CF Europe ensures that the patient’s perspective is included and considered in any future improved European Standards of Care. In addition to this, CF Europe supports its members to achieve access to the best possible care in their country by informing patients and professionals, by organising training, lobbying on a European level, and supporting lobbying on a national level.
VERA: Enhancing digital healthcare and access to health data for people with cystic fibrosis in Europe
We believe that people with CF should have access to their own data in order to manage their care and partner with their care team.
Our new initiative, VERA Europe, aims to improve the collection and accessibility of data by enhancing the capabilities of CF patient organisations in their own countries. We have called the initiative VERA, meaning ‘true’ in Spanish, Italian and Portuguese, to reflect our Europe-wide approach and our aim of showing what it’s really like to live with CF.
Why better data collection and accessibility are needed?
People with CF live with their condition and project manage their self-care at home for 360 days per year when they are not in a coordinated care context (their CF centre). Outside of this time, they have very little support and there are many aspects of care, health and adherence that are not measured and recorded. As with many other areas of our lives, digital advances have created an anticipation that digital health will support people with CF and their families better in this process.
As a result, patient organisations over time will have an increasing role to play providing information and education for patients on how they can use their data to project manage their self care and partner with their care teams.
There is also a challenge in ensuring equal access to new precision medicines. This is an unprecedented time for CF care, with new medications ushering in a new era of personalised healthcare. Ensuring that all persons with CF across all European countries have access to optimal treatments and care is a massive challenge that must be acknowledged and met at a European level through better data availability.
What data could be collected and what would be the benefits
Any data not collected in conventional randomised controlled trials could be collected and made accessible. For example, it could be observational data, predominantly carried out through patient reporting and or recording of their healthcare experience and it should include data validated in a clinical setting.
This data describes a patient’s health, wellbeing, care, and medications throughout the year. It has the potential to provide a deeper understanding by supporting and developing CF care guidelines, assisting care teams providing care to individuals with CF. It would also enable research into CF treatments and outcomes and new ways of designing CF clinical trials.
Benefits could also go beyond clinical measures. For example, collecting information on day-to-day needs, such as mobility (getting to the hospital), internet access to allow digitally connecting, adherence to treatment regimes.
Where CF Europe can make an impact
CFE is striving to provide CFE members the information and tools they need to harness the advantages and to adapt to changes presented by improved data collection.
Projects initiated through the VERA programme will be led by patient organisations in their own countries where CFE members wish to enhance their capabilities.
VERA will reflect best practices happening in many European countries today.
Data working for the patient
Of utmost importance to CF Europe is ensuring that data is being collected with the aim of improving care and access to treatments for people with CF. To this end, we are working with organisations that promote collaboration with an ecosystem approach in which interoperability, standardised protocols and open-source (basic) technology are all self-evident.
Most importantly, patients must have ownership of their own data to improve trust between people and organisations.
CF Europe is working with the support of the King Baudouin Foundation and is applying the 8 Principles of Caring Technology in Belgium.
Collaborating across disease areas to put patients in control of their health data
This is a crucial time for health non profit organisations to ensure that the framework for collecting and storing health data is robust and safe and that it facilitates access to optimal care and treatment, according to patients’ needs. This is particularly important for people with diseases like cystic fibrosis, who need to interact regularly with healthcare services.
For this reason, at the start of 2022, CF Europe initiated an active partnership with the European Haemophilia Consortium, the European Muscular Sclerosis Platform, and World Duchenne to highlight the needs of patients when collecting, storing and accessing health data.
Our aim is to ensure effective learning and decision making across disease areas to protect the rights of patients and enhance care. On the topic of data, we ´think as one´ and drive change together through strategic leadership and operational pilots for national and international data initiatives. Our shared vision is for patient organisations to have a clear ‘data role’. Specifically, we believe patient organisations should:
1) Provide education tools to improve healthcare and data literacy, for patients to use their data effectively to manage their self-care and partner with increasingly more remote care teams.
2) Deliver feedback to healthcare on data sharing practices preferred by patients, so that patient trust can be maintained.
3) Deliver data in order to advocate for better care and access to medicines alongside patients.
