ISPOR 16th Annual International Meeting: ISSUE PANEL PRESENTATIONS
 

ISSUE PANEL PRESENTATIONS

ISSUE PANELS - SESSION I Monday, May 23, 2011: 11:00 AM-12:00 PM

Health Policy Development Using Outcomes Research Issues

IP1: IS FDA/CMS PARALLEL REVIEW WORTHWHILE? IS IT FEASIBLE?
Moderator: Peter J Neumann, ScD, Professor and Director, The Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA
Panelists: Scott Gottlieb, MD, Resident Fellow, American Enterprise Institute for Public Policy Research, Washington, DC, USA; Sean R. Tunis, MD, MSc, Founder and Director, Center for Medical Technology Policy, Baltimore, MD, USA; James Chambers, MPharm, MSc, Project Director, The Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA
ISSUE: To discuss the prospects for parallel Food and Drug Administration (FDA)/Centers for Medicare and Medicaid Services (CMS) review of medical products as proposed in the recent memorandum of understanding between the two agencies, and how it may change the development, coverage, and reimbursement for drugs and devices. Panelists will debate this issue from the perspectives of government and the pharmaceutical industry (Dr. Tunis and Dr. Gottlieb) and will address data on consistencies and discrepancies between FDA and CMS review (Mr. Chambers).
OVERVIEW: The FDA and CMS make independent judgments with respect to approval and coverage of products.  The FDA approves technologies based upon safety and efficacy.  In contrast, the CMS covers and pays for technologies only if they are ‘reasonable and necessary’ for the diagnosis or treatment of an illness or injury.  Differences in “safety and efficacy” versus “reasonable and necessary” has manifested in different types of evidence considered by the agencies.  The FDA typically relies upon randomized placebo controlled trials; however, the CMS considers the totality of evidence, ranking studies with respect to design and quality.  In some instances, there have been delays or inconsistent decisions between FDA approval and CMS coverage and reimbursement.  In September, 2010, the FDA and CMS issued a proposal to initiate parallel review of medical products to help streamline and coordinate reviews.  Such a process could help expedite the path from FDA approval to coverage and reimbursement at CMS.  Given the different agency remits, it is unclear if parallel review is feasible, or will cause further delays to the processes.  This issue panel will provide a forum to debate the prospects for parallel review, including the likely political hurdles and the implications of agency coordination and data sharing.  Research will be presented describing the consistency, or lack of, between FDA indication and CMS coverage and reimbursement.   

IP2: PAYING FOR VALUE - WHICH TO GO FOR: THE NEW UK APPROACH OR THE NEW GERMAN LAW OR NEITHER?
Moderator: J. Jaime Caro, MDCM, FRCPC, FAC, Senior Vice President of Health Economics, United BioSource Corporation, Lexington, MA, USA
Panelists: Peter L. Kolominsky-Rabas, MD, PhD, MBA, Director, Centre for Health Technology Assessment (HTA) and Public Health (IZPH), University of Erlangen-Nurnberg, Erlangen, Germany; Alistair J. McGuire, PhD, Professor and Head of Social Policy, LSE Health and Social Care, London, UK
ISSUE: Everyone wants to payan appropriate amount for health care interventions. But what is “appropriate”? One answer is that it should depend on the value of the effects obtained. Recent changes in the English and German regulations governing HTAs aim to implement this idea and capture value for the public funds expended. Are they likely to achieve this or are they doomed to fail? The panellists with a broad range of experience in HTA will discuss the recent changes with a view to addressing this question. The panel will also debate what, if any, implications these initiatives may hold for the implementation of health care reform in the United States.
OVERVIEW: Substantial changes are going on in the European HTA landscape, influencing market access strategies and reimbursement decisions for drugs. The UK government is considering plans to radically overhaul the way drugs will be paid for, introducing a so-called “value-based” pricing system within the next three years. After that drug prices will be set according to the value medicines provide, with access likely to be no longer limited by NICE-set cost-effectiveness thresholds, although NICE will still have a role in providing information. In Germany, the federal government added a fourth hurdle at the beginning of 2011. This is a significant change for the introduction of drugs in Germany. Among other measures, it ends the period of free pricing in Germany and initiates the use of benefit/cost assessments. Rapid assessments of the clinical evidence (benefit assessment) and price negotiations for all drugs entering the German market are now implemented. Both sets of reforms will dramatically affect pharmaceutical pricing in countries characterised as currently having "free-pricing" regimes. The implications of these changes will ripple across various European countries and will even impact the United States.

