Issue Panels
Monday, May 21, 2018
11:00 AM - 12:00 PM
BREAKOUT SESSION
Patient-Reported Outcomes & Patient Preference Research Issues

11:00 AM - 12:00 PM
Room: Hall A (Level 100)

IP1: DESIGNING A PATIENT-CENTERED VALUE FRAMEWORK TO GUIDE SHARED DECISION MAKING IN ONCOLOGY: WHY, WHAT, AND HOW?

Moderator:

Jalpa A. Doshi, PhD, Professor of Medicine, Perelman School of Medicine; Director, Economic Evaluations Unit, Center for Evidence-based Practice; Director, Value-based Insurance Design Initiatives, Center for Health Incentives and Behavioral Economics, University of Pennsylvania, Philadelphia, PA, USA

Panelists:

Henry A. Glick, PhD, Professor of Medicine, Perelman School of Medicine; Professor of Health Care Systems, Wharton School, University of Pennsylvania, Philadelphia, NY, USA

Ellen Sonet, JD, MBA, Chief Strategy and Alliance Officer, Strategy, CancerCare, New York, NY, USA

Charles E. Phelps, PhD, MBA, University Professor & Provost Emeritus, Office of the Provost, University of Rochester, Rochester, NY, USA

ISSUE:

The past decade has seen unprecedented pharmacological breakthroughs in oncology. Nevertheless, the high costs of these medications have placed considerable financial pressures on both the U.S. healthcare system and patients. Recognition that costs and value should be important considerations in cancer treatment discussions has led several U.S. clinical organizations including the American Society of Clinical Oncology and the National Comprehensive Cancer Network to develop value frameworks. Such efforts to inform shared patient/physician decision making are laudable but have attracted a variety of criticisms, including being insufficiently patient-centered.

OVERVIEW:

This panel will debate the need for patient-centered value frameworks to support shared patient/physician decision-making surrounding cancer treatments and discuss the challenges and potential solutions for creating such tools. Jalpa Doshi will moderate the panel, providing an overview of the current landscape and posing key questions for the panelists to debate: What do current frameworks capture and why do we need a more patient-centered framework? How do definitions of value vary both within and between key stakeholder groups? What is important to patients, and how can we develop and implement a decision tool that truly meets the goals and priorities of individuals in daily clinical practice? Can we agree on a scoring approach to help inform decision-making based on the various factors? Henry Glick will discuss challenges in designing a patient-centered value framework, including addressing tensions between the societal and patient perspective. Ellen Miller Sonet will represent patient perspectives, offering insights from patient-centered research conducted by CancerCare. She will highlight factors important to cancer patients and approaches to collecting such information in clinical practice to support a customized patient-centered decision tool. Charles Phelps will argue that methods such as multi-criteria decision analysis can be used to develop a scoring approach to inform shared decision-making using a customized tool.

Economic Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Ballroom I (Level 400)

IP2: WHAT ROLE FOR VALUE ASSESSMENT IN US PRIVATE PAYERS’ SPECIALTY DRUG COVERAGE DECISIONS?

Moderator:

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

Susan A. Cantrell, CAE, Chief Executive Officer, Academy of Managed Care Pharmacy, Alexandra, VA, USA

Sara Traigle van Geertruyden, JD, Executive Director, Partnership to Improve Patient Care, Washington, DC, USA

Randy Burkholder, BA, Vice President of Policy and Research, PhRMA, Washington, DC, USA

ISSUE:

There is increasing use of value assessment (e.g., reports issued by Institute for Clinical and Economic Review) throughout US health care. This issue panel will debate the appropriate role of these assessments in informing payer specialty drug in the US private sector. James Chambers will moderate and frame the issue by summarizing new research examining commercial payers’ reported use of value assessments in their specialty drug coverage policies. Susan Cantrell will address payer evidence requirements and how value assessments inform decision-making. Sara Traigle van Geertruyden will provide a patient’s perspective, and will discuss the need for clarity as to how payers use value assessments to guide patients’ access to medical technology. Randy Burkholder will describe how uncertainty in payers’ use of value assessment can lead to challenges in bringing innovations to the market.

OVERVIEW:

In recent years value assessments have increasingly informed debate among patients, providers, payers and policy makers about the value of medical technology. However, how these assessments inform payer drug coverage policy is unclear. This panel will discuss research (based on a database of more than 4,000 drug medical policies) that has found variation in payers’ reported use of value assessments in their specialty drug coverage decision-making. The panel will debate whether the increased use of value assessments in the US reflects their greater influence, and whether their influence varies across payers. This issue panel will provide a forum to debate and discuss how value assessments can be used to complement other forms of evidence, and input from patients, to optimize payer decision-making. Panelists’ presentations will be limited to three PowerPoint slides, after which the moderator will encourage debate through Q&A and audience participation.

3:45 PM - 4:45 PM
BREAKOUT SESSION
Use of Real World Data Issues

3:45 PM - 4:45 PM
Room: Ballroom II (Level 400)

IP3: THE MACHINE LEARNING DEBATE: PANACEA OR THE NEW ALCHEMY?

Moderator:

Sreeram Ramagopalan, Ph.D, Director, Center for Observational Research and Data Sciences (CORDS), Bristol-Myers Squibb Pharmaceuticals Ltd, Uxbridge, UK

Panelists:

Andrew Briggs, DPhil, William R. Lindsay Chair of Health Economics, Health Economics & Health Technology Assessment, University of Glasgow, Glasgow, UK

Gorana Capkun-Niggli, PhD, Head of HEOR Innovation, Novartis Pharma AG, Basel, Switzerland

Radek Wasiak, Ph.D, Vice President, General Manager, Real-World Evidence, Evidera, London, UK

ISSUE:

The use of machine learning (ML) in biomedicine is increasing at a rapid rate. However, there is a substantial and well-documented division of opinion on its value; critics argue that the methodology is not transparent and too often leads to mistaken inference, calling it the new alchemy. Supporters argue that ML is a potential game-changer and enables powerful insights when traditional approaches fall short.

OVERVIEW:

This debate has not yet fully addressed the area of market access and health economics and outcomes research. Whilst common in other industries and widely used in the drug discovery process, ML application to patient medical record data presents several specific challenges, particularly when it is used as a diagnostic tool. In 2018, the new European legislation on data protection (General Data Protection Regulations) will potentially prohibit some current uses of ML (e.g., automated individual decision making and profiling). Considering the current real-world data explosion, the benefit of ML is potentially substantial; however, we also need to acknowledge the potential for incorrect application and thus the subsequent likelihood of mistaken insights.

The purpose of this panel will be to represent the spectrum of opinions on ML in our industry and will aim to explore opportunities and challenges with the participation of the audience. Examples of poor and successful applications of ML in the public domain will be used to make the debate accessible to a non-technical audience. Specifically, we plan to address

  • hypothesis generation (e.g., informing drug development) vs decision making (e.g., regulatory, reimbursement, treatment choice by doctors)
  • validation processes (how does ML compare to traditional statistical methods?)
  • interpretation of ML findings (how often are they spurious and can we trust results even if we cannot explain them?)

