|Moderator: Joakim Ramsberg, Agency for Health and Care Services Analysis, Sweden|
|CO1||Mapping The Landscape Of Covid-19 Clinical Trials In The US: Assessing The Opportunities And Gaps In Trial Designs, Interventions And Endpoints|
Simankova I1, Wilke T2|
1Ingress-Health HWM GmbH, Wismar, MV, Germany, 2IPAM - Institut für Pharmakoökonomie und Arzneimittellogistik e.V., Wismar, Germany
|CO2||WHEN Could a Second Wave of COVID-19 Appear in Germany and How Strong Will IT be? a Simulation Analysis|
MArS Market Access & Pricing GmbH, State University Baden-Wuerttemberg, University of Applied Sciences Weingarten-Ravensburg, Weil am Rhein, Germany
|CO3||Effectiveness of Government Measures to Reduce COVID-19 Mortality across 5 Different Countries|
S1, Ray B2, Holy C3,
Sakthivel M1, Elangovanraaj N1,
Krishnan D4, Gupta S5, Trivedi P1, Devulapally M1, Mohapatra A1, Coplan P6|
1Mu Sigma, Bangalore, KA, India, 2Mu Sigma, Bangalore , India, 3Johnson & Johnson, Somerville, MA, USA, 4Mu Sigma, Bengaluru, KA, India, 5Mu Sigma, Bangalore, India, 6Johnson & Johnson, New Brunswick, NJ, USA
|CO4||An Evaluation of the Differences in Vaccine Approval and Procurement Processes between Healthcare Systems during a Pandemic|
Farrington J, Longworth L
*Top 10% Average Review Score
Discussion Leaders: Lea Trela-Larsen, PhD, National Centre for Pharmacoeconomics, Dublin, D, Ireland; Raquel Aguiar-Ibáñez, MSc, Center for Observational and Real World Evidence, Economic and Data Sciences team, Merck Sharp & Dohme Ltd, Haarlem, NH, Netherlands; Howard Thom, BA, MSc, PhD, University of Bristol, Bristol, GLS, UK; Rose Hart, PhD, Health Economics Analysis Team, BresMed Health Solutions Ltd., Sheffield, UK
PURPOSE: To demonstrate the benefits of investing in R as an integrated statistical and economic modelling solution for the health economics and outcomes research community and health technology assessment (HTA). Our workshop will present the advantages (why), and the practicalities of use (how), such as graphical user interfaces and upskilling your team.
DESCRIPTION: The programming language R is well established within health economics. Its flexibility, efficiency and transparency make it ideal for performing the complex data analyses that are required to inform models. However, there is often a disconnection between the software used for statistical analyses and that used for modelling; results are often copied into Microsoft Excel for model building. This is a suitable method if done infrequently to reduce inefficiency or human error; if the modeller has a clear understanding of the source and analysis of the copied data; and if it is presented to key stakeholders and decision-makers in a transparent and justifiable way. The demands of our industry, however, with frequent strategy updates and short turnaround times, do not always make this possible. We therefore invite delegates to consider R as a potential start-to-finish analysis and modelling solution.
This workshop will offer both technical and non-technical participants a rounded demonstration of R from the perspective of HTA, industry, academia, and consultancy. Lea Trela-Larsen will explain the benefits of using R for transparency within HTA. Raquel Aguiar-Ibáñez will highlight the efficiency of updates, country adaptations, and the potential for continuity across product development from an industry perspective. Following these arguments for ‘why’ to use an integrated framework in R, Howard Thom will present the ‘how’ with a practical demonstration of the speed, transparency, and flexibility advantages. Finally, Rose Hart will discuss learning pathways for modelling, version control, and graphical user interfaces in R for technical and non-technical users.
|Moderator: Katja Rudell, BSc, MSc, PhD, Parexel, United Kingdom|
|CL1||The Additional Burden of Caring for Adults with Depression and Suicidal Ideation in Five Large European Countries|
BL2, Yue L3, Mulhern-Haughey
S4, Anjo J5
*Top 10% Average Review Score
|CL2||Measuring Health-Related Quality of Life in the Context of Episodic Conditions|
V1, on behalf of the
1Erasmus University Rotterdam, 's-Hertogenbosch, NB, Netherlands, 2NB, Netherlands
|CL3||Efficiency of Triage By a Rheumatologist in Primary Care for Patients Suspect for Inflammatory Arthritis: Preliminary Results|
E1, Han KH1, Hazes
JMW2, Lopes Barreto D3, Weel-Koenders
*Top 10% Average Review Score
|CL4||Concerns, Quality of Life, Access to Care and Productivity of the General Population during the First 8 WEEKS of the Coronavirus Lockdown in Belgium and the Netherlands|
Goossens LMA2, Bruin R1, van Ballegooijen H3, Krol
1IQVIA, Amsterdam, NH, Netherlands, 2Erasmus University Rotterdam, Rotterdam, Netherlands, 3IQVIA, Amsterdam, Netherlands
Moderator: Sachin Kamal-Bahl, PhD, COVIA Health Solutions, Lansdale, PA, USA
Panelists: Ellen Miller Sonet, JD, MBA, CancerCare, New York, NY, USA; Scott F Huntington, MD, MPH, MSHP, Section of Hematology/Department of Internal Medicine, Yale University School of Medicine, New Haven, CT, USA; Indranil Bagchi, PhD, Novartis Oncology, East Hanover, NJ, USA
ISSUE: The introduction of two chimeric antigen receptor therapies (CAR-Ts) in 2017 ushered a new era of transformational medicine in oncology. These treatments, typically administered in the inpatient setting, involve engineering a patient’s own immune cells to treat the cancer. While CAR-Ts have shown complete responses and prolonged disease-free survival in a substantial proportion of patients with refractory B cell malignancies in clinical trials, a large portion of patients even in high- and middle-income countries do not currently receive CAR-Ts due to multiple access barriers. At the same time, there are over 400 CAR-Ts in development with several companies working towards next-generation CAR-Ts, including those addressing cancers with high prevalence. Hence, concerns about payer affordability are likely to arise as patient access and availability of CAR-Ts is poised to increase in the coming years.
OVERVIEW: This issue panel will present a global perspective of patients, providers, payers, and manufacturers and debate the key access and affordability issues surrounding CAR-Ts and potential strategies to overcome these barriers. Sachin Kamal-Bahl will provide a brief overview of the current landscape and moderate the panel by posing the following questions to the panelists: What are the current access barriers to CAR-Ts? Can we better understand the unique access barriers to CAR-Ts from the varying perspectives on the panel and identify mid- to long-term practical solutions to address these barriers? What about payer affordability issues that will come along with expanded access? How can we strike a balance between these competing issues and foster life-saving innovation? Ellen Miller Sonet will address these questions from the patient perspective. Scott Huntington, an oncologist and health policy researcher focused on access and affordability issues, will represent both the health care system and payer perspective. Indranil Bagchi will present the perspective of a CAR-T pharmaceutical manufacturer in the debate.
Moderator: Matthew Taylor, PhD, MSc, York Health Economics Consortium, University of York, York, NYK, UK
Panelists: Mark Nuijten, MBA, PhD, MD, Istanbul Technical University, Istanbul, Turkey; Michelle Green, BA, MSc, York Health Economics Consortium, University of York, York, UK; Isabelle Durand Zaleski, MD, PhD, AP-HP / INSERM, Paris, France
ISSUE: England, France and the Netherlands are among the few countries in Europe dedicating a chapter or document within their health technology assessment (HTA) methods and processes to medical devices. The National Institute for Health and Care Excellence in England published its first medical technologies guidance in 2010 via its Medical Technologies Evaluation Programme (MTEP), set up specifically to undertake health technology assessment (HTA) of medical devices. The Haute Autorité de santé (HAS) issued a practical guide on medical device evaluation in 2009, last updated in 2019, and publishes a dashboard of device assessments. Zorginstituut Nederland (ZIN) dedicates a chapter of its guidelines for conducting economic evaluations in health care to medical devices.
This issues panel will consider the approaches taken in each of the three countries and provide an opportunity for debate around the processes and methods used for the HTA of medical devices. The panel will compare and contrast the experiences of the 3 countries as well as identifying key learnings identified to date.
OVERVIEW: Dr Matthew Taylor will introduce this issues panel by providing an overview of health technology assessment in the context of medical devices and introducing the panelists. Each panelist will speak for ten minutes. Professor Mark Nuijten will describe main differences between cost-effectiveness analyses for pharmaceuticals and devices at ZIN highlighting specific issues for devices. Michelle Green will discuss submissions to MTEP from the perspective of an External Assessment Centre for the programme at NICE. Professor Isabelle Durand-Zaleski will review the challenges for devices claiming a high medical benefit with non-generic pricing, and the HAS reports on the cost effectiveness models. Twenty minutes will be allowed for discussion with the audience following the presentations. The issues panel will be of interest to the medial device industry and HTA agencies evaluating medical devices.
Moderator: Michael Drummond, MCom, DPhil, Centre for Health Economics, University of York, York, YOR, UK
Panelists: Elena Nicod, PhD, Centre for Research on Health and Social Care Management, Bocconi University, Milan, MI, Italy; Andrew Lloyd, DPhil, Acaster Lloyd Consulting Ltd, London, UK; Ross Dent, MSc, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE), Manchester, UK
ISSUE: Professor Drummond will introduce the session and outline the challenges commonly encountered with patient-reported outcomes (PROMs) and health state utility values (HSUVs) in health technology assessment (HTA) of rare diseases. Elena Nicod will share findings from the EU-funded Horizon 2020 IMPACT-HTA project on use of PROMs/HSUVs in HTA of non-oncology rare disease treatments in England, Germany, Netherlands and France. Andrew Lloyd, as member of the ISPOR Task Force on use of HSUVs in cost-effectiveness models, will share his experiences in the development of PROMs/HSUVs in rare diseases for HTA and of PROM/HSUV methodologies generally. Ross Dent will provide an HTA perspective, bringing insights from the National Institute for Health and Care Excellence’s ongoing methods review, concerning whether and when departure from the reference case may be acceptable. The discussion will explore whether existing approaches are suitable for rare diseases, and the circumstances in which less conventional approaches (e.g. vignettes, analogue data, patient input) may be appropriate?
OVERVIEW: Measuring health benefits in rare conditions is extremely difficult due to small and heterogeneous populations and lack of disease knowledge and expertise. PROs, as subjective endpoints, are characterized by greater measurement error and require a lot of data to demonstrate clinically meaningful differences. Other challenges concern PROM availability or selection, data collection, or concept validity. To better incorporate PROs in HTA for rare diseases, a clearer understanding of these challenges, and the possible solutions, is needed. Different perspectives on the use of PROMs in HTA will be presented and feed into a debate concerning the circumstances in which special approaches may be needed (rare vs ultra-rare, children vs adult, acute vs chronic, etc). These will include questions around whether challenges in rare diseases are more nuanced than for prevalent conditions, the potential solutions, barriers to implementation, need for different methodologies and policy implications.