IP3: ASSESSMENTS OF RELATIVE EFFICACY: DO THE BENEFITS JUSTIFY THE COSTS?
Moderator: Benedikte Lensberg, MSc, Project Leader, i3 Innovus, Uxbridge, Middlesex, UK
Panelists: Michael Drummond, MCom, DPhil, Professor of Health Economics, Centre for Health Economics, University of York, Heslington, York, UK; Kalipso Chalkidou, MD, Director, NICE International, National Institute for Health and Clinical Excellence, London, UK; Adrian Towse, MA, Director, Office of Health Economics, London, UK
ISSUE: The European Medicines Agency (EMA) has called for more assessments of relative efficacy as part of the drug licensing process. The aim is to produce clinical information of more relevance to payers. This has several implications, however, for the clinical development plans of pharmaceutical companies. Do the likely benefits of this policy justify the costs? Panelists will debate on this topic from the perspective of the researcher, HTA/payer and the pharmaceutical industry.
OVERVIEW: In its Roadmap to 2015, the EMA identified the need to increase the relevance to payers of its European Public Assessment Reports (EPARs). A key suggestion is to request pharmaceutical companies more often to submit assessments of relative efficacy of their products, as compared to active comparators. Companies are concerned, however, that this will add to the length and cost of the clinical development process, requiring clinical trials to be larger, or longer in duration. In addition, it is possible that such studies may still not meet the needs of payers, who have an interest in relative effectiveness, involving the measurement of different clinical endpoints and assessments of the performance of the product in a regular clinical, as opposed to trial-based, setting. Indeed, if payers’ needs were to be met more completely through registration trials, this would require even more fundamental and costly changes in trial design. Furthermore, there are fears in some quarters that a movement towards centralized production of more comprehensive assessments of clinical value is the first step along the road towards a centralized European reimbursement process. Therefore, a policy of requiring more comparative studies for licensing purposes requires careful evaluation.

Economic Outcomes Research Issues

IP4: WHAT CAN WE LEARN FROM SUCCESSES AND FAILURES IN PERSONALIZED MEDICINE? IMPLICATIONS FOR EVOLVING HEALTH ECONOMICS AND OUTCOMES RESEARCH PRACTICES
Moderator: Eric C Faulkner, MPH, Senior Director, Market Access and Outcomes Strategy, RTI Health Solutions, Research Triangle Park, NC, USA
Panelists: Bruce Quinn, PhD, MD, Senior Health Policy Specialist, Foley Hoag, Boston, MA, USA; Kostas Trakas, PhD, MSc, Senior Director, Worldwide Health Economics & Pricing, CNS, Johnson & Johnson Pharmaceutical Services LLC, Toronto, ON, Canada; Lou Garrison, PhD, Professor, Pharmaceutical Outcomes Research & Policy Program, Department of Pharmacy, University of Washington, Seattle, WA, USA
ISSUE: As payers face increasing pressure to limit pharmaceutical access to appropriate subpopulations, personalized medicine represents one way to meet this goal. Decision maker requirements, however, are still evolving and pursuit of drug-diagnostic combinations does not guarantee success. How have personalized medicine products been handled by HTA agencies and payers? What factors have influenced reimbursement? How do assessments differ from conventional treatments? What is different from other technologies targeted to subpopulations such as orphan drugs or end of life therapies? What lessons can be learned from successes and failures in personalized medicine? What are the implications for HEOR in an era of global health care reform, comparative effectiveness and value-based purchasing? The moderator will represent an HTA perspective and will present an overview of HTA recommendations from North America, Europe and Australia on personalized medicines, compare them to conventional pharmaceuticals and set the stage for debate; Dr. Quinn will represent a payer's perspective and discuss decision factors that influence payer acceptance and should be considered as new HEOR approaches and policies evolve; Dr. Trakas will provide a drug manufacturer's perspective on challenges in personalized medicine and how HEOR planning is being retooled for value demonstration; and Dr. Garrison will represent a health economist's perspective and discuss whether new HEOR methods will be necessary to support decision making or existing methods are satisfactory.
OVERVIEW: Despite the implicit assumption that personalized medicine approaches increase the likelihood of cost effectiveness, these products must navigate hurdles similar to conventional pharmaceuticals. Making a value case for personalized medicines, while perhaps complex, still requires basic demonstration of efficacy, safety and cost-effectiveness.  The panel will consider recent global HTA recommendations on test/treatment combinations, identify clinical and economic decision factors, and debate whether these factors are specific to personalized medicine and new approaches are warranted to address HEOR challenges.

