Workshops
Monday, 6 November 2017
11:15 - 12:15
BREAKOUT SESSION
Health Policy Development Using Outcomes Research

11:15 - 12:15
Room: Hall 2

W1: WHERE DO WE NEED GOOD RESEARCH PRACTICE GUIDANCE IN HEALTH TECHNOLOGY ASSESSMENT?

Discussion Leaders:

Finn Børlum Kristensen, MD, PhD, Professor, Faculty of Health Sciences, University of Southern Denmark, Hilleroed, Denmark bio

Mirjana Huić, MD, PhD, Assistant Director, Department for Development, Research and Health Technology Assessment, Agency for Quality and Accreditation in Health Care and Social Welfare, Zagreb, Croatia

Wim Goettsch, PhD, Director EUnetHTA JA3, EUnetHTA JA3 Directorate, The National Healthcare Institute (ZIN), Diemen, The Netherlands

Sophie Werkö, PhD, MSc, Project Director, Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU), Stockholm, Sweden

PURPOSE:

An overview of existing good practices related to translating evidence to policy has been done by a multidisciplinary group of ISPOR and non-ISPOR members from all regions of the world and undergone two rounds of review including a public review. Emphasis was mainly on approaches to inform population-based purchasing, reimbursement, and formulary decisions on pharmaceuticals and medical devices while not excluding clinical practice guideline or pathway development. Next step is to identify areas in need of guidance and good research practice documents, and discuss approaches of how to address them. The workshop offers an opportunity to discuss where there is most need to improve the situation taking a perspective of those producing HTA, e.g., national and regional HTA agencies.

DESCRIPTION:

Informing population-based health care decisions using evidence after regulatory approval is a fast-growing policy –- and scientific –- field. It is necessary to identify good practices in this field to advance the science of informing decision making and promote appropriate capacity building and education. Workshop participants will be presented with examples of best practices and guidance identified through a scoping review. The workshop will discuss where good practices have not yet been identified, and how the situation could be improved at European and global scale.

Presented by the ISPOR Health Technology Assessment (HTA) Council Working Group


11:15 - 12:15
Room: Hall 1

W2: PATIENT POWERED REGISTRIES: USEFUL FOR HEALTH TECHNOLOGY ASSESSMENT OR NOT?

Discussion Leaders:

Gurmit Sandhu, B Pharm (Hons), MBA, MPH, Patient Engagement Specialist, Gurmit Sandhu Consulting GmbH, Basel, Switzerland

Elisabeth M. Oehrlein, BA, PhD Candidate, Dept. of Pharmaceutical Health Services Research, University of Maryland School of Pharmacy, Baltimore, MD, USA

Robert N. McBurney, PhD, Co-Principal Investigator, iConquerMS™ - the MS Patient-Powered Research Network, Accelerated Cure Project for MS, Waltham, MA, USA

Chantal Guilhaume, PharmD, Scientific Project Manager, EUnetHTA JA3; Direction de l'Evaluation Médicale, Economique et de Santé Publique (DEMESP), Haute Autorité de Santé, Saint-Denis La Plaine, France

PURPOSE:

Patient powered registries (PPR’s) are emerging data sources that collect information directly from patients on a specific disease or condition. These data are potentially useful to health technology assessment (HTA) bodies, in particular for understanding natural history of disease, current management, and utilization across subpopulations. However, researchers and decision-makers alike may be unfamiliar with these new sources of data. This workshop will introduce participants to PPRs and consider possible applications to HTA. Audience members will be invited to participate in a case study of multiple sclerosis (MS), including an overview of evidence utilized by a sample of European and North American HTAs for recently approved MS medications and a presentation of data capabilities of an MS PPR.

DESCRIPTION:

Participants will be provided an overview of the breadth of PPRs, possible applications of PPRs, and tools for identifying appropriate PPRs. Considerations include opportunities to improve patient representativeness in HTA decision-making and how PPR data could be complementary to existing data already incorporated into HTA decision-making. This workshop will include a case study comparing recent HTA evaluations with data available in the iConquerMS PPR. The workshop will conclude with an audience discussion on the role of PPRs in HTA. Discussion themes include how and when it is appropriate to incorporate PPR’s; minimum standards; types of data that can be gleaned from PPRs, and stakeholder perspectives.

14:15 - 15:15
BREAKOUT SESSION
Patient-Reported Outcomes & Patient Preference Research

14:15 - 15:15
Room: Hall 3

W3: MAKING PATIENT REPORTED OUTCOMES MEASUREMENT MEANINGFUL: BEST PRACTICES FOR PRESENTING PATIENT REPORTED OUTCOMES DATA TO PATIENTS, CLINICIANS, AND DECISION MAKERS

Discussion Leaders:

Jakob B. Bjorner, MD, PhD, CSO, Optum Patient Insights, Johnston, RI, USA

Michael Brundage, FRCPC, MD, Professor and Director, Division of Cancer Care and Epidemiology, Cancer Research Institute, Queen's University, Kingston, ON, Canada

Claire Snyder, MHS, PhD, Professor of Medicine and Program Director, Johns Hopkins School of Medicine, Baltimore, MD, USA

Martha Bayliss, MSc, CSO, Optum Patient Insights, Johnston, RI, USA

PURPOSE:

The purpose of this workshop is to describe and evaluate different displays of Patient reported outcomes (PRO) data to gauge their ability to convey key messages from patient-centered research. PROs are used routinely in clinical trials and pharmacoeconomic studies and increasingly in clinical practice and population health management. However, many intended users of PRO evidence are unfamiliar with interpretation of the source data. Also, PRO instruments vary in metric and direction of scoring, further complicating the communication of important messages from the data. This session will present results from qualitative and quantitative studies of presentation formats that most effectively communicate PRO results.

DESCRIPTION:

This session will start with an outline of challenges commonly encountered in presentating PRO data:, direction of scoring, standardization, using norm data and disease specific benchmarks, minimal important differences and responder thresholds. We will address challenges in interpreting individual-level scores as well as group- and population-level data. Discussion leaders will share their experience in selecting the best data visualization approaches for different audiences or stakeholder groups. We will present results of a recent study to develop stakeholder-driven, evidence-based standards for presenting PRO data, in which patients, providers and PRO researchers were shown hypothetical clinical trial data, asked to interpret the results and rate the clarity of the different formats.

