A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS)
A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS)
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DOI：
10.1093/annonc/mdv249
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发表时间：
2015-08-01
影响因子：
50.5
通讯作者：
Piccart, M. J.
中科院分区：
文献类型：
--
作者：
Cherny, N. I.;Sullivan, R.;Piccart, M. J.
任何新的治疗策略或治疗方法的价值都取决于其临床效益与成本的平衡程度。新治疗方案的临床获益证据来自临床研究，特别是III期随机试验，这些试验产生了关于新治疗方法的疗效、获益和安全性的无偏倚数据。到目前为止，还没有标准的工具来对癌症治疗的临床获益程度进行分级，其范围可能从微不足道（中位无进展生存期优势仅为几周）到显著（改善的长期生存期）。事实上，在缺乏标准化的方法来对临床获益的程度进行分级的情况下，从研究中得出的结论和建议经常引起激烈的争议，并且经常将非常温和的增量进展作为重大进展或“突破”进行介绍、讨论和推广。认识到对来自高质量临床试验的新治疗方法的临床获益程度进行明确和公正陈述的重要性，欧洲肿瘤医学会（ESMO）开发了一种经过验证的可重复工具来评估癌症药物的临床获益程度，ESMO临床获益程度量表（ESMO-MCBS）。该工具使用合理、结构化和一致的方法，得出新抗癌治疗预期的临床有意义获益幅度的相对排名。ESMO-MCBS是解决癌症护理价值的关键公共政策问题的重要第一步，有助于合理使用有限的公共和个人资源，以提供具有成本效益和负担得起的癌症护理。环境管理组织监测和平衡系统将是一个动态工具，其标准将定期修订。
The value of any new therapeutic strategy or treatment is determined by the magnitude of its clinical benefit balanced against its cost. Evidence for clinical benefit from new treatment options is derived from clinical research, in particular phase III randomised trials, which generate unbiased data regarding the efficacy, benefit and safety of new therapeutic approaches. To date, there is no standard tool for grading the magnitude of clinical benefit of cancer therapies, which may range from trivial (median progression-free survival advantage of only a few weeks) to substantial (improved long-term survival). Indeed, in the absence of a standardised approach for grading the magnitude of clinical benefit, conclusions and recommendations derived from studies are often hotly disputed and very modest incremental advances have often been presented, discussed and promoted as major advances or 'breakthroughs'. Recognising the importance of presenting clear and unbiased statements regarding the magnitude of the clinical benefit from new therapeutic approaches derived from high-quality clinical trials, the European Society for Medical Oncology (ESMO) has developed a validated and reproducible tool to assess the magnitude of clinical benefit for cancer medicines, the ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS). This tool uses a rational, structured and consistent approach to derive a relative ranking of the magnitude of clinically meaningful benefit that can be expected from a new anti-cancer treatment. The ESMO-MCBS is an important first step to the critical public policy issue of value in cancer care, helping to frame the appropriate use of limited public and personal resources to deliver cost-effective and affordable cancer care. The ESMO-MCBS will be a dynamic tool and its criteria will be revised on a regular basis.