Cited by 20Statistical Methods in Clinical TrialsCancer Genomics and DiagnosticsEthics in Clinical Research

The National Clinical Trials Network: Conducting Successful Clinical Trials of New Therapies for Rare Cancers

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Schott, Anne F., Welch, John J., Verschraegen, Claire F., Kurzrock, Razelle·2015·Seminars in Oncology

Abstract

Rare cancers account for 27% of neoplasms diagnosed each year, and 25% of cancer-related deaths in the United States. However, rare cancers show some of the highest response rates to targeted therapies, probably due to identification of oncogenic drivers with little interpatient variability. Although the low incidence of rare cancers makes large-scale randomized trials involving single histologies difficult to perform, drugs have been successfully developed in rare cancers using clinical trial designs that combine microscopic histologies. Such trials are being pursued within the National Clinical Trials Network (NCTN), which possesses unique qualifications to perform widespread molecular screening of tumors for patient enrollment onto therapeutic clinical trials. When larger clinical trials are needed to determine optimum treatment strategies in rare cancers, the NCTN's broad reach in North America and internationally, and their ability to partner with both United States-based and international research organizations, can make these challenging studies feasible.

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  • The observed lower female representation in HIV clinical trials depends, among other factors, on disadvantaged awareness about treatment and enrolment options compared with men.

    “The observed lower female representation in HIV clinical trials depends, among other factors, from the disadvantaged awareness about treatment and enrolment options compared with men129–131.”

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  1. [6]Synergizing Epidemiologic Research on Rare Cancers 2007 Epidemiology and Genomics Research Program NCI, National Institutes of Health Bethesda, MD (at http://epi.grants.cancer.gov/workshops/synergizing/.). September 8, 2014

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Authors
Schott, A. F., Welch, J. J., Verschraegen, C. F., & Kurzrock, R.
Affiliations
  • University of Michigan (US)
  • National Cancer Institute (US)
  • University of Vermont (US)
  • University of California San Diego (US)
Corresponding author
Schott, Anne F.
Venue
Seminars in Oncology
Publisher
Elsevier BV
Published
2015-10
Volume
42
Issue
5
Pages
731-739
ISSN
0093-7754 (ISSN-L)
OpenAlex
W2153116947
Cited by
20
References
29
Citations by year
2026: 2 · 2025: 1 · 2024: 1 · 2023: 2 · 2022: 1 · 2020: 2 · 2019: 1 · 2018: 2 · 2017: 4 · 2016: 3 · 2015: 1
FWCI
0.56
Type
journal article
Language
en
Open access
Locations
  • Seminars in Oncology (publishedVersion)
  • PubMed (publishedVersion)
  • PubMed Central (acceptedVersion)
Funders
National Cancer Institute (U10CA180888, U10CA037429)
Topics
  • Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Cancer Research › Cancer Genomics and Diagnostics
  • Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics in Clinical Research
Keywords
rare tumor, National Clinical Trials Network, clinical trial design, targeted therapy, tumor molecular profiling, oncogenic drivers, international research collaboration
MeSH terms
Clinical Trials as Topic, Cooperative Behavior, Humans, Neoplasms, Research Design, United States, Rare Diseases, Biomedical Research, National Cancer Institute (U.S.), Molecular Targeted Therapy

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