How Will New Guidance Impact the Statistical Analyses and Interpretation for Patient-Reported Outcomes (PRO) in the Context of HA and HTA Submissions


Moderator: Katja Rudell, PhD, MSc, Parexel Access Consulting, Parexel International, London, UK 
Panelists: Olivier Chassany, MD, PhD, Patient-Centered Outcomes Research, University Paris-Diderot, Paris, 75, France;Michael Schlichting, Diplom, Global Biostatistics, Epidemiology & Medical Writing, Merck KGaA, Darmstadt, HE, Germany; Corneel Coens, MSc, Quality of Life Department, EORTC, Brussels, Belgium


ISSUE: We observe high levels of variability from one sponsor, as well as across sponsors, in the analyses and presentations of PRO data for regulatory & HTA submissions. Whilst qualitative data and design of instruments have been highly regulated, the approach in statistical analysis and interpretation is mainly left to the sponsors – which can lead to flexibility but also inefficiency in submissions. We will debate from sponsor, clinical and PRO research perspectives how new guidance on statistics can aid forthcoming PRO submissions. We look forward to debate the topic among the audience involved in HTA and regulatory submissions.

OVERVIEW: Moderator Dr Katja Rudell will introduce the topic with an example overview of statistics from one product and highlight the difficulties for patients and HCPs to understand the nuances in the analyses (10mins) and then invite each panellist to present perspectives on how to improve presentations, as well as the impact of the latest guidance including estimands on this topic (10mins each). Dr Olivier Chassany will describe the clinician and reviewer perspectives from his experience with EMA/HAS; Michael Schlichting will frame it from the sponsor perspective; and Corneel Coens will describe the EORTC perspective. 20mins will be allocated to audience discussion and debate, for a total of 60 minutes. Stakeholders who will benefit attending include HTA scientists involved in review of PRO submissions as well as industry specialists involved in designing the submissions

Conference/Value in Health Info

2020-11, ISPOR Europe 2020, Milan, Italy




Clinical Outcomes

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