Sun 28 Sep
8:00 - 12:00
Health Technology Assessment for Rare Diseases
Session Type: Short Course
Level: Intermediate
Separate registration required.
This short course is designed for professionals with a basic understanding of health technology assessment (HTA) who seek to deepen their knowledge and skills in evaluating interventions for rare diseases. Participants will gain an appreciation of the unique challenges that rare diseases pose for HTA due to limited evidence, small patient populations, and high treatment costs. A case study from Europe will be shared. The session will close with a discussion on the methodological challenges of HTA in rare disease.
Speakers
-
Bruce Crawford, MA, MPH
Vista Health Japan KK, Tokyo, Japan
-
Josh Byrnes, BEc, PhD
Griffith University, Brisbane, Australia
Introduction to Applied Generative AI for HEOR
Session Type: Short Course
Level: Introductory
Separate registration required.
The rapid advancement in generative artificial intelligence (Gen AI) presents an opportunity for transformative potential in the field of Health Economics and Outcomes Research (HEOR). This course provides an introductory understanding of generative AI models with a particular focus on large language models (LLMs), which are revolutionizing the field of HEOR. Participants will be provided with an overview of the most appropriate ways to access LLMs, going beyond the use of chatbots. Further, they will be given insights into how to use prompt engineering to conduct scientific research and gain an understanding on issues pertaining to privacy and security when using Gen AI for HEOR. Participants will further explore specific applications of these models for conducting robust scientific HEOR research in systematic literature review (SLR), real-world evidence analysis, and economic evaluation. The course aims to equip participants with the knowledge to begin to use generative AI techniques for specific HEOR contexts and to appreciate how these innovative approaches can enhance HEOR activities. Practical exercises using Python and relevant AI frameworks will be incorporated for participants to follow along. Participants who wish to gain hands-on experience are required to bring their laptops with Python installed.
PREREQUISITES: Students should have a general understanding of common HEOR concepts such as SLRs and cost-effectiveness models. Knowledge of Python or similar programming languages such as R is considered a benefit, but not required.
Speakers
-
William Rawlinson, MPhysPhil
Estima Scientific, London, United Kingdom
Will is a senior health economist at Estima Scientific holding a degree in Physics and Philosophy from the University of Oxford. Will has 4 years’ experience developing cost-utility models and has specialized in applications of generative AI to health economic modelling. Will has published on the automation of R modelling using large language models (LLMs), and more recently has focused on applications of LLMs to Excel modelling and model reporting.
-
Tim Reason, MSc
Estima Scientific, London, United Kingdom
Tim Reason is co-founder of Estima Scientific and specializes in AI and evidence synthesis, having spent 15 years in the field of HEOR and technology. Tim is managing director of Estima, driving business activities, innovation and strategy for the company. Tim’s specializes in the intersection of HEOR, software development and AI to drive better outcomes for patients. Tim is the lead author on 2 seminal papers in AI for HEOR, showing that AI can be used to automate health economic modelling and NMA.
13:00 - 17:00
Causal Inference and Causal Estimands From Target Trial Emulations Using Evidence From Real-World Observational Studies and Clinical Trials
Session Type: Short Course
Level: Advanced
Separate registration required.
In recent years, real-world evidence (RWE) has been increasingly used to inform regulatory, payer, and health technology assessment (HTA) decisions, as well as clinical guideline development. In addition, it has been recognized that the analysis of hypothetical estimands in clinical trials is necessary when the standard intention-to-treat (ITT) analysis does not answer the decision problem, usually because of treatment switching. An innovative framework for causal inference methods, target trial emulation, causal estimands and causal modeling guides the design and analysis of observational studies and clinical trials. This course will (1) introduce causal principles, causal diagrams (directed acyclic graphs; DAGs), and target trial emulation to avoid self-inflicted biases (eg, time-zero bias, immortal time bias), (2) provide an overview of causal methods for baseline confounding (multivariate regression, propensity scores) and time-varying confounding (eg, g-formula, marginal structural models with inverse probability of treatment weighting, and rank-preserving structural failure-time models with g-estimation), (3) propose appropriate estimands to ensure decision problems are directly addressed when analyzing observational data or data from clinical trials affected by treatment switching, (4) present lessons learned from applied case examples in HTA, such as single arm-trials with external control arms or trials affected by treatment switching, (5) provide recommendations regarding the use of causal inference methods and estimands and their application in causal modeling, and (6) discuss acceptance and barriers from an HTA agency perspective in Asia and globally. The target audience includes all stakeholders and researchers from all fields in health and healthcare.
PREREQUISITE: Students are expected to have a basic knowledge in epidemiologic studies and methods (including the concept of confounding).
Speakers
-
Uwe Siebert, MPH, MSc, ScD, MD
UMIT TIROL - University for Health Sciences and Technology; Harvard Chan School of Public Health, Hall in Tirol, Austria
Uwe Siebert, MD, MPH, MSc, ScD is a physician by training and professor of Public Health, Medical Decision Making & Health Technology Assessment, chair of the Dept. of Public Health, Health Services Research and HTA, and director of HTADS Continuing Education (www.htads.org) at UMIT TIROL, and adjunct professor of Epidemiology and Health Policy & Management at the Harvard Chan School of Public Health, Boston, MA, USA. He is currently a member of several Boards of Directors, European Commission Expert Groups, and he advises several HTA/government agencies in different countries. In the past, he was a member of the ISPOR Board of Directors, ISPOR Annual Meeting chair, and president of the Society for Medical Decision Making (SMDM).
His research interests include applying evidence-based quantitative and translational methods from health economics, health services & outcomes research, public health, epidemiology, artificial intelligence, modeling, causal inference from real-world data, and health data & decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, public health policies and patient guidance. He teaches courses for these topics at several universities and for industry in Europe, USA, South America, and Asia. He has worked with several HTA Agencies in Europe, Brazil, US and Canada and he advises public and government agencies, academic institutions, and industry regarding the conduction of HTAs and their impact on policy and reimbursement decisions. He has authored more than 400 publications (> 30,000 citations, H index > 80) including HTA reports, textbook chapters, scientific articles, policy briefs and editorials, and is editor of the European Journal of Epidemiology and editorial board member of several scientific journals.
