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Understanding the Efficacy-Effectiveness Gap in Multiple Myeloma Treatments: A Real-World Perspective

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Medriva Correspondents
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Understanding the Efficacy-Effectiveness Gap in Multiple Myeloma Treatments: A Real-World Perspective

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Contrasting Efficacy in Clinical Trials and Real-World Settings

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A recent Canadian study has brought to light a stark contrast in the effectiveness of standard treatments for multiple myeloma patients in clinical trials versus real-world clinical settings. The study, presented at the American Society of Hematology annual meeting, discovered a significant efficacy-effectiveness gap across multiple standard-of-care regimens for multiple myeloma. The results indicated a worse progression-free survival (PFS) and overall survival (OS) in real-world patients compared to those in randomized controlled trials (RCTs), with differences as high as 44% for PFS and 75% for OS.

The Efficacy-Effectiveness Gap

The term 'efficacy-effectiveness gap' refers to the discrepancy between the results of treatments in ideal clinical trial settings and their performance in real-world settings. This gap was evident in the study conducted by Alissa Visram MD MPH and her research team. They found the median PFS was 3 to 18 months longer and the median OS was at least 19 months longer in the clinical trial cohort compared to real-world patients. Furthermore, the risk of progression or death was 44% higher, and the risk of death was 75% higher in the real-world compared to the clinical trial setting.

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Implications of the Study

The findings of this study have significant implications for a range of stakeholders, including policymakers, clinicians, regulators, and patients. Understanding the real-world effectiveness of multiple myeloma treatments is crucial to make informed treatment decisions and to identify the key contributors to the efficacy-effectiveness gaps. Further, this understanding will help in devising strategies to overcome these gaps.

Real-World Treatment Outcomes

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The retrospective population-based study included 3,951 adults with multiple myeloma treated between January 2007 and December 2020 using seven standard-care regimens. The results showed that patients with multiple myeloma treated in real-world clinical practice had significantly shorter median PFS and worse OS compared with those treated during randomized controlled trials for six of seven regimens evaluated in the study. However, the frequency of serious treatment-related adverse events was comparable for all seven regimens included in the study.

Other Real-World Studies

Similar real-world studies have been conducted, such as the phase 2 MajesTEC 1 trial, which analyzed the effectiveness of teclistamab in the treatment of relapsed refractory multiple myeloma. The study found similar safety and efficacy to the trial, with an overall response rate of 66% and a complete response or better in 29%. Furthermore, a study on the real-world outcomes of patients with multiple myeloma refractory to anti-CD38 monoclonal antibody therapy showed a median PFS of 4.6 months and a median OS of 13.3 months, emphasizing the urgent need for the development of and access to more effective therapeutics.

Conclusion

These findings underscore the importance of understanding the real-world effectiveness of standard multiple myeloma treatments. The efficacy-effectiveness gap revealed in these studies calls for a deeper understanding of the factors contributing to it, paving the way for more effective treatment strategies for multiple myeloma patients in real-world clinical settings.

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