Prof Ivo de Blaauw, ERN eUROGEN Sub-Coordinator, spoke in the session “Achieving Full Reach: Overcoming The Last Challenges To Access Highly Specialised Care” on 16 May 2024 during the 12th European Conference on Rare Diseases and Orphan Products (ECRD).
Over 700 people registered for the conference, representing many different stakeholder groups, including over 370 patients, advocates, and patient organisations, over 160 ERN/Academics/Healthcare Professionals, over 120 industry representatives, and over 25 Payer bodies/ HTA agencies/regulators/policymakers. The positive feedback from conference participants attending both in-person and online was overwhelming, with one participant, in particular, describing the conference as “fruitful, instructive and productive” and another remarking on the “wind of change” that was felt throughout.
The primary policy objective of ECRD 2024 was to ensure that rare diseases remain a top priority for the upcoming European political leadership, as the Conference set a decisive platform to champion the integration of a comprehensive European Action Plan on rare diseases into the new EU legislative agenda, rooted in the recommendations of Rare 2030. Participants were able to make their own personal commitments to the future of rare disease policy and had the opportunity to sign the EURORDIS Open Letter to EU Institutions and Country Leaders.
The session “Achieving Full Reach: Overcoming The Last Challenges To Access Highly Specialised Care” emphasised that healthcare services should be organised as close to the population as possible to meet local needs best. This principle also applies to rare diseases, but due to the small number of patients, planning for these diseases is more efficient at a national or pan-regional (European) level where there are enough cases to understand their needs.
In most European countries, Expert Centres handle enough cases of the most prevalent rare diseases to develop local expertise. However, cross-border collaboration at a pan-European or international level is necessary for less frequent and lower prevalent diseases and rare, complex surgical interventions.
The session discussed the feasibility and benefits of enhanced pan-European cooperation to plan, fund and deliver highly specialised healthcare services through Expert Centres connected to national, European, and international networks. The goal is to ensure safe, accessible, and sustainable high-quality care for all.
Participants learned about unmet needs in cross-border care, the need for greater EU solidarity, and the components required to establish an EU system for commissioning highly specialised services.
The session was chaired by Enrique Terol (Permanent Representation of Spain to the EU, who previously led the ERN Team at DG SANTE at the European Commission) and was developed by EURORDIS (our Supporting Partner), Holm Graessner (one of the trio of chairs of the ERN Coordinators Group), and RaDiOrg – Rare Diseases Belgium.