-
Nicholas Latimer
SCHARR, University of Sheffield, Sheffield, United Kingdom
-
Felicitas Kuehne, MSc
Pfizer Pharma GmbH, Wanzleben-Börde, Germany
Critical Steps to Construct a Budget Impact Analysis
Session Type: Short Course
Level: Introductory
Separate registration required.
This introductory course will describe the methods and critical steps involved to estimate the health insurance reimbursement budget impact when a novel healthcare technology will be incorporated into the reimbursement list. The course will present 6 critical steps for estimating budget impact: (1) estimating the target population; (2) selecting a time horizon; (3) identifying current and projected treatment mix; (4) estimating current and future drug costs; (5) estimating change in disease-related costs; and (6) estimating and presenting changes in annual budget impact and health outcomes. Along the 6 steps, critical scenario analysis planning will also be illustrated. Both static and dynamic methods for estimating the budget and health impact of adding a new drug to a health plan formulary will be presented. These critical steps will be illustrated using actual budget impact models. This course is designed for those with some experience with pharmacoeconomic analysis.
Speakers
-
Jianwei Xuan, PhD
China Southern Medical University, School of Pharmaceutical Sciences, Guangzhou, China
-
C. Daniel Mullins, PhD
University of Maryland School of Medicine, Baltimore, MD, United States
C. Daniel Mullins is a Professor at the University of Maryland School of Pharmacy. He is Founder and Executive Director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, a community-academic partnership for patient-driven research. Dr. Mullins has received approximately $25 million in funding as a Principal Investigator from AHRQ, FDA, NCI, NHLBI, NIA, NIMHD, the Patient-Centered Outcomes Research Institute (PCORI) and various patient advocacy organizations and pharmaceutical companies. At the University of Maryland Baltimore (UMB), he received the Dr. Patricia Sokolove Outstanding Mentor Award and the Dr. Martin Luther King Jr. Faculty Diversity Award. He was named Researcher of the Year at UMB and was awarded a University System of Maryland Wilson H. Elkins Professorship. At ISPOR, he has served as Editor-in-Chief of Value in Health since 2010 and received the Marilyn Dix Smith Leadership Award in 2017 and the Avedis Donabedian Lifetime Achievement Award in 2024.
Mon 29 Sep
8:00 - 9:15
Realizing RWE Potential in Asia Pacific: Opportunities, Challenges, and Progress
Session Type: Plenary
This plenary session examines the evolving landscape of Real-World Evidence (RWE) across the Asia Pacific region, where adoption varies significantly between countries despite growing interest. Speakers will explore how RWD/RWE is increasingly supporting decision-making in life sciences, regulatory approvals, and reimbursement. The session will highlight regional efforts while addressing critical challenges including data availability, data quality, interoperability, privacy concerns, and potential biases. Attendees will gain insights into how RWE can play a role in decision-making process, and discover practical lessons for infrastructure development and capacity building specific to Asia Pacific healthcare environments.
9:15 - 9:45
Networking Break
Session Type: General Meeting
9:45 - 10:45
Harnessing RWD for Evidence-Based HTA in Asia-Pacific: Data Challenges, Evolving Paradigms and Empirical Applications
Session Type: Workshop
Topics: Health & Insurance Records Systems
Level: Intermediate
PURPOSE: The quality, integrity, and methodological rigor of real-world data (RWD) are essential for generating real-world evidence (RWE) to support health technology assessment (HTA). Data sources such as electronic medical records, claims databases, health surveys, and patient registries provide valuable insights for healthcare stakeholders. However, regional disparities in RWD implementation can weaken outcome reliability and impede the transfer of evidence across regions. This workshop examines evolving paradigms, applications and challenges of RWD in China, Japan, and Australia. By offering regional insights, it aims to optimize RWD integration into HTA frameworks and provide global perspectives to support evidence-based decision-making.
DESCRIPTION: Attendees will gain an understanding of RWD use in HTA, with emphasis on data quality, integration, and regional practices. The session includes: a) the generation, quality, and accessibility of RWD in China, Japan, and Australia, and its role in healthcare decision-making; b) methodological approaches to ensure RWE meets HTA evidence standards; c) case studies on how RWD informs market access and reimbursement. Prof. Gordon G. Liu will chair the session, introducing the RWD-driven HTA landscape (10 min). Prof. Jing Wu will discuss RWD and RWE use in China, focusing on data quality and HTA applications (12 min). Prof. Isao Kamae will share the current status, challenges, and opportunities for RWD and RWE in Japan (12 min). Dr. Feili Zhao will present how RWD supports product development, evidence generation, and market access in Australia from the industry perspective (12 min). A 14-minute audience interaction will feature structured discussion and problem-solving based on an RWD-driven HTA scenario. The session will offer key insights into regional RWD ecosystems and inform evidence-based HTA decision-making.
Speakers
-
Isao Kamae, DrPH, MD
The University of Tokyo, Tokyo, Japan
-
Feili Zhao, PharmD, PhD
Global Value, Access & Pricing, BeiGene, Sydney, Australia
Translating Real-World Evidence Into Policy: A Comparative Analysis of Adoption Mechanisms in China and the US Drug Price Negotiations
Session Type: Issue Panel
Topics: Health & Insurance Records Systems
Level: Intermediate
ISSUE: Addressing the escalating costs of pharmaceuticals is critical to ensuring the sustainability of healthcare financing systems worldwide. The vast and growing pool of real-world data offers a valuable opportunity to generate real-world evidence (RWE), complementing the limitations of randomized controlled trials and informing evidence-based decision-making. In China and the U.S.—the world’s two largest pharmaceutical markets—national payers have engaged in drug price negotiations with manufacturers, with preliminary steps toward incorporating RWE. However, the effective integration of RWE into price negotiations and broader drug policy decisions remains an area requiring deeper exploration. This session will examine the application of RWE in drug price negotiations in both China and the U.S., addressing key questions such as: What types of RWE should be submitted? How can RWE be evaluated? And how can it be leveraged to support pricing and reimbursement decisions?
