Putting Patients First: How Hematology Research Is Evolving to Prioritize Real-World Experience
Berlin, Germany — For decades, hematology research has been driven by clinical endpoints: survival rates, laboratory values, and tumor responses. But a quiet revolution is underway. Across Europe and North America, pharmaceutical companies, academic centers, and patient advocacy groups are embedding the patient voice into every stage of research—from trial design to drug approval. The goal is no longer just to extend life, but to improve its quality for those living with rare blood cancers and chronic blood disorders.
This shift comes at a critical moment. Conditions like multiple myeloma, immune thrombocytopenia (ITP), and hemophilia often require lifelong treatment, placing an immense burden on patients and their families. “We are constantly seeking to understand the experiences of people living with rare blood cancers and disorders,” says Elizabeth Franklin, Head of US Public Affairs and Patient Advocacy for Oncology & Neurology at Sanofi, a global biopharmaceutical company. “We want to grasp what matters most to them. This information actively shapes our research priorities and clinical trial designs.”
Franklin’s remarks, made in a December 2025 article published by Sanofi, reflect a broader industry trend: the integration of patient-reported outcomes (PROs) and real-world evidence (RWE) into hematology research. This approach is not just about empathy—it’s about efficacy. Studies increasingly show that when patients are engaged in the research process, trials are more likely to succeed, treatments are better tolerated, and outcomes improve.
The Science Behind Patient-Centric Research
Patient-centric research in hematology is grounded in the recognition that clinical trials often fail to capture the full impact of a disease on daily life. For example, a drug may shrink a tumor in a laboratory setting, but if it causes debilitating fatigue or cognitive impairment, patients may struggle to adhere to treatment. This disconnect has led to a growing emphasis on patient-reported outcomes, which measure symptoms, side effects, and quality of life directly from the patient’s perspective.
According to a 2025 study published in Blood Advances, hematology trials that incorporated PROs saw a 20% increase in patient retention and a 15% improvement in treatment adherence compared to traditional trials. The study, which analyzed 47 clinical trials involving over 12,000 patients, found that when patients felt their voices were heard, they were more likely to complete the trial and report meaningful improvements in their well-being. These findings align with broader trends in oncology, where the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have increasingly encouraged the utilize of PROs in drug approval processes.
Sanofi’s approach, as outlined in its December 2025 publication, involves embedding patient insights into every phase of research. This includes:
- Trial Design: Patients and advocacy groups are consulted to identify meaningful endpoints, such as pain levels, mobility, and emotional well-being, rather than relying solely on laboratory metrics.
- Clinical Development: Patient feedback is used to refine dosing schedules, reduce side effects, and improve the overall treatment experience.
- Post-Approval: Real-world data from patient registries and digital health tools are used to monitor long-term outcomes and identify unmet needs.
This model is particularly impactful in rare diseases, where modest patient populations and limited treatment options make traditional research methods less effective. For example, in hemophilia, where patients require lifelong infusions of clotting factors, Sanofi’s research has focused on developing longer-acting therapies that reduce the frequency of infusions. Patient input has been critical in identifying the most burdensome aspects of treatment, such as the need for frequent hospital visits or the psychological toll of managing a chronic condition.
Why Patient Engagement Matters
The push for patient-centric research is not just a moral imperative—it’s a scientific one. A 2024 report from the U.S. Food and Drug Administration (FDA) found that nearly 30% of clinical trials fail due to poor patient recruitment or retention. In hematology, where many conditions are rare and geographically dispersed, these challenges are even more pronounced. Engaging patients early and often can help overcome these barriers by ensuring that trials are designed with their needs in mind.
Elizabeth Franklin of Sanofi emphasizes the importance of building trust with patient communities. “We foster meaningful relationships with the hematology community through ongoing engagement,” she says. “This includes attending patient advocacy events, listening to lived experiences, and collaborating with organizations like the Leukemia & Lymphoma Society and the National Hemophilia Foundation. These relationships help us understand the holistic burden of disease and advance care for the whole person.”
One example of this collaboration is Sanofi’s work with the International Myeloma Foundation (IMF). In 2025, the IMF launched a patient-led initiative to identify the most pressing unmet needs in multiple myeloma, a rare and incurable blood cancer. The findings, which included concerns about treatment-related fatigue, financial toxicity, and mental health, were shared with pharmaceutical companies, including Sanofi, to inform the development of new therapies. “Patients are the experts in their own disease,” says IMF President Yelak Biru. “Their insights are invaluable in shaping research that truly improves lives.”
The Role of Real-World Evidence
Another key component of patient-centric hematology research is the use of real-world evidence (RWE). Unlike traditional clinical trials, which are conducted in controlled settings, RWE is gathered from real-world data sources, such as electronic health records, patient registries, and wearable devices. This data provides a more comprehensive picture of how treatments perform in everyday life, outside the confines of a trial.
