PSI Scientific Meeting: Patient-focused drug development

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Date: Wednesday 18th & Thursday 19th October 2023
Time: Wed 18th 13:00-15:40 | Thurs 19th 13:30-16:10 BST
Location: Online
Speakers: Peter Mol (University Medical Center Groningen), Caroline Vass (RTI Health Solutions), Hannah Penton (OPEN Health), Marco Boeri (OPEN Health), Christine Sturchler (Novartis), Byron Jones (Novartis), Brett Hauber (Pfizer) and Bennett Levitan (Johnson & Johnson)

Who is this event intended for? Statisticians in the pharmaceutical industry, HTAs and regulatory bodies, working with registrational clinical trials.
What is the benefit of attending? Learn about latest developments in patient-focused drug development from various stakeholders' perspective.


This is event is free to attend for both Members of PSI and Non-Members. To register your place, please click here.


The meeting will consider the importance of patient-centric input into regulatory and HTA decision-making discussing the latest developments in the field and presenting industry examples. Speakers from EMA, the pharmaceutical industry, and those working with HTAs and patients will give their perspective on the importance and practical aspects of selecting relevant for patients outcomes in the evaluation of clinical trials.

Over the course of two half days we will have a range of speakers providing insight into the process of involving patients as research partners and how Patient Preference Studies can yield deeper understanding on patients’ perspectives for decision-making in clinical trials. To set the stage, we will discuss the regulatory perspective, review published guidance and existing framework, and give overview of the logistical and methodological challenges faced by researchers and HTA bodies. We will present case studies assessing the importance to patients of improving symptoms in chronic obstructive pulmonary disease and fatigue in multiple sclerosis, both using discrete choice experiments. We will learn about a conceptual framework for addressing patient preferences in dose selection in the absence of full information on risks and benefits, also involving discrete choice experiments. The event will finish with a panel discussion, where experts will deliberate on the key takeaways and future directions as well as the role of the statistician in patient-focused drug development.

Speaker details





Peter Mol

Peter Mol is the Committee for Medicinal Products for Human Use (CHMP) member for the Dutch Medicines Evaluation Board. He was from 2012 to 2023 member (vice chair 2016-2022) of EMA’s Scientific Advice Working Party. He has coordinated over 300 EMA and national scientific advice procedures for drug development programs for cardiometabolic, gynecology and hematology products. He was chair of the EMA Cross-Committee Task force on Registries (2016-2023)
He is also professor of drug regulatory science at the University Medical Center Groningen. His research focus is on developing new tools to support regulatory decision-making and the exchange of knowledge between regulatory authorities, health care professionals and lay people. He is currently involved in projects around personalized medicine, new data sources (RWE), patient-centric ways to weigh drug benefit-risk; e.g., using Patient Relevant Outcomes, Quality of Life, and Patient Preference information, and on risk communication; e.g., Direct Healthcare Provider Communication.  He is Principal Investigator of the HORIZON More-EUROPA project.

The perspective of an EU regulator

Regulatory decisions are made to enhance public health in the European Union (EU). Favourable and unfavourable effects of drugs are valued at a population level, with the emphasis that for an individual patient benefits of treatment should outweigh the risks. In patient-focused drug development and evaluation preferences of patients should be incorporated in the various decisional moments along the drug lifecycle. European regulators have already early on realized it is important to involve patients in activities of the various committees of the European Medicines Agency. Increasing attention for patient reported outcomes to evaluate drug effects, and patient preference studies (PPS) to capture patient’s valuation of drug outcomes led to the global 2021 ICH reflection paper on patient-focused drug development, and the qualification of the IMI PREFER PPS framework in 2022 by the EMA. Now, the challenge is in the implementation of these methods into the daily routine of drug development and evaluation. Key case studies will help determine situations where and how PPS and PROs are contributing most to decision-making. These cases will also help understand strengths and methodological challenges of incorporating the relevant patient voice in the regulatory decision. I will provide a regulator perspective on a possible way forward.