Patient-Reported Outcomes & Patient Preference Research Issues

3:45 PM - 4:45 PM
Room: Hall A (Level 100)

IP4: INCORPORATING QUANTITATIVE PATIENT PREFERENCE INFORMATION INTO HEALTHCARE DECISION-MAKING PROCESSES: IS HEALTH TECHNOLOGY ASSESSMENT FALLING BEHIND?

Moderator:

Paula Lorgelly, BSc(Hons), PhD, PGCAP, Deputy Director, Office of Health Economics, London, UK

Panelists:

David J. Mott, MSc, Economist, Office of Health Economics, London, UK

A. Brett Hauber, PhD, Senior Economist & Vice President, Health Preference Assessment, RTI Health Solutions, Research Triangle Park, NC, USA

Deborah A. Marshall, PhD, Canada Research Chair, Health Services and Systems Research, Professor, Department of Community Health Sciences, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada

ISSUE:

The use of quantitative patient preference information (PPI) in regulatory decision-making is increasing. In contrast, its application in health technology assessment (HTA) is less frequent. The aim of this issue panel is to debate whether there are opportunities for increased use of quantitative PPI in HTA. The panelists will draw upon their experiences in the United Kingdom, the United States and Canada. David Mott will provide an overview of the use of quantitative PPI to inform health care decision-making and put forward a number of reasons as to why HTA appears to be falling behind. Brett Hauber will discuss the recent progress in the regulatory space, explaining how PPI in regulatory decisions has become better defined and how this may differ to the type of information that is required for HTA. Deborah Marshall will consider the challenge for HTA assessment processes in incorporating and weighing PPI with clinical, cost-effectiveness and budget impact information. In the remainder of the session, the audience will be encouraged to put forward their thoughts in an open debate on the subject.

OVERVIEW:

Quantitative PPI can be generated using a range of stated preference methodologies including, but not limited to: time trade-off; contingent valuation; discrete choice experiments; and best-worst scaling. The data can be used within, or considered alongside, analyses that aim to inform health care decision-making, such as benefit-risk assessments and HTAs. In the United States, the Food and Drug Administration (FDA) has taken major steps forward to encourage the incorporation of the patient perspective into regulatory decision-making. With related research projects currently ongoing, regulatory agencies in Europe may follow the FDA’s lead in the near future. However, despite the potential and the progress being made in the regulatory space, there is currently little to suggest that HTA agencies will encourage greater utilisation of quantitative PPI.

5:00 PM - 6:00 PM
BREAKOUT SESSION
Economic Outcomes Research Issues

5:00 PM - 6:00 PM
Room: Ballroom II (Level 400)

IP5: SHOULD WE MOVE BEYOND CONVENTIONAL COST-EFFECTIVENESS ANALYSIS? IF SO, HOW?

Moderator:

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

Panelists:

Peter J. Neumann, ScD, Professor & Director, 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

Charles E. Phelps, PhD, MBA, University Professor & Provost Emeritus, Office of the Provost, University of Rochester, Rochester, NY, USA

Jeroen P Jansen, PhD, Lead Scientific Advisor - Open Source Value Project, Innovation and Value Initiative; Precision Health Economics, Los Angeles, CA, USA

ISSUE:

Cost-effectiveness analysis (CEA) is often considered the “gold standard” for the assessment of value for health care decision-making, but even its proponents acknowledge its limitations. As decision-makers increasingly seek to understand value, is it time to move beyond conventional CEA, and, if so, how? Darius Lakdawalla will moderate and provide an overview of recent debates about recommended methods for assessing relative value and making resource decisions in health care. Peter Neumann will shed light on the evolving field of CEA and its current challenges. Charles E. Phelps will share expertise on multi-criteria decision analysis (MCDA), which has been proposed as a way of evolving health care decision analysis and addressing some of CEA’s shortcomings. Jeroen Jansen will provide perspective from recent efforts to incorporate both CEA and MCDA into value-focused open-source decision tools designed, among other reasons, to improve the ability of CEA to respond rapidly to the emergence of new evidence.

OVERVIEW:

As health care systems increasingly move toward value-oriented decision-making, decision-analytic approaches must provide more flexibility than they have previously. Calls for inclusion of broader components of value – such as health equity, for example – challenge researchers to think beyond the traditional structure of CEA, which compares health interventions in terms of incremental costs relative to incremental gains (or losses) in quality-adjusted life years (QALYs). Meanwhile, some observers argue that the QALY does not adequately capture treatment attributes important to decision-makers, especially patients. Expansions of CEA and alternative approaches, such as multi-criteria decision analysis, have been proposed, but application of these approaches to real-world value comparisons – where diverse stakeholder perspectives may call for multiple approaches – presents considerable challenges. The panel will discuss alternative approaches to improving the flexibility and responsiveness of healthcare technology assessment, with particular focus on the role of MCDA and of flexible, open-source decision models.

Use of Real World Data Issues

5:00 PM - 6:00 PM
Room: Ballroom I (Level 400)

IP6: INCREASING MEDICAL DEVICE EVIDENCE FOR PAYERS, PROVIDERS, AND HTA BODIES: WHO’S RESPONSIBLE FOR GENERATING THE EVIDENCE?

Moderator:

Karen Worley, PhD, Research Manager, Comprehensive Health Insights, Humana, Cincinnati, OH, USA

Panelists:

Bruce Gingles, BA, Vice President, Global Technology Assessment and Healthcare Policy, Cook Medical, Bloomington, IN, USA

Suzanne Belinson, PhD, Executive Director, Blue Cross and Blue Shield Association, Chicago, IL, USA

Harindra C Wijeysundera, MD, PhD, Vice President, Medical Devices and Clinical Interventions, CADTH, Ottawa, ON, Canada

ISSUE:

Clinical evidence available at market authorization for medical devices is not meeting the needs of hospital payers and providers. As a result, HTA bodies and clinical groups are now looking to incorporate other forms of evidence, including real world data, into their assessment processes. Questions are arising as to what evidence should be include, who should produce and fund the research, and who has ownership of the evidence. Karen Worley will moderate, providing a brief overview of the issue. Bruce Gingles will address the current evidence production system through the manufacturer lens. Suzanne Belinson will address payer needs when evaluating coverage options. Harindra Wijeysundera will address the state of currently available evidence for devices and how observed gaps impede evaluation.

OVERVIEW:

Medical devices are regulated and reviewed in a manner different from pharmaceuticals. In the US, devices typically progress through an FDA clearance, providing data demonstrating that the new device is “substantially equivalent” to a device on the market, rather than an approval process, resulting in a paucity of clinical data in all phases of a technology’s life cycle. Where research does exist, published studies are highly variable in design and outcome evaluation. Standardization of clinical and economic research for medical devices could potentially be very beneficial for payers, providers, and HTA bodies, but there is no consensus as to who should oversee such an initiative. This panel will discuss the importance of standardization of clinical evidence for devices and debate the question of who should be sponsoring and directing such a standardization process.