Moderator: Katja Rudell, PhD, MSc, Parexel Access Consulting, Parexel International, London, UK
Panelists: Olivier Chassany, MD, PhD, Patient-Centered Outcomes Research, University Paris-Diderot, Paris, 75, France; Michael Schlichting, Diplom, Global Biostatistics, Epidemiology & Medical Writing, Merck KGaA, Darmstadt, HE, Germany; Corneel Coens, MSc, Quality of Life Department, EORTC, Brussels, Belgium
ISSUE: We observe high levels of variability from one sponsor, as well as across sponsors, in the analyses and presentations of PRO data for regulatory & HTA submissions. Whilst qualitative data and design of instruments have been highly regulated, the approach in statistical analysis and interpretation is mainly left to the sponsors – which can lead to flexibility but also inefficiency in submissions. We will debate from sponsor, clinical and PRO research perspectives how new guidance on statistics can aid forthcoming PRO submissions. We look forward to debate the topic among the audience involved in HTA and regulatory submissions.
OVERVIEW: Moderator Dr Katja Rudell will introduce the topic with an example overview of statistics from one product and highlight the difficulties for patients and HCPs to understand the nuances in the analyses (10mins) and then invite each panellist to present perspectives on how to improve presentations, as well as the impact of the latest guidance including estimands on this topic (10mins each). Dr Olivier Chassany will describe the clinician and reviewer perspectives from his experience with EMA/HAS; Michael Schlichting will frame it from the sponsor perspective; and Corneel Coens will describe the EORTC perspective. 20mins will be allocated to audience discussion and debate, for a total of 60 minutes.
Stakeholders who will benefit attending include HTA scientists involved in review of PRO submissions as well as industry specialists involved in designing the submissions
Discussion Leaders: Colm Leonard, MD, National Institute for Health and Care Excellence, Manchester, UK; Paula Rojas, MA, Dept of Economics, University of La Rioja, Logroño, LO, Spain; Simon Van der Pol, PharmD, Health Sciences, University of Groningen, University Medical Center Groningen, Groningen, GR, Netherlands; Isabelle Feldhaus, MSPH, PhD, The Organisation for Economic Co-operation and Development (OECD), Paris, France
PURPOSE: Diagnostic testing is an essential part of modern medicine, but only recently, due to the COVID-19 outbreak, did it gain substantial attention among the general public. VALUE-Dx is an IMI-funded consortium with the objective to assess and demonstrate the value of diagnostic-driven treatment of community-acquired acute respiratory tract infections (CA-ARTI) for both individual patients and public health. Within this consortium, academic, private and (inter)governmental institutions collaborate in many scientific fields: ranging from microbiology and economics to sociology and data science.
Inappropriate prescribing of antibiotics can lead to greater antimicrobial resistance of infectious pathogens. More rapid and accurate diagnoses can reduce inappropriate prescribing and thereby combat antimicrobial resistance. The workshop will focus on the current state of economic models and the appraisal of point-of-care (POC) diagnostics, as well as provide practical guidance for modellers, in vitro diagnostic companies, and appraisal agencies to value diagnostic innovations.
DESCRIPTION: The workshop will cover the full process of assessing a new POC diagnostic: A) the appraisal of POC diagnostic tests, reflecting on what NICE have learned about the challenges of assessing the value of POC testing in the UK setting. B) the specific elements to consider when conceptualizing a health-economic model for POC diagnostics of infectious disease, based on a review of the literature; C) an example of a health-economic model to value POC diagnostics for CA-ARTI, which explains how the considerations of B were used to develop a flexible, individual-based model applicable to several European countries; D) the assessment of broader public health and economic benefits with respect to antimicrobial resistance over the medium- to long-term using advanced modelling techniques. During the workshop, the audience will be asked questions using live polling, which will also be used to inform ongoing research of the VALUE-Dx project.
Discussion Leaders: Maureen Rutten-van Mölken, PhD, Erasmus School of Health Policy & Management (ESHPM), Erasmus University Rotterdam, Rotterdam, Netherlands; Balázs Nagy, PhD, Economic Modelling Division, Syreon Research Institute, Budapest, PE, Hungary; Matthijs Versteegh, PhD, Institute for Medical Technology Assessment, Rotterdam, Netherlands; Sarah Wordsworth, PhD, Health Economics Research Centre, University of Oxford, Oxford , OXF, UK
PURPOSE: 1) To discuss recommendations for health economic modelling of precision medicine (PM) and demonstrate their application using 3 case-studies – maturity-onset diabetes of the young, tumor-agnostic NTRK inhibitors, and DPYD genotyping (ToxNav) prior to fluorpyrimidine-based chemotherapy. 2) To provide an overview of innovations in R&D funding and provider-payments for PM and discuss the way forward in risk-sharing agreements.
DESCRIPTION: There is an increase in the development of healthcare interventions described as PM, with the EU investing €3.2 billion in PM research in 2017 alone. PM holds great promises for improved health outcomes and reduced costs because it enables targeting of therapies. Despite this, PM has been slow to enter routine clinical care, partly due to a lack of effectiveness evidence and because therapies are often priced at the margin, based on ‘what the market can bear’. Although health economic modelling methods are suited to assess the value of PM, the myriad of challenges that may converge in modelling PM calls for additional guidance.
This workshop invites responses to the preliminary recommendations of the EU-funded HEcoPerMed project for modelling of PM, such as “If a genetic test is not part of routine care, include the downstream costs and benefits of testing for both individuals who test positive and individuals who test negative in the model” and “When effectiveness is estimated relative to a historical cohort, obtain data on the distribution and prognostic value of the gene mutation in that cohort”. It demonstrates the application of these recommendations in three case studies. The workshop concludes with an overview of financing and payment models for PM and discusses their suitability to increase the development and uptake of PM.
The workshop benefits: a) health economists working in PM, b) manufacturers of PM, c) payers, d) regulatory authorities, e) international organizations such as ICPerMed, EUnetHTA, EMA.
|Moderator: Jacoline Bouvy, NICE, London, United Kingdom|
|HTA1||Adaptive Pathways for Tumour Agnostic Medicines in Various Developed Markets|
|RodesSanchez M, Henderson N, Steuten L|
The Office of Health Economics, London, UK
|HTA2||Joint HTA in Europe to What Extent Are Reports Meeting Agency NEEDS?|
National Institute for Health and Care Excellence (NICE), Manchester, UK
|HTA3||Innovation Rating in Italy: Analysis of 77 Drug/Indication Reports (2017-2020) from AIFA|
Aiello A1, Sironi E2|
1Certara, Milano, MI, Italy, 2Università Cattolica del Sacro Cuore, Milano, Italy
|HTA4||Clinical Development and HTA Approval of MULTI-Indication Oncology Products: Evidence from Germany, France, England, Scotland, the US, Canada, and Australia.|
M, Michaeli D, Miracolo A, Kanavos
London School of Economics and Political Science, London, UK
Moderator: Dana Goldman, PhD, University of Southern California, Los Angeles, CA, USA
Margaret Anderson, MA, Deloitte Consulting LLP, Washington, DC, USA
Sir Andrew Dillon, National Institute for Health and Care Excellence, London, United Kingdom
Tomas Philipson, PhD, MA, White House Council of Economic Advisers, Washington, DC, USA
Moderator: Jan Hansen, PhD, Genentech, South San Francisco, CA, USA
Marc Boutin, JD, National Health Council, Washington, DC, USA
Rachael Callcut, MD, MSPH, University of California, San Francisco (UCSF) Medical Center, Center for Digital Health Innovation, San Francisco, CA, USA
Nigam Shah, MBBS, PhD, Stanford University, Stanford, CA, USA
Moderator: Deborah Freund, MA, MPH, PhD, Claremont Graduate University, Claremont, CA, USA
Anirban Basu, PhD, University of Washington, Seattle, WA, USA
Susan Griffin, PhD, University of York, York, United Kingdom
Darius Lakdawalla, PhD, University of Southern California, Los Angeles, CA, USA
Moderator: Howard Thom, BA, MSc, PhD, University of Bristol, Bristol, GLS, UK
Panelists: Jeroen Paul Jansen, PhD, University of California – San Francisco, San Francisco, CA, USA; Stephane Regnier, MBA, PhD, HEOR, Novartis Pharma AG, Basel, Switzerland; Sabine E Grimm, PhD, Department of Clinical Epidemiology and Medical Technology Assessment (KEMTA), Care and Public Health Research Institute (CAPHRI), Faculty of Health, Medicine and Life Sciences (FHML), Maastricht University Medical Centre+, Liege, WLG, Belgium
ISSUE: Inadequate handling of uncertainty in health technology assessment and how it can lead to inappropriate decision making. We will debate reasons for these inadequacies and the panelists’ proposed solutions.
OVERVIEW: Health technology assessment (HTA) is often faced with uncertainty, for example on the correct parameter values or structure for a model, extrapolation of short-term effects, differences in risk aversion between patients, or variation in individual responses. Healthcare decision making often takes inadequate account of this uncertainty. Reasons include that well known techniques for addressing uncertainty, such as probabilistic analyses, model averaging, or value of information (VoI), are inadequately employed and that uncertainty communication remains challenging. Our panel will draw on the latest research to debate the impact and reasons for these inadequacies, and the best approaches to managing uncertainty. This will be done through 5-10 minute presentations by the moderator and panelists, who each will share their experience and research on uncertainty assessment and management. Howard Thom will present a general overview and research on worst-case impact of ignoring uncertainty, Sabine Grimm will present the decision maker’s perspective and research linking VoI to managed access agreements, Stephane Regnier will provide an industry perspective and research on inferring causal treatment links, and Jeroen Jansen will discuss variation in patient response and risk appetites.
Panelists will poll the audience to determine which type of uncertainties can have the biggest consequence and whether they believe current methodologies are appropriate to deal with the most serious uncertainties. There will be 30 minutes for debate and audience participation. Audience members will be able to submit questions through live polling or in person.
Healthcare decision makers, manufacturers preparing technology submissions, and consultants advising on submissions would benefit practically from attending this panel. However, anyone working in HTA will find our discussion relevant and informative.
