Spotlight sessions highlight timely topics in HEOR and promote areas of innovation of interest to the ISPOR community.
Monday, 12 November 14:15-15:15
IP4: Healthcare X.0: Digital Technologies and Creation of Value
The very nature of health care valuation and delivery is being redefined with the rise of digital technologies. Participants in this ISPOR Spotlight session in Barcelona will take a closer look at the game-changing potential of digital technologies in creating and delivering better value in healthcare, and the opportunities for health economics and outcomes researchers to participate in co-creation of the “next healthcare system” by discussing the following questions: How does digital technology contribute to healthcare innovation? How does the understanding of competing on value and evidence-based medicine change with digitalization? Are there tools, processes, and new strategic concepts to adjust the ways we manage and measure impact of digital health technologies?
Andrew Rosner, MBA, Vice President, Real-World & Late Phase, Syneos Health, Toronto, Canada
- John Hernandez, PhD, Research Scientist, Research Scientist, Google Brain, Mountain View, CA, USA
- Violeta Isabel Perez-Nueno, PhD, Research Programme Officer, EU Policies, Unit H3, eHealth, Well-being & Ageing, DG CONNECT, Communications Networks, Content and Technology, European Commission, Brussels, Belgium
- John G. Singer, Global Head of Strategy, Innovation and Technology, Health Business Unit, Wipro Limited, New York, NY, USA
- Egge van der Poel, PhD, Clinical Data Scientist, Erasmus University Medical Centre, Rotterdam, Associate Dean, Permanent Education for Professionals, Jheronimus Academy of Data Science, Rotterdam, The Netherlands
Monday, 12 November 14:15-15:15
IP5: Prove It With PROs
OVERVIEW: PRO data are increasingly included in HTA submissions, but are rarely a decision driver. The extent to which PROs are being assessed and the influence the data has on HTA recommendations varies between HTA bodies as their data requirements and assessment methods differ. Moderator Anke van Engen will first describe the impact of PRO data in HTAs in different therapeutic areas and different countries. IQWiG has clear guidelines on the assessment of PRO data and Germany is one the exceptions where the impact of PRO data on HTA decision-making is more transparent. Professor Yvonne-Beatrice Boehler will present IQWiG’s methodology for assessing symptoms and functional scales. She will highlight common issues seen with the submitted PRO data and set out what manufacturers need to do in order to be successful. Stefan Holmstrom will offer the industry perspective, discussing an example highlighting some of the key challenges in convincing HTA bodies of patient-relevant improvements and illustrating that better guidance from HTA bodies is required. The industry also must present evidence in a more user-friendly format. Professor Finn Boerlum Kristensen will present the value assessment of the patient and social aspects in the Core Model developed by EUnetHTA and argue that there can be more consensus and emphasis on PRO by including it more prominently in the HTA Core Model. The audience will be invited to share their thoughts on how to increase the impact of PROs on HTA decisions, and share best practices in collecting and assessing PRO data.
Anke van Engen, MSc, Senior Principal, IQVIA, Amsterdam, Netherlands
- Yvonne-Beatrice Boehler, MD, MBA, Vice Dean for Science and Knowledge Transfer, Professor for Pharmamanagement, Faculty of Applied Natural Sciences, TH Koeln, University of Applied Sciences, Leverkusen, Germany
- Stefan Holmstrom, BSC, MSc, Senior Director, Global HTA Strategy, PRO Excellence, Astellas, Leiden, Netherlands
- Finn Børlum Kristensen, MD, PhD, Professor, Department of Public Health, Research Unit of User Perspectives, Faculty of Health Sciences, University of Southern Denmark, Hillerod, Denmark
Monday, 12 November 14:15-15:15
W3: Adjusting for Post-Randomization Confounding and Switching in Phase III and Pragmatic Trials to Get the Estimands Right: Needs, Methods, Sub-Optimal Use, and Acceptance in HTA
DESCRIPTION: HTA should investigate both the effect of actual and intended treatment to guide clinical and policy decisions. Our focus is on situations with treatment switching or adherence influenced by time-varying post-randomization factors such as progression or side effects. In these cases, traditional methods may fail and causal inference methods must be used. Recently, HTA agencies have accepted and recommended using causal methods, and a paradigm shift is taking place. Selection of the appropriate adjustment method has become crucial to inform decision-making on patient access to innovative and cost-effective treatments. However, adjustment methods are often used poorly and their full potential is not being realized. This workshop has four parts: 1) Uwe Siebert will describe post-randomization confounding/selection bias in trials, introduce causal inference concepts and adjustment methods, and present case examples of biases occurring from inappropriate analyses. 2) Nicholas Latimer will demonstrate that the full potential of methods used to adjust for treatment switching is not being realized: He will explain how the use of adjustment methods can be improved by discussing key assumptions and application choices, and how these relate to health economic models. 3) Amanda Adler, Chair of NICE Technology Appraisal Committee B, will discuss the challenges of using statistical methods to address treatment switching in the HTA process using examples from NICE. We will actively engage participants in the discussion and use the ISPOR polling app to collect the audience’s view on discussed issues.
