Issue Panels Session I - Monday, May 16, 2005 

Health Policy/Health Care Reimbursement/Coverage Issues

SHOULD MANUFACTURERS CONSULT WITH CMS STAFF ON THE PHASE III STUDY DESIGN, AS CMS IS NOW REQUESTING?

Moderator: Diane Simison PhD, Executive Director, MEDTAP Center for Pricing and Reimbursement, Arlington, VA USA
Panelists: Steve Phurrough MD, MPA, Director, Coverage Analysis Group, Centers for Medicare and Medicaid Services, Baltimore, MD USA; Beth A. Hahn PhD, Managing Director, MEDTAP Center for Pricing and Reimbursement, Arlington, VA USA

ISSUES: Should manufacturers consult with CMS staff on the Phase III study design, as CMS is now requesting? How will the "Coverage with Evidence Development" option work in practice? Will the new coverage provisions CMS is considering for Part B also be applied to Part D?

OVERVIEW: For those new technologies for which the manufacturer wishes Medicare coverage, CMS has requested that manufacturers meet with CMS officials to discuss the Phase III trial design. This will ensure that CMS requirements can be considered in various aspects of design, such as patient inclusion and exclusion criteria, and other design elements. Technology sponsors may resist this request for a variety of reasons including increased timelines, possible conflicting opinions with FDA, and increased resource requirements. This issue panel is to debate the reasons behind both perspectives.

AMCP FORMAT FOR FORMULARY SUBMISSIONS: PERSPECTIVES ON DOSSIER PREPARATION AND REVIEW

Moderator: Shahnaz Khan, MPH, Health Outcomes Communications Specialist, RTI Health Solutions, Research Triangle Park, NC, USA
Panelists: Iris Tam PharmD, Medical Communication Scientist, Genentech, Inc, South San Francisco, CA, USA;
C. Mihalic, 3M.Plan, Indianapolis, IN, USA 

ISSUES: What is the process for receiving and fulfilling requests for dossiers? How do most manufacturers evaluate the usefulness of the dossier with payer groups or customers? What are unique issues with dossiers to support high cost injectable products? What level of deviation from the Format is acceptable? How do you make a scientific, evidence based document easy to understand? Should non-US data be included in an AMCP dossier? What is the degree of adoption of AMCP Format by health plans? (e.g., Does the health plan feel that the pharmacoeconomics data are presented in a balanced and easy-to-understand manner? Do health plans generally find clinical information in an AMCP based dossier accurate and adequate? What would a health plan recommend to facilitate review of an AMCP-based dossier?

OVERVIEW: The AMCP Format for Formulary Submissions provides helpful guidance for, as well as presents unique challenges to, pharmaceutical manufacturers, consultant agencies, and managed care review committees. Key contentious topics will include issues that manufacturers face in development and provision of dossiers; issues that consultant agencies face when working with a manufacturer to prepare a dossier; and the health plan's perspective on reviewing data presented according to the Format.

Moderator: Penny Mohr MA, Health Economist, Centers for Medicare and Medicaid Services, Baltimore, MD, USA
Panelists: Christopher McCabe PhD, Senior Lecturer, The University of Sheffield, Sheffield, UK ; Bernard Bloom PhD, Research Professor, University of Pennsylvania, Philadelphia, PA, USA

ISSUES: How does Medicare currently use cost-effectiveness analyses (CEA)? What are the barriers to expanded use of CEA for Medicare policy making? What are some lessons that have been learned for the Medicare program in the UK and other countries?

OVERVIEW: Medicare has faced barriers to using CEA for coverage decisions for many years, but CEA have been and are being used within the program. Section 641 of the Medicare Prescription Drug Improvement and Modernization Act of 2003 explicitly calls for CEA to be done and CEA have been built into collaborative research between CMS and other Federal agencies. This session offers the perspectives of a health economist working within the Medicare program, a US academic who has surveyed other payors about their use of CEA, and a UK academic who has worked closely with NICE to understand how CEA is being used within Medicare and shed light on how others are using it and pitfalls to avoid.

 
Outcomes Research (Economic, Clinical, Humanistic) Issues

Moderator: Michael Drummond PhD, Director, University of York, York, UK

Panelists: P. Tugwell, University of Ottawa, Ottawa, Ontario, Canada; Mark Sculpher MSc, PhD, Professor, University of York, York, North Yorkshire, UK; A. Maetzel, Amgen (Europe) GmbH, Lucerne, Lucerne, Switzerland
 

ISSUES Are there benefits from the greater methodological specification contained in disease-specific reference cases? Are disease-specific reference cases unnecessarily restrictive, or burdensome, to sponsoring or conducting research? Do the reference cases developed by OMERACT provide a suitable template for other diseases?

