Issue Panels
Monday, June 2, 2014
11:00 AM - 12:00 PM
ISSUE PANELS - SESSION I
Clinical Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Room 710b (7th Floor)

IP1: CROSS-FUNCTIONAL COLLABORATION ON CER - WHY CAN'T WE WORK TOGETHER?

Moderator:

Leona E. Markson, ScD, Executive Director, Comparative & Outcomes Evidence, Merck, West Point, PA, USA

Panelists:

Sebastian Schneeweiss, MD, ScD, Professor of Medicine and Epidemiology, Harvard Medical School, Boston, MA, USA

Robert Epstein, MD, MS, CEO & President, Epstein Health, LLC., Woodcliff Lake, NJ, USA

Lesley H. Curtis, PhD, Professor in Medicine, Duke University School of Medicine, and Duke Clinical Research Institute, Durham, NC, USA

ISSUE:

The perception of the potential strengths and limitations that “BIG DATA” and comparative effectiveness research (CER) can bring to evidence-based medicine and evidence-based decision making can vary significantly across different stakeholder groups.  Issues such as CER feasibility, level of pre-work needed before conducting CER and precision of findings are among those that may be perceived differently, along with timeliness, ability to proactively communicate results, and costs of evidence generation.  In particular, interpretation of what constitutes valid results in the presence of imperfect data and uncertain assumptions may be points of contention.   

OVERVIEW:

Cross-functional collaboration is essential to develop the CER agenda and data that will be useful for decision makers, but rarely are different perspectives directly discussed.  Understanding these perspectives and the rationale for such perspectives is a first step to overcoming the challenges. While the perspectives of various stakeholders may be quite different, usually there are areas of agreement that create opportunities for collaboration with few friction points.  Sometimes tools for assessing feasibility of CER can be useful to make different stakeholders more comfortable with such research and its interpretation. Panel Discussion: Each panelist will discuss the type of collaboration needed in order to design, conduct and interpret CER within the specific organizational settings they will represent in this session (academic; health system provider/payer; and cross-institutional perspectives).  The key issues that lead to obstacles to CER will be identified as well as potential strategies to overcome these obstacles.  Each panelist will be challenged to identify broad ways in which the overlap of interest and agreement among essential collaborators could grow to accommodate the interests of other parties involved.  Through the panel discussion, guiding principles will be suggested to support the conduct, interpretation and use of CER data for decision making.

Economic Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Room 518abc (5th Floor)

IP2: DOES HEALTH ECONOMICS HAVE A ROLE IN THE NEW ERA OF PERSONALIZED MEDICINE?

Moderator:

Scott Ramsey, MD, PhD, Member, Public Health Sciences, Fred Hutchinson Cancer Research Center, Seattle, WA, USA

Panelists:

Anirban Basu, MS, PhD, Professor, Health Services, University of Washington, Seattle, WA, USA

John Watkins, PharmD, MPH, BCPS, Pharmacy Manager, Formulary Development, Premera Blue Cross, Mountlake Terrace, WA, USA

David L. Veenstra, PharmD, PhD, Professor, Pharmaceutical Outcomes Research and Policy Program, University of Washington, Seattle, WA, USA

ISSUE:

What are the implications for health economics of the advent of personalized medicine empowered by Next Generation Sequencing, which allows very inexpensive multiplex assessment of multiple genetic variants that could influence therapy choices and outcomes?  As the cost of this technology falls while the clinical implications expand, what health economic approaches will be needed to address the important challenges in this rapidly evolving field? Dr. Ramsey, the moderator, will provide an overview of personalized medicine and the intersection with health economics methods. Dr. Veenstra will argue that the pace of innovation and paucity of clinical evidence in personalized medicine have incapacitated the use of cost-effectiveness analysis to inform policy decisions for the vast majority of upcoming personalized medicine technologies. Dr. Watkins will present the case for cost-effectiveness studies of personalized medicine and their utility for decision making in managed care. Dr. Basuwill present a health economist's perspective and discuss whether novel approaches are needed.  He will present a refined Expected Value of Individualized Care (EVIC) framework and outline future research needs and methods development. 

OVERVIEW:

Personalized medicine is undergoing a second, critical revolution with the introduction of a disruptive technology: Next Generation Sequencing.  Sequencing costs have dropped by orders of magnitude in the past few years, and clinicians and patients are confronted with the possibility of routine whole genome sequencing.  Generating evidence to inform the use of tens or even hundreds of clinically actionable genomic results will not be feasible.  The use of traditional cost-effectiveness analysis to inform reimbursement decisions and research investment will be challenging.  The panelists will discuss these challenges and outline novel approaches to understanding the uptake of personalized medicine, identifying stakeholder preferences, and delineating evidence thresholds.

Health Policy Development Using Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Room 520cf (5th Floor)

IP3: WHAT IS THE BEST WAY TO USE MCDA IN HEALTH TECHNOLOGY APPRAISAL?

Moderator:

Nancy Devlin, PhD, Research Director, Office of Health Economics, London, UK

Panelists:

Mark J. Sculpher, MSc, PhD, Professor of Health Economics, Centre for Health Economics, University of York, Heslington, York, UK

Stuart Peacock, DPhil, Co-Director, Cancer Control Research, BC Cancer Agency, Canadian Centre for Applied Research in Cancer Control, Vancouver, BC, Canada

Josie Godfrey, MAs, Associate Director, Highly Specialised Technologies, National Institute for Health and Care Excellence (NICE), London, UK

ISSUE:

There is increasing interest in the potential usefulness of multiple criteria decision analysis (MCDA) methods to inform pricing and reimbursement decisions. Most HTA systems consider multiple criteria, and the broad set of methods constituting MCDA arguably provide a means of supporting such decisions in a more transparent and consistent manner; however, there are sharply divergent views on how MCDA is best used as part of HTA processes.