Caroline Vass

Hannah Penton

Marco Boeri

Caroline Vass
Caroline is a Senior Economist in the Health Preference Assessment team at RTI Health Solutions. Her research focuses on stated preference research, particularly using discrete choice experiments (DCEs) to elicit preferences for benefit-risk trade-offs. She has methodological interests in both the qualitative and quantitative components of stated preference research. Prior to joining RTI Health Solutions, Caroline completed her PhD and post-doc research at the University of Manchester. Caroline has given oral presentations of her research at international conferences and has peer-reviewed publications including systematic reviews, editorials, empirical applications, and pedagogical pieces in journals such as PharmacoEconomics, Medical Decision Making, Value in Health, and the Journal of Choice Modelling. She currently sits on the honorary editorial board for The Patient journal. She has experience of conducting preference research to support regulatory decision making and has provided paid consultancy services to NICE Scientific Advice.

Hannah Penton
Hannah Penton is a Senior Scientist in the Patient-Centered Outcomes team, based in the Rotterdam office. Hannah is specialized in health state utility and preference-elicitation projects.

Hannah has experience in multiple health utility assessment projects, preference elicitation projects, psychometrics and qualitative content validation studies. Hannah served as a member of a NICE Evidence Review Group for three years, during which she reviewed and critiqued company cost-effectiveness submissions and advised committee, with particular focus on HRQoL, across a range of disease areas. She has also led a EuroQol funded project investigating the use of the recall period of the EQ-5D as well as being involved in several EuroQol psychometrics studies.

Hannah holds an MSc in Health Economics and Decision Modelling and a PhD in Health Economics and Decision Science from the School of Health and Related Research (ScHARR) at the University of Sheffield.

Marco Boeri
Marco Boeri, PhD, is Director of Preference Research at OPEN Health and honorary professor in practice in Health Economics at Queen’s University Belfast. He has over 15 years of experience in preference assessment in environmental and health economics. Marco co-chaired the key project on preference heterogeneity promoted by the ISPOR’s Health Preference Special Interest Group in 2021-2022, is chair-elect of the ISPOR Health Preference Special Interest Group, and is co-chairing the Benefit-Risk Special Interest Group at the PSI conference.

Marco has co-authored the first applications of the Random Regret Minimization model in both environmental and health economics, and published over 50 peer-reviewed papers, including several in applied economics journals, such as Pharmacoeconomics, Journal of Health Economics, Health Economics, Patient Prefer Adherence, Social Science and Medicine, Value in Health, Medical Decision Making, Preventive Medicine, Environmental and Resource Economics, Energy Economics, Transportation Research Part A, and the Journal of Economic Behavior and Organization.

Incorporating Patient outcomes, Preferences, and Perspectives into HTA

Like many authorities, health technology assessment (HTA) bodies are increasingly advocating for patient-centred decision-making. This talk will explore the role of patients’ perspectives and preferences in HTA by reviewing published guidance and case studies from the UK, Europe and Australia. We will explore how qualitative accounts are being incorporated into committee decisions and examine the growing, but still contained, role of quantitative patient preference data in technology appraisals. The talk will finish with a discussion of the logistical and methodological concerns that have been raised by researchers and the HTA bodies themselves.

Christine Sturchler

Christine holds the position of Director of Patient Engagement Scientific Excellence at Novartis, where her role involves overseeing the creation and implementation of patient-centric solutions for Drug Development. She collaborates closely with patients to gather their valuable insights on various Novartis drug development initiatives, with a particular focus on obtaining their input on patient preference study materials.

The perspective of Patients as Research Partners in Patient Preference Studies

By the conclusion of this presentation, attendees will have a clearer grasp of patients’ and external environment's expectations concerning Patient Focused Drug Development, with a specific emphasis on Patient Preference Studies. The focus will be on highlighting the advantages of a robust, early collaboration with Patients As Research Partners during the development of study materials for Patient Preference studies. Real feedback will be shared through a specific example as an illustration.

