Since the introduction of CFTR modulators over ten years ago, treatment and care of people living with cystic fibrosis (CF) has significantly changed. These treatments targeting the root cause of CF has led to better health outcomes and improved quality of life (1,2). The European Medicines Agency, who are responsible for approving treatments across Europe, have approved four CFTR modulators to date – Kalydeco (2012), Orkambi (2015), Symkevi (2018) and Kaftrio (2020) (3). However, years later there is still great disparity in accessing these treatments across Europe, with a particular divide between Eastern and Western European countries. 

This health inequity has been further exacerbated by the COVID-19 pandemic, but the cause is fundamentally due to the different reimbursement processes in each European country. The time it takes to access new medicines across the European Union is widening. For example, the average time it takes to access new treatments in Germany is 133 days compared to Romania where it is 899 days. Additionally, less than 33% of treatments are available in smaller European States (usually in the Eastern region) compared to larger states. A 2021 CFE survey showed only 46% of respondents in Western Europe have access to a modulator therapy compared to only 24% of respondents in Eastern Europe. Overall, the low uptake of CFTR modulators across Europe is unacceptable but these figures also demonstrate the difference between Eastern and Western States.   

Health inequity in accessing CF care across Europe far extends the CFTR modulators, with many of our members telling us that people living with CF in Europe often struggle to access standard treatments to alleviate their symptoms, to consult a CF specialist (let alone specialists from other disciplines such as an endocrinologist or ear-nose-throat doctor, or allied health professionals such as a psychologist, dietician etc.) and to afford the support they need. For example, a CFE survey showed that 44% of respondents from Eastern Europe struggled to access airway clearance therapies, compared to 19% of respondents in Western Europe. Even without CFTR modulators, access to standard treatments and appropriate healthcare professionals is essential to provide people with CF with the best quality of life and improve life expectancy. It is therefore imperative that CF services across Europe are assessed for gaps and the EMA and other regulatory bodies assist EU member states in improving access to such care. This is particularly important because if we are to be considered a European Union then medical products should be available to move cross borders to help support people living with chronic conditions like CF.  

As an organisation, CF Europe has been working with our members to tackle this inequity to ensure every person living with CF across Europe, specifically between Eastern and Western States, has access to the care they need. For example, we held a European Parliamentary event in December 2022 with the European Respiratory Society (4). We also have been running surveys to collect the necessary data to highlight the extent of inequity across Europe to help inform decision-making. For more information on our campaigning efforts, please visit our campaigning webpage: https://www.cf-europe.eu/what-we-do/advocacy/  

We will continue our advocacy and campaigning until this inequity is tackled. We are calling for: 

  • When EMA approves a treatment, greater consideration should be given to the capacity for individual countries to roll out new and existing treatments and what support they may need where there isn’t sufficient capacity to do so.   
  • Pharmaceutical companies should become a member of EFPIA and commit to submitting a reimbursement dossier within 2 years after market authorization. 

 

 

1 Regard et al, CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France, June 2022: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9179538/  

2 Page et al, Lived experiences of individuals with cystic fibrosis on CFTR-modulators, January 2022: https://bmcpulmmed.biomedcentral.com/articles/10.1186/s12890-022-01825-2#:~:text=Clinical%20trial%20data%20show%20that,lives%20of%20individuals%20with%20CF 

3 EMA, Marketing Authorisation: https://www.ema.europa.eu/en/medicines/human/EPAR/orkambi#:~:text=Orkambi%20received%20a%20marketing%20authorisation,EU%20on%2019%20November%202015 

4 CFE Access to Medicines European Parliamentary Event, December 2022: https://www.youtube.com/watch?v=WACcwEYnQmE