Emilia-Romagna Faces Scrutiny Over Delays in Home Therapy for Rare Diseases
Families battling rare metabolic lysosomal diseases in Italy’s Emilia-Romagna region are facing ongoing challenges in accessing vital enzyme replacement therapy (ERT) at home. Despite a regional decree (Delibera 1415/2022) intended to facilitate home-based treatment, implementation remains stalled, forcing patients to travel to hospitals for infusions – a significant disruption to their lives.
The Burden on Patients and Families
ERT requires regular infusions, typically lasting 2-3 hours, every 1-2 weeks. For many, hospital visits mean missed school days, frequent work absences and a substantial strain on family resources. Currently, almost all other Italian regions offer home therapy, making Emilia-Romagna and Piedmont outliers. Regional Councilor Elena Ugolini has been vocal about the issue, questioning why the region continues to impede access to a service readily available elsewhere.
A History of Advocacy and Unfulfilled Promises
The current situation stems from years of advocacy by patient associations. In July 2025, organizations including the Italian Glycogen Storage Disease Association (AIGlico), the Italian Anderson-Fabry Association (AIAF), the Italian Gaucher Association (AIG), and the Italian Mucopolysaccharidoses Association (AIMPS) jointly appealed to the regional health assessor, Massimo Fabi, highlighting the complex and often impossible path to accessing home therapy. They pointed to a lack of alignment with national guidelines from AIFA (the Italian Medicines Agency), which have authorized home administration of ERT since 2020.
Following an interpellance presented by Councilor Ugolini on October 28, 2025, Fabi acknowledged the need for full implementation of the 2022 decree and announced a working group to address the issue, with the inclusion of patient associations. However, progress has been unhurried, and concerns are mounting.
Missed Opportunities for Collaboration
A recent regional congress on rare diseases, held on February 5, 2026, further highlighted the lack of engagement with patient groups. None of the associations advocating for home therapy were invited to participate in the public discussion, raising questions about the region’s commitment to a collaborative approach.
Patient Support Programs Remain Untapped
A potential solution lies in utilizing Patient Support Programs (PSPs) offered by pharmaceutical companies, which are already successfully implemented in other regions. These programs provide specialized assistance with home infusions. However, Emilia-Romagna has yet to embrace this option, leaving families without a viable alternative.
Looking Ahead: A Crucial Meeting on the Horizon
A modern meeting with regional technical staff is scheduled for March 20, 2026. Patient associations are hoping for concrete indications on progress and operational solutions to finally make home therapy accessible. They emphasize the need for a simplified, uniform process applicable across all provinces, ensuring qualified nursing staff and avoiding undue burden on caregivers.
The associations stress that the existing decree must be fully implemented and updated, with patient organizations actively involved in technical and decision-making processes. They maintain that the gap between policy and reality remains “unsustainable” for families.
FAQ
Q: What is ERT?
A: Enzyme Replacement Therapy (ERT) is a treatment for lysosomal storage diseases, involving regular infusions to replace missing enzymes.
Q: What is Delibera 1415/2022?
A: A regional decree intended to enable home-based ERT for eligible patients in Emilia-Romagna.
Q: Why is home therapy important?
A: It reduces disruption to patients’ and families’ lives by eliminating the need for frequent hospital visits.
Q: What are PSPs?
A: Patient Support Programs offered by pharmaceutical companies to assist with the administration of specialized therapies at home.
Q: What is the current status of home therapy in Emilia-Romagna?
A: Implementation of the regional decree is delayed, and access to home therapy remains limited.
Did you understand? Nearly 2 million people in Italy live with rare diseases, often requiring complex and lifelong treatment.
Pro Tip: Patient advocacy groups can be valuable resources for information and support when navigating complex healthcare systems.
Have you or a loved one been affected by delays in accessing home therapy? Share your experience in the comments below.
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