ISPOR Forums
Monday, May 21, 2018
6:15 PM - 7:15 PM
BREAKOUT SESSION

6:15 PM - 7:15 PM
Room: Ballroom I (Level 400)
F1: HEALTH STATE UTILITY (HSU) GOOD PRACTICES TASK FORCE RECOMMENDATIONS FOR IDENTIFICATION AND USE OF HSU DATA IN COST-EFFECTIVENESS IN DECISION MODELLING
Moderator:

John E. Brazier, PhD, MSc, Dean, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

Speakers:

Andrew Lloyd, DPhil, Director, Acaster Lloyd Consulting Ltd., London, UK

Bruce Crawford, MPH, MA, Vice President, Real World Evidence, APAC, Syneos Health, Tokyo, Japan

A. Simon Pickard, PhD, Professor, Department of Pharmacy Systems, Outcomes and Policy, University of Illinois at Chicago, Chicago, IL, USA

DESCRIPTION:

PURPOSE: Attendees will learn how to identify and critically appraise utilities in a systematic and transparent way to meet the needs of the model, as well as reimbursement agencies, if/when to synthesize, considerations and assumptions when using HSU estimates in cost-effectiveness models, and reporting standards. The audience work through several systematic review examples to illustrate the “hows” and “whys” behind the task force’s proposed checklist.
DESCRIPTION: HSU estimates can drive results of cost effectiveness analysis. Currently, little attention is paid to evidence sources and methods used to apply these estimates in models. The absence of rigorous standards can lead to ”cherry picking” data that produce the most favorable cost per QALY. We will describe the methods for identification and appraisal of utilities to answer the question: Are comprehensive or structured searches best? We will then discuss the considerations in HSU evidence synthesis answering questions on if/when to synthesize, the appropriateness of combining estimates obtained from different measures, minimum data points needed, the use of mapped values, and alternative methods of synthesis. We then will work through the factors and assumptions to consider when using HSU estimates in cost-effectiveness models, including comorbidities, treatment related adverse events, concurrent clinical events, and the importance of capturing uncertainty. In our final example, we will examine reporting of the selection and synthesis of HSU estimates, including the search strategy, relevance of the criteria applied, and strengths and limitations of the evidence.
Presented by the ISPOR HSU Estimates in Cost Effectiveness Models Good Practices Task Force


6:15 PM - 7:15 PM
Room: Ballroom II (Level 400)
F2: HANDLING MISSING VALUES IN REAL-WORLD DATA: ARE THERE CHALLENGES FOR REGULATORY DECISIONS FOR MEDICAL PRODUCTS?
Moderator:

Laurence Djatche, PharmD, Health Economics and Outcomes Research Post-Doctoral Fellow, College of Population Health, Thomas Jefferson University, Philadelphia, PA, USA

Speakers:

Nneka C. Onwudiwe, PhD, PharmD, MBA, PRO/PE Scientific Reviewer, US Food and Drug Administration, Silver Spring, MD, USA

Helene Karcher, PhD, Managing Vice-President and Global Head of Modeling, Analytica Laser, Basel, Switzerland

Judith J. Stephenson, SB, SM, Principal Scientist, Survey Research, HealthCore, Inc., Wilmington, DE, USA

Samuel Wilson, PhD, BS, MS, Associate Director-Statistics, Astellas Pharmaceuticals US, Inc., Northbrook, IL, USA

DESCRIPTION:

Purpose: Section 3022 of the 21st Century Cures Act directs FDA to evaluate and issue guidance on the use of real world evidence to support approval of a drug or to satisfy post approval study requirements. However, the problem of handling missing values in real-world healthcare datasets is not completely solved. Namely, the flexibility in methods to handle missing data in analyses and the assumptions made about the data can lead to different results and/or introduce biased estimates. Are there challenges ahead?

