Key Takeaways
- Real-World Data (RWD) and Real-World Evidence (RWE) have become central themes across hematology research, complementing findings from clinical trials and supporting evidence-based decision-making.
- The implementation of the EU Health Technology Assessment (HTA) Regulation is increasing the focus on patient-centered outcomes and real-world evidence to complement clinical trial data in reimbursement and access decision-making.
- Patient voice, quality-of-life measures, and patient-reported outcomes are gaining importance alongside traditional clinical endpoints, reflecting a broader shift toward value-based healthcare.
- Real-world evidence featured prominently at EHA 2026, with new studies presented across multiple haematological conditions, including PNH, ITP, HLH, myelofibrosis, haemophilia, von Willebrand disease, and other rare disorders.
- Von Willebrand Disease (VWD) illustrated how real-world data can bridge the gap between symptoms and diagnosis, helping clinicians identify diagnostic delays, quantify disease burden, understand patient journeys, and generate evidence that supports more timely, personalized, and patient-centered care.
- The future of hematology will depend not only on breakthrough therapies but also on the ability to transform healthcare data into actionable evidence that improves outcomes for real patients in real-world settings.
The annual congress of the European Hematology Association (EHA) is widely recognized as one of the most influential global forums for advances in hematology, bringing together clinicians, researchers, patient advocates, regulators, and industry leaders to shape the future of care for patients with blood disorders.
At EHA 2026 in Stockholm, scientific sessions showcased practice-changing Phase III clinical trials, novel targeted therapies, and cutting-edge CAR-T cell therapies across leukaemia, lymphomas, myelofibrosis, and other haematological diseases. Beyond therapeutic innovation, however, the congress highlighted a broader transformation in healthcare: the growing importance of patient-centered care, the implementation of the new European Union Health Technology Assessment (HTA) Regulation, and the increasing role of Real-World Data (RWD) and Real-World Evidence (RWE) in bridging the gap between clinical trial results and outcomes achieved in everyday clinical practice.
Across multiple disease areas, discussions increasingly focused not only on whether treatments work under controlled study conditions, but also on how they perform in diverse patient populations, how they impact quality of life, and how healthcare systems can generate evidence that supports more informed clinical, regulatory, and reimbursement decisions.
The Evolution of Evidence Generation
Randomized clinical trials remain the foundation of modern medicine. They provide the rigorous evidence required to demonstrate efficacy and safety and remain essential for regulatory approval.
However, the patients enrolled in clinical trials often differ significantly from those treated in routine clinical practice. Real-world patients may be older, present with multiple comorbidities, receive concomitant medications, and face challenges related to healthcare access, adherence, and long-term disease management.
As innovative therapies reach broader populations, healthcare stakeholders increasingly need answers to questions that clinical trials alone cannot fully address:
- How effective are therapies in routine clinical practice?
- Which patient populations benefit most?
- What are the long-term outcomes?
- How do treatments impact quality of life?
- What is the burden on healthcare systems?
These questions have elevated the importance of Real-World Data and Real-World Evidence as complementary sources of clinical insight.
Real-World Evidence Takes Center Stage
One of the most visible trends at EHA 2026 was the growing number of real-world evidence studies presented across multiple disease areas. Examples included:
- Paroxysmal Nocturnal Haemoglobinuria (PNH)
- Immune Thrombocytopenia (ITP)
- Hemophagocytic Lymphohistiocytosis (HLH)
- Myelofibrosis
- Haemophilia von Willebrand disease, and other inherited bleeding disorders
These studies examined treatment patterns, clinical outcomes, disease burden, healthcare utilization, and patient experiences outside controlled clinical trial environments.
The message was clear: evidence generation does not end when a therapy receives regulatory approval. Real-world evidence is increasingly essential for understanding how innovations perform in everyday clinical practice and for informing clinical guidelines, reimbursement decisions, and healthcare policy.
The New EU HTA Framework Creates New Evidence Requirements
Since becoming applicable in January 2025, the EU Health Technology Assessment (HTA) Regulation has significantly increased the focus on patient-centered outcomes and real-world evidence as key components of reimbursement, market access, and healthcare decision-making across Europe.
The new framework represents an important shift in how innovative therapies are assessed, strengthening collaboration between EU Member States through Joint Clinical Assessments while placing greater emphasis on outcomes that matter to patients and healthcare systems. Beyond demonstrating efficacy in clinical trials, healthcare stakeholders increasingly seek evidence on comparative effectiveness, long-term outcomes, quality of life, treatment pathways, and healthcare resource utilization in routine clinical practice.
As a result, high-quality Real-World Data (RWD) and Real-World Evidence (RWE) are expected to play an increasingly important role in complementing clinical trial findings and supporting more informed, patient-centered healthcare decisions throughout the product lifecycle.