Health Policy Development Using Outcomes Research

14:15 - 15:15
Room: Forth (Armadillo)

W4: BRIDGING THE GAP: BEST PRACTICES IN NAVIGATING DIVERGING PERCEPTIONS OF REGULATORS AND PAYERS ON NON-TRADITIONAL CLINICAL TRIAL ENDPOINTS

Discussion Leaders:

Mark Chalmers, PhD, Principal & EU Lead, CBPartners, London, UK

Caroline Solon, MSc, Senior Associate, CBPartners, San Francisco, CA, USA

Andrew Walker, PhD, Director, Salus Alba Consulting, Glasgow, UK

Lung-I Cheng, PhD, Director, Market Access, Takeda, Zurich, Switzerland

PURPOSE:

The objective of this workshop is to cultivate potential solutions for the incongruity between regulatory acceptance and payer resistance to evidence review based on non-traditional endpoint selection. Perspectives from both the payer and manufacturer vantage points will be shared.

DESCRIPTION:

Payers are accustomed to reviewing safety and efficacy evidence based on established outcome metrics, such as PFS or OS in oncology. Recently, manufacturers have explored non-traditional clinical trial endpoints, e.g., PFS2, DFS, either because reliance on historically accepted metrics is not feasible for the patient population in question or the treatment addresses disease pathology and health outcomes in a new way. The main justification for the willingness of new therapy developers to use non-traditional endpoints is that regulators have advised on, accepted, and sometimes encouraged these new approaches to more accurately pursue marketing authorisation decision-making. However, the more diverse payer environment has been less willing to accept endpoint innovation, leading to a misalignment on evidence generation strategies across marketing authorisation and access decision-making.

Guided by recent examples, this workshop will explore the key drivers for non-traditional metric development, and the corresponding payer perceptions. The discussion will then turn to identification of steps that manufacturers can take to mitigate payer objections. Discussion leaders will invite the audience to share their experiences and opinions on why regulator and payer opinions diverge about non-traditional endpoints, as well as how trial designers can maximise the likelihood of payer acceptance through validation efforts and early-consultation. Additionally, discussion will focus on how clinical and economic opinion leaders can shape the literature domain and proactively educate future evidence reviewers.

The main output of the workshop will be a set of best practices for developers to communicate the rationality and improve review potential for using new endpoints in the absence of traditional outcome metrics.

15:45 - 16:45
BREAKOUT SESSION

15:45 - 16:45
Room: Lomond Auditorium (Loch Suite)

W5: SUSTAINABLE FUNDING AND FAIR PRICING FOR ORPHAN DRUGS: WHAT ARE THE SOLUTIONS?

Discussion Leaders:

Martina Garau, MSc, Principal Economist, Office of Health Economics, London, UK

Michael Drummond, MCom, DPhil, Professor of Health Economics, Centre for Health Economics, University of York, Heslington, York, UK bio

Saskia Knies, PhD, Senior Advisor Pharmacoeconomics, National Health Care Institute (ZiN), Diemen, The Netherlands

Olivier Ponet, MBA, Region Europe VD&A Lead, Shire plc, Zug, Switzerland

PURPOSE:

Since the inception of the European regulation on orphan drugs, there has been a debate as to whether conventional economic evaluation methods should be applied to assess these treatments and whether their funding is sustainable in healthcare systems facing substantial budgetary pressures. The evidence in support of paying a premium for orphan drugs is mixed but in practice many countries in Europe provide access to them. From a manufacturer’s perspective, it might be challenging to recoup R&D costs and earn a return on investment at the standard cost-per-QALY thresholds given small patient populations and development risks. Recent changes in the NICE Highly Specialised Treatments introduce a higher cost effectiveness threshold to judge value-for-money of treatments for very rare conditions, which represent only a fraction of orphan drug approvals.

The purpose of this workshop is to discuss options to make the funding of valuable orphan drugs sustainable for healthcare systems and to provide a ‘fair’ reward to manufacturers investing in areas of high unmet need.

DESCRIPTION:

Martina Garau will start the session providing evidence on the rate of HTA approval and reimbursement of orphan drugs across Europe.

Saskia Knies will present insights on and learnings from the Dutch current approach to assess and appraise orphan drugs.

Mike Drummond will propose a new method to adjust the cost effectiveness threshold to reflect the difference between the population size of orphan and non-orphan treatments.

Ulf Staginnus will discuss the topic from an industry perspective, including an illustration of the R&D cost drivers and risks in the context of rare conditions.

Attendees can provide their views on the methods presented in an interactive voting and Q&A session.

Use of Real World Data

15:45 - 16:45
Room: Clyde Auditorium (Armadillo)

W6: GENERATING REAL-WORLD EVIDENCE FOR REAL-WORLD DECISIONS: APPLICATION OF ADVANCED METHODS

Discussion Leaders:

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

Richard Grieve, PhD, Professor of Health Economics Methodology, London School of Hygiene and Tropical Medicine, London, UK

Anirban Basu, PhD, Professor & Director, Pharmaceutical Outcomes Research and Policy Program, University of Washington, Seattle, WA, USA

Stephen Oneill, MEconSc, PhD, Lecturer, Economics, National University of Ireland, Galway, Galway, Ireland

PURPOSE:

HTA agencies around the world have started demanding evidence on the effectiveness and cost-effectiveness of new interventions on real-world population. Two specific types of evidence, often sought for, are: What impacts would access to a new intervention produce in the population that is cared for by an agency? How can we generate causal information about the heterogeneity of intervention impacts outside an RCT? Harnessing the power of advanced statistical and econometrics methods in answering these question and also understanding their limitations can provide a transparent way to generate such real-world evidence and build confidence of HTA agencies in these results.

DESCRIPTION:

This session is organized for discussions of application of three advanced statistical/econometric methods to generate real-world evidence for real-world decision making. They will cover the following topics: 1) projecting results from an RCT to a target population in the context of the cost-effectiveness of Pulmonary Artery Catherization (PAC), 2) projecting decision model results, that span multiple trials, to a target population in the context of the comparative effectiveness of a long-acting antiphychotic, and 3) estimating causal treatment effect heterogeneity using advanced instrumental variables models in the context of the use of intensive care units for deteriorating ward patients.

