Past and current projects

EURORDIS has a long history of participation in key EU-level projects relevant to rare diseases and orphan medicines

Current projects


SCREEN4CARE is an international consortium that aims to tackle the major hurdle for rare disease patients – the lengthy and convoluted diagnosis journey – via an innovative research approach based on two central pillars: genetic newborn screening and artificial intelligence (AI)-based tools. The project launched in October 2021 and will run for a period of five years with a total budget of EUR 25 million provided by the Innovative Medicines Initiative (IMI 2 JU), a joint undertaking of the European Union and the European Federation of Pharmaceutical Industries and Associations (EFPIA). The role of EURORDIS in this project is substantial, as we bring expert multi-stakeholder perspectives and ensures patient engagement.

Learn more about the project

PARADIGM (Patients Active in Research and Dialogues for an Improved Generation of Medicines)

There is increasing consensus among stakeholders that patient engagement at different points of the medicines lifecycle is critical to fostering patient access to innovative therapeutic solutions, and delivering better health outcomes for patients.

PARADIGM was a public-private partnership, co-led by the European Patients’ Forum and EFPIA, whose mission is to provide a unique framework that enables structured, effective, meaningful, ethical, innovative, and sustainable patient engagement (PE) and demonstrates the ‘return on the engagement’ for all players.

Building on advances at international level, PARADIGM integrated the needs, perspectives and expectations of all stakeholders (including vulnerable populations) involved to co-produce a set of tools and recommendations toplan, conduct and evaluate patient engagement in medicines development in a meaningful and sustainable manner. The toolbox includes a monitoring and evaluation framework and metrics to measure the impact of patient engagement for all stakeholders involved in medicines development.

EURORDIS was a PARADIGM partner and leader of the work package developing a sustainability roadmap to optimise patient engagement. 

The PARADIGM partnership has developed a series of tools that all stakeholders involved in the development of medicines - including patient organisations -  can use to facilitate patient engagement:



RARE IMPACT is a multi-stakeholder consortium working to improve patients’ access to gene and cell therapies (advanced therapies).  EURORDIS chairs the initiative with the support of Dolon Consulting.  Through the engagement with HTA agencies, regulatory bodies, payers, patient groups, clinicians, manufacturers and other experts across Europe the consortium, RARE IMPACT partners aim to identify the challenges and will propose the solutions for a better access to advanced therapies in Europe.



C4C (Conect 4 Children)

c4c (conect4children) is a large collaborative European network that aims to facilitate the development of new drugs and other therapies for the entire paediatric population. EURORDIS is a member of the C4C consortium.




HTx is a Horizon 2020 project focussing on the framework for the Next Generation Health Technology Assessment (HTA) to support patient-centred, societally oriented, real-time decision-making on access to and reimbursement for health technologies throughout Europe. EURORDIS is a partner of HTx, ensuring the patient voice is heard in the development of the project. HTx will receive input from experts as well as a direct involvement of stakeholders in the progress of the project. Their objectives are facilitating the development of methodologies to deliver customised information of health technologies, providing methods to support personalised treatment advice, and leading the eventual implementation of these methods.


Past involvement


EURORDIS participates in the European Clinical Research Infrastructures Network (ECRIN),  a sustainable, infrastructure supporting multinational clinical research projects in Europe.


The five-year European Patients' Academy on Therapeutic Innovation (EUPATI) project (2012-2017) led by the European Patients' Forum (EFP) and funded by the Innovative Medicines Initiative (IMI), is educating patients on therapeutic innovation. EURORDIS is actively contributing to the pedagogic content, face-to-face training and e-learning.


EURORDIS led a Work Package focusing on developing and managing supranational biobanks in the Network of Excellence for rare inherited neuromuscular diseases, Treat-NMD.


EURORDIS co-created and ensured the coordination of the European Network of DNA, Cell and Tissue Banks for Rare Diseases (EuroBioBank) and continues to be involved in network activities.


The European Network for the Advancement of Clinical Gene Transfer and Therapy (CLINIGENE) was developed to integrate multidisciplinary research and development in gene therapy. EURORDIS played an advisory role, exchanging information on the potentials of gene therapy.

Page created: 07/08/2013
Page last updated: 19/10/2021
The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseasesRare Disease International Bringing together patients, families and experts to share experiences in a moderated multi-language forum. RareConnect The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases