The president of the Rete Civica group, Elena Ugolini, brings the Associations’ complaint to the Regional Council: “It is not a question of costs, but of putting the patient at the centre”
Three years after the approval of Resolution 1415/2022, home enzyme replacement therapy remains inaccessible for the majority of patients in Emilia-Romagna. On October 28, the matter will be discussed in aula thanks to the question presented by councilor Elena Ugolinipresident of the Rete Civica group and candidate for President of the Emilia Romagna Region in 2024, today an institutional reference voice for associations that ask for healthcare that is closer to the needs of people with rare diseases.
The document, filed on August 8, born from one joint letter from the associations AIGlico (Italian Glycogenosis Association), AIAF (Italian Anderson-Fabry Association), AIMPS (Italian Mucopolysaccharidosis Association) and AIG (Italian Gaucher Association)delivered to the regional health councilor Massimo Fabi on 21 July. It is from that solicitation that the question takes shape: a political initiative that collects and relaunches the requests of the associations, which have so far remained unanswered.
In the letter, the associations ask for theurgent activation of home therapy for lysosomal metabolic diseaseshighlighting how the regional route is “extremely complex and effectively impassable”. They denounce the inadequacy of the ADI (Integrated Home Care), often lacking sufficient staff and incompatible with the duration of the infusions, and underline the paradox of a Region which, despite having formally implemented the AIFA recommendations, does not guarantee their application.
“Many children could avoid missing school, and adults would no longer have to give up work to receive therapy. It is a question of equity and proximity of care”, says Ugolini.
In the text, the councilor questions the Council on three key points:
- if you intend to adopt concrete measures to guarantee the home administration of enzyme replacement therapy (ERT), in line with the provisions of Resolution 1415/2022;
- if she is willing to evaluate the use of public or private tools, including free services offered by some pharmaceutical companies in other Regions;
- if you deem it sustainable model that responds to patient needs without a significant increase in healthcare spending, overcoming current organizational barriers.
“It’s not a question of inventing new routes,” continues the group leader, “but of making those already authorized accessible. If other Regions do it, it is not clear why Emilia-Romagna should lag behind”.
The regulatory basis exists, both at national and regional level, but remains largely theoretical. AIFA authorized the home administration of enzyme replacement therapies with a first decision dated 30 March 2020, then confirmed and extended on 9 May 2023. Among the active ingredients explicitly included are the laronidasi (mucopolysaccharidosis type I), theRecombinant acid alpha-glucosidase (Pompe disease) and alpha flashlight (lysosomal acid lipase deficiency).
At the regional level, Resolution no. 1415 of 29 August 2022 approved a technical document that defines the path for the administration of ERT in different care settings, including home care. But that path, as the associations highlight and as the interpellation reiterates, never became operational.
“Home therapy is not a luxury,” concludes Ugolini. “It is a concrete response to real needs, which today are not listened to. And this, for public health, is unacceptable. For this reason, the session on 28 October will be an opportunity to understand whether the Region finally intends to unlock the implementation of home therapy. The associations are waiting for answers and the patients are waiting for a therapy that does not force them to choose between health, school and work”.
