ArticleJournal of clinical oncology : official journal of the American Society of Clinical Oncology2013
Prevalence of off-label use and spending in 2010 among patent-protected chemotherapies in a population-based cohort of medical oncologists.
Article in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 58 papers, 2 of them syntheses that pooled it.
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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.
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Who cites it
58 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- National Comprehensive Cancer Network Guideline Recommendations of Cancer Drugs With Accelerated Approval.JAMA network open · 2023Guideline
- Genomic Heterogeneity and Exceptional Response to Dual Pathway Inhibition in Anaplastic Thyroid Cancer.Clinical cancer research : an official journal of the American Association for Cancer Research · 2017Pooled it
- Variation in guideline decision-making: a cross-sectional study of oncology compendium panels.Health affairs scholar · 2026Article
- Second-Level Appeals of Denied Anticancer Medication Claims in Medicare Part D.JAMA network open · 2026Article
- A Taxonomy for Assessing Real-World Targeted Cancer Therapy Options in the Context of Broad Genomic Profiling.Journal of the National Comprehensive Cancer Network : JNCCN · 2026Article
- Off-label policy through the lens of trazodone usage and spending in the United States.Health affairs scholar · 2025Article
- The landscape of use of NCCN-guideline chemotherapy regimens in stage I-IIIA breast cancer in an integrated healthcare delivery system.Breast cancer research and treatment · 2024Article
- Patient Characteristics Associated with Intended Nonguideline Chemotherapy in Women with Stage I to IIIA Breast Cancer.Cancer epidemiology, biomarkers & prevention : a publication of the American Association for Cancer Research, cosponsored by the American Society of Preventive Oncology · 2024Article
- Clinical value of guideline recommended molecular targets and genome targeted cancer therapies: cross sectional study.BMJ (Clinical research ed.) · 2024Article
- Use of pembrolizumab among older adults with cancer in the United States, before and after FDA approval of its tumor-agnostic indication.Pharmacoepidemiology and drug safety · 2024Article
- Off-label use of immune checkpoint inhibitors for the treatment of solid tumors: analysis of a nationwide patient sample.Journal of cancer research and clinical oncology · 2023Article
- Nab-Paclitaxel and Gemcitabine as First-Line Treatment of Metastatic Ampullary Adenocarcinoma with a NovelBiomedicines · 2023Article
- A multinational, drug utilization study of lipegfilgrastim use in real-world setting in Europe.Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer · 2022Article
- Characteristics and survival of patients with cancer with intended off-label use-a cohort study.BMJ open · 2022Article
- Liquid Biopsy and Artificial Intelligence as Tools to Detect Signatures of Colorectal Malignancies: A Modern Approach in Patient's Stratification.Frontiers in oncology · 2022Review
- Characterization of the Early Years of Bevacizumab Use for First-Line Treatment of Ovarian Cancer in the United States.JCO oncology practice · 2021Article
- Article
- Immune Checkpoint Inhibitor Uptake in Real-World Patients With Malignant Pleural Mesothelioma.JTO clinical and research reports · 2021Article
- Association of Supporting Trial Evidence and Reimbursement for Off-Label Use of Cancer Drugs.JAMA network open · 2021Article
- Factors Associated With Off-Label Oncology Prescriptions: The Role of Cost and Financing in a Universal Healthcare System.Frontiers in pharmacology · 2021Article
Corrections and comments
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Authors and funding
6 authors.
Funding
Abstract
purposeThe prevalence of off-label anticancer drug use is not well characterized. The extent of off-label use is a policy concern because the clinical benefits of such use to patients may not outweigh costs or adverse health outcomes.
methodsPrescribing data from IntrinsiQ Intellidose data systems, a pharmacy software provider maintaining a population-based cohort database of medical oncologists, was analyzed. Use of the most commonly prescribed anticancer drugs ("chemotherapies") that were patent protected and administered intravenously to patients in 2010 was examined. Use was classified as "on-label" if the cancer site, stage, and therapy line met the US Food and Drug Administration (FDA)-approved indication. All other use was "off-label." Off-label use was divided by whether it conformed to National Comprehensive Care Network (NCCN) Compendium recommendations, a basis of insurer coverage policies. IMS Health National Sales Perspectives was used to estimate national spending by use category.
resultsTen chemotherapies met inclusion criteria. On-label use amounted to 70%, and off-label use amounted to 30%. Fourteen percent of use conformed to an NCCN-supported off-label indication, and 10% of off-label use was associated with an FDA-approved cancer site, but an NCCN-unsupported cancer stage and/or line of therapy. Total national spending on these chemotherapies amounted to $12 billion (B; $7.3B on-label, $2B off-label and NCCN supported; $2.5B off-label and NCCN unsupported).
conclusionCommonly used, novel chemotherapies are more often used on-label than off-label in contemporary practice. Off-label use is composed of a roughly equal mix of chemotherapy applied in clinical settings supported by the NCCN and those that are not.
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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.