17:00 - 18:00
BREAKOUT SESSION
Health Policy Development Using Outcomes Research

17:00 - 18:00
Room: Hall 2

W7: VALUE OF INFORMATION (VOI) ANALYSIS FOR RESEARCH DECISIONS: EMERGING GOOD PRACTICE RECOMMENDATIONS FROM THE ISPOR VOI TASK FORCE

Discussion Leaders:

Elisabeth Fenwick, PhD, Senior Principal, Health Economics, Health Economics & Epidemiology, ICON plc, Abingdon, UK

Saskia Knies, PhD, Senior Advisor Pharmacoeconomics, National Health Care Institute (ZiN), Diemen, The Netherlands

Hendrik Koffijberg, PhD, Associate Professor Health Economics, Health Technology & Services Research, University of Twente, Enschede, The Netherlands

Claire Rothery, PhD, Senior Research Fellow, Centre for Health Economics, University of York, York, UK

PURPOSE:

Value of Information (VOI) methods provide essential tools to quantify the value of additional evidence to reduce uncertainty in decision making and inform research prioritization decisions. In this workshop, the ISPOR Task Force members present its initial good practice recommendations. We focus on the role of VOI in supporting different types of health care decisions. Members of the task force will: 1) describe sources of uncertainty that can be addressed with VOI; 2) present wide applications of VOI from multiple decision making perspectives – clarifying how results of VOI analysis can be embedded into the decision making process to inform adoption and research decisions and address issues associated with conditional coverage options; and 3) identify key steps for reporting, presenting and interpreting VOI results.

DESCRIPTION:

Specifically, the task force will cover three key topics: (1) VOI’s role for informing HTA conditional reimbursement decisions following recent developments of EMA’s “Adaptive Pathways” approach; (2) VOI applications through specifying a pay-off function allowing the use of different decision criteria (e.g. comparative effectiveness, cost-effectiveness, or alternative metrics not using a cost per QALY value); and (3) barriers to current application, how to overcome them, and future research directions. The workshop will be valuable to researchers, HTA agencies, decision makers, and industry analysts involved in supporting and making research decisions. The audience will participate in a real-time online poll. Following the presentation of (1) and (2) topics, feedback is requested on the recommendations. Before topic (3), participants will indicate the resources, skills, and software that they should use/require for VOI analysis and how they think these requirements may be met. Following topic (3) participants will rank the presented future research topics in order of importance, and bring forward additional topics. The workshop will conclude findings and Q&A.

Economic Outcomes Research

17:00 - 18:00
Room: Hall 3

W8: MODELING SEPARATE LINES OF TREATMENT VERSUS TREATMENT SEQUENCES IN CANCER

Discussion Leaders:

Agnes Benedict, MSc, Executive Director, Evidera, Budapest, Hungary

Matthew Stevenson, PhD, Professor of Health Technology Assessment, School of Health and Related Research, University of Sheffield, Sheffield, UK

Sonja Sorensen, MPH, Senior Director, Senior Research Leader, Modeling & Simulation, Evidera, Bethesda, MD, USA

PURPOSE:

Treatment sequences have been incorporated in economic models of several therapeutic areas including autoimmune diseases, cardiology, and neurology, but this has not been a common approach in oncology where comparisons are often specific to line of treatment. Given the number of life-extending, but often high-cost, oncology therapies available, exploring whether to treat in earlier or later line or when to use a specific treatment sequence is of growing interest. There are substantial challenges to sequential modeling in oncology, however, including technical considerations, data requirements and their interpretation, and use in decision-making. This workshop will delineate the role of sequential versus line-specific modelling in oncology, detail the challenges, and propose solutions.

DESCRIPTION:

Workshop leaders will define alternative approaches to treatment-sequence modeling versus specific treatment-line models and provide an overview of their use in oncology. The merits and challenges of sequential versus line-specific modelling will be covered. We will discuss the questions that each type of model can answer and identify when it is appropriate to undertake sequential modeling. Finally, we will discuss issues related to the interpretation and presentation of these results. Examples will be used throughout the workshop to illustrate the issues.

Participants will gain an understanding of both the benefits and challenges of sequential-treatment oncology evaluations. The workshop is directed at health care decision makers and individuals commissioning pharmacoeconomic model evaluations, with the goal of highlighting the advantages and disadvantages of the different approaches.

Tuesday, 7 November 2017
8:45 - 9:45
BREAKOUT SESSION
Patient-Reported Outcomes & Patient Preference Research

8:45 - 9:45
Room: Forth (Armadillo)

W9: PATIENTS´ PREFERENCES IN THE EUROPEAN REGULATORY ENVIRONMENT: A CRITICAL REVIEW

Discussion Leaders:

Axel C. Mühlbacher, PhD, MBA, Professor, Health Economics and Health Care Management, Health Economics and Health Care Management, Hochschule Neubrandenburg, Neubrandenburg, Germany

Kevin Marsh, PhD, Executive Director, Outcomes Research, Evidera Ltd, London, UK

Janine A. van Til, PhD, Assistant Professor Preference Research, Health Technology and Services Research, MIRA institute for Biomedical Technology and Technical Medicine, University of Twente, Enschede, The Netherlands

PURPOSE:

Approval, reimbursement and pricing decisions involve trade-offs between multiple outcomes. Beyond clinical outcomes, value judgments are required on the importance of these outcomes. Stated preference methods are increasingly used to elicit patient and other stakeholders‘ preferences to support these benefit-risk trade-offs. Stakeholders across Europe need a better understanding of the methods and practices for incorporating preferences into the development and assessment of new health technologies.

DESCRIPTION:

There is no systematic mapping of the use of preference methods in EU decision making. For this reason, the ISPOR Stated Preference Methods Special Interest Group launched a project on the use of preference data in approval, reimbursement and pricing decisions in the EU to answer questions such as: What method(s) were used, for which decisions? Whose preferences are elicited? When is its use required? How is data validity ensured? Is there data aggregation on technology performance? Valuable lessons can be learned and recommendations developed based on this research.

We will provide an overview of our findings and present 3 comparisons of case studies designed to critically appraisal the appropriateness of preferences methods in different decision contexts. In each case, the audience will be polled to determine which method they think most appropriate. The case studies will include: 1) IQWiG’s cost-benefit assessments incorporate patient preferences elicited by discrete choice experiment (DCE) or the analytical hierarchy process (AHP); 2) EMA’s swing weighting pilot project to elicit decision makers’ preferences and their pilots of survey methods to elicit patient preferences; and 3) Hungarian HTA’s use of direct weighting methods to elicit decision makers’ preferences compared with NICE’s combination of cost-effectiveness analysis and committee deliberation.

Presented by the ISPOR Stated Preference Special Interest Group.


8:45 - 9:45
Room: Hall 3

W10: EQ-5D: IS NICE READY FOR THE NEXT LEVEL?