Description: Missing data can represent a potential source of bias and a substantial loss of precision and power in randomized trials and observational studies. Missing data in healthcare datasets such as claims, registries, and electronic medical records can present unique challenges. Missing data is generally categorized as missing completely at random (MCAR), missing at random (MAR) or missing not at random (MNAR). The credibility of the analysis will depend on the amount of missing data, reasons for missingness (e.g., outcome data, baseline covariates), assumptions made about the data, and the methods used to handle missing data. One approach to handle the missing values is to conduct a complete case analysis. This approach, however, assumes that the missing data are MCAR and can possibly result in less precision and often biased estimates. There are various imputation techniques for missing data such as last observation carried forward (LOCF), multiple imputation, regression imputation, etc. Nevertheless, complete case analysis is considered the most common approach to handling missing data. ISPOR’s Statistical Methods in Health Economics and Outcomes Research Special Interest Group has set out to provide statistical leadership for strengthening the use of appropriate statistical methodology in health economics and outcomes research and improve the analytic techniques used in real world data analysis. This forum will discuss the best possible methods for handling missing data and potential drawbacks from their perspectives, depending on the context of the analysis. The speakers will also include their experiences in handling missing data, and provide recommendations for conduct and reporting. The presentations will focus specifically on the reasons for missingness (e.g., outcome data, baseline covariates), assumptions made about the data, possible percentage of missing values, the methods used to handle missing data, and the intended use of the results. The speakers will address how incorrect assumptions underlying the mathematical model used to fix the missingness can lead to biased estimates and eventually to misleading recommendations. Finally, speakers will discuss the regulatory impact and challenges when informing regulatory decisions related to approval and promotion.

The panel will discuss the best approach and provide recommendations for conduct and reporting, and allow the audience to participate in the discussion and provide feedback through questions posed to attendees.

Presented by the ISPOR Statistical Methods in Health Economics and Outcomes Research Special Interest Group


6:15 PM - 7:15 PM
Room: Ballroom III (Level 400)
F3: DEFINING PATIENT CENTEREDNESS AND ENGAGEMENT IN HEOR: PROPOSED DEFINITION AND STAKEHOLDER RESPONSE
Moderator:

Rachel L. Harrington, BA, Pre-Doctoral Fellow, Institute for Health Research and Policy/College of Pharmacy, University of Illinois at Chicago, Chicago, IL, USA

Speakers:

Eleanor M. Perfetto, PhD, RPh, MS, Senior Vice President, Strategic Initiatives, National Health Council, Washington, DC, USA and Professor, Pharmaceutical Health Services Research, School of Pharmacy, University of Maryland School of Pharmacy, Baltimore, MD, USA

Suzanne Schrandt, JD, Director, Patient Engagement, Arthritis Foundation, Atlanta, GA, USA

Sarah Donelson, MA, Lead, Patient-Centered Research, Genentech, San Francisco, CA, USA

DESCRIPTION:

PURPOSE: To present the proposed consensus definition of “patient engagement” suitable for use in the context of health economics and outcomes research (HEOR). The definition derived from a multi-step process, including a systematic review and qualitative analysis of existing definitions, and a multi-stakeholder review. The implications of a consensus definition, and response to the proposed definition, will be shared from key stakeholder perspectives: Patients, Policy/Research and Industry. Attendees will come away with an understanding of the relevance and impact of adopting a standard definition, as well as organizational and research implications.

DESCRIPTION: Many stakeholders are implementing patient-related initiatives - including engagement and involvement - in drug development, research, and delivery of healthcare. Quantifying and measuring the impact of initiatives related to “patient engagement” or “patient centeredness” has become difficult, as these terms are used interchangeably and are often not defined. This can lead to a misunderstanding of concepts and expectations. This lack of a consistent, clear, definition has been highlighted in outcomes research, pharmacoepidemiology and related fields, as a barrier to implementing and measuring patient engagement in the field. A group comprised of researchers and patient representatives undertook a process to identify or develop a consensus definition that could be adopted for the HEOR area. The proposed definition represents an analysis of the current literature, as well as input from stakeholder representatives. Members of the panel will share their impressions of the definition, as well as implications for its adoption in their area, such as metrics for measuring engagement activities, and shared engagement principles for industry-patient collaborations.

