India, Ireland, and Malta Advance Rare Disease Policies and Treatment Access
India convened a national conference on rare diseases, Irish lawmakers debated Skyclarys access, and Malta unveiled its first rare disease strategy. New measures include expanded Centres of Excellence, ₹50 lakh assistance, and givinostat reimbursement.
Governments in India, Ireland, and Malta have taken new steps to address rare diseases. India’s Ministry of Health and Family Welfare convened a two-day National Conference on Rare Diseases in New Delhi on 5–6 May 2026, Irish lawmakers debated access to the Friedreich’s ataxia drug Skyclarys in the Dáil, and Malta’s Minister for Health and Active Ageing announced the country’s first National Strategy for Rare Diseases.
Opening the conference, the Union Health Secretary said the core objective is to understand challenges faced by stakeholders, encourage innovations, and generate new ideas for strengthening the management of rare diseases. She noted that the need for addressing rare diseases was first highlighted in the National Health Policy 2017 and institutionalized through the National Policy for Rare Diseases 2021. The policy is implemented through Centres of Excellence (CoEs), which expanded from 8 to 15, including two in North-East India. Financial assistance under the policy has been enhanced to ₹50 lakh per patient. The government has also exempted life-saving drugs from basic customs duty, with further expansion announced in the recent Union Budget. Approximately 1,800 patients have already received treatment support under the rare disease policy.
The Secretary of the Department of Health Research emphasised that India should develop context-specific models for diagnosis, prevention, and treatment rather than relying solely on approaches developed in higher-income countries. Digital technologies, including artificial intelligence and social media platforms, are being explored to support earlier diagnosis and public awareness. Research institutions are working to expand domestic treatment options, including indigenous development of therapies, collaboration with industry partners, and clinical evaluations through Centres of Excellence. Work is underway to identify more affordable alternatives to expensive medicines and explore repurposed drugs. Officials also highlighted ongoing work with the Department of Biotechnology and the Council of Scientific and Industrial Research to advance gene therapy research; CAR-T cell therapy was cited as demonstrating India’s expanding biomedical capabilities.
In Ireland, a Dáil debate on rare diseases focused on access to Skyclarys, a medication approved by the European Medicines Agency in 2024 for Friedreich’s ataxia, a rare inherited neurological condition that causes progressive damage to the nervous system and loss of muscle function. A Mayo TD said the condition continues to worsen as time passes, and another TD noted that over 200 people in Ireland live with the condition. Skyclarys is already available in several countries including the UK, France, Germany and Austria, while patients in Ireland continue to wait for a funding decision. The Minister for Health said the Government is examining new pathways for earlier access to orphan drugs and carrying out a comprehensive review of Ireland’s medicines approval and reimbursement system. No decision has yet been made on the reimbursement of Skyclarys; the application remains under active consideration by the HSE. The debate also heard that the drug givinostat has been approved for reimbursement for Duchenne muscular dystrophy following a recommendation by the HSE’s drugs group.
In Malta, the Minister for Health and Active Ageing announced that the Ministry has developed the first National Strategy for Rare Diseases, which will soon be launched for public consultation. He noted that while each rare disease affects a small number of people, collectively these conditions impact approximately 30,000 people in Malta. He explained that 80% of rare conditions are genetic and often require highly specialized care. The Minister highlighted Malta’s role in European Reference Networks, through which Maltese clinicians can consult with leading experts on complex cases. Digital eHealth services, including the exchange of patient summaries and electronic prescriptions, strengthen continuity of care outside Malta. The government continues to invest in national capacity, early diagnosis, professional training, and research.