Clinical Outcomes Research Issues

IP5: DEBATING THE VALUE AND FUTURE DIRECTIONS OF PATIENT REGISTRIES IN COMPARATIVE EFFECTIVENESS RESEARCH
Moderator: Nancy Dreyer, PhD, MPH, Chief of Scientific Affairs, Senior Vice President, Outcome, Cambridge, MA, USA
Panelists: Charles E. Barr, MD, MPH, Medical Director, Head of Registries, Medical Affairs, Genentech, Inc., South San Francisco, CA, USA; John Spertus, MD, MPH, Professor, Clinical Director of Outcomes Research, University of Missouri-Kansas City, Mid America Heart Institute of Saint Luke's Hospital, Kansas City, MO, USA; Robert McDonough MD, JD, MPP, Head, Clinical Policy Research and Development, Aetna, Hartford, CT, USA
ISSUE: What are the value and “appropriate” uses of patient registries by health care stakeholders, including the clinical, manufacturer and payer perspectives? What are the methodological considerations of patient registries?
OVERVIEW: As more therapeutic options become available and the cost of health care continues to increase, comparative effectiveness research (CER) may be critical in supplementing and confirming the evidence base for health care decision makers. Patient registries offer useful contributions to CER by providing data on populations often excluded from randomized controlled trials and tracking “real world” practice patterns and associations with patient outcomes, to help health care providers make informed treatment choices for their patients. There are, however, limitations of patient registries and observational research, including potential confounding and selection biases. Following a short introduction by the moderator (5 min), each panel member will first present the uses and value of patient registry data from a clinical, manufacturer, and US payer perspective (20 min). Panel members will then debate current and future uses and limitations of patient registries, including a discussion of what a registry should and should not be used for and why (15 min).  During this session, there will be probes on each panelist’s viewpoints on the methodological considerations and interpretation of registry data, as well as the perceived credibility of the evidence. The final session (20 min) will be allotted to audience questions and answers for the panel members.

