Value-based healthcare systems and the need to address the unmet medical needs of patients with rare bleeding disorders

On World Haemophilia Day we sat down with MEP Elisabetta De Blasis and Sobi to discuss how value-based healthcare approaches can address the unmet needs faced by people living with rare bleeding disorders such as Haemophilia in an equitable manner

By Sobi™

Sobi™ is an international biopharmaceutical company focused on rare diseases.

17 Apr 2024

Healthcare system resilience has emerged as a priority issue in Europe, as the continent faces an increase in demand and pressure stemming from demographic shifts, the rising prevalence of chronic diseases and funding constraints. Patients across Europe still face many unmet medical needs, especially when healthcare systems fall short in providing what patients truly need. To ensure a robust and adaptable system, that delivers patient-centric solutions, value-based healthcare approaches are needed.

Among the many rare bleeding disorders, haemophilia is an example of a disease where patients still report unmet medical needs throughout their life in spite of the significant scientific progress. The World Haemophilia Day, celebrated today on April 17 provides an opportunity to reflect on the imperative role that value-based approaches can have in driving change and address the needs faced by people living with rare bleeding disorders in an equitable manner. We invited Elisabetta De Blasis, Member of the European Parliament involved in the Committee on Industry, Research and Energy (ITRE) and the Subcommittee on Public Health (SANT) who plays an active European role in strengthening healthcare systems and supporting innovation, to join the Haemophilia community and industry partners, such as Sobi (Swedish Orphan Biovitrum AB) in this reflection.

Haemophilia is a genetic bleeding disorder caused by deficiencies in clotting factors (VIII and IX respectively for Haemophilia A and B), leading to bleeding into large joints such as elbows, knees, and ankles. Whilst several therapies are available for these rare bleeding disorders, patients living with this condition still face unmet medical needs. Beyond chronic pain, children and adults living with haemophilia often suffer from progressive joint deterioration, impaired mobility and mental health issues, which impacts all facets of their lives. It is evident that significant gaps persist in meeting the holistic needs of haemophilia patients and there is a need and an opportunity to elevate the standard of care. Addressing these challenges requires a concentrated effort from Member States to adopt a more comprehensive approach to healthcare: value-based healthcare can play an important role, by focusing more on the outcomes that are relevant to patients and by creating an environment which reflects clinicians’ preferences and patient individual unmet medical needs.

As a cardiologist and former hospital director in Italy, I understand the critical importance of listening to patients' individual needs and focusing on solutions that address these and add value to patients’ lives. Value-based healthcare systems put patients in the center and offer a pathway to ensure more personalised care, fostering inclusivity within our healthcare systems

– MEP Elisabetta De Blasis

Value-based approaches are also drivers of innovation. The perception that haemophilia is a resolved disease has led to a lack of recognition of the added value introduced by innovative therapies, despite the impact they might have on improving patients’ quality of life. Suboptimal clotting factor levels can lead to irreversible disease complications, causing productivity loss, higher healthcare resource utilisation, and overall poorer outcomes. In contrast, value-based procurement procedures can create an environment which sustainably rewards bidders whose treatments address today’s unmet medical needs. In doing so, ultimately the outcomes of people living with haemophilia can be improved while at the same time creating the environment for further development of innovation.

MEP Elisabetta De Blasis
MEP Elisabetta De Blasis, (ECR, IT).

By rewarding the value of innovative treatments, which can ultimately improve patients`s outcomes, we can enhance European healthcare systems’ resilience in the long-term.

The short-term savings from selecting the cheaper therapeutical options might be outweighed by the increased costs associated with disease complications and ultimately end up compromising future innovation. Innovation is essential in addressing the complex needs of individuals with rare bleeding disorders. As we rethink the General Pharmaceutical Legislation the time is now to ensure the EU can provide the right environment for companies to further develop innovative treatments. Value-based approaches play a pivotal role in making this possible 

– MEP Elisabetta De Blasis

Decision-makers need to broaden their perspective beyond short term budgetary considerations and acknowledge the wider societal and economic benefits of placing the patient at the center of healthcare systems. The adoption of value-based healthcare approaches represents a pivotal opportunity to change the treatment landscape for individuals with rare bleeding disorders such as haemophilia. By prioritising outcomes that matter most to patients while incentivising innovation, healthcare systems can bridge the gap between the unmet medical needs of patients with rare bleeding disorder and effective delivery of care.


Read more about World Haemophilia Day here.

Read the most recent articles written by Sobi™ - HLH Disease Burden Report


EU Institutions