Prerelease Sessions


Can RWE Observational Research Replicate RCT Results, and Should We Care?

Moderator: Lucinda Orsini, DPM, MPH, COMPASS Pathways, Skillman, NJ, USA

Panelists: Barbara Bierer, MD, Multiregional Clinical Trials Center, Brigham and Women's Hospital, Harvard University, Cambridge, MA, USA; David Thompson, PhD, Real World & Late Phase, Syneos Health, Boston, MA, USA; Jeroen Paul Jansen, PhD, PRECISIONheor, Oakland, CA, USA

ISSUE: There are many studies and reviews focused on the ability of real world evidence studies to replicate and reproduce randomized clinical trials. However, we often use RWE to answer questions that an RCT is not designed to answer. Can RWE replicate RCTs and if they do does that really give us comfort that RWE is credible and reliable?

OVERVIEW: RCT replications studies using secondary data sources are currently producing much awaited data to see how well RWE can produce ‘accurate’ results as defined by replicating RCT findings. DUPLICATE is sponsored by the FDA and intends to replicate 30 completed Phase III or IV trials and to predict the results of seven ongoing Phase IV trials using Medicare and commercial claims data. OPERAND conducted by Brown University and Harvard Pilgrim Health Care Institute (funded by unrestricted grants from a consortia of pharmaceutical companies) intends to replicate the ROCKET-AF trial for atrial fibrillation and the LEAD-2 trial for Type 2 diabetes control using claims from commercial and Medicare Advantage plans and electronic medical record data from OptumLabs Data Warehouse. This panel will explore viewpoints from both RWE experts involved in these duplication studies and as RWE experts, and from the experts who understand the strength of evidence that is needed in order to make causal inference or comparative effectiveness/efficacy claims. The moderator will open the session with a 5 minute introduction to the issue and each speaker will have 10 minutes to discuss their view point. There will be interactive use of polling through the zoom platform and at least 20 minutes for audience Q&A or for the panelists to query each other on their viewpoints if on-demand. This session will be of interest to HEOR professionals in life sciences industry, academia, regulatory or payer space who are interested in RWE.

What Is Research-Grade Real-World Evidence and Why Does It Matter?

Moderator: Dan Riskin, MD, MBA, Verantos, Menlo Park, CA, USA; 
Panelists: Tina Hernandez-Boussard, M.P.H, Ph.D., M.S., School of Medicine, Stanford University, Stanford, CA, USA; Nancy A Dreyer, MPH, PhD, Real-World Solutions, IQVIA, Cambridge, MA, USA; Keri L Monda, PhD, Center for Observational Research, Amgen Inc, Thousand Oaks, CA, USA

ISSUE: Increasingly, real-world evidence (RWE) is used to make clinical assertions and influence the standard of care through regulatory, reimbursement, and clinical decision-making. High validity RWE is increasingly desirable and required. But, the industry has not yet reached consensus on what it means to produce “research-grade” evidence using “fit-for-purpose” data. As physicians, insurance, and regulators increasingly scrutinize RWE for quality, it is now an industry imperative to understand issues of data quality and design a strategy to produce high-validity evidence.

OVERVIEW: This panel will seek to unpack this complex issue. Dan Riskin, the panel moderator, will set the stage for discussion by highlighting key questions, including: What are the differences between traditional and advanced RWE? What factors influence credibility of RWE? Where are there still gaps in data accuracy and population generalizability? Tina Boussard will describe scientific efforts to use advanced data sources and how this influences data quality. Keri Monda will address how the pharmaceutical industry is innovating in advanced RWE while maintaining and even growing efforts in traditional RWE. Nancy Dreyer will comment on advances in evidence validity and outline current thinking on research-grade RWE in the era of the 21st Century Cures Act. The moderator will ensure that at least 15 minutes of the session is devoted to soliciting audience interaction and feedback on the competing viewpoints.

When Patients and Clinicians Talk, How Are We Listening? A Discussion of How Patient and Clinician Experience Shapes HTA Recommendations

Moderator: Alexandra Chambers, BSc, MA, Novartis, Dorval, ON, Canada

Panelists: Paul Wheatley-Price, BSc, MBChB, FRCP (UK), MD, Ottawa Hospital Cancer Centre, Ottawa, ON, Canada; Sarah Berglas, BSc, BA, CADTH, Ottawa, ON, Canada; Christina Sit, BSc, Lung Cancer Canada, Toronto, ON, Canada

ISSUE: Innovative therapies are continually being developed, and Health Technology Assessments (HTA) agencies are evolving to keep up with the pace of innovation. A key tenant of HTA is the engagement of clinicians and patients who provide context and experience with the disease and therapy under review. Are we optimizing the perspectives of these key stakeholders into the HTA processes as novel therapies continue to enter the health system?

OVERVIEW: HTAs are designed to inform decision making through a multi-stakeholder approach considering many perspectives. For many years, the health care system has committed to focusing on patient-centred care, and that priority should also apply to HTA. The Canadian Agency for Drugs and Technologies in Health (CADTH) has grown to become a leader in patient engagement in HTA, where now there is a call for patient and clinician input for every review. Yet today’s treatment paradigms have been transformed by innovation and novel clinical trial designs, and the pace of new drug discovery continues to increase, putting pressure on the HTA process to further evolve. Real-world insights and the treatment values that patients and clinicians share can be enhanced. For this session, a diverse group of panelists will examine the strengths and opportunities that exist to meaningfully incorporate the experiences that patients and clinicians into HTA.

Risks and Rewards of Tokenization: Clinical Trial Data Meets the Real World

Moderator: Arnaub Chatterjee, MHA, MPA, Acorn AI at Medidata, a Dassault Systèmes company, Boston, MA, USA

Panelists: John Concato, MD, MS, MPH, BEng, CDER, FDA, Silver Spring, MD, USA; Andrew Kress, BA, HealthVerity, Philadelphia, PA, USA; Najat Khan, PhD, Janssen R&D Strategy and Operations, Johnson & Johnson, Malvern, PA, USA

ISSUE: The efficacy of new drugs is usually established by clinical trials (CTs) that are conducted with a finite follow-up period at the end of the CT, patients are often no longer followed. Data tokenization allows for patients in a CT to be followed using RWD, which in turn will facilitate accurate understanding of post-clinical trial treatment patterns and healthcare resource utilization, as well as providing information on long-term effectiveness and safety. These benefits would be specifically of importance when: It may not be feasible or ethical to run large and lengthy CTs (as often occurs in oncology and rare disease) or a situation warrants urgency, such as during the COVID-19 pandemic. At the same time, merging data from various sources in a secure manner is challenging due to: Lack of interoperability among existing healthcare systems or concerns regarding privacy and potential vulnerability of patients’ personal and health-related information. An urgent need therefore exists to explore this topic from the perspectives of relevant stakeholders.

OVERVIEW: The panel will discuss the opportunities and challenges associated with combining the data of patients enrolled in the clinical trial with real-world data sources.

Improving Validity and Relevance of Indirect Comparisons and Network Meta-Analysis: Implications for HTA Submissions

Discussion Leaders: David M Phillippo, PhD, Bristol Medical School, University of Bristol, Bristol, BST, UK; Jeroen P Jansen, PhD, School of Pharmacy, University of California San Francisco, San Francisco, CA, USA, and PrecisionHEOR, Oakland, CA, USA; Nicky J Welton, PhD, Bristol Medical School, University of Bristol, Bristol, UK 

PURPOSE: Indirect comparisons and network meta-analysis (NMA) estimate relative treatment effects between competing health technologies, however these methods may lack validity if there are differences in patient populations between included studies. Furthermore, in Health Technology Assessment (HTA) the estimates need to be relevant to the target population for the decision. This workshop will provide an overview of methods for indirect comparisons and NMA that aim to account for population differences. Advantages and disadvantages of the different methods and implications for future HTA submissions will be discussed.

DESCRIPTION: This workshop will begin with an overview of different approaches to population adjustment for indirect comparisons and NMA. If individual patient data (IPD) are available for all studies then IPD-level network meta-regression is the gold standard approach. However, in HTA IPD are typically only available for one or a subset of studies. Available methods in this case include matching-adjusted indirect comparison (MAIC), simulated treatment comparison (STC), network meta-regression, and the recently developed multilevel network meta-regression (ML-NMR). The methods will be compared as to their: validity; flexibility to synthesise networks of any size and availability of IPD; and ability to produce estimates in a given target population of interest. Their performance in simulation studies will be presented. Recommendations on when to use these methods for future HTA submissions will then be discussed based on hypothetical yet representative case-studies characterized by different evidence scenarios. Dr Jansen will introduce current methods for population adjustment in HTA. Dr Phillippo will then describe ML-NMR and compare the methods. Finally, Prof Welton will discuss recommendations for HTA. Presentations will last 40 minutes, followed by an interactive Q&A session, providing the audience an extended opportunity to discuss the issues raised with the panel. This workshop will benefit those involved in preparing or critiquing HTA submissions that utilise population adjustment methods.

Identifying Health Innovation Gaps: A Landscape Analysis

Discussion Leaders: Laura Elisabeth Gressler, B.A., M.S., Pharmaceutical Health Services Research, University of Maryland, School of Pharmacy, Baltimore, MD, USA; Elise Berliner, PhD, Center for Outcomes and Evidence, Agency for Healthcare Research and Quality, Rockville, MD, USA 

PURPOSE: Funding and activity in health innovation may not be aligned with the areas of highest public health burden and costs to the system. This workshop will focus on an attempt to develop of a reliable and valid model that helps to forecast, identify, and prioritize critical health and innovation gaps.

DESCRIPTION: The Department of Health and Human Services (HHS) and FasterCures convened a group of health experts and data scientists from public and private sectors to develop an objective, data-based methodology to identify critical health innovation gaps in order to inform future programs and policies. The multidisciplinary group aimed to develop a reliable and valid model to help identify critical health innovation gaps as measured by three domains: public health impact, healthcare costs, and innovation activity. The workshop will outline the process for developing the metrics from data sources and methodology for an analytic model and data analyses. Results from the pilot study will also be presented. The first discussion leader will present in 15 minutes the overarching goal of the project and the methodologies employed by each of the subgroups. The second presenter will then present for 10 minutes the detailed methodology used within the public health impact subgroup to identify conditions with the highest public health impact for the pilot and calculate research disparities. Workshop participants will then contend with issues in choosing data sources and developing metrics to measure public health burden and contribute feedback on methods of the pilot. Finally, how the data from all three domains were brought together in a multicriteria decision modeling will be presented. The final model can be leveraged to identify the areas that have relatively low innovation activity compared to public health burden and costs and allow the HHS to consider prioritization of resources to these important areas.

