ERN input to ACT-EU priorities- Survey!

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ACT-EU* – Accelerating Clinical Trials in the EU

Please contribute to ACT-EU survey

Target group: Academic Stakeholders

The aim of the survey is to identify needs and priorities of academic stakeholders involved in clinical trials, integrating them into the work of ACT EU. It seeks to address challenges in conducting clinical trials in the EU faced by ERNs and academics, highlight topics needing further clarification or discussion, and propose solutions for improving and accelerating clinical trials. Academic stakeholders are encouraged to contribute ideas for improvement across various aspects of clinical trial governance and implementation, clinical trial methodologies [...]


Patient Partnership Webinar – Unlocking ERN Potential: Disease Specific Measures

Eurordis webinar 24 july

The increasing recognition and diagnosis of rare diseases over the past decade have led to a growing body of knowledge within specialised clinical teams and centers. This expansion is evident in the European Reference Networks (ERN), which connect over 1,600 clinical teams from more than 300 specialised hospitals in the EU/EEA region.

The challenge now is to transform this collective expertise into real-world evidence that can enhance diagnosis, care, and treatment. The key lies in developing disease-specific outcome measures that reflect the effectiveness of care and treatment for each rare condition. These measures provide a common framework for experts to [...]


Approaches to the Evaluation of Evidence for Rare Disease and Complex Conditions Guidelines


It is well acknowledged that a different approach is needed to evaluate new Orphan Medicines compared to standard medicines and vaccines, due to the small population numbers that are impacted by rare diseases.

With limited published evidence available, how can we adopt an enhanced approach to reviewing all the available information on new rare disease medicines in order to support clinical decision making?

This webinar will bring together experts and methodologists experienced in successfully navigating the underlying issues. They will propose solutions for developing effective guidelines for the evaluation of evidence to support clinical decision making for people living with [...]


ERICA WP4 Webinar: c4c expert advice, patient and public involvement, data standards


ERICA WP4 Clinical Trial Support webinar

Friday, May 3rd From 13:00 to 14:00 CEST


Conect4children” (c4c) is a IMI2 project and a Dutch non-profit that provides services to academia and industry that accelerate the design and conduct of paediatric clinical research. This webinar introduces two of the services: expert advice about the design of paediatric trials, including the voice of children, young people, and data standards. We invite you to learn about how these services can support your research, adapt your work to the paediatric population, and how you can [...]


ERICA GA & ERN Research Conference


The ERICA 4th General Assembly & ERN Research Conference will take place from December 11th to December 13th, 2024 in Udine, Italy.

Hosted by MetabERN.

The aim of the ERICA consortium, in which all 24 European Reference Networks (ERNs) take part, is to build on the strength of the individual ERNs and create a platform that integrates all ERNs research and innovation capacity. ERICA Conference serves as a great opportunity to ‘meet and greet’ the active ERN research community, to present the research projects involving ERNs and to [...]


World Orphan Drug Congress Europe 2024 in Barcelona


The World Orphan Drug Congress Europe is the largest and most established orphan drug & rare disease event worldwide. Meet over 2000 attendees, hear from 250 leading speakers, and connect with 130 exhibitors as we bring together experts from the start-to-finish of orphan drugs. From regulation and policy, to global pricing and gene therapy.

If you are a member of an ERN, patient group organization, charity or government (including public health bodies, HTAs, regulators), you are eligible for a free VIP pass. Apply now:

Date & Location

22 October 2024 | Pre-Congress Workshops | Hotel Catalonia Barcelona Plaza


ERICA WP4 Webinar: c4c trials, education and training


ERICA WP4 Clinical Trial Support webinar

Thursday, April 11th From 13:00 to 14:00 CEST


Conect4children” (c4c) is a IMI2 project and a Dutch non-profit that provides services to academia and industry that accelerate the design and conduct of paediatric clinical research. This webinar introduces two of the services: support for clinical trials and education & training.
We invite you to learn about how these services can support your research and how you can inform the implementation of c4c for rare disease research.

Mark Turner, c4c

Chloe Bickerstaff,c4c

Francesca Rocchi, [...]


EMA/ACT EU Training: Transitioning trials to the CTR (CTIS) for non-commercial sponsors

ACT EU Training

ACT EU Training for non-commercial sponsors: Transitioning trials to CTIS

9 February 2024 10:00-13:00 CET

Location: Online/ European Medicines Agency, Amsterdam, the Netherlands, Live Broadcast

Event summary

This training event aims to support non-commercial sponsors of clinical trials in transitioning their clinical trials that are expected to continue after 30 January 2025 from the Clinical Trials Directive to the EU Clinical Trials Regulation (Regulation (EU) No 536/2014). Moreover, the event will provide useful training in preparing new clinical trials following the Clinical Trial Regulation (CTR).

From 31 January 2025 onwards only the CTR and its Delegated Acts will [...]


EMA Webinar on Rare World Diseases Day

EMA RD Day webinar

Orphan Medicines Development – ask the European regulator

Thursday, 29 February 2024 Virtual meeting via WebEx / EMA, Amsterdam

The EMA’s Orphan Medicines Office is hosting an interactive webinar on Rare Disease Day on 29 February 2024, to answer questions that developers of products for rare diseases or researchers in this field might have on orphan designations and rare disease development. The webinar will feature:

short presentations on the background of orphan designation and the benefits it has brought to patients; a live question-and-answer session enabling participants to ask their questions on orphan medicines development to a panel of regulatory [...]