OVERVIEW: The session will commence with an overview of RWE's growing importance in comprehensive drug value assessment presented by Dr. Wen Chen (10 minutes). Dr. Jifang Zhou will then provide an overview of China's evolving RWE ecosystem, including current data infrastructure and analytical capabilities. This will be followed by Dr. Luying Zhang's presentation of case studies demonstrating RWE's actual application in China's national reimbursement drug list and price negotiations. Dr. Lizheng Shi will offer the U.S. perspective, focusing on Medicare's experience with RWE in coverage and price determinations. The session will culminate in a panel discussion synthesizing cross-country experiences to identify: (1) the most impactful RWE categories for pricing decisions; (2) robust methodologies for RWE generation and validation; and (3) practical strategies for integrating RWE into policy-making processes. These insights will provide actionable guidance for healthcare systems seeking to balance drug affordability with sustainable innovation.
Speaker
-
Lizheng Shi
Tulane University, New Orleans, LA, United States
Fit for Purpose Data in Value Assessment in Japan: How Best to Leverage Real-World Evidence (RWE) and Qualitative Value Elements to Optimize Decision Analysis
Session Type: Issue Panel
Topics: Data Protection, Integrity, & Quality Assurance
Level: Intermediate
ISSUE: Value assessment is a key component of decision-making on newly innovative medicines in Japan with considerable potential impact on the healthcare ecosystem. Innovative technologies are quickly evolving to better treat complex and targeted conditions, resulting in the generation of new types of clinical trial designs, including non-RCT designs, and evidence with novel or intermediate endpoints. For example, in oncology and rare diseases, including regenerative medicines, it is not always possible or ethical to conduct randomized controlled trials (RCT). Therefore, we need to rely on evidence from non-traditional trial designs, novel endpoints, and evaluative methods such as indirect treatment comparisons (ITCs) and real-world evidence to inform value assessment. How then, should these data and methods be used and balanced with qualitative decision-analysis in the Japan HTA system to ensure value recognition and thus reduce the drug lag and loss of new medicines to realize optimized innovative care for patients?
OVERVIEW: This panel will critically evaluate how RWE should be appropriately used in the value assessment of innovative medicines, leveraging the Japanese system as a specific example. Dr. Demia will begin the conversation by describing the aims of the Japan HTA approach, and how it currently utilizes RWE and novel evidence to evaluate innovative medicines where there are limited RCT data. Christoph Glaetzer will then provide the developer’s perspective on the R&D process, the considerations that drive decision-making for clinical trial design and approaches to evidence generation. Finally, Dr. Igarashi will provide an HTA perspective on how best to use RWE and HTA evaluation methods to support clinical evidence assessment in Japan. This group will debate pragmatic approaches to achieving an optimized format for future HTA regarding healthcare sustainability for rapidly aging population.
Moderator
Speakers
-
Christoph Glaetzer, Dipl. Kfm.
Johnson & Johnson Innovative Medicine, New Brunswick, NJ, United States
-
Sven Demiya, MBA, MSc, PhD
IQVIA, Minato-ku, Japan
-
Ataru Igarashi, PhD
The University of Tokyo, Tokyo, Japan
10:45 - 11:15
Networking Break
Session Type: General Meeting
11:15 - 12:15
HTA System in Japan: Progress and Issues
Session Type: Other Breakout Session
Topics: Health & Insurance Records Systems
Level: Introductory
PURPOSE: The objective of this session is to share the progress and current issues of the Japanese new Health Technology Assessment (HTA) system, which started in 2019 from the perspectives of the government, academia and industry. DESCRIPTION: The new HTA system, which is the cost-effectiveness evaluation of pharmaceuticals and medical devices, officially started in 2019. During 6 years, over 40 products ware assessed, and some of which resulted in reimbursement price adjustment. In this session, after the brief introduction of the system by the moderator (5 min.), Dr. Shiroiwa will introduce the background, progress and current issues of the system from the perspective of the government and public assessor (15 min.). Dr. Ikeda will make a presentation about the issues and future possible directions from the academic perspective (15 min.). Finally, xxx will provide the views about the system from industry perspective (15 min.). The session will be concluded by questions and discussions between audience and panel members (10 min.). This session may benefit policymakers and HTA agencies, as well as academia and industry.
Moderator
-
Takashi Fukuda, PhD
National Institute of Public Health, Wako-shi, Japan
Speakers
-
Takeru Shiroiwa, MPH, PhD
C2H, National Institute of Public Health, Saitama, Japan
-
Shunya Ikeda, MSc, PhD, MD
International University of Health and Welfare, Narita, Japan
Equity-Informative Real-World Evidence: Learning From Global Lessons and Exploring Future Initiatives
Session Type: Workshop
Topics: Data Protection, Integrity, & Quality Assurance
Level: Introductory
PURPOSE: Health equity is one of the most pressing issues facing the HEOR community. Despite widespread recognition of the need for disaggregated data to monitor health equity, most real-world data remain aggregated, creating enormous challenges in assessing the reality of inequity. This workshop will showcase equity-informative real-world evidence (RWE) generation in diverse contexts to assess inequalities by subgroup, share lessons relevant to policy makers, practitioners and researchers from the Asia-Pacific region and beyond, and explore future directions.
DESCRIPTION: Workshop attendees will obtain a working knowledge of current methods together with relevant evidence of the equity-informative RWE in Asia-Pacific and beyond. This workshop will review: A) the landscape of equity-informative real-world data in diverse contexts; B) the state of equity research and analysis using real-world data (e.g., evidence, challenges and opportunities); and C) future research agendas for promoting equity-informative RWE. Dr Shimamoto will moderate the session and introduce the topic by outlining the global agenda on equity-informative RWE (10 min). Prof Doran will present equity-informative RWE from the UK and discuss recent advances in evidence generation and policy decision making (10 min). Dr He will present the social deprivation index and the stratified Quality-Adjusted Life Expectancy (QALE) in China (10 min). Lastly, Mr. Ota will share his experience of equity analysis from pharmaceutical perspectives in Japan (10 min). Audience participation will include an interactive polling session eliciting participants' views on equity-informative RWE (5 min), followed by a moderated Q&A session (10-15 min). This interactive and informative workshop will be valuable to researchers, industry analysts, policy analysts and clinicians who are interested in RWE for advancing a health equity focus in HEOR.