OVERVIEW:

Some argue that MCDA should be used to provide a systematic way of taking into account performance on a standard set of criteria, which can be weighted using social preferences i.e. the stated preferences of the general public regarding the relative importance of each criterion. Others argue that the most appropriate role of MCDA is to help decision makers structure the decision problem, with the criteria and weights to be used in each decision emerging from the deliberative process. These approaches differ in important ways. Which is argued to be preferred depends, among other things, on (a) normative views about whose weights should count in social decisions about new technologies and (b) views on whether decision makers’ role should be restricted to making scientific judgments (i.e., judging the quality of the evidence), or should also extend to exercising social value judgments on behalf of taxpayers (c) the feasibility of reducing complex HTA decisions to a fixed set of criteria, or whether ‘special cases’ are inevitable, and (d) the benefit, in terms of improved decision making, weighed up against the cost and practicality of implementing each approach. In this issues panel, the panelists will each present their own views on these issues, and invite the audience to consider the arguments for and against these different ways of using MCDA in their own HTA systems.


11:00 AM - 12:00 PM
Room: Room 710a (7th Floor)

IP4: IS IT TIME FOR A NEW DRUG DEVELOPMENT PARADIGM?

Moderator:

Adrian Towse, MA, MPhil, Director, Office of Health Economics, London, UK

Panelists:

Penny Mohr, MA, Senior Vice President, Program Development, Center for Medical Technology Policy, Baltimore, MD, USA

Matthew Rousculp, PhD, Senior Director, Comparative Effectiveness and Health Policy Research, GlaxoSmith Kline, Research Triangle Park, NC, USA

Robert McDonough, MD, JD, MPP, Head, Clinical Policy Research and Development, Aetna, Hartford, CT, USA

ISSUE:

Drug development costs have increased markedly in recent years, causing manufacturers to seek creative solutions to improve efficiency.  At seemingly cross purposes with these aims, payers and health care systems are demanding comparative or relative effectiveness research (CER/RE) to aid coverage and pricing decisions.  Working in manufacturers’ favor, the  environment for conducting this type of research is changing rapidly-with innovative study designs and growing access to electronic data.  Some have argued that the time is ripe for a new drug development paradigm (NDDP) that synthesizes evidence development across the life cycle of a drug and takes advantage of the investment in electronic health records and efforts to harmonize regulatory and payer requirements for evidence to markedly change the way drug companies design evidence. Others feel the regulatory environment is the primary force driving evidence development, and they anticipate no major changes in regulatory demands, thus, no need to markedly change the way drug companies design trials.

OVERVIEW:

This panel debates a vision of how the future demand for and capacity to produce CER/RE will look in the United States and Europe.  One perspective draws from an intensive modified-Delphi process including key informant interviews with 90 payers, regulators, patient advocates, experts in health information technology, health system change, research methods and personalized medicine, senior executives in the life science industry and thought leaders in CER and RE.   An NDDP is defined for two drug archetypes based on facilitated discussions between executives and senior leaders from five major life science companies. According to this vision, large simple trials and adaptive licensing will play a much larger role in drug development in the future. Reaction and debate about the likelihood of this future vision is presented by a representative from the pharmaceutical industry and a representative from a health care payer in the United States.

Use of Real World Data Issues

11:00 AM - 12:00 PM
Room: Room 519ab (5th Floor)

IP5: REAL-WORLD EVIDENCE: IS IT FIT FOR PURPOSE? AND IF SO, WHEN?

Moderator:

Jennifer S. Graff, PharmD, Director, Comparative Effectiveness Research, National Pharmaceutical Council, Washington, DC, USA

Panelists:

Raj Sabharwal, MPH, Senior Manager, AcademyHealth, Washington DC, USA

Steven Pearson, MD, MSc, FRCP, President, Institute for Clinical and Economic Review, Boston, MA, USA

Reed Tuckson, MD, FACP, Managing Director, Tuckson Health Connections, Edina, MN, USA

ISSUE:

Despite the demand for better evidence on how treatments work in the real-world, few payers use real-world evidence (RWE) in coverage or formulary decisions. This is complicated by evidence hierarchies which base evidence utility on study design rather than whether the evidence fits the purpose needed (“fit for purpose”). Better understanding is needed of the scenarios or circumstances when a specific research design is preferred. When are both needed? When is information from a variety of sources complementary? Without greater clarity there is the potential for misuse or non-use of evidence to inform payer decision making.

OVERVIEW:

A framework for determining when a Randomized Controlled Trial (RCT) or RWE is fit for purpose was developed based upon a literature scan, expert interviews, and a facilitated discussion roundtable of 12 payers and other decision makers. The core components of the framework include the type of clinical question (e.g., safety, effectiveness, adherence), payer decision (e.g., determine coverage and tier, remove coverage, alter utilization management), an understanding of the strength of the existing evidence, and contextual factors, all of which help to clarify and identify the types of new evidence desired. The framework and issues will be presented (Sabharwal). Panelists will consider and challenge this framework from the vantage point of a large national managed care organization (Tuckson) and a consortium working with state Medicaid directors (Pearson). They will reflect on the utility of the framework and debate the types of evidence needed using illustrative case studies. Audience participation will be sought on these case studies and on the broader utility of the framework for the researchers and payers. Greater clarity and understanding of what evidence is fit for purpose may improve the availability of the desired types of evidence needed for payer decisions and medical management policies.

Tuesday, June 3, 2014
11:00 AM - 12:00 PM
ISSUE PANELS - SESSION II
Economic Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Room 518abc (5th Floor)

IP6: VALUING TARGETED THERAPIES AND THEIR COMPANION TESTS – ARE WE LEAVING SOME OF THE VALUE ON THE BENCH (OR AT THE BEDSIDE)?

