Monday, May 22nd, 11:30am-12:30pm
Issue Panels-Session I
I
IP1: WILL THE QALY SURVIVE? - Salon E & F
(Invited Issue Panel)

Moderator: Michael Drummond PhD, Professor of Health Economics, University of York, Centre for Health Economics, Heslington, York, UK

Panelists: Daniel Kahneman PhD , Eugene Higgins Professor of Psychology; Professor of Psychology and Public Affairs Woodrow Wilson School, Princeton University, Princeton, Bank of Sweden Prize in Economic Sciences in Memory of Alfred Nobel, Princeton, NJ, USA; Dennis Fryback, PhD Professor of Population Health Sciences and Industrial Engineering , Population Health Sciences, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, USA; Alistair McGuire PhD, Professor in Health Economics, London School of Economics, London, UK


HEALTH CARE REIMBURSEMENT/COVERAGE

IP2: THE NEW NATIONAL INSTITUTE OF HEALTH AND CLINICAL EXCELLENCE (NICE) SINGLE TECHNOLOGY APPRAISAL (STA) PROCESS: EXPERIENCE FROM THE FIRST ROUND OF SUBMISSIONS - Salon A

Moderator: Shahnaz Khan MPH, Senior Health Outcomes Communications Specialist, RTI Health Solutions, RTP, NC, USA
Panelist: Sorrel Wolowacz PhD, Senior Health Economist, RTI Health Solutions, United Kingdom; Mark Sculpher PhD, MSc, Professor, Centre for Health Economics, University of York, UK

ISSUE: To provide an introduction to the new STA process for submission of clinical and pharmacoeconomics evidence to NICE, to discuss the experience of the presenters in engaging with it, and the opportunities and challenges presented by the new process.
OVERVIEW: The STA process, announced by NICE in November 2005, is designed to produce high quality guidance through a shorter, more efficient process. It will be used largely for new interventions (or new indications for existing interventions) where a single technology is being compared to current standard care. The presenters were involved in the first submission under the process in January 2006. The STA route presents manufacturers with new opportunities and challenges. The process will take around 6 months to complete, in comparison to around 14 months for the existing process (now termed the multiple technology appraisal [MTA] process). Thus a NICE recommendation is expected many months earlier. The expedited process is a single technology appraisal in which the intervention is compared with current practice only, and not with new competitor interventions. The STA differs from the MTA in the level of detail specified for the submission, the mandatory submission of models, and arrangements for feedback on draft guidance. Discussion topics will include issues that manufacturers may face in development of the STA submission, and in the consultation process, and the Institute's perspective on the STA process and review pf submitted evidence.


USE OF HEALTH ECONOMIC/ PHARMACOECONOMIC INFORMATION BY DECISION-MAKERS

IP3: PAYOR ORIENTED EVIDENCE GUIDELINES - Salon B

Moderator: Joseph Singer MD, Vice President Integrated Research, HealthCore, Inc, Wilmington, DE, USA

Panelists: Brian Sweet, MBA, BS Pharmacy, VP Clinical Services, WellPoint Pharmacy Management, West Hills, CA, USA; Dennis Raisch PhD, RPh, MS, Associate Center Director, Scientific Affairs, VA Cooperative Studies Program Clinical Research Pharmacy, Albuquerque, NM, USA

ISSUE: To explore and characterize the utilization of health outcomes and pharmacoeconomic research requirements of payors in their pursuit of evidence based guidelines and the identification of additional information requiremented to support regulatory approval, formulary placement and reimbursment criteria for pharmecuticals, biotechnology products and medical devises on a global basis.
OVERVIEW: This will be the third of three panel group discussions managed by this ISPOR Working Group (Florence, Shanghai and Philadelphia ISPOR meetings) where payors will present their research needs with regards to the development of reimbursment guidelines / criteria. Researchers in the audience will be given an opportunity to interact with panel members after their initial presentations. The recorded discussions will be included in White Papers produced by the Value Based Reimbursement Special Integest Group (Diane Simison, Chair). This panel discussion will run 60 minutes with the initial 45 minutes being presentations from the payors followed by the audience question and answer period. Representitives of CMS, the V.A. and managed care will participate in this panel.


