The Italian Medicines Agency, by means of Decision No. 816 of 15 June 2026, has approved the ranking for the independent research call on rare diseases for the year 2025, funded through resources provided for in the Consolidated Law on Rare Diseases (Law 175/2021).

A total of 19 projects have been selected, with a total funding equal to €17,554,208, intended to support independent studies in a sector characterised by high clinical need and limited opportunities for therapeutic development. Rare diseases represent one of the main challenges in research, both due to the complexity of the research pathways and the difficulty in attracting investment in areas involving a limited number of patients.

“Promoting and supporting independent research is an integral part of AIFA’s mission; it was the first European regulatory agency to include this among its institutional objectives,” states AIFA President Robert Nisticò. “Rare diseases are one of the areas where public support for research can make the greatest difference. With this funding, we are supporting projects of high scientific quality that have the potential to expand existing knowledge and foster the development of new therapeutic opportunities for patients who often have limited or no treatment options.”

As set out in the call for proposals, the funded projects cover two lines of research: preclinical and clinical studies aimed at developing drug therapies for orphan diseases, including studies on the repurposing of existing medicines to investigate and support new therapeutic indications for rare diseases, and preclinical and clinical studies aimed at developing plasma-derived orphan drugs.

“Independent research is an essential tool for generating scientific evidence in areas that are often overlooked in traditional industrial development pathways”, says AIFA’s Technical and Scientific Director, Pierluigi Russo. “The approved projects address significant clinical needs and promote innovative approaches, including the repurposing of existing medicines and the development of new therapeutic strategies. This is an investment that strengthens the contribution of public research to innovation and the protection of health.”

The initiative confirms AIFA’s commitment to supporting independent research and promoting the development of scientific evidence in areas characterised by high clinical need, helping to expand treatment options for people with rare diseases. The AIFA Independent Research Call 2025, which addresses antimicrobial resistance and precision medicine, is also currently being validated.

“Managing resources allocated to independent research is an important responsibility for the Agency. The goal is to ensure that funding is allocated according to rigorous criteria of transparency and quality, helping to create the conditions for the best projects to translate into beneficial results for patients and the entire healthcare system,” says AIFA’s Administrative Director, Giovanni Pavesi.