Diffusion and Use of Genomic Innovations in Health and Medicine Workshop Summary (2008) / Chapter Skim
Currently Skimming:
3 Practical Incentives and Barriers to Translation
3 Practical Incentives and Barriers to Translation
Pages 25-46
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From page 25... ...
To be covered by Medicare, an item or service must be determined to be reasonable and necessary. The working definition for reasonable and necessary is that there is adequate evidence to conclude that the item or service improves net health outcomes, is generalizable to the Medicare population, and is as good as or better than currently covered alternatives.
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From page 26... ...
Furthermore, the small falsenegative rate of PET scans might lead to withholding treatment and might lead to worse outcomes than empirically treating anyone with a clinical diagnosis of dementia. In light of this, Medicare policy is to not cover PET scans for Alzheimer's disease except in the context of a prospective clinical trial that would evaluate whether the information from PET scans changed patient management in other important ways.
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From page 27... ...
The important point is that there is no national policy from Medicare about how much evidence CMS will consider adequate to conclude that there is clinical utility on this test or any molecular diagnostic. Different payers have different evidence requirements for what is sufficient to determine the clinical utility of a diagnostic test, which makes it very difficult for a company developing a product to know how to design its clinical research portfolio.
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The documents are analogous to FDA guidance documents, but FDA guidance documents articulate evidence requirements for regulatory approval. The idea behind the CMPT documents is that they should serve as companion documents to these FDA documents and articulate the specific evidence requirements for reimbursement or for coverage.
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Blue Cross and Blue Shield Association In examining evidence it is important to distinguish between three kinds of policy, Aronson said: medical policy, coverage policy, and payment policy. Medical policy is based on scientific evidence and does not consider costs or coverage issues.
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Assessments of specific emerging technologies have included gene-expression profiling of breast cancer and genetic testing for longQT Syndrome (LQTS)
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The TEC assessment found that the genetic test is accurate in identifying the presence of a mutation but that the diagnostic accuracy for identifying LQTS is not clear because there is no true gold standard for clinical diagnosis of LQTS. The assessment did find, however, that genetic testing identifies more individuals that may have LQTS than are identified through clinical diagnosis alone.
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From page 32... ...
There are many intervening variables that contribute to warfarin dosing, and there is a narrow window between an effective therapeutic dose that prevents clotting and a too-high dose that leads to bleeding. For these reasons, one needs direct evidence, and that can only be accumulated with prospective trials of dosing algorithms to compare personalized warfarin starting doses with standard dosing in terms of bleeding outcomes.
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From page 33... ...
For diagnostic tests, indirect evidence can be used if that evidence is based on performance, in which inferences can be made about clinical utility. But when there are complex associations and intervening variables, direct evidence is necessary.
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Genetic tests differ in penetrance of the genotype from low to high. Historically, most applications of medical genetics have involved high-penetrance genotypes -- that is, genotypes where, in those cases where genetic information is available, there is a great deal of certainty about what the clinical experience of a patient is going to be.
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One is hereditary non-polyposis colon cancer (HNPCC) , which has a prevalence of one person in 500 and is correlated with an 80 percent lifetime risk of colorectal cancer.
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There may be some risk if information about different members of the family is missing. Based on knowledge of colorectal cancer epidemiology, however, individuals in that middle family are likely to benefit from starting routine colon cancer screening at 40 instead of 50.
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This is the kind of situation where additional data are needed. Another issue in pharmacogenetic testing is ancillary risk information.
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From page 38... ...
Because those contributions are predicated on the goal of improving health outcomes, the technology assessment issues are analogous to the technology assessment issues for other kinds of health care and pose the same kinds of challenges. An interesting question is the extent to which tests for gene variance that are associated with increased risk will become an important modulator of either disease classification or innovative therapies.
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From page 39... ...
View from the Trenches: Challenges and Opportunities in Personalized Medicine Brad Gray Genzyme Genetics Diffusion of genomic innovations into the practice of health care through new product launches requires a balancing of economic risk and reward, Gray said. The old paradigm in medicine is a series of actions: observation of a disease condition and action to treat it; an observation of response; and then a correction if the desired response is not achieved immediately.
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Which Drug Should I Use? Breast Cancer Tamoxifen ® ER/PR Breast Cancer Herceptin ® HER2 ® Leukemia, Chronic Myelogenous Gleevec BCR-ABL Colorectal Cancer Erbitux® EGFR Lung Cancer Tarceva ® EGFR ® Leukemia,MDS Revlimid Deletion (5q)
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From page 41... ...
In 2005 Genzyme Genetics made a significant push in the field of personalized medicine, focusing explicitly on tests that could be directly tied to a therapeutic intervention. The company was aggressive in licensing technologies with early but promising clinical data that had been published in reputable journals.
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From page 42... ...
Therefore, $1,000 is the value Genzyme Genetics would assign for the value of the test. The company, however, is reimbursed based on the current procedural terminology (CPT)
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guidelines for non-small-cell lung cancer include the test, a point which Genzyme Genetics believes will help community physicians gain comfort with the utility of the test. Based on past experience, then, Genzyme Genetics has revised its criteria for bringing new personalized medicine tests to market.
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From page 44... ...
In situations where only a non-exclusive product is offered, the company will not be able to justify the investment required to perform clinical research or to navigate the regulatory system. Concerning licensors and inventors, whether in academic medical c enters or in small companies, Gray said that they must be educated about the experience of Genzyme Genetics in this area.
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From page 45... ...
Burke responded that it will be important to stratify and recognize differences in genomic medicine. When the possible result of a test is a terminated pregnancy, it is likely that there will still be a need for counseling, but the level of counseling needed will be different, for instance, in the case of a pharmacogenetic test.
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From page 46... ...
Tunis replied that, for certain clinical applications, obtaining definitive evidence of clinical utility is going to be extremely lengthy, burdensome, and costly. Part of the new paradigm may require that the payer become comfortable with reimbursement tied to indirect evidence or to some threshold of clear clinical validity plus promising evidence of clinical utility with the subsequent documentation or verification of clinical utility occurring in post-market.
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