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Frequency and level of evidence used in recommendations by the National Comprehensive Cancer Network guidelines beyond approvals of the US Food and Drug Administration: retrospective observational study.
The BMJ ( IF 93.6 ) Pub Date : 2018-03-07 , DOI: 10.1136/bmj.k668 Jeffrey Wagner 1 , John Marquart 1 , Julia Ruby 1 , Austin Lammers 2 , Sham Mailankody 3 , Victoria Kaestner 2 , Vinay Prasad 4, 5, 6
The BMJ ( IF 93.6 ) Pub Date : 2018-03-07 , DOI: 10.1136/bmj.k668 Jeffrey Wagner 1 , John Marquart 1 , Julia Ruby 1 , Austin Lammers 2 , Sham Mailankody 3 , Victoria Kaestner 2 , Vinay Prasad 4, 5, 6
Affiliation
Objective To determine the differences between recommendations by the National Comprehensive Cancer Network (NCNN) guidelines and Food and Drug Administration approvals of anticancer drugs, and the evidence cited by the NCCN to justify recommendations where differences exist.
Design Retrospective observational study.
Setting National Comprehensive Cancer Network and FDA.
Participants 47 new molecular entities approved by the FDA between 2011 and 2015.
Main outcome measures Comparison of all FDA approved indications (new and supplemental) with all NCCN recommendations as of 25 March 2016. When the NCCN made recommendations beyond the FDA’s approvals, the recommendation was classified and the cited evidence noted.
Results 47 drugs initially approved by the FDA between 2011 and 2015 for adult hematologic or solid cancers were examined. These 47 drugs were authorized for 69 FDA approved indications, whereas the NCCN recommended these drugs for 113 indications, of which 69 (62%) overlapped with the 69 FDA approved indications and 44 (39%) were additional recommendations. The average number of recommendations beyond the FDA approved indications was 0.92. 23% (n=10) of the additional recommendations were based on evidence from randomized controlled trials, and 16% (n=7) were based on evidence from phase III studies. During 21 months of follow-up, the FDA granted approval to 14% (n=6) of the additional recommendations.
Conclusion The NCCN frequently recommends beyond the FDA approved indications even for newer, branded drugs. The strength of the evidence cited by the NCCN supporting such recommendations is weak. Our findings raise concern that the NCCN justifies the coverage of costly, toxic cancer drugs based on weak evidence.
中文翻译：
国家综合癌症网络指南在建议中使用的证据的频率和水平超出了美国食品和药物管理局的批准：回顾性观察研究。
目的确定国家综合癌症网络（NCNN）指南与食品和药物管理局批准的抗癌药物之间的建议之间的差异，以及NCCN引用的证据来证明存在差异的建议之间的差异。
设计回顾性观察研究。
建立国家综合癌症网络和FDA。
参加者2011年至2015年间，FDA批准了47种新的分子实体。
主要结果指标截至2016年3月25日，所有FDA批准的适应症（新的和补充的）与所有NCCN建议的比较。当NCCN提出的建议超出FDA的批准时，该建议进行分类并注明引用的证据。
结果对2011年至2015年间FDA最初批准的用于成人血液学或实体癌的47种药物进行了检查。这47种药物被批准用于FDA批准的69种适应症，而NCCN建议将这些药物用于113种适应症，其中69种（62％）与69种FDA批准的适应症重叠，另外44种（39％）是其他推荐。超出FDA批准适应症的推荐平均数为0.92。23％（n = 10）的额外建议基于随机对照试验的证据，而16％（n = 7）则基于III期研究的证据。在21个月的随访期间，FDA批准了14％（n = 6）的其他建议。
结论NCCN经常建议使用FDA批准的适应症以外的药物，甚至对于较新的品牌药物也是如此。NCCN引用的支持此类建议的证据不足。我们的发现引起了人们的关注，即NCCN基于证据不足而证明昂贵，有毒的癌症药物的覆盖范围是合理的。
更新日期：2018-03-08
Design Retrospective observational study.
Setting National Comprehensive Cancer Network and FDA.
Participants 47 new molecular entities approved by the FDA between 2011 and 2015.
Main outcome measures Comparison of all FDA approved indications (new and supplemental) with all NCCN recommendations as of 25 March 2016. When the NCCN made recommendations beyond the FDA’s approvals, the recommendation was classified and the cited evidence noted.
Results 47 drugs initially approved by the FDA between 2011 and 2015 for adult hematologic or solid cancers were examined. These 47 drugs were authorized for 69 FDA approved indications, whereas the NCCN recommended these drugs for 113 indications, of which 69 (62%) overlapped with the 69 FDA approved indications and 44 (39%) were additional recommendations. The average number of recommendations beyond the FDA approved indications was 0.92. 23% (n=10) of the additional recommendations were based on evidence from randomized controlled trials, and 16% (n=7) were based on evidence from phase III studies. During 21 months of follow-up, the FDA granted approval to 14% (n=6) of the additional recommendations.
Conclusion The NCCN frequently recommends beyond the FDA approved indications even for newer, branded drugs. The strength of the evidence cited by the NCCN supporting such recommendations is weak. Our findings raise concern that the NCCN justifies the coverage of costly, toxic cancer drugs based on weak evidence.
中文翻译：
国家综合癌症网络指南在建议中使用的证据的频率和水平超出了美国食品和药物管理局的批准：回顾性观察研究。
目的确定国家综合癌症网络（NCNN）指南与食品和药物管理局批准的抗癌药物之间的建议之间的差异，以及NCCN引用的证据来证明存在差异的建议之间的差异。
设计回顾性观察研究。
建立国家综合癌症网络和FDA。
参加者2011年至2015年间，FDA批准了47种新的分子实体。
主要结果指标截至2016年3月25日，所有FDA批准的适应症（新的和补充的）与所有NCCN建议的比较。当NCCN提出的建议超出FDA的批准时，该建议进行分类并注明引用的证据。
结果对2011年至2015年间FDA最初批准的用于成人血液学或实体癌的47种药物进行了检查。这47种药物被批准用于FDA批准的69种适应症，而NCCN建议将这些药物用于113种适应症，其中69种（62％）与69种FDA批准的适应症重叠，另外44种（39％）是其他推荐。超出FDA批准适应症的推荐平均数为0.92。23％（n = 10）的额外建议基于随机对照试验的证据，而16％（n = 7）则基于III期研究的证据。在21个月的随访期间，FDA批准了14％（n = 6）的其他建议。
结论NCCN经常建议使用FDA批准的适应症以外的药物，甚至对于较新的品牌药物也是如此。NCCN引用的支持此类建议的证据不足。我们的发现引起了人们的关注，即NCCN基于证据不足而证明昂贵，有毒的癌症药物的覆盖范围是合理的。
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