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 [...]
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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 [...]
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ERICA WP4 Clinical Trial Support webinar
Friday, May 3rd From 13:00 to 14:00 CEST
Speakers
“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 [...]
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The ERICA 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 discuss the future of [...]
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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: www.terrapinn.com/WODC/ERICA
Date & Location
22 October 2024 | Pre-Congress Workshops | Hotel Catalonia Barcelona PlazaRead
ERICA WP4 Clinical Trial Support webinar
Thursday, April 11th From 13:00 to 14:00 CEST
Speakers
“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, [...]
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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 [...]
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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 [...]
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The Joint Heads of Medicines Agencies (HMA)/European Medicines Agency (EMA) Multistakeholder workshop on Patient Registries will bring together representatives of registry holders, regulatory agencies, pharmaceutical companies, patients, healthcare professionals, academia, and health technology assessment bodies to address the following objectives:
Day 1 – 12 February afternoon: Discuss the
EMA qualification procedure for patient registries with the aim to clarify the benefits, identify current limitations, and propose measures to optimise the process; Day 2 – 13 February all day: Establish the value and enable the use of patient registries for regulatory decision-making by considering contexts of use for which registry [...]
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The European Conference on Rare Diseases & Orphan Products (ECRD) is the event that allows the dialogue and collaboration of over 1500 stakeholders in the rare disease community. The 12th edition coordinated by EURORDIS and co-organized by Orphanet will be celebrated next summer, from 15th to 16th May 2024.
The ECRD is the largest, patient-led, rare disease policy-shaping event held in Europe.
By bringing together people with rare diseases and patient advocates, policy makers, healthcare industry representatives, clinicians, regulators and Member State representatives, EURORDIS harnesses [...]
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