Biomarker Tests for Molecularly Targeted Therapies Key to Unlocking Precision Medicine (2016) / Chapter Skim
Currently Skimming:
Summary
Summary
Pages 1-22
The Chapter Skim interface presents what we've algorithmically identified as the most significant single chunk of text within every page in the chapter.
Select key terms on the right to highlight them within pages of the chapter.
From page 1... ...
Such tests are key to unlocking the promise of precision medicine. Biomarker tests for molecularly targeted therapies represent a crucial area of focus for developing methods that could later be applicable to other areas of precision medicine.
|
From page 2... ...
Addressing these challenges will enable biomarker tests for molecularly targeted therapies to realize their potential to advance the clinical practice of precision medicine. CONCLUSIONS The first conclusion from the committee's deliberations is that the full potential of precision medicine will not be realized without accurate, reliable, clinically useful, and appropriately implemented biomarker tests for molecularly targeted therapies.
|
From page 3... ...
Though the recommendations focus on diverse areas for improvement, they are linked together by a common understanding: properly validated, appropriately implemented biomarker tests hold the potential to enhance patient care and improve outcomes, and therefore addressing the challenges facing such tests is critical. The committee's 10 recommendations are presented as interrelated components of a rapid learning system for biomarker tests for molecularly targeted therapies (see Figure S-1)
|
From page 4... ...
7. Develop and maintain a sustainable national database for biomarker tests for molecularly targeted therapies through biomedical informatics technology to promote rapid learning for the improvement of patient care.
|
From page 5... ...
FIGURE S-1 Rapid learning system for biomarker tests for molecularly targeted therapies. NOTES: Numbers in parentheses refer to committee's recommendations.
|
From page 6... ...
, which also would involve the flow of patient biomarker test data and information into a national data repository. The committee's recommended integrated review process for coordinated regulatory, coverage, and reimbursement decisions for biomarker tests for molecularly targeted therapies also would be aligned with common evidentiary standards.
|
From page 7... ...
Common Evidentiary National Database for Biomarker Tests EHR Standards for Molecularly Targeted Therapies LIS Recommendations Ongoing Assessment of Clinical Utility of Biomarker Tests Integrated Improved Communication Strengthened Regulatory and regarding Test Performance and Laboratory Reimbursement Use through Standardized Labels Accreditation Review clinical practice rapid learning from research and Expanded Equity in Improved Processes for Enhanced Specimen Access to Biomarker Developing Clinical Standards Tests Practice Guidelines Key: Policy Environment Data Infrastructure Patient Care Processes Recommendations 1-5 Recommendations 6-7 Recommendations 8-10 FIGURE S-2 Interrelationship of components of rapid learning system for biomarker tests. NOTE: EHR = electronic health record; LIS = laboratory information system.
|
From page 8... ...
should facilitate the development of com mon clinical utility evidentiary standards that are applied for initial and ongoing coordinated regulatory, coverage, and reimbursement decisions for biomarker tests for molecularly targeted therapies. One mechanism for development of these evidentiary standards could be convening one or more independent, public–private, mul tistakeholder bodies.
|
From page 9... ...
; encourage and incorporate competency Actors/ assessments for effective use of biomarker tests for molecularly targeted therapies (8c) Organizations • Professional schools and training programs: Ensure that providers are skilled in point-of-care decision support tools (6d)
|
From page 10... ...
• HHS should continue to support ongoing refinement of common evidentiary standards as they evolve. Integrated Regulatory and Reimbursement Review The inefficiencies created by the misalignment of the regulatory and reimbursement decision processes represent a significant challenge to the effective and timely implementation of appropriate biomarker tests for molecularly targeted therapies into clinical practice.
|
From page 11... ...
Goal 2: Establish a more coordinated and transparent federal pro cess for regulatory and reimbursement decisions for biomarker tests for molecularly targeted therapies. Recommendation 2: The Secretary of the Department of Health and Human Services should facilitate the development of a new integrated federal review process involving the Food and Drug Administration and the Centers for Medicare & Medicaid Services, as a pathway for coordinated regulatory, coverage, and reimburse
|
From page 12... ...
Goal 3: Enhance communication to patients and providers about the performance characteristics and evidence for use of specific biomarker tests for molecularly targeted therapies. 2 This coordinated pathway is designed to reflect the current predominant fee-for-service reimbursement system for clinical tests.
|
From page 13... ...
. Regulatory oversight under CLIA is widely viewed as insufficient for increasingly complex biomarker tests for molecularly targeted therapies.
|
From page 14... ...
It is important that CMS and other payers develop payment models to support ongoing data collection required to establish sufficiently robust evidence to confirm the clinical utility of promising biomarker tests. These data would be instrumental for evolving payment determinations, includ Reimbursement Policy Mechanism to Support Ongoing Assessment of Biomarker Tests for Molecularly Targeted Therapies Ongoing data collection Continued Adequate coverage Investigational evidence for initial Experimental clinical utility No longer covered Analytic Clinical Validity Validity Assessing Clinical Utility Strength of Evidence High Low FIGURE S-5 Evidence continuum.
|
From page 15... ...
, which has potential to be an effective policy lever to generate evidence to support reimbursement decisions for promising technologies such as biomarker tests for molecularly targeted therapies. Goal 5: Ensure ongoing assessment of the clinical utility of bio marker tests for molecularly targeted therapies.
|
From page 16... ...
Goal 6: Ensure development and use of EHRs and related biomedi cal informatics tools and assessments that support the effective clinical use of biomarker tests for molecularly targeted therapies. Recommendation 6a: Electronic health record (EHR)
|
From page 17... ...
that facilitate point-of-care decision support for biomarker test ordering, report ing, and clinical decision making. This point-of-care decision sup port should align with available evidence-based clinical practice guidelines.
|
From page 18... ...
Goal 7: Develop and maintain a sustainable national database for biomarker tests for molecularly targeted therapies through bio medical informatics technology to promote rapid learning for the improvement of patient care. Recommendation 7: The Secretary of the Department of Health and Human Services (HHS)
|
From page 19... ...
Moreover, collaboration between community health care providers and larger health care centers or academic medical centers should be examined to determine potential impact on access for patients in remote and/or underserved areas. Goal 8: Promote equity in access to biomarker tests for molecularly targeted therapies and the expertise for effective use of the results in clinical decision making.
|
From page 20... ...
Development Processes Increasingly, a broader base of interdisciplinary expertise is needed to generate trustworthy CPGs related to complex biomarker tests. Consistent with the committee's vision of a rapid learning system, CPGs serve an important educational purpose -- both for clinical decision making as well as for test and drug labeling -- and should consider the evolving nature of evidence of clinical utility for biomarker tests for molecularly targeted therapies.
|
From page 21... ...
. The committee's proposed rapid learning system is expressly designed to promote the proper development, effective and ongoing assessment, and appropriate use of biomarker tests for molecularly targeted therapies.
|
Key Terms
This material may be derived from roughly machine-read images, and so is provided only to facilitate research.
More
information on Chapter Skim is available.