Moderator
-
Kyoko Shimamoto, PhD
Keio University, Tokyo, Japan
Speakers
-
Tim Doran, PhD
University of York, York, United Kingdom
-
Riku Ota, MBA, MSPH
Novo Nordisk, Brøndby, Denmark
As a Global Payer Evidence Lead at Novo Nordisk A/S in site Soborg, Denmark, Riku is dedicated to driving the generation and dissemination of clinical and economic evidence with a specific focus on global markets and liver therapeutic areas. His responsibilities are geared towards securing patient access and enhancing value treating liver diseases in diverse and dynamic markets worldwide.
His academic background includes a Master's in Public Health from King's College London and Global MBA from IE Business School, providing him with a robust foundation in global health, business administration, and management. Over the years, his expertise in real-world evidence, health economic outcome research, and project management has been meticulously honed through diverse roles in both the consulting and pharmaceutical sectors.
Riku's professional ethos is rooted in an unwavering passion for health equity, driving my endeavor to harness my expertise in public health and health economics to enhance health outcomes and bridge the gaps in healthcare accessibility for patients across the globe. This commitment not only anchors my career path but also propels me forward, fueling my aspiration to effect tangible, meaningful change in the healthcare landscape from a holistic and inclusive perspective.
Harnessing Big Data, AI, and Real-World Evidence to Transform Regulatory and Health Technology Assessment in APAC
Session Type: Issue Panel
Topics: Data Protection, Integrity, & Quality Assurance
Level: Intermediate
ISSUE: Real-World Evidence (RWE), derived from sources outside traditional clinical trials, is transforming healthcare decision-making in the Asia-Pacific region. RWE accelerates regulatory approvals and optimizes reimbursement decisions, drawing from electronic health records, claims data, and patient-generated information. Despite growing recognition, significant barriers hinder the full utilization of big data and artificial intelligence in developing robust evidence across APAC countries. Fragmented healthcare systems with inconsistent data standards impede cross-border RWE utilization. Regulatory frameworks vary substantially, with South Korea implementing parallel review processes while China pilots early-access programs, complicating multi-country evidence strategies. Stakeholders often remain skeptical of RWE compared to randomized controlled trials, despite increasing cost-effectiveness demands. The integration of artificial intelligence with RWE offers opportunities to overcome these barriers through advanced analytics and automated data validation. This convergence aligns with ISPOR's consideration of establishing a global health economics institution to support international organizations.
OVERVIEW: This panel will explore how APAC countries are leveraging RWE or AI-enhanced RWE for regulatory and reimbursement decisions. We will 1) Share case studies of successful RWE implementations, 2) Identify remaining challenges in RWE adoption, 3) Propose frameworks aligning with ISPOR's vision of a globally integrated health economics community. By examining RWE implementation in Health Technology Assessment (HTA), we aim to contribute to developing standardized methodologies that reflect Asia's unique healthcare contexts. This will advance evidence-based decision-making in the region and support ISPOR's initiatives in global health economics.
Speakers
-
Eun Young Bae, MSc, PhD
School of Pharmacy, Gyong-Sang National University, Seoul, Korea
-
Kelvin Bryan Tan, PhD
Policy Research and Evaluation Division, Ministry of Health, Singapore, Singapore
12:15 - 13:15
Lunch
Session Type: General Meeting
13:15 - 14:15
Informing Health Policy Decisions Through Meta-Analysis of Economic Evaluations: Why and How to Do It? How to Connect with Users?
Session Type: Other Breakout Session
Topics: Reproducibility & Replicability
Level: Introductory
PURPOSE: The objectives of this session are to introduce the concept and methodology of meta-analysis of economic evaluations (MAEE), to demonstrate the application of MAEE using a relevant case study, to discuss existing gaps and methodological challenges, and to engage the audience in a discussion on how the proposed MAEE method can improve future healthcare decision-making by addressing existing gaps and challenges.
DESCRIPTION: Evidence from economic evaluation studies (EEs) is crucial for informing health policy decisions. Meta-analysing EEs (MAEE) can summarize the evidence quantitatively, and may support clear policy recommendations, facilitating decision-making especially in resource-limited countries where context-specific EEs are unavailable. A novel method for MAEE has been recently developed and applied in several therapeutic areas. However, data preparation for MAEE can be challenging due to inconsistent reporting of parameters and multiple sources of heterogeneity in EEs.
This session will start with an introduction to the overall concept. A step-by-step process for extracting data from EEs and preparing it for meta-analysis will be discussed (15 minutes, Nathorn). In the next section, a case study of GLP1-RA and obesity will be presented demonstrating how MAEE can help policymakers make informed decisions (10 minutes, Teerapon). This will be followed by a discussion of existing gaps and challenges, as well as proposed future mechanisms to address them, as endorsed by the recent Immunization and Vaccine-related Implementation Research Advisory Committee (March 2024) (10 minutes, Sajesh). The session will conclude with audience participation to identify their perceptions, the demand for its application, and any additional challenges, along with proposed mechanisms to address them (25 minutes, Nathorn). This session may offer value to stakeholders seeking to generate economic evidence that can inform decision-making in the absence of context-specific data.
Moderator
-
Nathorn Chaiyakunapruk, PharmD, PhD
University of Utah, Salt Lake City, UT, United States
Dr.Chaiyakunapruk is a Professor in the College of Pharmacy at The University of Utah. His expertise is in Health Technology Assessment and Health Policy. He has applied health economics, real world data analysis, and evidence synthesis to support national and global policy, especially his contributions to the World Health Organization and Centers for Disease Control. His current research works have been focused on health equity, health economics, and advanced methodology in evidence synthesis. He is a co-author of CHEERS 2022 (Consolidated Health Economic Evaluation Reporting Standards Statement) and WHO guide for standardization of economic evaluations of immunization programmes, 2nd edition. He was a co-founder of the ISPOR Asia Consortium, an adviser of National Essential Drug List Selection Subcommittee of Thailand and a member of Health Economic Working Group of Benefit Package Selection Committee of National Health Security Office Thailand. Dr.Chaiyakunapruk has an H-index of 62 and is an author of more than 300 international publications in peer-reviewed journals.