ISSUE PANELS - SESSION II Tuesday, May 24, 2011: 2:45 AM-3:45 AM

Health Policy Development Using Outcomes Research Issues

IP6: WHEN IS THE EVIDENCE ADEQUATE? DIFFERENT PERSPECTIVES FROM KEY HEALTH CARE DECISION-MAKERS
Moderator: Robert W. Dubois, MD, PhD, Chief Science Officer, National Pharmaceutical Council, Washington, DC, USA
Panelists: Bryan R. Luce, PhD, MBA, Senior Vice President, Science Policy, United BioSource Corporation, Bethesda, MD, USA; Steven Pearson, MD, MSc, President, Institute for Clinical and Economic Review, Boston, MA, USA; Robert S. Epstein, MD, MS, Chief Medical Officer, Senior Vice President, Medical Affairs, Medco Health, Franklin Lakes, NJ, USA
ISSUE: A series of comparative effectiveness research (CER) reports become available with a diversity of findings related to the efficacy/effectiveness/safety/resource utilization for a new and costly biopharmaceutical agent.  When does a payer have adequate evidence to approve coverage, deny coverage, or grant conditional coverage (e.g., risk sharing, coverage with evidence development)? 
OVERVIEW: When does a payer have adequate evidence to approve coverage, deny coverage, or grant conditional coverage (e.g., risk sharing, coverage with evidence development)?  It is likely a function not only of the convincing nature of that evidence (based upon number of studies, consistency of results, directness, precision, and risk of bias), but also upon contextual issues (e.g., ethical, moral, legal) inherent to the decision makers and their organizations.  These factors might include: 1) How many patients would be affected by the acceptance (or negation) of the current evidence? 2) How much “better” is the new therapy compared to existing ones? 3) What is the comparative “value” of the new therapy? 4) Is it a politically important population of patients (or an under-served population)? 5) What is the downside if we are "wrong" in our understanding of the evidence?  (both Type I and Type II errors)? 6) What would it cost to get additional information and what would be the value of that information? 7) How long would it take to get that additional information?  This Issue Panel will explore how different stakeholders (i.e., payer, pharmacy benefit managers (PBM), researcher, industry) consider the above issues and will debate:  relative importance of the factors, transparency of the decision making process, whether new research methods are needed, and a “middle ground” of coverage with evidence development.  The audience will react to a hypothetical example and be challenged to discuss the pros and cons of proceeding with the available evidence versus waiting for more information.

IP7: HOW DO WE STOP PAYING FOR LOW-VALUE CARE?
Moderator: Sarah Garner, PhD, Associate Director R&D NICE, Research and Development, NICE, London, UK
Panelists: Peter J. Neumann, ScD, Professor and Director, The Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA; Mark Fendrick, MD, Professor/ Co-Director, Division of General Medicine, Department of Internal Medicine and Department of Health Management and Policy, University of Michigan Center for Value-Based Insurance Design, Ann Arbor, MI, USA; Adam Elshaug, MPH, PhD, Harkness Fellow/Senior Research Fellow, School of Population Health and Clinical Practice, Adelaide University/AHRQ, Rockville, MD, USA
ISSUE: All health systems are seeking to control costs without compromising the quality of care that they provide. To date, most efforts have concentrated on managing the entry of new interventions. Interest is however increasing in identifying and disinvesting from existing practice that is either clinically or cost ineffective. Such efforts are however proving controversial and difficult to implement.
OVERVIEW:  Despite the apparent agreement that there is substantial overuse, under use, and misuse of existing medical care any attempts to address it have proved controversial. Particularly if there is a proposal for complete disinvestment.  Therefore most activities have inevitably focused technologies that are less controversial for example cosmetic surgery, self-limiting diseases or conditions that are not life-threatening such as dermatology or surgery for varicose veins.  This introduces a paradox; newer technologies are being rigorously evaluated yet there has been very little attention on existing – legacy - technologies that were introduced prior to relatively new stringent assessment methodologies. If different evaluation mechanisms are used, many technologies and services that are being disinvested in may in fact be more valuable.  Both the Australia and the United Kingdom have introduced national efforts to identify and ‘disinvest’ from low-value healthcare so that the resources may be re-invested in more valuable care. There is interest in the United States on using ‘value-based’ insurance design to provide dis-incentives for consumption of specified low-value practices, for example by increasing co-payment. However there has been little activity to date in part due to political concerns.  This issue panel will bring together experts from Australia, the United States and United Kingdom to discuss the experiences and debate the challenges with current initiatives and highlight the lessons learnt.  Dr Elshaug and Dr Garner have gained additional insight into the US context as both have been based in the US on Harkness Fellowships focussing on low-value technologies.