Moderator: Nancy Joy Devlin, PhD, Centre for Health Policy, University of Melbourne, Melbourne, VIC, Australia
Panelists: Mike Paulden, PhD, School of Public Health, University of Alberta, Edmonton, AB, Canada; Mikel Berdud, PhD, Office of Health Economics, London, LON, UK; Laura Vallejo-Torres, PhD, Departamento de Métodos Cuantitativos en Economía y Gestión, Universidad de Las Palmas de Gran Canaria, Las Palmas, Spain
ISSUE: In many healthcare systems, reimbursement decisions are based on health technology assessments using a cost-effectiveness threshold (CET) to support decisions. The CET acts as a price ceiling to the value-based price. What should the CET reflect and how does that split value between developers and payers? Should payer and developer surplus coming from multiple indications or across multiple health systems be considered when setting the CET value? Should payer and developer bargaining power be considered too?
OVERVIEW: This panel will discuss the implications of setting CETs for value distribution between payers (consumers) and developers (producers). They will debate how CETs should be determined.
Nancy Devlin will introduce the issue and moderate the panel, ensuring debate and audience participation.
Mike Paulden will discuss theoretical work that informed a recent technical report for the Canadian PMPRB. This work implies that short run payer benefit is maximised when the CET is set below the supply side opportunity cost threshold. Mike will also describe issues for considering payer and developer surplus arising from health technologies with different market size, across multiple indications, and across multiple public health systems.
Mikel Berdud, from an economics of innovation perspective, will argue that different degrees of payer and developers bargaining power affect the optimal CET (i.e. maximises access in the short run without compromising innovation in the long-run) and, if some conditions hold, it may make sense to set the CET above the supply side CET.
Laura Vallejo Torres will comment on the supply and demand perspectives for setting a CET, based on her work estimating a CET in Spain using both approaches. She will discuss methodological aspects and issues around different decision-making contexts in relation to the use of CETs to inform decisions. Implications for the share of value apportion to payers and developers will be discussed.
Discussion Leaders: Sven L Klijn, MSc, Pharmerit - an OPEN Health Company, Rotterdam, Netherlands; Katharina Nickel, MSc, Pharmerit - an OPEN Health Company, Berlin, Germany; Bill Malcolm, MSc, Bristol Myers Squibb, Uxbridge, UK; Nicky Welton, PhD, Bristol Medical School, University of Bristol, Bristol, UK
PURPOSE: Extrapolation of survival curves for cost-effectiveness analyses often rely on a single parametric distribution fitted to relatively short-term trial data. Extrapolations can be very sensitive to the choice of distribution, even when the distributions fit equally well to the short-term data, causing structural uncertainty. Sensitivity analyses to address parameter uncertainty do not account for this structural uncertainty. Further, the selection of the parametric distribution is frequently scrutinized by decision-makers, especially for therapies exhibiting a long tail in survival, e.g. immuno-oncology therapies. Methods available to address structural uncertainty in survival extrapolation include Bayesian Model Averaging (BMA) which pools results from different distributions and use of external evidence to provide constraints. This workshop aims to 1) answer the question, how BMA and external data can be utilized in survival extrapolations, and 2) debate the value of this approach.
DESCRIPTION: This workshop will present how BMA and external data can be utilized to reduce uncertainty in survival extrapolations, based on practical examples. First, an overview of current practices and related uncertainties will be presented from an industry perspective (15 minutes). The concept of BMA will be introduced based on an illustrative case study using BMA for several indications (15 minutes). The BMA approach will also be discussed within the context of HTA appraisals. Further, appropriate external data sources and how these can be incorporated in the BMA will be presented (20 minutes). The workshop will conclude with a debate, involving the audience, to discuss the suitability of the BMA approach to reduce uncertainty (10 minutes). Throughout the workshop, the audience will be encouraged to participate in the discussion either in-person or via polling, evaluating the suitability of the presented approaches. The workshop will provide illustrative examples and will be relevant for a broader audience including data analysts, industry representatives, and decision-makers alike.
Discussion Leaders: Rita Faria, MSc, Centre for Health Economics, University of York, York, UK; Elisabeth Fenwick, PhD, Pharmerit International, Oxford, OXF, UK; Jon Tosh, PhD, Evidence Synthesis, Modelling and Communications, Evidera, London, UK; Rebecca Albrow, MPH, Diagnostics Assessment Programme, National Institute for Health and Care Excellence, Sale, UK
PURPOSE: To share and compare approaches to the design and communication of cost-effectiveness models of complex pathways of care, to ensure stakeholder engagement and enhance the models’ value for decision-makers.
DESCRIPTION: Even with rising costs of pharmaceuticals and procedures, the majority of health budgets globally continue to be allocated to health professionals and service delivery. Changes to complex pathways and health services represent cost-effective opportunities to improve population health, however there are many challenges with estimating the costs and benefits of changes to pathways and services.
This workshop will shine a light on a relatively under-researched topic, and will also highlight how cost-effectiveness models can inform decisions about pathway reconfiguration and/or further data collection/research before real-world testing and implementation.
Elisabeth Fenwick will chair the session and introduce the topic, drawing on her experience in cost-effectiveness modelling.
Rita Faria will present a cost-effectiveness model of cascade screening of relatives of known patients with genetic familial hypercholesterolaemia, specifically the journey from the model's conceptualisation to implementation and how stakeholders informed its design and parameterisation.
Jon Tosh will present a cost-effectiveness model of service improvements for longer-term depression. A conceptual and simulation model were developed in collaboration with service users, to ensure the analysis best reflected their experience and ideas for improvements.
Rebecca Albrow will present real-world examples of uncertain care pathways that have been encountered when developing NICE diagnostics guidance, and explore how uncertainties were reconciled by the diagnostics advisory committee during its discussions on whether, on balance, to recommend technologies for use in the National Health Service in England.
This interactive workshop will be equally relevant for those tasked with developing cost-effectiveness models and for those commissioning or using their results.
Discussion Leaders: Venediktos Kapetanakis, PhD, Evidera, London, UK; Murtuza Bharmal, MD, Global Evidence & Value Development, EMD Serono Inc., Rockland, MA, USA; K. Jack Ishak, PhD, Evidera, St-Laurent, QC, Canada
PURPOSE: This workshop will demonstrate through an application in patients with metastatic urothelial carcinoma in the second-line setting, the use and impact of assessment schedule matching (ASM), a novel method for adjusting for assessment time bias (ATB) when conducting unanchored indirect treatment comparisons of progression-free survival.
DESCRIPTION: Methods to adjust for imbalances in baseline patient characteristics between trials are available and often used in health technology assessments (HTAs). However, these methods cannot adjust for trial design differences including the schedule of assessments for disease progression. This can impact the comparison of progression-free survival (PFS), as studies can adopt different schedules of assessment depending on the length of treatment cycles. This in turn leads to progressions being detected at different intervals. Thus, even if two treatments had an identical risk of progression and death, the PFS curves observed in the two trials can appear different, which can then affect cost-effectiveness assessments. We demonstrate the effect of differences in assessment schedules in indirect comparisons of PFS between avelumab and four other immunotherapies (atezolizumab, nivolumab, durvalumab, pembrolizumab). Matching adjusted indirect comparisons are carried out to account for between-study differences in effect modifiers and prognostic factors on the originally reported PFS outcomes. The comparisons are then repeated after aligning the first assessment time for avelumab to each comparator’s schedule with the ASM method. We describe the relative influence of adjusting for population and schedule differences and show that the magnitude of change from the latter can equal or exceed distortions due to population differences.
Participants will gain an understanding of how to account for ATB when comparing PFS between trials, the underlying assumptions of ASM and how to use it appropriately. The workshop is directed at healthcare decision-makers, statisticians and individuals commissioning health-economic evaluations.
|Moderator: Ramiro E. Gilardino, MD, MHS, MSc., Managing Director – HE-Xperts Consulting, Miami, Florida, USA|
|CV2||Patients' Preferences for Once-Daily Oral Versus Once-Weekly Injectable Diabetes Medications: The REVISE Study|
Ross M2, Mody R3, Konig M3, Gelhorn H4|
1Eli Lilly and Company, Greenwood, IN, USA, 2Evidera, Bethesda, MD, USA, 3Eli Lilly and Company, Indianapolis, IN, USA, 4Evidera, Winter Park, CO, USA
|CV4||Patients with Hypercholesterolaemia or MIXED Dyslipidaemia and High or VERY High Cardiovascular Risk According to ESC/EAS Guidelines – a Population-Based CROSS–Sectional Study from the Netherlands|
|Heintjes E1, Kuiper J1, AnastassopoulouA2, Penning-van Beest F1,
Bilitou A2, Kerschnitzki
M2, Herings R1, Postma M3,
1PHARMO Institute for Drug Outcomes Research, Utrecht, Netherlands, 2Daiichi Sankyo Europe, Munich, Germany, 3University Medical Center Groningen, Groningen, Netherlands, 4Leiden University Medical Center, Leiden, Netherlands
Moderator: Daniel Ollendorf, PhD, Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA
Panelists: Valentina Strammiello, MA, • European Patients’ Forum, Brussels, Belgium; Michael Drummond, MCom, DPhil, Centre for Health Economics, University of York, York, YOR, UK; Adrian Griffin, MSc, HTA & Reimbursement Policy, Johnson & Johnson, Buckinghamshire, UK
ISSUE: HTA is a growing phenomenon worldwide. As these enterprises mature and seek additional efficiencies, there has been an increase in “importation” of the outputs of assessments of the value of health technology conducted in a different setting, including multi-country evaluation, reference pricing, and other elements. Yet countries may differ widely in economic indicators, health system and reimbursement structure, HTA approach and governance, and sociopolitical context. These differences collectively present challenges to appropriate application and transfer of value assessment across settings.