Organized by the ISPOR Health Technology Assessment – Pharmacy and Therapeutics (HTA-P&T) Roundtable – North America


5:00 PM - 6:00 PM
Room: Hall A (Level 100)

IP7: THE CHANGING LANDSCAPE OF HEALTHCARE DECISION MAKING: HOW CAN WE LEVERAGE THE USE OF REAL-WORLD EVIDENCE FOR REGULATORY, COVERAGE, AND CLINICAL DECISION MAKING?

Moderator:

Rachael Fleurence, PhD, Executive Director, National Evaluation System for Health Technology Coordinating Center (NESTcc), Medical Device Innovation Consortium (MDIC), Arlington, VA, USA

Panelists:

Owen Faris, PhD, Clinical Trials Director, Office of Device Evaluation, Center for Devices and Radiological Health, U.S. Food and Drug Administration, Silver Spring, MD, USA

Kathleen Blake, MD, MPH, Vice President, Healthcare Quality, American Medical Association (AMA), Washington, DC, USA

Naomi Aronson, PhD, Executive Director of Clinical Evaluation, Innovation, and Policy, Blue Cross and Blue Shield Association, Chicago, IL, USA

ISSUE:

The rise of digital and health technologies is changing the landscape of decision-making in the health care system. Stakeholders are now faced with a dichotomy by which more information is readily available, but the systems in place for harnessing this information are decentralized and inefficient. To combat this, NESTcc is working to be the leading, go-to, organization within the medical device ecosystem for conducting efficient, timely, and high-quality Real-World Evidence (RWE) studies throughout the Total Product Life Cycle (TPLC) for the purpose of enhancing decision-making to improve patient health and outcomes.

Rachael Fleurence will lead the discussion, providing an overview of NESTcc and its goal to establish functional and efficient pathways for key stakeholders to generate lower-cost, nearer real-time evidence of sufficient quality for regulatory, coverage, patient, and clinical decision-making. Kathleen Blake will address the need for increased clinician access to information in order to enhance quality of patient care. Naomi Aronson will provide a unique perspective on the importance of Real-World Evidence to coverage decision-making while Owen Faris will speak to the impacts of Real-World Evidence on premarket clearance and regulatory decision-making.

OVERVIEW:

The changing landscape of decision-making in health care is catalyzing the need for high-quality Real-World Evidence. For patients, this means more transparent access to information regarding appropriate device use and potential health outcomes. Clinicians benefit from having access to nearer real-time and high-quality information about the quality of care they provide. From a regulatory standpoint, Real-World Evidence can enhance support for premarket clearances and enhancing device safety monitoring. This panel will explore how to ensure Real-World Evidence generation is useful for regulatory, coverage, and clinical decision-making.

Tuesday, May 22, 2018
11:00 AM - 12:00 PM
BREAKOUT SESSION
Clinical Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Ballroom IV (Level 400)

IP8: HOW IMPORTANT ARE CORE OUTCOME SETS TO MEASURING VALUE OF INNOVATIVE NEW DRUGS?

Moderator:

Elise Berliner, PhD, Director, Technology Assessment Program, Center for Evidence and Practice Improvement, Agency for Healthcare Research and Quality, Rockville, MD, USA

Panelists:

Chuck Shih, PhD, MHS, Associate Director, Healthcare Reform & Public Policy, Biogen, Washington, DC, USA

Elizabeth Seeley, MS, PhD, Adjunct Lecturer on Health Policy and Management, Department of Health Policy and Management, TH Chan School of Public Health, Harvard University, Boston, MA, USA

Sean R Tunis, MD, MSc, President and Chief Executive Officer, Center for Medical Technology Policy, Baltimore, MD, USA

ISSUE:

Drug costs have been a concern among policymakers due in part to the recent introduction of innovative new treatments, including gene therapies. Understanding the effectiveness of drugs is key to value based purchasing, but the lack of agreement on the most critical outcomes and the data infrastructure to measure these outcomes hampers progress. Chuck Shih will address how industry chooses which outcomes to measure in clinical studies. Elizabeth Seeley will discuss the payer perspective on outcome-based contracting and the limited availability of meaningful metrics to measure effectiveness. Sean Tunis will review his work with international efforts such as COMET (Core Outcome Measures for Effectiveness Trials) and ICHOM (International Consortium of Health Outcome Measurement) to drive consensus among stakeholders on adoption of core outcome sets across the healthcare system. The panelists together with the participants will debate how core outcome sets could be implemented, including issues of funding and governance.

OVERVIEW:

Systematic reviews and technology assessments often find that clinical trials use a range of different outcomes measures, making it difficult to compare the effectiveness and value of different treatments. With this uncertainty, payers have started to look at outcomes based contracting for high cost drugs, where payment is tied to real world outcomes of treatment. However, the lack of consensus on outcomes measures sometimes leads to unclear impact of these models in the real world setting as well. Consensus on core outcome sets that could be used for research studies and for implementation of a real world data infrastructure would address these problems but there are questions about funding and aligning incentives for adoption.

Organized by the ISPOR Health Technology Assessment – Pharmacy and Therapeutics (HTA-P&T) Roundtable – North America

Economic Outcomes Research Issues

11:00 AM - 12:00 PM
Room: Ballroom III (Level 400)

IP9: LIES, DAMNED LIES AND COST-EFFECTIVENESS: OPEN-SOURCE MODELS ARE ESSENTIAL IF COST-EFFECTIVENESS ANALYSES ARE TO BE WIDELY ACCEPTED

Moderator:

Neil Hawkins, PhD, Professor of Health Technology Assessment (HTA), Health Economics & Health Technology Assessment, University of Glasgow, Glasgow, UK

Panelists:

Renée J G Arnold, PharmD, Director, Life Sciences, Navigant Consulting, Inc., San Francisco, CA, USA

J. Jaime Caro, MDCM, Adjunct Professor, Medicine, Epidemiology, and Biostatistics, McGill University, Montreal, QC, Canada

ISSUE:

Cost-effectiveness models typically synthesize a wide range of evidence to enable extrapolation over time and from intermediate to final decision endpoints1. These models are often complex, statistically sophisticated, and require assumptions that are not always directly testable. This can lead to decision-makers questioning the validity of models and “discounting” their results, particularly if the developer is seen as partial.

OVERVIEW:

The development of open-source cost-effectiveness models has the potential to improve the quality and impact of cost-effectiveness models by both allowing investigators to access a range of candidate models and by facilitating the internal and external validation of these models2,3. However, there are concerns with the potential impact on intellectual property right, trust issues and the effort involved in developing and maintaining them4.

Against this background, Jaime Caro will argue that the development of open-source models is essential for cost-effectiveness analysis to to be viewed as a reliable input to decision-making and Renée Arnold will argue that the impact on intellectual property rights, model “ownership”and oversite and, consequently, the incentives and resources available to develop and maintain models4 , are prohibitive.

Finally, we will consider whether the submission of working models should be mandated by journals and HTA agencies. There will be time for the audience to cross-examine the panel and a vote will be taken.