Moderator:

Scott Ramsey, MD, PhD, Member, Fred Hutchinson Cancer Research Center and Professor, School of Pharmacy School of Medicine Institute for Public Health Genetics, University of Washington, Seattle, WA, USA

Panelists:

Adele R. Weston, PhD, Executive Vice President & Senior Scientist, Health Economics and Outcomes Research, Optum, Sydney, Australia

Deborah Marshall, PhD, Associate Professor, Community Health Sciences, University of Calgary, Calgary, AB, Canada

ISSUE:

To debate the ‘evaluation of value’ of targeted therapies and their companion diagnostic tests. Are we capturing all the value of the combined test-drug pathway to the health system and clinicians, as well as to patients and their families? Are the methods we currently use to evaluate the cost-effectiveness for broader population-based therapies appropriate for the more personalized approach?

OVERVIEW:

Dr. Weston will summarize the current methods for evaluating the value for money of targeted test-drug combinations. She will use the Australian Government’s Co-dependent Technology evaluation process as an example, as this remains the only formal system internationally that assesses test-drug technologies together.  She will argue that, with careful consideration, current cost-effectiveness methods can largely accommodate targeted test-drug pathways and that we should not make exceptions or change methodology for targeted therapies. In contrast, Dr. Marshall will present evidence and examples to demonstrate that there are important additional value considerations that are not typically or easily quantified through standard economic evaluation approaches. Further, results from conjoint analysis studies suggest that the value of targeted tests differ considerably between clinicians and patients and the general public – whose values should be used to inform decisions? Professor Drummond will summarize the arguments and invite the audience to comment on whether different methods are needed to truly determine value of the personalized approach and inform coverage and reimbursement decisions.

Health Policy Development Using Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Room 520cf (5th Floor)

IP7: HOW CAN WE DEFINE AND ACQUIRE SUFFICIENT EVIDENCE TO SUPPORT REIMBURSEMENT DECISIONS FOR MEDICAL DEVICES?

Moderator:

Mark Sculpher, MSc, PhD, Professor of Health Economics, Centre for Health Economics, University of York, Heslington, York, UK

Panelists:

Pascale Brasseur, EconD, Health Economics and Reimbursement Director, Medtronic International Trading Sarl, Tolochenaz, Switzerland

Mirella Marlowe, MA, MBA, Programme Director, Devices and Diagnostics Systems, National Institute for Health and Care Excellence (NICE), London, UK

Stuart Mealing, MSc, Director of Health Economics, ICON Health Economics, Oxford, UK

ISSUE:

Health Technology Assessment (HTA) in many jurisdictions is predicated on a formal assessment of clinical and cost-effectiveness, with randomized clinical trials (RCTs) being the preferred source of efficacy estimates. However, the evidence available at launch to support funding decisions is often characterized by gaps and other forms of uncertainty. This is often particularly acute with medical devices where evidence generation is scientifically and financially challenging. In such an environment, the question arises as to what constitutes a sufficient level of evidence in order for health systems to make appropriate decisions. Should ‘sufficiency’ be defined differently for devices than for pharmaceuticals? 

OVERVIEW:

A range of perspectives will be presented on what constitutes sufficient evidence in health technology assessment of medical devices. The first speaker will present a view from industry and in particular the relevant commercial challenges and regulatory barriers to generating evidence. The second speaker will then present the issues faced by reimbursement agencies in reaching funding decisions for medical devices. In particular, they will discuss how the agency makes recommendations in situations where RCT evidence is unavailable. The panel will then consider analytic approaches to defining evidence sufficiency based on the costs and benefits of further research, and discuss potential roles for health systems in incentivizing research. The objective of this session is to increase awareness of the challenges in evidence generation for medical devices, discuss how sufficiency in evidence can be defined/operationalized and determine how the gaps between actual and sufficient evidence can be filled. Attendees will have the opportunity to consider the issues faced by reimbursement agencies and manufacturers in this challenging area.


11:00 AM - 12:00 PM
Room: Room 710a (7th Floor)

IP8: GUIDELINES AND STANDARDS FOR OBSERVATIONAL STUDIES: ARE WE HEADED FOR DISCOURSE OR HARMONY?

Moderator:

Robert W. Dubois, MD, PhD, Chief Scientist, National Pharmaceutical Council, Washington, DC, USA

Panelists:

Rachael Fleurence, PhD, Program Director, Comparative Effectiveness Research (CER) Methods and Infrastructure Program, Patient-Centered Outcomes Research Institute (PCORI), Washington, DC, USA

Sally C. Morton, PhD, Professor & Chair, Biostatistics, University of Pittsburgh, Pittsburgh, PA, USA

Jean Slutsky, PA, MSPH, Director, Center for Outcomes and Evidence (COE), Agency for Healthcare Research and Quality (AHRQ), Rockville, MD, USA

ISSUE:

The need for determining how to conduct high-quality observational studies is particularly acute now given the desire to produce valuable evidence for comparative effectiveness research decisions. While research standards for conducting observational studies have evolved and proliferated, currently there are not uniform standards for observational studies. Do we need standards for observational studies or will such standards stifle innovation? If we need standards, should there be one set or will many suffice and be more supportive of high-quality research?

OVERVIEW:

As a foundation for the panel, a systematic review of 18 guidelines and standards for conducting observational studies will be shared highlighting areas of commonality and disagreement. While most guidelines and standards covered similar elements, the level of detail and degree to which the standards or guidelines are actionable differed. Panelists will provide their perspective and the implications of one or many existing research best practices and standards as funders of research (Dr. Rachael Fleurence from the Patient-Centered Outcomes Research Institute (PCORI)), conductors of research (Dr. Sally Morton from the University of Pittsburgh), and decision makers seeking to disseminate the results of the research (Ms. Jean Slutsky from PCORI and previously the Agency for Healthcare Research and Quality (AHRQ)).  They will discuss 1) whether a common and agreed upon set of standards is needed; and if so, 2) whether one set of standards or guidelines is desirable; and if so, and 3) how to harmonize the current guidelines standards via a consensus process. The moderator will encourage audience participation regarding the policy implications of multiple standards and the need for harmonization moving forward.