OVERVIEW: This panel will discuss the challenges and appropriate application of the transfer of value assessment outputs between settings. Dan Ollendorf will moderate the session and will briefly (5-7 minutes) present findings of recent research on economic, political, and health-system differences across 6 diverse countries, their approach to HTA, and examples of differences in the structure and outcome of value assessments across them. Each panelist will then be given 12 minutes to speak. Valentina Strammiello will discuss concerns in the patient community with imported and multi-country value assessments in terms of medication adoption and access. Mike Drummond will examine the transferability of value assessment, with a focus on what is and is not feasible, the potential benefits and drawbacks of transferred value assessments within HTA, assessment elements to be most concerned about, and red flags for transferability. Adrian Griffin will discuss the merits of locally tailored value assessments and challenges to transferring such assessments across markets from an industry perspective. Panelists will discuss and debate the feasibility, cultural context, and appropriateness of importing different elements of value assessments from other settings, how stakeholder definitions of “appropriate” may differ, and potential implications for access, reimbursement decisions, and price negotiations.
|Moderator: Paul Revill, Research Fellow, University of York, United Kingdom|
A Model-Based Estimation of the Cost-of-Illness Associated with Neisseria gonorrhoea in England and the USA: Assessing the Potential Impact of Antimicrobial Resistance and Long-Term Health Problems
*Top 10% Average Review Score
L1, Peultier AC2, Whelan J3, Beck E4|
1GSK, Wavre, Belgium, 2Erasmus University Rotterdam, Rotterdam, Netherlands, 3GSK, Amsterdam, Netherlands, 4GSK, Munich, BY, Germany
|IN3||Impact of Government Measures on COVID-19 Incidence of Reported New Cases: An Analysis of 9 Countries|
S1, Ray B2, Holy C3,
Sakthivel M1, Elangovanraaj N1,
Krishnan D4, Gupta S5, Trivedi P1, Devulapally M1, Mohapatra A1, Coplan P6|
1Mu Sigma, Bangalore, KA, India, 2Mu Sigma, Bangalore , India, 3Johnson & Johnson, Somerville, MA, USA, 4Mu-Sigma, Bangalore, India, 5Mu Sigma, Bangalore, India, 6Johnson & Johnson, New Brunswick, NJ, USA
|IN4||Economic Impact of Routine Childhood Immunization in the 2017 United States Birth Cohort|
J1, La E1, Talbird SE1, Chen YT2, Nyaku MK2, Carias C2,
Marshall GS3, Roberts C4
*Top 10% Average Review Score
|Moderator: Praveen Thokala, MASc, PhD, Senior Research Fellow, School of Health and Related Research (ScHARR), The University of Sheffield, UK|
|ML1||Do Machines Perform Better THAN Humans at Systematic Review of Published Literature? a Case Study of Prostate Cancer Clinical Evidence.|
L1, Witzmann A2, Bednarski M3, Sumner M4, Baehrens D4|
1F. Hoffmann La Roche, Basel, Switzerland, 2F. Hoffmann La Roche, Kaiseraugst, Switzerland, 3Roche Polska Sp. z o.o., Warsaw, Poland, 4Averbis GmbH, Freiburg, Germany
|ML2||Network Meta-Analysis (NMA) Powered By Artificial Intelligence.|
|Hvingelby R, Joshi S, Holm-Larsen T|
Silvi.ai (A-Evidence), Copenhagen, Denmark
|ML3||Detection of Quality of Life IMPACT in Health-Related Messages in Social MEDIA|
T, Khadhar M, Renner S, Foulquié
P, Voillot P, Mebarki A, Texier N, Schück S|
Kap Code, Paris, France
|ML4||Application of Machine Learning Models to Evaluate COVID-19 Related ICU Utilization in a US Population|
Chan C1, Munsell M1, Menzin J2|
1Boston Health Economics, Boston, MA, USA, 2Boston Health Economics, LLC, Boston, MA, USA
|Moderator: Nicky Welton, Professor of Statistical and Health Economic Modelling, University of Bristol, United Kingdom|
|NM1||A Comparison on Different Network-Meta Analysis (NMA) Models Using Randomized Clinical Trials (RCTS) in Transplant-Ineligible (TIE) NEWLY Diagnosed Multiple Myeloma (NDMM)|
van Beekhuizen S2, Verhoek A2,
Postma MJ3, Ouwens M4, Heeg B2|
1Ingress-health, Tilburg, NB, Netherlands, 2Ingress-health, Rotterdam, Netherlands, 3University of Groningen, University Medical Center Groningen, Groningen, Netherlands, 4AstraZeneca, Mölndal, Sweden
|NM2||Using Randomized and Observational DATA to Predict Heterogeneous Treatment Effects|
Pellegrini F2, Salanti G1|
1University of Bern, Bern, Switzerland, 2Biogen International GmbH, Baar, Switzerland
|NM3||Extending the Network Meta-Analysis (NMA) Framework to Multilevel Network Meta-Regression (ML-NMR): A Worked Example of ML-NMR VS Standard NMA|
S1, Sharma A2,
1Parexel International, Mohali, PB, India, 2Parexel International, Mohali, India
|NM4||Flexible Generic Framework for Evidence Synthesis in Health Technology Assessment|
T1, Pellegrini F2,
Subramaniam S3, Salanti G4|
1University of Bern, Bern, BE, Switzerland, 2Biogen International GmbH, Baar, Switzerland, 3University of Basel, Basel, Switzerland, 4University of Bern, Bern, Switzerland
|Moderator: Beth Woods, Senior Research Fellow, TEEHTA, Center for Health Economics, University of York, York, UK|
|MS1||Cost-Effectiveness Analysis of Colorectal Cancer Screening Programmes:How Omissions of Relevant Comparators CAN Lead to Inefficient Policies|
Pokharel R, O'Mahony JF|
Trinity College Dublin, Dublin, Ireland
|MS2||Methods to Optimise Survival Predictions Using Multistate Models in Oncology|
|WigfieldP1, Verhoek A2, Ouwens DM3, Heeg B1|
1Ingress-Health Nederland B.V., Rotterdam, ZH, Netherlands, 2Ingress-Health, Rotterdam, ZH, Netherlands, 3AstraZeneca, Mölndal, Sweden
|MS3||Reproduction of Published Health Economic Obesity Models: Assessment of Facilitators, Hurdles and Barriers|
M2, Hiligsmann M3, Evers S4|
1AHEAD GmbH, Lörrach, Germany, 2Istanbul Technical University, Istanbul, Turkey, 3Maastricht University, Maastricht, LI, Netherlands, 4Maastricht University / Trimbos Institute, Maastricht / Utrecht, Netherlands
|MS4||How to Assess the Lifetime IMPACT of Lifestyle Interventions for Type 2 Diabetes in Germany? a Markov MODEL Design and Validation|
M1, Dubois C1, Altin S2, Leppert N3,
Altpeter B3, Amelung
1Private Institute of Applied Health Service Research (inav GmbH), Berlin, BE, Germany, 2AOK Rheinland/Hamburg, Düsseldorf, Germany, 3Deutsches Institut für Telemedizin und Gesundheitsförderung (DITG), Düsseldorf, Germany
Moderator: Esther de Bekker-Grob, PhD, Erasmus University Rotterdam, Rotterdam, Netherlands
Panelists: Caroline M Vass, PhD, RTI Health Solutions, Manchester, UK; Gemma Shields, BSc, MPH, Manchester Centre for Health Economics, The University of Manchester, Manchester, LAN, UK; Lucy Abraham, MSc, Pfizer Ltd, Tadworth, Surrey, SRY, UK
ISSUE: Healthcare decision makers are increasingly interested in incorporating patient preference information into their research and policy-related activities. However, many guidelines and working documents often neglect to define what a ‘patient’ is, despite the desire to elicit data from them. The panel will debate the role of preference data specifically from patients and discuss circumstances when preferences from alternative stakeholders could be acceptable. The panel offers academic, industry, and methodological perspectives. Esther de Bekker-Grob will introduce and moderate the panel. Caroline Vass will outline the need for a broad definition of the ‘patient’, drawing on her experience of eliciting preferences for screening and diagnostic tests. Lucy Abraham will provide an industry perspective, illustrating how Pfizer used patient preferences for chronic pain treatments. Gemma Shields will critically summarize how the public’s preferences are currently incorporated within the quality-adjusted life-year (QALY) framework of health technology assessments (HTA). Each panelist will speak for 12-15 minutes on the opportunities and challenges associated with eliciting patients’ and, when relevant, other stakeholders’ preferences for HTA and regulatory decisions. A 15-minute question-and-answer session will follow.
OVERVIEW: Patient preference data are increasingly being used to inform healthcare decisions. Although most documentation by regulators advocates the use of preference data from patients, there exist few definitions of what constitutes a ‘patient’. For some technologies, such as cancer screening or genetic testing, individuals without the condition may become a patient after consumption. In other instances, the patient may not be able to reliably state their preferences, such as young children or those with significant cognitive impairments. For many decisions, even individuals with the disease may not have consumed the technology unless they were in the treatment arm of a trial. Should clinical and economic decision makers consider patient preference data exclusively or should the views of other stakeholders be considered?
Moderator: Wim Goettsch, PhD, WHO Collaborating Centre for Pharmaceutical Policy, Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht University, Diemen, Netherlands
Panelists: Milou Hogervorst, PharmD, MSc, Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht University, Utrecht, Netherlands; Georgia Salanti, Prof., PhD, University of Bern, Bern, Switzerland; Alric Rüther, Dr. med., Institute for Quality and Efficiency in Health Care (IQWiG), Cologne, Germany
ISSUE: According to HTA organisations, many challenges in HTA are expressed during the relative effectiveness assessment (REA). These challenges iterate in the cost-effectiveness assessment (CEA) and decision making (appraisal) on policies and reimbursement. A recent detailed assessment as part of the H2020 HTx project shows that the roots of these challenges often lie in the (un)availability of data, e.g., immature data, surrogate outcomes, lack of QoL data, etc. What are the solutions to address these challenges and increase certainty in policy and reimbursement decisions? On the one hand, the quality and quantity of available data can be increased for example with trial guidelines and more scrutiny from regulatory institutions. On the other hand, methods for evidence synthesis using only ‘limited data’ can be improved by for example focusing on indirect data comparisons and combining data sources.
