Intelligent ecosystem to improve
the governance, the sharing,

and the re-use of health data for rare cancers

IDEA4RC launches its Community of Interest starting from patients

Since the inception of IDEA4RC, the involvement of a large and diverse group of stakeholders has been deemed crucial for the success of the project. Values and expectations of people who can benefit from the IDEA4RC data ecosystem have been considered through focus groups and interviews, but now, as the platform begins to take shape, stakeholders can be actively engaged in its development.

This is why IDEA4RC is establishing a Community of Interest, which in mid-June started welcoming its first members among the patients’ representatives of EURACAN, the European Reference Network (ERN) devoted to rare adult solid cancers. In the future, the Community of Interests will involve other professional categories, such as healthcare providers, scientific societies, healthcare and data protection authorities, pharma and IT companies.

To kick-start the Community, EURACAN patients’ representatives were invited to take part in a survey developed by Alleanza Contro il Cancro, IDEA4RC partner in charge of the stakeholder engagement work. The survey is intended to understand how patient advocacy groups can contribute to shape IDEA4RC data ecosystem and what is the best way to elicit such contributions. The survey is introduced by a summary of the project developed specifically for patients, which you can find in the project website page dedicated to the Community of Interest. In the same page, you can join the Community by filling the registration form.

We spoke with two experts about the role of patients’ representatives in clinical and basic research and how IDEA4RC plans to engage with them.

The first guest is Ariane Weinman, member of the Public Affairs team of EURORDIS, an alliance of more than 1000 rare disease patient organisations from 74 countries. Weinman is supporting the European Patient Advocacy Groups (ePAGs) in the four ERNs related to rare cancers.

Read more about EURACAN and ePAGS

EURACAN is one of the 24 European Reference Networks (ERNs), established by the European Commission to promote cross-border collaboration among centres of expertise specialised on rare, low-prevalence, and complex diseases.

Rare cancers are targeted by four among the 24 ERNs, one of them being EURACAN, devoted to rare adult solid tumours. IDEA4RC is focused on two among the 10 rare tumour families which are represented in EURACAN: head and neck cancers and soft tissue sarcomas.

ePAGS were constituted by EURORDIS together with the entire rare disease community to make the voice of patients heard within the different ERNs.

The second guest is Lidia Villanova, project manager at Alleanza Contro il Cancro who is leading the activities related to the IDEA4RC Community of Interest.

Ariane, what kind of role are patients’ advocates playing within EURACAN and ERNs in general?

ERNs are clinical networks whose main goal is to facilitate the access to cross-border care for people suffering from rare and complex diseases. Due to the rarity and complexity of these conditions, medical experts are scarce and scattered across Europe. Getting in touch with and being treated by them physically is extremely challenging, also given the difficulty that people suffering from debilitating conditions must face to move to another country. ERNs were conceived to offer virtual access to highly specialised care across Europe and also to facilitate knowledge sharing among experts in different countries. The final goal is for every patient to receive a timely diagnosis and adequate treatments.

In order to engage the patient organizations in the network and act as ambassadors between EURACAN and the wider patient community, EURORDIS has involved strong national and European patient organizations who were already very knowledgeable on European policies and who can be the voice of the patients within the network and also liaise with the wider patient community to explain what the network EURACAN is doing for them.

EURACAN, as well as all the other ERNs, are equipped with a Clinical Patient Management System provided by the Commission, which allows doctors to share in a secure way the clinical records of their patients with other experts to consult with them virtually.

How rare cancer patients’ engagement in ERNs has evolved in the last few years?

Rare cancer patients have always been very knowledgeable about their condition. Since it’s difficult to find the right experts and treatments are less available than for more frequent cancers, patients do a lot of searches, they engage with medical doctors to know more, to learn more. They can also reach out to established patient organisations in their country or to European federations. Over the last ten or twenty years, these patient organisations as well as other types of organisations have developed training courses for patients and caregivers, and for their representatives. Trainings can be for beginners or more advanced and concern different topics such as the drug development process, how clinical trials are designed and conducted, and how health technology assessment is performed. I would say that the people are in demand to be well informed in general. EURACAN also offers training opportunities to patients with the aim of making their contribution as impactful as possible.

What do patients expect from being involved in research initiatives?

A survey we conducted in 2018 among rare disease patients, thus not only rare cancers patients, showed that their primary motivation to get involved in research is to find treatment options, for example by joining clinical trials. Given the scarcity of treatment options for rare diseases, enrolling in a clinical study is often the only chance to be treated.

However, the same survey revealed that rare disease patients are well aware that among the obstacles to rare disease research there is not only the lack of funding and the small dimension of the patient population, but also the lack of collaboration and data sharing between different groups of researchers. This is a field where EURACAN is putting effort, by building a rare cancer registry.

What kind of contribution have patients given to the set-up of the EURACAN registry?

The work on the EURACAN registry was pioneered by the STARTER project and then continued by the BlueBerry project and both these projects involved patients all along their path. Patient representatives were involved in defining several aspects of the registry governance, they have a seat in the steering committee and also in the data access committee. Patients are convinced that the registry is a solid basis to advance research on rare cancers and thus improve patient outcomes in the future.

Lidia, why did you decide to start from patient representatives in building the IDEA4RC Community of Interest?

Patients are the ultimate beneficiaries of the research studies IDEA4RC wishes to make possible, and patient organisations will be among the users of the data ecosystem. Therefore, we want to make sure that their voices are heard, and their values and expectations considered from the onset.

Currently, we are defining the governance model of the platform, and we believe that patients should have the first say in such discussion. Who should have access to a health data ecosystem for rare cancers? Should users be asked for a financial contribution to access the data? How can we ensure that IDEA4RC data ecosystem is useful to researchers, while protecting patients’ personal data? Who should evaluate and approve data access requests to conduct scientific research, and how? All these questions encompass ethical aspects, such as the equity in the access to high-quality care, the economic value of health data and the balance we should find between private and public actors.

With patients as well as with the other stakeholders invited to the Community, we would like to establish an open dialogue that may be enriching on both sides. Members of our Community can benefit from the IDEA4RC data ecosystem while providing valuable feedback and recommendations within their areas of expertise, through a co-creation approach.

What are the objectives of the survey offered to EURACAN patient representatives?

Our primary goal is to raise awareness of the project within the community of patient advocates, starting from EURACAN. We also aim to encourage patient representatives to advocate for a cross-border data space for rare cancers, which is the overarching goal of IDEA4RC. Currently, the attention of policymakers on health data and its potential for improving care and research is very high, especially with the recent adoption of the Regulation establishing the European Health Data Space (EHDS). The coming years will be crucial to ensure that the implementation of the EHDS by Member States meets its initial objectives, hence facilitating cross-border care and collaboration among researchers in Europe.

Through the survey, we also aim to gather initial inputs on IDEA4RC governance, potential users, and economic sustainability. Additionally, we would like to know their preferences about communication and engagement channels, as well as areas of interest.

What are the next steps you plan to take once the survey results will be available?

The survey outputs will help us tailor the content and channels of the engagement activities addressed to patient representatives within the Community of Interest. As a project manager for other European initiatives focused on cancer, I have witnessed the invaluable contributions that patient representatives can make. Their perspective often harmonises a personal experience as a patient or caregiver with robust scientific, legal, and policy-related technical skills, thus providing a great added value. This is why I look forward to collaborating with them on IDEA4RC.