  1. Bertagnolli MM, Sartor O, Chabner BA, et al. Advantages of a Truly Open-Access Data-Sharing Model. New England Journal of Medicine. 2017;376(12):1178-1181
  2. Dunlop WCN, Mason N, Kenworthy J, Akehurst RL, Mason N. Benefits, Challenges and Potential Strategies of Open Source Health Economic Models. Pharmacoeconomics. 2017;35(1):125–8.
  3. Arnold RJ, Ekins S. Time for cooperation in health economics among the modelling community. Aug 1 2010;28(8):609-613.
  4. Eddy DM, Hollingworth W, Caro JJ, Tsevat J, McDonald KM, Wong JB. Model transparency and validation: a report of the ISPOR-SMDM Modelling Good Research Practices Task Force--7. Value Health. 2012;15(6):843–50.

2:15 PM - 3:15 PM
BREAKOUT SESSION
Health Policy Development Using Outcomes Research Issues

2:15 PM - 3:15 PM
Room: Ballroom III (Level 400)

IP10: PRIOR AUTHORIZATION POLICIES FOR MANAGING SPECIALTY DRUG SPENDING IN THE UNITED STATES: CAN WE STRIKE A BALANCE BETWEEN APPROPRIATE UTILIZATION AND APPROPRIATE ACCESS?

Moderator:

Jalpa A. Doshi, PhD, Professor of Medicine, Perelman School of Medicine; Director, Economic Evaluations Unit, Center for Evidence-based Practice; Director, Value-based Insurance Design Initiatives, Center for Health Incentives and Behavioral Economics, University of Pennsylvania, Philadelphia, PA, USA

Panelists:

Steven Miller, MD, MBA, Senior Vice President and Chief Medical Officer, Express Scripts, St. Louis, MO, USA

Cat Davis Ahmed, MBA, Vice President, Policy and Outreach, The FH Foundation, Pasadena, CA, USA

Seth J. Baum, MD, President, Affiliate Professor of Medicine, Schmidt College of Medicine, Florida Atlantic University (FAU), American Society for Preventive Cardiology; Chief Medical Officer, Excel Medical Clinical Trials, Boca Raton, FL, USA

ISSUE:

Prior authorization (PA) is an increasingly common gatekeeping strategy used by U.S. payers to manage specialty drug utilization. At the same time, burdensome PA requirements may create barriers to access for patients in need. For example, recent analyses of a new class of cholesterol-lowering medications called proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9is) report that PA requirements for PCSK9is were substantially more extensive than those for other cardiometabolic drugs and more than half of PCSK9i prescriptions were rejected even among clinically appropriate patients. As more and more expensive specialty drugs become available to treat chronic conditions, the challenge of ensuring appropriate access within real world budget constraints has come into sharp focus. There is an urgent need to explore this issue from the perspectives of all relevant stakeholders.

OVERVIEW:

This panel will debate the pros and cons of PA requirements and explore approaches to handling the competing priorities of balancing costs and access, using PCSK9is as a case study. Jalpa Doshi will moderate the panel and provide an overview of the current landscape of PA requirements as well as pose key questions for the panelists to debate: What are best practices for ensuring appropriate use among patients? What types of PA requirements are blunt tools for cost containment versus necessary for ensuring prescribing in patients for whom the drugs offer high value? Should high budget impact drugs be handled differently? Steven Miller will discuss PA efforts to balance costs and access from a PBM/payer perspective. Cat Davis Ahmed will represent the patient perspective, offering insights on patient experiences and the human consequences of restrictive PA policies. Seth Baum will represent clinical societies calling for PA reform and offer a provider perspective on the burden of administrative requirements and their impact on patient care as well as a physician’s practice.

Economic Outcomes Research Issues

2:15 PM - 3:15 PM
Room: Ballroom I (Level 400)

IP11: “MAKING MEDICINES AFFORDABLE: A NATIONAL IMPERATIVE” – REFLECTIONS ON THE NATIONAL ACADEMIES REPORT (INVITED ISSUE PANEL)

Moderator:

Guru Madhavan, PhD, MBA, Senior Program Officer, Health and Medicine, National Academies of Science, Engineering, and Medicine, Washington, DC, USA

Panelists:

Norman Augustine, MS, Retired, Lockheed Martin Corporation, Bethesda, MD, USA

Charles E. Phelps, PhD, MBA, University Professor & Provost Emeritus, Office of the Provost, University of Rochester, Rochester, NY, USA

Stacie B. Dusetzina, PhD, Associate Professor, Health Policy, Ingram Associate Professor of Cancer Research, Vanderbilt University Medical Center, Nashville, TN, USA

Robert W. Dubois, MD, PhD, Chief Science Officer & Executive Vice President, National Pharmaceutical Council, Washington, DC, USA

ISSUE:

To approach the proper balance between affordabil­ity and future availability of medicines in the inter­est of public health, Making Medicines Affordable offers a set of eight specific recommendations, with interlinked imple­mentation actions in the biopharmaceutical sectors, regarding: federal government negotiation of prices, access to off-patent drugs, consumer information, insurance benefit designs, the Orphan Drug Act, and reimbursement incentives. What are the key rationales behind these recommendations? Can we be confident – and how could we evaluate – whether implementing them would achieve a desirable balance between affordability and innovation?

OVERVIEW:

Over the past several decades, the U.S. biopharmaceutical sector has been very successful in developing and delivering effective drugs for improving health and fighting disease. Many medical conditions that were long deemed untreatable can now be cured or managed effectively. Yet this success has come at a cost: Spending on prescription drugs has been rising dramatically, to the point that many people have difficulty paying for the drugs that they or their family members need. Drug costs are a significant part of the nation’s total spending on health care. With support from a host of sponsors, the National Academies of Sciences, Engineering, and Medicine conducted a study to recommend policy actions that address drug price trends, improve patient access to afford­able and effective treatments, and encourage innovations that address significant needs in health care. The resulting report, Making Medicines Affordable: A National Imperative, provides several strategies to tackle the rising costs of prescription drugs without discouraging the development of new and more effective drugs for the future.

3:45 PM - 4:45 PM
BREAKOUT SESSION
Health Policy Development Using Outcomes Research Issues

3:45 PM - 4:45 PM
Room: Ballroom I (Level 400)

IP12: WHAT RESEARCH SHOULD BE UNDERTAKEN TO SUPPORT DECISIONS REGARDING THE VALUE OF CURING OR ERADICATING DISEASE?

Moderator:

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

Panelists:

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

Steven D. Pearson, MD, MSc, Founder and President, Institute for Clinical and Economic Review, Boston, MA, USA

Carleigh B Krubiner, PhD, Research Scholar and Project Director, Berman Institute of Bioethics, Johns Hopkins University, Baltimore, MD, USA

ISSUE:

Many health care interventions are focused on reducing the impact of a disease on individuals’ health. Methods of health technology assessment and economic evaluation are widely used support health systems’ decisions regarding the funding of such interventions. There is now a prospect of medical technologies curing disease, for example gene therapy for diseases such as haemophilia, and innovative approaches that may remove HIV in treated patients. Eradicating further tropical infectious diseases, such as visceral leishmaniasis, is also a prospect. Given the likely high cost of such interventions, appropriate research methods to support funding decisions will be important. But are current methods fit for purpose when major step-changes in health improvement are feasible? Furthermore, how should improvements in other aspects of wellbeing associated with cure be assessed when evaluating the value for money of these interventions against lower cost disease management options?