OVERVIEW: The debate will be introduced with a presentation (10 minutes) on recent results from the H2020 HTx project, that identified challenges in HTA of advanced health technologies, like gene therapies, combined therapies and sequences on the basis of information received from 22 HTA agencies around Europe. The panelists will then each present their viewpoints on the most preferable solution for these challenges (2 x 10 minutes). Hereafter, the audience will debate with the panelists, guided by the moderator, on the most relevant and feasible for implementation solutions (30 minutes). The aim of the panel session is to provide stakeholders dealing with this dilemma, e.g. regulatory institutions, HTA organisations, industry representatives, academics in HTA / evidence synthesis methods, epidemiologists, etc., with solutions for and future perspectives on the data challenges that eventually result in uncertainties in policy decisions.
|Moderator: Aleksandra Torbica, PhD, Associate Professor, Bocconi University, Italy|
|SP1||Matching Techniques in Stated Preferences for Health|
AJ1, Vass C2
*Top 10% Average Review Score
|SP2||What Is the Relative VALUE of Carer and Patient Quality of Life? a Person Trade-Off (PTO) Study with the UK Public|
Wittenberg E2, Donaldson C3, Brouwer W4|
1University of Birmingham, Birmingham, UK, 2Harvard School of Public Health, Boston, MA, USA, 3Glasgow Caledonian University, Glasgow, UK, 4Erasmus School of Health Policy & Management, Rotterdam, Netherlands
|SP3||Should I Choose Artificial Intelligence or Clinicians' Diagnosis? a Discrete Choice Experiment of Patients' Preference UNDER COVID-19 Pandemic in China.|
T1, Tsang W2,
Huang F2, Ming WK2|
1University of Groningen, Liyang, 32, China, 2Jinan University, Guangzhou, China
|SP4||A Longer Life or a Quality Death? a Discrete Choice Experiment to Estimate the Relative Importance of Different Aspects of END-of-Life Care|
1Office of Health Economics, London, UK, 2University of East Anglia, Norwich, UK
|Moderator: Aureliano Finch, Scientist, EuroQol Research Foundation, Netherlands|
|PR1||Comparison of the Psychometric Performance of a New Condition-Specific Preference-Based Measure Derived from the Cfq-R (CFQ-R-8D) to EQ-5D-3L and SF-6D to Evaluate Health-Related Quality-of Life (HRQOL) in People with Cystic Fibrosis (CF)|
Rowen D1, Brazier JE1, McGarry L2, Quittner A3, Lou Y2, Sosnay P2, Acaster S4
*Top 10% Average Review Score
|PR3||Patient-Reported Outcome Measures to Prioritize Surgical Lists during Pandemic Events|
Rosis S1, Lungu DA2, PennucciF2, Nuti S2|
1Sant'Anna School, pisa, PI, Italy, 2Sant'Anna School, Pisa, PI, Italy
|PR4||The IMPACT of Removing Immediate Death from TTO on CHILD Health State Valuation|
1SHE, Brussels, VBR, Belgium, 2EuroQol Research Foundation, Rotterdam, Netherlands
|Moderator: David Epstein, Associate Professor, Department of Applied Economics, University of Granada, Granada, Spain|
|ON1||Comparison of Overall Survival (OS) Extrapolations of Immune-Checkpoint Inhibitors (ICI) in Efficiency Opinions of French 'Haute Autorite de Sante' (HAS) with Updated OS data: Were the Projections Accurate?|
S2, Chevalier J3, Gaudin AF4, Branchoux S5
*Top 10% Average Review Score
|ON2||Assessing the Performance of Landmark Response Analysis to Improve Overall Survival Extrapolation in Nivolumab NSCLC Trials|
MA1, Rafiq S2,
Siddiqui MK3, Vanderpuye-Orgle J4,
Edmonson-Jones M2, Teitsson S2,
Juarez-Garcia A5, Penrod JR1
*Top 10% Average Review Score
|ON3||Comparison of External Control Arm (ECA) Critiques in Regulatory and Health Technology Assessment (HTA) Oncology Submissions|
Vondeling G2, Martin L3, Draganigos A3, Louder A1, Rosenlund M3
*Top 10% Average Review Score
|ON4||Heterogeneity in Survival with Immuno-Oncologic Therapy and Its Implications for Survival Extrapolations: A Case Study in Untreated Metastatic Melanoma|
V1, Kurt M2, Zhang
L3, Amadi A4, Hernlund E5, Johnson HM4, Moshyk A6, Borrill J4|
1ICON plc, New York, NY, USA, 2Bristol Myers Squibb, Princeton, NJ, USA, 3ICON plc, London, UK, 4Bristol-Myers Squibb, Uxbridge, UK, 5ICON plc, Stockholm, Sweden, 6Bristol-Myers Squibb, Lawrenceville, NJ, USA
|Moderator: Amr Makady, PhD, PharmD, Health Economics & Market Access Manager, Johnson & Johnson, Breda, Netherlands|
|RW1||Bridging The Gap Between Clinical Trials And Real World Data: Evidence On Replicability Of Efficacy Results Using German Claims Data|
Maywald U2, Wilke T3, Heeg B4
*Top 10% Average Review Score
|RW2||Synthetic Control Arms: Hype Versus Reality, the Payer Perspective|
R1, Cable G2, Siddiqui MK3, Lucas J4|
1Parexel International, London, UK, 2Parexel International, Billerica, MA, USA, 3Parexel International, Mohali, India, 4Parexel International, Basel, Switzerland
|RW3||The Use of Real-World Evidence in HTA of CLASS III Medical Devices in Europe.|
P1, de Groot S2, Blommestein HM3, Pongiglione
B4, Torbica A5, Al MJ2
*Top 10% Average Review Score
|Moderator: Katarina Steen Carlsson, Associate Professor, Lund University, Sweden|
|PA1||Different Levels of Care for Follow-up of Adults with Congenital Heart Disease|
F2, Goossens E2, De Groote K3,
Budts W2, Moniotte
S4, de Hosson M3, Van Bulck L2, Marelli A5, Moons P2,
De Backer J3, Annemans L6|
1Ghent University, Gent, VOV, Belgium, 2Leuven University Hospitals, Leuven, Belgium, 3Ghent University Hospital, Gent, Belgium, 4Cliniques Universitaires Saint-Luc, Brussels, Belgium, 5McGill University, Montréal, QC, Canada, 6Ghent University, Ghent, Belgium
|PA2||Treatment Intensification and Risk of Cardiovascular Events in Insulin-Treated Patients with Type 2 Diabetes; A United Kingdom Retrospective Cohort Study|
Hag Hersi M1, Seidu
S2, Khunti K2, Zaccardi F2, Gillies C3, Webb DR3,
Lubwama R4, Boss A4, Dex T4
*Top 10% Average Review Score
|PA3||New Primary Care Incentive Scheme for Type 2 Diabetes in Denmark|
CV, Olsen KR|
University of Southern Denmark, Odense, 83, Denmark
|PA4||Delaying Assistance Intensification Among Elderly with Assisted Living Services: Care Costs, Target Group Profile, and IMPACT Estimation for a Potential Social IMPACT Bond (SIB) in Finland|
R2, Häkkinen E3, Vähäkangas P4, Hammar
M5, Björndahl-Öhman J4, Pyykkö M6, Tonteri A6|
1ESiOR Oy, Kuopio, 15, Finland, 2ESiOR Oy, Kuopio, Finland, 3The South Savo Social and Health Care Authority, Mikkeli, Finland, 4Vaasa Hospital District, Vaasa, Finland, 5Vaasa Central Hospital, Vaasa, Finland, 6Ministry of Economic Affairs and Employment of Finland, Helsinki, Finland
|Moderator: Nick Latimer, MSc, PhD, Reader in Health Economics, ScHARR, University of Sheffield, United Kingdom|
|EX1||How to Select the Appropriate Survival Extrapolation Method? a Proposed Framework|
Oostrum I1, Pham HA1,
Verhoek A1, Garcia A2, Postma MJ3, Ouwens M4, Heeg B1|
1Ingress-Health Nederland B.V., Rotterdam, ZH, Netherlands, 2Ingress-Health Nederland B.V., rotterdam, Netherlands, 3University of Groningen, University Medical Center Groningen, Groningen, Netherlands, 4AstraZeneca, Mölndal, Sweden
|EX2||Structural Uncertainty in Survival Extrapolation: Exploring the Impact of Four Model Averaging Methods and Adjusting for Data Maturity|
Lee D1, Sly I2, Kearns B3|
1BresMed Health Solutions, Sheffield, DBY, UK, 2NHS England, London, UK, 3University of Sheffield, School of Health and Related Research, Sheffield, UK
|EX3||Investigating Time to Next Treatment As a Surrogate Endpoint for Overall Survival in Previously Untreated Intermediate- to Poor-Risk Advanced Renal Cell Carcinoma Patients: An Insight from the Phase III CheckMate-214|
CL2, Kurt M3, Gaudin AF4, Italiano A5, Rondeau V2, Bellera C6|
1Bristol Myers Squibb, RUEIL MALMAISON, France, 2Bordeaux Population Health Center, ISPED, Centre INSERM U1219, Bordeaux, France, 3Bristol Myers Squibb, Princeton, NJ, USA, 4Bristol-Myers Squibb, Rueil-Malmaison, France, 5Institut Bergonié Comprehensive Cancer Centre, Bordeaux, France, 6Epicene Team (Cancer & Environnement), Bordeaux Population Health Center, ISPED, Centre INSERM U1219, Bordeaux, France
|EX4||Bayesian MULTI-Parameter Evidence Synthesis to Improve LONG TERM Survival Extrapolation with EARLY Follow up DATA from Nivolumab NSCLC Studies|
S1, Chaudhary MA2,
Siddiqui MK3, Vanderpuye-Orgle J4,
Edmonson-Jones M1, Teitsson S1,
Juarez-Garcia A5, Penrod JR2|
1Parexel International, London, LON, UK, 2Bristol-Myers Squibb, Princeton, NJ, USA, 3Parexel International, Mohali, India, 4Parexel International, Billerica, MA, USA, 5Bristol-Myers Squibb, Uxbridge, UK
|Moderator: Thor-Henrik Brodtkorb, PhD, RTI Health Solutions, Sweden|
|MT1||Cost-Effectiveness Analyses of an Absorbable Antibacterial Envelope for Use in Patients at Increased Risk of Cardiac Implantable Electronic Device Infection in Three European Countries|
Kennergren C2, Tarakji
KG3, Wright DJ4, Ahmed FZ5, McComb JM6, Goette A7,
Blum T8, Biffi M9, Green M10,
Shore J10, Carion PL11, Wilkoff BL3|
1University of Modena and Reggio Emilia, Policlinico di Modena, Modena, Italy, 2Sahlgrenska University Hospital, Göteborg, Sweden, 3Cleveland Clinic, Cleveland, OH, USA, 4Liverpool Heart and Chest Hospital, Liverpool, UK, 5Manchester University NHS Foundation Trust, Manchester, UK, 6The Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK, 7Saint Vincenz Hospital, Paderborn, Germany, 8Universitaets-Herzzentrum Freiburg Bad Krozingen, Bad Krozingen, Germany, 9Policlinico Sant' Orsola – Malpighi, Bologna, Italy, 10York Health Economics Consortium, York, UK, 11Medtronic International Trading Sàrl, Paris, France
|MT2||Discrepancy between the Cost of an Innovative Medical Procedure and Its Payment: The Case of Mechanical Thrombectomy Procedures in ACUTE Ischemic Stroke in France|
K1, Chauvin P1, Piotin M2, Lapergue
1University Paris Descartes, Paris, France, 2Fondation Ophtalmologique Adolphe de Rothschild, Paris19, 75, France, 3Hôpital Foch, University Versailles Saint Quentin en Yvelynes, Suresnes, France
|MT3||A Review of the National Institute for Health and Care Excellence (NICE) Medical Technologies Guidance over the Last 10 Years|
Harper S, Wright DL
*Top 10% Average Review Score
|MT4||The Cost-Effectiveness of Selective Internal Radiation Therapy (SIRT) for Treating Advanced Unresectable Hepatocellular Carcinoma in the United Kingdom|
M1, Claxton L2,
Sharif-Hurst S2, Wade R2, Eastwood A2,
*Top 10% Average Review Score
