National Conference On Rare Diseases Marks 10 Years Of Collective Advocacy In Malta

The National Alliance for Rare Diseases Support – Malta, in collaboration with the Ministry for Health and Active Ageing and the University of Malta, hosted its annual national conference on rare diseases, bringing together patients, healthcare professionals, researchers, policymakers, and international experts to discuss key priorities affecting the rare disease community.

This year’s conference holds special significance as it marks the 10-year anniversary of the National Alliance for Rare Diseases Support – Malta, celebrating a decade of unified advocacy, patient support, and collaboration aimed at improving the lives of people living with rare conditions.

The conference theme, Together Through the Uncommon, reflects the collective effort required to address the complex medical, social, and policy challenges faced by individuals and families affected by rare diseases.

The event featured keynote contributions from national and international leaders in the field, including the Chair of Rare Diseases International, highlighting Malta’s active engagement within the global rare disease community. Discussions throughout the day focused on newborn screening and early diagnosis, equal access to care and treatment, cross-border healthcare, and the importance of holistic support that extends beyond clinical care to education, social inclusion, and quality of life.

Addressing the conference, Founder and President of the National Alliance for Rare Diseases Support – Malta, Michelle Muscat, emphasised that the conference comes at a pivotal moment for the rare disease community in Malta. She welcomed the growing national attention being given to rare diseases and expressed hope that ongoing discussions may lead to strengthened investment, structured policy development, and the launch of a national framework that responds to the real needs of patients and families.

“Any move towards a comprehensive national policy discussion document would represent an important step forward,” she noted. “What matters most is that this process remains inclusive, evidence-based, and shaped by the lived experiences of patients, alongside the expertise of healthcare professionals and researchers.”

Over the past decade, the Alliance has played a central role in uniting patient organisations, families, clinicians, and researchers under one umbrella, strengthening representation and awareness of rare diseases in Malta. Key achievements include collaboration with national and international stakeholders, the promotion of registries and directories for rare conditions, and continuous one-to-one support for patients navigating complex and often uncertain diagnostic and treatment journeys.

Despite progress, significant challenges remain. Diagnostic delays, limited treatment options, and the need for stronger integration with European Reference Networks and cross-border healthcare pathways continue to affect patients in small Member States such as Malta.

The conference therefore provided an essential platform for dialogue between patients, policymakers, and professionals on how to translate awareness into concrete, sustainable action.

The presence of international experts further underscores Malta’s commitment to aligning with European and global developments in rare disease policy, research, and care. By fostering collaboration across sectors and borders, the conference aims to ensure that no person living with a rare disease in Malta feels isolated or overlooked.

Concluding her address, Michelle Muscat reiterated the Alliance’s readiness to work in close partnership with national authorities and stakeholders to advance a comprehensive, patient-centred approach to rare diseases.

She stressed that meaningful progress will depend on sustained political commitment, coordinated services, and active patient involvement in policy development.

The conference stands as a strong reminder that while each rare disease is uncommon, the collective voice of the community is powerful. Through continued collaboration, Malta can ensure that rarity never equates to invisibility and that every patient receives the recognition, care, and support they deserve.

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