Rare diseases: 100 projects submitted for AIFA's independent research call

One hundred research projects have been submitted to AIFA under the call for independent research into rare diseases. The call, which closed on July 23, allocates a total funding of €17.8 million for research aimed at developing therapies for conditions with no specific treatment.

"One hundred projects is a significant achievement, testifying to the vitality and high level of Italian research," emphasized Robert Nisticò, President of the Italian Medicines Agency. "Globally, there has been a significant decline in nonprofit studies, including our country, likely due to the difficulty in raising the necessary resources," he added. "In this context, the Agency wanted to send a signal by strengthening its commitment to supporting independent research in less marketable areas, such as rare diseases."

The proposals received by the Agency come mainly from hospitals, healthcare companies and university polyclinics (41), universities (20), IRCCS (18), IRCCS/hospital foundations (10). Northern Italy is the most represented area, with Lombardy in the lead, followed by Lazio, Emilia-Romagna, Piedmont, Tuscany, Veneto, Friuli-Venezia Giulia, Puglia, Liguria, Campania, Sicily, Umbria, Marche, Molise, Sardinia and Trentino-Alto Adige.

In the coming weeks, the study proposals will undergo a validation phase by AIFA's Independent Research Office to verify their compliance with the call for proposals. A committee will then be established to evaluate the projects.

"It's necessary to continue along this path and make the publication of calls for proposals a regular feature," Nisticò stated. "An annual event to allow researchers to plan their projects and encourage the development of effective therapies for rare diseases, improving patients' health and quality of life."