IP8: IDENTIFICATION, WEIGHTING AND PRIORITIZATION OF MULTIPLE ENDPOINTS FOR COMPARATIVE EFFECTIVENESS RESEARCH – WHAT HAVE WE LEARNED FROM GERMANY?
Moderator: Michael Drummond, MCom, DPhil, Professor of Health Economics, Centre for Health Economics, University of York, Heslington, York, UK
Panelists: Axel Mühlbacher, PhD, Harkness Fellow in Health Care Policy and Practice, Duke Clinical Research Institute/ Fuqua School of Business, Duke University, Durham, NC, USA; John F.P. Bridges, PhD, Assistant Professor, Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA; Maarten J. IJzerman, PhD, Professor & Chair, Department of Health Technology & Services Research, University Twente, Enschede, The Netherlands
ISSUE: While many countries use cost-effectiveness analysis and the cost-per-QALY as their primary method for health technology assessment (HTA), both Germany and the United States (US) have opted for comparative effectiveness research (CER) that aims to promote patient-centered outcomes.  While the United States has not identified how they will make their CER patient-centered, IQWiG, the German HTA agency, has explored both conjoint analysis and analytic hierarchy process (AHP) as methods to prioritize and weight patient-centered outcomes.  This panel will compare the approaches of the United States and Germany and will debate the relative benefits of conjoint analysis and AHP as means of involving patients’ preferences and facilitating the decision making process.
OVERVIEW: This session will involve three brief presentations on comparing the HTA methods proposed by IQWiG and the United States and the potential role of conjoint and AHP methods in prioritizing and weighting patient-centered outcomes.  The session will also accommodate a rigorous debate on patient-centered outcomes and questions from conference participants.  Dr. Bridges will discuss the importance of involving patients in the choice of outcomes in HTA and CEA and highlighting the similarities and differences between the approaches taken by US and other countries in Europe.  Dr. Mühlbacher will discuss how conjoint analysis can be used in CER and HTA, drawing from the Germany experience. Dr. IJzerman will discuss the relative merits of using AHP in CER and HTA, drawing from studies from the Netherlands and Germany.  The panel with then discuss the relative merits of these methods and the likely impact that they will play the United States and Europe.

IP9: WILL THE PROPOSED VALUE BASED PRICING WORK IN THE UK?
Moderator: Stephen Beard, MSc, Head of Health Economics (Europe), RTI Health Solutions, Sheffield, UK
Panelists: Ron Akehurst, Professor of Health Economics, Dean of School of Health & Related Research, ScHARR, The University of Sheffield, Sheffield, UK; James Raftery, PhD, Professor of Health Technology Assessment, Southampton Health Technology Assessments Centre (SHTAC), University of Southampton, Southampton, UK; Trevor Leighton, Vice President, Pricing & Reimbursement, Shire AG, Eysins, Switzerland
ISSUE: Recent developments from the United Kingdom’s (UK’s) National Institute for Health and Clinical Excellence (NICE) will be discussed, including proposed changes to the Institute’s role in the planned introduction of 'value based pricing' in 2014.  An overview of the proposed process will be presented, key aspects of new evidence requirements will be discussed and recommendations for research planning and implementation to fulfill the changing requirements will be presented.  The Issue Panel will conclude with an open session for audience debate.
OVERVIEW: In November 2010, the UK government announced its intention to introduce 'value based pricing', which will come into effect when the Pharmaceutical Price Regulation Scheme expires in 2014.  Details of the proposals were issued for consultation in December 2010.  In these proposals, NICE will continue to have a central role in undertaking pharmacoeconomic assessments and in providing advice to the National Health Service on the relative clinical and cost effectiveness of treatments.  Higher cost-effectiveness thresholds will be applicable for medicines that are indicated for diseases with a higher burden of illness (defined as a combination of the severity of the condition and the level of unmet need) and that can demonstrate greater therapeutic innovation and improvement.  The Panel will summarise and interpret these new initiatives, and participate in discussion and debate with the audience. 

