Horizon Europe rare disease partnership to launch first calls in December

A new Horizon Europe rare disease partnership will launch its first calls in December, as it embarks on a seven-year mission to consolidate research and improve treatment.

The rationale for a joint European effort is that there are 7,000 different rare diseases, each with very few patients in every country. However, in total 30 million people in Europe are affected by a rare disease, for most of which there are currently no therapies.

“For the majority of rare diseases in one country you never have a critical mass that is needed to do the research on a specific disease. That's why strong collaboration is very much needed,” says Daria Julkowska, coordinator of the European Rare Diseases Research Alliance (ERDERA).

The partnership is rooted in the EU’s Horizon Europe research programme, from which the EU will contribute €150 million. Another €230 million will be provided directly by the participating countries, bringing the total estimated budget of the partnership to €380 million.

ERDERA builds on more than 20 years of previous EU efforts in rare disease research, including Horizon 2020’s European Joint Programme on Rare Diseases (EJP RD). They encompass funding for projects, dedicated support services, regulatory and ethics advice, and collaboration with patients and policymakers, says Julkowska.

The partnership’s goal is to create a functioning ecosystem to enable rare disease research to thrive in Europe. “The overall objective is not necessarily to focus on specific diseases or groups of diseases,” says Julkowska.

There are three main goals to address by 2031: accelerate time to diagnosis from an average of four years to six months; accelerate the development of treatments; and improve the lives of patients with rare diseases. 

How the partnership works

ERDERA has two workstreams: competitive funding calls and in-house research.

The competitive funding comes in three forms: transnational calls for projects; calls for clinical trials; and a networking support scheme for building rare disease research communities (Julkowska describes this as a mini–COST Action).

The first transnational funding calls will be announced in November, before a December launch. This is in line with previous rare disease research calls funded under EJP RD, which concluded in August 2024.

ERDERA is a co-funded, which means researchers will need one of the partnership’s member organisations in their country to co-fund their participation. Organisations that are partnership member will be listed with the call, but here’s a list of those that took part in the 2023 EJP RD call.

The wait for other ERDERA funding streams will be a little longer. The networking scheme is due to open in early 2025, while the calls on clinical trials won’t be announced until 2026. Here, the partners are giving themselves more time to set up the calls to address the bottlenecks that tend to impact the success rates clinical trials.

The inhouse research is more like a clinical research network, which brings together the technical expertise of research institutes and clinical expertise of hospitals and research organisations to advance diagnosis, therapeutics and clinical readiness.

These inhouse groups will pool knowledge on rare diseases and define patient outcomes to get rare disease groups to a point where they may be considered for later-stage research and trials.

The idea is to set up pilot projects that can then “be expanded to all types of rare diseases and demonstrat[e] that we have the capacity as a clinical research network to accelerate this clinical trial readiness of the different rare diseases or groups of rare diseases,” says Julkowska.

ERDERA will involve more than 170 organisations in 37 Horizon Europe countries. The members are research institutes, patients’ organisations, European research infrastructures, pharmaceutical and technology companies, public funding agencies, philanthropic and private funders, regulatory institutions, healthcare authorities, hospitals, universities and medical associations.