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Background and Objectives <p>The value of new therapies depends on the magnitude of their clinical benefit. In multiple myeloma (MM), numerous drugs have been approved in the past two decades, yet the extent of clinical benefit at authorization remains uncertain. This study evaluated MM drugs approved by the European Medicines Agency (EMA), the Food and Drug Administration (FDA), and the Brazilian Health Regulatory Agency (Anvisa).</p> Methods <p>This retrospective cohort study assessed MM drugs approved between 2003 and 2024. Data were extracted from publicly available regulatory documents. Clinical benefits were assessed using the European Society for Medical Oncology Magnitude of Clinical Benefit Scale for Haematological Malignancies (ESMO-MCBS:H v 1.1) based on pivotal trials. Descriptive statistics, Kaplan–Meier curves and log-rank tests were performed.</p> Results <p>During the study period, the EMA approved 17 drugs for MM, with 11 (64.7%) supported by single-arm studies and 4 (23.5%) through accelerated assessment. The FDA also approved 17 drugs, with 12 (70.6%) based on single-arm studies, 11 (64.7%) through expedited pathways, and 8 (47.1%) designated as Breakthrough Therapies. Anvisa approved 12 drugs, with 7 (58.3%) under priority registration. According to the ESMO-MCBS:H v1.1 scoring, only 4 of 17 drugs (23.5%) demonstrated meaningful clinical benefit, defined as a grade ≥4 in the non-curative setting. Accelerated timelines in Brazil were significantly associated with biologicals, monoclonal antibodies, and Breakthrough Therapy status (all p <i>&lt;</i> .05).</p> Conclusion <p>Most MM therapies approved entered the market with limited evidence of meaningful clinical benefit. These findings support using the ESMO-MCBS:H to guide regulatory and reimbursement decisions in resource-constrained settings.</p>

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Clinical Benefit of Multiple Myeloma Drugs at Regulatory Approval in Brazil, Europe, and the USA: A Retrospective Cohort Study (2003–2024)

  • 期刊论文

  • 发表日期：

    2026年4月21日

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  • Marina Alacoque Rodrigues,
  • Cristiane Aparecida Menezes de Pádua,
  • Paula Lana de Miranda Drummond,
  • Pedro Henrique Carvalho de Souza,
  • Jéssica Soares Malta,
  • Adriano Max Moreira Reis

摘要

Background and Objectives

The value of new therapies depends on the magnitude of their clinical benefit. In multiple myeloma (MM), numerous drugs have been approved in the past two decades, yet the extent of clinical benefit at authorization remains uncertain. This study evaluated MM drugs approved by the European Medicines Agency (EMA), the Food and Drug Administration (FDA), and the Brazilian Health Regulatory Agency (Anvisa).

Methods

This retrospective cohort study assessed MM drugs approved between 2003 and 2024. Data were extracted from publicly available regulatory documents. Clinical benefits were assessed using the European Society for Medical Oncology Magnitude of Clinical Benefit Scale for Haematological Malignancies (ESMO-MCBS:H v 1.1) based on pivotal trials. Descriptive statistics, Kaplan–Meier curves and log-rank tests were performed.

Results

During the study period, the EMA approved 17 drugs for MM, with 11 (64.7%) supported by single-arm studies and 4 (23.5%) through accelerated assessment. The FDA also approved 17 drugs, with 12 (70.6%) based on single-arm studies, 11 (64.7%) through expedited pathways, and 8 (47.1%) designated as Breakthrough Therapies. Anvisa approved 12 drugs, with 7 (58.3%) under priority registration. According to the ESMO-MCBS:H v1.1 scoring, only 4 of 17 drugs (23.5%) demonstrated meaningful clinical benefit, defined as a grade ≥4 in the non-curative setting. Accelerated timelines in Brazil were significantly associated with biologicals, monoclonal antibodies, and Breakthrough Therapy status (all p < .05).

Conclusion

Most MM therapies approved entered the market with limited evidence of meaningful clinical benefit. These findings support using the ESMO-MCBS:H to guide regulatory and reimbursement decisions in resource-constrained settings.