The AIFA 2025 call for proposals for independent research on rare diseases has been successfully concluded. By the deadline of 23 July, 100 projects had been submitted, for a total funding of €17.8 million.

The proposals come primarily from hospitals, healthcare facilities, and university hospitals (41), universities (20), IRCCS (18), IRCCS/hospital foundations (10), other entities such as institutes and networks (5), foundations (4), and IRCCS/university hospitals (2). Northern Italy is the most represented region, with Lombardy leading the way, followed by Lazio, Emilia-Romagna, Piedmont, Tuscany, Veneto, Friuli-Venezia Giulia, Puglia, Liguria, Campania, Sicily, Umbria, Marche, Molise, Sardinia, and Trentino-Alto Adige.

 “The first AIFA call for proposals dedicated exclusively to rare diseases closed with a round figure: 100 projects represent a great result, which testifies to the vitality and high level of Italian research, which responded with great interest,” emphasises Robert Nisticò, president of the Italian Medicines Agency. “Globally, there has been a significant decline in non-profit studies, which has not spared our country, probably due to the difficulty in finding the necessary resources. In this context, the Agency wanted to send a signal by strengthening its commitment to supporting independent research in areas that are less attractive to the market, such as rare diseases: it is necessary to continue along this path and make the publication of calls for proposals structural. An annual event to allow researchers to plan their projects and encourage the development of effective therapies for rare diseases, improving the health and quality of life of patients.”

The 100 study proposals will undergo a validation phase by the AIFA Independent Research Office to verify compliance with the requirements of the call for proposals. A commission will then be set up to evaluate the candidate projects. The call for proposals focuses on two lines of investigation: preclinical and clinical studies aimed at developing drug therapies for diseases without specific treatments, including projects involving the repositioning of existing medicines to investigate new therapeutic indications for rare diseases, and preclinical and clinical studies aimed at developing plasma-derived orphan drugs. Strict evaluation and monitoring mechanisms have been put in place with the aim of facilitating the transfer of results from the laboratory to clinical practice.