Please note: the hybrid event is open in person only to invited people. The event is open for all online.

Event Highlights:

• Dates: Monday, May 27 – Tuesday, May 28, 2024
• Venue: Hotel Excelsior Bari, Via G. Petroni, 15, Bari / Puglia, Italy
• Registration deadline: March 31st, 2024 – Registration is open here.

Agenda Coming Soon: Stay tuned as we prepare to unveil the detailed agenda, promising a comprehensive exploration of the impact of EJP RD.

More information on the event here.

Registration is open here (early bird deadline: January 31st, 2024)

Don’t miss the unique opportunity to be part of a transformative event that is paving the way for advancements, collaborations, and hope in the realm of rare diseases organized by Rare Diseases Greece (RDG), member of EURORDIS-Rare Diseases Europe and Boussias Events following the World Rare Disease Day 2024.

Under the theme “Navigating the Uncharted: Advancements, Collaborations, and Hope in Rare Diseases”, the International Congress on Rare Diseases and Orphan Drugs aspires to stimulate dialogue and cooperation across all stakeholders – patient representatives, policymakers, clinicians, researchers, industry, payers and regulators – towards advancing a comprehensive rare disease strategy that promotes sustainability and equity within healthcare ecosystems.

Rare Disease Moonshot’s mission: Scaling up public-private partnerships to accelerate research by enhancing the translational research ecosystem, optimising clinical trials and regulatory pathways and develop infrastructure to accelerate the journey to diagnosis and treatment.

The paper is available here.

This open-access publication, reflecting the collaborative efforts of the IRDiRC Drug Repurposing Task Force and the IRDiRC Therapies Scientific Committee over the past 1.5 years, is a significant milestone in advancing drug repurposing for rare diseases.

The paper delves into key insights gained from analyzing cases of drug repurposing in small population conditions. Lessons learned include the importance of sharing clinical observations, early engagement with regulatory scientific advice, and fostering public-private collaboration.

IHI – call 6
IHI – call 6 is a two-stage call for proposals with the following topics:

• Topic 1: Support healthcare system resilience through a focus on persistency in the treatment of chronic diseases
• Topic 2: Development of evidence based practical guidance for sponsors on the use of real-world data / real-world evidence

Deadline for short proposals: 16 April 2024 at 17:00 Brussels time
Deadline for full proposals: 10 October 2024 at 17:00 Brussels time
Call web page: https://europa.eu/!RjXdcH

IHI – call 7
IHI – call 7 is a single-stage call for proposals with the following topics:

• Topic 1: Improving clinical management of heart disease from early detection to treatment
• Topic 2: User-centric technologies and optimised hospital workflows for a sustainable healthcare workforce
• Topic 3: Clinical validation of biomarkers for diagnosis, monitoring disease progression and treatment response

Deadline for full proposals: 22 May 2024 at 17:00 Brussels time.
Call web page: https://europa.eu/!64yx8Y

Find out more
All topic texts, call documentation (including a comprehensive guide for applicants), details of how to apply and guidance on how to find project partners can be found on the IHI website.

Info sessions and networking
The IHI Call Days for calls 6 & 7 are running from 10 January to 25 January 2024 inclusive. They include info sessions on the topics, our rules & procedures, and preparing the financial parts of the proposal, as well as pitching sessions on the topics. Participants can also use the Call Days website to identify and meet potential project partners. The recordings of these info sessions are also available online. Find out more: https://europa.eu/!RTJc7m

Why apply?
IHI calls for proposals represent an excellent opportunity for researchers to take part in ground-breaking cross-sector collaborative projects that aim to deliver tangible benefits for patients. Scientists from academia, diverse industry sectors, SMEs, mid-sized companies, hospitals and patients’ organisations are encouraged to get involved in our projects.

Pre-proposals must be submitted no later than 12:00h C.E.T. on March 5, 2024, via the electronic submission tool.

Funding: The total made available for this call is about 18 million euros.

Call text, participating countries and more information here.

The European Partnership for Personalised Medicine (EP PerMed), supported by the European Union under Horizon Europe (Grant Agreement N° 101137129), launches its first joint transnational call (JTC2024) for proposals on “Identification or Validation of Targets for Personalised Medicine Approaches (PMTargets)”. In total, 39 funding organisations participate in this call with an available budget of over 45 Mio€.

Timeline, contact, and more information here.

The European Joint Programme on Rare Diseases is organizing the training webinar: “Real-World data, Machine learning and Deep analytics in rare diseases: Regulatory grade data collection for marketing authorization submissions – what is buzz, what is realistic?” that will be held online on 26 January 2024 from 2:00pm to 4:00pm CET.

Registration is open here before 25 January 2024 at 23:45 p.m

Marc Van Dijk (UCB) and Luis Pinheiro (EMA) will present views of industry and regulators on various facets of collecting, processing, and using data for research, regulatory and healthcare decisions. The two keynote talks will provide an overview of patient needs and challenges in rare disease R&D and of data and technologies that offer solutions to those challenges. 

The panel discussion between representatives of patients, academia, regulatory scientists, industry and clinicians and with the audience will provide different perspectives on challenges and solutions in the context of regulatory realities and imperatives. They will also provide examples of solutions already used and their limitations.

More information here.

Virginie brings an extensive professional background in rare diseases, scientific research, and patient advocacy, combined with personal insights from her niece’s experience with Congenital Disorders of Glycosylation.

EJP RD is proud to have collaborated with her at EJP RD, and excited to see her drive positive change in the rare disease community!

“I am privileged to re-join EURORDIS as CEO. My goal will be to build on both my professional experience and personal insights in rare disease research and patient advocacy to further EURORDIS’s mission, ensuring that the voices of all people living with a rare disease in Europe are heard and their needs are met,” Virginie stated.

Yann Le Cam, outgoing Chief Executive Officer of EURORDIS, expressed his full confidence in Bros-Facer’s leadership:

“I am thrilled to pass the baton to Virginie. Her unique blend of professional expertise and personal commitment to rare diseases makes her the ideal leader to guide EURORDIS into the future.”

Source: EURORDIS