Strengthening Global Ties: MDI Joins FSHD Europe, Advances Registry Work, and Supports Patient Voice in Access to Treatments
MDI has joined FSHD Europe, a key alliance supporting patients with facioscapulohumeral muscular dystrophy across the continent, aiming to improve diagnosis, care, and treatment access. We also recently took part in the TREAT-NMD Annual Curators Meeting, gaining valuable insights as we continue to develop a national neuromuscular registry for Ireland. Lastly, MDI highlights the role of patient organisations in Health Technology Assessments (HTAs) — a crucial step in securing access to new treatments. We may seek patient input for upcoming HTA submissions, so stay tuned for future opportunities to share your voice.
FSHD Europe
MDI is delighted to become a member of FSHD Europe. FSHD Europe unites and amplifies the voice of FSHD patients across Europe, raising awareness and interest among all stakeholders, including researchers, clinicians, pharmaceutical companies, regulators, payors, and national patient organisations. Its mission is to improve the quality of life for all patients and their families, and facilitate the easy diagnosis of FSHD, and enable all patients have access to the best treatment. We look forward to working more closely with our counterparts in Europe, sharing resources and knowledge in line with the organisation’s objectives.
TREAT-NMD Registry
Dympna recently attended TREAT-NMD Annual Curators meeting. This meeting brings together members of TREAT-NMD Global Registry Network, a federated network of individual, independent, national (or regional) patient registries that collect data on neuromuscular patients from more than 40 countries worldwide. As MDI continues to work on developing a national registry for Ireland, attending this meeting was invaluable for learning from other established registries and gaining a deeper understanding of the importance and value of a registry at both the national and international level.
Patient Organisation Involvement in National Health Technology Assessment
In Ireland the Health Service Executive (HSE) is responsible for decisions regarding the reimbursement of new drugs. Once a medicine has been approved at a European level by the EMA (European Medicines Agency), the pharmaceutical company that makes the medicine must then make separate applications for the drug to be considered for reimbursement at a national level in each EU or EEA country. In Ireland, applications are made to the HSE Corporate Pharmaceutical Unit (CPU) which then commissions the National Centre for Pharmacoeconomics (NCPE) to carry out an assessment of that medicine.
Many medicines, especially those for rare conditions, usually require a Health Technology Assessment (HTA). An HTA is an assessment of the clinical and cost-effectiveness of a medicine. During this process, input from patients and patient organisations is often sought, and sharing the lived experience can have a significant impact on the assessment outcome.
As we await the outcome of several treatments currently undergoing EMA assessment, we may reach out for patient testimonials in the future to support an HTA application for their reimbursement in Ireland. Please stay tuned to our website and social media for any updates or calls for participation.