IP4: LOST IN SPACE: REIMBURSEMENT FOR PHARMACOGENOMICS - Salon C

Moderator: Louis Rossiter PhD, Senior Research Fellow, The College of William & Mary, Williamsburg, VA, USA

Panelists: C. Randal Mills PhD, President and Chief Executive Officer, Osiris Therapeutics, Inc, Baltimore, MD, USA; Kathryn A. Phillips PhD, Professor of Health Economics & Health Services Research, University of California, San Francisco, San Francisco, CA, USA

ISSUE: Is reimbursement for pharmacogenomics caught between regulatory issues at the Food and Drug Administration, few good models of approved cost-effective products and services, and payers who are waiting for someone to tell them what to do?
OVERVIEW: Panelists will examine the status of pharmacogenomics, current and pipeline products, and the evidence for cost effectiveness. Up for debate is whether science can produce some excellent examples of pharmacogenomic products or services that substitute for current costly therapies, and whether the business model for pharmacogenomics will remain unclear. Without a blockbuster product, with unassailable outcomes, that is a clear substitute for existing “fix up” therapies, for a large number of patients; the field may be lost in space for some time. Possible reimbursement models for future products and services will be discussed. The question of whether Medicare Part D changes everything will be debated.
 

Tuesday, May 23rd, 9:15am-10:15am
Issue Panels-Session II

HEALTH POLICY

IP5: ORGANIZATION OF A TECHNOLOGY ASSESSMENT INSTITUTE FOR THE UNITED STATES - Salon A

Moderator: Frank J Papatheofanis MD, MPH, PhD, Associate Professor and Director, UCSD, San Diego, CA, USA

ISSUE: Would the US health care system benefit from a central organization for technology assessment? How would such an organization differ from existing government and commercial organizations? Who would formulate the policy agenda for such an agency? How should such an institute be funded (all government, mix of public and private monies, etc.)? What can we learn from NICE and EPTA, particularly in health economic evaluation, and should there be collaboration with outside agencies?
OVERVIEW: The US Congressional Office of Technology Assessment (OTA) closed in 1995 after serving lawmakers for over 23 years. Several initiatives (e.g. MCAC) sponsored by CMS since then have made significant contributions toward providing government decision makers with guidance concerning complex medical technology issues. Other governments increasingly rely on such government-sponsored agencies for guidance as well. However, other government agencies advise their sponsors on the basis of clinical and economic vale. Cost-effectiveness analysis remains an elusive and controversial tool for providing guidance in this setting. As health economic results become increasingly valuable in deciding technology adoption and diffusion, there is an opportunity to enhance US-based technology assessment by consolidation and the formation of a dedicated organization similar to those used elsewhere. Such an institute would potentially offer guidance to US government and commercial payors as well as other stakeholders.


IP6: CONSEQUENCES OF DRUG COST CONTAINMENT: NEW EVIDENCE ON CO-PAYMENTS, CO-INSURANCE, AND THERAPEUTIC SUBSTITUTION - Salon B

Moderator: Sebastian Schneeweiss MD, ScD, Associate Professor or Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA

Panelists: Colin Dormuth ScD, MA, MS, Assistant Professor, University of British Columbia, Vancouver, BC, Canada; Amanda Patrick MS, Decision Scientist, Brigham and Women's Hospital, Boston, MA, USA