Healthcare decision-makers increasingly seek evidence on:
- Comparative effectiveness
- Long-term outcomes
- Quality of life
- Real-world treatment patterns
- Healthcare resource utilization
While clinical trials remain fundamental, they cannot always provide complete answers to these questions. As a result, high-quality RWD and RWE are expected to play an increasingly important role throughout the product lifecycle, supporting reimbursement, market access, and post-launch evidence generation.
Integrating the Patient Voice into Clinical Advancement
Another major theme at EHA 2026 was the growing recognition that patients should play a more active role in defining treatment success.
Historically, clinical endpoints such as response rates, progression-free survival, and overall survival have dominated clinical research.
Today, these outcomes remain essential, but they are increasingly complemented by patient-reported outcomes and measures that capture the lived experience of disease.
Patients often prioritize outcomes such as:
- Daily functioning
- Fatigue
- Ability to work and maintain social activities
- Treatment convenience
- Emotional well-being
- Overall quality of life
The integration of patient perspectives is helping create a more holistic understanding of treatment value and aligns closely with broader healthcare trends toward patient-centered and value-based care.
Von Willebrand Disease: A Powerful Example of the Value of Real-World Data
One of the most compelling examples discussed during EHA 2026 involved Von Willebrand Disease (VWD), the most common inherited bleeding disorder worldwide.
Despite being described more than a century ago, VWD remains substantially underdiagnosed. Many patients experience symptoms for years before receiving a definitive diagnosis.
Patients may present with:
- Recurrent nosebleeds
- Heavy menstrual bleeding
- Easy bruising
- Gastrointestinal bleeding
- Surgical bleeding complications
- Iron deficiency anaemia
- Chronic fatigue
The complexity of VWD contributes to these diagnostic challenges. Disease severity can vary significantly between patients, symptoms may fluctuate over time, and laboratory results can be influenced by numerous biological and environmental factors.
Traditional clinical studies often struggle to capture this complexity.
Real-world data offers a unique opportunity to better understand the disease as it exists in routine clinical practice.
By analysing longitudinal healthcare records, laboratory results, physician notes, and patient-reported outcomes, researchers can identify diagnostic patterns, quantify disease burden, and better understand patient journeys.
Importantly, RWD can help reveal opportunities for earlier diagnosis by identifying common pathways and warning signs that precede diagnosis. This evidence can support more proactive disease recognition, improve care pathways, and ultimately reduce the burden experienced by patients.
The VWD example highlights a broader opportunity across hematology: using healthcare data not only to evaluate treatments but also to improve diagnosis, disease management, and patient outcomes.
From Data to Better Outcomes
A recurring message throughout EHA 2026 was that the future of hematology will not be built on scientific innovation alone.
It will depend on the ability to connect multiple sources of evidence, including:
- Clinical trial data
- Real-world clinical data
- Patient-reported outcomes
- Biomarker and genomic information
- Healthcare utilization data
- Digital health technologies
The goal is not to replace randomized clinical trials but to complement them, creating a more complete picture of how therapies perform in real-world healthcare settings.
As healthcare systems increasingly embrace value-based care and implement the new EU HTA framework, organizations capable of transforming healthcare data into actionable evidence will play a critical role in supporting better decisions and improving patient outcomes.
EHA 2026 demonstrated that the future of evidence generation is no longer simply about proving that treatments work. The challenge now is understanding how they work for real patients, in real healthcare systems, and how evidence can be used to improve care across the entire patient journey.
EHA 2026: Questions & Answers
Why is Real-World Evidence becoming so important in hematology?
Because clinical trials alone cannot fully represent the diversity and complexity of patients treated in routine practice. RWE helps clinicians, researchers, regulators, and payers understand how therapies perform in broader populations and real healthcare settings.
How does RWE complement clinical trials?
Clinical trials demonstrate efficacy and safety under controlled conditions. RWE evaluates effectiveness, treatment patterns, long-term outcomes, quality of life, and healthcare resource utilization in everyday clinical practice.
What is driving the increased focus on RWD and RWE in Europe?
The implementation of the new EU HTA Regulation, together with growing interest in value-based healthcare, is increasing demand for evidence that reflects real-world patient outcomes and healthcare system impact.
Why is patient voice becoming more important?
Patients often value outcomes that are not fully captured by traditional clinical endpoints, such as fatigue, daily functioning, emotional well-being, treatment burden, and quality of life. These perspectives are increasingly informing clinical research and healthcare decision-making.
Why was Von Willebrand Disease highlighted at EHA 2026?
VWD illustrates how real-world data can help uncover diagnostic delays, quantify disease burden, and better understand patient journeys. It provides a powerful example of how healthcare data can support earlier diagnosis and more patient-centered care.
How can artificial intelligence support RWE generation?
AI and natural language processing can extract valuable information from unstructured healthcare data sources such as physician notes, pathology reports, laboratory reports, and discharge summaries, transforming them into research-ready evidence at scale.
What was the overarching message from EHA 2026?
The future of hematology will be shaped not only by breakthrough therapies but also by the ability to generate meaningful evidence from real-world clinical practice, integrate patient perspectives, and translate innovation into measurable improvements in patient outcomes.