Byron Jones

Until recently Byron Jones was a Senior Biometrical Fellow and Executive Director in the Statistical Methodology and Consulting Group at Novartis Pharma AG in Basel, Switzerland. He now works as an external biostatistician in the Patient Engagement Science function at Novartis. He is a Fellow of the American Statistical Association and the co-author of nine statistical textbooks. For the twenty-five years before joining the pharmaceutical industry he worked in academia, ultimately holding the position of Professor of Medical Statistics at De Montfort University, UK. After leaving academia he held Honorary Professorial positions at four UK universities: University College London, the London School of Hygiene and Tropical Medicine, University of Leicester and Queen Mary, University of London. Prior to joining Novartis in 2011 he held senior positions at GSK and Pfizer. Byron has been a PSI Board of Directors member and in 2016 led the successful campaign to save PSI from being overtaken by a larger statistical society. He was a member of the ICH Expert Working Group that wrote the revised ICH E8 guidance on “General Considerations for Clinical Studies”. He is a Series Editor for the Chapman and Hall/CRC Press' Biostatistics book series, was a Founding Editor-in-Chief of the PSI journal Pharmaceutical Statistics, and was formerly an Associate Editor of JRSS Series B and a Regional Editor of the Journal of Biopharmaceutical Statistics.

The importance of different symptoms to people living with Chronic Obstructive Pulmonary Disease (COPD): results of a multi-country patient preference study.
Industry case (Novartis).

Understanding patient needs and expectations related to new treatments is essential to ensure that drug development addresses what matters most to patients. The selection of endpoints in clinical trials should therefore be informed and driven by robust patient-based evidence exploring priorities for people living with the disease in question.

Previous research using Social Media Listening and Online Bulletin Boards highlighted the importance persons living with Chronic Obstructive Pulmonary Disease (COPD) place on symptoms that affect their daily lives (chronic cough, excess mucus, sleep disturbance and urinary incontinence), in addition to the more commonly measured COPD clinical endpoints of shortness of breath (SOB) and exacerbations.

We describe the results obtained from a patient preference study that sought to quantify the relative importance patients placed on improving these symptoms using a discrete choice experiment (DCE) with different health states based on the six COPD symptoms. In total, 1050 persons living with COPD in the United Kingdom, United States, France, Australia, and Japan participated in the study.

All six attributes were considered important when patients determined their preferences in the DCE: the relative importance, in order, was: Exacerbations, Sleep Quality, Shortness of Breath, Urinary Incontinence, Mucus Clearance and Cough.

In a health-state preference simulation, two profiles with qualitatively equivalent improvements in cough + mucus or shortness of breath, there was a clear preference for the cough + mucus improved profile. When comparing two profiles with improvement of daily symptoms (cough + mucus + SOB) or improvement in exacerbations, there was a clear preference for the daily symptoms improved profile.

A newly developed COPD-focused health utility measure derived from the DCE demonstrated face validity, with values negatively correlated with self-perceived COPD disease severity.

The new measure was also compared to the the generic quality of life instrument, EQ-5D. The COPD health utility measure showed an only moderate correlation with EQ-5D-3L. This discrepancy was largely attributable to the impact of patient comorbidities on EQ-5D-3L, whereas the COPD health utility score was less impacted by comorbid conditions.

The study showed patient preference studies early in the product lifecycle, focused on disease symptoms, are a useful way to assess which aspects of a disease matter most to patients and therefore inform the selection of endpoints for patient-centric clinical trials.

Brett Hauber

Brett Hauber, PhD, is the Senior Director, Patient Preferences in Worldwide Medical and Safety at Pfizer. He is also Affiliate Associate Professor in the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute at the University of Washington School of Pharmacy. Dr. Hauber was the Principal Investigator on the Catalogue of Patient Preference Methods developed by the Medical Device Innovation Consortium in 2015. He has been a member of multiple ISPOR Task Forces and was the Chair of the ISPOR Task Force for Good Research Practices for the Statistical Analysis of Discrete-Choice Experiments. He is past Benefit-Risk co-chair of the Benefit-Risk Assessment Communication and Evaluation special interest group of the International Society for Pharmacoepidemiology. He sits on the Editorial Boards of Pharmacoeconomics and The Patient. He was active in IMI-PREFER. Within Pfizer, he is the leader of the Patient Preference Studies Community of Practice, co-chair of the Patient Preference Studies Technical Advisory Committee, and co-Chair of the Benefit-Risk Advisory Council.

