Tag: Regulatory Affairs

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.
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.
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

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.
Pharmaceutical products

EU institutions agree next steps on reform of pharmaceutical legislation

Following the 11 December 2025 political agreement, EU institutions are moving towards formal adoption of a new Directive and Regulation that will reshape how medicines—including orphan and paediatric medicines—are developed, authorised and supplied across the European Union.
Task-Forces-IRDiRC

IRDiRC launches call for experts for four new rare disease activities

Rare disease researchers, clinicians, patient advocates and data experts are invited to help shape four new IRDiRC activities linked to the new IRDiRC Roadmap, with applications closing on 20 February 2026.
Publication cover image

ICH E22 Guideline on general considerations for patient preference studies

Open for consultation from 12 December to 12 April, this draft ICH guideline sets out how to design and submit robust patient preference studies to inform regulatory decision‑making across a medicine’s lifecycle.
Non-human primate hand via pickpik.com (CC0)

EMA public consultation on non‑human primate use in safety testing

EMA seeks stakeholder input on new reflection paper to reduce and replace non‑human primate use in medicines safety testing through 3Rs approaches.
Molecular therapy

Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation

Outside traditional drug development pathways, clinicians and scientists face the challenge of systematically evaluating whether individual patients with severe ultrarare diseases might be eligible for and potentially benefit from individualized mutation-specific RNA therapies.
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