Discussion Leaders:

Rosemary Lovett, PhD, Senior Scientific Adviser, Science Policy and Research Programme, National Institute for Health and Care Excellence (NICE), London, UK

Allan Wailoo, MA, MSc, PhD, Professor of Health Economics, School of Health and Related Research, University of Sheffield, Sheffield, UK

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

PURPOSE:

There are 2 versions of EQ-5D, each with an associated valuation set: the 3-level version (3L) and the newer 5-level version (5L). NICE’s methods guide says that the 3L valuation set should be used ‘until an acceptable valuation set for 5L is available’. A 5L valuation set for England has recently been produced. However, the two instruments have different characteristics, and for most technologies the 5L gives a higher incremental cost-utility ratio than the 3L (using the UK valuation sets). This is a fast-moving field; by November we anticipate that NICE will have published an interim position statement on the 5L valuation set. Through its Decision Support Unit, NICE has commissioned work to examine the sources of differences between instruments and quality-assure the 5L. The EuroQol Foundation has also funded research into these issues and established a Taskforce to examine differences between 3L and 5L. The workshop will provide an overview of these developments and explore the implications for different stakeholders, including issues of relevance for those outside the UK.

DESCRIPTION:

Rosie Lovett will explain NICE’s interim position on the 5L valuation set and the rationale behind it, and provide guidance for company submissions. The workshop will then discuss the research that informed NICE’s interim position, along with work relevant to a longer-term position and the use of 5L more broadly. Allan Wailoo will present the Decision Support Unit’s work comparing 3L and 5L, including cost-effectiveness case studies from past NICE appraisals. He will explain methods for linking 3L and 5L, both descriptive systems and utilities, to allow all evidence to be incorporated into technology assessment. Nancy Devlin, representing the EuroQol Research Foundation, will discuss why the 5L was developed, the evidence for its advantages over 3L, and ongoing research into the characteristics of the valuation sets.

14:00 - 15:00
BREAKOUT SESSION
Economic Outcomes Research

14:00 - 15:00
Room: Clyde Auditorium (Armadillo)

W11: DETERMINING THE VALUE OF LONG TERM OUTCOMES ASSOCIATED WITH IMMUNO-ONCOLOGY THERAPIES - CHALLENGES AND APPROACHES FOR OS EXTRAPOLATIONS

Discussion Leaders:

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

Pralay Mukhopadhyay, PhD, Statistician, Astrazeneca, Gaithersburg, MD, USA

Andrew Briggs, DPhil, Visiting Investigator, Center for Health Policy and Outcomes, Department of Epidemiology & Biostatistics, Memorial Sloan-Kettering Cancer Center, New York, NY, USA

Nicholas Latimer, MSc, PhD, Senior Research Fellow, ScHARR, University of Sheffield, Sheffield, UK

Min Huang, Ph.D., Principal Scientist, Predictive & Economic Modeling, Center for Observational and Real-world Evidence, Merck & Co., Inc., North Wales, PA, USA

PURPOSE:

Estimating the value of immuno-oncology (IO) treatments is challenging. It is common for the OS benefit to represent a major source of uncertainty due to the immaturity of data from clinical trials. Typically extrapolation of OS relies on fitting a parametric curve under a set of standard distribution assumptions. The emergence of IO agents and their new mechanisms of action add further layers of complexity to OS extrapolation. The objective of this workshop is to discuss additional challenges in fitting survival models for IO treatments and evaluating emerging approaches to overcome existing challenges from both statistical and clinical perspectives. The workshop will build upon previous workshops held at 2016 and 2017 ISPOR meetings, assessing survival projection methods for IO agents.

DESCRIPTION:

Both standard and emerging methodological approaches used to date will be applied to the same dataset from a clinical trial of an IO agent to fit the observed OS data and to extrapolate the OS outcome beyond the trial. The model performance and extrapolations will be compared and contrasted. Based on the empirical findings, IO-specific data gaps and uncertainties, alongside strengths and weaknesses, will be examined and recommendations will be provided for additional methodological considerations.

Use of Real World Data

14:00 - 15:00
Room: Forth (Armadillo)

W12: GETTING TO THE HEART OF THE MATTER: REAL-WORLD EVIDENCE AS AN INDISPENSABLE SOURCE FOR THE ONGOING ASSESSMENT OF CARDIOVASCULAR TREATMENTS

Discussion Leaders:

Kathleen E. Hughes, MBA, Vice President, Avalere Health LLC, Washington, DC, USA

Colin Berry, FRCP, PhD, Professor, University of Glasgow, Glasgow, UK

Pall Jonsson, PhD, Associate Director, Research and Development, National Institute for Health and Care Excellence (NICE), Manchester, UK

Yi Qian, PhD, Director, Amgen, Thousand Oaks, CA, USA

PURPOSE:

Much attention has been paid to the augmentation of data from randomized controlled trials (RCTs) with real world evidence (RWE) in oncology; however, cardiovascular disease (CVD) also presents an interesting case. This workshop demonstrates the importance of a process for all evidence suppliers and users, including manufacturers, professional societies, and HTA organizations, to be undertaking. Panelists show how their organizations use RWE to augment data from RCTs to evaluate the clinical, cost-effectiveness, and patient-reported outcomes of CVD therapies and make recommendations on furtherance of a more meaningful effort in the future. Additionally, the chair will facilitate a series of debate questions with audience participation, culminating with a vote on the strongest answer to each question

DESCRIPTION:

Presenter 1 (cardiologist) will present the rationale for continuous RWE review of CVD: 1) high prevalence of CVD conditions; 2) significant burden on patients and society; and 3) put RCT data in the context of RWE. He will describe the role that professional associations can play in the update process – informing providers, patients, and HTA bodies. Presenter 2 (manufacturer) will outline strength and challenges of RWE, and show how the RWE-informed update process is carried out in CVD research and development within his company. Presenter 3 (HTA representative) will describe the appropriate use of RWE and feasibility of HTAs in performing systematic CVD therapeutics updates and discuss means that could be used, including particulars on structure, process, and use of results. Session Chair (consultant) will act as organizer of workshop participants, posing questions to the presenters to illustrate different points of view and facilitating the audience participation period. She will also add an American perspective to points the European presenters make for additional workshop depth.