OVERVIEW:

There are several ways in which standard economic evaluation may fall short in evaluating interventions with the prospect of curing or eradicating disease. These include the assumption of a linear relationship between gains in health and its value, a failure to recognize the importance of the burden of disease in assessing value and, in the case of disease eradication, not considering the value to future generations. Members of the panel will take different perspectives on appropriate methods in this context. Watkins will moderate and set the scene in terms of the characteristics of research to support payers’ decisions. Sculpher will argue that, when appropriately implemented, existing methods are entirely suitable for these interventions. Krubiner will present a range of ethical considerations that highlight the limitations of existing methods. Pearson will suggest an approach whereby the pricing of cures can be adjusted in a systematic fashion to allow innovators and society to share fairly in potential large downstream cost offsets. Organized by the ISPOR Health Technology Assessment (HTA) Council

Clinical Outcomes Research Issues

3:45 PM - 4:45 PM
Room: Hall A (Level 100)

IP13: SURROGATE OUTCOMES IN ONCOLOGY: HOW CAN THEY BE USED TO PREDICT OVERALL SURVIVAL IN CLINICAL PRACTICE AND PAYER DECISION MAKING?

Moderator:

Jipan Xie, MD, PhD, Vice President, Analysis Group, Inc., New York, NY, USA

Panelists:

Yiduo Zhang, PhD, Director, Health Economics and Payer Analytics, Global Payer Evidence and Pricing, AstraZeneca, Gaithersburg, MD, USA

Russell Hales, MD, Director, Department of Radiation Oncology and Molecular Radiation Sciences, The Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD, USA

Louis P. Garrison, PhD, Professor, Pharmaceutical Outcomes Research & Policy Program, School of Pharmacy University of Washington, Seattle, WA, USA

ISSUE:

Overall survival (OS) is a final patient-centric outcome valued by clinicians and health technology assessment (HTA) agencies/payers. However, immature OS is a common issue for new oncology drugs, and using surrogate outcomes to support decision-making is a practical challenge. Dr. Xie will provide an overview of the issue, summarize recent research (using lung cancer and immuno-oncology as examples), and moderate the session. Dr. Zhang will present the pharmaceutical company perspective, looking at how such research is harnessed to demonstrate the value of innovative drugs, and evaluate the pros and cons of different methodologies, focusing on the challenges in early health benefit assessment for the pharmaceutical industry. Dr. Hales will provide a clinical view of surrogate outcomes and patient-centric outcomes and their relevance in clinical practice. Dr. Garrison will present different HTA/payers’ perspectives and discuss methods and evidence used by HTA/payers to assess the clinical benefits of oncology drugs in the context of economic evaluations. Panelists will debate the standard procedures to generate relevant evidence based on surrogate outcomes in oncology from three distinct perspectives and will invite the audience to share their views.

OVERVIEW:

Surrogate outcomes, such as response rate and progression-free survival, have been increasingly used by regulatory agencies to approve new oncology drugs. While much research has been devoted to associating surrogate outcomes with final patient-centric outcomes, such as OS, there has not been a standard method in oncology to establish the associations. In addition, there is little guidance on what evidence should be generated based on surrogate outcomes and OS for decision-making by clinicians and HTA/payers, partially due to inconsistent findings across treatments and disease areas. This panel will debate potential standard methodologies used to associate surrogate outcomes with OS and call for more guidance on evidence that can be accepted by clinicians and HTA/payers.

Economic Outcomes Research Issues

3:45 PM - 4:45 PM
Room: 310 (Level 300)

IP14: DOES MEASURING THE ECONOMIC VALUE OF CLINICAL GENOMIC SEQUENCING REQUIRE NEW METHODOLOGICAL APPROACHES?

Moderator:

Kathryn A. Phillips, PhD, Professor, Center for Translational and Policy Research on Personalized Medicine (TRANSPERS), University of California – San Francisco, San Francisco, CA, USA

Panelists:

Deborah A. Marshall, PhD, Canada Research Chair, Health Services and Systems Research, Professor, Department of Community Health Sciences, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada

Dean A. Regier, PhD, Scientist, Canadian Centre for Applied Research in Cancer Control (ARCC), British Columbia Cancer Agency, Vancouver, BC, Canada

Sarah Wordsworth, PhD, Associate Professor, Nuffield Department of Population Health, University of Oxford, Oxford, UK

ISSUE:

We address whether measuring the economic value of clinical genomic sequencing (NGS) requires new methodological approaches. Panelists are members of an international working group preparing a theme section for VIH publication. Panelists will debate the following as “true” or “false”:

  • Standard decision analytic models can be used to model the costs and outcomes for tests that provide multiple clinical results, including secondary findings;
  • QALYs are sufficient to capture the full value of NGS tests;
  • Economic evaluations should incorporate the value of identifying genetic variants that currently do not provide clinical utility but may do so in the future;
  • Economic evaluation guidelines (e.g. CADTH, CEA in Health/Med 2nd ed) apply equally as well to NGS as new pharmaceuticals;
  • “Big Data” offers an ideal way to obtain data for measuring the costs and benefits of NGS.
We will elicit audience “voting” on these statements before/after the debate, and conclude with a discussion of how to move ahead with approaches to address the challenges identified.

OVERVIEW:

Genomic tests analyzing multiple genes using NGS, which can improve diagnostic and prognostic information, are entering clinical care. However, it is unclear whether these tests provide economic value for money, and few economic evaluations of NGS have been conducted. One reason for this lack of data is that health economists are uncertain about whether existing approaches to economic evaluation are sufficient to evaluate NGS technologies.

5:00 PM - 6:00 PM
BREAKOUT SESSION
Patient-Reported Outcomes & Patient Preference Research Issues

5:00 PM - 6:00 PM
Room: Hall A (Level 100)

IP15: HOW CAN WE ADDRESS THE CURRENT CHALLENGES IN VALUE ASSESSMENT?

Moderator:

Sachin Kamal-Bahl, PhD, VP & Head, Center for Health Systems Innovation & Leadership, Pfizer, Inc., New York, NY, USA

Panelists:

Josh J. Carlson, MPH, PhD, Associate Professor, The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington, Seattle, WA, USA

Shelby D Reed, PhD, RPh, Professor in Medicine, Preference Evaluation Research Group, Duke Clinical Research Institute, Durham, NC, USA

Gillian D Sanders Schmidler, PhD, Deputy Director, Academics and Professor of Population Health Sciences, School of Medicine, The Robert J. Margolis, MD, Center for Health Policy, Duke University, Washington, DC, USA

ISSUE:

Concern over rising U.S. health care costs in recent years has increased interest in promoting high-quality care, while avoiding low value or inefficient care. In response, many initiatives aiming to drive value in health care have emerged, but few offer transformative solutions that reflect patient preferences and real-world clinical practice. The PhRMA Foundation launched its Value Assessment Initiative in 2017 to address issues in methodology and patient engagement. The panel will discuss and debate three potential approaches: Dr. Sanders Schmidler will discuss contexts or situations where perspective could lead to important differences affecting decision making. Dr. Reed will identify opportunities to broaden the discussion on value by quantifying the relative importance of hope in comparison to other aspects of value. Dr. Carlson will discuss methods that seek to address limitations in health-state utility estimation within current cost-effectiveness models.

