CLINICAL OUTCOMES RESEARCH ISSUES

HEALTH CARE POLICY DEVELOPMENT USING OUTCOMES RESEARCH ISSUES

IS NICE ALL THAT NASTY? COMPARISONS OF ACCESS TO CANCER THERAPY IN UK AND US HEALTH CARE
Moderator: Lee N Newcomer MD, Senior Vice President, Oncology, UnitedHealthcare, Edina, MN, USA.
Panelist(s): Mike F Drummond PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, Heslington, UK; Scott Ramsey MD, PhD, Associate Member, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Dennis W Raisch PhD, Associate Center Director, Department of Veterans Affairs Cooperative Studies Program, Clinical Research Pharmacy Coordinating Center, Albuquerque, NM, USA.
ISSUE: Would the guidance issued by the National Institute for Clinical Excellence (NICE) in the United Kingdom lead to restrictions in access to cancer drugs if implemented in the United States?
OVERVIEW: One of the concerns about the spread of health technology assessment in the United States is that it will lead to restrictions in access to approved therapies. The decisions of NICE in the UK are often cited as examples of the rationing of care that would be unpopular and perhaps unacceptable in the US. In this session, NICE guidance on new cancer therapies will be compared and contrasted with the corresponding formulary decisions of major public payers in the US.
Specifically, we will discuss the timing of NICE guidance and US health insurer coverage decisions in relation to regulatory approval by each country's regulatory agency. In addition, the coverage rules and restrictions, if any, imposed on therapies for given indications, and trends in restrictions or openness in coverage will be compared. Ultimately, we address the question: Would the implementation of NICE guidance in the US negatively influence access to therapy for US cancer patients.

NOW WHAT FOR GENOMICS? TURNING PROMISE INTO PRACTICE
Moderator: Clifford Goodman PhD, Senior Vice President, The Lewin Group, Falls Church, VA, USA.
Panelist(s): Deborah Marshall PhD, Vice President, i3 Innovus, Global Health Economics and Outcomes, Burlington, ON, Canada; Gurvaneet Randhawa MD, MPD, Medical Officer and Senior Advisor, Center for Outcomes and Evidence, Genomics & Personalized Medicine, Rockville, MD, USA; Emily S. Winn-Deen PhD, Vice President, Cepheid, Sunnyvale, CA, USA.
ISSUE: Despite the widespread promotion of genomic technologies in medical practice and public health settings, few genomic applications have demonstrated evidence-based applications. The only genomic application in primary practice that is recommended by the US Preventive Services Task Force is BRCA1/2 testing for breast and ovarian cancer susceptibility. What evidence is required to accelerate the integration of genomics into health care practice?
OVERVIEW: There is substantial interest and opportunity for conducting translation research that will move promising genomic technologies into practice. In the US, the NIH and CDC have identified the critical need for evidence-based evaluation of the use of genomic technologies to assess both the positive and negative impacts on health care outcomes and costs. The DHHS Secretary's Advisory Committee on Genetics and Health in Society (SACGHS) is releasing national reports in 2008 on clinical and public health implications of pharmacogenomics and on oversight of genetic testing. The panelists will debate the evolving pathways for translating genomic breakthroughs into practice, including evidence requirements for demonstrating clinical validity, clinical utility and cost-effectiveness and for securing market access and adequate reimbursement.

PATIENT-REPORTED OUTCOMES RESEARCH ISSUES

PATIENT-REPORTED OUTCOMES, HEALTH-STATE UTILITIES, OR STATED-PREFERENCES? SIMILARITIES, DIFFERENCES AND ROLES IN DEMONSTRATING PRODUCT VALUE
Moderator: A. Brett Hauber PhD, Senior Economist and Head, RTI Health Solutions, Health Preference Assessment, Research Triangle Park, NC, USA.
Panelist(s): Paul Kind PhD, Professor of Economics, Centre for Health Economics, University of York, York, Heslington, UK; William Furlong MSc, Research Coordinator, McMaster University and Health Utilities Inc, Hamilton, ON, Canada; F. Reed Johnson PhD*, Senior Fellow and Principal Economist, RTI International, Research Triangle Park, NC, USA.
ISSUE: The increased focus on patient-reported outcomes (PROs) in medical research in recent years has heightened awareness of the importance of patients' perspectives in assessing health care interventions. Health-state utility elicitation methods such as standard gamble (SG) and time tradeoff (TTO), usually considered to be preference-based measures of health-status assessment, and sometimes considered patient-reported outcomes in and of themselves, are widely used in cost-utility analysis. Recently, stated-preference methods including contingent valuation and conjoint analysis or discrete-choice experiments have been used increasingly to quantify patient preferences for health and health care. Often, however, there is misunderstanding as to the similarities and differences among methods and how these different patient-centered measurement techniques contribute to outcomes research. For example, patient-reported outcomes often are used to quantify health outcomes, but do not measure patient preferences. Some health-state utility measurement techniques apply published preference weights to patient-reported health-status measurements to calculate multi-attribute health-state utilities. In some cases, conventional health-state utility measurement techniques are unable to capture the impact of acute conditions, treatment risks, or process-related factors such as method of administration or treatment duration. Stated-preference measures can quantify preferences for both health outcomes and process-related factors, but importance weights may not be generalizable across therapeutic areas.
OVERVIEW: PROs, health-state utilities, and stated-preference assessment were developed to answer different questions but similarities among these approaches have created confusion about the appropriate role of each method in outcomes research. In this panel, experts in each of these fields, Paul Kind (PRO), William Furlong (health-state utilities), and Reed Johnson (stated-preference assessment) will clarify the roles of these methods and discuss the extent to which these methods are redundant, complementary, or possibly contradictory in demonstrating the value of health care interventions. *Panelist represent members of the ISPOR PRO SIG: Patient Preference Methods – Conjoint Analysis Working Group: John FP Bridges PhD (Chair); Brett Hauber PhD; Joel Hay PhD; F. Reed Johnson PhD; Emily Lancsar; Prof.dr. Maarten J. Ijzerman; Andrew Lloyd PhD; Deborah Marshall PhD; Ebere Akobundu Onukwugha PhD; Lisa A. Prosser PhD; Dean Andrew Regier PhD