Advancing Employer Engagement on Value Assessment

Discussion Leaders: Margaret Rehayem, MA, National Alliance of Healthcare Purchaser Coalitions, Washington, DC, USA; Elisabeth Oehrlein, PhD, MS, Research and Programs, National Health Council, Washington, DC, USA; Kimberly Westrich, MA, National Pharmaceutical Council, Washington, DC, USA; Jennifer Bright, MPA, Innovation and Value Initiative, Alexandria, VA, USA 

PURPOSE: This workshop will provide lessons learned from three projects focused on improving employer evidence needs and engagement in value assessment (VA). These findings will inform how current methods can better account for health plan attributes that matter to employers. 

DESCRIPTION: Employers are key healthcare purchasers in the United States. They have a vested interest in ensuring employees have access to timely care that is most likely to meet individual employee and/or family needs. As healthcare costs rise, employers are doubling down on achieving improved value for their dollar. Health care value, from the employers’ perspective, is broader than that considered by many stakeholders and includes the relative impact on clinical outcomes, employee productivity, and organizational value proposition. However, employers have not traditionally been included in discussions about VA. As VA frameworks are increasingly used to inform health care decision-making, it is important that employers and VA bodies engage in meaningful discussions with one another on what is important to them, discuss alignment in perspectives and needs, and identify strategies to improve current approaches. This workshop is relevant for VA bodies, researchers, patients, students, healthcare purchasers, payers and policymakers.

Successfully Selecting, Developing, and Deploying Digital Clinical Measures of Health

Discussion Leaders: Elizabeth (Nicki) Bush, MHS, Global Patient Outcomes and Real World Evidence, Eli Lilly and Company, Indianapolis, IN, USA; Elizabeth Tschosik, PhD, Patient-Centered Outcomes Research, Genentech, South San Francisco, CA, USA; Bray Patrick-Lake, MFS, Evidation Health, San Mateo, CA, USA; Jennifer Goldsack, MS, MBA, DiMe, Boston, MA, USA 

PURPOSE: To support the audience in selecting, developing, and deploying fit-for-purpose, meaningful digital clinical measures across clinical research, patient care, and public health.

DESCRIPTION: The global COVID-19 pandemic has brought new urgency to the drive toward devising a common framework for the development and deployment of high quality, patient centric, digital clinical measures. Multiple digital technologies for symptom tracking and contact tracing are currently in use and/or development, and clinical researchers are now designing new decentralized clinical trial strategies using telemedicine and remote patient monitoring to minimize pandemic-related disruption of the clinical research enterprise. Adoption of a comprehensive "how-to" roadmap to synthesize best practices in the digital health field is essential for guiding these efforts and safeguarding public health.

Building the Real-World Ecosystem for Safety Signal Management: It Takes a Village!

Discussion Leaders: Mary Beth Ritchey, PhD, FISPE, Med Tech Epi, LLC, Philadelphia, PA, USA; Danica Marinac-Dabic, MD, PhD, MMSc, FISPE, CDHR/OCEA, U.S. Food and Drug Administration, Silver Spring, MD, USA; Christopher Tieche, PhD, Medtronic, Chicago, IL, USA; David Wamble, PhD, Bristol Myers Squibb, Lawrence Township, NJ, USA 

PURPOSE: In the world of medical device surveillance, the processes of signal management (detection, discernment and evaluation) are complex and driven by dynamic convergence of device-, provider-, setting- and patient- related characteristics. This workshop will focus on identification of actionable factors for building the (1) capacity of device ecosystem to ascertain and characterize the signal; (2) efforts to utilize novel methods including artificial intelligence/machine learning (AI/ML) and blockchain; (3) transparent and timely communication between stakeholders; and (4) value of patient-centric, interdisciplinary approaches that integrate the medical product ecosystem.

DESCRIPTION: Varying levels of unfamiliarity with real-world data (RWD), study designs, and analytic approaches appropriate for signal management has resulted different levels of trust across the medical device ecosystem, underscoring the need for education and training. This workshop will engage workshop participants in building an ecosystem-driven model for signal management, using examples from paclitaxel-coated devices for the treatment of peripheral arterial disease and diagnostics for identification of biomarkers for oncology treatments. Dr. Richey will discuss methodological capacity and identify needs, some early successes and introduce early- and long-term opportunities. Dr. Marinac-Dabic will present the integration of digital health solutions including blockchain and AI/ML algorithms to advance the value of RWD in active surveillance. Dr. Tieche will highlight the need for responsible transparency and communication across health care sectors and stakeholder perspectives. Dr. Wamble will detail the importance of characterizing new and ongoing risk-benefit balance from a market access perspective. The workshop participants will work interactively in four groups to identify tactical approaches in each of the four domains discussed (i.e., methodologic capacity, integration of digital solutions, communications, and interdisciplinary approach). Interested participants will then also be invited to join the post-workshop mirror stakeholder group to engage in further discussions on this topic.

Clinical Outcome Assessment Studies

Moderator: James Murray, PhD, Eli Lilly and Company, Indianapolis, IN, USA
CO1The Natural History, Clinical Outcomes and Unmet Needs of Patients with Arginase 1 Deficiency (ARG1-D): A Systematic Review of Case Reports

Bin Sawad A1;Pothukuchy A1;Badeaux M1;Hodson V1;Bubb G1;Lindsley K2;Uyei J3, Diaz GA4
1Aeglea BioTherapeutics, Austin, TX, USA, 2IQVIA, Windsor Mill, MD, USA, 3IQVIA, San Francisco, CA, USA, 4Division of Medical Genetics and Genomics in the Department of Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai, New York, NY, USA

*Top 5% Average Review Score

CO2Healthcare Utilization, Cost, and Quality Among High-Need, High-Cost Medicare Beneficiaries in Medicare Fee-for-Service Versus Medicare Advantage

Teigland C1;Pulungan Z2, Mohammadi I3
1Avalere Health, an Inovalon Company, Washington, DC, USA, 2Avalere Health - An Inovalon Company, Washington, DC, USA, 3Avalere Health, Washington, DC, USA

*Top 5% Average Review Score

CO3Comparison of Goal Attainment and Measures of Function in Two Dementia Clinical Trials.

Stanley J1;Howlett SE2;Dunn T1;Stanojevic S1;Chapman C1, Rockwood K2

1Ardea Outcomes, Halifax, NS, Canada, 2Dalhousie University, Halifax, NS, Canada

*Top 5% Average Review Score

CO4Predictors of Inpatient Relapse in Multiple Sclerosis Patients Using First-Line Disease Modifying Therapies: A Machine Learning Study of Real World Data

Icten Z;Friedman M, Menzin J
Panalgo, Boston, MA, USA

*Top 5% Average Review Score


Patient and Quality Reported Outcomes Studies

Moderator: Janine A. Van Til, MSc, University of Twente, Enschede, The Netherlands
PR1Capturing the Health-Related Quality of Life of Children Living with Aadc Deficiency through a Vignette Study: A French Experience
 Smith A1;Hanbury A2;Beitia Ortiz de Zarate I3;Hammes F4;De Pouvourville G5, Buesch K6
1RB, Hull, ERY, UK, 2York Health Economics Consortium, York, UK, 3PTC Therapeutics France, Paris, 75, France, 4PTC Therapeutics France, Paris, France, 5ESSEC Business School, Cergy-Pontoise, France, 6PTC Therapeutics, Steinhausen, Switzerland

*Top 5% Average Review Score

PR2Concordance of Preferences and Treatment Choice Using Choice Based Adaptive Conjoint Analysis
 Jayadevappa R;Chhatre S;Malkowicz SB, Schwartz JS
University of Pennsylvania, Philadelphia, PA, USA


*Top 5% Average Review Score

PR3Identifying Preferences for Closed-Loop Insulin Systems Among Children with Type 1 Diabetes and Their Parents: A Discrete Choice Experiment

Kelley M1;Wadwa P2;Buckingham B3;Thankamony A4;Campbell F5;Weinzimer S6;Mauras N7;DiMeglio L8;Denvir L9;Davis N10;Ghatak A11;Besser R12, Fox DS13
1University of Southern California, Santa Monica, CA, USA, 2University of Colorado, Aurora, CO, USA, 3Stanford University, Palo Alto, CA, USA, 4University of Cambridge, Cambridge, Cambridge, UK, 5St. James University Hospital, Leeds, West Yorkshire, UK, 6Yale University, New Haven, CT, USA, 7Nemours Children’s Clinic, Jacksonville, FL, USA, 8Indiana University, Indianapolis, IN, USA, 9Nottingham Children’s Hospital, Nottingham, Nottinghamshire, UK, 10Southampton Children’s Hospital, Southampton, Hampshire, UK, 11Alder Hey Children’s Hospital, Liverpool, North West England, UK, 12Oxford University Hospitals NHS Foundation Trust, Oxford, Oxford, UK, 13University of Southern California, Los Angeles, CA, USA

*Top 5% Average Review Score

PR4Health Utility Assessment of Different Options for Treatment of Iron Deficiency Anemia: Results from Time-Trade-Off Study in China

Wu D1;Zhang Y2;Boegelund M3, Hu S4
1First Affiliated Hospital of Soochow University, Suzhou, China, 2Shanghai Institute of Technology, Shanghai, China, 3Incentive, Holte, Denmark, 4School of Public Health, Fudan University, Shanghai, China


*Top 5% Average Review Score


Reimbursement and Access Policy Studies

Moderator: Kimberly Westrich, National Pharmaceutical Council, Washington, DC, USA
RE1Performance-Based Risk-Sharing Arrangements in Diagnostics and Devices in the United States

Chen Y1, Carlson J2
1University of Washington, Seattle, WA, USA, 2University of Washington, SEATTLE, WA, USA



RE2Modeling the Impact of Drug Pricing Policies on Innovation 
 Nichols D1;Toliver J2, Wang R1
1Genentech, South San Francisco, CA, USA, 2The University of Texas at Austin, Authin, TX, USA

RE3Expectations Versus Reality: How Real-World Data Fits into Budget Impact Analyses

Willms K1;Jamet N1;Shek N2;Achrafie L1;Haddlesey A2, Bureau M2
1Amaris Consulting Inc., Toronto, ON, Canada, 2Hoffman-La Roche Limited, Mississauga, ON, Canada


