Europe Strengthens Rare Disease Research: New Opportunities for Lithuanian Researchers and Innovators
In June 2026, the conference Advancement of Treatments for Rare Diseases was held in Nicosia as part of the programme of the Cyprus Presidency of the Council of the European Union. It brought together representatives of European institutions, the research and clinical communities, patient organisations, and the pharmaceutical and biotechnology sectors. The discussions focused primarily on the development of new therapies, clinical trials, regulatory processes, biobanks, health data and patient access to treatment.
Although each rare disease affects a relatively small proportion of the population, rare diseases collectively affect between 27 and 36 million people in the European Union. Cooperation at EU level is particularly important, as knowledge, patient data and specialised expertise are dispersed across different countries and treatment centres.
From European Strategies to Clinical Trials
The conference highlighted that funding fundamental research alone is not sufficient to advance rare disease research. Scientific discoveries must be systematically translated into preclinical and clinical studies, while promising therapies must ultimately reach patients.
This ambition is reinforced by the European Commission’s Life Sciences Strategy, published in 2025, which aims to make Europe the world’s most attractive place for life sciences by 2030. The strategy also provides for an EU investment plan designed to facilitate funding for clinical trials conducted across several countries. The European Commission’s proposal for a European Biotech Act also seeks to simplify the regulatory environment for clinical trials and cell and gene therapies, while improving access to finance for start-ups and small and medium-sized enterprises.
Horizon Europe remains the EU’s principal research funding programme, with rare diseases identified as one of its health-related funding priorities. European Partnerships and the European Platform on Rare Disease Registration also play an important role in supporting research and cooperation by helping to reduce data fragmentation and creating better conditions for the use of data in scientific research.
ERDERA Brings Together the Rare Disease Research Community
One of the most important initiatives in this field is the European Rare Diseases Research Alliance, ERDERA. It brings together more than 170 public- and private-sector organisations from 37 countries. The Alliance’s total projected budget through 2031 amounts to approximately €380 million. ERDERA’s activities cover research funding, clinical trial readiness, the development of diagnostics and therapies, and the use of data, artificial intelligence and digital technologies.
Following the conference, on 1 July 2026, ERDERA launched a clinical trials call supporting multinational Phase I, Phase I–II and Phase II interventional studies of medicinal products for rare diseases. Up to €30 million has been allocated to the call. Eligible applicants include universities, research institutes, hospitals, clinical centres, non-profit organisations, patient organisations, and small and medium-sized enterprises. The mandatory Expression of Interest stage is open until 10 September 2026. Lithuanian institutions may participate as funded partners.
Lithuania’s Experience Presented at the Conference
Lithuanian expertise was represented at the conference by Birutė Tumienė, Coordinator of the Centres of Expertise at Vilnius University Hospital Santaros Klinikos. She presented a clinical model for interdisciplinary knowledge and innovation sharing aimed at supporting the development of therapies for rare diseases.
Giedrė Kvedaravičienė, Director of the Lithuanian Population and Rare Disease Biobank, discussed the role of national biobanks in population and rare disease research. Her presentation highlighted the importance of biobanks in collecting high-quality biological samples and data that are essential for advancing diagnostics and developing new treatment solutions.
What Does This Mean for Lithuanian Researchers and Innovators?
Lithuanian researchers, healthcare institutions, patient organisations, and small and medium-sized enterprises have new opportunities to join international consortia, participate in clinical trials and secure EU funding. It is particularly important to begin identifying partners and preparing project proposals before calls are officially launched.
The Research Council of Lithuania participates in European Partnerships, provides funding to Lithuanian partners involved in selected projects, and advises applicants and project beneficiaries. The Lithuanian RDI Liaison Office in Brussels, LINO, supports the Lithuanian research and innovation community in developing international cooperation, identifying project partners, and keeping track of relevant EU funding opportunities and policy initiatives.
Last updated: 30-07-2026
Related news:
RCL Launches Call for Personalised Medicine Research
RCL Launches a Call to Uncover the Layers of Vilnius Cathedral’s History
OECD and European Commission Invite Researchers to Take Part in a Survey on Research Careers
Chair of the RCL Expert Committee Prof. Vilmantė Borutaitė Receives a State Decoration