OVERVIEW:

Value assessment in health care comprises a broad set of methods to synthesize and evaluate the relative benefits and costs of health care interventions. The goal of value assessment is to assist stakeholders, including patients, providers and payers, in making informed decisions to improve health and care efficiency. These assessments are conducted using varied methodological approaches to synthesize data from various sources to guide individual or population-level decisions. Stakeholders have suggested that patients’ perspectives on value are not adequately represented in many value frameworks. However, there are limitations with available data sources, methods and measures applied in value assessment. Existing methods for value assessment typically apply the same preference weights across patients even though there may be wide variations in how individual patients value alternative treatments. This issue panel will provide a constructive debate for approaches for integrating value into the United States health care system in a holistic, patient-centered manner.

Use of Real World Data Issues

5:00 PM - 6:00 PM
Room: Ballroom IV (Level 400)

IP16: IN THE ELECTRONIC HEALTH RECORD ERA, DO WE STILL NEED CLINICAL REGISTRIES?

Moderator:

Lisa M. Wruck, PhD, Director, Center for Predictive Medicine, Duke Clinical Research Institute, Durham, NC, USA

Panelists:

Sharon Hensley Alford, PhD, Associate Chief Health Officer, IBM Watson Health, Cambridge, MA, USA

Marc Berger, MD, Consultant, Self Employed, New York, NY, USA

Tracy Wang, MD, MHS, Associate Professor of Medicine, Duke Clinical Research Institute, Duke School of Medicine, Durham, NC, USA

ISSUE:

Researchers and research sponsors alike have an interest in how expanding EHR data can be used to generate registry-like patient insights quicker and at lower cost with greater generalizability. Can EHRs replace clinical registries? Sharon Hensley of IBM Explorys will address the potential to replicate registry data efficiently through EHRs. Tracy Wang of the Duke Clinical Research Institute will argue for the continuing value of registries to answer research questions. Marc Berger, who headed real world data and analytics at Pfizer, will share the perspective of pharmaceutical companies that commission clinical research on the cost-benefit of each approach. The panelists will debate before the discussion is opened to audience participation.

OVERVIEW:

Clinical registries, a well-established mechanism for collecting standardized data on a well-defined study population, are considered the gold standard for understanding natural history, quality of care, clinical effectiveness and safety. But registries require a dedicated research infrastructure, which can be expensive, time consuming and lack generalizability. Utilizing EHR data for research is appealing as sample sizes are large, data are readily available and populations are representative. However, limitations include selection bias toward sicker patients, issues with accuracy and completeness of data, privacy considerations, challenges identifying when patients are present in the health system, and misclassification errors due to dependence on coding.

Health Policy Development Using Outcomes Research Issues

5:00 PM - 6:00 PM
Room: Ballroom II (Level 400)

IP17: HAS THE TIME COME TO REPLACE RANDOMIZED CONTROLLED TRIALS WITH REAL-WORLD DATA -- A CASE OF MEDICAL DEVICES?

Moderator:

Rok Hren, PhD, MSc, Assistant Professor, University of Ljubljana, Ljubljana, Slovenia

Panelists:

Katarzyna Kolasa, PhD, Professor of Health Economics, Head of Department, Health Economics and Healthcare Management, Kozminski University, Warszawa, Poland

Rosanna Tarricone, Associate SDA Dean, Government, Health and Non Profit Division, Centre for Research on Health and Social Care Management (CeRGAS), SDA Bocconi School of Management, Milan, Italy

Cynthia Iglesias, PhD, Senior Research Fellow, Centre for Health Economics, University of York, York, UK

ISSUE:

How to robustly compare alternative interventions involving different medical devices (MDs) when randomized controlled trials (RCTs) are missing and only single arm observational studies are available? Why should RCTs matter at all if patients’ heterogeneity and clinical proficiency have such a formidable influence on the MD’s performance? Isn’t it better to assess the value of MDs with real world data (RWD) alone instead? The methodological concerns with the analysis of RCTs and RWD will be discussed from academic academic (RT) and HTA agency (CI) perspective. The challenges with the collection of RCTs and RWD will be addressed from manufacturer standpoint (KK). In addition to that, the panel will discuss whether there is a need for a new framework for value assessment of MDs. Panelists will present their experiences and perspectives related to the question whether RWD alone can ensure objectiveness and feasibility of the estimation of clinical and economic benefits of MDs.

OVERVIEW:

In 2015, there were more than twice as many patents granted for new MDs than drugs. As the market grows, there is an increased understanding of the need to assess the value of MDs. Following new MD directive (MDR 2017/745), some experts reinforce the importance of clinical data in the search of MD’s value. It is provoking to ask whether RCTs can truly provide the relevant evidence for the clinical and economic benefits. MD’s efficacy and safety is in fact determined not only by its technical capabilities but also patients’ characteristics as well as end user experience. The challenge to assess MD’s value is related to the difficulties with the choice of a comparator too. The complexity of clinical pathways introduces multiple alternative treatment options. Sometimes they are used simultaneously for the benefit of patient. Consequently, clinical efficacy can differ significantly from the real life effectiveness. The recent developments such as the introduction of FDA guidelines ”Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices” draw more and more attention to the usefulness of RWD in the decision making processes. It can therefore be asked whether RWD can substitute RCTs in the assessment of the value of MDs or we need to pursue both. This panel will attempt to address the pros and cons of the replacement of RCTs with RWD in the pricing & reimbursement process for MDs.

Wednesday, May 23, 2018
8:30 AM - 9:30 AM
BREAKOUT SESSION
Health Policy Development Using Outcomes Research Issues

8:30 AM - 9:30 AM
Room: Ballroom I (Level 400)

IP18: WHEN RESEARCH QUESTIONS COLLIDE: HOW MUCH IS THE UNITED STATES ACTUALLY SPENDING ON DRUGS?

Moderator:

Michael Kleinrock, MA, Research Director, IQVIA Institute for Human Data Science, Plymouth Meeting, PA, USA

Panelists:

Sean Keehan, MA, Economist, Office of the Actuary, Centers for Medicare and Medicaid Services, Baltimore, MD, USA

Cynthia Cox, MPH, Director, Program for the Study of Health Reform and Private Insurance, Kaiser Family Foundation, Washington, DC, USA

Sara Sadownik, MSc, Deputy Director, Research and Cost Trends, Massachusetts Health Policy Commission, Boston, MA, USA

ISSUE:

As stakeholders wrestle with policies related to U.S. health care spending, significant attention is paid to the portion of spending attributable to prescription drugs. There are many numbers bandied about, ranging from 10.1% to 27.7%. The vast range of these estimates, however, renders them useless for the ongoing health care spending discussion. If stakeholders are unable to agree on a number, how can they even begin to agree on a policy?

