Time: 14:00 - 15:30 UK Time Presenters: Kim Cocks (Adelphi Values), Andrew Thompson (EMA) and Christoph Gerlinger (Bayer)
Clinical Trials are increasingly featuring a wide variety of Patient Reported Outcomes (PROs). PROs are important endpoints to both regulatory and health technology bodies in their assessments and approvals. There are standard ways in which PRO instruments are developed and validated, as well as important concepts for designing and interpretation of PRO data in clinical trials such as assessing the Minimally Clinically Important Differences (MCIDs/MIDs) and the use of a Responder definition.
The intent of this webinar is to describe an overview of how PROs tools are developed and used in clinical trial settings and how results can be clinically and meaningfully interpreted. In addition, a regulatory viewpoint will be shared on the key considerations for PROs. The talks will also include examples and case-studies showing the derivation of a validated thresholds for treatment responder and interpretation of PROs in the context of regulatory approval.
Kim Cocks is a Director and Principal Statistician at Adelphi Values, a global healthcare consultancy. She has worked as a medical statistician and clinical trial methodologist for over 20 years across pharmaceutical, academic and consultancy environments. She specialises in PRO interpretation and analysis and is an active member of both the EORTC Quality of Life group and ISOQOL.
Abstract Overview of PROs and clinically meaningful interpretation
PROs are validated instruments designed to provide a direct report from the patient on aspects of their health including symptoms and functioning. This talk will provide a brief overview of how PROs are developed, how they provide a score for multi-dimensional aspects of a patient’s health and why these can be difficult to interpret. The variety of quantitative and qualitative methods available to aid interpretation will be introduced.
Andrew Thomson (EMA)
Andrew Thomson is a statistician at the EMA, in the Human Medicines Research & Development Support Division. Prior to joining the EMA in 2014, he spent 7 years at the MHRA, initially as a Statistical Assessor in the Licensing Division, and subsequently Head of Epidemiology in the Vigilance & Risk Management of Medicines Division.
Abstract Regulatory Considerations for PROs In this talk I will present some regulatory considerations on the use of PROs in regulatory assessment, and how they can be developed. These will be discussed alongside a recent case example that has been through the qualification procedure at EMA.
Christoph Gerlinger (Bayer)
Christoph Gerlinger is Bayer’s Expert Statistician for Health Technology Assessment and Women’s Health. He is the regulatory chair of the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI) and a work package leader for the IMI BigData@Heart project. Christoph worked as statistician over 25 years in the pharmaceutical industry and in his spare time he teaches at the University Medical School of Saarland.
Abstract Empirical derivation of the minimal important difference for PROs In this talk I will present some methods on how to derive the minimal important difference for PROs from a clinical trial. I will present a worked example where we used this empirical minimal important difference to formulate a responder definition that was then accepted by the FDA.
PSI, the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI) and the Biopharmaceutical Section of the American Statistical Association (ASA) are jointly organising a webinar on Estimands in Practice. Speakers from regulatory authorities (FDA and EMA) and industry will present on their experience on this topic to date.
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