Speakers
-
Nathorn Chaiyakunapruk, PharmD, PhD
University of Utah, Salt Lake City, UT, United States
Dr.Chaiyakunapruk is a Professor in the College of Pharmacy at The University of Utah. His expertise is in Health Technology Assessment and Health Policy. He has applied health economics, real world data analysis, and evidence synthesis to support national and global policy, especially his contributions to the World Health Organization and Centers for Disease Control. His current research works have been focused on health equity, health economics, and advanced methodology in evidence synthesis. He is a co-author of CHEERS 2022 (Consolidated Health Economic Evaluation Reporting Standards Statement) and WHO guide for standardization of economic evaluations of immunization programmes, 2nd edition. He was a co-founder of the ISPOR Asia Consortium, an adviser of National Essential Drug List Selection Subcommittee of Thailand and a member of Health Economic Working Group of Benefit Package Selection Committee of National Health Security Office Thailand. Dr.Chaiyakunapruk has an H-index of 62 and is an author of more than 300 international publications in peer-reviewed journals.
-
Sajesh K Veettil, PhD
University of Utah, Salt Lake City, UT, United States
-
Teerapon Dhippayom, PharmD, PhD
Naresuan University, Muang Phitsanulok, Thailand
Learning From the COVID-19 Pandemic and Designing a New HEOR Research Model: What is the Role of Real-World Evidence and How Does It Work?
Session Type: Other Breakout Session
Topics: Health & Insurance Records Systems
Level: Intermediate
PURPOSE: The world has experienced the COVID-19 pandemic since 2020. However, still the risk of a new pandemic exists. Hence, it is necessary to learn from the lessons of the past and design new HEOR research. This session will present an original model for infection control and economic evaluation, showing examples from Japan and Korea, and deepen participants' knowledge of the role and application of real-world evidence (RWE). DESCRIPTION: Attendees will gain practical knowledge of RWE utilization for HEOR studies of non-pharmaceutical intervention such as diagnostic testing. The session will: a) explore the processes of viral transmission, regarding epidemiological multiple factors such as virus virulence, test accuracy, vaccinations, etc. and show the formula to estimate the number of infected cases with multiple variables of RWE; b) using the formula, develop a model for cost-effectiveness analysis; c) demonstrate case studies on how RWE informs policymakers of value-based strategies for pandemic control. Dr. Kobayashi will chair the session, introducing the landscape of roles and challenges of RWE (10 min). Prof. Isao Kamae will present the methods for pandemic-control modelling based on RWE, illustrating how to transform an analytic model to the formula in which multiple variables of RWE are integrated. Also, he presents a simple prototype of the cost-effectiveness analysis with the Japanese case (20 min). Prof. Jeonghoon Ahn will present and discuss the Korean case with the same model, but different RWEs in Korea (15 min). The audience interactive session (15 min) will make the audience understand a new approach to pandemic HEOR study and learn how to apply it for their own country through an exercise by EXCEL sheet. This session will offer critical insights into the importance of regional RWE availability and enhance the capability of coping with the next pandemic in the world.
Moderator
-
Makoto Kobayashi, PhD
CRECON MEDICAL ASSESSMENT INC., Tokyo, Japan
Speakers
-
Isao Kamae, DrPH, MD
The University of Tokyo, Tokyo, Japan
-
Jeonghoon Ahn, MA, PhD
Department of Health Convergence, Ewha Womans University, Seoul, Korea
Nordic-Asia Pacific Collaboration in Real-World Evidence Studies: Lessons Learnt and Opportunities for Future Research Networks
Session Type: Workshop
Topics: Distributed Data & Research Networks
Level: Intermediate
PURPOSE: Real-world evidence (RWE) is increasingly used by regulators in shaping healthcare decisions, offering valuable insights into medication safety and treatment effectiveness across diverse populations. The Nordic countries, with their comprehensive nationwide registries, and the Asia-Pacific region, with its rapidly expanding healthcare data infrastructure, present unique opportunities for collaboration in generating robust RWE. This session is designed for researchers, regulators, and policymakers interested in leveraging international cooperation to strengthen RWE. Attendees will gain practical insights, establish new connections, and contribute to shaping future research directions in this rapidly evolving field.
DESCRIPTION: This workshop presents existing and potential future Nordic-Asia Pacific collaborations, focusing on how real-world data can be leveraged to address key research questions in perinatal and pediatric health. We will reflect on key lessons from existing collaborations, including data harmonization and methodological innovations. Case studies will highlight successful navigated cross-border research partnerships, demonstrating how diverse healthcare systems and population-based registries have contributed to clinically relevant, high-impact findings. An interactive session will encourage attendees to explore innovative ways to enhance inter-continent partnerships, strengthen institutional ties, and align research priorities between the two regions. Dr van Gelder will start with a brief introduction (5 min). Professor Hsiao will present real-world data sources in the Asia-Pacific region (15 min). Professor Nordeng will follow with RWE activities in Nordic countries and examples of multinational studies in Norway and Asia (15 min). Dr Trinh will present data harmonization efforts to facilitate multinational studies with a focus on OMOP common data model and perinatal expansion tables (10 min). The workshop will be concluded by an interactive session (15 min).
Moderator
-
Marleen van Gelder
Nijmegen, Netherlands
Speakers
-
Fei-Yuan Sharon Hsiao, PhD
College of Medicine, National Taiwan University, Taipei, Taiwan
-
Hedvig Nordeng
University of Oslo, Norway
Hedvig Nordeng is Professor and Head of the Pharmacoepidemiology and Drug Safety Research Group at the Department of Pharmacy, University of Oslo. She has over 15 years of experience in pharmacoepidemiological research using Norwegian and Nordic health registry data, with a focus on vulnerable populations such as pregnant individuals and children. She has led multiple interdisciplinary and international projects and has developed an analytical pipeline at UiO for rapid response to pharmacovigilance signals and is a partner in European Medicines Agency (EMA)s federated Data Analysis and Real World Interrogation Network (DARWIN EU). Professor Nordeng is editor of a clinical pharmacology textbook and author of over 200 scientific publications, many centered on perinatal drug safety. Since 2018, she has served as an EU commission-appointed expert on the PRAC committee at the EMA. She is a Fellow of ISPE and, since 2024, holds an adjunct professorship in Clinical Pharmacology at the University of Southern Denmark.