11:00 AM - 12:00 PM
Room: Room 710b (7th Floor)

IP9: THE NEXT FRONTIER FOR RISK-SHARING AGREEMENTS IN THE UNITED STATES: DOES THE CHANGING HEALTH CARE LANDSCAPE BRING NEW PROMISE FOR PARTNERSHIPS BETWEEN MANUFACTURERS AND PAYERS?

Moderator:

Josh J. Carlson, MPH, PhD, Assistant Professor, Pharmaceutical Outcomes Research and Policy Program, University of Washington, Seattle, WA, USA

Panelists:

Lou Garrison, PhD, Professor, Pharmaceutical Outcomes Research and Policy Program, School of Pharmacy, University of Washington, Seattle, WA, USA

Adrian Towse, MA, MPhil, Director, Office of Health Economics, London, UK

Peter J. Neumann, ScD, Professor & Director, The Center for the Evaluation of Value and Risk in Health, The Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA

ISSUE:

Risk-sharing agreements (RSAs) have been leveraged frequently in Europe, and offer the potential for earlier access to new products by linking coverage and reimbursement levels to real-world performance or utilization of the product.  There are potential advantages for patients, payers, and manufacturers; however, the dearth of schemes in United States raises questions as to why their use has been limited and also as to what might become feasible in the rapidly evolving health care system, especially with the new emphasis on accountable care organizations.

OVERVIEW:

Lou Garrison will present findings from recently completed interviews with United States and European manufacturers and payers regarding their RSA experiences and a comprehensive RSA database review.  Specifically, he will highlight what has been done in both regions, and the perceived benefits and challenges in the use of RSAs across regions.   RSAs potentially promote earlier access to new products but face challenges in terms of the lack of data infrastructure, heterogeneous patient populations, and patient compliance, among other factors.  Adrian Towse will elaborate on the European experience: what has been done, and whether the United States can really learn from the European examples.  Is the use of RSAs in Europe motivated in large part by the desire of manufacturers to keep discounts confidential because of the reference pricing system?  What are the key considerations to be taken into account to fulfill the promise of RSAs in the U.S. context? Peter Neumann will provide a contrasting United States viewpoint, discussing the barriers to using RSAs, whether these barriers can be overcome, and whether the use for these agreements will expand as the health care system evolves.  Does the multi-payer system exacerbate the effort required to implement an RSA?  Will accountable care organizations find RSAs more attractive than traditional payers?

Use of Real World Data Issues

11:00 AM - 12:00 PM
Room: Room 519ab (5th Floor)

IP10: ARE WE GETTING REAL ABOUT REAL-WORLD EVIDENCE REQUIREMENTS?

Moderator:

Kathleen E. Hughes, MBA, Vice President, Health Economics and Outcomes Research, Avalere Health LLC, Washington, DC, USA

Panelists:

John P. Graham, PharmD, Executive Director, Health Services, Health Economics and Outcomes Research, Bristol-Myers Squibb, Plainsboro, NJ, USA

Richard Lieblich, RPh, Vice President, Industry Relations, MedImpact Healthcare Systems, Inc., San Diego, CA, USA

John Watkins, PharmD, MPH, BCPS, Pharmacy Manager, Formulary Development, Premera Blue Cross, Mountlake Terrace, WA, USA

ISSUE:

Avalere had experts rank the intermediate-term probability that the following parameters would be employed and affect market access decisions made by regulators and/or payers, including those in the private sector. All parameters directly or indirectly involve the use of RWE and were ranked on a scale of 1-6, with 6 being the highest. The results are: Safety program requirements (6), Registry requirements (6), More comparative effectiveness techniques in coverage decisions (6), Effectiveness, not efficacy (5), Heterogeneity of treatment effects (5), Pragmatic real-world settings (4), Conditional approvals (coverage with evidence development, risk plans) (4), Focus on effect size, not just superiority (4), Patient involvement in other post-marketing activity  (including adverse event reporting) (4), More patient-centered outcomes (3), Patient involvement in trial results use (3), Patient involvement in research design (3), Bayesian and adaptive approaches to design, accounting for prior probabilities (2). Our panel seeks to go beyond generalities and present specifics on what private payers and manufacturers are actually requiring and generating in terms of RWE.  Both sides will present future predictions and practical advice for others.

OVERVIEW:

Panelist 1 (moderator) will review the recently-completed research results on anticipated use of RWE.  Panelist 2, a representative from a pharmaceutical manufacturer’s HEOR function, will discuss what he has seen thus far in terms of developments in the field, i.e., regulatory and payer requirements, and how these have affected how his company prepares and uses RWE.  Panelists 3 and 4, a payer medical director and a primary user of RWE at a large insurer, will explore how RWE is used at their plans, trends in the future, and practical advice to manufacturers in demonstrating value using RWE.   All presenters will engage in a dialogue facilitated by the moderator, and the audience will be able to direct questions to the panelists.