Patient-Reported Outcomes Research Issues

IP10: HOW CAN PATIENT-REPORTED OUTCOMES BECOME A PART OF COMPARATIVE EFFECTIVENESS RESEARCH?
Moderator: C. Daniel Mullins, PhD, Professor, Pharmaceutical Health Services Research Department, University of Maryland School of Pharmacy, Baltimore, MD, USA
Panelists: Amy Abernethy, MD, Associate Professor, Duke University, Durham, NC, USA; Albert Wu, MD, MPH, Professor, Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA
ISSUE: What is the optimal approach to integrating patient-reported outcomes (PROs) in comparative effectiveness research (CER)?
OVERVIEW: The patient perspective is an essential component of comparative effectiveness research (CER) and is noted in the legislation which created PCORI -- yet it is not included in most CER research.  There is no standard approach to selecting and administering specific PRO measures in CER.  Methods of data collection differ from preapproval trials conducted in the regulatory context, because real-world populations may be less willing or able to self-report information.  Standards for mixing modes and methods may also differ from the preapproval setting, and use of backup data collection approaches may be more important.  Moreover, measures of interest differ in this area, including assessments of quality of life, satisfaction, value of delivered care (i.e., “was it worth it”), as well as symptoms, compliance, and reasons for non-compliance.  Stakeholders differ in the CER context, meriting different analytic approaches.  This session will review methods used to collect PROs in CER and for regulatory purposes, and debate how to make PRO a part of CER.  Specific examples will be provided.    

ISSUE PANELS - SESSION III Wednesday, May 25, 2011: 8:45 AM-9:45 AM

Health Policy Development Using Outcomes Research Issues

IP11: COVERAGE WITH EVIDENCE DEVELOPMENT IN THE PRIVATE INSURANCE SECTOR: IS IT A PIPEDREAM OR INEVITABLE FOR SOUND POLICY?
Moderator: Penny Mohr, MA, Vice President, Program Development, Center for Medical Technology Policy, Baltimore, MD, USA
Panelists: Seema Sonnad, PhD, Associate Professor, Department of Surgery, University of Pennsylvania, Philadelphia, PA, USA; Russell Teagarden, PharmD, Vice President, Scientific Affairs, Medco Health Solutions, Inc, Franklin Lakes, NJ, USA; Jens Grueger PhD, Vice President, Head Global Market Access Pfizer Ltd, London, UK

ISSUE: Coverage with evidence development (CED) conditions payment for potentially beneficial, yet unproven, medical technologies on enrollment in studies that will support more informed coverage policies.  The term was coined for initiatives begun in the Medicare program, and is part of formal policy in many countries under different names.  Although early successful experiments with CED in the United States were done by private insurance companies, there are few recent examples.  Skeptics suggest barriers are too great within the fragmented private insurance market to support CED.  Proponents argue CED is a proactive approach to managing medical technology that is an essential complement to the yes/no dichotomy of traditional coverage.

OVERVIEW: This issue panel brings together three different perspectives – one from the private insurance world, one from the pharmaceutical industry, and one from academia – to debate the viability of private sector CED.  Dr. Grueger from Pfizer will discuss how CED can accelerate access for patients to breakthrough medicines. Dr. Teagarden, from a pharmaceutical benefit management firm, MedCo Health Solutions, Inc., argues that in the current economic climate the time is ripe to aggressively pursue this policy option – with many of their large employer clients pushing them to adopt innovative approaches to managing technology costs.  Their views are tempered by Dr. Sonnad, a researcher, who led a private sector CED pilot project conducted by the private sector Center for Medical Technology Policy (CMTP). This project came near success to implementing CED in two US health plans as a complement to a Medicare CED policy.  The CMTP approach highlights the importance of having a neutral, arbiter to provide a firewall for the selection of topics and to ensure credibility of the research being conducted.  She provides a more cautionary note.

