Date: Wednesday 29th May 2024
Time: 14:00-16:00 BST | 15:00-17:00 CEST
Location: Online via Zoom
Speakers: Oriana Ciana (SDA Bocconi School of Management), Rod Taylor (University of Glasgow), Anthony Manyara (Bristol University), Ray Harris (Retired) and Mario Ouwens (AstraZeneca).
Who is this event intended for? Clinical trial statisticians involved in evaluation of surrogate outcomes.
What is the benefit of attending? Update on extension of CONSORT/SPIRIT guidance on protocol writing and reporting of randomized controlled trials in which surrogate outcomes are used. Update on expectation setting for surrogacy validation for chronic diseases.
This event is free to both Members of PSI and Non-Members. To register your place for this event, please click here.
The webinar will start with providing an overview of surrogacy for licensing and reimbursement. This will articulate the need of extensions of the SPIRIT and CONSORT statement related to surrogacy. Both the process to define the extensions as well as the extensions themselves will be presented. The importance of right communication of the interpretation of surrogate outcomes to patients will be articulated by a patient representative. Finally, it will be shown by results from a simulation study that maturity influences the probability of success of surrogacy validation, among other factors like variation in estimated treatment effects. Time is reserved for panel discussion and Q&A.
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Oriana Ciani is Associate Professor of Practice, Public Management and Policy Area, Health Economics and HTA at SDA Bocconi School of Management. Her research interests focus on methodological aspects of Health Technology Assessment (HTA), evidence synthesis and surrogate endpoints validation. She has led the development of CONSORT and SPIRIT extensions for RCTs with surrogate endpoints (MRC CONSORT-SPIRIT). Oriana is currently PI for the three-year HORIZON Europe HI-PRIX project (no. 101095593), on innovative pricing and payment schemes for health technologies. In 2020 Oriana is Fulbright Research Scholar at the Yale School of Medicine and the Yale School of Public Health. Oriana is Associate Editor of Value in Health. For her research activity she has received the Egon Jonsson Award (2016), the Bocconi Research Award (2023), the Exeter Impact Award (2013), Best Student Podium Presentation Award at the ISPOR Conference in Dublin (2013), the AIES Young Researcher Award (2010). |
A significant proportion of clinical trials across different therapeutic areas use surrogate endpoints as primary outcomes. After introducing a published definitional framework for surrogate endpoints in trials, the presentation will highlight the advantages and challenges of using them for clinical and regulatory decision-making. Finally, an overview of available validation approaches will be provided. |
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Rod Taylor is Professor of Population Health Research and co-Director of Centre for Excellence in Trials Collaboration, University of Glasgow; Adjunct Professor, Faculty of Health Sciences – National Institute of Public Health and Department of Psychology, University of South Denmark. He has published over 500 peer review articles the field of health services research and has an H-index of 125 and i10-index 416. Rod’s main research interests and expertise include cardiovascular secondary prevention/rehabilitation trials: REACH-HFpEF Trial, PERFORM trial, ACROSS trial and trial methodology. He is currently leading the development of CONSORT and SPIRIT extensions for RCTs with surrogate endpoints (MRC CONSORT-SPIRIT) and he is member of the Strategy Committee of the UK Medical Research Council (MRC) and National Institute for Health Research (NIHR) Trial Methodology Research Partnership (TMRP). He is the Director for the Cardiac Rehabilitation Cochrane Review Centre (CochraneCardiacRehabCentre). His postgraduate qualifications include PhD Clinical Physiology (Glasgow), MSc in Medical Statistics (London) and Postgrad. Dip. Health Economics (Aberdeen). His postgraduate qualifications include PhD Clinical Physiology (Glasgow), MSc in Medical Statistics (London) and Postgrad. Dip. Health Economics (Aberdeen). |
This presentation will describe the process by which we developed extensions to the SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) and CONSORT (Consolidated Standards of Reporting Trials) reporting guidelines to improve the design and completeness of reporting of RCTs and their protocols using a surrogate endpoint as a primary outcome. In brief, the project used the 4-stage process recommended for development of methods guidance by EQUATOR: stage 1 (literature reviews) to identify candidate reporting items to be rated in a Delphi study; stage 2 (e-Delphi study) to rate the importance of items identified in stage 1 & receive suggestions for additional items; stage 3 (consensus meeting) to agree on final set of items for inclusion in the extensions; stage 4 (knowledge translation) to engage stakeholders and disseminate the project outputs through various strategies including peer-reviewed publications. Patient and public involvement was embedded into all project phases. |
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Anthony Manyara is a Research Associate and Epidemiologist at the Global Health and Ageing Research Unit, Bristol Medical School, University of Bristol and Honorary Research Fellow at the School of Health and Wellbeing, University of Glasgow. His research interests include surrogate endpoints use and reporting, evidence synthesis, noncommunicable disease prevention and control. He is a member of the MRC-NIHR Trials Methodology Research Partnership Surrogate Endpoints subgroup. Anthony coordinated the development of the SPIRIT and CONSORT extensions for reporting trials using surrogate endpoints as a postdoctoral researcher and is keen to see the extensions implemented to improve trial reporting, reduce research waste, and better patient care and population health. |
After introducing the development of the SPIRIT|CONSORT-Surrogate extension, the extension items to be reported for both protocols and trial reports will be presented. A rationale for reporting each item and examples of good reporting will be provided. |
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Ray Harris is retired from a career in various statistical roles, mostly in the pharmaceutical industry. He worked across all stages of drug development. He is an honorary member of PSI and a Chartered Statistician. He continues to use his experience in a variety of volunteer roles. He is a lay member of an ethics committee and an expert member of the Oxford Primary Care Hosted Research Datasets Independent Scientific Committee. He is a PPI member in a number of groups. He has found that years of asking questions as a non-clinical member of a research team transitions very well to asking questions from a patient and public interest point of view. |
From the beginning of the project patient and public involvement (PPI) was recognised as a key part of the process, engaging representatives of a key stakeholder community. The presentation will outline the PPI involvement; representing the views, interests and concerns of the public; and the benefits of PPI engagement. |
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Mario Ouwens is group director in medical and statistical evidence statistics at AstraZeneca. He is co-leading the ISPOR surrogacy taskforce and has participated in the development of the CONSORT and SPIRIT extensions. |
A simulation study will be presented showing that it is quite unlikely to satisfy the IQWiG rules for more chronic diseases. The impact of the following factors will be evaluated for a time-to-event late outcome and continuous early outcome: a) Trial duration b) Sample size c) Number of trials d) Variation in treatment effect on early outcome e) Rate of mortality f) Extend to which late outcome can be explained by relation between early and late outcome Treatment effect on late outcome |