15:30 - 16:30
BREAKOUT SESSION
Economic Outcomes Research

15:30 - 16:30
Room: Lomond Auditorium (Loch Suite)

W13: WHY IS YOUR OUTCOME DIFFERENT FROM MINE? DISCUSSING THE POTENTIAL IMPACT OF DESIGN CHOICES IN THE DEVELOPMENT OF DISCRETE EVENT SIMULATION MODELS

Discussion Leaders:

Elisabeth Fenwick, PhD, Senior Principal, Health Economics, Health Economics & Epidemiology, ICON plc, Abingdon, UK

Hendrik Koffijberg, PhD, Associate Professor Health Economics, Health Technology & Services Research, University of Twente, Enschede, The Netherlands

Beate Jahn, PhD, Senior Scientist, Department of Public Health, Health Services Research and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall i.T., Austria

Koen Degeling, MSc, PhD Candidate Health Economics, Health Technology & Services Research, University of Twente, Enschede, The Netherlands

PURPOSE:

Discrete event simulation (DES) is increasingly being used to evaluate the health economic impact of healthcare interventions. Report 4 of the ISPOR-SMDM-Modeling-GRP Task Force includes a number of best practices for modeling using DES. This interactive workshop will elaborate on the design choices underlying several of these best practices, and discuss additional design choices required for DES. Topics covered include design choices regarding the model structure, analysis of (censored) individual patient data to parameterise the model, number of individuals to simulate, handling competing risks, and performing probabilistic sensitivity analysis. Workshop participants will not only become more familiar with these underlying design choices, but also with the corresponding options available to address them and their impact on modeling outcomes. The workshop would therefore benefit those who (are learning to) perform DES modeling studies, review DES models, or with a general interest in health economic modeling.

DESCRIPTION:

The workshop will commence with an introduction by Dr. Fenwick to the concept of DES as well as to several underlying design choices. Next, different options for addressing these design choices will be discussed in detail by discussion leaders Dr. Jahn and Dr. Koffijberg. Participants will be asked to vote on their preferred option for every design choice both before and after the detailed discussion. The latter vote will also include participants’ expectations regarding the impact of the discussed options on modeling outcomes, which will be identified through a single comprehensive case study on treatment in metastatic colorectal cancer patients. The subsequent discussion by mr. Degeling will address the actual impact of the alternative options to the design choices for the case study. In addition to active engagement of participants in the discussion of the design choices and corresponding options for addressing them, the workshop will conclude with an interactive Q&A session.

Patient-Reported Outcomes & Patient Preference Research

15:30 - 16:30
Room: Hall 2

W14: IMPACT OF PATIENT INVOLVEMENT IN HEALTH TECHNOLOGY ASSESSMENTS: A CASE STUDY

Discussion Leaders:

Matthew May, BS, EUPATI Coordinator, EPF, 1000 Brussels, Belgium

Joan Jordan, EUPATI Fellow, Multiple Sclerosis Society of Ireland, Dublin, Ireland

Michael Barry, MB, FRCPI, PhD, Director, Trinity Centre for Health Sciences, St. James’s Hospital, National Centre for Pharmacoeconomics (NCPE), Dublin, Ireland

Orlaith Brennan, Director of Commercial Affairs, Irish Pharmaceutical Healthcare Association (IPHA), Dublin, Ireland

PURPOSE:

The purpose of this workshop is to present a case study that demonstrates the impact of patient involvement in the health technology assessment (HTA) process and the value a trained patient can provide to improve decisions. A multi-stakeholder panel including a patient representative will present their experiences from the same HTA case study.

DESCRIPTION:

Different stakeholder perspectives are crucial; these perspectives ultimately result in more meaningful decisions for all engaged. Much has been written, discussed, and debated about a patient’s or patient organization’s ability to provide input into the HTA process. The European Patient Academy (EUPATI) has developed a “Guidance for patient involvement in HTA” in addition to patient educational material. Joan Jordan will present a case study on Ocrelizumab, a new MS treatment recently approved, where the patient’s voice was included in the reimbursement evidence. Dr. Barry and Roche TBD will present the HTA and industry perspectives, respectively. Matthew May will identify publicly accessible resources such as the EUPATI Toolbox, Patient Expert Training course and the guidance document on HTA, which were utilized in preparing the patients’ input. Speakers will engage the audience by polling on their experiences in involving patients in the HTA process, their perspective on the case study, and the need for guidance documents on effective involvement of patients.

16:45 - 17:45
BREAKOUT SESSION
Economic Outcomes Research

16:45 - 17:45
Room: Clyde Auditorium (Armadillo)

W15: TIME FOR A CHANGE? ALTERNATIVE APPROACHES TO MODELLING IN CANCER VALUE ASSESSMENTS

Discussion Leaders:

Meindert Boysen, PharmD, MSc, Professor, Health Services Research & Policy, London School of Hygiene & Tropical Medicine, London, and Programme Director, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE), London, UK

Robert Hettle, MMath, Vice President (Technical) of Health Economic modelling, HERON Commercialization, PAREXEL International, London, UK

Beth Woods, MSc, Senior Research Fellow, Team for Economic Evaluation and Health Technology Assessment (TEEHTA), Centre for Health Economics, University of York, Heslington, York, UK

Marta Soares, MSc, Senior Research Fellow, Team for Economic Evaluation and Health Technology Assessment (TEEHTA), Centre for Health Economics, University of York, Heslington, York, UK

PURPOSE:

One approach – partitioned survival analysis – has come to dominate assessments of value in oncology. Over seventy percent of cancer appraisals conducted by NICE currently use this approach which is intuitive and easy to implement, but makes strong assumptions which have not been recognised by those using the approach to inform pricing and reimbursement decisions. In particular it assumes independence between clinical endpoints – which has implications for the reliability of long term survival projections, the ability to interrogate key areas of uncertainty, and transparency with respect to model predictions. The objectives of this workshop are to discuss the limitations of the partitioned survival approach and the potential for innovative methods, such as the use of multi-state survival analysis, to provide a more robust basis for assessing the value of cancer drugs.

DESCRIPTION:

The workshop will commence with an overview of the key findings of a recent NICE Decision Support Unit Technical Support Document (TSD). This will summarise the strengths and limitations of partitioned survival analysis, and review the potential for state transition modelling using multi-state survival analysis to overcome these challenges. We will highlight three technical challenges faced when applying these alternative methods: (i) producing unbiased estimates of transition probabilities; (ii) the discord between commonly reported data and model parameters; and (iii) implementation of time-dependent transition probabilities. The second part of the workshop will explore what a move away from partitioned survival analysis would mean for industry and health care decision makers internationally. We will discuss how use of alternative modelling approaches may change the package of evidence presented by industry, reimbursement body decision-making processes, and the nature of funding decisions made. There will be allocated time after each part of the workshop for the audience to share their own experiences and ask the panellists questions.

