Spotlight Sessions

Moderator:
Andrew Rosner, MBA, Vice President, Real-World & Late Phase, Syneos Health, Toronto, Canada

Speakers:

• 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 

Moderator:
Anke van Engen, MSc, Senior Principal, IQVIA, Amsterdam, Netherlands

Speakers:

• 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

Speakers:

• 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
  

Moderator:

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

Speakers:

• Jan Geissler, MBA, Co-Founder, CML Advocates Network and Founder and CEO, Patvocates, EUPATI, Munich, Germany
• Suzanne Schrandt, JD, Director, Patient Engagement, Arthritis Foundation, Atlanta, GA, USA
• Nicola Bedlington, Secretary General, European Patients Forum (EPF), Brussels, Belgium 
  

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.

Moderator:

Steven D Pearson, MD, MSc, President, Institute for Clinical and Economic Review, Boston, MA, USA

Speakers:

• 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

   

  Health Economics, University of York, York, United Kingdom

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.

Speakers:

• 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