IP12: EXPERIMENTAL VS. OBSERVATIONAL STUDIES: WHICH SHOULD HAVE A HIGHER RANK IN HEALTH CARE DECISIONS?
Moderator: Edward Kim, MD, MBA, Executive Director, Health Economics and Outcomes Research, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA
Panelists: C. Daniel Mullins, PhD, Professor, Pharmaceutical Health Services Research Department, University of Maryland School of Pharmacy, Baltimore, MD, USA; Rachael Fleurence, PhD, Director, Oxford Outcomes, Bethesda, MD, USA; Winston Wong, PharmD, Associate Vice President, Pharmacy Management, CareFirst BlueCross BlueShield, Baltimore, MD, USA
ISSUE: Comparative Effectiveness Research (CER) is increasingly used to inform reimbursement decisions worldwide.  While randomized controlled trials (RCTs) have traditionally been considered the gold standard with respect to scientific validity, observational research methods have evolved to demonstrate both internal and external validity.
OVERVIEW: Health care decision-makers must make determinations of comparative effectiveness by evaluating and integrating the best available evidence.  This process is challenged by assumptions regarding evidence hierarchies and best practices for integrating the totality of experimental and observational study results. One view accepts the traditional hierarchy that considers RCTs to be  the gold standard and only requires modifications based on study design quality to adjust the ranking of individual studies.   Under this hierarchy, a poorly designed RCT may receive a lower ranking than a well-designed observational study. An alternate view holds that a methodology-based hierarchy (RCT vs. observational) is inherently flawed and should be replaced by a continuum of studies that address whether the treatment works, for whom, and under which conditions.  This approach focuses on the quality of the study to answer the question, and whether the question is appropriate to the stakeholder need.  Payers’ decisions are influenced by their assumptions about a hierarchical versus a continuum approach to evidence.  The payer’s conundrum in making decisions based on incomplete evidence will be discussed in the context of these competing views. Manufacturers must make strategic decisions on post-approval research investments based on market demands.  As the criteria for evaluating evidence evolve and US evidentiary standards begin to take shape, attention to and participation in these discussions will become critical to commercial success. This session will include a debate regarding whether or not the traditional evidence hierarchies exist within CER with insights from a medium-sized insurance company, a pharmaceutical manufacturer, and a consultant and academic who work in related areas.

Economic Outcomes Research Issues

IP13: ARE WE READY FOR A PRIME TIME TO CONDUCT RETROSPECTIVE COMPARATIVE EFFECTIVENESS STUDIES FOR ORPHAN DRUGS AND DISEASES
Moderator: Lou Garrison, PhD, Professor, Pharmaceutical Outcomes Research & Policy Program, Department of Pharmacy, University of Washington, Seattle, WA, USA
Panelists: Zhimei Liu, PhD, Associate Director, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA; Amy Guo, PhD, Senior Director, Health Economics & Outcomes Research, US CD&MA, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA; Peter Sun, MD, PhD, Vice President, Health Economics & Outcomes Research, Kailo Research Group, Fishers, IN, USA
ISSUE: Payers are increasingly seeking real-world information on the comparative effectiveness of orphan drugs.  Is the outcomes research community ready to meet this need?   Do we have enough new empirical databases, new methods, and new statistical tools to conduct scientifically valid retrospective data analysis for orphan drugs or diseases?
OVERVIEW: Prospective studies of orphan drugs are often very limited.  Hence, there is great interest in retrospective data analyses of orphan drugs or diseases, which are also difficult due to lack of sufficient data, adequate study design and analytical methods. Over time, more retrospective data sets, innovative study approaches, and advanced statistical tools have become available to us. We ask:  are we now prepared and able to conduct such studies for orphan drugs and diseases from health economic and outcomes research perspectives? The panel will discuss this readiness issue from three different angles: 1) Not yet, because of many daunting challenges; 2) Maybe, because of more new data sets and methods, 3) Yes, because we have done it (a real world cases). During the session, the moderator will review current status of and demand for retrospective data analysis of orphan drugs or diseases. The first panelist will deliver his not-yet-view through summarizing the major challenges in using retrospective data analysis for orphan drugs or diseases. The second panelist will present her may-be-view through discussing the availability of new empirical data sets and new study approaches that can help researchers to overcome some of the challenges. The third panelist will demonstrate his yes-view through further discussion of new data, new methods, and through a real world case.  During the last 10 minutes, audience will debate the readiness for retrospective CER of orphan drugs and diseases.