Patient-Reported Outcomes & Patient Preference Research

16:45 - 17:45
Room: Lomond Auditorium (Loch Suite)

W16: UNDERSTANDING THE VALUE AND FEASIBILITY OF ENGAGING PATIENTS IN THE DESIGN OF CLINICAL TRIALS AND CLINICAL OUTCOME ASSESSMENT MEASUREMENT STRATEGIES: INSIGHTS FROM EXPERIENCES IN ONCOLOGY AND GOUT

Discussion Leaders:

Bryan Bennett, PhD, Director, Patient-Centered Outcomes, Adelphi Values Ltd, Bollington, Cheshire, UK

Sophi Tatlock, MA, Associate Director, Patient-Centered Outcomes, Adelphi Values Ltd, Bollington, Cheshire, UK

Stephanie Manson, PhD, Senior Director, HEOR Excellence, Novartis, East Hanover, NJ, USA

Katja Rudell, PhD, Teaching Fellow, Queen Mary University of London, London, UK

PURPOSE:

The purpose of this workshop is to discuss the value of engaging patients in both clinical trial design and clinical outcome assessment (COA) strategies, as well as examining the types of stakeholders who benefit, by drawing on case study examples. Additionally, the logistical considerations around the feasibility of patient engagement initiatives will be explored.

DESCRIPTION:

Patient engagement acknowledges that the patient is an expert in their own health and care. Encouraging patient engagement may increase activation, promote positive patient behavior and thereby in turn improving outcomes. The presenters in this workshop will draw upon two case study examples of patient engagement: 1) the development and use of the Clinical Trial Feedback Questionnaire (which Dr. Stephanie Manson and Dr. Bryan Bennett developed); and 2) the engagement of a patient advisory group as well as video evidence in the critical evaluation of COAs in supporting endpoints in gout clinical trials (as conducted by Dr. Katja Rudell and Sophi Tatlock). By engaging patients beyond their traditional ‘passive’ role as subjects in trials, or interview participants in COA validation studies, the presenters will discuss the added value and insights obtained, as well as exploring the range of stakeholders who benefitted, in each case study. Participants will be asked to participate in a discussion around the feasibility of patient engagement, notably the logistical challenges which may need to be considered and how they can be overcome. Throughout the interactive discussions with the audience we will discuss our solutions to logistical challenges with our participants. Participants who attend the workshop will therefore leave the session with experience based innovative methods of engaging with patients in clinical trial design and COA research.

Wednesday, 8 November 2017
8:45 - 9:45
BREAKOUT SESSION
Economic Outcomes Research

8:45 - 9:45
Room: Hall 1

W17: COMPARING, CONTRASTING, AND VALIDATING HEALTH ECONOMIC DECISION MODELS: EXPERIENCES FROM THE LATEST MT. HOOD CHALLENGE IN DIABETES AND LESSONS FOR OTHER DISEASE AREAS

Discussion Leaders:

Alan Brennan, PhD, Professor of Health Economics and Decision Modelling, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

Mark Lamotte, MD, Cardiologist, Senior principal, RWES, QuintilesIMS, Zaventem, Belgium

Talitha Feenstra, PhD, Senior Researcher / Assistant Professor, RIVM /UMCG, Bilthoven, The Netherlands

Michael Willis, PhD, Research Director, The Swedish Institute for Health Economics, Lund, Sweden

PURPOSE:

In 1999, two groups of diabetes simulation modelers met on the slopes of Mt Hood near Portland, Oregon, simulated their models for a set of identical scenarios, and compared the results. Now, there have been 8 Mt. Hood Challenges, with up to 10 modeling groups meeting biennially to validate their models, and discuss methodology and data in what is arguably the most collegiate and yet competitive environment for debating models in the HEOR community. The purpose of this workshop is to share the concept of disease-specific, cross-modeling working meetings with modelers from all disease areas and to discuss practical considerations and best methods for this kind of forum.

DESCRIPTION:

This session will start by reviewing the purpose, scope, structure, and practical implementation of the Mt. Hood Challenge congresses, at a level relevant for modelers without diabetes experience. A brief summary of the methods and results of the transparency challenge from 2016 will then be presented, illustrating the difficulty of reproducing results based on publications (even when the same model was used). The 2016 challenge has led to development of guidelines for the reporting of model-based results in diabetes. With a focus on a minimum set of key parameters and scenario characteristics, they substantially extend existing more general model transparency guidelines. The draft version will be presented and the floor will be opened for discussion. Important issues around academic intellectual property versus model sharing and transparency will be discussed. A similar venture for cross model validation and comparison in COPD will also be briefly discussed, with key learnings shared. The workshop will include an extended interactive Q&A session and live electronic voting via smartphone app, during which the audience will have the opportunity to discuss aspects of validation by cross model comparisons as in the Mt Hood Challenges with the panel.

Health Policy Development Using Outcomes Research

8:45 - 9:45
Room: Forth (Armadillo)

W18: FROM ONE TO MANY: WHEN GROUPS – NOT CZARS – MAKE DECISIONS

Discussion Leaders:

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

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

PURPOSE:

To help participants understand alternative methods for ranking and choosing among alternative "candidates" when groups (not “czars”) make health care investment decisions and to help participants choose among those systems for potential use in their future work.

DESCRIPTION:

We will first discuss key elements of social choice methods – systems to rank or choose among alternatives. This will discuss Arrow’s Impossibility Theorem (and how to avoid it), issues of strategic voting and agenda manipulation. and how different voting systems work. This will highlight two key aspects of voting rules -- what type of input voters provide and how that information is transformed into a group priority statement.

Then, using a set of hypothetical technologies (all having multiple important dimensions of value) participants will prioritize the alternatives using four different methods for eliciting preferences: (1) Rank order (using Borda Count and perhaps other voting rules to aggregate preferences); (2) Approval voting (indicate all approved candidates -- priority based on total number of approvals); (3) Majority Judgement (a categorical grading method using median-like rules to prioritize choices) and (4) cumulative voting (aka “dotmocracy, where voters assign 100 points across the alternatives, total points determining the priorities).

After participants have voted using each of the four methods, we will summarize and present their votes, and discuss with participants their use of and views about the different methods of ranking and choosing, and compare the actual voting outcomes of each system. This discussion will emphasize both the traditional social choice theory aspects of each voting rule and also the human factors from an engineering design sense – ease of use, ability to express one’s preferences with clarity, and ease of understanding how individual ballots are converted to a group preference in each of the four choice mechanisms.

