Research
The Ethics of Health Technology Appraisal
Summary: This line of research and policy development seeks to address the ethical challenges associated with the appraisal for insurance coverage of new drugs, devices, and procedures. The appraisal of new health technologies has been a controversial flashpoint where the goals of access, innovation, and cost containment have often been in conflict. Although much of the process of appraisal is based on an analysis of scientific evidence of the effectiveness of new technologies, many aspects of technology appraisal include important, if sometimes less visible, ethical components.
Specific questions addressed by this line of research include: how to balance consideration of possible risks and benefits of new technologies; how to manage the tension between concerns for safety and effectiveness with patient desires for broad access to new technologies; how to judge and apply perspectives of the value of new technologies when these perspectives differ between patients, clinicians, and payers; and how to consider the ethical ramifications of the dominant utility-maximizing perspective of cost-effectiveness analysis. The overall objective of this line of research is to provide empirical and normative analyses of approaches to balancing access, innovation, and cost control in support of an equitable and sustainable health care system.
| Section: | Ethics and Health Policy | ||||||||||
| Principal Investigator: | Steven D. Pearson, M.D., M.Sc. | ||||||||||
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Background: At a time of rapid innovation, increasing health care premiums, and growing patient responsibility for health care costs, all health care decision makers need better evidence on the value of new health care technologies. Policy makers and proponents of health care system reform thus routinely argue that cost control and improved value within the health care system will require more explicit appraisal of the clinical effectiveness and cost-effectiveness of new medical technologies.
Yet the process of appraising new technologies in order to make decisions regarding insurance coverage and reimbursement carries with it important ethical dimensions whose delineation and consideration remain very controversial. The controversies arise due to the complex interplay between the goals of quality of care improvement, technological innovation, and cost control.
The major policy issues that arise can be categorized as pertaining to either the technical methods of technology appraisal or to the overall political process in which technology appraisal is embedded. The methods of technology appraisal have developed along a technical path dominated by the principles of evidence-based medicine. Some appraisal efforts also include formal efforts to judge value by using cost-effectiveness models to produce estimates of the marginal benefit of the new technology as measured by the cost per life year gained, or per quality-adjusted life year gained. None of the methods of evidence-based medicine and of cost-effectiveness analysis are free from inherent considerations of ethical values, including relative weights given to risks and benefits, reasonable boundaries of uncertainty in evidence, and considerations of the relative severity and “need” of patients with different illnesses.
Departmental Research Initiative: The ethics of health technology appraisal can be seen as a component of the general departmental effort in resource allocation, but with the arrival of Steve Pearson in 2005 this line of policy development and research gained a lead investigator and increasing visibility within the department.
Dr. Pearson’s work in this area began when he was at Harvard as part of his early collaborative efforts with the department that led to the birth of the Center for Ethics in Managed Care (CEMC) at Harvard Medical School. Launched in 1996, and co-directed by Dr. Pearson and Dr. Emanuel, the CEMC played an important role as an “honest broker” at a time when the ethical, legal, and policy challenges over managed care became particularly intense. The CEMC hosted several national conferences on ethical and professional challenges confronting the health care system in the United States, allowing a venue for researchers, health care leaders, and policy advocates to meet to exchange views and ideas for constructive policy development.
The CEMC’s most notable research endeavor was in the area of organizational ethics in managed care. In 2003 Pearson and Emanuel, working with other colleagues at NIH and at Harvard authored the book, No Margin, No Mission: Health Care Organizations and the Quest for Ethical Excellence, published by Oxford University Press. For two consecutive years this book was highlighted as the standard text at the annual meeting of the Organizational Ethics Interest Group within the American Society of Bioethics and Humanities. One of its chapters was dedicated to the problems faced by private insurers of making “medical necessity” decisions, the decisions of whether a new technology would be covered by insurance or not. Presenting several distinctive approaches to technology appraisal found among private health plans, this chapter was highlighted at several conferences as an important research effort that framed the key evidentiary and ethical issues for future policy development.