Clinical Outcomes Research Issues

IP14: WHO SHOULD FUND AND HAVE ACCESS TO SECONDARY DATA LINKAGES FOR ONCOLOGY OUTCOMES RESEARCH?
Moderator: Steven B. Clauser, PhD, Chief of the Outcomes Research Branch at the US National Cancer Institute, Outcomes Research Branch, US National Cancer Institute, Bethesda, MD, USA
Panelists: Joseph Lipscomb, PhD, Professor, Department of Health Policy & Management, Emory University, Rollins School of Public Health, Atlanta, GA, USA; Cathy J. Bradley, PhD, Professor, Department of Healthcare Policy and Research, Virginia Commonwealth University, Richmond, VA, USA; Kathleen Foley, PhD, Director, Strategic Consulting, Healthcare, Thomson Reuters, Cambridge, MA, USA
ISSUE: Current efforts to develop comprehensive linked data files for oncology outcomes research (OOR) are fragmented, resulting in limited access for researchers across government, academia, non-profit, commercial, and industry settings.  Funding and technical barriers limit academic/commercial collaborations. This issues panel will provide the academic and commercial perspectives on the role of data linkages, challenges with funding, barriers to broader data linkage and access to linked data, and ways to increase collaborative efforts linking public and private data with population-based cancer incidence and death registries. 
OVERVIEW: The use of secondary data for OOR is limited by the lack of comprehensive information from any given data source and multiple barriers to the creation of linked datasets.   Secondary data linkages have the potential to address comparative effectiveness questions, and are necessary for exploring treatment effectiveness, particularly in populations under-represented in cancer clinical trials.  Dr. Lipscomb will discuss his current federally funded project to strengthen the empirical base for cancer care quality evaluation in the state of Georgia by linking state cancer registry files with multiple public and private administrative data sources.  He will focus on administrative, legal, and fiscal challenges in developing an enduring state-level data infrastructure for OOR, and discuss practical approaches for addressing each challenge.  Dr. Foley will discuss the barriers to data linkages with private insurance claims from the perspective of commercial data management and research firms, and the potential benefits of increasing collaborations providing access to linked files for academics, non-profits and pharmaceutical companies. Dr. Bradley will discuss barriers to data linkages including access to private/commercially insured lives, and issues that prevent consolidation of datasets and collaborations across academic, government, and private institutions.  The panelists will offer solutions to these barriers and engage the audience in a discussion of ways to improve collaborations. 

Patient-Reported Outcomes Research Issues

IP15: DO PATIENT-REPORTED OUTCOMES ADD VALUE IN HEALTH CARE DECISIONS?
Moderator: Andrea LaFountain, PhD, CEO, Mind Field Solutions Corp., Fairfax, VA, USA
Panelists: Joseph C. Cappelleri, PhD, MPH, Senior Director, Biostatistics, Pfizer Inc., New London, CT, USA; Mitch Golant, PhD, Senior Vice President, Research and Training, Cancer Support Community, Washington, DC, USA
ISSUE: Patient-reported outcomes (PROs) as value statements in product claims, patient care and reimbursement.
OVERVIEW: The costs of healthcare continue to accelerate without parallel improvements in population health.  A measure of cost containment that is being instituted is reimbursement for outcomes.  While philosophically this idea is admirable, practically it creates a necessity for (1) adequate demonstration of outcomes in ‘uncontrolled’ clinical settings, (2) valid and sensitive measurements of benefit, perhaps beyond the restricted purview of clinical trial endpoints, and (3) accountability to achieve and demonstrate impact. This panel will debate (1) parameters of outcomes that are worthy of ‘claim’ and how these can be effectively measured and documented as product claims, (2) practical considerations of achieving clinically meaningful outcomes in ‘uncontrolled’ real-world settings, and (3) the role of respective healthcare constituents (manufacturer, physician, patient) in driving to effective outcomes with associated reimbursement. Panelists will highlight their positions on the ownership and accountability of driving improvement in Patient Reported Outcomes from assessment of need, intervention, and demonstration of impact.