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 »
FDA releases draft guidance on natural history studies for rare diseases
The US Food and Drug Administration (FDA) has released its proposed guidance to industry, aiming to assist companies in the … read more »
Parliament debates NICE appraisals of rare disease treatments
Last Thursday MPs debated a motion that this House believes that NICE appraisal processes do not properly address the medical … read more »
DHSC publishes 2019 update for rare disease strategy plan
The Department of Health and Social Care (DHSC) has published its 2019 update for the UK strategy for rare diseases. … read more »