Growing interest in the ethical and scientific issues associated with technology appraisal led to Dr. Pearson's receiving an Atlantic Fellowship to spend a year as a Senior Fellow at the National Institute for Clinical Excellence (NICE), the body within the National Health Service of England that makes all of its national coverage decisions. At NICE, Dr. Pearson led several initiatives, including one examining the integration of societal values into appraisal decisions. Interest in bringing to Medicare in the US lessons from the experience both in managed care and in the UK led to the creation of a shared post for Dr. Pearson in Washington after his year at NICE. In 2005, therefore, while on continued leave from Harvard, he came to Washington with a dual role: Special Advisor, Technology and Coverage Policy, at the Centers for Medicare and Medicaid Services (CMS); and as Visiting Senior Scientist in the Department of Bioethics. The goal of establishing a split position was to provide an intellectual and practical bridge between the work of the Department and that of the policy makers at CMS who were now beginning to wrestle with various new approaches to technology appraisal.
During the first year 2005-2006 the research and policy development initiative on technology appraisal encompassed two distinct areas of emphasis:
1) A National Institute for Clinical Excellence (NICE) for the US?
Interest in the methods and organizational structure of NICE has been growing in the United States for some time as it became clear that NICE had achieved a status as the acknowledged world leader in health technology appraisal. For JAMA, Dr. Pearson wrote the first article in the United States medical literature summarizing NICE as an organization and analyzing its applicability to the U.S. setting. This publication has been frequently cited and led to invitations to speak at many conferences and seminars, particularly in Washington, DC where many policy makers have been keen to discuss NICE as one example of what the United States might do to improve the value of its health care system. At these conferences much attention has been paid to the role NICE accords cost-effectiveness analysis in its decisions. The use of cost-effectiveness raises important ethical issues related to its reliance on a utilitarian model that weighs the sum of health benefits across a population without attention to how those health benefits are distributed. Thus this area of research and policy development includes key ethical issues that often overlap with scientific and political considerations.
Dr. Pearson’s ongoing work in relation to NICE also led to a publication exploring the options for using technology appraisal not only to evaluate new technologies but to identify obsolete or less effective therapies in current use. This emerging application of technology appraisal, called “disinvestment” in the UK, overlaps extensively with efforts in the United States to use variation in the utilization of certain tests and treatments to identify inappropriate use, both overuse and underuse. Again, in the application of technology appraisal for this purpose there are many ethical issues that arise, including consideration of how to weigh public and patient values when technologies are being evaluated for removal from the health care system.
2) The Ethics of Coverage with Evidence Development (CED)
One central problem underlying the application of evidence-based analytic approaches to medical policy decisions is the dearth of high quality evidence. Therefore, one central policy mechanism to develop better evidence on the risks, benefits, and costs of new technologies is to link insurance coverage with a requirement that patients participate in relevant research, including registries and clinical trials. Medicare has introduced its own version of this approach, called “coverage with evidence development” (CED), and has used it in several high-profile coverage decisions. But the policy has been seriously questioned as being coercive and unfair. Dr. Pearson, as a bridge between Medicare and the Department, was in an ideal position to recognize the importance of the ethical component to the debates over CED, and he led an effort to write a paper that appeared in JAMA analyzing the ethical basis for CED. This article argued strongly that CED, if properly based on clear evidence thresholds, was not coercive because Medicare beneficiaries had no entitlement to new technologies that did not make the evidence threshold to be considered eligible for unlimited coverage. This article is the first in the bioethics or health policy literature to address the ethical issues of linking insurance coverage to requirements for participation in research, and has achieved wide international attention as many other nations are in the midst of their own fledgling attempts to build systems to gain ongoing evidence on the safety and effectiveness of new medical treatments. Dr. Pearson co-authored another article, this time in Health Affairs, that addressed the historical context for the CED policy and reiterated the importance of framing CED within a clear set of evidentiary thresholds determining insurance coverage decisions. Further work to better define these thresholds and their integration with ethical concerns is the focus of a future research initiative discussed below.
Future Research Initiatives: Both of the two previously described areas of emphasis continue to provide opportunities for further research and policy development. In addition, two other areas of research have been emerged within this portfolio of activities.
1) The Evidence-based Roadmap Project
Dr. Pearson is now leading a multi-stakeholder project whose goal is to create a new framework to guide evidence-based medical policy decisions. Participants include America's Health Insurance Plans (AHIP), Merck and Co., Johnson and Johnson, Aetna, Boston Scientific, and the Agency for Health Care Quality and Research (AHRQ). This project will create a clear and consistent terminology for the assessment of scientific evidence on the effectiveness of new technologies, and then provide a normative guide to the integration of “contextual considerations” into a final medical policy decision such as coverage or reimbursement. The contextual considerations will include cost, equity, severity of illness, and other issues that are important components of any medical policy decision but which have for too long operated in an opaque fashion. The broad participation of key stakeholders will lend significant weight and visibility to the product of this project.
