Genome-Based Diagnostics Demonstrating Clinical Utility in Oncology: Workshop Summary (2013) / Chapter Skim
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3 Perspectives from Stakeholders
3 Perspectives from Stakeholders
Pages 11-28
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From page 11... ...
• A provisional period of several years during which payers cover part of the costs of a molecular diagnostic's use would allow additional evidence to be gathered, after which the test could be accepted only if it produces substantial improvements in health outcomes. • Situations may arise when a randomized controlled clinical trial is not needed for the approval, use, and reimbursement of a biomarker, but these situations must be chosen with great care.
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From page 12... ...
Clinical guideline developers Clinical guidelines are not prescriptions for care, said Al Benson, professor of medicine and associate director for clinical investigations at the Robert H Lurie Comprehensive Cancer Center, Northwestern University.
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From page 13... ...
An increase in patient numbers may result in the development of stronger evidence for an intervention, but will also lead to increased expenses for screening eligible populations. An increased reliance on tumor banks of appropriate tissues, whether metastatic or primary, will become greater as well, perhaps with serial biopsies and the use of multiple markers over time to deal with tumor heterogeneity.
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From page 14... ...
. Guideline Development in the National Comprehensive Cancer Network The National Comprehensive Cancer Network (NCCN)
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From page 15... ...
Clinical trials become complicated as populations are continuously subdivided, adding such expenses as screening for marker positive and negative individuals and evaluating markers over time to judge whether tumor biology is changing. "That comes at enormous cost.
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From page 16... ...
Development of Guidelines by the American Society of Clinical Oncology Like Benson, Gary Lyman, professor of medicine and director of comparative effectiveness and outcomes research–oncology at the Duke University School of Medicine and the Duke Cancer Institute, observed that clinical practice guideline recommendations face particular challenges with molecular diagnostics. Many tests are already in existence; new tests and data are emerging rapidly; and analytic validity, clinical validity, and clinical utility all have to be established.
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From page 17... ...
A major challenge, said Lyman, is to learn within the evidence-based structure established by the IOM and ASCO to appraise and update oncology biomarkers to enhance their trustworthiness and impact on clinical practice. As an example of how impact on practice can be measured, Lyman briefly described the Quality Oncology Practice Initiative (QOPI)
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From page 18... ...
ASCO is also looking at quality measures, rapid systematic reviews for guidelines panels, and a point-of-care guide on regimen benefits, toxicities, and costs. The goal, said Lyman, is "to bring the membership and practicing oncologists real-time data -- updated, current, yet properly validated and assessed by an expert group -- as they care for their patients." Additional barriers involve the lack of awareness, slow dissemination of new recommendations into clinical practice, inadequate access to the guidelines, reluctance to accept guidelines, and lack of accountability.
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From page 19... ...
The goals of the evidence reviews during the guidelines development process are to permit flexibility of choice, find the balance point that maximizes patient benefits but maintains accountability for health care expenditures, ensures the ability of patients to participate in clinical trials, integrates cancer care with physicians' workloads, and remains current. Flexibility is particularly important, Everson emphasized.
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From page 20... ...
Analyses have shown that the pathways can save money without adversely affecting outcomes. According to Everson, level 1 pathways can reduce variation in patient care, improve the predictability of costs for health plans, offer up-to-date clinical tools for documentation and reporting, prepare oncologists to succeed in pay-for-performance relationships, and demonstrate fiscal responsibility to patients and payers.
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From page 21... ...
Also, the customers of the payers, most of which are self-funded businesses, would need to agree to such a system, because they would be the ultimate funders of such an approach. Finally, current health care legislation limits payers to using 15 percent of premium revenues for administrative costs,2 and if the provisional funding were considered an administrative cost rather than a medical cost, it probably would not be a viable option.
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From page 22... ...
According to Medicare, a demonstration of clinical utility requires that a test or intervention improve patient outcomes by such measures as better functional status, improved quality of life, reduced disability, or changes in the physician's management of a patient. But published evidence for clinical utility or evidence-based decisions is lacking.
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From page 23... ...
He proceeded from insights into a particular disease to more general observations about where randomized controlled trials of clinical utility are needed. About 22,280 new cases of epithelial ovarian cancer4 occur annually in the United States, with about 15,500 deaths despite advances in surgery and chemotherapy.
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From page 24... ...
Biomarkers can help distinguish malignant from benign pelvic masses. A risk-of-malignancy index for ovarian cancer was developed in 1990 that incorporates the biomarker CA125, ultrasound, and menopausal status, providing a sensitivity of 71 to 88 percent and specificity of 74 to 97 percent (Jacobs et al., 1990)
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From page 25... ...
. With Karen Lu at MD Anderson and in collaboration with seven different sites, Bast has participated in a smaller trial evaluating the use of CA125 and transvaginal ultrasound in postmenopausal women at average risk for developing ovarian cancer.
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From page 26... ...
Several potential candidate biomarkers exist, but given the potential toxicity and cost of treatment, a test with high negative predictive value would be very useful, Bast noted. These treatment issues raise the more general question of whether accurate prediction of failure to respond to a toxic drug is adequate evidence of clinical utility, or are prospective, randomized controlled trials required to validate biomarkers or panels of biomarkers?
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From page 27... ...
Collyar said, "I regularly get the question, ‘Why should we support cancer research if they are just going to produce drugs that nobody can afford? '" Finally, little information is available about the clinical utility of molecular tests even after a decade or more of use (Sparano and Solin, 2010)
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From page 28... ...
In addition, test results need to be updated quickly as the test or a person's condition changes, with medical personnel receiving adequate training to keep up to date with rapidly changing technologies. Patients also need to have the choice to receive test results because some people will want to know a result even if no intervention is available, but others will not.
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