Industry case (Pfizer)

Patient preferences can be used to inform many decisions during the drug-product lifecycle. One such decision is dose selection. Ultimately, this decision can be described as comparing preferences for two products. However, at the time of dose selection, information about the risks and benefits of each dose may be limited, even if the case where Phase 3 data exist for both doses. This presentation will describe a conceptual framework for addressing this question and for applying data from DCE to support dose selection decisions.

Bennett Levitan

Bennett Levitan, MD-PhD is Senior Director, Benefit-risk Assessment, Department of Epidemiology at Janssen R&D, Pharmaceutical Companies of Johnson & Johnson. He introduced state of the art patient-focused benefit-risk assessment to Johnson & Johnson and his group has led numerous clinical teams in preparation of benefit-risk assessments and patient preference studies for regulatory submissions and health authority advisory meetings. He has co-led cross-disciplinary teams to implement processes to support growing regulatory requirements for patient-focused benefit-risk assessment both during development and post-approval. Bennett has published widely on both theoretical and pragmatic aspects in benefit-risk and patient preference studies and is a frequent speaker on these topics in national and international conferences. He co-led development of the PhRMA Benefit Risk Action Team (BRAT) Framework for drug benefit-risk assessment, the Medical Device Innovation Consortium (MDIC) Patient Centered Benefit-Risk Framework, the Clinical Trials Transformation Initiative’s financial value of patient engagement project and the Innovative Medicines Initiative (IMI) PREFER project on patient preference studies. Bennett serves on several organizations and committees that inform policy on benefit-risk methods including co-leading the Brighton Collaboration BRAVATO Benefit-Risk Working Group, MDIC’s Science of Patient Input Initiative and serving as a member of the International Academy of Health Preference Research and the PhRMA Patient-Focused Drug Development Work Group. Bennett received his B.Sc. (Electrical Engineering) from Columbia University in New York and his M.D.-Ph.D. (Bioengineering) from the University of Pennsylvania and was a postdoctoral fellow at the Santa Fe Institute.

The relevance of fatigue to relapse rate in multiple sclerosis: Applying patient preference data to the OPTIMUM trial
Industry case (Johnson & Johnson)

Background: Patients with multiple sclerosis (MS) experience multiple symptoms including physical and cognitive fatigue, dysesthesia, gait difficulties, vision problems, dizziness, pain, and depression. Fatigue is the most common MS symptom, occurring in about 80% of MS patients.  In the OPTIMUM trial in patients with relapsing MS, treatment differences in annualized relapse rate (0.088) and change in fatigue at week 108 (3.57 points, measured using the Fatigue Symptoms and Impacts Questionnaire–Relapsing Multiple Sclerosis, symptom domain (FSIQ-RMS-S)) both favored ponesimod over teriflunomide. However, the importance of the fatigue outcome to patients was unclear.

Objective: To assess the importance of the OPTIMUM FSIQ-RMS-S results using data from an MS discrete choice experiment (DCE).

Methods: The DCE included components to correlate levels of physical and cognitive fatigue with FSIQ-RMS-S scores. Changes in relapses/year and time to MS progression equivalent to the treatment difference in fatigue in OPTIMUM were determined for similar fatigue levels as mean baseline fatigue in OPTIMUM.

Results: DCE participants would accept 0.06 more relapses/year or a 0.15–0.17 year decrease in time to MS progression for a 3.57-point difference in physical fatigue on the FSIQ-RMS-S. To improve cognitive fatigue by 3.57-points on the FSIQ-RMS-S, DCE participants would accept 0.09–0.10 more relapses/year or a 0.24–0.28 year decrease in time to MS progression.

Conclusion: MS patients would accept 0.06 more relapses/year to change their fatigue by a similar magnitude as the between-treatment difference observed in the OPTIMUM trial.  These results suggest that, for MS patients, in the OPTIMUM trial, the between-treatment difference in relapse rate is equal in importance to the between-treatment difference fatigue measured with the FSIQ-RMS-S.


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