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American Reinvestment and Recovery Act

Comparative Effectiveness Research: Recovery Act Investment Report

November 2009

Public Health Burden of Cancer

Cancer is the second leading cause of death in the United States after heart disease. In 2009, it is estimated that nearly 1.5 million new cases of invasive cancer will be diagnosed in this country and more than 560,000 people will die of the disease.

Cancer-Related CER and ARRA

Although a wide variety of cancer prevention, screening, and treatment interventions have been shown to be effective, evidence is less complete regarding the effectiveness of these interventions in actual community practice, where populations, treatment settings, and techniques and practices may differ markedly from those of the initial controlled clinical trials. Comparative effectiveness research (CER) is widely defined as an evaluation of the impact of different options for treating or managing a given condition in a particular patient group. Most health care experts and policymakers believe CER results can improve 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.

Screening/Diagnosis

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 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), a consortium of 14 health plans across the United States. Approaches to effectively deliver cancer screening to populations to enhance detection, diminish adverse effects, and, ultimately, reduce mortality will be studied.(1)
  • A project to analyze existing data on 2 million women from the Breast Cancer Surveillance Consortium (BCSC) for whom information is available on breast cancer risk factors, mammography frequency and type, health outcomes, comorbid conditions, and healthcare utilization. The influence of screening intervals, screening modalities, and risk factors on breast cancer detection and mortality and associated costs will be determined. For example, the clinical effectiveness of 1- versus 2- year mammography screening intervals for subgroups of women, the downstream health care utilization and costs of digital versus film screening mammography, and the clinical and cost-effectiveness of various breast cancer screening strategies will be compared. A prospective data collection system designed to capture clinically and scientifically relevant data on breast magnetic resonance imaging (MRI) and expansion of the BCSC Statistical Coordinating Center's capacity to design and conduct CER in community settings will also be implemented.(2)
  • A project to develop a Center for Comparative Effectiveness Research in Cancer Imaging, a multi-institutional research center. The effectiveness of advanced imaging technologies for detecting cancer and determining treatment, with attention to the consequences of incidental diagnostic findings that can result from the use of these technologies, will be studied.(3)

Treatment

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:

  • Androgen deprivation therapy (ADT) has become increasingly used as a primary treatment in older men with newly diagnosed, localized prostate cancer, despite the fact there is no proven mortality benefit from clinical trials for the use of ADT in this setting. In a comparative effectiveness study, researchers will examine data from two integrated health delivery systems to assess the risks and potential benefits of immediate ADT compared with observation (no treatment) in men diagnosed with localized disease. All-cause and prostate cancer-specific mortality and progression-free survival are the medical outcomes of interest. Longitudinal direct medical care costs, to capture the impact of ADT, and the cost-effectiveness (cost per life years saved) and cost-utility (quality-adjusted life years) of this intervention will be calculated.(4)
  • Despite the frequency of breast cancer surgery, few data are available on surgical outcomes discernable at the surgeon or hospital level. Recent measures of quality have been proposed for breast cancer surgery, but no healthcare administrative database now exists that will allow CER of initial breast cancer surgery. This is because existing databases lack sufficient detail about critical data elements that drive surgical decisions. ARRA funding is allowing researchers 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 outcomes of initial breast cancer surgery.(5)

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 Cancer Genomics (CANCERGEN), will be developed that will generate high-quality evidence supporting the clinical utility and economic value of genomic applications that are designed to assess cancer risk and prognosis and to predict drug response.(6)
  • A project to develop a comprehensive registry of biological samples from, and health and economic data for, lung and breast cancer patients treated at a single university. This information will be used to evaluate the ability of novel gene expression biomarkers to direct treatments to those most likely to respond and to reduce the toxicity associated with ineffective treatments.(7)

Selected References

  1.  1RC2CA148576-01 — SEARCH: cancer screening effectiveness and research in community-based healthcare — Buist, Diana S.M. (WA)
  2.  1RC2CA148577-01 — Comparative effectiveness of breast imaging strategies in community practice — Miglioretti, Diana L. (WA)  
  3.   1RC2CA148259-01 — Comparative effectiveness of advanced imaging in cancer — Tosteson, Anna N. (NH)
  4.   1RC1CA146238-01 — Cost-effectiveness of hormonal therapy for clinically localized prostate cancer — Van den Eeden, Stephen K. (CA)
  5.  1RC1CA145402-01- Improving breast cancer surgery quality through a collaborative surgery database — McCahill, Laurence (MI)
  6.  1RC2CA148570-01 — Center for comparative effectiveness research in cancer genomics—CANCERGEN —  Ramsey, Scott D. (WA)
  7.  1RC2CA148041-01 — Programs in clinical effectiveness of cancer pharmacogenomics — Ginsburg, Geoffrey S. (NC)