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17 July 2019

Tara Symonds

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Tara Symonds This introduction to PRO measurement will initially cover what a PRO measure is and is not, its history and importance in drug development from various stakeholder perspectives (patient, clinician, payer and regulator). The latter half of the presentation will concentrate on PRO development and validation. The presentation will outline the basic psychometric properties to investigate to demonstrate a measure is reliable, valid, and sensitive-to-change. An overview of deriving a meaningful change threshold using an anchor-based approach will also be discussed. Tara Symonds is Strategic Lead at Clinical Outcomes Solutions providing advice on Clinical Outcome Assessments (COA) strategy from development to dissemination. Tara has 25+ years of experience in the COA field both negotiating labelling and in disseminating results effectively with payers. She has worked in numerous therapeutic areas with extensive expertise in Sexual Health, Women’s Health, Pain, and Rare Diseases. She worked in industry for 16 years, prior to this she worked in academia teaching undergraduate & postgraduate psychology. She has published 80+ peer reviewed articles and co-authored the book: Cappelleri et al (2014) Patient Reported Outcomes: Measurement, Implementation and Interpretation, CRC Press. Tara holds a Doctorate in Health Psychology from the University of Huddersfield, UK.

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17 July 2019

Melanie Calvert

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Melanie Calvert Patient-reported outcome (PRO) results from clinical trials can provide valuable evidence to inform clinical decision making, pharmaceutical labelling claims and health policy. Despite this, reviews of PRO protocol content and trial reporting demonstrate that the quality of PRO components remains highly variable. The aim of this presentation is to highlight current challenges in trial design and reporting, share examples of good practice, and discuss approaches to implementation and uptake of SPIRIT-PRO and CONSORT-PRO guidance. Professor Melanie Calvert, PhD, is Professor of Outcomes Methodology at the University of Birmingham UK. She is Director of the Centre for Patient Reported Outcomes Research (www.birmingham.ac.uk/cpror) which aims to optimize the use of PROs in clinical trials and routine care, to improve service delivery, enhance patient care and outcomes and ensure that the patient perspective is at the heart of health research and healthcare decision-making. She is the cross-cutting theme lead for PROs research within National Institute for Health Research infrastructure including the Biomedical Research Centre Birmingham, Surgical Reconstruction and Microbiology Research Centre and MedTech Co-operative and is a member of the National Research Ethics Advisory Panel. After serving on the International Society for Quality of Life Research (ISOQOL) Board of Directors she currently Chairs the ISOQOL Standards and Best Practice and Committee and is a member of the Best Practice for PROs in Clinical Trials Task force. She has >150 peer reviewed publications in journals including the NEJM, BMJ, JAMA and the Lancet including: the SPIRIT-PRO Extension (JAMA. 2018; 319(5):483-494) CONSORT-PRO (JAMA. 2013; 309(8):814-822) PRO-Alerts (JAMA. 2013; 310(12):1229-1230) and pathways to impact for medical research (PLoS Med. 2017 Aug 9;14(8):e1002370). Her highly cited work has informed European Society of Cardiology guidelines, NICE and EMA guidance. Prof Calvert works closely with a wide range of national and international collaborators to maximise the reach, influence and impact of her research. She sits on a number of international committees leading national and international strategy for PROs research/implementation including collaborative research with the EORTC led SISAQOL initiative.

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17 July 2019

Ingolf Griebsch

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Ingolf Griebsch In my talk, I will discuss the current methodology available to devise PRO data collection strategies and the analytical approaches used to analyse these data in Oncology clinical studies. I will also briefly discuss different evidentiary standards employed by regulatory and health technology assessment agencies to evaluate these data and highlight the need to derive consensus recommendation for the analysis of these data in Oncology to increasingly use these data in decision making. Study of pharmacy, public health and health economics. Work as research associate at the MRC HSRC, based at the Department of Social Medicine, University of Bristol. from 2001 to 2005. PhD in health economics from the University in Bristol in 2009. Move to the pharmaceutical industry in 2005. Since 2012, I have been leading the Global market access team for Oncology at Boehringer Ingelheim. Lead of the industry consortium for the IMI 2 application (Call 18) “Establishing international standards in the analysis of patient reported outcomes and health-related quality of life data in cancer clinical trials”.

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17 July 2019

Kim Cocks

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Kim Cocks This session will cover two topics, firstly an overview of new guidelines aimed at standardizing the analysis of PROs in oncology clinical trials and, secondly, presenting some practical examples of longitudinal analyses dealing with informative missing data. Dr Kim Cocks is a Director in the Patient-Centered Outcomes team at Adelphi Values, heading up their statistics and programming team in the UK. Kim has over 20 years’ experience as a medical statistician working in clinical trials and associated methodological research. She has worked across pharmaceutical, medical device, academia and consultancy companies. Kim is a Board member for ISOQOL (International Society for Quality of Life Research) and part of an international consortium seeking to standardise the analysis of patient reported outcome data in oncology trials (SISAQOL).