-
Nhung Trinh
University of Oslo, Oslo, Norway
14:15 - 15:15
Poster Session
Session Type: General Meeting
15:15 - 16:15
How to Collect PROs in RW Studies and Leverage Them for Robust Evidence Generation? ISPOR Good Practices Task Force on Patient Reported Outcomes (PROs) in Prospective Real-World Studies: Emerging Recommendations & Next Steps
Session Type: Workshop
Topics: Data Protection, Integrity, & Quality Assurance
Level: Intermediate
PURPOSE: Real-world evidence (RWE) and patient-centred research, as captured through patient-reported outcomes (PROs), are critical to the ISPOR community. Prospective real-world (RW) studies offer an opportunity to collect PRO data, providing valuable insights into effectiveness, safety, and tolerability from the patient perspective. Global regulators, health technology assessors, and policymakers recognize the importance of PROs in RWE; however, there is limited guidance on their optimal use in RW studies. An ISPOR Task Force is developing a good practices report to address this gap. A key component of this effort is gathering multi-stakeholder perspectives on the value, use, and acceptability of RW-PRO data across different geographies. This session aims to consolidate feedback from ISPOR members on the draft report and explore how RW-PRO data can meet stakeholder needs. Additionally, we seek recommendations on ensuring the report’s relevance across regions and identifying next steps for further research and guidance.
DESCRIPTION: This session will present the task force’s findings on maximizing the value of PROs in RW studies. Key emerging recommendations include: working with patients as partners, defining appropriate study population, ensuring PRO instrument suitability in RW settings, maintaining data quality, implementing PROs effectively, analyzing PRO data, leveraging technology, and engaging with regulators and payers. Audience input will be sought on these good practices, helping refine the recommendations for RW-PRO data collection and use. The session will conclude by identifying evidence gaps and areas for further research to enhance the integration of PROs in RWE. Designed for researchers, payers, regulators, technology assessors, and industry professionals, this session provides a unique opportunity to discuss the evolving landscape of PROs in RW studies and their role in patient-centred evidence generation.
Moderator
-
Konrad Maruszczyk, MSc
University of Birmingham, UK, Birmingham, United Kingdom
Speakers
-
Jessica Roydhouse, PhD
Menzies Institute for Medical Research, University of Tasmania, Hobart, Australia
-
Antony Martin, BSc, MSc, PhD
Health Economics & Real World Evidence, QC Medica, Liverpool, United Kingdom
Use of Real-World Evidence in Medical Device HTA Evaluations: Lessons Learned From Australia and Japan
Session Type: Other Breakout Session
Topics: Health & Insurance Records Systems
Level: Intermediate
PURPOSE: The objectives of this session are to introduce the audience to how health technology assessment (HTA) bodies in incorporate real-world evidence (RWE) into their evaluation of medical devices and discuss lessons learned in leveraging RWE in HTA and market access in Australia and Japan. DESCRIPTION: RWE is becoming increasingly important for reimbursement and market access. However, globally, HTA bodies vary in their required levels of evidence and their willingness to consider and standards they use to evaluate RWE in their decision-making process. This session will discuss lessons learned from the Australian and Japanese approaches and highlight the case study of the Micra leadless pacemaker, a medical device technology that was recently evaluated by both country HTA bodies and was the first medical device technology to be evaluated through Japan’s new cost-effectiveness evaluation schema. Dr. Strachan will begin with a global overview of RWE use and acceptance in HTA (10 min). Dr. Igarashi will introduce the Japanese HTA process and approach to RWE (10 min) and then Mr. Tilden will provide a similar introduction for Australia (10 min). Mr. Tilden and Dr. Igarashi will then introduce the Micra leadless pacemaker case study, how RWE was leveraged in these evaluations, and compare and contrast across the two countries (15 min). Dr. Strachan will then lead the panelists in a discussion of best practices for leveraging RWE for medical device HTA in future before concluding with audience questions (15 min).
Moderator
-
Liesl Strachan, BSc, PhD
Medtronic, Sydney, Australia
Speakers
-
Ataru Igarashi, PhD
The University of Tokyo, Tokyo, Japan
-
Dominic Tilden, MPH
THEMA Consulting, Sydney, Australia
Enhancing Expert Education in HTA: Challenges and Lessons From Thailand
Session Type: Workshop
Topics: Distributed Data & Research Networks
Level: Introductory
PURPOSE: Since the introduction of the cost-effectiveness evaluation system in Japan in 2019, six years have passed, and various challenges in its implementation have been identified. Among these, the education and training of experts with specialized knowledge in Health Technology Assessment (HTA) remains a critical issue. In Japan, several universities play a key role not only in conducting public assessments but also in fostering HTA expertise. This workshop aims to review the current state of HTA education in Japan and explore what can be learned from international experiences in countries with more established HTA systems.
DESCRIPTION: The session will begin with an overview of Japan’s HTA system and educational initiatives presented by Manabu Akazawa (5 min). This will be followed by presentations from Drs. Rei Goto (15 min) and Kensuke Moriwaki (15 min) on their respective institutional approaches to HTA expert training. Additionally, Dr. Sitaporn Youngkong will provide insights into the history and development of HTA education in Thailand (20 min). Finally, an interactive discussion will engage the audience in identifying key challenges and opportunities for strengthening HTA education in Japan. Lessons from Thailand and other countries will be examined to consider potential improvements. This workshop is expected to provide valuable insights not only for academia but also for policymakers and industry stakeholders involved in HTA in Japan.