Presented by the ISPOR Patient Centered Special Interest Group


6:15 PM - 7:15 PM
Room: 309 (Level 300)
F4: THE ISPOR HEOR COMPETENCIES FRAMEWORK™: GUIDING PROFESSIONAL DEVELOPMENT FOR THE FIELD OF HEOR
Moderator:

Bill Marder, PhD, MA, BA, Chief Data Strategist, IBM Watson Health, Cambridge, MA, USA

Speakers:

James F. Murray, PhD, Research Fellow, Global Health Outcomes - COE, Eli Lilly and Company, Carmel, IN, USA

Laura T. Pizzi, PharmD, MPH, Professor and Director, Center for Health Outcomes Policy and Economics, Rutgers University, Piscataway, NJ, USA

DESCRIPTION:

This Forum will present the ISPOR HEOR Competencies Framework. As part of its core mission, ISPOR seeks to provide educational and experiential offerings that assist early career and HEOR professionals build their skills and experiences relevant to their own HEOR career path. A Competency Assessment Inventory was developed to guide these individuals as well as educators and hiring managers in the recruitment fields. The long-term goal of the Competency Assessment Inventory is to better define the HEOR discipline and the scope of ISPOR’s career development offerings.

This initiative is a collaboration between ISPOR staff and the ISPOR Institutional and Faculty Advisor Councils. Since its inception in 2017, the Initiative has identified an inaugural Inventory of the HEOR Competencies. The Forum will present the structure and contents of the Inventory. It will also present the results of two surveys from Academic and Industry ISPOR members. The Academic survey will present how the representative HEOR degree granting programs match and fulfill the competencies within the ISPOR Competency Inventory. The Industry survey results will benchmark the validity and relevance placed on the competencies by industry members. Finally, the Forum will present its plans for future development and use of the Competency Inventory.

Future work will include:

  • A final set of key topics within each Competency
  • Assessment of whether the Competency is best learned didactically or experientially, and
  • Defining specialties within the Competency Inventory that practitioners should master to be competent in select areas (e.g., Patient Reported Outcomes, Real World Evidence, etc.)

With the fully developed and validated inventory, ISPOR, in collaboration with many excellent academic institutions and member companies will be able to provide educational and experiential offerings.

Presented by the ISPOR HEOR Competency Assessment working group


6:15 PM - 7:15 PM
Room: 310 (Level 300)
F5: ADVANCING PATIENT ACCESS TO INNOVATIVE HEALTH TECHNOLOGIES IN ASIA – THE ROLE OF REAL-WORLD DATA IN THE VALUE FRAMEWORK
Moderator:

Boxiong Tang, MD, PhD, Senior Director, Global Health Economics and Outcomes Research (GHEOR), Teva Pharmaceutical, Frazer, PA, USA

Speakers:

Jianwei Xuan, PhD, Professor and Director, Health Economic Research Institute, Sun Yat-sen University, Guangzhou, China

Larry Liu, PhD, MD, Executive Director, Merck, Rahway, NJ, USA

Makoto Kobayashi, PhD, Director and Chief Operating Officer, CRECON Medical Assessment Inc., Tokyo, Japan

Sang-Soo Lee, PhD MBA, Director, Corporate Affairs, Medtronic Korea, Seoul, Korea, Republic of (South)

DESCRIPTION:

With the new development of healthcare systems toward the Value Framework for reimbursement in the world, Real World Evidence (RWE) is becoming a more important to be used by decision making process for reimbursement authorities in Asian countries. At this critical point, it would be important to have a firm grasp on what evidence can be presented, as well as the acceptable, in the value framework for reimbursement.

To support and promote this initiative, Asia Consortium Industry Committee has launched two related projects in Real World Data (RWD) development and Health Technology Assessment (HTA) requirements in Asia. At this forum, speakers will present the objectives, approaches, methods of these projects, and provide status updates, and expectation of the deliverables, with usefulness of the outcomes to support regional reimbursement decision making, especially for researchers who need to collect and provide the RWD evidences.