10:00 - 11:00
BREAKOUT SESSION

10:00 - 11:00
Room: Hall 1

W19: GENERATING EVIDENCE TO SUPPORT OFF-LABEL HIGHER VALUE CANCER TREATMENT REGIMENS

Discussion Leaders:

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

Mark J. Ratain, MD, FASCO, Leon O. Jacobson Professor, Department of Medicine, The University of Chicago, CHICAGO, IL, USA

Peter Clark, MA, MD, FRCP, Professor, The Clatterbridge Cancer Centre NHS Foundation Trust, Wirral, UK

Virginia Acha, PhD, Executive Director, EU, EMEA, APAC Regulatory Policy, MSD Ltd., Hertfordshire, UK

PURPOSE:

The price of modern oncology drugs presents global payment challenges. While there have been many proposals aimed at constraining pricing through commercial arrangements between payers and companies, there has been little attention given to opportunities to reduce costs of treatment that would be incremental to any reduction in unit acquisition cost. This workshop will present an alternative approach: generating evidence to support the development of higher value (i.e., comparable clinical benefit at much lower cost) off-label treatment regimens that employ lower doses, shorter durations, and/or therapeutic substitutions.

DESCRIPTION:

In this workshop (chaired by Dr. Sculpher), we discuss the global funding challenge of modern oncology drugs, resulting from advances in our understanding of cancer biology and immunology. We also present a potential contribution to the solution of the problem, the development of treatment regimens that aim to decrease drug costs by 50-90%. Specific examples will be presented, such as the use of food to enhance bioavailability of poorly absorbed drugs labeled to be taken fasting (e.g., abiraterone). Strategies to fund and conduct such studies will also be discussed, including discussion of both governmental (e.g., the NHS England Research Plan) and non-governmental (e.g., Value in Cancer Care Consortium) opportunities, as well as the perspective and potential response from the pharmaceutical industry.

Clinical Outcomes Research

10:00 - 11:00
Room: Forth (Armadillo)

W20: DISCONNECTED OR LIMITED EVIDENCE IN NETWORK META-ANALYSIS: WHAT CAN BE DONE?

Discussion Leaders:

Howard Thom, BA, MSc, PhD, Research Fellow in Statistical Modelling, School of Social and Community Medicine, University of Bristol, Bristol, UK

Joy Leahy, BSc, PhD Student, School of Computer Science and Statistics, Trinity College Dublin, Dublin 2, Ireland

Jeroen P. Jansen, PhD, MSc, Chief Scientist of Evidence Synthesis and Decision Modeling, Evidence Synthesis and Decision Modeling, Precision Health Economics, Vancouver, BC, Canada

PURPOSE:

It is often difficult or impossible to use network meta-analysis (NMA) to compare the treatment effects of competing interventions using randomized controlled trial (RCT) evidence alone. Our purpose is to describe methods to supplement RCT evidence using single-arm studies, non-randomized trials, or observational evidence and clarify when they are appropriate.

DESCRIPTION:

NMA uses multiple RCTs to simultaneously estimate many relative treatment effects and models exist for various outcome types; it is thus our preferred framework. However, RCT evidence for interventions of interest can be disconnected or provide only highly uncertain estimates for relative effects. Several alternative methods of indirect comparison and extensions of NMA itself have been proposed to supplement the RCT evidence. When only aggregate data (AD) is available, NMA can be extended to 3-level hierarchical models, with the additional level representing observational or RCT effects. Random effects could also be placed on the study baseline to include single arm studies in the NMA framework. When individual patient data (IPD) is available from at least one RCT alternative indirect comparison methods have been proposed, such as propensity score reweighting (matching adjusted indirect comparison) and outcome-regression models (simulated treatment comparison). We will discuss these methods in the context of specific clinical applications from our own research and from the literature, and explain where they produce biased estimates or make inappropriate assumptions.

14:15 - 15:15
BREAKOUT SESSION
Use of Real World Data

14:15 - 15:15
Room: Hall 1

W21: COMPARING TREATMENTS BY COMBINING DATA FROM VARIOUS RANDOMIZED AND OBSERVATIONAL STUDIES: INTRODUCTION TO CONCEPT, METHODS, AND APPLICATION

Discussion Leaders:

Viktor Chirikov, PhD, Scientist, Pharmerit International, Bethesda, MD, USA

Susanne Schmitz, PhD, Postdoctoral Fellow, Department of Population Health / Health Economics and Evidence Synthesis Research Unit, Luxembourg Institute of Health, Strassen, Luxembourg

Farhan Mughal, MRPharmS, MSc, Associate Director, Health Economics and Outcomes Research, Celgene Ltd, Uxbridge, UK

PURPOSE:

The aim of this workshop is to introduce the audience to recent developments in cross-design synthesis of randomized and non-randomized data. Opportunities and disadvantages of conducting treatment comparisons mixing data from non-randomized and randomized trials will be highlighted and discussed in the context of timely health care decision-making.

DESCRIPTION:

Requirements for value-based dossiers has created demand to increase the availability of indirect pooled data of treatment comparisons in order to support reimbursement decisions. Research in meta-analysis and related techniques is ongoing to provide solutions for the increasingly complex evidence base of mixing data from multiple studies with often different designs and patient populations. However, multiple challenges remain, most prominent of which being the need to account for uncertainty when dealing with heterogeneous data across various dimensions. The wealth of complex methodologies can be confusing to the untrained eye as different types of evidence require alternative incorporation techniques. The audience will be presented with an overview of existing methods (naïve pooling techniques, design-adjusted evidence synthesis and propensity score weighting, Bayesian hierarchical models) to synthesize evidence from different data sources and study designs. The audience will be engaged to discuss opportunities and limitations of the proposed methods.

Health Policy Development Using Outcomes Research

14:15 - 15:15
Room: Lomond Auditorium (Loch Suite)

W22: NEGOTIATING PRICE AND DATA IN AN ERA OF CONDITIONAL APPROVAL: “STICK” OR “TWIST”?