Moderator
-
Manabu Akazawa, MPH, PhD
Meiji Pharmaceutical University, Kiyose, Japan
Speakers
-
Rei Goto, PhD, MD
Graduate School of Health Management, Keio University, Tokyo, Japan
-
Kensuke Moriwaki, BS, MS, PhD
Ritsumeikan University, Kyoto, Japan
-
Sitaporn Youngkong, PhD
Mahidol University, Bangkok, Thailand
16:15 - 16:45
Networking Break
Session Type: General Meeting
16:45 - 17:45
Educational Symposia
Session Type: General Meeting
17:45 - 18:45
Networking Reception
Session Type: General Meeting
Tue 30 Sep
8:00 - 9:00
Generative AI in RWE: Real-World Impact and the Road to Reliable Evidence for Decision Making
Session Type: Workshop
Topics: Data Protection, Integrity, & Quality Assurance
Level: Intermediate
PURPOSE: This workshop will critically evaluate the tangible impact of Generative AI (GenAI) in RWE over the past two years since the introduction of GPT3.5 and beyond. It will address key questions regarding the technology advancements, real-world applications, limitations, and barriers of GenAI in key areas of RWE, including quality data curation, insight generation and evidence synthesis. Furthermore, we will explore how close GenAI is to being considered trustworthy and reliable for regulatory and payer decision-making,
DESCRIPTION: HEOR leverages real-world data (RWD) to improve patient outcomes, yet faces challenges such as data heterogeneity, scalability constraints, and underutilization of unstructured data. Emergent AI technologies, particularly GenAI and large language models (LLMs), offer potential solutions by enhancing data curation, natural language processing, and predictive analytics. The session begins with an overview of the current state of GenAI and LLMs in HEOR. Dr. Wang from IMO/Tulane will present her research on developing and evaluating RWE generation, particularly how LLMs have powered "social listening" on disease care journeys and patient-reported outcomes for hard-to-reach populations. Dr. Shi from Tulane will compare traditional approaches to GenAI-powered applications in systematic literature reviews, highlighting time and cost savings in different approaches. Dr. Wang-Silvanto from Astellas will offer an industry perspective on implementing GenAI-enabled evidence generation and synthesis solutions. Finally, all panelists will discuss trustworthiness, transparency, scalability, and readiness of GenAI in RWE for decision-making. The session concludes with an interactive Q&A and digital polling, fostering engagement and diverse perspectives on the current and future role of GenAI in HEOR. This panel provides actionable insights into GenAI’s potential to transform evidence generation and synthesis and decision-making in HEOR.
Moderator
-
Xiaoyan Wang, PhD
Tulane University, Westport, CT, United States
Speakers
-
Xiaoyan Wang, PhD
Tulane University, Westport, CT, United States
-
Lizheng Shi
Tulane University, New Orleans, LA, United States
-
Jing Wang-Silvanto, PhD
Astellas Pharma, Addlestone, United Kingdom
The Promise of OMOP and Database Research at Scale in APAC: Opportunities and Gaps
Session Type: Issue Panel
Topics: Distributed Data & Research Networks
Level: Introductory
ISSUE: The Observational Medical Outcomes Partnership Common Data Model (OMOP-CDM) has emerged as a valuable framework for large-scale database research and real-world evidence (RWE) generation within the Asia-Pacific (APAC) region. However, acceptance and implementation levels vary significantly, resulting in distinct gaps and missed opportunities for robust data utilization. Understanding regional variations, including areas of high acceptance, limited uptake, and clear gaps, is essential for optimizing OMOP-CDM’s potential to facilitate evidence-based healthcare decisions, effective health technology assessments, and efficient market access processes across APAC.
OVERVIEW: The moderator introduces the session (approximately 15 minutes), emphasizing the global importance of OMOP-CDM for large-scale healthcare database research and outlining significant regional opportunities and adoption gaps within APAC. Speaker 1 (10 minutes) will share academic experiences from Taiwan, detailing successes, challenges, and critical gaps in OMOP-CDM implementation. Discussion will focus on the impact of these gaps on data integration quality, research collaboration, and evidence generation. Speaker 2 (10 minutes) provides an industry-focused viewpoint from Japan, exploring practical implementation successes and barriers encountered with OMOP-CDM. Key impacts on market access, health technology assessment strategies, and database research capabilities will be highlighted. Speaker 3 (10 minutes), representing OHDSI, will discuss lessons from OMOP-CDM implementations across APAC, addressing best practices, common challenges, and strategies for improving data infrastructures and collaboration. The session concludes with a 25-minute interactive discussion involving panelists and the audience, focusing on actionable steps to close identified gaps, enhance OMOP-CDM adoption, and strengthen collaborative database research throughout APAC.
Moderator
-
Sven Demiya, MBA, MSc, PhD
IQVIA, Minato-ku, Japan
Speakers
How to Address Evidence Uncertainties via Real-World Evidence Generation in Cancer Drug Fund in Taiwan
Session Type: Issue Panel
Topics: Health & Insurance Records Systems
Level: Intermediate
ISSUE: Taiwan Government introduced Cancer Drug Fund (CDF) to enhance access to new cancer drugs.
OVERVIEW: This is meant for selected cancer drugs based on high unmet needs with early trial data, which poses uncertainty in evidence. Therefore, CDF requires Real World Evidence (RWE) collection to address the evidence uncertainty. However, RWE collection has its own challenges to address the evidence uncertainties. Each panelist from different background will debate the challenges and potential solutions from their respective perspectives.
Moderator
-
Wonjoo Jung, BSc, MSc
Johnson & Johnson, Singapore, Singapore
Speakers
-
Fei-Yuan Sharon Hsiao, PhD
College of Medicine, National Taiwan University, Taipei, Taiwan
-
Joann Tseng, PharmD
Johnson & Johnson, Taipei, Taiwan
9:00 - 9:30
Networking Break
Session Type: General Meeting
9:30 - 10:30
Culture-Specific Health-Related Quality of Life Measurement: Is It Necessary for Asia?
