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ARRA Investments in Cancer: Comparative Effectiveness Research

Public Health Burden
Cancer is the second leading cause of death in the United States after heart disease. In 2010, it is estimated that more than 1.5 million new cases of invasive cancer will be diagnosed in this country and approximately 570,000 people will die of the disease.

Comparative Effectiveness Studies
Comparative effectiveness research (CER) assesses the relative impact of different options for the detection, treatment, or management of a medical condition in a specific patient population. Most experts and policymakers believe CER can provide the evidence needed to make truly informed health care decisions and optimize patient outcomes, while improving overall health care value. ARRA funding is supporting efforts to build capacity and accelerate scientific progress in the area of cancer-related CER. These efforts include building coherent teams of interdisciplinary researchers, leveraging and integrating existing data and health system research resources, and advancing measurement techniques and methodology.

The effectiveness of cancer screening and diagnostic tests in real-world settings depends not only on the efficacy of the individual tests but also on the behavior of patients and health care providers, as well as the context in which health care is delivered. ARRA-funded CER related to cancer screening and diagnosis includes:
  • A project to determine the comparative effectiveness of virtual colonoscopy (computed tomographic [CT] colonography) versus standard colonoscopy, flexible sigmoidoscopy, and fecal occult blood testing for colorectal cancer screening and surveillance.1
  • A project to develop an innovative and sustainable multidisciplinary and multi-institutional virtual center for cancer SEARCH—Screening Effectiveness and Research in Community-based Healthcare—within the NCI-funded Cancer Research Network (CRN) to 1) study approaches to the effective delivery of cancer screening in community-based settings; 2) develop methodological capacity for future large-scale, population-based CER studies; and 3) demonstrate an ability to conduct CER in the area of cancer screening to address important gaps in existing evidence.2
  • A project to analyze existing data available for two million women from the Breast Cancer Surveillance Consortium (BCSC) to assess breast cancer risk factors, mammography frequency and type, health outcomes, comorbid conditions, and healthcare utilization. The influences of screening intervals, screening modalities, and risk factors on breast cancer detection and mortality and associated costs will be determined.3
  • A project to develop a multi-institutional center for CER in cancer imaging to assess the effectiveness of advanced imaging technologies for detecting cancer and determining treatment.4
Clinicians must sometimes make cancer treatment decisions in the absence of clear information about what the best treatment might be for a given patient. ARRA-funded research is examining issues related to the comparative effectiveness of a variety of cancer treatments. For example:
  • A project to optimize the staging, surgical management, and follow-up of patients with melanoma. Various components of the project will provide histopathology quality review, molecular data for patient stratification, biostatistics support, and operation centers for two phase III clinical trials.5
  • A project to examine data from regional cancer registries, linked to Medicare enrollment and insurance claims reports, to study the characteristics of Medicare beneficiaries with myelodysplastic syndromes (MDS), patterns of treatment, safety, effectiveness of erythropoietic stimulating agent therapy, and costs of care.6
  • A project to examine data from two integrated health delivery systems to assess the risks and potential benefits of immediate androgen deprivation therapy (ADT) compared with observation (no treatment) in men diagnosed with localized disease.7
  • A project to develop a multicenter electronic breast cancer surgery outcomes database that will allow for an assessment of measures of surgical quality by examining variation in the outcomes of initial breast cancer surgery.8
Genomics and Personalized Medicine
Advances in cancer genomics and progress in identifying susceptibility genes for a wide variety of cancers are ushering in a new era of personalized cancer care and prevention. However, no systematic research has been conducted to test the clinical utility and cost-effectiveness of cancer care and prevention based on genomic tools and markers against existing standards of care and prevention not based on genome-based approaches. ARRA funding is helping to fill this gap in knowledge. For example:
  • A collaborative consortium, the Center for Comparative Effectiveness Research in Genomic Medicine (CCEGM), will be developed to generate and synthesize evidence to support the translation of genomic tests into improvements in cancer prevention, screening, diagnosis, treatment, and survivorship.9
  • A project that builds a consortium of investigators from several member sites of the NCI-funded Cancer Research Network (CRN) and academic partners to investigate the comparative effectiveness of several genomic and molecular tests related to colorectal cancer.10

  1. 5RC1CA147256-02 – CT colonoscopy screening for CRC: comparative effectiveness vs traditional models – Gazelle, G Scott (MA)
  2. 1RC2CA148576-01 – SEARCH: cancer screening effectiveness and research in community-based healthcare – Buist, Diana S.M. (WA)
  3. 1RC2CA148577-01 – Comparative effectiveness of breast imaging strategies in community practice – Miglioretti, Diana L. (WA)
  4. 1RC2CA148259-01 – Comparative effectiveness of advanced imaging in cancer – Tosteson, Anna N. (NH)
  5. 5P01CA029605-28 – New approaches to surgical oncology – Morton, Donald L. (CA)
  6. 5RC1CA145831-02 – Comparative effectiveness: erythropoietic stimulating agents in treatment of MDS – Davidoff, Amy J. (MD)
  7. 1RC1CA146238-01 – Cost-effectiveness of hormonal therapy for clinically localized prostate cancer – Van den Eeden, Stephen K. (CA)
  8. 1RC1CA145402-01 – Improving breast cancer surgery quality through a collaborative surgery database – McCahill, Laurence (MI)
  9. 1UC2CA148310-02 – Comparative effectiveness in genomic medicine – Armstrong, Katrina (PA)
  10. 1UC2CA148471-02 – Comparative effectiveness in genomic and personalized medicine for colon cancer – Goddard, Katrina (OR)

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Page Last Updated on June 30, 2018 NIH...Turning Discovery Into Health®