Projects

Combining diagnostic data and interventional approaches for futureproof cardiology care 

COMBINE will close the gaps in the existing workflow for the treatment of Coronary Artery Disease patients. We propose an end-to-end workflow that will unlock the full potential of Coronary Computed Tomography Angiography (CCTA) to improve hospital efficiency and patient outcomes. AI-powered CCTA algorithms will enable definitive ischemia diagnosis, accurate patient stratification and efficient planning of the interventions, as well as patient specific follow-up. While our focus on CAD is justified by the large patient population, the CCTA-enabled diagnostic accuracy and seamless workflow, backed by the clinical evidence generated in five multicentre clinical trials, will be directly transferable to other clinical domains using CT.

Gene Therapy for treatment of rare inherited Arrhythmogenic Cardiomyopathy 

The GEREMY consortium will use a novel parallel approach leveraging the transformative potential of gene therapy (GT) to deliver the first cardiac GT for treatment of rare inherited forms of ACM (desmosomal and non-desmosomal). Our approach also involves development of new preclinical models, validation of novel delivery vehicles and gene editing approaches, and the revolutionary development of pig models for in vivo GT validation.

Within the GEREMY project, we will develop GTs for PLN- and PKP2-mutation induced ACM. These will serve as models for other inherited heart diseases and contribute directly to the portfolio of treatments for rare cardiac diseases. Social embedding of all stakeholders will ensure value sensitive design and an innovative/modernised clinical trial design will contribute towards expedited timelines and increased probability of clinical development success. A business strategy will be established with commercialisation partners to market the selected GT approach; ultimately lowering mortality rates and reducing disease burden and healthcare costs.

The importance of patient engagement in Health Technology Assessment (HTA) is today widely recognised, and patients and patient organisations will play a vital role in the implementation of the new EU framework for HTA Regulation (EU 2021/2282).

To facilitate this, patients will require access to training, education and information on HTA and the ways in which they can be involved. Over the past 10 years, The European Patients’ Academy on Therapeutic Innovation (EUPATI) has become a key multistakeholder actor for patient engagement in Europe, with an extensive track record in patient education in medicines research and development (R&D).

EUPATI has trained over 200 patient experts (EUPATI Fellows), 450 online learners via EUPATI Open Classroom and 500 professionals (through EUPATI Fundamentals). EUPATI has become the ‘go to’ portal for independent, user-friendly information on medicines R&D (6 million users of the Toolbox in 14 languages), along with becoming the main point of contact at national level through its network of 24 National Platforms across Europe.

In this project EUPATI will:

1. Update EUPATI’s existing patient training content on HTA aligning with the new Regulation
2. Design an e-learning training course for patients on HTA and the specific requirements for the new Regulation
3. Develop interactive online training sessions (in 5 languages) preparing participants to take part in joint clinical assessments and joint scientific consultations with national HTA bodies
4. Develop and implement sustainability measures to ensure the continuity of the project outputs into the future.

The new HTA Regulation represents a unique opportunity to enhance the role of patients and patient representatives in HTA processes. In this respect, this project is in full alignment with EUPATI’s mission to further patient engagement in medicines R&D through education.

Since the beginning of 2022, EUPATI has been a part of the 360° HTA Patient Involvement project, together with EPF and HTAi.

The 360° HTA Patient Involvement project is funded by the European Federation of Pharmaceutical Industries and Associations aims to research the current methods and processes for patient involvement in HTA in Europe and analyse how they are perceived by the different stakeholders. Expected outcomes of this project include a 360° review of these methods and processes and a list of best practices. This research might be further expanded to other regions.

EUPATI’s role in the project is contributing to the survey on the role of patient engagement in HTA and gather responses. For this, EUPATI is working closely with EUPATI Fellows  contributing to and shaping the development of the project.

The project is coming to its end by December 2023, and the final report and recommendations are now available. Link to the final document: https://htai.org/wp-content/uploads/2023/10/360-HTAi-PCIG-Research-in-Patient-Involvement-Practices-in-HTA-in-Europe-Oct-2023-1.pdf

A lot of good practices exist, but there is lots of room for improvement. This report shows that we can learn from the great work already being done and embed this more widely. It also points to the need for flexibility in processes and for a constant cycle of learning. Covering European practices, this report comes at a timely moment when the implementation of the HTA Regulation in Europe is underway.

