London School of Economics and Political Science, London, England.
Harvard Medical School, Boston, Massachusetts3Division of Hematology-Oncology, Department of Medicine, Massachusetts General Hospital, Boston.
JAMA Oncol. 2017 Mar 1;3(3):382-390. doi: 10.1001/jamaoncol.2016.4166.
There is a dearth of evidence examining the impact of newly licensed cancer medicines on therapy. This information could otherwise support clinical practice, and promote value-based decision-making in the cancer drug market.
To evaluate the comparative therapeutic value of all new cancer medicines approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) between 2003 and 2013.
DESIGN, SETTING, AND PARTICIPANTS: We used a narrative synthesis approach to systematically synthesize and analyze English, French, and Australian health technology assessments (HTAs) of all new cancer medicines licensed in the United States and Europe between 2003 and 2013.
Sixty-two new molecular entities with a primary oncology indication.
Overall survival (OS), quality of life (QoL), and safety.
Of the 62 new active cancer molecules approved by the FDA and EMA between 2003 and 2013, 53 were appraised by English, French, or Australian HTA agencies through May 2015. Of these 53 drugs, 23 (43%) increased OS by 3 months or longer, 6 (11%) by less than 3 months, and 8 (15%) by an unknown magnitude; there was no evidence to suggest that the remaining 16 (30%) increased OS over best alternative treatments. Where overall survival gains could be quantified, all new cancer drugs were associated with a mean (SE) total increase in OS of 3.43 (0.63) months over the treatments that were available in 2003. Drug-related improvements in OS were, however, widely distributed across therapeutic targets-ranging between 0 (thyroid, ascites) and 8.48 months (breast cancers)-and were sometimes based on modeled data, indirect or nonactive comparisons, or nonvalidated evidence. Although 22 (42%) of 53 new medicines were associated with an increase in QoL, 24 (45%) were also associated with reduced patient safety. Of the 53 new cancer drugs, 42 (79%) were associated with at least some improvement in OS, QoL, or safety.
Although innovation in the oncology drug market has contributed to improvements in therapy, the magnitude and dimension of clinical benefits vary widely, and there may be reasons to doubt that claims of efficacy reflect real-world effectiveness exactly. These findings raise important questions for clinical decision-making and value-based policy.
目前缺乏有关新批准的癌症药物对治疗影响的证据。这些信息可以支持临床实践，并促进癌症药物市场基于价值的决策。
评估 2003 年至 2013 年间，美国食品和药物管理局（FDA）和欧洲药品管理局（EMA）批准的所有新癌症药物的相对治疗价值。
设计、地点和参与者：我们采用叙述性综合方法，系统地综合分析了 2003 年至 2013 年间在美国和欧洲获得许可的所有新癌症药物的英文、法文和澳大利亚健康技术评估（HTA）。
62 种新的分子实体，主要用于肿瘤学。
总生存期（OS）、生活质量（QoL）和安全性。
在 2003 年至 2013 年间，FDA 和 EMA 批准的 62 种新的活性癌症分子中，有 53 种通过 2015 年 5 月前的英语、法语或澳大利亚 HTA 机构进行了评估。在这 53 种药物中，有 23 种（43%）将 OS 延长了 3 个月或更长时间，6 种（11%）延长了不到 3 个月，8 种（15%）延长了未知的时间；没有证据表明其余 16 种（30%）能延长 OS 超过最佳替代治疗。在能够量化总生存获益的情况下，所有新的癌症药物与 2003 年可获得的治疗相比，总生存平均（SE）增加了 3.43（0.63）个月。然而，药物相关的 OS 改善广泛分布于治疗靶点之间，范围在 0（甲状腺、腹水）到 8.48 个月（乳腺癌）之间，并且有时基于模型数据、间接或非活性比较或未经验证的证据。尽管 53 种新药物中有 22 种（42%）与 QoL 提高有关，但也有 24 种（45%）与患者安全性降低有关。在 53 种新的癌症药物中，有 42 种（79%）至少在 OS、QoL 或安全性方面有所改善。
尽管肿瘤药物市场的创新为治疗带来了改善，但临床获益的幅度和范围差异很大，并且可能有理由怀疑疗效的说法是否完全反映了实际效果。这些发现为临床决策和基于价值的政策提出了重要问题。
