Observational studyBMJ (Clinical research ed.)2018
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.
Observational study in BMJ (Clinical research ed.), 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 1 synthesis or guideline pooled it.
- National Comprehensive Cancer Network Guideline Recommendations of Cancer Drugs With Accelerated Approval.JAMA network open · 2023Guideline
- Association of National Cancer Institute-Sponsored Clinical Trial Network Group Studies With Guideline Care and New Drug Indications.JAMA network open · 2019Trial
- The lifecycle and evolution of new regimens on the National Comprehensive Cancer Network Guidelines for newly diagnosed multiple myeloma.The oncologist · 2025Article
- Clinical value of guideline recommended molecular targets and genome targeted cancer therapies: cross sectional study.BMJ (Clinical research ed.) · 2024Article
- Pathways for non-manufacturers to drive generic drug repurposing for cancer in the U.S.Frontiers in pharmacology · 2024Article
- Use of suboptimal control arms in randomized clinical trials of investigational cancer drugs in China, 2016-2021: An observational study.PLoS medicine · 2023Observational
- Real-world treatment outcomes of medicines used in special situations (off-label and compassionate use) in oncology and hematology: A retrospective study from a comprehensive cancer institution.Cancer medicine · 2023Article
- Evidence of clinical benefit of WHO essential anticancer medicines for children, 2011-2021.EClinicalMedicine · 2023Article
- An urgent call to raise the bar in oncology.British journal of cancer · 2021Review
- Assessment of Drugs Approved by Public Knowledge-Based Applications (Kouchi-shinsei) During the Last Two Decades in Japan.Clinical pharmacology and therapeutics · 2021Article
- Regulatory and clinical consequences of negative confirmatory trials of accelerated approval cancer drugs: retrospective observational study.BMJ (Clinical research ed.) · 2021Observational
- Association of Supporting Trial Evidence and Reimbursement for Off-Label Use of Cancer Drugs.JAMA network open · 2021Article
- Inclusion of Performance Parameters and Patient Context in the Clinical Practice Guidelines for Heart Failure.Journal of cardiac failure · 2021Article
- Status Update on Data Required to Build a Learning Health System.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2020Article
- Estimation of Percentage of Patients With Fibroblast Growth Factor Receptor Alterations Eligible for Off-label Use of Erdafitinib.JAMA network open · 2019Article
- When is off-label off-road?Annals of oncology : official journal of the European Society for Medical Oncology · 2019Article
- Level of evidence used in recommendations by the National Comprehensive Cancer Network (NCCN) guidelines beyond Food and Drug Administration approvals.Annals of oncology : official journal of the European Society for Medical Oncology · 2019Article
- Adaptive, Iterative, Long-Term Personalized Therapy Management in a Case of Stage IV Refractory NSCLC.Journal of personalized medicine · 2019Article
- Cancer drug development: The missing links.Experimental biology and medicine (Maywood, N.J.) · 2019Review
- Estimation of the Percentage of US Patients With Cancer Who Benefit From Genome-Driven Oncology.JAMA oncology · 2018Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
Abstract
objectiveTo 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.
designRetrospective observational study.
settingNational Comprehensive Cancer Network and FDA.
participants47 new molecular entities approved by the FDA between 2011 and 2015.
main outcome measuresComparison 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.
results47 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.
conclusionThe 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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.