Sean Keehan will take the perspective of a government agency seeking to quantify health care spending for the entire U.S. population. The CMS estimate is 10.1%.

Cynthia Cox will take the perspective of a policy analysis group seeking to estimate health care spending for those with health insurance coverage through a large employer. The KFF estimate is 21.0%.

Sara Sadownik will take the perspective of an individual U.S. state seeking to understand their state’s health care spending trends. The MHPC estimate is 17.0%.

Michael Kleinrock will share the health plan perspective, using AHIP’s estimates of 22.0% and 27.7%, and, as moderator, will facilitate the exploration of these differing perspectives and their resultant discordant estimates of the share of the health care dollar attributable to drugs, and provide a bridge to reconcile the differences where possible.

OVERVIEW:

Many policy discussions on health care spending are focusing on controlling the share of health care spending allocated to prescription drugs. However, estimates of this share stem from very different research questions and can vary considerably from each other. Informed policy discussions require a mutual understanding of the data and an estimate that is relevant to both the research and policy questions at hand. This panel will debate how the share of health care spending attributable to drugs should be measured and whether there is a “right” number for policy discussions.


8:30 AM - 9:30 AM
Room: Ballroom II (Level 400)

IP19: VALUE ASSESSMENT FRAMEWORKS IN AN ERA OF PERSONALIZED MEDICINE: SHARED OBJECTIVES OR IRRECONCILABLE DIFFERENCES?

Moderator:

Kristen Migliaccio-Walle, BS, Director, Global Health Economics & Outcomes Research, Xcenda, LLC, Palm Harbor, FL, USA

Panelists:

Donna Cryer, JD, President and CEO, Global Liver Institute, Washington, DC, USA

Daniel A Ollendorf, PhD, Chief Scientific Officer, Institute for Clinical and Economic Review, Boston, MA, USA

Robert W Dubois, MD, PhD, Chief Science Officer, National Pharmaceutical Council, Washington, DC, USA

ISSUE:

In purpose, personalized medicine and value assessment frameworks (VAFs) are clearly aligned in seeking to increase the efficiency of health care, but that can only happen if frameworks incorporate the key elements of personalized medicine that demonstrate its value. How these frameworks consider personalized medicine is not yet clear. Introduced before 25% of new drugs were personalized medicines, VAFs may not fully account for the roles of diagnostic testing and patient-centered practice-based evidence in targeting therapeutic use. Kristen Migliaccio-Walle will moderate and provide an overview of the current environment. Donna Cryer will address the importance of personalized medicine from the patient perspective based on her own experience as a patient and patient advocate. Steve Pearson will address the VAF developer perspective describing the ICER framework methodology and the solutions and challenges of incorporating key elements of personalized medicine. Robert Dubois will address the manufacturer perspective noting the increasing focus on targeted therapies in oncology and the evolving definition of value.

OVERVIEW:

By identifying which medical treatments and procedures will work best for each patient, personalized medicine improves efficiency at both the individual and health system levels. In an environment where the US has paid increasing attention to health care costs, VAFs aim to systematically quantify which treatments provide the most benefit for patients and the health system at a reasonable cost. Indeed, the two share many objectives related to improving health outcomes. Nevertheless, the current methodologies of VAFs may not sufficiently capture the value of personalized medicine, focusing instead on population health, thereby overlooking efficiencies in patient-level health care and potentially under-valuing innovative therapies. This panel aims to expose and debate the challenges of valuing personalized medicines in the current VAF environment and identify areas of consensus on which newer methodologies can be built.

1:45 PM - 2:45 PM
BREAKOUT SESSION
Patient-Reported Outcomes & Patient Preference Research Issues

1:45 PM - 2:45 PM
Room: Ballroom I (Level 400)

IP20: THE PATIENT PERSPECTIVE AND VALUE ASSESSMENT: EASY TO IDENTIFY THE NEED, HARD TO AGREE ON THE SOLUTION

Moderator:

Mike Ciarametaro, MBA, Vice President, National Pharmaceutical Council, Washington, DC, USA

Panelists:

Ilene L. Hollin, PhD, MPH, Health Policy Fellow, National Pharmaceutical Council and University of Southern California Schaeffer Center for Health Policy and Economics, Washington, DC, USA

David Wamble, MS, MBA, Director, Health Economics, RTI Health Solutions, Durham, NC, USA

Josh Seidman, PhD, MHS, Senior Vice President, Avalere, Washington, DC, USA

ISSUE:

While most would agree that the patient perspective is an important aspect of value assessment, consensus is lacking on how to achieve this. Patient heterogeneity, individual preferences, and incorporation of indirect benefits have all been identified as potential solutions to the problem, but we fail to agree on the best approach going forward. Which solution is best? Are all required? Furthermore, we often stop short of understanding how to incorporate any of these solutions into actual value assessment tools. Michael Ciarametaro will moderate and provide an overview of the challenges associated with incorporating the patient perspective in value assessment. Ilene Hollin (patient preference researcher perspective) will address patient heterogeneity in terms of how patients think about value. David Wamble (indirect benefits researcher perspective) will speak to how assessments differ when considering indirect benefits in value assessment. Josh Seidman (framework developer perspective) will address how their patient-centered value assessment framework incorporates patient perspectives.

OVERVIEW:

Value assessment frameworks are becoming a tool increasingly used by decision-makers to evaluate the use of healthcare services. It is widely accepted that value assessment, an already complex pursuit, is complicated further by the inclusion of the patient perspective. Many argue that for value assessment tools to achieve maximum utility they should incorporate the patient point-of-view. Few agree, however, on the best way to achieve this goal. The list of challenges in doing so are long and include reconciling individual preferences and needs with population-level decision-making, accounting for long-term benefits on a short-term time horizon, and acknowledging indirect benefits in a healthcare system perspective that is traditionally aligned to consider more tangible clinical benefits. Lack of consensus about the appropriate approach puts us at risk for ignoring patient perspectives altogether. Continual debate as well as trial and error are necessary to ensure forward movement. This panel, with participation from the audience, will attempt to answer the question about what aspects of the patient perspective should be included in value assessment frameworks and how to best incorporate them.

Use of Real World Data Issues

1:45 PM - 2:45 PM
Room: Ballroom II (Level 400)

IP21: IS ARTIFICIAL INTELLIGENCE THE NEXT BIG THING IN HEALTHCARE DECISION MAKING?

Moderator:

Santiago Ernesto Herran, MD, Market Access Head, Bayer Andina, Bogota, Colombia

Panelists:

Rafael Alfonso Cristancho, MD, MSc, PhD, Senior Director, Value Evidence Outcomes, R&D, GlaxoSmithKline, Collegeville, PA, USA

Juan-David Rueda, MD, MS, Doctoral Candidate, Pharmaceutical Health Services Research, University of Maryland School of Pharmacy, Baltimore, MD, USA

Carrie Savage Bennette, PhD, MPH, Senior Methodologist, Flatiron, NY, NY, USA

ISSUE:

Artificial Intelligence (AI), software and algorithms that analyzes medical data, has been used for diagnosis, therapeutic, and predictive models. AI's optimal use is still controversial. From privacy concerns to accuracy and validity of algorithms and their results are debatable. Dr. Rueda will present examples of successful use of AI in healthcare and explore the potential benefits of this technology, focusing on optimization of available data, early adoption and potential decrease of human error analyzing large amounts of complex data. Dr. Alfonso will focus on the limitations of data privacy and the challenges with the validations of the algorithms used in AI, which could be seen as a "black box" for the user, and the potential lack of accountability from developers versus the healthcare provider or decision maker who is using them. The session will be moderated by Dr. Herran who will highlight critical points and encourage participation from the audience.