|Moderator: Martin Henriksson, Associate Professor, Linköping University, Linköping, Sweden|
|RE1||Financing and Reimbursement for Personalised Medicine - Are WE There YET?|
|Koleva-KolarovaR1, Buchanan J1,
Wordsworth S2, Tsiachristas A1|
1University of Oxford, Oxford, UK, 2University of Oxford, Oxford , OXF, UK
|RE2||A Review of Recently Proposed Policy Innovation in the US Aimed at Driving the Implementation of VALUE-Based Pricing Agreements|
Amaris Consulting, London, LON, UK
|RE3||Advancing Outcomes-Based Pharmaceutical Reimbursement Models Using Structured Risk and Investment Funding|
T1, Brar SS1, Ray A1, HinkelJM2|
1Lydion Research, Los Angeles, CA, USA, 2University of Oxford, Incline Village, NV, USA
|Moderator: David Epstein, Associate Professor, Department of Applied Economics, University of Granada, Granada, Spain|
|RO1||Barriers and Potential Solutions for Implementation of Outcome-Based Spread Payments for High-Cost, One-SHOT Curative Therapies|
S1, Nachi S2, Van
Dyck W3, Simoens S2, Huys I2|
1University of Leuven, Leuven, VBR, Belgium, 2University of Leuven, Leuven, Belgium, 3Vlerick Business School, Brussels, Belgium
|RO2||Analysis of Reimbursement Outcomes for Orphan Drugs with EMA Exceptional Circumstances Designation in France, Germany, and the UK|
Pisarczyk K1, Beckerman R2, Meletiche D3|
1Maple Health Group, LLC, Krakow, Poland, 2Maple Health Group, LLC, New York, NY, USA, 3Maple Health Group, LLC, Cambridge, MA, USA
|RO3||Using Proms in Health Technology Assessment for RARE Disease Treatments|
Nicod E1, Meregaglia
M2, Drummond M3|
1Bocconi University, Milan, MI, Italy, 2Bocconi University, Milan, Italy, 3University of York, York, YOR, UK
|RO4||Comparison of Health Technology Assessments and Time to Reimbursement for Orphan Drugs from FOUR HTA Agencies: Opportunities and Challenges in France, Germany, England and Scotland|
Poon C, Raj N, Kelly S|
Charles River Associates, London, UK
Moderator: Katja Rudell, PhD, MSc, Parexel Access Consulting, Parexel International, Chittering, UK
Speakers: Lynda Doward, Master of Research, Patient-Centered Outcomes Assessment, RTI Health Solutions, Didsbury, UK; Bryan Bennett, PhD, Bristol Myers Squibb (BMS), Uxbridge, Middlesex, UK; Angela J Jane Rylands, PhD, CPsychol, Kyowa Kirin International plc, UK, Marlow, BKM, UK; Ana Maria Rodriguez, PhD, MSC, PT, RWE Patient-Centered Endpoints CoE, IQVIA, Madrid, Spain
Outcomes Assessment (COA) Special Interest Group’s focused this past year on harmonizing COA nomenclature and value as well as align guidance for COA generation in real world (RW) studies which are often presented to regulatory and HTA agencies.
Bryan Bennett and Lynda Doward will discuss the results of a membership survey which identifies how different stakeholders value PRO data in their professional decision-making based on their current and projected use of COA data . Additionally as part
of the key project, they will present the preliminary findings from a review of HTA terminology used for COA from different countries that will help to inform our understanding with an aim to a degree of harmonize the way Health Economic and Outcome Research
scientists and COA specialists describe COA value. Angela Rylands and Ana Maria Rodriguez would like to involve COA experts attending ISPOR in aligning next steps in their proposal of new guidelines and best practices for COA in real world evidence (RWE)
studies. They will share a draft manuscript framework, which aims to summarize the output from the virtual expert meeting and international survey with industry, regulatory and HTA/payer agencies hosted at the end of 2019 which collected views on the
use of COA in RWE.
Moderator: Mickaël Hiligsmann, PhD, Department of Health Services Research, School for Public Health and Primary Care, Maastricht University, Maastricht, LI, Netherlands
Speakers: Tamas Agh, MD, PhD, MSc, Syreon Research Institute, Budapest, Hungary; Andrew Peterson, PharmD, PhD, FCPP, Substance Use Disorders Institute, John Wyeth Dean Emeritus, Professor of Clinical Pharmacy and Professor of Health Policy, University of the Sciences, Philadelphia, PA, USA
non-adherence is prevalent across most clinical conditions causing major medical and economic challenges. Although several studies have demonstrated that medication adherence enhancing interventions (MAEIs) may improve health outcomes, existing
evidence on the effectiveness of MAEIs remains of poor quality. Important policy decisions about MAEIs are therefore currently based on inadequate or suboptimal information. The Medication Adherence and Persistence Special Interest Group (MAP-SIG)
is conducting a systematic evaluation of the evidence associated with MAEIs by identifying and prioritizing relevant criteria for their value assessment from the perspectives of different stakeholders. Attendees interested in medication adherence
and developing consensus-based criteria to assess value of MAEIs are welcomed to join this forum. A brief presentation on the purpose of the initiative and findings of the systematic literature review on criteria for assessing MAEIs will be
presented. This will be followed by an open discussion on the criteria. The attendees will be asked to: 1) share their opinions on identified criteria, 2) suggest improvements to criteria presented, and 3) recommend additional criteria. After
a final list of criteria is obtained, the audience members will rank the presented criteria in order of importance, via the polling app. Lastly, the group will be asked to share their perspectives and discuss ideas on how these criteria can
be used to improve medication adherence studies. Grab your lunch and join us for this informative discussion.
Moderator: Richard Charter, MSc, MedTech Market Access Europe & Asia Pacific, Alira Health, Basel, Switzerland
Speakers: Federico Augustovski, MSc, PhD, Institute for Clinical Effectiveness and Health Policy, Buenos Aires, B, Argentina; Giuditta Callea, PhD, Centre for Research on Health and Social Care Management (CeRGAS), SDA Bocconi School of Management, Milan, MI, Italy; Luqman Tariq, PhD, Stryker, Amsterdam, Netherlands; Gastón Gabín, MD, Hospital Universitario Austral, Buenos Aires, Argentina
Preparation for the pandemic, or even subsequent waves of the existing pandemic, raises several related and pressing questions about the role of breadth, scope, and flexibility of value assessment for Medical Devices & Diagnostics. This discussion can help inform the procurement and distribution of critical medical technologies at the local and international level. Related issues and solutions need to be discussed and assessed using an integrated approach of various key stakeholders. Hence the panelists reflect an international WHO perspective, an HTA agency perspective, and academic / health economic perspective, an industry / manufacturer perspective and a health care provider perspective in order to discuss related key issues including:
• How healthcare systems can coordinate, globally, to help ensure that healthcare efficiency has the potential to be maintained even in challenging, emergency situations? Who should have an active role on value assessment
of new technologies in emergency situations?
• How can non-governmental organizations, national governments, and international pan-national bodies help to provide a framework for more efficient decisions and allocations of resources in a pandemic emergency? How should these organizations receive feedback from local procurement?
• How can public and private collaborate and co-design solutions to ensure affordability, sustainability and overall resilience?
• How to define strategic procurement strategies, based on the creation of a portfolio of reliable suppliers, in order to avoid the lack of critical goods in case of emergency?
• What should be the ideal role of the academics and specifically health economists be in similar future contexts? Which role plays value-based healthcare in this context?
• How can the industry and policymakers contribute to address these critical questions? Value-based procurement needs an appropriate place in decision making to ensure organizational and health care systems are prepared for subsequent pandemics in a timely manner.
Moderator: Rita Karam, PharmD, PhD, Faculty of Medical Sciences, Lebanese University, Hadath, Lebanon
Speakers: Yacine Sellam, Pharm.D, Ph.D, Algerian Society for Regulatory Affairs & Pharmacoeconomics (SAARPE), ALGER, 16, Algeria; Kasem Akhras, PharmD, Astrazeneca, Dubai, United Arab Emirates; Mahmoud Diaa Elmahdawy, PharmD, HEOR/RWE APAM Region, Novartis, Cairo, Egypt; Sherif Abaza, BPharm, MBA, MENA for Syreon President Elect Egypt ISPOR Chapter, Syreon Middle East, Cairo, C, Egypt; Sarine Aderian, PHARM D, Levant and Egypt, Abbvie biopharmaceuticals. Member of ISPOR Lebanon chapter, Beirut, Lebanon
The cost to deliver healthcare in developing countries has been rising exponentially. Governments around the world are searching
for alternative mechanisms to reduce costs while increasing the medical capacity of the system with significant investments in infrastructure. Governments especially in Europe, have increasingly used private sector involvement in developing, financing
and providing public health infrastructure and service delivery through public–private partnerships (PPPs). Use of PPPs in MENA is a relatively new phenomenon. Policy makers and governments are very much interested in studying and understanding
the relationship between health care expenditure and health outcomes. Public healthcare systems may not be technically and allocative efficient due to several challenges, a push for private funding is usually needed. The goal of this forum is to explore
motivations, challenges, and lessons learned during PPP partnerships: engagement, formation, operations, financing and knowledge management. The forum objectives is to engage in forward-looking dialogue on the evolving models of partnerships for improving
health outcomes in MENA region; to explore opportunities for identifying and applying lessons learned from experiences of success and failure; and to discuss how the evolving model of partnerships and lessons learned can impact the future of the healthcare
system in MENA, help meet developmental challenges, and sustain improved outcomes.