The objectives are:

  1. To assess HTA value evidence requirements in the Asian region via information on current HTA status in selected countries and their trend in 3-5 years.
  2. To provide a data source platform to support RWE/RWD research in the region, by conducting a landscape evaluation of current information on the availability and accessibility of various existing RWD in Asia and performing a general quality assessment and contents evaluation of each type of the RWD.

During the forum, reimbursement negotiation cases from selected countries with outcomes data communication and recommendation on the value framework to generate the right evidence will be provided. How RWD evidence generation meets the needs for growing HTA demand in Asia and how to incorporate RWE in market access strategies will also be discussed.

Presented by the ISPOR Asia Consortium Industry Committee

Tuesday, May 22, 2018
6:15 PM - 7:15 PM
BREAKOUT SESSION

6:15 PM - 7:15 PM
Room: Ballroom IV (Level 400)
F6: VALUE OF INFORMATION (VOI) ANALYSIS FOR RESEARCH DECISIONS EMERGING GOOD PRACTICES TASK FORCE: FINAL RECOMMENDATIONS
Moderator:

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

Speakers:

Lotte Steuten, PhD, MSc, Associate Professor & Director, Health Economics, Fred Hutchinson Cancer Research Center, Seattle, WA, USA

Anirban Basu, PhD, Stergachis Family Endowed Professor & Director, The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington, Seattle, WA, 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

James F. Murray, PhD, Research Fellow, Global Health Outcomes - COE, Eli Lilly and Company, Carmel, IN, USA

DESCRIPTION:

Value of Information (VOI) methods provide essential tools to quantify the value of additional evidence to reduce uncertainty in decision making and inform research prioritization decisions. In this session, members of the task force will 1) provide an overview of VOI and the types of decisions where it can be used, 2) present the good practice recommendations from the task force papers. This will be followed by a panel discussion, incorporating a range of perspectives, on how VOI can be integrated in practice, highlighting possible barriers and potential solutions and detailing the implications for decision-making of incorporating VOI methods.

This interactive forum will be informative for anyone involved in supporting and making research decisions including researchers, HTA agencies, decision makers, and industry analysts. Come along and join in the debate!

Presented by the ISPOR Value of Information (VOI) Emerging Good Practices Task Force


6:15 PM - 7:15 PM
Room: Ballroom I (Level 400)
F7: ENABLING ISPOR MEMBER AND PATIENT ADVOCACY GROUP RESEARCH COLLABORATION: A VOLUNTEER PLAN
Moderator:

Richard J. Willke, PhD, Chief Science Officer, ISPOR, Lawrenceville, NJ, USA bio

Speakers:

Laura T. Pizzi, PharmD, MPH, Professor and Director, Center for Health Outcomes Policy and Economics, Rutgers University, Piscataway, NJ, USA

Jason Harris, BA, Associate Director of Policy and Programs, National Health Council, Washington, DC, USA

Brian Ung, PharmD, MS, Post-Doctoral Fellow, US Health Economics and Outcomes Research, Celgene Corporation, Summit, NJ, USA

DESCRIPTION:

Greater patient engagement in research is being heavily encouraged, and health economics and outcomes research is a key area for patient involvement. However, mechanisms to link patient advocacy groups and researchers are not yet fully developed. In some cases, a patient group may need to do such research itself, for either internal or external purposes, but may lack the expertise and funding do it. There may be ISPOR members interested in helping such groups - e.g., students needing a small project, or more experienced members who are willing to donate their time and expertise to a patient-oriented cause. From the ISPOR researcher’s perspective, many funding opportunities now require patient input, meaning that advance knowledge of, and a mechanism to connect with, interested patient groups would be of significant value.

