Category: Clinical Research

Publicaciones (1)

EU rare disease action plan- European Added Value Assessment

This study investigates possible measures that could be taken at EU level to address these challenges. It finds significant European added value in harmonising coordination and access across the 27 Member States, mainly in terms of improved diagnostic tools and availability of medical treatment, better health outcomes, particularly lower infant mortality, and improved well-being of family members and caregivers.
Rare Disease Day 2026 visual representation

One year of ERDERA: the European alliance built to accelerate rare disease research reports first results

28 February, across Europe and beyond: one year into delivery, ERDERA is advancing towards shorten diagnostic journeys and improved therapies for people living with a rare disease.
the power behind erdera2 (1)

Undiagnosed Day 2026 | Where Diagnosis Becomes Hope: The Power of Collaboration and Technology in Rare Diseases

This meeting will focus on practical approaches to phenotyping and diagnosis in undiagnosed conditions, including how to define next steps when a diagnosis remains uncertain, and how to strengthen pathways and collaboration around undiagnosed care.
Genetic Mapping

ERDERA unites 10 000 rare disease datasets in a single, secure European resource

In its first year, ERDERA has brought together 10 000 harmonised genomic and phenotypic datasets from unsolved rare disease cases across Europe, creating a secure, standardised and scalable resource.
Medicines

EMA starts review of Tavneos for rare autoimmune diseases GPA and MPA

The European Medicines Agency has begun a formal review of Tavneos (avacopan) after emerging information raised questions about the integrity of key clinical trial data supporting its EU authorisation, with potential implications for adults living with rare autoimmune vasculitis.
Young Patients Training

ERDERA opens call for young rare disease advocates to join training in Paris

Taking place on 23–25 April at Institut Imagine in Paris
Scientific paper

Human genomics technologies in clinical studies – the research landscape: report on the 1990–2024 period

Published on 14 December, this WHO technical document maps global trends in registered clinical studies using human genomic technologies from 1990 to 2024, including patterns of inclusion and equity.
EMA

Webinar on the use of platform technologies in the non-clinical and clinical domains

dna-8895875_1280

New WHO report highlights gaps in human genomics research

Greater efforts are needed to invest in infrastructure, foster inclusive study designs and align research agendas.
European Medicines Agency (EMA)

ICH opens consultation on draft E22 guidance for patient preference studies

A draft ICH E22 guideline on patient preference studies is now open for public comment via the European Medicines Agency until 12 April 2026.
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