Multiple myeloma (MM) is a rare and complex blood cancer that affects plasma cells in the bone marrow. These malignant cells accumulate and interfere with normal blood cell production, leading to complications such as anemia, kidney dysfunction, bone lesions, and increased susceptibility to infections. The disease affects a significant number of people worldwide, with estimates of around 188,000 new cases annually.
While significant advancements in MM treatment have improved patient outcomes, challenges persist. Despite the introduction of innovative therapies, patients continue to face obstacles such as delayed access to novel treatments, variability in therapeutic adoption across regions, and the complexities of managing relapsed or refractory disease. Regulatory considerations further exacerbate these disparities, leaving some patients without timely access to life-extending therapies. Understanding how treatment patterns have evolved and their impact on survival outcomes is crucial in addressing these ongoing issues.
TriNetX Oncology: Providing A Data-Driven Look at MM Care
At the recent American Society of Hematology annual meeting (ASH 2024), my colleague, Guillaume Azarias, Senior Data Scientist at TriNetX Oncology, and I presented findings from our study titled: “Evolution of Treatment Patterns and Survival Outcomes in European Patients With Multiple Myeloma From 2012-2023 Through the HONEUR Federated Data Network.” This large-scale analysis provides valuable insights into how MM treatment has evolved over the years and the real-world impact of these changes on patient outcomes.
Key Findings from the Study Fueled by Real-World Data
Conducted under the umbrella of the HONEUR network, this study analyzed real-world data (RWD) from more than 29,000 newly diagnosed MM patients across five registries in France, Germany, the Czech Republic, and the UK. Spanning 11 years, the research categorized patients into three cohorts: 2012–2015, 2016–2019, and 2020–2023, the latter influenced by the challenges of the COVID-19 pandemic.
The study highlighted a significant shift in frontline MM treatment patterns.
Over time, there was a clear transition from proteasome inhibitor-based regimens to the increasing adoption of anti-CD38-based therapies. By 2023, nearly 40 percent of patients were receiving anti-CD38 regimens as frontline therapy, a stark rise from just 1.2 percent in 2012. However, the rate of adoption varied by country; while France and Germany saw rapid uptake (63 percent and 30 percent, respectively), access in the Czech Republic and the UK lagged due to delayed regulatory approvals and reimbursement policies.
Alongside these changes in treatment patterns, MM survival outcomes also improved.
Median overall survival increased significantly, with the 2012–2015 cohort reaching 75 months, while the 2016-2019 and 2020–2023 cohorts’ median survivals were not yet reached. Time to next treatment similarly improved, growing from 29.6 months in the earliest cohort to 32.4 months in the most recent group. These trends were most evident in France and Germany, where earlier access to anti-CD38 regimens and lenalidomide maintenance therapy contributed to better outcomes. In contrast, survival benefits were less pronounced in the Czech Republic and the UK due to treatment access delays.
Implications for Future MM Care
This study underscores the critical role of timely access to innovative therapies in improving patient outcomes. The disparities observed between countries highlight the urgent need for more uniform regulatory pathways and reimbursement frameworks to ensure equitable treatment access across Europe.
The research also demonstrates the power of real-world evidence (RWE) and collaborative data-sharing networks such as HONEUR. By leveraging large-scale RWD while maintaining stringent privacy standards, we can generate meaningful insights that complement clinical trial findings and inform future treatment strategies.
At TriNetX, we hope to contribute to the growing body of evidence that supports equitable access to innovative therapies for all MM patients. The study results not only highlight the strides made in treatment but also underscore the challenges that remain in ensuring that all patients, regardless of location, can benefit from advancements in care.
Discover how TriNetX’s non-U.S. oncology RWD and services are benefiting clinical cancer research and download a sample report for multiple myeloma.
About Markus Rückert, PhD
Markus is a seasoned biopharmaceutical expert specializing in oncology and rare diseases. Proficient in clinical development and Medical Affairs, as well as experimental and RWE generation, Markus has successfully navigated various stages of drug development—from discovery to market launch—with a keen focus on advancing oncological and rare disease therapies for tangible patient benefits. Having achieved significant milestones in oncology and rare disease therapeutics, Markus now channels his diverse expertise towards broader pursuits in epidemiology, health service research, and the real-world impact of oncological treatments.
