could be shaping clinical evaluations in the future. The discussion will highlight both the new opportunities and risks that JCAs present for orphan medicines. Thought a multi-stakeholder discussion, strategies for overcoming potential challenges will be proposed, including the early identification and simulation of PICOs and approaches for aligning evidence generation with JCA and national HTA requirements. Attendees will gain actionable insights into maximizing access and positive outcomes for patients with rare diseases in the context of rapidly evolving clinical assessment landscape