Genome-Based Diagnostics Clarifying Pathways to Clinical Use: Workshop Summary (2012) / Chapter Skim
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2 Calls for Change
2 Calls for Change
Pages 5-16
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From page 5... ...
• Venture capital companies are no longer investing in the devel opment of molecular diagnostic tests because of the complexity in and lack of clarity for both regulatory and reimbursement pathways. • A predictable and efficient pathway, not necessarily an easier one, from regulatory approval to reimbursement could help attract further venture capital investment in this space.
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From page 6... ...
. This undervaluation has led to limited use of these diagnostics by health care providers and poor reimbursement when a marker has been able to navigate the regulatory environment to be brought to market.
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From page 7... ...
Hayes focused his recommendations for breaking the "vicious cycle" of undervalued tumor biomarkers on two areas: the regulatory environment and marker reimbursement. Requiring FDA Approval of Laboratory Developed Tests LDTs can currently be introduced into clinical practice while only meeting Clinical Laboratory Improvement Amendments (CLIA)
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From page 8... ...
That's against the law, and I think it should be against the law to develop a new assay and use it to treat my patients differently without having had it vetted by some regulatory body." TABLE 2-1 Comparison of CLIA and FDA Regulatory Pathways CLIA FDA Research Phase No Yes Analytical Validation Post hoc sampling Yes Clinical Validation No Yes Report Adverse Events No requirement; no system Yes Transparent Results No public information Published review summary NOTE: CLIA, Clinical Laboratory Improvement Amendments; FDA, U.S. Food and Drug Administration.
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From page 9... ...
Instead of including clinical validity and intended use in their assessment, he suggested that FDA should review diagnostics for analytic validity and clinical utility. While he acknowledged this will increase the time and resources needed to get FDA approval, tests will have demonstrated clinical value for patients upon entrance to clinical practice.
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From page 10... ...
Instead of a standing board, ad hoc committees of experts without a "corporate memory" review devices, which means that "there is no consistent approach toward how one device is approved versus the other." Hayes did note that while the ODAC has significant oncologic expertise it lacks analytical expertise and proposed combining the review of all oncologic products into a single FDA Oncology Office. Combining all oncologic products into a single office would require a fundamental reorganization of FDA, Hayes observed, which is a substantial obstacle to moving forward on this recommendation.
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From page 11... ...
" Many earlier tests were developed with support from venture capital, including the Oncotype DX breast cancer assay for predicting chemotherapeutic benefit and metastasis risk, the MammaPrint assay for the risk of metastasis following breast cancer surgery, and the HER-2/neu test for directing Herceptin treatment of women with metastatic breast cancer. More broadly, Siegel observed, the venture capital community plays a crucial role in the economy, spurring innovation that benefits the quality and efficiency of the health care system.
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From page 12... ...
Figure 2-3 The Venture Capital Process Siegel gave a brief overview of the venture capital process. It begins with limited partners (LPs)
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From page 13... ...
As an early stage investor, the firm expects a life science or health care company to have a capitalefficient business model, to produce a product, and to be generating revenue within 3 to 5 years. It also prefers the company to have a strong intellectual property position, limited regulatory risks, a clear path toward reimbursement, and a convincing health economics model (a strong rationale for why a product could help the whole system of health care decrease costs)
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From page 14... ...
5 100 Total VC Investment (Billions) 4 80 3 60 2 40 1 20 0 0 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 Biotechnology Medical Devices Health Care Services Total FIGURE 2-5 Venture capital investments in the life sciences and health care have declined significantly in recent years.
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From page 15... ...
This will allow venture capital firms to continue to support health care entrepreneurs who bring innovative ideas and business models that can help transform our current health care system into one that offers improved quality of care and increased access at lower costs." 1 The Roundtable on Translating Genomic-Based Research for Health held a prior workshop on July 22, 2010, titled Establishing Precompetitive Collaborations to Stimulate GenomicsDriven Product Development, which examined the value, utility, and ethical challenges in using biospecimens in developing medical products, including diagnostics (IOM, 2011a)
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