World Haemophilia Day 2022: Sobi's position statement

7 April, 2022 01:00

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

Haemophilia: the disease and evolution

In 2020 the World Federation of Hemophilia (WFH) identified 165,379 people with haemophilia A and 33,076 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 leading 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 death to the potential of a normal life⁶

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. More recently, 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 surgery.⁷

Currently, factor replacement therapies as well as non-factor therapy are available in most European countries. However, haemophilia products in most of the EU countries are under significant price pressure due to strict approaches such as ‘winner takes all’ in national tenders that have seen single manufacturers awarded the total allocation of product, which threatens the diversity of supply and long-term sustainability of drugs. 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 term. This will help ensure a more efficient and sustainable health system⁹.

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 processes 10 . As such their preference must be a key factor in purchasing decisions in order to improve outcomes and address remaining unmet patient and 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 2021, the WFH celebrated reaching the milestone of 1B IU of factor being donated through the Program, of which close to 600 million IUs of factors had been donated by Sobi and Sanofi. Over 18,880 people have been treated with factors donated by Sobi and Sanofi through the programme since 2015. 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 is setting new ambitions: normal haemostasis as a new, optimal standard of care⁶. A ‘normalised’ haemostasis and lifestyle should be the aim of haemophilia care. High to normal factor levels could lead to absence of bleeding, long-term preservation of joint function and full capacity for enjoying an active life. This would also enable an easier disease management as result of the superior 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 influence the future of people living with haemophilia towards a truly liberated one¹⁴. A diverse, science driven treatment landscape can help eliminate patient uncertainties, improve stability in clinical outcomes and ensure a more predictable healthcare resource utilisation.

References

Report on the WFH annual global survey 2020. Accessed on 7 March 2022 at: https://www1.wfh.org/publications/files/pdf-2045.pdf
Berntorp E, Fischer K, Hart DP, et al. Haemophilia. Nat Rev Dis Primers. 2021;7(1):45.
O'Hara J, Walsh S, Camp C, et al. The relationship between target joints and direct resource use in severe haemophilia. Health Econ Rev. 2018;8(1):1.
Shapiro S, Makris M. Haemophilia and ageing. Br J Haematol. 2019;184(5):712-720.
O’Mahony B. Ageing and Haemophilia. Accessed on 7 March 2022 at: https://haemophilia.ie/living-with-haemophilia/adults-with-haemophilia/ageing/aging-and-haemophilia/
Skinner MW, Nugent D, Wilton P, et al. Achieving the unimaginable: Health equity in haemophilia. Haemophilia. 2020;26(1):17-24.
Aledort L, Mannucci PM, Schramm W, et al. Factor VIII replacement is still the standard of care in haemophilia A. Blood Transfus. 2019;17(6):479-486.
Mancuso ME, Mahlangu JN, Pipe SW. The changing treatment landscape in haemophilia: from standard half-life clotting factor concentrates to gene editing. Lancet. 2021;397(10274):630-640.
Expert Panel on effective ways of investing in health. Accessed on 7 March 2022 at: https://ec.europa.eu/health/expert-panel-effective-ways-investing-health/overview_en
APPG on Haemophilia and Contaminated Blood: Inquiry into Access to Treatment; Putting people with bleeding disorders at the heart of care. APPG_report_web.pdf (haemophilia.org.uk)
The WFH Humanitarian Aid. Accessed on 7 March 2022 at: https://www.wfh.org/en/wfh-humanitarian-aid-program
Franchini M, Mannucci PM. Past, present and future of hemophilia: a narrative review. Orphanet J Rare Dis. 2012;7:24.
Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition [published correction appears in Haemophilia. 2021 Jul;27(4):699]. Haemophilia. 2020;26 Suppl 6:1-158.
Hughes T, Brok-Kristensen M, Gargeya Y, et al. Treating for stability: an ethnographic study of aspirations and limitations in haemophilia treatment in Europe. J Haem Pract. 2020;7(1):165-172.