More information on the project here.

8.8 million euros for accelerated drug repurposing for rare neurological disorders

SIMPATHIC Consortium receives grant from European Commission.

The SIMPATHIC Consortium, led by the Dutch Radboud University Medical Center and Amsterdam University Medical Centers, has developed a new approach to expedite the use of existing drugs for groups of patients with rare neurological disorders. The consortium has been awarded an 8.8 million euros grant from the Horizon Europe program to further develop this innovative method.

Traditionally, drugs are developed one disease at a time, which is costly and time-consuming. It often takes a long time before patients can use a new drug. The international SIMPATHIC Consortium, which comprises 22 international partners, has created a novel method for accelerating the use of existing drugs for other conditions, based on screening tissues from individual patients. The consortium’s approach has been recognized and funded by the European Commission with a 8.8 million euros grant.

The researchers will use a new technology to test the efficacy of existing drugs in patients with neurological disorders, requiring only a tube of blood or a small piece of skin from the patient. These materials contain stem cells that the researchers culture into nerve cells. They subsequently test how they respond to a variety of existing drugs.

If the researchers observe a positive effect of a drug, they will launch a clinical study in a group of patients with different disorders but similar clinical symptoms. As existing drugs have already been tested in humans, animal studies may not be necessary, which significantly accelerates the use of drugs in new applications and reduces research costs.

Project leader and bioinformatician Prof. Peter-Bram ‘t Hoen of Radboudumc explains, “The project will significantly improve the efficiency and speed of discovery, evaluation, and approval of new applications for existing drugs.”

Project leader and pediatrician-biochemical geneticist Prof. Clara van Karnebeek of Amsterdam UMC adds: “In addition, it will have a huge impact on the care of patients with rare neurological disorders, for example, by accelerating drug availability for these debilitating conditions.”

The SIMPATHIC Consortium comprises 22 partners, including research teams from Radboudumc, Amsterdam UMC, and other academic centers in Europe and Canada, European patient organizations, companies, and a European infrastructure network. The consortium’s innovative approach is expected to revolutionize drug development.

More information here. 

EUPATI Reload was an EIT Health funded project (2020-2021) focusing on the re-packaging of the EUPATI Patient Expert Training Programme to make it available on-demand by patients, patient representatives and other stakeholders. For more information, please see here.

EFOEUPATI stands for Ensuring the Future of EUPATI. It was an IMI-EFOEUPATI project launched on 1 September 2018 that ran for 25 months, until end of September 2020.

Ensuring the Future Of EUPATI aimed to ensure optimal exploitation and sustainability of the core achievements of the IMI-EUPATI Project 2012-2017: Patient Expert Training Course, the multilingual public Toolbox, and the EUPATI National Platforms Network. It is imperative that these pillars exist in the medium- and long-term to address the growing field of patient engagement. The project was co-lead by the European Patients’ Forum (EPF) and Bayer. For more information, please see here.

More information on the project here

PARADIGM was a public-private partnership project co-led by the European Patients’ Forum and EFPIA (2018-2020). PARADIGM’s mission was 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. The objective was to develop much needed processes and tools for three key decision-making points: research priority setting, design of clinical trials and early dialogue. Building on advances at international level, PARADIGM sought to integrate the needs, perspectives and expectations of all actors (including vulnerable populations) involved and produce a set of metrics to measure the impact of patient engagement. Project website: https://imi-paradigm.eu/

EUPATI collaborated with PARADIGM on sharing the developed tools for patient engagement via its Toolbox and on organising the Patient Engagement Open Forum.

EUPATI was involved (in 2022) in the ‘Framework for clinical trial participants data reutilisation for a fully transparent and ethical ecosystem project’ (FACILITATE). It explores whether the GDPR has limited Europe’s capabilities in innovative drug development and looks for ways to allow patients’ data to be accessed, used and reused.

Started in 2022, the project is conducted by a consortium of 27 partners from 17 Member States including patient associations, hospitals, universities, subject matter experts and members of the European Federation of Pharmaceutical Industries and Associations (EFPIA). The project is set to end in 2025.

As of 2023, EUPATI continues its involvement in the project through the Italian EUPATI National Platform. More information is available at the project website: https://facilitate-project.eu/