ISPOR and National Health Council have been collaborating to understand 1) the interest of ISPOR members and patient advocacy groups in voluntary research collaborations, and 2) pilot and operationalize a mechanism to match ISPOR volunteers and patient groups on identified research projects. Recently, an interview-style survey of 10 NHC member groups has revealed significant interest on patient advocacy group side, and an on-line survey of selected ISPOR groups elicited 235 responses, of which 90% showed clear interest in doing volunteer research work, either short-term or long-term. Pilot collaborations are now being organized. At a Forum, this effort and the draft matching mechanism would be explained, both to solicit feedback on the plan and to make more direct contact with potentially interested members.

Presented by the ISPOR Patient-Research Matchmaking Group


6:15 PM - 7:15 PM
Room: Ballroom II (Level 400)
F8: REAL-WORLD EVIDENCE AND LOCAL EVIDENCE GENERATION: HOW SHOULD IT BE APPROACHED IN ASIA PACIFIC?
Moderator:

Jitendar Sharma, PhD, CEO, Andhra Pradesh MedTech Zone Limited, Andhra Pradesh, India

Speakers:

Sukyeong Kim, PhD, Senior Research Fellow, Evidence-based Research Department, National Evidence-Based Healthcare Collaborating Agency, Seoul, Korea, Republic of (South)

Makoto Kobayashi, PhD, M.Eng., Director and COO, CRECON Medical Assessment Inc., Tokyo, Japan

Jianwei Xuan, MD, PhD, Director, Health Economic Research Institute, School of Pharmacy, Sun Yat-Sen University, Guangzhou, China

Bor-Sheng Ko, MD, PhD, Attending Physician and Clinical Assistant Professor, BMT Unit and Hematology Division, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan

DESCRIPTION:

Real World Evidence (RWE) helps inform health care decisions by improving quality and safety and reducing costs. The use of RWE to support decision-making is quite common in Europe and North America. It becomes increasingly prevalent in the Asia-Pacific region; however, there are many factors adding barriers to its use in the region, such as limited data sources, poor data quality. Is using global evidence sufficient for decision making in the Asia-Pacific? What does local evidence tell us? In this forum, speakers from different sectors in Asia will share their perspectives and experiences on RWE, and discuss challenges faced when generating and using RWE.
Presented by the ISPOR Asia Consortium HTA Agencies, Health Care Policymakers & Payers Committee


6:15 PM - 7:15 PM
Room: Ballroom III (Level 400)
F9: NEW DEVELOPMENTS OF VALUE-BASED PAYMENT ARRANGEMENTS AND VALUE DEMONSTRATION IN LATIN AMERICAN HEALTH SYSTEMS
Moderator:

Juan Carlos Alandete Manotas, MD, MSc, MPH, MACS LATAM, Bayer, Whippany, NJ, USA

Speakers:

Louis Garrison, PhD, Professor Emeritus, CHOICE Institute, University of Washington, Seattle, WA, USA

Ramiro E. Gilardino, MD, MSc, Director, Global Networks Latin America, ISPOR, Lawrenceville, NJ, USA

Boxiong Tang, MD, PhD, Senior Director, Global Health Economics and Outcomes Research (GHEOR), Teva Pharmaceutical, Frazer, PA, USA

DESCRIPTION:

PURPOSE:

To increase awareness of new developments in value-based payment (VBP) arrangements and value demonstration in Latin American health systems.

Increasing healthcare costs—and particularly for innovative medicines—are a growing concern in Latin American countries. Value-based pricing and payment have been proposed as one of the strategies to improve outcomes while controlling costs. The proposed cost-containment strategies include: risk-sharing arrangements, outcomes-based contracting, and high-cost drug funds. These are in addition to the use of health technology assessment, pharmacoeconomic guidelines, reference pricing, and care pathways. Meanwhile, health plan administrators and industry executives are exploring the full range of these options in an effort to understand VBP applicability in regional healthcare systems. In this proposed workshop, speakers from academia, pharmaceutical industry, and international organizations will describe and discuss the history, current development, and trends in VBP in Latin American markets. Lessons from other regions will also be presented. The final 15 minutes of the workshop will be devoted to discussion and Q&A with the audience about future developments of VBP in the region.

Presented by the LA Consortium Technology Producers Committee

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