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Your Tag: Regulatory

ERDERA Accelerator webinar (1)

ERDERA launches Ethics & Regulatory webinar series with introductory webinar on ethics across the research continuum

ERDERA’s ethics and regulatory experts are launching a dedicated webinar series to help the rare disease research community navigate two fast-moving areas of biomedical research: Advanced Therapy Medicinal Products (ATMPs) and Artificial Intelligence.
webinar series ERDERA

Introductory webinar: Situating ethics in the research continuum

July 24 @ 3:00 pm - 4:00 pm
This webinar will present a theoretical framework for situating ethics across research activities. The framework will then be brought to life through a relevant case study discussion, giving participants a concrete reference point for the episodes ahead.
ECRD 2026

ECRD 2026 in Prague strengthens the push for a coordinated EU Action Plan on rare diseases

On 3–4 June, EURORDIS–Rare Diseases Europe and Orphanet convened the rare disease community at ECRD 2026 in Prague around a shared call for coordinated European action, including the forthcoming European Blueprint for Rare Diseases.
BMC Medical Ethics

Ethics and regulatory guidance to address the challenges encountered in research on rare diseases in a multinational dimension: the experience of a European funded programme

This BMC Medical Ethics article examines ethics and regulatory guidance across EJP RD-funded rare disease research projects.
Female scientist in protective gear using lab equipment for research.. Photo by ima Miroshnichenko via Pexels (CC0)

Biotech Act II: European Commission opens Call for Evidence on industrial biotechnology and biomanufacturing

The European Commission has opened a Call for Evidence on the upcoming Biotech Act II — with feedback open until 10 June 2026, inviting stakeholders to submit input that will help shape the initiative before a legislative proposal is prepared.
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EMA launches consultation on proof-of-concept data in paediatric oncology

This initiative is also highly relevant from a rare diseases perspective, as paediatric cancers are rare conditions where small patient populations make robust non‑clinical proof‑of‑concept data essential for responsible and ethical trial initiation.
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Towards animal-free testing: virtual control groups gain regulatory acceptance

For rare diseases, where research often relies on limited animal models and small development teams, the regulatory acceptance of virtual control groups could help streamline early non‑clinical studies while maintaining a strong focus on patient safety.
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.
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