2) The Institute for Clinical and Economic Review (ICER)
In June 2006, Dr. Pearson was awarded a grant from the Blue Shield Foundation of California to launch the Institute for Clinical and Economic Review (ICER) to develop and test new methods of technology appraisal for US decision-makers.
Pearson has also recently received grant support to develop and test a new format for technology assessment that can integrate clinical and cost-effectiveness reviews. The use of cost-effectiveness in policy decisionmaking has raised many ethical and political issues in the past, and Pearson’s project is an explicit attempt to forge a new method that can overcome these difficulties and provide decisionmakers with the information on health care value that can support a more equitable and sustainable health care system.
Publications:
Since 2005 Pearson Arrival in the Department
Pearson SD, Miller F, Emanuel EJ. Medicare’s requirement for research participation as a condition of coverage: Is it ethical? JAMA. 2006;Aug 23;296(8):988-91.
Tunis SR, Pearson SD. Coverage options for promising technologies: Medicare's “coverage with evidence development.” Health Aff (Millwood). 2006;Sep-Oct;25(5):1218-30.
Pearson SD, Littlejohns P. The challenge of disinvestment: Methods for NICE to support cost-saving efforts in the NHS. Journal of Health Services Research & Policy. 2006. (In Press).
Pearson SD, Rawlins MD. Quality, innovation, and value for money: NICE and the British National Health Service. JAMA .2005;294:2618-2622.
Earlier Related Publications
Pearson SD, Sabin JE, Emanuel EJ. No Margin, No Mission: Health Care Organizations and the Quest for Ethical Excellence in Competitive Markets (New York: Oxford University Press, 2003).
Pearson SD. Patient reports of coverage denial: Association with ratings of health plan quality and trust in physician. American Journal of Managed Care. 2003;9:238-244.
Randel L, Pearson S, Sabin J, Hyams T, Emanuel EJ. How Managed Care Can Be Ethical. Health Affairs. 2001;20:43-56.
Emanuel E. Justice and Managed Care: Four Principles for the Just Distribution of Health Care Resources. Hastings Center Report. 2000;30:8-16.
Major Presentations since July 2005
Ethics and Evidence: A New Model for Technology Appraisal. Invited plenary presentation at the annual conference of the National Institute for Health and Clinical Excellence. London, 2006.
Allocating Health Care Resources: The American Experience. Panel presentation to the annual meeting of the German National Ethics Council. Berlin, 2006.
Is the Evidence Adequate for Coverage? A New Framework of Evidentiary Standards. Biannual meeting of the International Society for Priorities in Health Care. Toronto, 2006.
Paying for Emerging Therapies: New Trends in How Private and Public Payers Make Coverage Decisions. Avalere Health and Health Affairs national audio conference. Washington, DC, 2006.
The Learning Healthcare System: Implications for Standards of Evidence. Invited panel presentation at The Learning Healthcare System workshop of the Institute of Medicine Roundtable on Evidence-based Medicine. Washington, DC, 2006.
Health Care Bridges to Nowhere? NICE for the US. Invited panel presentation at Does the United States Need a NICE? Perspectives on the UK model of drug reimbursement. American Enterprise Institute. Washington, DC, 2006.
Evidence and Medicare Coverage: Old Statutes and New Trends. Invited panel presentation at the Avalere Evidence Conference. Washington, DC, 2006.
What relevance for NICE in the US? Invited presentation at a meeting on Integrating Cost-Effectiveness into Health Policy Decisions, Agency for Health Care Research and Quality. Washington, DC, 2006.
Technology Assessment in the United States. Invited panel presentation at Health Industry Forum's conference: Future Strategies for U.S. Technology Assessment. Washington, DC, 2006.
Regulator/Payer Viewpoints: Re-thinking Regulation and Health Technology Assessment. Invited plenary presentation to the Health Technology Assessment International Policy Forum. Leesburg, Virginia, 2005.
Disinvesting in Ineffective Practice: Concentrating on What Works. Invited panel presentation at the annual conference of the National Institute for Health and Clinical Excellence. Birmingham, England, 2005.
