Pharmaceutical industry pledges to ‘leave no patients behind’ in rare diseases
An infographic developed by Farmaindustria has been published describing what the last twenty years in research and development for orphan … read more »
BIA launches report with PwC on rare medicines appraisal
The Bioindustry Association’s (BIA) Rare Disease Industry Group has launched a report A Rare Chance for Reform in collaboration with … read more »
EMA to host workshop on regulatory support for development of orphan medicines
The European Medicines Agency (EMA) is hosting a workshop on 30 November to discuss the benefits and impact of early … read more »
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 »
