Tag: Regulatory Affairs

abstract publication image on science health

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.
World Orphan Drug Congress USA 2026

World Orphan Drug Congress USA 2026

More than 280 speakers from industry, regulators, patient organisations, investors and healthcare providers, and aims to showcase new science, technologies and policies that can accelerate orphan drug development and improve access to therapies for people living with rare conditions.
generic publication image

Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval

With over 300 million people affected by rare diseases, timely access to effective therapies is critical. Despite strong alignment in regulatory expectations, patients in many regions face years of delays highlighting the need for improved international coordination.
EDERA WODC & GAM - Preview- JVDPhotography-1-min

ERDERA at WODC Europe 2025: wrap up and next steps

Sharing patient‑centred methods, early diagnostics, and data‑driven trial innovation to accelerate rare disease research across Europe.
Training online

Information Webinar – ERDERA Joint Transnational Call 2026

This information webinar on the 2026 Joint Transnational Call for Proposals is scheduled for 16 December 2025, 15:00–17:00 CET, focusing on resolving unsolved rare genetic and non‑genetic diseases.
Ai Exhibition

EMA opens public consultations on patient experience data and use of external controls

EMA invites feedback to strengthen how the EU generates and uses evidence that reflects what matters to patients, including in rare diseases
Exterior of European Medicines Agency in Amsterdam

EMA concludes pilot to support repurposing of established medicines

Accelerating evidence-driven innovation to unlock fresh indications for established therapies.
XVII Foresight Training Course

XVII Foresight Training Course | From research to access: how does Europe speed up the availability of medicines for unmet needs?

The course will focus on the current status and advancements of clinical research especially to address unmet medical needs. Innovative methodologies, regulatory challenges, and the involvement of adolescents in clinical trials will be discussed.
Magda Cheblus

“The notion that public-private collaboration is inherently problematic doesn’t align with current realities. Not only is it possible—it is necessary, beneficial, and more straightforward than many assume.”

ERDERA's

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