OVERVIEW:

Increased availability of electronic medical information and higher computing power led IT developers and healthcare researchers to combine efforts to develop software and algorithms to identify and predict patterns using complex medical data, known as AI. The use and implementation of AI in healthcare is variable and controversial. The algorithms and software can be proprietary and not fully transparent to the users, limiting the ability to reproduce it's findings or standardize its approaches. The quality and quantity of the data become critical elements for the predictive power of AI but is unclear how to control potential data privacy issues and incomplete or inaccurate data that often lingers in EHRs. In other fields, AI has been transformational but the role in healthcare is still to be determined. Early successes and clear identification of the challenges should help us to enhance its use and inform better decisions to improve the population health.

3:00 PM - 4:00 PM
BREAKOUT SESSION

3:00 PM - 4:00 PM
Room: Ballroom I (Level 400)

IP22: CHALLENGES AND OPPORTUNITIES IN THE USE OF REAL-WORLD EVIDENCE FOR REGULATORY DECISION MAKING FOR IN VITRO DIAGNOSTICS

Moderator:

Tyler J. O'Neill, DVM, MSc, PhD, Scientist, Diagnostics Information Solutions, Roche Diagnostics, Pleasanton, CA, USA

Panelists:

Michael S. Waters, PhD, SHIELD Team Lead; CDRH RWE Tactical Team – Diagnostics, Office of in vitro Diagnostics and Radiologic Health (OIR), Food and Drug Administration (FDA), Silver Spring, MD, USA

Brad Spring, BS, Vice President, Regulatory Affairs, BD Life Sciences, Sparks, MD, USA

Asif Jan, PhD, Lead, Diagnostics Information Solutions, Roche Diagnostics, Pleasanton, CA, USA

ISSUE:

Evidence from “real world” practice can align healthcare decisions more closely with individual patient needs. How this evidence can be used to support regulatory decision making for IVDs is less well defined. The panel will present, debate, and reflect on challenges and opportunities to leverage RWE to address this gap. Dr. Alain Silk will present an overview of current uses and case examples of regulatory use of RWE, including FDA RWE Guidance for IVDs. Brad Spring will describe its challenges, barriers, and limitations. Finally, Dr. Asif Jan will provide insight into future opportunities and identifying gaps for RWE in regulatory claims (and how do we get there?). Each panelist will speak for 10 minutes followed by a 15-minute moderated discussion and 15 minutes of audience Q&A.

OVERVIEW:

Current frameworks for IVD regulatory approval rely almost exclusively on evidence from randomized clinical trials (RCTs) that use narrow patient populations, circumscribed clinical settings, and limited patient journeys. Use of RWE for regulatory decision making, including new approvals and label expansions, may be suitable in settings where trial conduct is impractical and can address evidentiary gaps not addressed in RCTs (e.g. long-term outcomes). Use of real-world data (RWD) may also reduce time and cost of evidence development and is expected to be a cornerstone of future IVD regulatory submissions. Current FDA evidentiary standards for regulatory decision making evaluate whether available evidence is of sufficient quality to address the decision being considered. Nevertheless, current standards of analytic methods and associated data to generate RWE for regulatory decision making present hurdles to full realization of its utility including access and adequacy of relevant, reliable, valid and timely data on patient outcomes. Thus, opportunities exist to take advantage of a more pragmatic approach to evidence generation of regulatory decision making (e.g. 2017 FDA RWD guidelines, 21st Centuries Cures Act). Critical understanding of how RWE will redefine the landscape of regulatory strategies will be necessary for competitive advantage in the future and demonstrate clinical utility of IVD to improve patient outcomes.

Patient-Reported Outcomes & Patient Preference Research Issues

3:00 PM - 4:00 PM
Room: Ballroom II (Level 400)

IP23: ESTIMATING SAMPLE SIZE FOR QUALITATIVE RESEARCH IN CLINICAL OUTCOME ASSESSMENT RESEARCH: ONE SIZE DOES NOT FIT ALL!

Moderator:

Helen Kitchen, MSc, Senior Consultant, Clinical Outcomes Assessment, DRG Abacus, Manchester, UK

Panelists:

Kathryn Lasch, PhD, Executive Director, Patient Reported Outcomes, Pharmerit International, Newton, MA, USA

Helen Doll, PhD, Strategic Lead, Quantitative Science, Clinical Outcomes Solutions, Folkestone, UK

Katy Benjamin, PhD, Director, HEOR - Patient Reported Outcomes, Abbvie Inc, Chicago, IL, USA

ISSUE:

Recent regulatory guidelines for collecting patient-focused data emphasize that samples must be representative of the target patient population.[1] However, sample size estimation methods for qualitative studies to develop tools for patient-centered research have been questioned.[2] The need to identify appropriate sample sizes is highlighted within ISPOR PRO Good Research Practices yet few practical guidelines are available. Recommendations from various researchers are inconsistent.[3]

OVERVIEW:

This panel discusses the theoretical underpinnings and practical considerations for sample size selection in qualitative research studies and will debate different approaches for COA development and validation. Kathy Lasch will present qualitative approaches to sample size selection for COA development and preference studies. Helen Doll will address alternative methods, including recent advances in quantitative social science methods (e.g. Fugard and Potts, 2015)[4] and mixed method approaches. Katy Benjamin will debate the PROs and CONs of these approaches. All panelists will highlight clinical and practical factors which may influence sample size estimation for qualitative studies including study type, condition prevalence, number and size of subgroups of interest (e.g., condition or relevant demographic subtypes), complexity of the research question, interview length, and resource constraints. The panelists will debate methods and broaden discourse to advance qualitative research methodology.

[1] FDA PATIENT-FOCUSED DRUG DEVELOPMENT PUBLIC WORKSHOP ON GUIDANCE COLLECTING COMPREHENSIVE AND REPRESENTATIVE INPUT. December 18, 2017.

[2] Lundy J. Sample Optimization in Scale Development. SIXTH ANNUAL PRO CONSORTIUM WORKSHOP April 29 - 30, 2015.

[3] Patrick DL, Burke LB, Gwaltney CJ, et al. Content validity - Establishing and reporting the evidence in newly-developed PRO instruments for medical product evaluation: ISPOR PRO good research practices task force report: Part 2 – Assessing respondent understanding. Value Health 2011;14; 978-988.

[4] Fugard AJB, Potts, HWW (2015) Supporting thinking on sample sizes for thematic analyses: a quantitative tool. International Journal of Social Research Methodology. Vol. 18 (6)

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