We strongly believe that patients should be the owners of their data and remain in control of data access and management and that the GO FAIR principles should be applied to ensure appropriate use of aggregate anonymous data.
Data collection and the sharing of successful practices
During 2021, CFE consulted widely to assess current capabilities and opportunities across all 39 member patient organisations in Europe.
CFE consulted with CFE member organisations through workshops, meetings and an online survey (Survey Monkey). The survey asked what support patient organisations would like in order to enhance data collection in their country. 59 percent of organisations responded to the online survey (23 out of 39). We also carried out interviews with member organisations to discuss ongoing projects and examples of best practice.
In some countries, data collection in CF is leading the way, and others are seeking to enhance the collection of data. Who collects the data and how it is stored differs from country to country. It is clear that there are a lot of great practices in CF and other disease areas.
The survey asked patient organisations what their perfect scenario would be with respect to data collection and what support they would like from CFE. The responses reflect the many different experiences of CF organisations across Europe. Feedback from patient organisations included:
“Promote exchanging of good practices, ensure data can be extrapolated to European level, organise informative events for member organisations”.
“Get deeper insight on effects of CFTR-modulators: how do they really influence patients’ lives? Psychologically, physically and effect on their participation in society (labour market, education, parenthood, …)”.
“We do not have any kind of official Registry for Cystic Fibrosis (regional, national, nothing). Everything … is voluntarily from doctor to doctor, or Clinic to Clinic. Our Association collects everything in one place, and when someone needs some data, we send what we have, but nothing of that is official”.
Some best practice examples
The German CF patient organisation is currently working on documenting PROs into the German CF Registry by expanding their online registry tool MUKO.web with a new interface (MUKO.PRO patient portal). The PROs will complement the medical data documented in the registry in a patient-specific dataset. The questionnaires will be browser-based and can be answered on a PC or mobile device during, or shortly after, their outpatient clinic visit. A pilot project with 5 outpatient clinics is planned in late 2022.
In the Netherlands, UMC Utrecht, together with the Dutch CF Foundation, have developed a patient-specific PROM, the Q-Life app, that assesses quality of life based on individual outcomes chosen by people with CF. In the app, people with CF can define and rank up to 5 personal indicators they find most important for their own quality of life (QoL). In a first validation study including 89 clinically stable patients of 14 years and older, participants could score for each personal indicator to what extent they felt limited by CF on a 5-point Likert scale, ranging from not limited to completely limited. After 14 days, this was repeated, and the results became visible in the app. The pilot study showed that QoL assessment with the Q-Life app had a high internal consistency and reliability. Convergent validity was demonstrated by the correlation of Q-Life scores with CFQ-R scores and FEV1. The app has been translated in the languages of all people taking part in the HIT-CF clinical trials (English, German, French, Danish, Flemish, Spanish, Italian, Polish, Portuguese, Swedish and Hebrew). In early 2022, around 250 people with CF that recently started with Trikafta are tracking changes in their Q-Life scores over the next year.
Co-designing VERA with our members
We heard clearly that patient organisations across Europe want to collect more data to benefit people with CF, particularly to gain deeper insight into new innovative therapies. This is with the aim of improving access to new treatments, and improved patient monitoring to better see trends and predict exacerbations. Responses indicated that the potential gains for patients as well as other stakeholder groups is significant.
Three themes clearly emerged from the scoping work:
- It is clear that there is a need to establish an efficient RWD capabilities platform that CFE member patient organisations can rely on to always keep them up to date.
- It is desirable to offer support for individual CFE member patient organisations with their RWD set up.
- Whatever is put in place must be able to build European scale data.
Best-practice sharing platform
To support our members, we will provide up-to-date information on RWD projects across Europe and facilitate learning about data. The platform will include:
-bespoke learning packages
-webinars and other tools specifically on data collection in CF
-a web portal for CFE members to submit best practice examples.
Setting up pilot projects
Learning from pilot projects is invaluable to designing the VERA project. The first pilot project, VERA Belgium is now underway, using a new quality of life questionnaire to:
-collect patient data in a secure and structured way
-use these data to ensure continuity of therapy for patients on modulators
-render the collection of data consistent, coherent and comparable on a national and European level based on the same questionnaire
-Pseudonymize data so that clinical and patient data can be used from the same person to improve quality of care
-enable sharing of these data to the care teams for above mentioned use
-package the learnings to enhance collection of quality-of-life data in other countries.