2:15 PM - 3:15 PM
ISSUE PANELS - SESSION III
Economic Outcomes Research Issues

2:15 PM - 3:15 PM
Room: Room 710b (7th Floor)

IP11: MOVING BUDGET IMPACT ANALYSIS FORWARD: LEVERAGING BIG DATA TO ENABLE USE IN REAL TIME OPERATIONAL DECISION MAKING

Moderator:

Barry Chaiken, MD, MPH, Chief Medical Information Officer, Infor and Adjunct Professor, Health Information Technology, Boston University, New York, NY, USA

Panelists:

Elizabeth L. Meyers, BSN, MS, Industry Strategy Director, Healthcare Analytics, Infor and PhD Student/Guest Lecturer, Institute for Health Informatics, University of Minnesota, Baldwin, WI, USA

Stephanie Earnshaw, PhD, Vice President, Health Economics, RTI Health Solutions, Research Triangle Park, NC, USA

Andrew J. Knighton, MS, CPA, PhD Candidate, Instructor & Teaching Assistant, Institute for Health Informatics, University of Minnesota, Minneapolis, MN, USA

ISSUE:

Budget Impact Analysis (BIA) is a commonly performed economic analysis.  However, BIAs are not widely utilized by health care decision makers for everyday decisions. Meyers:  BIAs should be integrated into commercial business intelligence products and powered by big data so that they can be used for all levels of operational decision making; Earnshaw:  BIAs should continue to be performed on a case by case basis by experts; Knighton: Data science methods and techniques should be leveraged to integrate health data into decision making processes.

OVERVIEW:

ISPOR has developed a tool box for health care decision makers.  These tools are the summation of years of thought leadership and research by academic and subject matter experts.    Although they have been adopted for use in coverage determination by insurance and governmental providers, they are not widely used to improve the everyday decisions of health system decision makers.   The day-to-day decisions made by health care leaders are more likely to be based on expert opinion.  Quality based payments are driving value based care, and coverage decisions at a national level need to be augmented at the local level.  Each health care organization makes technology and intervention choices based on the needs of the specific patient population for which they hold accountability. And, because populations and providers vary, the adoption of technology may also vary at the local level.  The Budget Impact Analysis (BIA) is a tool that can bridge this gap to improve decisions, value and the quality of health care, because BIAs can be adjusted to meet the goals of a particular population with particular needs. Panelists will review the current uses of BIA and contrast them with current decision technologies utilized by health system leaders.  Discussion will include BIA automation for use in real-time health care decisions utilizing advanced technology, analytics and big data.

Health Policy Development Using Outcomes Research Issues

2:15 PM - 3:15 PM
Room: Room 710a (7th Floor)

IP12: IS THE US MEDICARE PROGRAM’S USE OF COMPARATIVE EFFECTIVENESS RESEARCH CONSISTENT WITH THE AFFORDABLE CARE ACT’S RESTRICTIONS?

Moderator:

Peter J. Neumann, ScD, Professor & 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 bio

Panelists:

Penny Mohr, MA, Senior Vice President, Program Development, Center for Medical Technology Policy, Baltimore, MD, USA

Art Small, MD, Head, Oncology Outcomes Research, US Medical Affairs, Genentech, Inc., South San Francisco, CA, USA

James D. Chambers, PhD, MPharm, MSc, Assistant Professor, 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:

The Affordable Care Act (ACA) prevents Medicare from using comparative effectiveness research (CER) as the sole basis to deny coverage of medical technology.  Research shows that Medicare’s use of CER in national coverage determinations (NCDs) varies by technology type and indication.  Panelists will discuss the implications of this inconsistency and research evaluating the relationship between the available evidence base and Medicare coverage policy.  Panelists will discuss this issue from the perspectives of industry (Art Small), health plans (Penny Mohr), and academic researchers (James Chambers). 

OVERVIEW:

The US Medicare program covers medical technology deemed reasonable and necessary for the diagnosis or treatment of illness or injury.  However, the Centers for Medicare and Medicaid Services (CMS) has not defined their interpretation of reasonable and necessary, nor what level of evidence is sufficient, leaving coverage policy uncertain.  This uncertainty has consequences for Medicare beneficiaries’ access to medical technology and for manufacturers as they bring innovations to the market.  The ACA’s restriction that CER findings should not be the sole determinant to deny coverage complicates matters.  New research, to be discussed by the panelists, is relevant to this issue as it attempts to map the evidence base reviewed by CMS for all Medicare NCDs.  It has identified that Medicare has had variable access to CER, and its availability varies by technology type and indication.  The panelists will discuss the findings of analyses evaluating the role of comparative effectiveness research on coverage outcome, and whether Medicare coverage policy is consistent with available evidence.  Further, the panelists will debate if CMS’ incorporation of CER into coverage policy is appropriate, and in accordance with the ACA’s restrictions.  This issues panel will provide a forum to discuss the Medicare program’s evolving use of evidence, and debate how evidence should be used to guide coverage policy.


2:15 PM - 3:15 PM
Room: Room 518abc (5th Floor)

IP13: IS HTA MORE SIMILAR THAN DIFFERENT ACROSS THE ATLANTIC? COMPARISON OF CANADA AND EUROPE

Moderator:

Lou Garrison, PhD, Professor & Associate Director, Pharmaceutical Outcomes Research & Policy Program, Department of Pharmacy, University of Washington, Seattle, WA, USA bio

Panelists:

Wim Goettsch, PhD, Project Leader, EUnetHTA WP5 on Relative Effectiveness of Pharmaceuticals and Advisor of International Affairs and Academia, Health Care Insurance Board (CVZ), Diemen, The Netherlands

Adrian D. Griffin, MSc, Vice President, HTA & Market Access Policy, Johnson & Johnson, Buckinghamshire, UK

Tammy Clifford, PhD, Vice President, Strategic Initiatives & Chief Scientist, Canadian Agency for Drugs and Technologies in Health (CADTH), Ottawa, ON, Canada

ISSUE:

Countries within the EU and at the pan-European level have been closely collaborating on how to improve HTA processes that are evolving to meet the needs of local health system decision makers.  HTA organizations often face similar challenges, including process guidelines, scientific advice, data sharing, assessment processes, etc.  In addressing these issues, HTA agencies also need to respond to the local needs of individual health systems and payers.  Are there enough similarities between the Canadian and European HTA systems to allow for collaborations to occur?

