Cystic Fibrosis Europe (CF Europe) is committed to advancing the interests and well-being of people with cystic fibrosis (CF) across Europe. As advocates for the CF community, we recognize the importance of collective efforts in shaping the future of pharmaceutical legislation within the European Union (EU). In line with the recommendations put forth by EURORDIS and the European Patients Forum, we present our position statement on the reform of EU pharmaceutical legislation: 

  1. Innovation driven by unmet needs: CF Europe endorses pharmaceutical legislation reform that places patient-centred innovation at the core of its objectives. We believe that any developments in research should be centred on what matters for people with CF and what they identify as an unmet need. The EU should prioritize creating an environment that encourages and accelerates the development of innovative therapies for rare diseases like CF. Regulatory frameworks should be adapted to facilitate the approval and equitable access to life-enhancing and life-saving therapies for cystic fibrosis.
  2. Accelerated Approval Processes: We echo the call for streamlined and expedited approval processes for CF medications within the EU. Swift access to new treatments is paramount for individuals with CF, and we support the recommendation to expedite assessments and approvals for therapies addressing unmet medical needs, such as CF. In particular, even though the advent of CFTR modulators has been truly transformative for the majority of people with CF living in those European countries with a performant healthcare system, there is still a significant portion of people with CF having to make do with only symptomatic treatment, because they don’t have access to these innovative treatments or because their CF is caused by (ultra)rare mutations. These people still have the highest unmet medical need. We highlight the importance of considering, in the revisions, the organoid technology and personalized medicine as a possible way to secure access to innovative treatment for people with ultrarare mutations that do respond to the modulators in organoid models. Additionally, for those people with CF who do have access to CFTR modulators but must stop them due to important side effects on mental health and liver dysfunction, new CFTR modulators need to be developed in the longer term. Moreover, transplanted patients, are often not receiving CFTR modulators due to drug-drug interactions with immunosuppressive medication and they represent a significant part of the adult CF population.
  3. Collaboration and Research Investment: CF Europe fully endorses the promotion of collaboration among stakeholders, including patient organizations, pharmaceutical companies, healthcare providers, and EU regulatory authorities. Collaboration and investments in CF research are vital for expediting the development and approval of life-saving therapies and ensuring their equitable distribution across Europe.
  4. Real-world Data Utilization: We strongly advocate for the integration of real-world data and patient insights into the EU’s regulatory decision-making processes. The use of real-world evidence is essential to assess the effectiveness and safety of CF treatments in real-life settings, ensuring that regulatory decisions are rooted in the experiences of CF patients.
  5. Access and Affordability: CF Europe supports the recommendations on addressing issues of access and affordability within pharmaceutical legislation reform. We emphasize the need to make CF treatments accessible to all individuals, regardless of their financial means or geographical location. Pricing transparency, fair pricing mechanisms, and patient-friendly reimbursement policies must be considered to ease the financial burden on CF patients and their families.
  6. Paediatric Access and Research: We stress the importance of ensuring that CF therapies are accessible to paediatric patients. Pharmaceutical legislation reform should incorporate provisions that prioritize paediatric access and encourage dedicated research efforts to meet the unique needs of children living with CF.

In summary, Cystic Fibrosis Europe acknowledges the recommendations made by EURORDIS and the European Patients Forum and fully supports their integration into the reform of EU pharmaceutical legislation. We call upon EU policymakers, regulatory bodies, and all stakeholders to work in unison to create a regulatory framework that embodies patient-centric innovation, expedited approvals, and equitable access to life-enhancing and life-saving therapies for cystic fibrosis within the European Union. 


To find out more:  

Reform of the EU pharmaceutical legislation 

Eurordis statement November 2022 

Eurordis statement March 2023 

Eurordis statement April 2023 

EPF statement April 2023