Paving the way for another decade of progress in hemophilia care through EU policy

On World Hemophilia Day, the hemophilia community sits together to reflect on progress to date in the care of patients with bleeding disorders, whilst pointing that challenges remain and several EU policy opportunities could be game changers to address these.

World Hemophilia Day serves as a reminder every year of how important it is to keep working collaboratively to improve patients’ diagnosis and access to specialist care and treatment across Europe.

MEP Deidre Clune Quote

What are the main challenges faced by bleeding disorders patients today and unmet needs? 

Inequalities in access to care and treatment still remain an issue in Europe, notes Laura Savini, from the European Hemophilia Consortium, also pointing at gender and geographical disparities. The complex care model for hemophilia is now a reference framework in Europe but certain aspects are lacking, for instance psychosocial support.

By their nature, bleeding disorders come with complexities and require involvement of many specialists to ensure a proper diagnosis as well as optimal care for people living with these conditions. This multi-disciplinary approach is crucial but not always in place in every EU country, explains Prof. Cedric Hermans, from St Luc University Hospital (Belgium). Current treatment options have brought improvements, but come with impact on patients’ health outcomes and quality of life due to the frequency of infusions that are needed to maintain a certain level of clotting factors [proteins that help control bleeding] in patients’ blood.

Specialised care through excellence centres is crucial in treating bleeding disorders, however more work needs to be done to ensure patients’ equal access to those centres across Europe, noted Karen Pinachyan, from CSL Behring.

MEP István Uhjelyi Quote

Which advances in the care of bleeding disorders can we celebrate today and what can we anticipate in the near future? 

“The journey of hemophilia in the last 50 years has been extraordinary”, underlines Laura Savini. “We went from no treatment to suboptimal treatment to replacement therapy, and now we have the prospect of gene therapy”. This has been possible thanks to collaboration and recovering from the trust crisis of the 90s resulting from contaminated blood, she added. The community of clinicians, patients, industry and broader health stakeholders has emerged stronger and joined forces to improve treatment and access to care for patients. There is still constant vigilance from the patient community, but also hope, she explained.

Biotechnology advances have helped to produce proteins artificially to reduce reliance on donors, now therapeutic innovation such as gene therapy holds the potential for patients to be cured and giving them a lot of freedom from burden of treatment, pain etc, underlined Prof. Hermans, adding that this evolution path makes hemophilia an area which can serve as an excellent example of what can be achieved for other rare diseases.

“It is important to address rare diseases holistically through a multi-disciplinary approach, involving all the relevant specialists. And hemophilia is a beautiful example of a learning space that should inspire other disciplines as it is a result of an excellent collaboration amongst all partners” - Prof. Cédric Hermans  

Karen Pinachyan also pointed that beyond therapeutic innovations, such as advanced therapies, the patient journey is evolving holistically, including systemic and health systems innovations, as well as technological innovation. Digital tools are now helping to provide comprehensive care for patients. This innovation has been possible by the community altogether.

How can we improve access to optimal care and treatment for patients across Europe in the years to come; and which policy opportunities can drive such change?   

“The EU has really been a driving force for policy in rare diseases”, underlines Laura Savini. “However, we would certainly like to see additional commitment, with additional investment in novel technologies as well as delivery of care”, she explained. At the European level, we are seeing a set of legislative proposals coming up [the revision of the general pharmaceutical legislation and orphan medicinal products legislation], which really give a once in a generation opportunity to make the system right to deliver better medicines for patients. There is a place to propose innovative models where we further use real-world evidence, generated by patients, she noted.

“We really are at a cross-road. Health systems need to be strengthened. […] With new technologies like gene therapy coming to Europe, we have to rethink the way we organise care, services, hospitals.” – Laura Savini

Cedric Hermans underlined that it will be important to continue focusing on cross-country collaboration, noting the value of the European Reference Networks that allow physicians to work together across Europe. These structures are unique in the world, he said. ERNs’ level of ambition shall contribute to helping patients have equal access to care and novel therapies, he outlined.

Karen Pinachyan noted that the whole treatment paradigm is shifting away from life-long administration of treatment to a one-time administration and very long-term low-intensity follow-up, allowing patients to live a normal life and improving their well-being. As such, adaptations in regulatory, HTA and payment models will be needed to assess how different models can be put in place and evolve to recognise innovation that is coming for patients, with systems that link payment to outcomes and value. These adjustments and novel models will be the keystone to making the system work and ensure that Europe remains at the forefront of hemophilia care and an attractive region for innovation. 

MEP Katalin Cesh underlined that policy changes shall continue improving hemophilia care in Europe.


MEP Cseh Quote

The future of hemophilia care looks bright. While hemophilia is regularly identified as a best practice in the rare disease field, policymaking should continue to support improvements in the years to come in patients’ care and access to specialist expertise and treatments, particularly to support the timely access of novel transformative therapies, such as gene therapy

“I am really excited for the next ten years, I think there is really the opportunity to change the paradigm for patients in how they receive their care, and also to enlarge it to other rare disease areas where there is currently no treatment and no hope for good quality of life” - Laura Savini

Expressing support for World Hemophilia Day, MEP István Uhjelyi reiterated this vision: "Here in the Parliament, we try to foster the creation of an environment that can address the unmet needs and provide proper access to the treatments and care patients need. I fully support World Hemophilia Day and the whole community. As a politician - to whom healthcare is extremely important - this is how I can help, thus in my work, I will continue helping to advance EU policies for the benefit of patients”.

This article is based on a Q&A debate recorded in the European Parliament in the context of World Hemophilia Day, moderated by EU journalist Jack Parrock working with DW News; organized with the support of MEPs Katalin Cseh and Deirdre Clune, and initiated by  CSL Behring. Watch the full debate here or below