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 the collection of Real World Data in Cystic Fibrosis
CF Europe’s new initiative, VERA, aims to spread the collection of Real World Data (RWD), led by patient organisations in their own countries, where CFE members wish to enhance their capabilities, or where no such capability currently exists. We will reflect best practices happening in many European countries today to enable the design of a Europe-wide model to advance access to optimal treatment and care for people with CF. We have called our Real World Data programme VERA, because we want to emphasize the word ‘real’, showing what it is really like living with CF, from the patients’ perspective. The word VERA, meaning ‘true’ in Spanish, Italian and Portuguese, reflects our priorities for this programme and our Europe-wide approach.
Why better data collection is needed
This is an unprecedented time for cystic fibrosis (CF) research, 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. Collecting RWD, understanding which platforms are used, what information is gathered, and how will be integral to driving up treatment access and care for all.
What is Real World Data and what are its benefits
RWD is any data not collected in conventional randomised controlled trials (RCTs). For example, it can be observational data, predominantly carried out through patient reporting and or recording of their healthcare experience and it can include data validated in a clinical setting. It 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 will 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.
Our ambition for Real World Data in CF
Data holds the key to helping people with CF understand their condition and will help to keep people with CF healthier across Europe. It provides an opportunity for autonomy and empowerment and will change the relationship with their healthcare, providing a rich picture of an individual’s health. Our vision is to create a ‘passport’ which will allow a care team to understand an individual’s health and needs. The data will be secure, putting the individual in fundamental control over who sees this data.
Alongside people with CF and a range of stakeholders, including other rare disease representatives, we are designing an approach so that what matters to patients gets measured. We want people with CF to be supported by patient organisations, to learn how to collect data to manage their self care and partner effectively with their care team 365 days of the year, rather than just at clinic visits once or twice a year. Building data collection around people with CF and supporting them will have many benefits, as the figure on the right shows.
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 their 8 Principles of Caring Technology in Belgium.
On a European level, CF Europe is learning from the Duchenne Data Platform and supporting the Go Fair principles.
What CF data is collected across Europe
During 2021, CFE consulted widely to assess current capabilities and opportunities across all 39 member countries. CFE consulted with CFE member organisations through workshops, meetings and an online survey (Survey Monkey). The survey asked about data collection practices, use of patient reported outcome measures and 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 and through anecdotal feedback for those who felt unable to fill in the survey questions.
These findings represent feedback from patient organisations and therefore highlights how patient organisations are looped into what is going on in their countries by the clinicians, hospitals and registries. Gaps in knowledge could indicate a need to improve collaboration within that country with healthcare stakeholders.
Clinical CF data collection – key findings from online survey
- There is a lot of variability across CFE members as to how clinical CF data collection is undertaken.
- 80% of patient organisations report that some or all patient demographics are routinely collected, including age, gender, and weight of pwCF. Three patient organisations reported that the most basic patient demographics are not collected into a registry or database.
- Patient organisations reported variability on the types of health measures collected into a registry or database beyond the basic demographics. 60% of patient organisations reported that respiratory measures were collected; 55% reported gastro-intestinal, and 25% said pain information was collected.
- 40% of patient organisations (8 respondents) stated that none of these health measures were collected.
- 66% of respondents said that drug names are collected into a registry or database but only 39% of respondents reported that information on dosage was collected, and 28% reported capturing side effects.
- Only 20% of respondents stated that pwCF had digital access to their own healthcare records.
Patient Reported Outcomes collection – key findings from online survey
- The survey found that 50% of CFE member organisations reported the collection of PROs in their country.
- We asked patient organisations what types of PROs were used in their country. The most common type used is the Cystic Fibrosis Questionnaire-Revised (CFQ-R) (44%).
- Other instruments used by respondents were; Generalised Anxiety Disorder 7 item questionnaire (GAD7) (6%), PHQ9 Patient Depression Questionnaire (6%), Patient Reported Symptom Diary (12%), EQ-5DL – a family of instruments to describe and value health (12%).
- 41% of members are currently working on digital projects that collect PROs in their country. 12% of patient organisations report being approached by MedTech companies to design digital tools for PROs.
What are Patient Reported Outcomes and why they matter
Patient Reported Outcomes (PROs) are data reported directly by a patient on his or her own health condition. Patient-reported outcome measures (PROMs) are the tools used to measure and collect data on PROs. PROMs are usually an established tool, such as a questionnaire, to monitor patients’ experiences and can help to support patient-clinician communication, and support future care. They can also capture the outcomes that are most important to people with CF in a standard way so that we can compare them and aid in establishing the cost-effectiveness of different treatments.
Real World Data collection and the sharing of successful practices
In some countries, RWD collection in CF is leading the way, and others are seeking to enhance the collection of RWD. 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 RWD 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 the pwCF. In the app, pwCF 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 pwCF 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 RWD 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.
Moving forwards, three work streams combined will make up the design phase for VERA in the coming 6 months and we will be inviting patient organisations and other stakeholders to co-design this work with us:
Alongside the three work streams, we are also working with CFE’s member patient organisations and providing practical support to set up or enhance RWD collection. We are aiming to set up three country or region level pilot programmes over the coming six months, where and when are to be agreed.
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.