On the occasion of World Rare Diseases Day, the councilor denounced the delays in the implementation of home therapy and the lack of involvement of associations
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Regional councilor Elena Ugolini brought attention to the delays which, in Emilia-Romagna, continue to prevent activation of home enzyme replacement therapy (ERT) for people with lysosomal metabolic diseases. The note released in those days highlights how, despite a regulatory framework defined for yearsthe route foreseen by the resolution 1415/2022 remains “effectively impracticable” and Integrated Home Assistance (ADI) is not able to guarantee the service.
A CLEAR REGULATORY PATH, BUT NEVER BECOME OPERATIONAL
The note reminds us that ERTs require 2-3 hour infusions, to be carried out every one or two weeks, and that home administration is already active in almost all Italian regions. Emilia-Romagna and Piedmont represent the only exceptions. According to what we read, For many families, this leads to school absences, continuous work permits and a significant impact on the quality of life.
“Why – asks Ugolini – do we have to hinder and make the lives of people who already have a difficult life even more complex? We cannot accept that what is foreseen and possible is in fact denied due to rigidity or ideological preconceptions”.
THE PROGRESS: FROM THE LETTER OF THE ASSOCIATIONS TO THE INTERPELLATION OF 28 OCTOBER
The situation had already been brought to the attention of the Legislative Assembly on October 28, 2025when Ugolini had presented ainterpellation born from a joint letter from the Italian Glycogenosis Association (AIGlico), the Italian Anderson-Fabry Association (AIAF), the Italian Gaucher Association (AIG) and the Italian Mucopolysaccharidosis Association (AIMPS)delivered to the regional health councilor Massimo Fabi on 21 July. The associations denounced an “extremely complex and effectively impassable” path, the inadequacy of the ADI and the failure to apply the AIFA recommendations, which from 2020 authorize the home administration of ERT.
During the meeting on 28 October, Councilor Fabi had recognized the need to make resolution 1415/2022 fully operational and had announced the convocation of a work table for 19 November 2025, with the involvement of the associations. It had also opened to possibility of using i Patient Support Programs (PSP) made available free of charge by pharmaceutical companies, already used in other regions.
THE DELAYS AND CONCERNS OF THE ASSOCIATIONS
As reported in the note, Despite the commitments made, the work of the technical group is proceeding “slowly” and the associations are expressing growing concern. A negative signal would also have come from the regional congress on rare diseases on 5 February 2026, dedicated to proximity pathways: none of the associations involved in the request for home therapy would have been invited to the public discussion.
“Resolution 1415/2022 – Ugolini strongly underlines – must be fully implemented and updated. The associations must be involved in the technical and decision-making tables. Patient Support Programs are a tool that is already available and safe: not activating them means leaving families alone”.
THE REQUESTS MADE TO THE REGION
The note calls for the immediate implementation of resolution 1415/2022, the full involvement of the associations in the technical tables and the activation of the PSPs to guarantee safe home infusions, with qualified healthcare personnel. According to what has been reported, the objective is to make an already recognized right enforceable, overcoming the organizational barriers that in recent years have prevented patients from accessing home therapy.
The four associations involved – AIGlico, AIAF, AIMPS e AIG – welcomed the renewed institutional attention, but underlined that the situation remains critical. In a shared position they highlight how “the distance between what is predicted on paper and what happens in reality continues to be unsustainable for families”. They recall that the therapy is authorized at a national and regional level, but that “without a clear operational model, without certain times and without the activation of the PSPs, the risk is that everything remains immobile”.
The associations also reiterate that healthcare proximity cannot depend on the availability of individual local services: “We need a simplified path and uniformapplicable in all provinces, which guarantees the presence of qualified nursing staff and which does not place improper responsibilities on caregivers. In almost all Italian regions there is the possibility of taking advantage of home care made available free of charge by some highly specialized companies, but the Emilia – Romagna region persists in not taking it into consideration, without however offering patients an alternative that guarantees the same standard of care quality”. In this context, they point out how an important step will be the new meeting scheduled with the regional technical staff for next March 20th, from which concrete indications are expected on the progress of the process and on the operational solutions to finally make home therapy accessiblehoping that the observations already made by the associations are truly taken into consideration.