Moderator: Diane Simison PhD, Co-Executive Director, MEDTAP, Arlington, VA, USA
Panelists: Ruben King-Shaw MA, President, The Solutions Institute, Bethesda, MD USA; Robert Navarro PharmD, Executive Advisory Group, Campbell Alliance, Raleigh, NC  USA

ISSUES: Will CMS begin to use economic data to evaluate new technologies for Medicare coverage? Will health plans offering both the Part D benefit and different formularies for commercial products actually use different criteria for evaluating formularies, and will the criteria include cost effectiveness or other cost evaluation measures? If a health plan uses internal plan data on the relative consumption of health care resources in the formulary decision process, are they in fact using economic data?

OVERVIEW: Economic data, including cost, is essential to evaluate the value of new technologies. In the past, CMS and other payers have limited their coverage evaluations whether there is an insurance benefit for the technology and medical necessity. Economic considerations have usually not been included in the coverage debate. Payments for new procedures may not reflect the cost of the new technology and does not allow an evaluation of its value. Increasingly, new technologies and treatments will need to demonstrate economic value. The key discussion issues are as follows: what actually occurs during CMS coverage for determinations? Are the current Medicare review procedures sufficient to determine economic value in the Medicare coverage decision? Can researchers provide evidence of value for CMS to consider?

Issues on Use of Health Outcomes Research Information by Decision-Makers
 

THE PEER REVIEW PROCESS: HOW EDITORS DRAW THE LINE BETWEEN SCIENCE AND ADVERTISING

Moderator: C. Daniel Mullins PhD, Director, Pharmaceutical Health Services Research, University of Maryland, School of Pharmacy, Baltimore, MD, USA
Panelists: Chris Carswell BSc, MSc, Editor, Pharmacoeconomics, Mairangi Bay, Auckland, New Zealand; Alan Lyles MPH, RPh, Editor, Clinical Therapeutics & Professor, University of Baltimore, Baltimore, MD, USA; Josephine Mauskopf PhD, Editor, Value in Health & VP, Health Economics, RTI Health Solutions, RTI International, Research Triangle Park, NC, USA

ISSUES: What are the criteria for performing sensitivity analyses for modeling papers? How do we utilize the ISPOR task force guidelines for database studies? How, when, and at what rate discounting should be performed? What is the use of accepted criteria for validating quality of life measures? How do we incorporate the best evidence from clinical trials and other sources into modeling studies? How do we present balanced conclusions rather than 'company value messages'? How do we present balanced conclusions rather than 'company value messages'? What is the process for determining authorship and acknowledgements? What are the issues with disclosure of real and potential conflicts of interest?

OVERVIEW: The editorial and peer-review process for pharmacoeconomics and outcomes research articles is designed to assure that published papers use sound methodology and appropriate data, and present results and conclusions that are substantiated, unbiased and useful for decision-making. There is a long history of controversy concerning whether these decisions are effective in separating science from marketing. The editors of three journals that publish significant numbers of pharmacoeconomics and outcomes research papers will debate ways to evaluate such papers to achieve these goals.

Moderator: Michael Drummond PhD, Director, University of York, York, UK
Panelists: Peter Neumann ScD, Associate Professor of Policy and Decision Sciences, Harvard University, Boston, MA, USA; John Watkins MPH, RPh, Pharmacy Manager, Premera Blue Cross, Mountlake Terrace, WA, USA; Adrian Towse MA, Director, Office ofHealth Economics, London, UK

ISSUES: What are the most useful aspects of the databases for researchers or decision-makers? What are the main shortcomings, or limitations, of the databases? What can be learned from similar enterprises, such as the reviews produced by the Cochrane Collaboration? Is there potential for more international collaboration?

OVERVIEW: A number of economic evaluation databases have been developed Worldwide. These include, in the USA, the Harvard Cost-Effectiveness Registry and, in Europe, the NHS Economic Evaluation Database (NHS EED), the OHE Health Economic Evaluations Database (HEED) and the Collège des Économists de la Santé CODECS database. In addition, a European Network of Health Economic Evaluation Databases (EURONHEED) is under development. The databases provide a handy literature source and, to different degrees, structured critical commentaries on published economic evaluations. Potentially they could be of use to researchers and industry sponsors, who may be planning studies, and to decision-makers, who need to interpret study results.

Moderator: Nancy Santanello, MD, MS, Executive Director, Epidemiology Department, Merck Research Laboratories, Blue Bell, PA, USA

Panelists: Bryce Reeve BS, MA, PhD, Psychometrician, National Institutes of Health, Bethesda, MD, USA; David Cella PhD, Professor and Director, Center on Outcomes Research and Education, Evanston Northwestern Healthcare, Evanston, IL, USA; H. Guess, University of North Carolina, Chapel Hill, Chapel Hill, NC, USA
 

ISSUES: Are item banking and computerized adaptive testing (CAT) the future of PRO assessment? Will item banking and CAT replace current methods and popular measures, or enhance their value? What can past experience teach us about moving forward with item banks?