- Felicitas Kühne, MSc, Junior 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
- Uwe Siebert, Prof, MPH, MSc, ScD, MD, Professor, Department of Public Health, Health Services Research & HTA / Department of Health Policy and Management / Institute for Technology Assessment and Department of Radiology, UMIT - University for Health Sciences, Medical Informatics and Technology / Harvard T.H. Chan School of Public Health / Harvard Medical School, Boston, MA, USA, Hall i.T., Austria
- Nicholas Latimer, PhD, Reader in Health Economics, Health Economics and Decision Science, ScHARR - University of Sheffield, Sheffield, United Kingdom
- Amanda Adler, MD, PhD, FRCP, Chair, Technology Appraisal Committee B, Addenbrooks’s Hospital/National Institute for Health and Care Excellence, Cambridge, United Kingdom
Tuesday, 13 November 14:00-15:00
IP15: Transforming Healthcare: The Impact of Patient Engagement
This panel will start with an overview of the global emergence of patient engagement, followed by a brief history of the patient movement towards evidence-based advocacy and meaningful and substantial patient involvement. The panelists will identify how patient engagement has influenced the EU perspective on health policy and is transforming the EU regulatory system. A view from the US will demonstrate the various ways patients are engaging in healthcare research. Highlighted by case studies, speakers will focus on identifying the impact of patient engagement and ways in which improvements in healthcare and patient outcomes can be attributed to patient engagement. The panel will conclude with a dialogue on the lessons learned and a path for the future.
Nancy Devlin, PhD, Research Director, Office of Health Economics, London, UK
Tuesday, 13 November 14:00-15:00
IP16: Valuing a Cure: Are New Approaches Needed?
ISSUE: The health technology assessment (HTA) community and payers now face an innovation pipeline containing many new drugs that offer the potential to cure significant chronic illnesses. Hemophilia, HIV, sickle cell disease, as well as many other more rare conditions, may have potential cures receiving regulatory approval over the next 3-5 years. Are the standard approaches of evidence assessment and economic evaluation fit for purpose for potential cures? Questions have already been raised about whether standard HTA methods capture all the elements of “value” represented by potential cures. Similarly, concerns exist about whether standard methods are able to reflect the uncertainty that will likely persist for many years following the introduction of these treatments. Standard HTA methods may also suggest that extremely high prices represent reasonable value for money when potential cures are targeted at conditions like hemophilia that have substantial lifetime costs. Consideration of how best to adapt HTA for the evaluation of potential cures is needed now before the hoped-for wave of new cures arrives.
OVERVIEW: This panel will present contrasting views on the best options for adaptation of HTA methods for potential cures. Pearson will moderate and also briefly describe a new proposal for "sharing" substantial cost offsets between innovators and health systems. Grueger will then describe how innovators think about the value of potential cures and how that should guide pricing and funding decisions. Garner will describe the view on the pricing of cures from the public health perspective, where access to innovative treatments in lower income countries is a top priority. Lastly, Sculpher will argue that, despite the issues raised earlier, existing HTA methods are adequate to evaluate these interventions and should not be radically changed. Moderated discussion will then explore these views and develop a list of key themes for future policy development.
Steven D Pearson, MD, MSc, President, Institute for Clinical and Economic Review, Boston, MA, USA
- Jens Grueger, PhD, Vice President and Head of Global Pricing and Market Access, Roche, Basel, Switzerland
- Sarah Garner, PhD, Co-ordinator, Innovation, Access and Use, Essential Medicines and Health Products, World Health Organization, Geneva, Switzerland
- Mark Sculpher, PhD, Professor of Health Economics, Centre for
Tuesday, 13 November 14:00-15:00
W10: Understanding and Addressing Potential Bias in Patient-Reported Outcomes from Clinical Trials
PURPOSE: We will share a variety of novel trial design and analytic techniques to understand or minimize bias in PRO data, with implications for understanding treatment effects and optimizing HTA and regulatory submissions.
DESCRIPTION: Bias within PRO data collected in trials has been a longstanding issue, which can confound conclusions drawn from such data. Certain diseases with event driven data collection (e.g. oncology) and trial designs (e.g. open label and single arm trials) have the potential for higher levels of bias. We will first discuss trial design including examples of how to optimize trial design in different situations, e.g. where it is not possible to have a double blinded study or when symptoms aren’t expected to coincide with planned site visits. We will also discuss approaches for minimizing the amount of missing PRO data including technology and effective site communication.
We will discuss a variety of analytic techniques that can be used to better understand PRO data, particularly in situations with large amounts of potentially informative missing data. A few examples will be shared to best understand these data, including pattern mixture models and extended pattern mixture models. In addition, more complicated (i.e., indirect/mediated) relationships often exist between treatment and PRO outcomes, but traditional analyses often don’t capture them. We will describe alternative approaches and present recent examples from oncology clinical trials.
Finally, we will discuss the importance of minimizing bias and missing data from an evaluator’s perspective: how can we anticipate or at least identify potential biases, and analyze, interpret and present results in a submission dossier for optimizing a submission for drug approval or reimbursement?
There will be polling throughout this workshop to ask the audience their priorities in terms of approaches to minimizing bias, the pros/cons of different approaches, and an opportunity for questions at the end.
- Stephanie Manson, PhD, Senior Director, HEOR Excellence, Novartis, East Hanover, NJ, USA
- Stefanie Knoll, PhD, Global Access Product Lead, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA
- Donald E Stull, PhD, Head, Data Analytics and Design Strategy, Health Economics, RTI Health Solutions, Durham, NC, USA
- Olivier Chassany, MD, PhD, Professor, Patient-Centered Outcomes Research, University Paris-Diderot, Paris, France