As VERA moves forward, additional pilots will be selected. The criteria for designing pilot projects are:
-They are supported by the national patient organisation in their country.
-They gather data not currently available.
-They gather data needed to reflect PWCF viewpoint.
-They are realistically scalable.
-Learnings can be taken across rare diseases and link to European level policy (European health data space, FAIR, EMA).
Find out more
To find out more, you can contact Claire Francis, Programme Manager for the VERA programme, at email@example.com or contact the CFE office.
Over the last few years, the ECFS-PR has been collecting clinical data from people with CF to identify changes to care needs. This data set provides a wealth of insight into clinical care, however it is limited in providing insight into people’s perception of access to medicines. To fill this gap, CF Europe ran surveys to patients, caregivers, and clinicians to gain insight into the overall picture and between country variation in access to vital cystic fibrosis (CF) medicines across Europe during the COVID-19 pandemic.
1.More people with CF live in urban than rural areas
2.Full time employment rates are low amongst people with CF
3.Problems accessing CF medicines are more common in Eastern than Western Europe
4.Healthcare systems in Western Europe ease the cost of CF treatments more than those in Eastern Europe.
A key focus of the analysis was comparison between Eastern and Western European countries. These results will steer the future strategy of CF Europe, and can be used by individual countries seeking to improve the lives of people with CF.
Overall, we found many people with CF and their carers (65%) are struggling with the cost of living. This was particularly clear for Eastern Europe (85%) compared to Western Europe (61%). This is further exacerbated as few respondents (26%) were in either full time or part time employment. This may change in the future as 33% of respondents were students reflecting the young CF population, but whether it will improve is dependent on their future health outcomes.
Improving health outcomes is reliant on access to the CFTR modulators (Kalydeco, Symkevi, Orkambi, Kaftrio) as 82% of respondents are eligible for at least one of the modulators. Yet, only 26% of respondents had access to a modulator before, with a particular contrast between Eastern (13%) and Western Europe (34%). This contrast is due to variation in availability in individual countries, and highlights the differences in outcomes we might see in the future if more countries don’t get access to the CFTR modulators.
COVID-19 and access to treatments
Across Europe, lockdowns and rules varied from country to country. However, most people with CF were instructed to remain indoors as much as possible to avoid contracting COVID-19. The impact of the pandemic had a large impact on people with CF, as 23% of people in Western Europe and 50% in Eastern Europe struggled to access treatments as they were unable to travel to clinics and the cost of the treatments. Such additional pressure had a detrimental impact on their physical and mental health.
Access to CF Treatments
Generally, CF treatments that help alleviate symptoms are generally accessible across the European CF Community, particularly to clear mucus (80%) and pancreatic enzyme replacement treatments (91%). However, there was still quite a contrast between Western and Eastern Europe, for example:
- 68% have access to airway clearance treatments but in Western Europe access was much higher (81%) compared to Eastern Europe (45%) due to lack of trained staff.
- 78% have access to IV antibiotics when they need it across Europe but, the barriers in Eastern Europe were predominantly due to cost (49%) and availability in the country (25%).
- 80% have access to treatment for pseudomonas with a large discrepancy between Eastern (71%) and Western Europe (85%).
- This discrepancy was even more noticeable for access to oral/inhaled antibiotics (92% in Western versus 58% in Eastern Europe) and access to Insulin for CFRD (94% in Western versus 73% in Eastern Europe).
Overall, people with CF don’t feel that the healthcare system in their country helps to ease the additional financial cost of CF medicines (43% in Western Europe and 80% in Eastern Europe).
These results particularly highlight the need for CF Europe to help support national patient organisations to overcome the identified barriers and ensure all people with CF have access to treatments when they need it to reduce avoidable delays which are detrimental to their health. Going forward, we are committed to steer forward oru advocacy work to highlight the identified barriers but also the discrepancy between countries which exacerbated health inequalities and poorer outcomes across European countries.