ISSUE: Medicare Part D drug coverage for seniors will use a cost-containment strategy that includes premiums, deductibles, co-payments, co-insurance and therapeutic substitution. A large number of policy evaluations have been published that so far culminate in a confusing mix of statements relevant only to the specific policy implementations studied. Yet policy makers need evidence-based advice that can be applied to their own specific setting. How do we best generate such evidence? Can we see trends that can be generalized? What are some of the most recent evaluation results?
OVERVIEW: Based on existing studies the workshop will point out problems in published evaluations and challenges in generalizing results of drug policy evaluations. This will lead to a set of hypotheses regarding generalizable principles relevant to cost-containment policies. These hypotheses will be tested in four brief presentations of most recent drug policy evaluations in elderly patients, including the effects of deductibles, co-payments and co-insurance on utilization and outcomes in patients using COPD medications, statins, anti-depressants, and therapeutic substitution of PPIs.

PHARMACOECONOMIC / HEALTH ECONOMIC STUDY METHODOLOGY

IP7: ASSESSING HETEROGENEITY IN COST-EFFECTIVENESS ANALYSIS - Salon C

Moderator: Andrew Briggs DPhil, Professor, University of Glasgow, Glasgow, United Kingdom

Panelists: Mark J Sculpher PhD, Professor, University of York, York, United Kingdom; John R Cook PhD, Senior Director Health Economic Statistics, Merck and Co, Blue Bell, PA, USA

ISSUE: What is the role and limits of sub-group analysis in cost-effectiveness analysis?
OVERVIEW: Health care systems and payers are inevitably concerned with maximising benefits to patients from their funding base. There is an abundance of evidence that there is heterogeneity in the cost-effectiveness of health care technologies. This heterogeneity relates to a number of factors. Most notably cost-effectiveness is known to vary according to the characteristics of patients at the time of treatment selection. This can be in terms of clinical factors, such as baseline disease severity, or demographic variables like age and gender. Cost-effectiveness is also known to vary by other factors such as the location of treatment (e.g. country or hospital). Decision makers are interested in identifying groups of patients who are likely to derive the greatest benefit per dollar spent. But there is a tradition of caution for some types of sub-group analysis in randomised trials when used as a source of efficacy data. This Issues Panel will consider the opportunities and limits to sub-group analysis for cost-effectiveness analysis. The panellists will debate the merits of Bayesian decision theoretical approaches versus conventional frequentist trial analysis.


RISK ASSESSMENT

IP8: HEALTH OUTCOMES APPROACHES TO RISK-BENEFIT ANALYSIS: HOW READY ARE THEY? - Salon D

Moderator: Richard J Willke PhD, Senior Director/Group Leader, Pfizer Inc, Bridgewater, NJ, USA

Panelists: Lou Garrison PhD, Professor, Pharmaceutical Outcomes Research and Policy Program, University of Washington, Seattle, WA, USA; F. Reed Johnson PhD, Senior Fellow and Principal Economist, Research Triangle Institute, Research Triangle Park, NC, USA; Larry Lynd PhD, Assistant Professor, Faculty of Pharmaceutical Sciences, University of British Columbia, Vancouver, BC, Canada

ISSUE: What are the principal health-outcomes approaches to quantitative risk-benefit analysis and are they robust enough to be considered for use in regulatory approval as well as the drug-development and clinical steps leading up to NDA submission?
OVERVIEW: Given the growing emphasis on risk-benefit decisions regarding new and existing medical interventions, and the parallels between risk-benefit and cost-benefit analyses, it is a natural progression to migrate some accepted health outcomes techniques to risk-benefit analysis. Some methods already being explored include incremental net health benefits (measured in monetary vs. quality-adjusted life years), contingent valuation, and discrete-choice experiments. The structured approaches to risk-benefit analyses that these methods offer will be considered in this session. For example, the potential value of more explicit and structured processes and methods will be contrasted with the current regulatory review process. However, those working in health outcomes are well aware of the limitations of these types of analysis. Additionally, differences in the types and numbers of risks between treatment options increase the complexity of providing one metric to capture risk-benefit. The discussion will consider how such limitations may affect the feasibility of applying these methods in marketing approval and go/no-go development decisions.