Discussion Leaders:

Daniel Gladwell, MSc, Principal Consultant, BresMed Health Solutions LTD, Sheffield, UK

Warren Cowell, MSc, National Market Access Policy Lead, Janssen Inc., High Wycombe, UK

Alan Brennan, PhD, Professor, University of Sheffield, Sheffield, UK

Ash Bullement, BSc, Health Economist, BresMed Health Solutions, Sheffield, UK

PURPOSE:

When the evidence base for a new oncology treatment leaves substantial uncertainty, the new Cancer Drugs Fund allows the National Institute for Heath and Care Excellence to give the manufacturer two options: (i) offer a low price based on conservative assumptions and obtain immediate approval (“stick”); or (ii) wait until the evidence base has further matured before finalising a potentially higher agreed price (“twist”). This workshop explores how simulation methods can be used to inform the manufacturer’s decision.

DESCRIPTION:

Warren Cowell will begin the workshop with an overview of recent changes to the regulatory and health technology assessment (HTA) landscapes, including the implications of these changes for key stakeholders. Mark Strong will provide a summary of how the expected value-of-information framework can be used by HTA organisations to assess whether an intervention should be approved, approved with research, or rejected. Ash Bullement will then present a hypothetical case study, illustrating how simulation methods can be used to estimate the expected commercial value of information derived from a trial extension. Finally, Daniel Gladwell will lead an extended Q&A session. In the Q&A session, the audience will have the opportunity to engage in discussion with the panel. The topics for discussion include: (i) the impact of increased regulatory and HTA flexibility on patients, payers and manufacturers; and (ii) areas where new methods could help to manage the large uncertainties that arise when using an immature evidence base. Prior to attending the panel, we invite the prospective audience to reflect on how their organisations approach uncertainty during the HTA process. This will enable an informed, interactive discussion about the practical steps required to enhance decision making when faced with substantial uncertainty.

15:30 - 16:30
BREAKOUT SESSION
Use of Real World Data

15:30 - 16:30
Room: Lomond Auditorium (Loch Suite)

W23: IMPROVING PERFORMANCE OF ALGORITHMS TO POWER UNMET NEED AND EFFECTIVENESS IN HEALTH ECONOMICS AND OUTCOMES RESEARCH USING ELECTRONIC HEALTH RECORDS AND HEALTH CARE CLAIMS DATA SOURCES

Discussion Leaders:

Hoa Van Le, MD, PhD, Senior Consultant, PAREXEL INTERNATIONAL, DURHAM, NC, USA

Aaron WC Kamauu, MD, MS, MPH, CEO, Anolinx LLC, Salt Lake City, UT, USA

Schiffon L Wong, MPH, Franchise Head Neurology, Global Evidence & Value Development, Global Research & Development, EMD Serono, Inc., Billerica, MA, USA

Monica Gaines Kobayashi, PhD, MBMA, Consultant, Real World Evidence & Data Analytics, PAREXEL International, Durham, NC, USA

PURPOSE:

The purpose of this workshop is to highlight best practices to improve observational research performance through 1) a methodological overview of algorithm development and validation in electronic health records (EHR) and healthcare claims-based databases, 2) an understanding of novel approaches using text-mining and natural language processing (NLP), and 3) case studies of algorithms developed to identify multiple sclerosis (MS) and its clinical subgroups to generate real world evidence (RWE). By offering an interactive forum, participants will be able to identify and share their successes and challenges in algorithm development and validation.

DESCRIPTION:

We will first present an overview of algorithm development methodologies in EHR and healthcare claims databases. Based on the nature of the research question and study outcomes of interest, we will also address the framework for identifying databases and coding systems with a focus on feasibility, data capture, and data quality. The algorithms’ development process, evaluation measures, and best optimization practices will be shared through examination of case studies that identify MS patient cohorts, subtypes, relapse, and disease activity status. The second topic will focus on novel text mining and natural language processing approaches to enhance performance. NLP-based and traditional medical chart review validation techniques will also be compared. Finally, we will provide insight into the potential benefits and obstacles of using algorithms to generate RWE for the value and safety of medicines. The audience will be engaged in an interactive discussion regarding their successes and challenges in using validated algorithms with contemporary data sources. This workshop will be valuable for healthcare researchers wanting to gain greater awareness and insight on how algorithms can be used to support unmet need and effectiveness studies in health economics and outcomes research (HEOR).

Health Policy Development Using Outcomes Research

15:30 - 16:30
Room: Hall 3

W24: POPULATION-ADJUSTED TREATMENT COMPARISONS IN HEALTH TECHNOLOGY ASSESSMENT: AN OVERVIEW OF APPROACHES AND PERSPECTIVES

Discussion Leaders:

David M. Phillippo, MSc, BSc, Research Associate in Evidence Synthesis, School of Social and Community Medicine, University of Bristol, Bristol, UK

Ahmed Elsada, MSc, BSc, Technical Adviser, National Institute for Health and Clinical Excellence, Manchester, UK

Mark Belger, BSc, Principal Research Scientist, Eli Lilly and Company, Surrey, UK

Nicky J. Welton, PhD, MSc, Reader in Statistical and Health Economic Modelling, School of Social and Community Medicine, University of Bristol, Bristol, UK

PURPOSE:

This workshop will: (i) provide an overview of population adjustment methods and outline recommendations for their use in Health Technology Assessment (HTA); (ii) present industry and reimbursement agency perspectives and practical experiences.

DESCRIPTION:

Treatment comparisons without head-to-head trials are frequently formed using indirect comparisons across multiple trials, making the assumption that any effect modifiers are balanced between trials. Recently proposed methods which relax this assumption, including Matching-Adjusted Indirect Comparison (MAIC) and Simulated Treatment Comparison (STC), are becoming increasingly common in industry-sponsored treatment comparisons, where a company has individual patient data (IPD) from its own trials but only aggregate data from competitor trials. These methods aim to adjust for between-trial differences to form population-adjusted indirect comparisons, and may even be used for comparisons involving single arm studies or disconnected networks. However, additional assumptions and complexities are introduced which must be carefully considered. Since the premise of population adjustment methods is that relative treatment effects can differ between populations, a core component of population adjustment in HTA is the definition of and adjustment to a specific target population for the decision; reimbursement agencies must consider the implications of this change in the decision scenario, whilst companies making submissions must be prepared to produce and justify a comparison in the correct target population.

This workshop will begin with an overview of population adjustment methods. Their assumptions and additional considerations over standard indirect comparisons will be explained along with recommendations on their use in HTA, drawing from a recently published NICE Decision Support Unit Technical Support Document. Following this, presentations from industry and reimbursement agency perspectives will explore the role of population adjustment in HTA through practical examples, providing insight on the experiences and challenges of the use of these new methods in HTA. The workshop will conclude with an interactive Q&A session.

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