RE4The Impact of Reimbursement for NON-Face-to-Face Chronic Care Management on Health Outcomes and Health Care Utilization Among Patients with Type 2 Diabetes in Louisiana 
 Shao Y1;Hong D2;Stoecker C2;Nauman E3;Bazzano A2;Kabagambe E4;Hu G5;Yoshida Y6;Fonseca V6;Katzmarzyk P7;Nigam S8;Carton T3;Gugel J6, Shi L2
1Tulane University School of Public Health and Tropical Medicine, River Ridge, LA, USA, 2Tulane University School of Public Health and Tropical Medicine, New Orleans, LA, USA, 3Louisiana Public Health Institute, New Orleans, LA, USA, 4Ochsner Health, New Orleans, LA, USA, 5The Pennington Biomedical Research Center, Baton Rouge, LA, USA, 6Tulane University School of Medicine, New Orleans, LA, USA, 7Pennington Biomedical Research Center, Baton Rouge, LA, USA, 8Blue Cross and Blue Shield of Louisiana, Baton Rouge, LA, USA


Artificial Intelligence Method Studies

Moderator: James Murray, PhD, Eli Lilly and Company, Indianapolis, IN, USA
AI1Quantifying Bias in ML-Extracted Variables for Inference in Clinical Oncology
 Lee J*1;Estevez M2;Segal BD2;Sondhi A2;Cohen AB2, Cherng ST2
1Flatiron Health, Baltimore, MD, USA, 2Flatiron Health, New York, NY, USA


AI2The Impact of Including Race and Ethnicity in Risk Prediction Models on Racial Bias
 Khor S1;Hahn EE2;Haupt EC2;Shankaran V3;Clark S1;Rodriguez P1;Chen Y1, Bansal A1
1University of Washington, Seattle, WA, USA, 2Southern California Permanente Medical Group, Pasadena, CA, USA, 3Fred Hutch, Seattle, WA, USA
AI3Disease-Free Survival (DFS) As a Surrogate Endpoint for Overall Survival (OS) in Adults with Resectable Esophageal or Gastroesophageal Junction Cancer: A Correlation Meta-Analysis


Leung L1;Kurt M2;Singh P2;Kim I2;Donnellan G1, Kanters S1
1Evidinno Outcomes Research Inc., Vancouver, BC, Canada, 2Bristol Myers Squibb, Lawrenceville, NJ, USA


AI4Comparing Machine-Learning Methods for the Prediction of Major Adverse Limb Events and Mortality after a Percutaneous Intervention
 Gressler L1;Marinac-Dabic D2;Dosreis S3;Goodney P4;Mullins CD3, Shaya FT5
1University of Maryland, Baltimore, MD, USA, 2U.S. Food and Drug Administration, Silver Spring, MD, USA, 3University of Maryland School of Pharmacy, Baltimore, MD, USA, 4Dartmouth-Hitchcock Medical Center, Lebanon, NH, USA, 5University of Maryland Baltimore, Baltimore, MD, USA


Are Value Frameworks in Latin America Fit For Purpose When Assessing COVID-19 Health Technologies?

Moderator: Homero Antonio Monsanto, PhD, Center for Observational and Real-World Evidence (CORE), Merck & Co., Inc., Cidra, PR, USA

Panelists: Claudia Brabata, MSc, Medtronic, USA, Miami, FL, USA; Diego F Guarin, MD, MPH, MA, Merck, Kenilworth, NJ, USA; Jose Y. Santiago, PhD, Abbott, USA, Des Plaines, IL, USA

ISSUE: The SARS-CoV-2 infection (COVID-19) has had profound health, social and economic effects across the world. By mid-December 2020, four countries in Latin America (Argentina, Brazil, Colombia and Mexico) had more than 1 million cases and were among the top 10 countries in the world in number of deaths due to COVID-19. Health innovations to tackle the pandemic, ranging from vaccines and antivirals to medical devices and diagnostics are advancing at incredible speed adding pressure to provide rapid and broad access to these technologies for those who need in order to manage the pandemic. Health Technology Agencies in Latin America follow explicit value frameworks to assess health technologies. These frameworks have a narrow definition of value, mainly focused in elements like health benefits (e.g. LYG, QALYs) and costs, disregarding other elements. COVID-19-related health technologies illustrate challenges and opportunities to advance the definition of what is value and which other elements should be considered based on international recommendations.

OVERVIEW: This panel will address challenges faced by decision makers to assess the value of COVID-19-related health innovations under the established value frameworks in three countries with established HTA agencies (Brazil, Colombia and Mexico). Experts from health technology producers including pharmaceuticals, medical devices, and diagnostics, will discuss the challenges and opportunities faced in the region highlighting those value elements that are not being considered yet.

Recruitment for Stated Preference Surveys. Do Respondents with Self-Reported Versus Physician-Confirmed Diagnoses Yield Similar Results?


Moderator: Michelle Tarver, MD, PhD, Center for Devices and Radiological Health, U.S. Food and Drug Administration, Silver Spring, MD, USA

Panelists: Shelby Reed, PhD, RPh, Preference Evaluation Research Group, Duke Clinical Research Institute, Durham, NC, USA; Olufemi Babalola, MHS, MSc, Center for Devices and Radiological Health, Food and Drug Administration, Silver Spring, MD, USA; Lisa Kaiser, BS, Dynata Healthcare Solutions, Warrington, PA, USA

ISSUE: Patients with a physician-confirmed diagnosis are the ideal respondents for stated-preference research in healthcare/regulatory evaluation. However, there are logistical challenges associated with recruiting patients from clinical sites. Recruitment of patients with a physician-confirmed diagnosis may be associated with longer study duration and greater cost. It has been suggested that national panels of patients provide direct access to willing patient participants which can translate to lower expenses and shorter study timeframes. It is not clear if preference results elicited from a self-reporting cohort are similar to preferences from patients recruited directly from the clinic setting. Are panels a suitable substitute for clinical enrollment to elicit preferences? The answer to this methodological question would potentially lead to greater efficiencies and may encourage the conduct of health preference studies to inform regulatory decisions.

OVERVIEW: This panel will debate the pros and cons of a utilizing a self - reporting cohort to elicit patient preferences. Dr. Tarver will moderate the panel. She will pose questions for the panelists to discuss. Some questions are: Do preference surveys administered to respondents via panels and clinically diagnosed patients deliver similar results? Are samples similar in terms of demographics, disease severity, health literacy, numeracy, and other factors? Does this influence the results? How do vendors who manage panels request information on members’ medical conditions? What are recruitment rates for panel-derived vs. physician-recruited samples? What’s the recruitment duration difference (panel vs. physician diagnosed)? What measures can be taken to optimize the reliability of a self-reported diagnosis? Dr. Reed will discuss methodological considerations in comparing preferences between samples. Olufemi Babalola will present an example from the CDRH that compares preference results from a panel (self-reported) to a clinically diagnosed cohort in the case of fibroids. Lisa Kaiser will highlight best practices around the recruitment into and management of their panel.

Impact of COVID-19 on Health Systems, Treatment, and Value

Moderator: William Padula, PhD, University of Southern California, Los Angeles, CA, USA
IN1Treatment Journey of COVID-19 Patients in Hospital Settings

Moon R1;Rosenthal N2, Brown H2
1Premier Inc., Ocoee, FL, USA, 2Premier Inc., Charlotte, NC, USA


*Top 5% Average Review Score

IN2Characteristics of Patients Diagnosed with Coronavirus Disease 2019 (COVID-19) Across the Three Waves in the US: A Claims-Based Study Using a Large National Sample
 Divino V
IQVIA, Falls Church, VA, USA

*Top 5% Average Review Score
IN3Economic Value and Health System Impact of Remdesivir in Treating Hospitalized COVID-19 Patients in the United States

Sun F1;Jeyakumar S2, Smith N2
1Gilead Sciences Inc., Foster City, CA, USA, 2Maple Health Group, LLC, New York, NY, USA


*Top 5% Average Review Score

IN4Non-Health Considerations in Economic Evaluations of COVID-19 Interventions: A Systemic Review 
 Podolsky M ;Kim D, Neumann PJ
Tufts Medical Center, Boston, MA, USA

*Top 5% Average Review Score


The Future and Evolution of Advanced Modeling Techniques to Support the Development of COVID-19 Mitigation and Prevention Policies

Moderator: Jacki Chou, MPP, MPL, PRECISIONheor, Los Angeles, CA, USA

Panelists: Pinar Karaca Mandic, PhD, Medical Industry Leadership Institute, University of Minnesota, Carlson School of Management, Minneapolis, MN, USA; Romilla Batra, MD, MBA, SCAN Health Plan, Long Beach, CA; Lance Waller, PhD, Emory University, Atlanta, GA, USA

ISSUE: The COVID-19 global pandemic is an unprecedented public health challenge requiring significant resources to evaluate the problem and determine the best solutions from a health and economic perspective. Much of the decision making related to COVID-19 have been centered around prevention, which requires forecasting. This issue panel will discuss the data and modeling needs for policy makers, health care decision makers, and the average US resident for the COVID-19 pandemic, specifically, with blue sky considerations for a non-specific future pandemic.

OVERVIEW: This panel will discuss the modeling and real-time data needs to support good decision making during the COVID-19 global pandemic. The panel will consider past challenges in data collection and forecasting, and the solutions that were used, as well as remaining challenges. The panel will also debate the role of the scientist in communicating critical data to decision makers at all levels of government and health system. Ms. Chou will moderate and provide an overview of the pandemic from a US perspective as well as a framework for thinking through the data and modeling challenges for COVID-19. Dr. Karaca Mandic will describe the challenges with real-time data collection and interpretation with the University of Minnesota COVID-19 Hospitalization Tracking Project. Dr. Singh will review advanced modeling techniques that have been or could be implemented in supporting COVID-19 decision making. Finally, Dr. Waller will describe his efforts relating to assessing inequity in spatial access to testing sites between racial and ethnic groups in Atlanta, Georgia.

