A new publication from the International Rare Diseases Research Consortium, “Rare disease preparedness: the time is now“, published in Rare Disease and Orphan Drugs Journal, calls for urgent, coordinated global action to transform how rare diseases are diagnosed, treated, and prioritized within healthcare systems worldwide.
Drawing lessons from the COVID-19 pandemic, the publication argues that the same level of political urgency, scientific collaboration, regulatory flexibility, and investment that accelerated pandemic responses must now be applied to rare diseases.
Although individually uncommon, rare diseases collectively affect an estimated 300–400 million people globally. Yet despite their scale and impact, approximately 95% of rare diseases still have no approved treatment.
Read the full publication, available open access: https://www.oaepublish.com/articles/rdodj.2025.39?utm_campaign=website&utm_medium=email&utm_source=sendgrid.com