World Haemophilia Day 2024: Sobi's position statement

People with haemophilia can live a rich and fulfilling life, but healthcare policies need to keep up with science.

On World Haemophilia Day, we are calling on policy and decision makers to reflect in their decisions the evolution of treatment goals, by securing tailored approaches which fit with patient needs and improve long-term stability in clinical outcomes.

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.

Haemophilia: the disease and evolution

In 2022 the World Federation of Hemophilia (WFH) identified 208,957 people with haemophilia A and 42,203 people with haemophilia B worldwide¹.

Haemophilia is a genetic bleeding disorder characterised by a deficiency of coagulation factor VIII (FVIII) which causes haemophilia A, or factor IX (FIX), which causes haemophilia B. The hallmark characteristic of haemophilia is bleeding, especially into large joints such as elbows, knees and ankles. Such joint bleeding can eventually cause painful and disabling haemophilic arthropathy² which can start early in life and lead to higher healthcare resource use, poorer health-related quality of life (QoL) and reduced productivity in people with haemophilia³. Regardless of the severity of their

disease, patients who don’t achieve optimal protection may also suffer from life-threatening bleeds, such as intracranial bleeds and in other internal organs².

While people with haemophilia had previously much lower life expectancy than that of the general population, advances in the development of effective and safe treatments over the past 50 years have resulted in a significant increase⁴. For more people with haemophilia to reach their 70s or even 80s national health policies need to keep up with science providing access to diverse treatment options and innovation fitting individual patient’s needs in order to advance closer to the goal of optimal function and health equity^5,6.

Haemophilia outcomes evolution: from survival to the potential of a normal haemostasis⁶

Current haemophilia management and the policy environment

There are currently factor replacement therapies that replace the missing FVIII or FIX protein, with different options available, including standard half-life (SHL) and extended half-life (EHL) factors, used as prophylaxis or on-demand treatment for haemophilia A and B.  Decades of clinical and real-world experiences prove that prophylaxis with factor therapies is highly effective in reducing bleeding and long-term complications such as arthropathy⁷. Non-replacement therapy has expanded the therapeutic options for patients with haemophilia A⁸, however, this treatment is not suitable for acute bleeding episodes or management of major surgery⁷.

Factor replacement therapies as well as non-factor therapy are available in most European countries whilst gene therapies are still undergoing health technology assessments and price & reimbursement discussions. As highlighted by the EU Commission report on public procurement, Member States together with public buyers and decision makers must develop purchasing strategies for innovation whereby value for money is supported by better health outcomes in the long term⁹. The Report for the European Parliament on the shortage of medicines also recognizes that “price only” tenders cause severe price erosions and can produce disruptions to the number of suppliers on the market¹⁶.

In the case of haemophilia procurement and tendering, patients and frontline healthcare providers, who use and benefit from what is purchased, are partially or inadequately consulted during these processes¹⁰. Value-based procurement 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.

To improve the level of care in developing countries, Sobi and Sanofi have donated their medicines to the WFH Humanitarian Aid Program since 2015.

By providing more predictable and sustainable access to humanitarian aid donations, the Program makes it possible for people with inherited bleeding disorders to receive consistent and reliable access to treatment and care. In 2022, the WFH reached the milestone of 1,5B IU of factor being donated through the Program, of which more than 800 million IUs of factors had been donated by Sobi and Sanofi. Over 22,000 people have been treated with factors donated by Sobi and Sanofi through the programme since 2015¹⁷. Additionally, the companies have provided financial support for initiatives such as treatment, access and education programs¹¹.

The future of the haemophilia landscape

Decades of clinical research and investments in improving factor replacement, engineering recombinant factors in particular extending factor half-life, have allowed people with haemophilia to move towards the achievement of their full potential, ultimately improving their quality of life12. However, unmet needs still exist hence this potential is not reached by all people living with haemophilia across Europe and around the world.

To achieve the best outcomes for patients and ultimately for healthcare systems, prophylaxis should be individualised to provide optimal protection, taking into consideration the bleeding tendency, joint status, individual pharmacokinetics and patient preference. This approach may enable people with haemophilia to lead healthy and active lives including participation in most physical and social activities, similar to the non-haemophilic population¹³.

Considering improvements in science and diversity of the treatment landscape, the haemophilia community has set new ambitions: normal haemostasis as a new, optimal standard of care6. Suboptimal clotting factor levels can lead to irreversible disease complications, causing productivity loss, higher healthcare resource utilisation, and overall poorer outcomes¹⁸. A normal haemostasis should be the aim of haemophilia care,  as this would enable easier disease management as a result of the comprehensive level of protection.

Based on decades of unequivocal clinical and real-world evidence, Sobi believes that factor replacement is a fundamental cornerstone to treat and prevent bleeds in people with haemophilia. As treatment goals evolve, tailored approaches must fit with patient needs and allow for better long term outcomes. In the light of existing and emerging data on clinical safety and effectiveness, policy and decision makers have the power to address the remaining unmet medical needs of people living with haemophilia¹⁴. By prioritizing 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.