CLINICAL OUTCOMES RESEARCH ISSUES

HEALTH CARE POLICY DEVELOPMENT USING OUTCOMES RESEARCH ISSUES

WHAT DID THE MEDICARE REPLACEMENT DRUG DEMONSTRATION TEACH US ABOUT THE ROLE OF COST-EFFECTIVENESS ANALYSES IN PUBLIC POLICY?
Moderator: Penny E Mohr MA, Director, Division of Research on Health Plans and Drugs, Centers for Medicare and Medicaid Services, Office of Research, Development and Information, Baltimore, MD, USA.
Panelist(s): William Lawrence MD, MS, Research Fellow, Agency for Healthcare Research and Quality, Center for Outcomes and Effectiveness Research, Rockville, MD, USA; Allan Wailoo BSc, MA, PhD, Senior Lecturer in Health Economics, The University of Sheffield, Health Economics and Decision Science, School of Health and Related Research, Sheffield, UK; Martin Zagari MD, Global Health Economics Head, Amgen, Thousand Oaks, CA, USA.
ISSUE: How can we improve upon the way cost-effectiveness studies are done to better serve public policy?
OVERVIEW: The Medicare Replacement Drug Demonstration provided coverage for selected drugs and biologics just prior to the introduction of the Medicare prescription drug benefit. As part of the evaluation of this demonstration, the Centers for Medicare and Medicaid Services funded two cost-effectiveness studies. The studies raised many issues including: 1) the strengths and weaknesses of complex models; 2) how best to communicate the results of analyses; 3) how best to portray uncertainty to policymakers; and 4) the role of stakeholders and the nature of the review process. This issues panel brings together three different viewpoints (industry, the reviewing organization, and the academic group who completed the studies) to discuss lessons learned from these studies. The panel will examine the implications of these findings and debates whether we can improve on ways cost-effectiveness analyses are done to better serve public policy.

QUANTITATIVE APPROACHES TO REGULATORY RISK-BENEFIT ASSESSMENT FOR FDA DECISION-MAKING: WHAT WOULD WORK?
Moderator: James T. Cross MS, Graduate Student, University of Washington, Pharmaceutical Outcomes Research and Policy Program, Seattle, WA, USA.
Panelist(s): F. Reed Johnson PhD, Senior Fellow and Principal Economist, RTI International, Research Triangle Park, NC, USA; Larry D. Lynd PhD, Assistant Professor, University of British Columbia, Faculty of Pharmaceutical Sciences, Vancouver, BC, Canada; Louis P. Garrison PhD, Professor of Pharmacy, University of Washington, Department of Pharmacy, Seattle, WA, USA.
ISSUE: Given recent concerns about the adequacy of FDA's regulatory review process, how should FDA evaluate alternative quantitative methods for drug risk-benefit assessment?
OVERVIEW: In the wake of the withdrawals of several widely marketed drug products, IOM and CIOMS reports charged the US Food and Drug Administration's (FDA) process for evaluating drug risks and benefits with being too subjective, inconsistent, and lacking transparency. In response, the US FDA hosted a working group meeting in late 2007 consisting of approximately 70 senior FDA and foreign regulators, academics, patient groups, physicians, and manufacturers to discuss possible institutional and analytical responses to this criticism. Six quantitative approaches to risk-benefit assessment were identified as possible alternatives: (1) Number Needed to Treat/Number Needed to Harm, (2) Incremental Net Health Benefits, (3) Health Outcomes Modeling with Quality-Adjusted Life-Years, (4) Stated Preference Risk-Benefit Trade-Offs, (5) Multi-Criteria Decision Analysis, and (6) Semi-Quantitative Structured Frameworks. Regulators, analysts, and industry representatives now are charged with determining which quantitative approaches, if any, could best aid in regulatory risk-benefit assessment. Each panelist presented a specific alternative at the FDA meeting and is participating in the ongoing FDA initiative to evaluate options. They will debate the pros, cons, and feasibility of potential approaches and the design of future research needed to test and validate the methods. Some key considerations for these policy alternatives include: transparency, objectivity, and adaptability.