OVERVIEW:

This panel will include representatives from Canadian and European HTA agencies.  The purpose is to provide an overview of the commonalities and challenges shared, as technologies change and HTA evolves.  Each panelist will aim to explore the following from each of her/his perspective: 1) Where are the best points of collaboration beyond borders?, 2) There are a lot of similarities in how HTA is performed and subsequently used in Canada through common drug review at the provincial level; how does this relate to possible joint activities in Europe?, 3) How do the EUnetHTA guidelines relate to the guidelines of Canadian Agency for Drugs and Technologies in Health?  Are there any similarities and differences?, 4) What is the role of the International Network of Agencies for Health Technology Assessment (INAHTA)?  Could INAHTA play a mediating role?, 5) Are there opportunities for joint meetings on specific products or emerging policy issues or processes?, 6) What, if any are the current activities underway on harmonization and sharing of best practices?, and 7) What are some lessons learned from past and current collaboration initiatives?


2:15 PM - 3:15 PM
Room: Room 519ab (5th Floor)

IP14: VALUE-BASED PRICING ACROSS INDICATIONS: ARE THE CONCEPTUAL AND IMPLEMENTATIONAL CHALLENGES WORTH IT?

Moderator:

Adrian Towse, MA, MPhil, Director, Office of Health Economics, London, UK

Panelists:

Ansgar Hebborn, PhD, Head, Global Market Access Policy, F. Hoffmann-La Roche AG, Basel, Switzerland

Jeffrey S. Hoch, PhD, Director, Pharmacoeconomics Research Unit & Co-Director, Canadian Centre for Applied Research in Cancer Control (ARCC), Toronto, ON, Canada

Paolo Daniele Siviero, MS, Head of Economic Strategy & Pharmaceutical Policy, Director of Pharmaceutical Policy Unit, Italian Medicines Agency (AIFA), Rome, Italy

ISSUE:

In this issues panel we will discuss the concept of value based pricing across indications (“multiple-indication” or “indication-specific” pricing), the case for and against indication-specific prices and how different prices for the same molecule could actually be implemented in a reimbursement system. Adrian Towse, the moderator of this panel, will briefly introduce into the subject and explain the theoretical benefits of indication-specific pricing as a means to provide relevant incentives for pharmaceutical R&D and for the optimal utilization of a medicine. Jeffrey Hoch will explore in theory and in practice how the issue has played out in Canada with a focus on oncology products. Paolo Siviero will address the topic from the perspective of a health care payer and will focus on experience with indication-specific pricing gathered so far in Italy.  Ansgar Hebborn will present, from an industry perspective, on the opportunities and challenges of indication-specific pricing as well as on the practical issues with the implementation of indication-specific pricing models.

OVERVIEW:

Most public payers request that reimbursed prices for medicines reflect the added clinical and economic value that the innovation delivers to patients, their families and societies. Where medicines have been approved in more than one indication, this value is likely to differ between indications. Many of today’s pricing and reimbursement systems are not sufficiently flexible to reflect these differences in the price of the medicine. As a consequence patient access to a medicine remains sub-optimal compared to its clinical potential when reimbursement is restricted to specific indications of perceived highest value. Should indications of the same medicine be priced differently? What needs to be done to realize the theoretical benefits of indication-specific pricing models? Will it be possible to overcome the practical barriers to the introduction of indication-specific pricing?

Patient-Reported Outcomes & Patient Preference Research Issues

2:15 PM - 3:15 PM
Room: Room 520cf (5th Floor)

IP15: IS PERSONALIZING ADHERENCE THE SOLUTION FOR HELPING ALL PATIENTS TAKE THEIR MEDICINES CORRECTLY?

Moderator:

Dana P. Goldman, PhD, Leonard D. Schaeffer Chair & Director, Schaeffer Center for Health Policy & Economics, University of Southern California, Los Angeles, CA, USA

Panelists:

Anupam B Jena, MD, PhD, Assistant Professor of Health Care Policy and Medicine, Harvard Medical School, Cambridge, MA, USA

Ross Maclean, MD, Chief Operating Officer, Precision Health Economics, Los Angeles, CA, USA

Seth Seabury, PhD, Associate Professor of Research, Department of Emergency Medicine, Keck School of Medicine of USC, Los Angeles, CA, USA

ISSUE:

Academic literature has consistently shown that patients utilize less than the prescribed dosages of their medications, even though adherence has been shown to significantly increase patient health outcomes and quality of life. Although poor adherence is a well-documented and costly problem, it is still largely unclear which interventions are most effective for which patient populations due to the individual factors affecting nonadherence. This panel will consider a variety of approaches to targeting the right intervention to the right patient, or “personalizing” adherence interventions. Dr. Goldman will moderate and provide an overview of the policy issues raised by poor medication adherence. ­­Dr. Jena, as a professor at Harvard Medical School will provide the physician’s perspective. Dr. Maclean, as a UK physician working in the pharmaceutical industry and member of the PhRMA Medication Adherence Advisory Council will provide the international and industry perspectives, and Dr. Seabury will provide the front-line perspective as a researcher at one of the largest emergency departments in the United States.

OVERVIEW:

The failure of patients to adhere to their medications as prescribed is widely recognized as a significant public health problem. Many interventions to raise adherence have been proposed, but most show only modest improvements. This is unlikely the result of such interventions having modest effects across all patients, but rather heterogeneity in the way patients respond to specific interventions. Effectiveness of an intervention varies across people with different cognitive and personality characteristics, and adherence is a complicated cognitive process. How can we identify patients at risk for nonadherence? What role do new payment schemes, such as insurance benefit design, play in adherence outcomes? Should efforts to improve adherence focus on reducing financial barriers, increasing patient-provider communication, or modifying patient behaviors? What lessons can be learned from other health behaviors that have substantially improved over time?

