The initiative, dedicated to funding clinical studies on rare diseases, will include active patient organization involvement at every stage of research.
Fondazione Telethon will oversee the funding of the new international call launched by ERDERA, the European rare disease research alliance bringing together over 170 organizations across 37 countries. The initiative will make up to €30 million available to support early-phase clinical studies for rare diseases for which therapeutic options are limited or unavailable.
The call will be open to international consortia consisting of at least three partners from three different countries. Patient organizations will be systematically involved and will actively contribute to the design, conduct, and dissemination of study results. This approach is considered essential to ensure both scientific quality and the real-world feasibility of clinical trials in the field of rare diseases.
Eligible projects will include Phase I, I/II, and II clinical trials across a range of therapeutic modalities, from small molecules to advanced therapies, provided they are supported by robust preclinical or preliminary data and a well-defined clinical development pathway.
The entire evaluation process will be managed by Fondazione Telethon through a centralized model aimed at overcoming the fragmentation typical of national projects and facilitating the sharing of expertise, data, and resources among clinical centers in different countries.
“Being selected to coordinate this call is a source of great pride for the Foundation: it represents international recognition of our expertise and our role as a catalyst for the development of therapies for rare and ultra-rare diseases. This is further confirmed by the UK Ministry of Research and Innovation’s decision to entrust the Foundation with the management of its financial contribution to the initiative,” commented Graciana Diez-Roux, Scientific Director of Fondazione Telethon.
The selection process, which is long and complex, will last over one year and is expected to conclude in February 2028. Full details of the call and application procedures are available on the ERDERA website.