PRACTICAL CONSIDERATIONS ON COVERAGE WITH EVIDENCE DEVELOPMENT: HOW WILL THE PIPER BE PAID?
Moderator: Stuart MacLeod MD, PhD, FRCPC, Executive Director, Children's & Women's Health Centre of British Columbia, Vancouver, BC, Canada.
Panelist(s): Mike F Drummond PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, Heslington, UK; Sean Tunis MD, MSc, Executive Director, Center for Medical Technolgy Policy, Baltimore, MD, USA; Pierre Philippe Sagnier MD, Vice President Global Health Economics, Bayer Schering Pharma, Outcomes and Reimbursement, Wuppertal, Germany.
ISSUE: There is increased interest in post-launch research as more jurisdictions require cost-effectiveness data beyond those available at the time of launch in the context of formal decision processes for listing and reimbursement of drugs. Given these developments, what are the practical considerations that need to be considered if regulators implement coverage with evidence development (CED) policies?
OVERVIEW: Many countries now require clinical effectiveness and cost-effectiveness data to support formal decision processes for listing and reimbursement of drugs. In the USA, the Center for Medicare and Medicaid Services (CMS) has introduced innovative approaches to encourage such data collection, by issuing special coverage status to encourage industry to gather additional data to supplement standard claims data as a condition of payment. Many other countries are also considering ‘conditional reimbursement' as a policy tool that will allow ongoing reimbursement of a drug to be linked to a future performance measure. Although listing and reimbursement authorities may call for post-launch data collection, they rarely specify who should carry out or fund the work. If reimbursement authorities are interested in encouraging manufacturers to collect additional data to help inform reimbursement decisions, there are many practical issues that need to be considered. Important issues include: 1) identifying the conditions when CED policies are appropriate and warranted; 2) establishing a consensus on the most appropriate methods (registry versus RCT-based studies); 3) creating positive incentives for manufacturers to invest in these programs; 4) determining appropriate success/ failure measures for programs; and 5)

PATIENT-REPORTED OUTCOMES RESEARCH ISSUES

QALYS GONE WILD?
Moderator: Peter I. Juhn MD, MPH, Vice President, Evidence and Regulatory Policy, Johnson & Johnson, New Brunswick, NJ, USA.
Panelist(s): Adrian Griffin MsC, Vice President, Strategic Affairs, LifeScan, Inc, High Wycombe, Buckinghamshire, UK; Peter J. Neumann ScD, Professor, Tufts-New England Medical Center, Institute for Clinical Research and Health Policy Studies, Boston, MA, USA; Michael Schlander, MD, MBA, Professor, Institute for Innovation & Valuation in Health Care (InnoVal-HC), University of Heidelberg, Eschborn, Germany.
ISSUE: This panel will explore the use and misuse of the QALY in cost-effectiveness analyses by highlighting specific product examples, their cost/QALY issues and related reimbursement decisions.
OVERVIEW: Why has the QALY become the defector standard outcomes metrics used in cost-effectiveness analyses? What are the potential alternatives to the QALY and what are the conditions under which these alternative measures might be the more appropriate measure? Are there fundamental differences between therapeutic technologies (i.e. drugs vs. devices) that make outcomes comparisons using QALYs, particularly in an ICER setting, unreliable? How do health- related cultural values impact the use of QALYs, including their relevance? The QALY methodology applies different weights to the degree of disutility experienced by patients. This has the advantage of allowing comparisons across a wide variety of disease states but assumes that the standard weighting used is both accurate and relevant to the question being posed. The key challenges with this approach are: 1) therapies that are primarily designed to improve the Quality of Life and improve functionality are weighted much less heavily than therapies that increase the duration of life; 2) when the period of disability is fairly brief before the intervention is performed, even if the disability is severe, this also has an adverse effect on the weighting accorded by the QALY approach; and 3) unless a new form of therapy can be shown to directly impact mortality, it may be deemed “ not worth paying for”. Moreover, the policy context within which QALYs are used can be problematic; particularly when ICER calculations are repeated after market maturation has readjusted the relative prices of the comparators. The unresolved regulatory issues of PRO/QOL claims create additional challenges. The panelists will illustrate these issues with a medical device example and a drug example and then discuss potential alternative approaches.