Wednesday, June 4, 2014
8:30 AM - 9:30 AM
ISSUE PANELS - SESSION IV
Health Policy Development Using Outcomes Research Issues

8:30 AM - 9:30 AM
Room: Room 518abc (5th Floor)

IP16: PERSONALIZED MEDICINE IN THE ERA OF BIG DATA: HOW WILL THIS AFFECT YOU?

Moderator:

Amalia M. Issa, PhD, MPH, Professor & Chair, Department of Health Policy and Public Health & Program in Personalized Medicine & Targeted Therapeutics, University of the Sciences in Philadelphia, Philadelphia, PA, USA

Panelists:

Gillian Bartlett-Esquilant, PhD, Associate Professor, Department of Family Medicine, McGill University, Montreal, QC, Canada

Martin Dawes, MD, FRCGP, Royal Canadian Legion Professor & Head, Department of Family Practice, University of British Columbia, Vancouver, BC, Canada

Gary E. Marchant, JD, PhD, Regents' Professor of Law, Sandra Day O’Connor College of Law, Arizona State University, Tempe, AZ, USA

ISSUE:

Big data that is increasingly being generated from whole genome sequencing and other personalized genomic medicine technologies has led to a tension between scientists, clinicians and other stakeholders who assert that using this information is premature, and those who contend that use of this big data will enable health care providers and patients to make better decisions about treatment options. 

OVERVIEW:

Advances in genomic technologies (the “-omics”) and associated diagnostic and therapeutic interventions have tremendously increased the volume and type of health care information that is pertinent to clinical decision making for treatment recommendations. This vast amount of information  (commonly referred to as “big data”) has the potential to improve outcomes and consequent cost savings. However, this increasingly voluminous amount of information presents with challenges to clinical interpretation (e.g., providers are not equipped to interpret the extensive data concerning patient conditions, diagnostic and imaging results, whole genome sequencing and other genomics, outcomes, etc), to economic reimbursement by insurers and payers, and the potential for suboptimal utilization of the data with possible consequent liability concerns. The moderator, Amalia M. Issa, will present an overview of the state of `big data’ in the field of personalized medicine and set the stage for debate on whether we are moving too fast in our quest after big data. Gillian Bartlett-Esquilant and Martin Dawes will use their experience of developing primary care pharmacogenomics and use of big data to discuss the opportunities and challenges to use and interpretation of this information by clinicians and the economic costs of not using this information vs. implementation in clinical practice. Gary Marchant will provide a perspective on emerging liability aspects of using big data to drive personalized medicine forward. Ideas will be debated and future research directions discussed. The panel will actively engage the audience in the discussion.


8:30 AM - 9:30 AM
Room: Room 519ab (5th Floor)

IP17: THE AFFORDABLE CARE ACT: WILL IT HELP OR HARM THE LANDSCAPE FOR BIOMEDICAL INNOVATION AND TECHNOLOGY?

Moderator:

Amitabh Chandra, PhD, Professor of Public Policy, John F Kennedy School of Government, Harvard University, Cambridge, MA, USA

Panelists:

Michael Chernew, PhD, Leonard Schaeffer Professor of Health Care Policy, Harvard Medical School, Boston, MA, USA

Anup Malani, PhD, JD, Lee and Brena Freeman Professor, University of Chicago Law School, Chicago, IL, USA

Darius N. Lakdawalla, PhD, Professor, Quintiles Chair in Pharmaceutical Development and Regulatory Innovation, University of Southern California, Los Angeles, CA, USA

ISSUE:

Will the Affordable Care Act (ACA) strike the right or wrong balance between stimulating innovation and ensuring patient and payer access to new medical technologies? Dr. Chandra, Director of Health Policy Research at Harvard University, will moderate the discussion and provide an overview of the pressing policy issues. Dr. Chernew, Vice Chair of the Medicare Payment Advisory Commission (MedPAC), will represent the payer’s perspective. Dr. Lakdawalla, the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation, will represent the innovator’s perspective, and Dr. Malani, Professor of Law and of Medicine at the University of Chicago, will provide a legislative perspective.

OVERVIEW:

Continued discovery of new biomedical technologies depends on maintaining sufficient rewards for investments in research.  However, paying more to innovators may translate into higher prices for patients and less access to valuable technologies. This results in a policy-making trade-off between the need to stimulate innovation for tomorrow’s patients and the need to ensure access to care for today’s patients. The ACA has a variety of competing effects on this trade-off.  It expands the Medicaid program, which commands the lowest prices for medical technology in the marketplace.  Yet it has avoided a move towards allowing the federal government to use its full buying power to negotiate health technology prices down, and it likely expands the size of the market for medical care.  At the same time, the ACA promises to dramatically alter the incentives that physicians face for prescribing health technologies to patients.  What will these competing effects mean both for today’s patients’ access to care, and tomorrow’s patients access to new health care technologies?


8:30 AM - 9:30 AM
Room: Room 520a (5th Floor)

IP18: RISK SHARING FOR MEDICAL DEVICES: HAS THE TIME COME?

Moderator:

Susan Garfield, DrPH, Senior Vice President, Market Access, Health, GfK Market Access, Wayland, MA, USA

Panelists:

Michael Ferguson, PhD, Director, Health Economics and Comparative Effectiveness Research, Health Economics & Reimbursement, Boston Scientific, Natick, MA, USA

David Ertel, MBA, MPH, Chief Financial Officer, Einstein Healthcare, Philadelphia, PA, USA

Maria Lopes, MD, Chief Marketing Officer, Geisenger Health Systems, Danville, PA, USA

ISSUE:

Risk sharing agreements have been used to enable drugs to gain access to markets, with payment conditional upon the drug delivering on pre-defined results. However, to date risk sharing has not been largely adapted to coverage of and payment for medical devices.   The emergence of Accountable Care Organizations in the U.S. may change that since these organizations both provide care and are financially at risk for long term clinical outcomes. With this shift, are risk sharing arrangements for medical devices more applicable?  Will med device companies be willing to bear the financial risk of device failure in order to gain access to certain parts of the U.S. market? The moderator will present an overview of the risk sharing concept. Dr. Lopes will provide the ACO perspective; Dr. Ferguson will represent the medical device industry and Mr. Ertel will discuss how these arrangements might impact hospitals.