Moderator: Katarzyna Kolasa, PhD, Health Economics and Healthcare Management, Kozminski University, Warszawa, Poland
Speakers: Anita Burrell, BA, MA, MBA, Anita Burrell Consulting LLC, Flemington, NJ, USA; Axel Christian Mühlbacher, PhD, MBA, IGM Institute Health Economics and Health Care Management, Hochschule Neubrandenburg, Neubrandenburg, BW, Germany; Bakul Patel, MSEE, MBA, Digital Health, FDA CDRH, Silver Spring, MD, USA; Laura Vinuesa, D.V.M. M.Sc., Decision Resources Group, London, LON, UK
Digital Health Interventions (DHIs) continue to be classified and categorized in different ways by different organizations. This inconsistency leads to confusion and can inhibit assessments for regulatory, market access, and pricing
and reimbursement (P&R) decision-making. This forum will give an update on the SIG’s special scoping review project, the aim of which is to collate and understand the numerous DHI categorization and definitions. This synthesis of available knowledge
shall contribute to a better understanding of how to place digital health technologies in healthcare systems and also represents a valuable contribution to ongoing P&R developments for DHIs in different jurisdictions. An overview of the P&R landscape
for DHIs worldwide and an FDA regulatory update will be presented. Starting in 2020, Germany’s Digital Care Act (Digitale Versorgung-Gesetz, DVG) has introduced the statutory health insurance (SHI) reimbursement process for digital health applications
(DiGA). We will discuss the implications of this law for manufacturers/operators of DHIs. Attendees will have opportunities to respond to specific questions and comments relating to DHI definitions and categorization, as well as answer general regulatory
and P&R questions. During the closing Q&A, participants will have the chance to ask questions about the future projects of the SIG. Dr. Kolasa will moderate and provide an overview of the P&R landscape; Dr. Odom, who will be speaking in an
individual capacity and not as a representative of her company, will give updates on the ISPOR DH SIG key project manuscript; Dr. Mühlbacher will provide pricing and reimbursement updates and news from Germany, discussing the Digital Care Act and
what it means for manufacturers and patients; Mr. Patel will provide FDA regulatory updates related to DH technologies; and Dr. Vinuesa will discuss member engagement work of the SIG.
Moderator: Eline van Overbeeke, PhD, Member Services, ISPOR, Brussels, VBR, Belgium;
Speakers: Elisabeth Oehrlein, PhD, MS, Education Initiatives, International Society for Pharmacoeconomics & Outcomes Research (ISPOR), Washington, DC, USA; Kevin Marsh, PhD, Evidera, Newport Pagell, BKM, Great Britain
The ISPOR New Professional Steering Committee, Staff, and Health Preference Special Interest Group invite you to an informative fireside chat style session that will focus on "Health Preference Research in Europe". The session was developed based on an article published in the July issue of Value in Health with the title, "Health Preference Research in Europe: A Review of Its Use in Marketing Authorization, Reimbursement, and Pricing Decisions". The session is geared towards New Professionals but is open to anyone who wishes to learn more about this high interest topic.
• Overview of New Professionals & the Health Preference Research SIG (5 Minutes)
• Discussion about the research paper Health Preference Research in Europe 15 minutes)
• Discussion about IMI PREFER, including a case study (10 minutes)
• Plug for paper for those who wish to read it to learn more and encouraging New Professionals to get involved with the Health Preference SIG or other Member Groups (5 minutes)
Discussion Leaders: Stephanie Manson, PhD, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA; Matt Michelson, Ph.D., Evid Science, La Canada, CA, USA; Meaghan Gibbs, BSc, MSc, Novartis, Dubai, United Arab Emirates; Pall Jonsson, BS, MRes, PhD, Science Policy and Research Programme, National Institute for Health and Care Excellence, Manchester, LAN, UK
PURPOSE: This workshop will present examples of how AI can be applied to automate the creation of components of HTA submissions and how these technologies are perceived from an HTA perspective. By bringing together discussion leaders from AI, HTA creation, and HTA evaluation, this workshop aims to give a well-rounded perspective on the topic.
DESCRIPTION: With a proliferation in artificial intelligence approaches, AI techniques are being applied to the preparation of evidence for inclusion in HTA submissions. Matthew Michelson will first share from a technical perspective how AI can be applied to screen and extract data within literature reviews, and how this process has recently evolved. Stephanie Manson will share how this AI approach has been used in practice to conduct systematic and targeted literature reviews and how this can support HTA submissions. Meg Gibbs will then discuss how AI is being used to digest detailed global value dossiers, clinical study reports, modelling reports and supplemental evidence in order to autocreate tailored summaries suitable for HTA submissions. Finally, Páll Jónsson will share the NICE perspective on how to ensure quality and appropriate application with these technologies. Each presenter will incorporate two-way audience dialogue into their perspective through polls about perceptions and barriers towards the adoption of AI in HTA submissions.
Discussion Leaders: Andrew Briggs, DPhil, Health Services Research & Policy, London School of Hygiene & Tropical Medicine, London, UK; Alexis Samantha Doyle, MA, Avalon Health Economics, Morristown, NJ, USA; Eric William Low, NA, Eric Low Consulting, Haddington, ELN, UK; Tanja Podkonjak, MBA, Takeda UK Ltd, London, UK
PURPOSE: Combination therapies are often used in diseases such as cancer. However, combination therapies often fail to be cost-effective, especially when the component drugs are priced independently by different manufacturers. Manufacturers of component drugs cannot coordinate prices since this is prohibited by anti-trust law. The purpose of this workshop is to illustrate and discuss a value attribution framework for combination therapies grounded in the standard rules of cost-effectiveness that can be used to assist in negotiations around prices when evaluating combination therapies by reimbursement authorities, such as the National Institute for Health and Care Excellence (NICE) in the UK.
DESCRIPTION: We will present a proposed value attribution framework that can be used to address the problem of pricing combination products fairly to manufacturers of the component products AND payers within the health system. We present solutions to four scenarios of combination therapies that are characterized by key features of the problem: perfect/imperfect information about the monotherapy effect of component therapies (individual effects are known/unknown) and balanced/unbalanced market power between their manufacturers (unbalanced being where one manufacturer already has market access and a new entrant seeks to add to this 'backbone' therapy) . Case studies are used to illustrate each scenario drawn from publicly available information of recent combination therapy submissions to NICE. Consideration will be given to the process of negotiation that can help navigate anti-trust rules designed to prevent price collusion.
Moderator: Dalia M Dawoud, -, National Institute for Health and Care Excellence (NICE), London, LON, UK
Speakers: Noluthando Nematswerani, -, Centre for Clinical Excellence, Sandton, South Africa; Oksana Pyzik, -, School of Pharmacy, University College London, London, UK; Asmaa Fouad Ismail, -, Egyptian Drug Authority, Cairo, Egypt; Tienie Stander, MBChB, MBA, TCD Outcomes Research Centurion, Pretoria, South Africa and Managing Director, Value In Research, Dubai, South Africa
To discuss the COVID-19 pandemic’s impact on the supply of and demand for biologics in low- and middle-income countries (LMICs), the resultant shortages and the role that accelerating access to biosimilars can play. Description: International emergencies,
such as the COVID-19 pandemic, come with unprecedented challenges that test healthcare systems’ preparedness and ability to function under and adapt to extreme conditions. One challenge that we witnessed is the shortage of many medicines as a result
of either repurposing these medicines for use as potential treatments for COVID-19 or the measures put in place to combat the spread of the pandemic such as closures of manufacturing sites, export bans and border closures. Some governments have responded
by stockpiling medications, which caused widespread condemnations across the world and concerns around the rise of black markets, and sub-standard and falsified medicines, as a result. The problem has been profound for biologics that have no alternative
biosimilar approved for use, particularly those currently being tested as potential COVID-19 treatments (e.g. IL-1/6 inhibitors such as tocilizumab) but also those used for other conditions. Innovative approaches and policy decisions to accelerate the
approval, manufacture and uptake of biosimilars could address some of these shortages and ensure patient access to effective treatments is not disrupted. Polling questions to assess the scale of the problem in the countries represented in the audience,
seek audience views regarding the potential impact of accelerating biosimilars’ approval and use on patients’ access to medications and the barriers and facilitators to achieving this potential will be used. Dr Nematswerani will present the
LMIC payer perspective on the topic, Dr Pyzik will provide a non-governmental organisation perspective, Dr Fouad will provide the regulator perspective and Dr Stander will provide a research consultancy perspective. Dr Dawoud will present the HTA perspective
and moderate the forum.
Moderator: Julia Slejko, PhD, University of Maryland School of Pharmacy, USA
|AD1||Compensating Patients, Caregivers, and Patient Groups for Their Patient-Engagement Activities: Development of the NHC FAIR-Market-VALUE (FMV) Calculator|
EM, Schoch S, Boutin M, Oehrlein E|
National Health Council, Washington, DC, USA
|AD2||Qualitative Interviews to Explore Drivers and Behaviours Associated with Medication NON-Adherence across a Range of Diseases, Treatment Modalities, and Countries|
E1, Bentley S1, Rossom R2,
Roche N3, Higgins V4, Piercy J5, Arbuckle R1,
1Adelphi Values Ltd, Bollington, UK, 2HealthPartners Institute, Minnesota, MN, USA, 3Paris-Descartes University, Paris, France, 4Adelphi Real World, Bollington, CHE, UK, 5Adelphi Real World, BOLLINGTON, CHE, UK, 6University of Leicester, Leicester, UK
Moderator: Yajaira Bastardo, PhD, Universidad Central de Venezuela, Caracas, Venezuela (Bolivarian Republic of)
Speakers: Alfonso Gutierrez Aguado, MD, INSTITUTO DE INVESTIGACION EN CIENCIAS BIOMEDICAS, UNIVERSIDAD RICARDO PALMA, LIMA, Peru; Selenia Gómez de los Santos, BSc, MBA, -, Mexico City, Mexico; Stephen Stefani, MD, Instituto OncoCLinicas, Porto Alegre, Brazil; Rafael Pinedo-Villanueva, PhD, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK
HEOR in Latin America continues to evolve dynamically and its implementation in practice is making a profound impact on policy in the region. In this forum, we will demonstrate the versatility and wide-ranging impact of
HEOR approaches and tools on Latin America policymaking through case studies from different jurisdictions across the region. Cases will include a look at HEOR’s role and contribution to key initiatives such as cancer treatment policy in Brazil,
centralized purchasing of high cost drugs in Mexico, and the HPV vaccination program in Peru. There will also be presentation sharing the recent development of a benefit and budget impact calculator for the implementation of secondary fracture prevention
services in Latin America. Informational presentations will be supplemented by an opportunity for Q&A and the end.
Moderators: Vasco M Pontinha, MPharm, MA, Pharmacotherapy and Outcomes Science, Virginia Commonwealth University School of Pharmacy, Richmond, VA, USA; Chintal Shah, MS, BPharm, University of Maryland, Baltimore, MD, USA; Soham Yande, B.Pharm, University of Houston, Houston, TX, USA; Ingrid Cox, MD, MSC, Menzies Institute for Medical Research, Bellerive, TAS, Australia;
Speakers: Mickaël Hiligsmann, PhD, Department of Health Services Research, School for Public Health and Primary Care, Maastricht University, Maastricht, LI, Netherlands; A.M. Sofie Berghuis, MSc, PhD, Van Asch van Wijckstraat, GSK, Amersfoort, Netherlands; Brian Godman, PhD, Strathclyde Institute of Pharmacy and Biomedical Sciences, University of Strathclyde, Glasgow, UK
The ISPOR Student Roundtable events have traditionally been done as an in-person event with 5 topics based on the Top 10 HEOR Trends reports. For this Virtual Student Roundtable we will invite 5 speakers and do 5 breakout rooms for attendees to be distributed amongst the topics. Once 10 minutes has passed they will move to the next breakout room to hear from the presenter on that topic. There will be open dialogue between the presenters and attendees to ensure engagement and a general Q&A at the end with all 5 presenters.