Machine Learning Applications in Health

Moderator: Manuel Cossio MSGC, MS, MSAI, HE -Xperts LLC, Miami, FL, USA
ML1Comparing Mortality in Cardiac Patient Surgical Clusters with Machine Learning Clusters in the National Inpatient Sample

Gala K1;Lodaya K2;Marinaro X2;Zhang X2;Hayashida DK2;Munson S2, D'Souza F2
1Deborah Heart and Lung Center, Browns Mills, NJ, USA, 2Boston Strategic Partners, Inc., Boston, MA, USA


ML2Supervised Machine Learning Predicts Mortality in COVID-19 Patients Using Electronic Health Records
 Marinaro X*;Meng Z; Zhang X; Lodaya K; Hayashida DK; Munson S, D'Souza F
Boston Strategic Partners, Inc., Boston, MA, USA

ML3LASSO (Least Absolute Shrinkage and Selection Operator) and XGBoost (eXtreme Gradient Boosting) Models for Predicting Depression-Related Work Impairment in US Working Adults

Li V1;Costantino H1;Rowland J2;Yue L3, Gupta S1
1Kantar, New York, NY, USA, 2Kantar, Bronx, NY, USA, 3Kantar, Jersey City, NJ, USA



ML4Association of Incident Cancer with Low-Value Care Among Elderly Medicare Beneficiaries Using Machine Learning 
 Iloabuchi C1;Dwibedi N2;LeMasters T2;Ladani A3;Shen C4, Sambamoorthi U4
1West Virginia University School of Pharmacy, Robert C. Byrd Health Sciences Center, WV, USA, 2West Virginia University School of Pharmacy, Morgantown, WV, USA, 3West Virginia University Medicine, Morgantown, WV, USA, 4Penn State College of Medicine, Hershey, PA, USA



Emerging Methods and Practices in Health Economics

Moderator: Chris Pashos, BS, MPP, PhD, Genesis Research, Winchester, MA, USA
SM1The Random Effects Warfarin Days' Supply (REWARDS) Model: Development and Validation of a NOVEL Method to Estimate Exposure to Warfarin Using Administrative Data
 Salmasi S1;Höegg T1;Safari A1;De Vera M1;Lynd LD1;Koehoorn M1;Barry A1;Andrade J1, Loewen P2
1University of British Columbia, Vancouver, BC, Canada, 2University of British Columbia, Vancouver, QC, Canada

SM2The Consequences of Greater Net Price Transparency for Innovative Medicines in Europe: Insights from a Multi-Agent Simulation Model
 Van Dyck W1;Riccaboni M2;Swoboda T3, Pistollato M4
1Vlerick Business School, Gent, Belgium, 2IMT School for Advanced Studies, Lucca, Italy, 3Vlerick Business School, Hamburg, HH, Germany, 4Charles River Associates, London, LON, UK
SM3A Technique for Meta-Analysis DATA Extraction with Time-Dependent Outcomes: A Hierarchical Approach
 Hebert A1;Kreaden U2;Yankovsky A3;Massachi S4;Guo D2;Li Y2;Soito A5, Slee A6
1Intuitive Surgical, San Marcos, CA, USA, 2Intuitive Surgical, Sunnyvale, CA, USA, 3Intuitive Surgical, Palo Alto, CA, USA, 4Stratevi, Santa Monica, CA, USA, 5Soito Consulting, Oakland, CA, USA, 6New Arch Consulting, Issaquah, WA, USA
SM4The New SMC Ultra-Orphan Pathway: HTA Best Practice for Very Rare Diseases?
 Carr D1, Macaulay R2
1Precision Advisors, Kingston, UK, 2Precision Advisors, London, UK


What Can Be Gained from Payer/HTA Involvement in the Multi-Stakeholder Consensus Development of Core Outcome Sets for Late Phase Clinical Trials?

Moderator: Donna A Messner, PhD, Center for Medical Technology Policy, Baltimore, MD, USA

Panelists: Foluso O Agboola, MBBS, MPH, Institute for Clinical and Economic Review, Boston, MA, USA; Emily Tsaio, PharmD, Premera Blue Cross, Mountlake Terrace, WA, USA; Jill Morrow-Gorton, MD, MBA, University of Pittsburgh (UPMC) Health Plan, Pittsburgh, PA, USA

ISSUE: Panelists will explore the potential value of payer and health technology assessor (HTA) participation in consensus-based core outcome set (COS) development for drug clinical trials, discuss hurdles to their participation and debate the potential benefits (or drawbacks) of COS to coverage and payment decision-making.

OVERVIEW: Gene therapies and biologics promise paradigm shifts for many diseases, though at considerably higher prices. Decision makers assessing the added value of these agents for patients, in relation to their high costs, encounter major challenges: a) heterogenous outcomes hinder comparisons with traditional therapies, or b) effective new therapies impact standards of care such that traditional outcomes lose relevance. COS development may address these and other challenges by bringing together multiple stakeholder perspectives to deliberate and reach consensus on critical outcomes for decision-making. Through this lens, payers and HTA are key stakeholders in these initiatives.

How the Time-Drive Activity-Based Costing Contributes to Increasing Value in Health Care? The Example of the Brazilian Stroke Care Pathway

Discussion Leaders: Carisi Anne Polanczyk, ScD, MD, National Health Technology Assessment Institute, Porto Alegre, Brazil; Sheila Ourique Martins, PhD, MD, Hospital Moinhos de Vento, Porto Alegre, Brazil; Ana Paula Beck da Silva Etges, PhD, Eng., National Health Technology Assessment Institute, Porto Alegre, RS, Brazil 


PURPOSE: This workshop intends to share with the audience how by applying the value-based health care (VBHC) framework, the Brazilian Institute for health technology assessment (IATS) is producing contributions for the scientific community and the brazilian stroke care pathway. The Time-driven Activity-based Costing methodology is one of the gold-standards recommendations of the value-agenda. However, its application in a real-world setting is still emergent and frequently registers methodological heterogeneity. Motivated by that, the TDABC in healthcare Consortium ( was created to improve the quality of projects that apply TDABC. The community already involves members from all continents, and in Brazil is working with the national stroke network in a research to evaluate value for the stroke-care pathway. By attending this workshop, the audience will learn why the TDABC is the gold-standard recommendation in the context of VBHC, how it is possible to explore the method to identify and measure cost-saving opportunities and consequently increase value, and how TDABC in addition to the stroke ICHOM standard set is being used to evaluate and increase value for the stroke care pathway. 

DESCRIPTION: The speakers will introduce the TDABC in the context of VBHC (20 min), explain the sequence of eight steps to apply it using real examples (20 min), and present the value analysis for the Brazilian stroke care pathway. The stroke case discussion will explore how the value-analysis performed can guide waste reduction for the stroke care pathway and identify opportunities to increase the quality of care (20 min).

Preparing for a Policy Sandbox: Engaging Stakeholders to Advance the Use of RWD in HTA

Discussion Leaders: Dalia Dawoud, PhD, Science, Policy and Research Programme, National Institute for Health and Care Excellence, London, UK; Johan Pontén, MA, Dental and Pharmaceutical benefits Agency, TLV, Stockholm, Sweden; Milou Amber Hogervorst, PhD, Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht University, Utrecht, Netherlands; Ayla Lokhorst, MA, Development, Science and International Affairs, Zorginstituut Nederland (ZIN), Diemen, Netherlands 

PURPOSE: This workshop introduces the sandbox approach and explains its possible applications in health technology assessment (HTA), using methodological and policy challenges around real world data (RWD) for HTA-decisions. Attendees should walk away with insight in why it would be beneficial and how it might work to engage all stakeholders in such a complex environment to test emerging methods, policies and processes.

DESCRIPTION: In 2022, a policy sandbox will be organised in the Horizon2020 funded project HTx (Next Generation Health Technology Assessment) to test new methods to make better use of RWD. There is a growing recognition of the value of RWD in addition to efficacy data from RCTs. However, some hurdles remain in benefiting from the use of RWD. A policy sandbox is a safe environment where the outcomes of the project can be translated into HTA practice while engaging stakeholders. We have seen that it is crucial for the acceptance and uptake of any new method that stakeholders are engaged during development. Despite its potential, this is the first time that a sandbox will be applied in HTA-setting.

Milou Hogervorst will give a background on the willingness to use RWD for HTA decisions in challenging circumstances (8 min.). Secondly, Johan Pontén will elaborate on the identified challenges of using RWD in HTA (10 min.). Thirdly, Ayla Lokhorst will outline useful change models for engaging stakeholders (10 min.). Finally, Dalia Dawoud will present the concept of policy sandboxes and its use as a controlled environment to test new HTA methods (13 min.). Q&A time will be given at the end and questions will be asked on other examples/situations where it would be useful to apply the policy sandbox format (19 min.). This workshop will be very relevant for attendees from various backgrounds including academics, HTA agencies and pharmaceutical companies.

Mixed Methods Research: Using Embedded Qualitative Interviews to Enhance Interpretation of Clinical Trial Outcomes

Discussion Leaders: Miriam Kimel, PhD, Patient-Centered Research, Evidera, Bethesda, MD, USA; Naomi Knoble, PhD, Food and Drug Administration, Silver Spring, MD, USA; Carla Dias Barbosa, MSc, Patient-Centered Research, Evidera, London , LON, UK; Laurie Eliason, MPH, Value Evidence and Outcomes, Patient Centered Outomes in Oncology, GlaxoSmithKline, Upper Providence, PA, USA 

PURPOSE: Conducting embedded interviews within clinical trials is a new research paradigm that can provide a variety of benefits, including understanding treatment benefit and safety/tolerability from the patient perspective, and estimating thresholds for meaningful change. Using a mixed method approach, embedded interview data can enrich our understanding of the patients’ experience to illuminate and complement the PRO and biomedical outcomes collected in the trial. This workshop will illustrate how embedded interviews can be utilized to generate in-depth and meaningful patient input through the drug development process.

DESCRIPTION: After a brief introduction of the purpose of this type of research and value for different stakeholders, the speakers will present examples of embedded interview use within clinical trials and address resulting contributions to drug development research in each case study. 
• (10 minutes) Introduction to description, uses and value of mixed methods embedded interview research to stakeholders. (Miriam Kimel)
• (10 minutes) Overview of value-added contributions of mixed method embedded interviews to support regulatory inquiries. (Naomi Knoble)
• (10 minutes) Case study on use of mixed methods assessment of patient treatment experience, including disease changes and tolerability in oncology. (Laurie Eliason)
• (10 minutes) Case study on use of longitudinal mixed methods approach to assess treatment benefit-risk assessment in a rare disease. (Carla Dias Barbosa)

Medical Technology Studies

Moderator: Harry Smolen, Medical Decision Modeling Inc., Indianapolis, IN, USA
MT1Telehealth Utilization and Multiple Sclerosis Imaging Utilization in Four MS Centers During the COVID Pandemic: Real-World Evidence from the MS-CQI Improvement Research Collaborative
 Chen A1;Molaei M2;Vaeth A3;Walsh K1, Oliver B4
1Thomas Jefferson University, Philadelphia, PA, USA, 2Thomas Jefferson University, Conshohocken, PA, USA, 3Massachusetts General Hospital; Harvard Medical School, Charlestown, MA, USA, 4Dartmouth-Hitchcock-Health, Lebanon, NH, USA


MT2Combining Systematic Literature Reviews; What Does the Effect Size Say?