OVERVIEW:

Risk sharing arrangements may be ideally suited to medical devices as it is easier to assess outcomes without concerns over whether patient adherence is impacting results. However, medical devices have historically been purchased by hospitals and reimbursed under a procedure-specific diagnosis related group by a third party payer or via a technology specific pass-through. New quality measures are shifting some of the risk onto hospitals, changing the paradigm where devices are selected based on physician preference or low cost. Simultaneously, ACOs and Integrated Delivery Networks are device purchasers uniquely positioned to achieve financial benefit from using high quality medical devices, especially if outcome assessment is supported by existing EMRs or simple registries. This panel will discuss the impact of ACOs, IDNs and the Medicare Quality initiative on the medical device industry and explore the potential for risk sharing to emerge as a strategy to make novel, clinically impactful medical devices available to patients more rapidly.


8:30 AM - 9:30 AM
Room: Room 520b (5th Floor)

IP19: CAN MOBILE HEALTH REVOLUTIONIZE HEALTH OUTCOMES RESEARCH?

Moderator:

Kathleen E. Hughes, MBA, Vice President, Health Economics and Outcomes Research, Avalere Health LLC, Washington, DC, USA

Panelists:

Betsy J Lahue, MPH, Vice President, Health Economics and Outcomes Research, Becton Dickinson, Franklin Lakes, NJ, USA

Yarmela Pavlovic, JD, Partner, Hogan Lovells US LLP, Philadelphia, PA, USA

Jo Carol Hiatt, MD, MBA, Chair, National Product Council, and Assistant Medical Director, Southern California Permanente Medical Group Business Administration, Pasadena, CA, USA

ISSUE:

Observers cite three stages of innovation and characterize the current state of mobile health (mHealth) as somewhere between 1) hype and 2) hope, but a long way from 3) happiness. Creative use of mobile and wearable health information and sensing technologies has potential to reduce health care costs and improve well-being.  mHealth also has potential to revolutionize health outcomes research through the expansion of existing methods and the creation of new data capture/analytic techniques. Disruptive technology like mHealth brings the challenge of introducing compatibility with the legacy aspects of health care, including health services research, regulation, and payment. A few challenges include: 1) Privacy: controls, concerns with privacy, 2) Electronics: hardware, software, telecommunications differences leading to interface/interoperability difficulties, 3) Content: variable types, formats, content, frequencies, distribution of data/reports, 4) Processes: manipulation differences for aggregation, disaggregation, extraction, calculation,  reporting formats, 5) Procedures: variously automated/manual because of different levels of electronic data processing capabilities, 6) Files and entire databases: multiple structures and capabilities, organizational, indexing differences, 7) Organizations/organizational components: differences in functions, forms, interfaces, intra-system interactions. Patients, clinicians, researchers, regulators, and payers need to participate in the introduction of mHealth. Our multi-stakeholder panel brings together major points of view to sort out mHealth's promises and realities.

OVERVIEW:

The moderator will provide an overview of recent developments in mHealth.  Panelist 2, a device manufacturer/HEOR specialist, will relate how her and like companies develop technologies that meet patient and research needs, including data for payers.  Panelist 3, a former regulator, will recap FDA stances to date on the technology and data needed to support safety and efficacy claims.   Panelist 4, a physician-administrator/technology assessor/payer will review progress in technology introduction within her IDN.  She will address the promise of using mHealth data integrated with other “big” data analytics to reduce costs and provide better quality, patient-centered care.


8:30 AM - 9:30 AM
Room: Room 520c (5th Floor)

IP20: AUSTERITY MEASURES VERSUS EARLY ACCESS TO INNOVATIVE TREATMENT: HOW TO STRIKE A BALANCE BETWEEN PATIENTS' NEEDS AND BUDGET AVAILABILITIES?

Moderator:

Vladimir Zah, Health Economist and Founder, ZRx Outcomes Research Inc., Belgrade, Serbia and Montenegro

Panelists:

Andrea Manca, MSc, PhD, Professor, Centre for Health Economics, University of York, York, UK

Zoltan Kalo, MD, MSc, PhD, Chief Executive Officer, Syreon Research Institute, Budapest, Hungary

Katarzyna Kolasa, PhD, Senior Research Fellow, Department of Pharmacoeconomics, Medical University of Warsaw, Warsaw, Poland

ISSUE:

In the era of austerity measures, it is very tempting to deny reimbursement to patients in the early stage of diseases. Are the savings justifiable because they release resources for other more severe ill patients? Or, are the savings unjustifiable because short term gains do not outweigh the costs incurred due to disease progression in the long term?

OVERVIEW:

In the time of economic crisis, the search for potential cost containment measures in the health care sector evolves. On one hand, it is very tempting for decision makers to postpone the treatment initiation for various health conditions or deny preventive measures across healthy individuals in order to limit budget spending.  It will release funds for more urgent needs such as treatment of severe ill patients. On the other hand, there are others who claim that it is irrational to postpone early initiation of the treatment. The argument is that the costs of long-term consequences of untreated diseases may outweigh any initial savings. It is easier to prevent than treat. There are cases to prove that costs of early detection are more than likely offset by projected future savings. Both sides of the issue will be debated. This session will elicit contributions and feedback from the participants.

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