Beyond Clinical Trials: Real-World Evidence Supporting Better Patient Outcomes in Transplant

As the Global Head of Real-World Evidence (RWE) at Sanofi General Medicines, I'm constantly inspired by the potential of RWE to transform patient care. My journey for the past couple of decades in addressing research questions with real world data has reinforced my belief that RWE serves a critical role in understanding the value of new therapies. Through data-driven insights, RWE empowers us to personalise treatment strategies informed by the identification of unmet needs, optimise treatment pathways, and improve healthcare efficiency.

Sanofi is deeply committed to addressing these unmet needs and improving patient outcomes. My team is dedicated to generating robust RWE that complements clinical trial data, providing a more complete picture of a treatment's impact and ensuring that more people can access the most suitable treatment for them.

The Value of RWE in Transplant

As the European Hematology Association (EHA) congress concludes, I have been reminded that RWE is a particularly powerful tool in the transplantation landscape. It allows us to understand the value and potential benefits of novel interventions in patient populations that may not be fully represented in traditional clinical trials. In Europe, decision-makers are increasingly recognising the value of RWE for contextual evidence to support regulatory submissions.[1] This shift underscores the growing importance of real-world data in shaping healthcare policy and access to innovative treatments.

RWE also presents exciting opportunities in the rare disease space. It can improve clinical trial efficiency, accelerating the time to market and patient access for much-needed therapies. Furthermore, RWE can be invaluable for evidence gathering prior to clinical development, helping to define the natural history of a disease and identify potential therapeutic targets.[2]

Addressing Unmet Needs in Chronic GVHD

Chronic Graft-versus-Host Disease (cGVHD) is one such rare condition where RWE can significantly improve our understanding of the disease and, ultimately, patient outcomes. GVHD is a serious complication affecting a significant proportion of patients who have undergone allogeneic hematopoietic stem cell transplantation, with many developing chronic GVHD within a year.[3]

cGVHD is a complex and heterogeneous disease. The disease burden is often multifaceted for these patients, resulting in physical, functional, and psychosocial deficits that negatively impact quality of life.[4] Many patients require multiple lines of therapy to control their disease, highlighting the challenges in managing this condition.[5]

Currently, a significant unmet medical need exists for new treatment options in the EU, particularly beyond second-line therapy, that can improve long-term outcomes for individuals living with cGVHD.

As a community, we need to embrace RWE to advance innovation and progress in the transplant space and to improve outcomes for individuals living with cGVHD.

Sanofi's pursuit of RWE excellence in transplant

At this year's #EHA2025 congress, we were excited to present new real-world evidence data from our transplant portfolio for patients with cGVHD. These data, together with results recently presented at #EBMT2025, provide valuable insights into unmet needs and the potential benefits of innovative treatments in real world settings. I want to express my sincere gratitude to all the study authors and Sanofi colleagues involved in the submission and presentation of these important data. Their dedication to generating and sharing RWE is crucial to advance our understanding of cGVHD and improve patient care. I am particularly proud of the research our team has conducted using innovative methods, such as target trial emulation. This approach allows us to estimate treatment effects more reliably using observational data, bridging the gap between randomized trials and real-world evidence.

At Sanofi, we remain committed to protecting the promise of transplant through rigorous real-world data generation and ongoing collaboration. We believe that by working together, we can keep expanding our shared scientific knowledge and discover new breakthroughs that change transplant patients’ lives. As a trusted partner within the transplant community, we will continue to chase the miracles of science to ensure that even more patients, including those with haematological conditions, can receive hope through a successful transplant. I look forward to seeing RWE data come to life at even more meetings in the future.

[1] Alana Hippensteele. Pharmacy Times. Bridging Evidence Gaps: The Expanding Role of Real-World Evidence in Regulatory and HTA Decision-Making. Accessible at: https://www.pharmacytimes.com/view/bridging-evidence-gaps-the-expanding-role-of-real-world-evidence-in-regulatory-and-hta-decision-making

[2] Cegedim Health Data. Rare diseases: How real-world data can help deliver new therapies. Accessible at: https://blog.cegedim-health-data.com/rare-diseases-how-real-world-data-can-help-deliver-new-therapies

[3] Lee JS, Classification systems for chronic graft-versus-host disease, Blood. 2017;129(1):30-37

[4] Hansen JL, et al. Psychological and physical function in allogeneic hematopoietic cell transplant survivors with chronic graft-versus-host disease. J Cancer Surviv. 2023 Jun;17(3):646-656

[5] Lee SJ, Nguyen TD, Onstad L, et al. Success of immunosuppressive treatments in patients with chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2018;24:555–562