Kreaden U1;Bossie H1;Yankovsky A2, Hebert A1
1Intuitive Surgical, Sunnyvale, CA, USA, 2Intuitive Surgical, Palo Alto, CA, USA


MT3Assessing the Potential Value of Wearable Digital Health Technologies in Chronic Kidney Disease Using Early HTA Methods 
 Gc VS1;Manca A1;Casson AJ2;Antrobus S2, Iglesias CP1
1University of York, Heslington, York, UK, 2University of Manchester, Manchester, UK



MT4Safety Warnings about Power Morcellation in Hysterectomy: A Simulation of National Impact
 Xu X1;Desai VB2;Schwartz PE1;Gross CP1;Lin H3, Wright JD4
1Yale University, New Haven, CT, USA, 2CooperSurgical Inc., Trumbull, CT, USA, 3Rutgers University, Newark, NJ, USA, 4Columbia University, New York, NY, USA



Patient Behavior Studies

PB1Differences in Adherence and Healthcare Utilization between Users of Analog Vial, Analog Pen, and Human Insulin
 Shuvo S1;Gatwood J2;Tolley E1;Surbhi S1;Kaplan C3, Bailey J1
1University of Tennessee Health Science Center, Memphis, TN, USA, 2University of Tennessee Health Science Center, Nashville, TN, USA, 3University of Southern California, Los Angeles, CA, USA

*Top 5% Average Review Score

PB2Real-World Adherence to Single-Inhaler Fluticasone Furoate/Umeclidinium/Vilanterol Versus Multiple-Inhaler Triple Therapy Among Asthma Patients in the US
 Averell C1;Germain G2;Laliberte F3;MacKnight S2;Jung Y2;Duh MS4, Abbott C5
1GlaxoSmithKline, Research Triangle Park, NC, USA, 2Groupe d’analyse, Ltée, Montréal, QC, Canada, 3Groupe d’analyse, Ltée, Montreal, QC, Canada, 4Analysis Group, Inc., Boston, MA, USA, 5GlaxoSmithKline, Research Triangle Park, Durham, NC, USA


*Top 5% Average Review Score

PB3Real World Adherence, Persistence, Relapse and Multiple Sclerosis Symptoms Among Patients Treated with Oral Disease-Modifying Therapies

Leist T1;Le H2;Verma S3;Cole M4;Afolabi M3;Tsai WL3, Keenan A5
1Jefferson University, Philadelphia, PA, USA, 2Janssen Scientific Affairs, LLC, Titusville, NJ, USA, 3STATinMED Research, Inc., Plano, TX, USA, 4Janssen Global Services, LLC, Southampton, PA, USA, 5Janssen Global Services, LLC, Titusville, NJ, USA

*Top 5% Average Review Score

PB4Patient Perspectives on Implementation of a Long-Acting Injectable Antiretroviral Therapy Regimen in HIV US Healthcare Settings: Final Month 12 Results from the CUSTOMIZE Study
 Flamm J1;Garris C2;D'Amico R3;Dalessandro M4;McHorney CA5;Mansukhani SG6;Benson P7;Thedinger B8;Salazar D9;Tanda N10;Fricker J11, Czarnogorski M3
1Kaiser Permanente, Sacramento, CA, USA, 2ViiV Healthcare, RTP, NC, USA, 3ViiV Healthcare, Research Triangle Park, NC, USA, 4ViiV Healthcare, Collegeville, PA, USA, 5Evidera, Bethesda, MD, USA, 6Evidera, Waltham, MA, USA, 7Be Well Medical Center, Berkley, MI, USA, 8KC CARE Health Center, Kansas City, MO, USA, 9University of Florida, Jacksonville, FL, USA, 10GlaxoSmithKline, Uxbridge, UK, 11GlaxoSmithKline, Brentford, UK


*Top 5% Average Review Score


Real World Data & Information Systems

Moderator: Pamela Blumberg, MPH, DrPH, TriNetX, Cambridge, SC, USA
RW1A Comparative Analysis of Recommendations for the Post-Reimbursement Collection of Real-World DATA (RWD) in Oncology Appraisals Issued By Six HTA Agencies

Gurjar K1;Harricharan S1;Nguyen K1, Forsythe A2
1Purple Squirrel Economics, Montreal, QC, Canada, 2Purple Squirrel Economics, New York, NY, USA


*Top 5% Average Review Score

RW2Real-World Adherence to Single-Inhaler Fluticasone Furoate/Umeclidinium/Vilanterol Versus Multiple-Inhaler Triple Therapy Among Asthma Patients in the US
 Wong W1;To TM1;Li M2;Lee W3;Veenstra D4, Garrison LP4
1Genentech, Inc., South San Francisco, CA, USA, 2The University of Texas MD Anderson Cancer Center, Houston, TX, USA, 3University of Washington, Pasadena, CA, USA, 4University of Washington, Seattle, WA, USA"

*Top 5% Average Review Score

RW3Quantitative Bias Analysis (QBA) for Comparative Effectiveness of Alectinib versus Ceritinib in Non-Small Cell Lung Cancer (NSCLC) 

Wilkinson S1;Gupta A2;Scheuer N3;Mackay E2;Arora P2;Thorlund K4;Wasiak R5;Ray J6, Ramagopalan S7

1Roche, Welwyn Garden City, UK, 2Cytel, Toronto, ON, Canada, 3Roche Products Limited, Welwyn Garden City, UK, 4McMaster University, Hamilton, ON, Canada, 5Cytel, London, UK, 6F. Hoffmann-La Roche, Basel, Switzerland, 7F. Hoffmann-La Roche, Basel, BS, Switzerland

*Top 5% Average Review Score

RW4Use of Diabetes, COPD and Asthma Medications Among Different Health Insurance Plan Levels
 Chen C1, Roberts M2
1University of New Mexico College of Pharmacy, Albuquerque, NM, USA, 2University of New Mexico College of Pharmacy, ALBUQUERQUE, NM, USA


*Top 5% Average Review Score


Mixed Diseases and Condition Studies

Moderator: Nana Numapau, Boehringer Ingelheim, Chandler, AZ, USA
SC1Increases in the Share of Spending on Pharmaceuticals Met with Decrease in Overall Spending for Elderly Americans with Diabetes
 Cohen B1, Tysinger B2
1Monument Analytics, Los Angeles, CA, USA, 2Sol Price School of Public Policy, University of Southern California, Los Angeles, CA, USA


SC2Trends in Diagnostic Prevalence, Treatment Patterns, and Healthcare Resource Utilization of Pediatric Food Allergy Patients in the United States, 2008-2018
 Mills K1;Nowak C2;Choong C2;Shan M2;Hoyt M2, Hunter T2
1Eli Lilly and Company, Ramsey, MN, USA, 2Eli Lilly and Company, Indianapolis, IN, USA


SC3The Drivers of Health State Utilities in Duchenne Muscular Dystrophy (DMD): A Study Using the Health Utilities Index Mark 3 (HUI3)
 Rogula B1;Filipovic Audhya I2;Szabo S3;Feeny D4;Bolatova T3, Gooch KL2
1Broadstreet Health Economics & Outcomes Research, Vancouver, Canada, 2Sarepta Therapeutics, Cambridge, MA, USA, 3Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada, 4McMaster University, Hamilton, BC, Canada


SC4The Clinical and Economic Impacts of Pharmacist-Initiated Pre-Exposure Prophylaxis for Men Who Have Sex with Men in the United States: A Cost-Utility Analysis
 Tran J1, Zimmermann M2
1University of Washington, Seattle, WA, USA, 2Institute for Disease Modeling, Bellevue, WA, USA



Value Assessment of Biosimilars: Challenges and Considerations

Discussion Leaders: Steven Simoens, MSc, PhD, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, VBR, Belgium; Evelien Moorkens, MSc PhD, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium; Dalia Dawoud, PhD, Science, Policy and Research Programme, National Institute for Health and Care Excellence, London, UK; Delphine Courmier, PhD, MBA, Global Health Economics, AMGEN, Thousand Oaks, CA, USA 

PURPOSE: To provide guidance to and raise awareness of workshop participants regarding methodological issues surrounding the value assessment of biosimilars

DESCRIPTION: The use of biosimilars faces significant gaps and challenges in terms of their health technology assessment, which have not yet received sufficient attention in the health economic literature and in guidelines issued by many health technology assessment agencies.
This workshop will explain the following methodological issues in the value assessment of biosimilars:

  1. The choice of the appropriate technique of economic evaluation (cost-minimization or other) and of the appropriate comparator.
  2. The approach to filling the clinical evidence gap when the marketing authorization of the biosimilar has been granted on the basis of extrapolation (and no phase 3 clinical trial data are available).
  3. The approach to value assessment of biosimilars in treatment naïve patients and in patients previously treated with biologics/biosimilars.
  4. Whether value assessment of biosimilars should account for the potential ‘nocebo’ effect and how.
  5. The management of uncertainty and barriers to uptake related to biosimilars and the role of managed entry agreements.
  6. The valuation of value-added services. 
  7. The societal valuation of expanding access to treatment in terms of capturing QALY gains at population level.

We Have the Technology: Building Better Comparative Effectiveness Methods for the Future

Discussion Leaders: Alan Brnabic, MS, Eli Lilly and Company, Indianapolis, IN, USA; David Madigan, PhD, Columbia University, New York, NY, USA; Anthony Zagar, PhD, Eli Lilly and Company, Indianapolis, IN, USA; Xiang Zhang, PhD, CSL Behring, Philadelphia, PA, USA 

PURPOSE: To present recently proposed methods for comparative effectiveness including the application of machine learning through frequentist model averaging (fMA) and doubly robust methods. Along with novel methods for estimation, we will discuss advances in evaluating the robustness of analyses to the potential for unmeasured confounding.

