UK orphan medicine designation to be implemented
The UK has designed an independent replacement for present system for officially designating a medicine as ‘orphan’, i.e. for rare … read more »
EC publishes report on orphan medicine regulation
The European Commission (EC) has published its evaluation of the regulations over 2000-2017 (medicines for rare diseases) and 2007-2017 (medicines … read more »
EURORDIS urges protective measures as lockdowns lift
EURORDIS, the European Rare Diseases organisation, has issued a statement explaining the measures needed to protect patients of rare diseases … read more »
Landmark pay-for-performance contract agreed in Germany
The Gesellschaft für Wirtschaftlichkeit und Qualität für Krankenkassen (GWQ) has announced it has reached a deal to supply Zolgensma through … read more »
ANVISA: post-registration clarification for rare diseases
ANVISA (the Agência Nacional de Vigilância Sanitária – National Health Surveillance Agency) has clarified the procedure for post-registration changes to … read more »
Rare Diseases Ireland publishes guide for action
Rare Diseases Ireland (RDI) has launched its guide to rare diseases in Ireland to assist in policymaking around these conditions. … read more »
UK strategy for rare diseases: 2020 update published
The 2020 update to the Government’s strategy for rare diseases has been published, explaining progress made to date and the … read more »
DHSC launches surveys to understand rare disease community
The Department of Health and Social Care has begun its ‘national conversation’ on rare diseases by developing surveys to understand … read more »
Brexit could harm rare disease research and collaboration
A patient organisation has written an open letter to the UK Health Secretary Matt Hancock and Brexit Minister Michael Gove … read more »
Welsh annual report 2018 on rare diseases
The 2018 Welsh annual report summarises the progress and achievements against the Welsh implementation plan for rare diseases. The key … read more »