Session Type: Issue Panel
Topics: Health & Insurance Records Systems
Level: Intermediate
ISSUE: Most Health-related quality of life (HRQoL) measures are developed in Europe or North America and subsequently introduced to other regions. There are concerns about this practice because what mattes to a person’s health and quality of life may be influenced by the culture the person lives in. Indeed, there is some evidence for the suboptimal measurement properties of some HRQoL instruments in Asia. Recently, Asian outcomes researchers started to develop culture-specific HRQoL instruments. Examples include Asia PBM seven dimensions (AP-7D) and China Health Related Outcomes Measures (CHROME).
OVERVIEW: The issue panel will include an initial vote for attendees in favour or against the idea of culture-specific HRQoL measurement. We will allocate 20 minutes for an interactive discussion with attendees. Attendees’ views on the role of culture-specific HRQoL instruments in Asia will be sought again at the end of the session.
Moderator
-
Nan Luo, PhD
National University of Singapore, Singapore, Singapore
Speaker
-
Yasuhiro Morii, PhD, MBA
National Institute of Public Health, Wako, Japan
Real-World Evidence in China: How Can It Transform China's Healthcare Landscape?
Session Type: Other Breakout Session
Topics: Health & Insurance Records Systems
Level: Introductory
PURPOSE: The utilization of real-world evidence (RWE) in China has gained significant momentum, influencing key aspects of the product lifecycle, from regulatory approval to market access and physician adoption. The experts in this session will discuss how RWE is being utilized across the product lifecycle in China, analyzing its impact on regulatory decisions, NRDL evaluations, and clinical adoption. We will also explore the opportunities and challenges associated with applying RWE in China’s evolving healthcare landscape.
DESCRIPTION: The session will begin with an interactive audience poll to capture initial thoughts on RWE in China, with a word cloud summary displayed on screen (3min, Zhang). Next, there is an introduction of policy initiatives, e.g., pilot zones in Hainan Boao and the Greater Bay Area and share examples of innovative products that have integrated RWE data into regulatory submissions for accelerated approval (9min, Zhang). Afterwards, we will explore the impact of RWE on reimbursement evaluations and decisions and potential future trends (20min, Liu). Following that, we will consider how RWE is influencing clinical practice, industry dynamics, and the broader healthcare landscape, highlighting the opportunities and challenges it presents (20min, Wu). The session will be concluded by a discussion between the audience and presenters (8 minutes). This discussion will benefit policymakers, researchers, and industry stakeholders by providing insights into the current state and future trends of RWE in China.
Moderator
-
Wenting Zhang, PhD
Trinity Life Sciences, San Francisco, CA, United States
Speaker
-
Chris Wu, MSc
ASCA I-DXd P&MA Lead, Daiichi Sankyo, Tokyo, Japan
How Is Real-World Evidence Supporting Conditional Listing and Health Technology Reassessment?
Session Type: Workshop
Topics: Reproducibility & Replicability
Level: Intermediate
PURPOSE: Increasing demand for timely, evidence-based access to innovative intervention has led to the growing adoption of conditional listing mechanisms for costly health technology with great uncertainty. Health technology reassessment (HTR) is urgent to ensure long-term effectiveness/safety and economic outcomes in real-world settings. Real-world evidence (RWE) becomes essential in supporting these adaptive reimbursement strategies. Across Asia and other regions, insurance payers and policy regulators are engaged in developing mechanisms to generate, evaluate, and apply RWE throughout the life cycle of health technology. This workshop will explore how RWE supports conditional coverage and HTR, with a focus on adaptation and application within specific country contexts. Participants will gain insights into methodological considerations, implementation challenges, and case studies illustrating how RWE supports decision-making under uncertainty. Challenges and considerations from multiple stakeholders will also be discussed.
DESCRIPTION: Attendees will gain a practical view of the strengths/limitations and key considerations of current RWE guidance applied in specific country contexts. We will begin with an overview of recent policy reforms in Taiwan and highlight the need for RWE (10 min). International guidance will be discussed, emphasizing methodological transferability and local adaptation. Taiwan’s RWE framework will be introduced as a regional case, illustrating how infrastructure, data governance, and stakeholder collaboration support evidence generation (15 min). Case examples will demonstrate practical approaches to study design, outcome selection, and flexible data strategies for conditional listing and HTR (20 min). The workshop will conclude with a multi-stakeholder panel featuring government, academia, and industry perspectives on implementation barriers and future directions (20 min). This workshop aims to strengthen the role of RWE in dynamic coverage models.
Moderator
-
Fei-Yuan Sharon Hsiao, PhD
College of Medicine, National Taiwan University, Taipei, Taiwan
Speakers
-
Elise Chia Hui Tan, MHA, MS, PhD
China Medical University, Taiwan, Taichung, Taiwan
-
Huang-tz Ou, PhD
National Cheng Kung University, Tainan, Taiwan
-
Bor-Seng Ko, MD & PhD
Department of Hematological Oncology, National Taiwan University Cancer Center, Taipei, Taiwan
10:30 - 11:00
Networking Break
Session Type: General Meeting
11:00 - 12:00
Educational Symposia
Session Type: General Meeting
12:00 - 13:00
Networking Break
Session Type: General Meeting
13:00 - 14:00
Poster Session
Session Type: General Meeting
14:00 - 15:00
Spotlight Session
Session Type: General Meeting
15:00 - 15:15
Break
Session Type: General Meeting
15:15 - 16:30
Building the Future: Advancing Real-World Evidence in Asia Pacific and Beyond
Session Type: Plenary
The use of real-world evidence (RWE) continues to increase but has yet to reach its potential and maximize value. There is a long history of RWE in post-market reviews to monitor drug safety, effectiveness and value-based pricing; however, RWE for decision-making such as drug approvals, health technology assessment and clinical practice remains in its development. Access to large – population level - real world databases is fundamental as is developing robust methods for standardized analysis of data and collaboration across jurisdictions remains in its infancy.
This plenary panel explores the future for RWE in HEOR and addresses questions around what do we, as HEOR experts, need to do to develop and optimise the use of RWE? How should we go about getting RWE into decision-making and clinical practice? What should we be doing to foster the development and use of RWE? Is there a place for using AI in RWE? If a harmonized HTA ecosystem was sought, can RW data and RWE provide the uplift needed to harmonize systems?
