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Tag: Research Design

ERDERA First Policy Think Tank meeting

ERDERA Policy Think Tank meets in Paris to advance data and registry policy for rare diseases

Bringing together policy makers, researchers and data experts to align on how registry data can better support rare disease research and evidence‑based policy across Europe.
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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.
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Women’s Day highlights widening gender inequalities in rare diseases

Drawing on Europe-wide patient and carer surveys, ERDERA highlights how gender can shape diagnostic delays, care burden and the evidence base for rare-disease research.
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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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.
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Clinical experts and patient advocates gather in Heidelberg to turn discussion into action on global rare disease research

From shared standards to local impact, the 2nd Clinical Research Networks Conference agreed concrete next steps for interoperable clinical research networks worldwide
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One month to go: register now for the 2nd International Conference on Clinical Research Networks

9-10 December, Heidelberg and online — last places for in person attendance; free livestream for registered participants.
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EMA publishes new guidelines for non-interventional studies

New EMA ICH M14 guidelines sets common international standards for non‑interventional real‑world data studies to strengthen medicine safety assessment.
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Shaping the future of global rare disease research: Join the 2nd International Conference on Clinical Research Networks

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