DESCRIPTION: Evidence from real world data (RWE) is increasingly becoming a vital component of decision health care decision making. However, limitations inherent with causal inference are challenging. One challenge is the need for selecting the ‘right’ statistical models both for treatment selection and outcome analysis. For treatment selection models, propensity scores estimated from logistic regression are the gold standard. However, new approaches using tree-based methods, balancing algorithms, and double scores have been proposed. Penalized regression has become popular for building outcome models and doubly robust methods bring the promise of more robust analyses. There is no consensus on the best approach and simulations suggest there is no single best method for all scenarios. Speakers will cover the above issues as well as future directions of including machine learning for causal inference using targeted maximum likelihood methods (TLME) and fMA. With fMA researchers specify many potential methods and models into the analysis and cross validation determines the better approaches in a data driven fashion. 
Another challenge in comparative effectiveness is addressing bias due to unmeasured confounding. In recent years a suite of methodologies has emerged ranging from methods quantifying the robustness to confounding to methods using external data to adjust the estimated causal effect. The speakers will facilitate an understanding of unmeasured confounding options by presenting different statistical procedures within a unified framework. Tools for selecting and implementing, along with simulations and real data examples will be discussed.

Value Assessment Gives Us Estimates – But Is It Answering the Right Questions?

Moderator: Samuel Nussbaum, MD, Innovation and Value Initiative, Alexandria, VA, USA

Panelists: Steven Pearson, MD, MSc, Institute for Clinical and Economic Review, Boston, MA, USA; Annie Kennedy, BS, EveryLife Foundation for Rare Diseases, Washington, DC, USA; Bruce Sherman, MD, FCCP, FACOEM, North Carolina Business Group on Health, Greensboro, NC, USA

ISSUE: In the US, value assessment is rapidly becoming a cornerstone of decision making within contracting and payment negotiation, even as debate continues about whether we have the right tools and methods to support the increasing demand for quantifiable estimates of value. These value assessments give us numbers – but are they answering all of the right questions? And if not, what additional elements and frameworks are needed?

OVERVIEW: This panel will explore the current capabilities and limitations of value assessment methods, consider the most important unmet needs in discerning high and low-value care, and debate the best way forward to answering questions that matter to all stakeholders. Samuel Nussbaum will moderate and provide insight on payers’ needs when making value-driven decisions. Steven Pearson will provide the value assessor’s perspective and describe current real-world use. Annie Kennedy will provide the patient community perspective on the alignment between value assessments and value delivered to patients. Bruce Sherman will provide insights from the perspective of the employer community. Panelists will provide brief remarks, reserving the majority of the session for panel discussion and audience questions.

Should We Be Talking "Embedded" Rather Than "Pragmatic" For Real-World Clinical Trials?

Moderator: Moderator: Nirosha Lederer, PhD, Aetion Inc, Boston, MA, USA

Panelists: David Thompson, PhD, Real World & Late Phase, Syneos Health, Boston, MA, USA; Lindsay Kehoe, MS, CGC, Clinical Trials Transformation Initiative, Durham, NC, USA; Lou Fiore, MD, VA System, Boston, MA, USA

ISSUE: Since first being described more than 50 years ago, the pragmatic clinical trial (PCT) has been considered the most scientifically rigorous of all real-world research designs. Yet, despite its promise, the PCT design has been relatively underutilized in comparison to other approaches for real-world evidence (RWE) generation, such as prospective observational research and retrospective studies of electronic health records (EHRs) and administrative claims databases. More recently, a variety of organizations have focused attention on the potential to embed clinical trials in real-world practice settings as a means of involving practicing physicians, recruiting real-world patients, and leveraging existing EHR systems for data capture. Is this approach better suited to RWE generation? Should we be talking “embedded” rather than “pragmatic” for real-world clinical trials?

OVERVIEW: In its RWE Program Framework, the US Food & Drug Administration highlighted the benefits of integrating clinical trials into real-world practice settings, indicating that real-world data systems can be leveraged in this fashion to generate regulatory-grade evidence. Other organizations, including the NIH Collaboratory, the Clinical Trials Transformation Initiative (CTTI) and the Australian Clinical Trials Alliance, have workstreams devoted to advancing these objectives. Nirosha Lederer, the panel moderator, will set the stage for the discussion by outlining the key questions, including: What are the defining characteristics of embedded clinical trials? In what ways are embedded trials similar or different from pragmatic trials? Is embedding clinical trials in real-world practice a more practical way of improving their generalizability? David Thompson will highlight similarities and differences between embedded and pragmatic trials. Lindsay Kehoe will describe CTTI’s progress to date to identify practical issues associated with embedded clinical trials. Lou Fiori will provide an overview of case studies of embedded clinical trials undertaken in the US Veterans Administration system. The moderator will solicit audience interaction and feedback on the alternative viewpoints.

Can Performance-Based Risk Sharing Arrangements (PBRSAS) for Medtech Address Procurement and Market Access Challenges? Scanning the Current Horizon and a View to the Future.

Discussion Leaders: Richard Charter, MSc, MedTech Market Access Europe & Asia Pacific, Alira Health, Basel, Switzerland; Mark Sculpher, PhD, Centre for Health Economics, University of York, York, YOR, UK; Giuditta Callea, PhD, SDA Bocconi School of Management, Rome, Italy; Payam Abrishami, PhD, Dutch National Health Care Institute, Diemen, Netherlands 


PURPOSE: This panel seeks to discuss performance-based risk-sharing agreements (PBRSA) for medical technologies (MedTech); to identify whether taxonomies of PBRSAs for pharmaceuticals are applicable for MedTech; and, through discussion, develop a nascent set of best practices for device-specific PBRSA. 
Mark Sculpher will present a taxonomy based on the the nature of the decisions taken by health systems and the type of evidence needed to support these. Susan Garfield, a consultant active in the area of PBRSA, will bring the industry perspective and provide insights the challenges of planning, execution, and evolution of these plans, and Payam Abrashimi will serve as a representative of a payer perspective into how PBRSA can address the needs of payers and procurement bodies; Richard Charter will moderate and provide an overview of PBRSA.

DESCRIPTION: Procurement bodies have increasingly sought rigorous evidence before accepting premium price products. Recent pandemic challenges have accelerated these trends of more cautious evaluation of MedTech prior to procurement. Yet, regulatory agencies in many instances allow for the commercialization of devices without rigorous trials providing the evidence sought by procurement. In the absence of such data, evidence development through performance-based risk-sharing agreements (PBRSA) have recently generated increased interest, development, and emergent use. 

This panel seeks to discuss recent years’ growing interest in PBRSA for MedTech and the roles, opportunities, and challenges within this landscape of procurement bodies, governments, health technology assessment (HTA) entities, and payers. Topics for discussion include how to deliver PBRSA, what is the role of health economics and outcomes research in reducing the clinical and economic uncertainty, and the role of real-world evidence in risk-share agreements.
Each speaker will speak for approximately 15 minutes. The audience is encouraged to bring their experience to the discussion; open questions highlighted during the panel will be tested with audience polling.

Oncology Studies

Moderator: Ramiro E. Gilardino, MD, MHS, MSc, HE-Xperts Consulting LLC, Miami, FL, USA
CN1Patterns of Utilization and Implementation Experiences of Oncology Monoclonal Antibodies (MABS) Biosimilars in the U.S.: From BOTH Payers and Healthcare Professionals (HCPS) Perspectives
 Yang J1;Blinzler K2;Lankin J2;Maculaitis M3, Shelbaya A4
1Pfizer Inc, New York, NY, USA, 2Kantar, New York, NY, USA, 3Kantar Health, Red Bank, NJ, USA, 4Pfizer Inc.; Columbia University Mailman School of Public Health, Heliopolis, Egypt

*Top 5% Average Review Score

CN2Eliciting Unreported Subgroup-Specific Survival from Aggregate Randomized Controlled Trial Data

Alagoz O1;Xiao H2;Singh P2;Gricar J2;Dixon M2, Kurt M2
1University of Wisconsin-Madison, Madison, WI, USA, 2Bristol Myers Squibb, Lawrenceville, NJ, USA


*Top 5% Average Review Score

CN3It's All in the Family: Microsimulation Modelling of Genetic Testing 

Petelin L1;Cunich M2;Procopio P3;Nickson C3;Campbell I4, Trainer AH4
1Peter MacCallum Cancer Centre & The University of Melbourne, Brunswick west, VIC, Australia, 2University of Sydney, Sydney, NSW, Australia, 3Cancer Council NSW & The University of Melbourne, Melbourne, VIC, Australia, 4Peter MacCallum Cancer Centre & The University of Melbourne, Parkville, VIC, Australia

*Top 5% Average Review Score

CN4Patient-Reported Outcomes from the Phase 3, Randomized Study of Acalabrutinib with or without Obinutuzumab Versus Chlorambucil PLUS Obinutuzumab for Treatment-Naïve Chronic Lymphocytic Leukemia (ELEVATE-TN)
 Walker P1;Sharman JP2;Jurczak W3;Munir T4;Banerji V5;Coutre S6;Woyach J7;Salles G8;Wierda WG9;Patel P10;Wang MH10;Emeribe U11;Flood E11;Byrd JC7, Ghia P12
1Peninsula Health, and Peninsula Private Hospital, LANGWARRIN, VIC, Australia, 2Willamette Valley Cancer Institute/US Oncology, Eugene, OR, USA, 3Maria Sklodowska-Curie National Research Institute of Oncology, Kraków, Poland, 4St James's Hospital, Leeds, UK, 5Max Rady Faculty of Health Sciences, College of Medicine at the University of Manitoba, Winnipeg, MB, Canada, 6Stanford University School of Medicine, Stanford, CA, USA, 7The Ohio State University Comprehensive Cancer Center and Division of Hematology, Columbus, OH, USA, 8Hospices Civils de Lyon, Centre Hospitalier Lyon Sud, Service d’Hématologie Clinique, Pierre-Bénite, France, 9The University of Texas MD Anderson Cancer Center, Houston, TX, USA, 10Acerta Pharma, South San Francisco, CA, USA, 11AstraZeneca, Gaithersburg, MD, USA, 12Università Vita-Salute San Raffaele and IRCCS Ospedale San Raffaele, Milano, Italy

*Top 5% Average Review Score


Caregiver Considerations in Health Economics

Moderator: Julia Slejko, PhD, University of Maryland School of Pharmacy, Baltimore, MD, USA
ST1Caregiver-Perceived Behavioral Challenges in Fragile X Syndrome (FXS) and Their Measurement Using the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS)

Merikle E1;Patel V2;Tich N3;Sebree T3;Hagerman R4, Palumbo J3
1Covance by Labcorp, Gaithersburg, MD, USA, 2Covance by Labcorp, Chicago, IL, USA, 3Zynerba Pharmaceuticals, Devon, PA, USA, 4UC Davis Health System, Sacramento, CA, USA

*Top 5% Average Review Score

ST2Exit Interviews with Caregivers and Clinicians Confirm Content Validity and Feasibility of Completing the Pediatric Respiratory Syncytial VIRUS (RSV) Electronic Severity and Outcome Rating System (PRESORS) for Pediatric RSV Clinical Studies