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17 July 2019

Helen Doll

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Helen Doll This session on the analysis and interpretation of PROs will introduce the main types of statistical analyses that can be applied to the analysis of PRO data and to flag the main issues that should be considered in these analyses, such as multiplicity of endpoints, repeated assessment timepoints, between-group or within-subject analysis, and treatment of missing data. Further discussion of assessment and application of existing thresholds for evaluating meaningful change, such as the use of PDFs and CDFs, and detailed tabular representation of outcome by anchor status, will also be included. The need to ensure that sufficient analyses for the relevant stakeholders are captured in the clinical trial/additional HTA SAP will also be covered, so that these analyses, and their interpretation, take account of local and regional review needs to support HTA submissions. Helen Doll is the Strategic Lead of Quantitative Sciences at Clinical Outcomes Solutions. Helen is a Medical Statistician and Psychometrician who has completed over 25 years of clinical research experience, both in academia and in the pharmaceutical industry. Prior to joining COS in the February of 2018, Helen had led the quantitative team within ICON’s Patient Centred Outcomes group for more than five years. She has also held Senior Medical Statistician and Lecturer positions at the Universities of Oxford and East Anglia. Helen has co-authored more than 130 peer-reviewed papers, is a Fellow of the Royal Statistical Society, and provides statistical reviews for journals such as the British Medical Journal and Quality of Life Research. Helen holds a BSc in Biological Sciences from the University of Warwick, and a Diploma in Applied Statistics, MSc and a DPhil in Medical Statistics from the University of Oxford.

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17 July 2019

Christoph Schürmann

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Christoph Schürmann

Assessing patient reported outcomes is part of the (added) benefit assessment of medical interventions. PROs usually are considered to analyse aspects of morbidity and health-related quality of life. For valid conclusions on PRO assessments, suitable and validated instruments are required. This talk gives an overview on PRO assessment criteria applied by IQWiG and commonly experienced challenges when interpreting aggregate data. In this context relevant matters are pre-planned periods of evaluation and cut-off choices along with strategies for handling missing data. Some examples from past assessments are  presented to illustrate these issues and their impact on the final assessment. Christoph Schürmann graduated from the University of Dortmund with a diploma in Statistics in 2002 and with a doctoral degree in 20018. He's been a research associate at the department of Medical Biometry at the Institute for Quality and Efficiency in Health Care (IQWiG) since 2019. His research interests are systematic reviews, meta-analysis, network meta-analysis, surrogate endpoint validation and health-related quality of life assessments.

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17 July 2019

Rachael Lawrence Q&A

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10 July 2019

Peter Thall and Kyu Ha Lee

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Discussion on survival analysis method with focus on the application of two Bayesian methods for time to event data.  Our two presenters are Peter Thall from MD Anderson and Kyu Ha Lee from Harvard. The discussants are: Axel Gandy, Imperial College London (RSS discussant) and Neby Bekele, Gilead Sciences (PSI discussant).

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04 July 2019

Jürgen Hummel, Kaspar Rufibach and Kit Roes

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Jürgen Hummel, Kaspar Rufibach and Kit Roes In September 2018 the FDA published a guidance on adaptive design for clinical trials of drugs and biologics, updating (not finalizing) its initial draft from February 2010.  The main focus of the webinar is to provide an overview of its main contents, which is presented by Jürgen Hummel (PPD). In addition, Kaspar Rufibach (Roche) introduces an open-source statistical software for adaptive designs, RPACT (an R package that enables the design and analysis of confirmatory adaptive clinical trials).  Kit Roes also comments on the guidance from a European Regulatory perspective.



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17 June 2019

Jim Whitwell, Darren Kidd and Robert Smith

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The in vivo Pig-a gene mutation assay is based on the endogenous X-linked Pig-a gene  (phosphatidylinositol N-acetylglucosaminyltransferase, subunit A). The Pig-a gene codes for an N-acetyl glucosamine transferase that is involved in the biosynthesis of Glycosyl phosphatidyl inositol (GPI) anchor for tethering a variety of mammalian cell surface protein markers. A single mutation in the Pig-a gene results in loss of the GPI anchor and the GPI-tethered protein markers and can be detected by fluorescent antibodies against the surface markers and flow cytometric analysis to quantify marker deficient cells. The Pig-a assay has been evaluated internationally and the data generated to date indicate that it is sensitive, robust and has high reproducibility and transferability. With this in mind, Working Groups of the International Workshop on Genotoxicity Testing (IWGT) and HESI Genetic Toxicology Technical Committee (GTTC) have been working towards the development of an OECD testing guideline for performing the Pig-a assay in rodents, which will allow the Pig-a assay to be used for regulatory assessments in drug development. Current guidance for the assessment of DNA reactive (mutagenic) impurities in pharmaceuticals (ICH M7(R1)) has already suggested the use of the Pig-a assay to investigate the in vivo relevance of in vitro mutagens (Ames positive).

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