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Raising the bar in haemophilia A care

Illustration of an outstretched hand cradling a large red blood drop, surrounded by floating red blood cells, set against a soft peach background.

Needs evolve, and so must care. For Dr. John Pasi, Global Franchise Head, Haematology and Rare Blood Diseases, Product Development at Roche, the future of haemophilia A care demands bold innovation, personalised solutions, and an unwavering commitment to the evolving needs of people affected by this disease.

Portrait of Dr. John Pasi, Global Franchise Head of Non-malignant Haematology, Product Development at Roche

Over years working directly with people with haemophilia A, I have seen first-hand the daily, different, and ever-changing realities they face. Understanding their lived experiences is what drives me in my role at Roche to keep pushing forward the science, because past breakthroughs don't diminish our responsibility to address all the remaining opportunities for improving care.

Dr. John Pasi

Global Franchise Head, Haematology and Rare Blood Diseases, Product Development at Roche

For people living with haemophilia A, every bleed matters. Though rare, the condition affects around 900,000 people worldwide, along with their families and loved ones.1,2

Haemophilia A occurs when the body lacks or has insufficient factor VIII, a clotting protein, which leads to uncontrolled bleeding.

This reality has fuelled decades of innovation, transforming a condition once defined by risk into a future defined by possibility. A future where care must continue to evolve to meet the changing needs of people with haemophilia A.

“Looking ahead, our next clinical challenge is to achieve full protection so a person can live as if they were not affected by haemophilia A at all,” says John. “This means treatments that can ‘normalise’ the blood clotting process, further reduce treatment demands, and provide freedom from the daily physical and psychological weight of managing haemophilia A.”

Abstract illustration symbolising scientific innovation and advanced therapies in haemophilia care.
Understanding ongoing unmet needs and new opportunities to improve care

Progress doesn’t erase the human realities of haemophilia A. Even as science advances, people still face physical, emotional and logistical burdens that influence every decision and shape their lives.

The definition of ‘unmet need’ itself evolves not only across a person’s life with haemophilia A, but also when advances in treatment raise expectations and reveal new challenges that were previously hidden.

Despite the availability of prophylactic therapies, some people with haemophilia A still experience bleeds and long-term complications such as chronic joint damage and haemarthrosis (bleeding into joints causing pain and swelling).

Beyond physical symptoms, haemophilia A can limit spontaneity and add logistical or financial strain – all pressures that evolve as life changes, from school to work to family. Fear of bleeds and vigilant contingency planning can be overwhelming.

Beyond the physical

When you speak with people living with haemophilia A, you understand how the impact goes beyond physical symptoms. Many describe having to organise their life around managing their condition, living with a psychological burden of planning for bleeds, and wanting to pursue personal goals without disease-related limitations. All of these can affect the quality of life for them and those around them.

There is no cure yet for haemophilia A, and these challenges persist globally, varying by disease severity, lifestyle and personal aspirations. As John explains, this drives a deeper question for the future of care. “Acknowledging this ongoing burden compels us to ask harder questions: how do we ensure that innovation translates into meaningful improvements for everyone living with haemophilia A, regardless of circumstances or location?”

Delivering treatment for all

Today, care must be patient-centred and personalised, moving beyond the traditional ’one-size-fits-all’ approach to provide the best possible outcomes for every person with a bleeding disorder.

Yet meeting these evolving needs across a lifetime means considering more than clinical variation. Many people living with haemophilia A in low- and middle-income countries have little access to diagnosis, care and therapies, so working towards an improved care environment for all is crucial.

Geographic, economic, and healthcare infrastructure realities shape access and adherence across populations. John notes that achieving this balance requires a broader definition of personalisation, “True equity in haemophilia A care means addressing personalisation at both ends of the spectrum. Tailoring treatment to individual patient needs while ensuring innovations are accessible across diverse healthcare systems worldwide.”

The critical role of innovation and collaboration

The haemophilia A community has seen remarkable progress, but work is far from complete. Each breakthrough opens new possibilities – and new challenges. Meeting them requires sustained long-term commitment and the bringing together of expertise from many different spheres.

Redefining innovation

Innovation means more than developing new treatments. It requires understanding of real-world experiences and barriers. From adherence challenges to navigating healthcare systems and coping with the psychological impact of living with a chronic condition.

Investment in research and development, rooted in the real and evolving needs of people living with haemophilia A, is essential to push boundaries and address unmet needs across all patient populations. But advancing haemophilia A care demands broad, multi-stakeholder collaboration, including patients and families, scientists, manufacturers, regulators, health technology assessors, patient organisations, and multidisciplinary care teams, all working together.

Illustration of a healthcare professional placing a plank to bridge a gap toward a hospital, with a diverse group of people waiting to cross, symbolising improved access to care.
Continuing the journey toward meaningful change together

At Roche, our vision is a haemophilia free life for all. Achieving this requires long-term commitment and partnership with the haemophilia A community.

For more than 20 years, Roche has been at the forefront of redefining treatment in haematology. Today, with more than 160 scientists dedicated to haemophilia research, we continue to innovate and develop medicines for people affected by the disease.

We are working closely with all corners of the global haemophilia A community. From clinical development partnerships to supporting healthcare professionals and people living with haemophilia A throughout their treatment journey, and partnering with organisations to create tailored access solutions. Our goal: transform how haemophilia A is treated and managed worldwide.

For John, delivering on this transformation and making real progress means bringing together innovation, partnership, and access. “The future of haemophilia A care lies in a combination of sustained innovation, personalised approaches, and dedication to meeting the evolving needs of people living with and affected by this bleeding disorder. Doing this requires both scientific excellence and a deep commitment to global health equity.”

References

  1. Srivastava, A, Santagostino, E, Dougall, A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020: 26 (Suppl 6): 1‐158.

  2. Iorio A et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males. Ann Intern Med. 2019 Oct 15;171(8):540-546

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