Tatlock S1;Arbuckle R2;Grimes R3;Santucci-Hutcheson E4;Sinha R5;Witek J5, Scott J6
1Adelphi Values Ltd, Cheshire, CHE, UK, 2Adelphi Values Ltd, Bollington, UK, 3Adelphi Values Ltd, Bollington, Cheshire, UK, 4Janssen Research and Development, Beerse, Belgium, 5Janssen Research and Development, Titusville, NJ, USA, 6Janssen Global Services, LLC, High Wycombe, UK

*Top 5% Average Review Score

ST3Development of a Survey-Based Predictive Model for Clinical Dementia Rating in Alzheimer's Disease

Yu JC1;Hlavka J2;Tysinger B2, Lakdawalla D3
1School of Pharmacy, University of Southern California, Los Angeles, CA, USA, 2Sol Price School of Public Policy, University of Southern California, Los Angeles, CA, USA, 3School of Pharmacy and Sol Price School of Public Policy, University of Southern California, Los Angeles, CA, USA

*Top 5% Average Review Score

ST4Psychometric Evaluation of the Pediatric RSV Severity and Outcomes Rating System Version 6 (PRESORSV6), Caregiver Observer-Reported [ObsRO] and Clinician-Reported [ClinRO] Questionnaires in Children Aged 1 to 36 Months Hospitalized with Respiratory Syncytial VIRUS (RSV) Infection
 Fennema H
Janssen Pharmaceutical Research and Development, Beerse, Belgium
*Top 5% Average Review Score


Economic Evaluation Applications: Burden and Value of Therapies

Moderator: Ramiro E. Gilardino, MD, MHS, MSc, HE-Xperts Consulting LLC, Miami, FL, USA
ED1Antimicrobial Resistance in US Hospitals: Burden and VALUE of Investment in Developing New Treatments
 Iacobucci W1;Siego CV1;Dall T1;Wallin Bernhardsson N2;Ulrich J3, Dougherty JS3
1IHS Markit, Washington, DC, USA, 2IHS Markit, London, UK, 3PhRMA, Washington, DC, USA

*Top 5% Average Review Score

ED2Elements of Value for Gene Therapy: A Systematic Review
 Kim E1;Raimundo K2;Marcum ZA1, Veenstra DL1
1University of Washington, Seattle, WA, USA, 2Genentech, South San Francisco, CA, USA
*Top 5% Average Review Score
ED3Societal Burden of Dementia-Related Psychosis in the US: A Cost of Illness Analysis

Rajagopalan K1;Rashid N2;Abler V2, Shah A3

1An-L-It-Iks Inc, Framingham, MA, USA, 2ACADIA Pharmaceuticals Inc., San Diego, CA, USA, 3An-L-It-Iks Inc, Somerville , MA, USA
*Top 5% Average Review Score
ED4Economic Burden of Attention-Deficit/Hyperactivity Disorder (ADHD) Among Children and Adolescents in the United States (US): A Societal Perspective
 Adler LA1;Childress A2;Cloutier M3;Gagnon-Sanschagrin P3;Davidson M3;Kinkead F3;Guerin A3;Lefebvre P4, Schein J5
1NYU Grossman School of Medicine, New York, NY, USA, 2Center for Psychiatry and Behavioral Medicine, Las Vegas, NV, USA, 3Analysis Group, Inc., Montreal, QC, Canada, 4Analysis Group, Inc., Montréal, QC, Canada, 5Otsuka Pharmaceutical Development & Commercialization, Inc., Princeton, NJ, USA

*Top 5% Average Review Score


Addressing Health Inequity in The U.S.: How Can Value Assessment (VA) Contribute to More Equitable Healthcare Resource Allocation?

Discussion Leaders: Susan dosReis, PhD, Center for Patient-Driven Values in Healthcare Evaluation, University of Maryland School of Pharmacy, Baltimore, MD, USA; Louis P. Garrison, PhD, The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington, Seattle, WA, USA; Stacey Kowal, MSc, Health Policy and Systems Research, Genentech, Alameda, CA, USA; Richard Xie, PhD, The Innovation and Value Initiative, Newton, MA, USA 

PURPOSE: The purpose of this workshop is to explore challenges in addressing health inequity as an objective in value assessment (VA), and to demonstrate and discuss the applications of potential methods in the US context using real-world model examples. 

DESCRIPTION: There is system-wide consensus and emphasis on the importance of tackling the widening health disparities in our society. Various promising modeling approaches, most notably the distributional cost-effectiveness analysis (DCEA), have been proposed to support more equitable resource allocation in health care. However, the extent to which these methods can be applied in the pluralistic, fragmented US healthcare system is unclear. 

In this workshop, the speakers will first discuss concepts and measures of health equity, and key challenges in addressing equitable resource allocation using existing methods (~10 minutes). To tackle these challenges, ensuring data representations from the underserved subpopulations is the necessary first step. Using the IVI’s model focusing addressing major depressive disorder (IVI-MDD) as an example, researchers will share strategies on how to recruit individuals of color and lower socioeconomic status in a component patient preference survey (~10 minutes). Discussions on available modeling will include: (1) how to build a Medicaid perspective in a VA model (~10 minutes), and (2) how to leverage US data to conduct DCEA through a case study on the impact of funding inpatient COVID-19 treatments on underlying health equality. (~10 minutes).
Insights from the workshop are applicable across all stakeholders: researchers will gain insight into available sources and methods for US equity assessment; innovators and patient groups can learn about how to encourage the traditionally underrepresented communities to participate in the data generation and collection process for health data; and payers and US HTA bodies can learn about data availability and suggested next steps for integration of equity assessment into decision-making.

Adaptive Reimbursement: Innovating for Patient Access and Better Outcomes for All

Discussion Leaders: Mark Trusheim, MS, BS, MIT Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, MA, USA; Angela Banks, MBA, MA, UnitedHealth Group, Washington, DC, USA; Ron Potts, MD, Kaiser Permanente, Portland, OR, USA; David Strutton, PhD, MPH, Center for Observational and Real-World Evidence, Merck, Philadelphia, PA, USA 

PURPOSE: Based on MIT NEWDIGS LEAPS Project experience, inspire participants to explore Adaptive Reimbursement opportunities to accelerate adoption of RWE-based patient regimen optimization to simultaneously improve quality-of-life and economic outcomes.

DESCRIPTION: Scientific, clinical development and regulatory advancements are fueling unprecedented biopharmaceutical innovation from gene therapies to immunotherapies. These upstream innovations have not been matched by patient-centered downstream innovations that employ payment and other incentive systems to implement patient specific regimen improvements informed by leveraging real-world evidence.

Adaptive reimbursement combines downstream innovations that:
(1) Creatively cover and finance therapies to ensure timely, appropriate access, enhance healthcare system sustainability and reward upstream innovation; and
(2) Reward optimized treatment regimens for each patient through the generation of real-world evidence that reduces uncertainties regarding biopharmaceutical product performance.
This Panel will explore current Adaptive Reimbursement examples from the MIT NEWDIGS LEAPS project, discuss developing future innovations, and describe approaches to overcome the significant implementation challenges. Hear how:
• Durable cell and gene therapy precision financing models could apply to other therapeutic areas
• Adaptive reimbursement mechanisms might fund RWE production platforms (indirectly or directly) by creating financial demand for their results
• Data needed to administer adaptive reimbursement contracts can also be used to de-risk market access, identify unmet medical needs, and monitor patient benefits from therapies
• RWE platforms could fuel the design and implementation of innovative adaptive reimbursement models.

Modeling and Simulations for Healthcare Decision Maker: Battling COVID-19 and Other Biomedical Challenges

Discussion Leaders: Jacki Chou, MPP, MPL, PRECISIONheor, Los Angeles, CA, USA; Bhramar Mukherjee, PhD, University of Michigan, Ann Arbor, Ann Arbor, MI, USA; Zhilan Feng, PhD, Purdue University, West Lafayette, IN, USA; Anuj Mubayi, PhD, PRECISIONheor, Los Angeles, CA, USA 

PURPOSE: This workshop will focus on advanced modeling tools and how they might be used to inform healthcare decision making, including COVID-19 intervention polices as a recent example. Workshop participants will understand the foundational constructs of simulation and modeling techniques and the reliance of these models on the quality of the data used to inform inputs and assumptions. Participants of this workshop will be able to understand the basics for key types of selected statistical and mathematical models as well as what types of questions these models are suited to answer in health evaluations.

DESCRIPTION: Health economic evaluation is used to support decision makers in areas including public health, epidemiology, infectious diseases and pharmacology. This workshop aims to provide some background and overview on cutting edge modeling and simulation methods to address continuously changing healthcare problems and data. Ms. Jacquelyn Chou will provide an introduction and overview for the workshop, as well as basic principles and objectives for simulations and models (5 minutes). Dr. Bhramar Mukherjee will provide an overview of the basics for statistical models alongside key considerations and limitations when there is delay or underreporting, as in the case of COVID-19. (15 minutes). Dr. Zhilan Feng will walk-through the fundamentals and considerations for key types of mathematical dynamic models including complex agent based models and infectious disease transmission models (15 minutes). Finally, Dr. Mubayi will review the applications of these models for health economics via methods such as decision trees and microsimulation models. He and Ms. Chou will also facilitate an interactive session where participants can help sort research questions into the type of model and data that is best suited to answer that question via polling, as well as submit their own research questions to be sorted into model types by fellow participants via polling (30 minutes).

