Listening to Lead: Patient Voices Driving Innovation in Hematology

Driving Transformative Solutions: A Differentiated Approach

Across hematology, our patient-centric research centers on exploring novel mechanisms and technologies to target underlying drivers of disease within the immune system, with the goal of advancing new treatments that have the potential to address the unmet needs for patients living with rare, chronic diseases.

In MM, we are exploring the potential of synergistic combinations – pairing therapies with different mechanisms of actions – to target MM cells, particularly for newly diagnosed patients, as earlier treatment intervention may contribute to improved disease management over time. As Yvonne Barnes, US Medical Head of Hematologic Malignancy notes, another key goal of our research is to match the right treatments to the right patients. By evaluating these combinations across diverse populations – such as frail and elderly patients – we want to better understand how to tailor treatment approaches for those who may not benefit from common treatment regimens. Beyond this, we aim to accelerate the delivery of new therapies by exploring novel endpoints like minimal residual disease negativity (MRD) – a powerful prognostic measure that can potentially demonstrate the clinical benefit of new medicines in MM.

For rare blood disorders, Sanofi continues to transform care. Our ambitions are driven by strategic innovation across key areas: exploring exciting pathways in hemophilia care with the aim of addressing current limitations; harnessing our deep immunoscience expertise to investigate potential novel mechanisms for rare autoimmune conditions; and pursuing the potential of emerging approaches in sickle cell disease (SCD) treatment to address significant unmet needs.

As Brad Ward, Interim Head of US Medical, Rare Blood Disorders articulates, our differentiated approach goes beyond traditional drug development by deepening our understanding of disease mechanisms, quality of life, and patient focused outcomes in addition to conventional endpoints. “This is critical in rare blood disorders, where complex mechanisms and a narrow focus on conventional measures have often led to suboptimal patient outcomes. We are committed to advancing treatments that seek to address unmet needs so patients can pursue their goals,” Ward notes.

Enhancing Patient Experience: Novel Administration and Real-World Data

Beyond therapeutic innovation, new modes of administration – including oral therapies and alternate administration methods – are a key focus across our teams. Particularly for patients managing chronic conditions that require ongoing disease management, identifying ways to improve the treatment experience is paramount to our goal of addressing the holistic burden. Similarly, real-world data is an important complement to our clinical research, providing a comprehensive view of the holistic treatment experience. This type of evidence generation is particularly important in disorders like hemophilia to help stakeholders understand what to expect when making a new treatment decision.

Sanofi’s Commitment to a Brighter Future

Our commitment to patients with difficult-to-treat blood cancers and rare blood disorders is unwavering, and we believe that true progress emerges from the powerful intersection of rigorous scientific research and dedicated patient advocacy. By continuing to integrate patient insights in clinical research, fostering open dialogue with the advocacy community, and pioneering innovative solutions, we strive to redefine what’s possible for individuals living with rare hematologic conditions.