Topics: RWE, Value-Based Alternative Payment Models, Drug Pricing & Transparency, Digital Technologies, Precision Medicine
Format: Introduction: 5 minutes)
Presentations (5 speakers): 10 minutes each with a different moderator for each topic asking questions that have been seeded from students
Open Q&A (5 minutes) for additional questions
Discussion Leaders: Judit Simon, Professor, Department of Health Economics, Medical University of Vienna, Vienna, Austria; Leona Hakkaart-van Roijen, PhD, Erasmus School of Health Policy & Management (ESHPM), Erasmus University Rotterdam, Rotterdam, Netherlands; Jan van Busschbach, PhD, Psychiatry, section MPP, Erasmus University Medical Center, Rotterdam, Netherlands
PURPOSE: The aim is to present the results of the PECUNIA project on harmonising methods and tools for outcome assessment in economic evaluations across countries in Europe. More specifically, the workshop addresses the challenges of cross-country measurements and valuation of outcomes using the EQ-5D as an illustrative example.
DESCRIPTION: Challenges of cross-country measurement and valuation of outcomes in economic evaluations include the lack of national value sets for the EQ-5D, and the absence of comparability of utility values across countries. For countries that do not yet have a national value set, it is a common practice to use another country’s value set as a proxy. Currently, there exists no definitive criteria to choose from for such a proxy value set, nor it is clear how large the differences are in health state values between countries. The main difficulty is to disentangle the possible influence of national cultural values from the methodological variation of developing value sets between the national studies. With an increasing importance of healthcare policy at the European level, a pooled value set for the European region might gain significance. This workshop presents an innovative, flexible method of deriving pooled regional value sets for the EQ-5D and its application: a pan-European and supra-national value sets for the EQ-5D. While pan-European value set pools all available national value sets from the European countries, supra-national value sets combine a selection of country-specific value sets on the basis of cultural, linguistic and other considerations. The applications of both approaches are discussed. This work provides a feasible and pragmatic solution for HTA bodies when national value sets are absent and can be of help when policy-making is done at the European level, and it is useful for a wide range of audience including health policy makers, health economists, national decision makers, and clinicians.
Moderator: Molly Purser, PhD, RTI Health Solutions, Durham, NC, USA
Speakers: Susan Snyder, PhD, MBA, Geisinger Department of Epidemiology and Health Services Research, Danville, PA, USA; Bettina Zimmermann, Access Global Lead, Roche, Basel, BS, Switzerland; Daryl S. Spinner, PhD, MBA, Precision Medicine and Population Health, Dept. of Health Policy and Behavioral Sciences, Georgia State University, Atlanta, GA, USA
Utilization of next-generation testing (NGT) that includes multiple
gene/genomic biomarkers including comprehensive cancer profiling provide a wealth of information to clinicals and patients. The increase in the number of disease-related biomarkers and targeted therapies can provide more timely clinical diagnosis and
efficient treatment. However, these innovative tests are often not reimbursed, and pathways for reimbursement are inconsistent, often fragmented, or nonexistent. The panel will discuss the applicability of current HTA frameworks for these tests, and why
and how NGT may or may not be different from other diagnostic testing modalities. Are new methodologies and/or criteria needed? What evidence may be needed to measure and assess their value, and how can manufacturers and payers more consistently evaluate
and share the risks and rewards of these innovated tests that is sustainable? This forum will introduce the new key project paper which is a collaboration between the Precision Medicine and Advanced Therapies SIG and the Medical Devices and Diagnostics
SIG and being co-chaired by Daryl Spinner and Susan Snyder. Following the forum, we will have a brief PMAT SIG meeting. The meeting will focus on activities of the SIG and varies ways to be involved and engaged with the SIG. We will poll attendees to
identify areas of greatest interest, and generate ideas for various methods for members to be involved and engaged with the SIG
Moderator: Olha Zaliska, PhD, DSci, Head of Department of Management and Economy of Pharmacy, Medicine Technology and Pharmacoeconomics, Postgraduate Faculty, Danylo Halytsky Lviv National Medical University, Lviv, Ukraine
Speakers: Iga Lipska, MD, PhD, Head of Health Care Services Monitoring Division, Health Care Services Department, National Health Fund, Warsaw, Poland; Oresta Piniazhko, PhD, State Expert Center of the Ministry of Health of Ukraine, Lviv, Ukraine; Bertalan Németh, MSc, Past President of the ISPOR Chapter Hungary, Syreon Research Institute, Budapest, Hungary; Finn Børlum Kristensen, MD, PhD, Faculty of Health Sciences, Danish Centre for Health Economics (DaCHE), University of Southern Denmark, Hilleroed, 84, Denmark
Compared to Western European nations, Central and Eastern European countries have even more limited funds available not only for purchasing health technologies, but for conducting analyses, for example health technology assessment (HTA) as well. Due to the generally worse health status in these countries, and the growing demand from the public to have access to innovative therapies, the need for adequate decision making is strong, but it is hindered by the aforementioned lack of funds and general lack of human capacities in the region. During the discussion, participants will debate on finding the possible compromise between practical feasibility and scientific accuracy. Proposed key elements of the discussion will be 1) tools and methods supporting optimal decision making 2) the transferability of health technology assessment methods and results 3) the model suggested by the European Network for Health Technology Assessment (EUnetHTA), on the local re-use of jointly produced HTA evidence.
Moderator: Bruce Crawford, MPH, MA, Real World & Late Phase, APAC, Syneos Health, Tokyo, 13, Japan
Speakers: Sang-Soo Lee, PhD, MBA, Medtronic North Asia (Korea and Japan), Seoul, 41, Korea, Republic of (South); Eugene Salole, PhD, MPH, Value-Based Access Pty Ltd, Sydney, Australia; Hwee-Lin Wee, PhD, Saw Swee Hock School of Public Health, National University of Singapore, Singapore, Singapore; Perry Bridger, -, Global Value, Payer Access & Public Policy, Edwards Lifesciences LLC, Irvine, CA, USA
The potential roles and opportunities for real world evidence (RWE) have been rapidly growing in global healthcare sector, including the Asia Pacific. The 21st Century Cures Act passed by the US Congress in 2016 required the
US FDA to expand the role of RWE and establish a framework for the RWE program. While RWE is being used for regulatory decision-making such as supporting approvals of new products and expansion of new indications or tracking the performance of products
already on the market, there are also significant opportunities in market access areas such as coverage and pricing. Compared with pharmaceutical products, medical technologies (medtechs) have relatively less robust clinical evidence mainly driven by
the different and diverse characteristics of the medtech industry and technologies themselves, which brings different regulatory and evidentiary requirements. The need for more practical and robust evidence of safety and effectiveness are ever increasing
and RWE could be the feasible solution to fill the evidence gap and optimize the clinical outcomes of medical technologies. In this session, a diverse panel of speakers will share their views and experiences on how to use RWE for medtech value assessment
including coverage and pricing determination. The specific challenges and opportunities for the Asia Pacific region will be highlighted.
Moderator: Nathaniel Smith, PhD, Maple Health Group, LLC, New York, NY, USA
Panelists: Fabrizio Gianfrate, PhD, MSc, University of Ferrara, Ferrara, Italy; Don Husereau, BScPharm, MSc, University of Ottawa, Ottawa, ON, Canada; Thomas J Stephens, PhD, Maple Health Group, Oslo, Norway
ISSUE: The cancer treatment landscape has been evolving with the introduction of products such as cell therapies that require higher upfront investment yet offer the potential for significant improvements in long-term health outcomes. These products often have trials with relatively short follow-up creating increased uncertainty around their long-term cost-effectiveness. With a growing need to balance the long-term effectiveness of these treatments with short-term affordability, payers are showing increased interest in innovative payment schemes, such as outcomes-based contracting, leveraging objective short-term outcomes to help balance the financial investment with the potential long-term benefits. HTA agencies are starting to discuss how appraisal frameworks need to be updated for cell and gene therapies, but the challenges of accounting for outcomes-based agreements in economic models for oncology products have not been explicitly defined.
OVERVIEW: Partitioned survival models have been the standard cost-effectiveness modelling approach in oncology in recent years, utilising data on disease progression and death directly from clinical trials. Innovative therapies offer the possibility of significant improvements in overall survival, and possibly even long-term cure; however, these benefits are often based on trials with short-term follow-up (<2 years) at the time of reimbursement assessment, with meaningful gains primarily demonstrated on surrogate endpoints like overall response rate. Given that a product’s cost-effectiveness and budget impact would be dependent on the reimbursement scheme, it may be appropriate to use shorter-term clinical outcomes, such as response rate, as the structural framework for models rather than survival. Do models need to explicitly account for the correlation between shorter-term outcomes that are metrics for reimbursement, and the longer-term drivers of cost-effectiveness, and capture the uncertainty in the relationship, in order to appropriately inform potential outcome-based agreements? This panel will discuss the potential modelling approaches, the results, their validity, and the implications for payers and HTA agencies.
Moderator: Zeba M. Khan, RPh, PhD, Celgene Corporation, Summit, NJ, USA
Speakers: Sujit KUMAR Sah, Doctor of Pharmacy, Department of Pharmacy Practice, Jagadguru Sri Shivarathreeswara University, Mysuru, KA, India; Yoon-Bo Shim, PharmD, School of Pharmacy, Sungkyunkwan University, suwon, South Korea; Valentine Grumberg, Master, Master Market-Access and Economic Evaluation, Université Paris-Saclay, Chatenay-Malabry, France; Naiya Patel, -, University of Louisville, LOUISVILLE, KY, USA; Samuel Austin Crawford, MS, University of Southern California, Los Angeles, CA, USA; Khalid Kamal, -, HEALTH SCIENCES, West Virginia University, Morgantown, WV, USA
This revamp of the Student Research Showcase will allow for increased participation
and interaction as it will feature 5 of the top student outcomes research studies submitted for the conference. Attendees of the event will hear a 3-5 minute elevator pitch of the summary of the research study, the methods, challenges, the so what, conclusions,
and the relevance of the research to the conference theme. There will be time at the end for questions from the Moderators.