Comparative Effectiveness Studies

Moderator: Douglas Faries, PhD, Eli Lilly and Company, Indianapolis, IN, USA
CE1A Modeled Health Outcomes Evaluation of Darolutamide Plus Androgen Deprivation Therapy for High-Risk NON-Metastatic Castration-Resistant Prostate Cancer in China
 Ming J1;Liu Y1;Lu W1;Wu Y1;Li W1;Han R2, Hu S3
1IQVIA, Shanghai, China, 2Bayer Healthcare Company Ltd., Beijing, China, 3School of Public Health, Fudan University, Shanghai, China

*Top 5% Average Review Score

CE2Effect of Different Variance Estimation Methods with Inverse Probability Treatment Weights (IPTW) on Comparative Effectiveness Measure in Multiple Sclerosis
 Earla J1;Hutton GJ2;Thornton JD3;Chen H4;Johnson ML4, Aparasu RR4
1Incyte Corporation, Chadds Ford, PA, USA, 2Baylor College of Medicine Medical Center, McNair Campus, Houston, TX, USA, 3University of Houston, The Prescription Drug Misuse Education and Research (PREMIER) Center, Houston, TX, USA, 4University of Houston, Houston, TX, USA

*Top 5% Average Review Score

CE3Systematic Review and Indirect Comparison of PD-(L)1 Inhibitors in Combination with Platinum-Based Doublet Chemotherapy (PT-DC) for the First-Line Treatment of Non-Squamous, Non-Small-Cell Lung Cancer (nsqNSCLC)

Zhang L1;Qian Y2;Li J2;Cui C2;Chen L2;Qu S3, Lu S4
1Eli Lilly and Company, Shanghai, 31, China, 2Eli Lilly and Company, Shanghai, China, 3Real World Solutions, IQVIA, Shanghai, China, 4Shanghai Chest Hospital, Shanghai, China

CE4Expanding Evidence Base VS Introducing Heterogeneity in Networks for Network Meta-Analyses: A Simulation Study
Luttenauer H1;Le Nouveau P2, Gauthier A3
1Amaris Consulting, London, UK, 2Amaris Consulting, Levallois-Perret, ON, France, 3Amaris Consulting, Barcelona, Spain

*Top 5% Average Review Score


Economic Evaluation in Oncology Studies

Moderator: Lisa Hess, PhD, Eli Lilly and Company, Indianapolis, IN, USA
EC1A Cost-Effectiveness Analysis of Nivolumab plus Ipilimumab plus Two Cycles of Platinum Doublet Chemotherapy Versus Platinum Doublet Chemotherapy in the First-Line Treatment of Stage IV or Recurrent Non-Small Cell Lung Cancer in the United States
 Polyzoi M1;Sandhu H2;Maervoet J2;Yuan Y3;Chaudhary M3;Varol N4;Lee A4;Dale P2;Jones C5;Lubinga SJ3, Penrod JR3
1PAREXEL International, Strängnäs, D, Sweden, 2Parexel International, London, ESS, UK, 3Bristol-Myers Squibb, Princeton, NJ, USA, 4Bristol-Myers Squibb, Uxbridge, Middlesex, UK, 5PAREXEL International, Stockholm, Sweden

*Top 5% Average Review Score

EC2Modeling Approaches to Estimate Realized Real Option Value of Ipilimumab in Metastatic Melanoma
 Lee W1;Li M2;Wong W3;To TM3;Garrison LP1, Veenstra D1
1University of Washington, Seattle, WA, USA, 2The University of Texas MD Anderson Cancer Center, Houston, TX, USA, 3Genentech, Inc., South San Francisco, CA, USA
*Top 5% Average Review Score
EC3Economic Burden of Cardiovascular Events in Patients with Chronic Lymphocytic Leukemia Treated with Novel Agents

Malangone-Monaco E1;Ryan K2;Marchlewicz E1;Lo J2, Huntington S3
1IBM Watson Health, Cambridge, MA, USA, 2AstraZeneca, Gaithersburg, MD, USA, 3Yale University School of Medicine, New Haven, CT, USA
*Top 5% Average Review Score
EC4Real-World Healthcare Costs in Patients with Classical Hodgkin Lymphoma Treated with Pembrolizumab and Nivolumab in the United States
 Laliberte F1;Raut M2;Germain G3;Desai K4;Nahar A5;Yang X6;MacKnight S3, Duh MS7
1Groupe d’analyse, Ltée, Montreal, QC, Canada, 2Merck & Co., Inc., Raritan, NJ, USA, 3Groupe d’analyse, Ltée, Montréal, QC, Canada, 4Merck & Co., Inc., Cranbury, NJ, USA, 5Merck & Co., Inc., Kenilworth, NJ, USA, 6Merck & Co., Inc., Rahway, NJ, USA, 7Analysis Group, Inc., Boston, MA, USA

*Top 5% Average Review Score


Health Technology Assessment Studies

Moderator: R. Brett McQueen, MA, PhD, University of Colorado, Skaggs School of Pharmacy and Pharmaceutical Sciences, Denver, CO, USA
HT1Effects of Stakeholder Engagement with Icer on Cost-Effectiveness of New Interventions: Lessons Learned from a Critical Review of Evaluations from 2018 to 2019
 Ronquest N1;Paret K2;Gould I3;Barnett CL4, Mladsi D5
1RTI Heatlh Solutions, Durham, NC, USA, 2RTI Heatlh Solutions, Research Triangle Park, NC, USA, 3RTI Health Solutions, Durham, NC, USA, 4RTI Health Solutions, Research Triangle Park, NC, USA, 5RTI Health Solutions, Waltham, NC, USA

*Top 5% Average Review Score

HT2Evaluation of Tucatinib for HER2-Positive Breast Cancer Patients with Brain Metastases: A United States-Based Cost-Effectiveness Analysis
 Dong L1;Nian D2;Huang Y2;Lin S2;Zhong L3, Xu X2
1Fujian Medical University, Fuzhou, China, 2First Affiliated Hospital of Fujian Medical University, Fuzhou, China, 3Texas A&M University, College Station, TX, USA
*Top 5% Average Review Score
HT3What is Value? A Systematic Review of Value Assessment Frameworks
 Zhang M1;Bao Y2;Lang Y3;Fu S4;Kimber M1;Levine M5, Xie F1
1McMaster University, Hamilton, ON, Canada, 2Gansu Provincial Hospital, Lanzhou, China, 3Dalian Medical University, Dalian, China, 4China Pharmaceutical University, Nanjing, China, 5McMaster University, Ancaster, ON, Canada
*Top 5% Average Review Score
HT4Patient-Relevance of Endpoints Other Than Overall Survival (Non-OS Endpoints) in Oncology Health Technology Assessments by the Federal Joint Committee (G-BA) in Germany
 Couybes N1;Agashe V2;Kulp W3, Ward J4
1AstraZeneca, Hamburg, Germany, 2Xcenda UK Ltd., Aldwych, UK, 3Xcenda GmbH, Hannover, Germany, 4AstraZeneca, Cambridge, UK

*Top 5% Average Review Score


Emerging Methods in Economic Evaluation

Moderator: R. Brett McQueen, MA, PhD, University of Colorado, Skaggs School of Pharmacy and Pharmaceutical Sciences, Denver, CO, USA
EM1When Are Breakthrough Therapies Cost-Effective?
 Olchanski N1;Lin PJ1;Yeh D2;Kowal S3, Cohen JT1
1Tufts Medical Center, Boston, MA, USA, 2Genentech, South San Francisco, CA, USA, 3Genentech, Alameda, CA, USA

*Top 5% Average Review Score

EM2Economic IMPACT of Compassionate Use of Medicines
Jommi C1;Pantellini F2;Stagi L2, Cavazza M1
1Bocconi University, Milano, Italy, 2Roche Spa, Monza (MI), Italy
*Top 5% Average Review Score
EM3Robustness of External Control Arm: WHEN to Use Them
 Ladouceur M1;Colby C2;Okala S3, Yue B4
1Evidera, Verdun, QC, Canada, 2Evidera, Oakland, CA, USA, 3Evidera, London, UK, 4Evidera, Orlando, FL, USA
*Top 5% Average Review Score
EM4Recommendations for Handling Uncertainty in Economic Evaluation: A Targeted Review of Pharmacoeconomic Guidelines
 Berdunov V1;Sammon C1, Ramagopalan S2
1PHMR Ltd, London, UK, 2F. Hoffmann-La Roche, Basel, BS, Switzerland

*Top 5% Average Review Score


Hospital and Clinical Practice Studies

Moderator: Laura Pizzi, MPH, PharmD, PhD, Rutgers University, Piscataway, NJ, USA
HP1Validated Models for Pre-Test Probability of Stable Coronary Artery Disease: A Systematic Review Suggesting How to Improve Validation Procedures
 Bodini A1;Mincarone P2;Tumolo MR2;Vozzi F3;Rocchiccioli S4;Pelosi G3;Caselli C4;Sabina S5, Leo CG6
1National Research Council - Institute for Applied Mathematics and Information Technologies “Enrico Magenes”, Milano, Italy, 2National Research Council - Institute for Research on Population and Social Policies, Brindisi, Italy, 3National Research Council - Institute of Clinical Physiology, Pisa, Italy, 4National Research Council - Institute of Clinical Physiology, Pisa, PI, Italy, 5National Research Council - Institute of Clinical Physiology, Lecce, Italy, 6National Research Council - Institute of Clinical Physiology, Lecce, LE, Italy"

*Top 5% Average Review Score

HP2Patient-Provider Cost Discussions and Out-of-Pocket Costs Among Cancer Survivors with Varying Levels of Cancer-Related Financial Hardships

Zhang H1;Zhang S;Chen S;Barner JC, Moczygemba L
The University of Texas at Austin, Austin, TX, USA"

*Top 5% Average Review Score

HP3Dental Opioid Prescribing Trends in the United States, 2012-2019: A Time Series Analysis
 Yan C, Lee TA, Hubbard CC, Calip GS, Sharp LK, Evans CT, Rowan S, McGregor JC, Campbell A, Gellad W, and Suda KJ
Does Medicaid Expansion Provide Affordability of Healthcare Service for Asthma Population
 Shi L, Luck J
Oregon State University, Corvallis, OR, USA
*Top 5% Average Review Score


Improving the Odds of a Successful Contract Research Project – Results from a Sponsor-Vendor Collaboration

Moderator: Bill Marder, PhD, IBM Watson Health, Winchester, MA, USA

Panelists: Ami R Buikema, MPH, Health Economics and Outcomes Research, Optum, Eden Prairie, MN, USA; David L. Van Brunt, PhD, MS, BA, Evidence and Analytics, Health Economics and Outcomes Research, AbbVie, Chicago, IL, USA; Khalid Kamal Kamal, M.Pharm., Ph.D., School of Pharmacy, West Virginia University, Morgantown, WV, USA

ISSUE:The pharmaceutical industry funds thousands of research projects each year. Most projects run smoothly but problems do arise. This panel will identify a set of common issues and recommend some best practices for successful engagements.

OVERVIEW: In 2020, the ISPOR Institutional Council (IC) undertook a study to better understand sponsor-vendor interactions. A qualitative survey of IC members was conducted, results of which were utilized to develop a quantitative survey questionnaire. The questionnaire was completed by 158 ISPOR members who were either sponsors or vendors and involved in contract research. Four major issues were identified: 1) managing changes to scope and time, 2) achieving the quality expected for the project, 3) prompt/ timely reply as issues arise, and 4) stability of staffing/leadership (and managing turnover).