Produced by the Global Coalition on Aging and the Global Rare Disease Policy Network, this report examines rare disease policy across 16 countries, focusing on diagnostics and screening, treatment access and reimbursement, and national policy and governance. It draws on literature review, policy analysis and stakeholder interviews.
The assessment identifies diagnostic delays, workforce shortages, fragmented care and uneven access to therapies. It considers how longer survival and population ageing affect care needs, while acknowledging limitations in cross-country comparability and evidence on rare diseases in older populations.
The report discusses the European Rare Diseases Research Alliance (ERDERA) within European research collaboration and the country profiles for France and Spain. It also references the International Rare Diseases Research Consortium (IRDiRC) in its policy timeline and discussion of international research participation. Daria Julkowska and Holm Graessner are among the acknowledged stakeholder interview contributors.
The report was developed in partnership by the two issuing organisations, with development costs funded by Biogen. Acknowledged interview contributors participated on an unpaid basis.
