Systems for Research and Evaluation for Translating Genome-Based Discoveries for Health Workshop Summary (2009) / Chapter Skim
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4 Current Practices in Moving from Evidence to Decision
4 Current Practices in Moving from Evidence to Decision
Pages 33-52
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From page 33... ...
RARE DISEASE MODEL James Perrin, M.D. Harvard Medical School and Massachusetts General Hospital Center for Child and Adolescent Health Policy The Evidence Review Workgroup provides timely information to the Secretary's Advisory Committee on Heritable Disorders in Newborns and Children to guide their recommendation decisions for adding conditions to uniform newborn screening panels.
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From page 34... ...
The Advisory Committee may choose to send the nomination to the Evidence Review Workgroup to carry out a more in-depth evidence review for that particular condition. The workgroup then reports back through the Maternal and Child Health Bureau to the Advisory Committee, which then makes its recommendations to the Secretary of Health and Human Services.
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From page 35... ...
are a main area of interest, but one for which in nearly all cases few data exist. Evidence Review Methodology As described above, the workgroup developed evidence questions, many of which apply broadly across conditions, although specific questions within a particular condition always arise, Perrin said.
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From page 36... ...
Other key questions involve the test itself: • How does the newborn screening test work? • What are the characteristics of the test?
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Moderator Burke asked Perrin about the decision to establish the explicit and formal separation of the evidence review from the process of making recommendations, noting that other processes often do not do this. Perrin said the statutory authority rests with the Secretary's Advisory Committee, which was developed in response to the Children's Health Act of 2000.
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From page 38... ...
R01538 Ginsburg said many of those recommendations could also apply to the vector, editable translation of genome-based technologies. Such changes would include the scaled as landscape above establishment of coordinated, perhaps centralized, biobanks with standards portrait below both for sample handling and informatics; the aggregation of genomic technologies into core facilities accessible to investigators; the development of interoperable informatics systems, including electronic health records and molecular, clinical, and imaging data; increasing the cadre of skilled biostatisticians and improving physician training in quantitative skills; and better Biomarker Clinical Assay Biomarker Clinical Trial Clinical Practice Clinical Practice Discovery Confirmation Development Validation Implementation Implementation Adoption DNA/RNA/ Leads Research Candidate Validated Clinical Clinical Protein Assay Marker Marker Decision Utility Tool Confirmed Lead Clinical Validated Clinical Clinical Clinical Markers Candidate Assay Marker Decision Utility Adoption GXP GXP Tool GXP GXP GXP 8 - 80+ years FIGURE 4-1 The translational continuum for biomarkers.
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From page 39... ...
Prospective genome-guided clinical trials are one means to develop the evidence required for clinical adoption. A prototype for such clinical utility studies is to consider areas where the current standard of practice is a choice between two or more therapies or combinations of therapies, where based on the clinical data there is clinical equipoise.
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From page 40... ...
Ginsburg cited an ongoing clinical trial being conducted by Duke and Eli Lilly on advanced-stage, non-small-cell lung cancer, for which the standard of care is a combination of cisplatin and gemcitabine. Individuals predicted to be sensitive to platinum-based therapies (including cisplatin)
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From page 41... ...
The genomic technology groups address assay standardization, ensure compliance with the Clinical Laboratory Improvement Amendments, develop the bioinformatics and algorithms necessary to deliver genomic information to the clinical trial, and establish longitudinal genomics data and sample repositories. Ginsburg offered a variety of approaches in addition to the prospective trials that were discussed that could further enable evaluation of genomic markers.
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From page 42... ...
To really drive clinical adoption of potentially valuable genetic tests into the Duke system, this type of clinical decision support tool needs to be integrated into the computerized order entry process. At this time, GTAC is focusing primarily on pharmacogenetic tests, and tests that are included in the label of an FDAapproved drug.
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The concept leads to clinical trials, the results of which can be used to generate guidelines and performance indicators, after which the concept enters clinical use.
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NCDR is a suite of hospital- and office-based registries and quality improvement programs focused on measuring outcomes and identifying gaps in the delivery of quality cardiovascular patient care. The mission of NCDR is to improve care, provide knowledge and tools, implement quality initiatives, and support research.
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From page 45... ...
The structure of NCDR is such that each program has a steering committee, a quality improvement subcommittee, and a research and publications committee. Each component reports to the NCDR Management Board and the Clinical Quality Council, which in turn are responsible to the ACC board of trustees.
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From page 46... ...
NCDR has developed a robust risk adjustment model that can be used to develop patient-centered consent forms that offer outcomes risk assessment based on the patient's clinical scenario to help the physician and patient make decisions. NCDR data are also being used to assess the safety and efficacy of performing angioplasty in facilities without onsite cardiac surgical facilities.
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From page 47... ...
NCDR and Genomics A variety of issues need to be considered regarding use of NCDR to aid translation of genomic technologies. NCDR operates under a quality improvement model that does not require Institutional Review Board (IRB)
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An increasing number of states are mandating participation to oversee quality, particularly related to angioplasty at sites without onsite cardiac surgical facilities. For some other registries, the financial models are weak.
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From page 49... ...
The question of whether these tests might be subject to regulatory oversight is one of the uncertainties that still pervades the field, Ginsburg responded. A key question is understanding whether the test is considered high risk and will require a prospective clinical trial in order to prove its clinical value or clinical validity, or whether it is low risk and could be subject to a lower bar, such as a 510k submission (allowed to market by demonstrating substantial equivalence to a device that has been already cleared for marketing by the FDA)
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From page 50... ...
She asked the panel to comment on limitations created by how data is recorded, and what registry data enable us to understand. Brindis said the quality of registry data is much higher than the quality of administrative data, which he said has greater challenges in terms of accuracy, particularly related to co-morbid conditions and other clinical descriptors that would be important in the genomics field.
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From page 51... ...
He noted that there are several ongoing, prospective clinical trials that will hopefully establish more definitive evidence as to whether these tests should be done. The warfarin example should be viewed as a test case for how to develop a system to integrate genetic testing information and decision making into physician ordering.
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