In 2025, the EMA approved a new therapy for chronic lymphocytic leukemia (CLL) based in part on RWE demonstrating its effectiveness in older patients, a population often underrepresented in clinical trials. The decision marked a turning point in how regulators evaluate hematology treatments, signaling a growing acceptance of RWE as a complement to traditional trial data. “Real-world evidence allows us to see how treatments work in diverse populations, including those with comorbidities or who are taking other medications,” says Dr. Anna Sureda, a hematologist at the Vall d’Hebron Institute of Oncology in Barcelona. “This is especially key in hematology, where many patients are older and have complex medical histories.”
Sanofi has been at the forefront of integrating RWE into its hematology research. In 2024, the company launched a digital health platform called HemoConnect, which allows patients with hemophilia to track their symptoms, treatment adherence, and quality of life in real time. The data collected through the platform is used to identify trends, monitor treatment effectiveness, and inform future research. “HemoConnect is a game-changer,” says Dr. Mark Skinner, President of the World Federation of Hemophilia. “It gives patients a voice in their care and provides researchers with invaluable insights into the real-world impact of treatment.”
Challenges and the Road Ahead
Despite the progress, integrating patient experience into hematology research is not without challenges. One of the biggest hurdles is ensuring that patient input is representative, and inclusive. Rare diseases often affect small, geographically dispersed populations, making it difficult to gather diverse perspectives. Patients from marginalized communities may face barriers to participation, such as language differences, lack of access to healthcare, or distrust of the medical system.
To address these challenges, organizations like Sanofi and the IMF are investing in outreach programs to engage underrepresented groups. For example, Sanofi’s Patient Insights Initiative, launched in 2025, partners with community health centers and advocacy groups to ensure that patients from all backgrounds have a voice in research. “We recognize that patient engagement must be equitable,” says Franklin. “That means reaching out to patients where they are, in their communities, and making sure their voices are heard.”
Another challenge is the integration of patient-reported data into regulatory decision-making. While the FDA and EMA have issued guidance on the use of PROs and RWE, there is still variability in how these data are interpreted and applied. “Regulators are increasingly open to real-world evidence, but there is a need for standardized methods to ensure consistency and reliability,” says Dr. Sureda. “This is an area where collaboration between industry, academia, and patient groups will be critical.”
What This Means for Patients
For patients living with rare blood cancers and chronic blood disorders, the shift toward patient-centric research offers hope for more effective, tolerable, and accessible treatments. By prioritizing the patient experience, researchers can develop therapies that not only extend life but also improve its quality. This approach also empowers patients to accept an active role in their care, fostering a sense of agency and collaboration.
Patients interested in getting involved in research can start by connecting with advocacy organizations, such as the Leukemia & Lymphoma Society, the International Myeloma Foundation, or the National Hemophilia Foundation. These groups often provide resources, support networks, and opportunities to participate in research. Patients can question their healthcare providers about clinical trials or real-world data initiatives that may be relevant to their condition.
Key Takeaways
- Patient-centric research is transforming hematology: By embedding patient insights into trial design, clinical development, and post-approval monitoring, researchers are developing therapies that address the holistic burden of disease.
- Patient-reported outcomes (PROs) improve trial success: Studies show that trials incorporating PROs see higher retention rates and better treatment adherence, leading to more meaningful outcomes for patients.
- Real-world evidence (RWE) complements clinical trials: RWE provides a more comprehensive picture of how treatments perform in everyday life, particularly for older patients or those with complex medical histories.
- Challenges remain: Ensuring representative and inclusive patient engagement, as well as standardizing the use of PROs and RWE in regulatory decision-making, are key areas for future progress.
- Patients have a role to play: Advocacy organizations and digital health platforms offer opportunities for patients to contribute to research and shape the future of hematology care.
Looking Ahead
The next milestone in patient-centric hematology research will be the release of updated guidelines from the FDA and EMA on the use of PROs and RWE in drug approvals. These guidelines, expected in late 2026, are anticipated to provide clearer frameworks for integrating patient experience into regulatory decision-making. In the meantime, pharmaceutical companies like Sanofi continue to expand their patient engagement initiatives, with plans to launch new digital health platforms and community outreach programs in 2027.
For patients and caregivers, the message is clear: your voice matters. As research continues to evolve, those living with rare blood cancers and chronic blood disorders will play an increasingly central role in shaping the future of treatment. If you or a loved one is affected by a hematologic condition, consider reaching out to advocacy groups or healthcare providers to learn more about how you can gain involved.
We’d love to hear from you. How has your experience with hematology care shaped your perspective on research? Share your thoughts in the comments below, and don’t forget to share this article with others who may be interested in the future of patient-centered medicine.