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\n Tara Symonds (Clinical Outcome Solutio ns) \; \; \; \; \; \; \; \; \ ; \; \; \; \; \; \; \; \; \; \; \; \; \; \; \; \; \; \; &n bsp\; \; \; \; \; \; \; \; \; &nbs p\; \; \; \; \; \; \; \; \; \ ; \; \; \; \; \; \; \; \; \; \; \; \; \; \; \; \; \; \; &n bsp\; \; \; \; \; \; \; \; \; &nbs p\; \; \; \; \; \; \; \; \; \ ; \; \; \; \; \; \; \; \; \; \; \;
Introductio n to Patient Reported Outcome (PRO) Measurement
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\nAbstract: This introduction to PRO measurement will initially cover what a PRO meas ure is and is not\, its history and importance in drug development from va rious stakeholder perspectives (patient\, clinician\, payer and regulator) . \; The latter half of the presentation will concentrate on PRO devel opment and validation. \; The presentation will outline the basic psyc hometric properties to investigate to demonstrate a measure is reliable\, valid\, and sensitive-to-change. \; An overview of deriving a meaningf ul change threshold using an anchor-based approach will also be discussed.
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\nBiography: 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 b oth negotiating labelling and in disseminating results effectively with pa yers. \; She has worked in numerous therapeutic areas with extensive e xpertise in Sexual Health\, Women&rsquo\;s Health\, Pain\, and Rare Diseas es. \; 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: Cap pelleri et al (2014) Patient Reported Outcomes: \; Measurement\, Imple mentation and Interpretation\, CRC Press. \; Tara holds a Doctorate in Health Psychology from the University of Huddersfield\, UK.
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\n Melanie Calvert (Birmingham University) &nbs p\; \; \; \; \; \; \; \; \; \ ; \; \; \; \; \; \; \; \; \; \; \; \; \;
| \n Abstract: Patient-reported outcome (PRO) resu lts from clinical trials can provide valuable evidence to inform clinical decision making\, pharmaceutical labelling claims and health policy. Despi te this\, reviews of PRO protocol content and trial reporting demonstrate that the quality of PRO components remains highly variable. The aim of thi s presentation is to highlight current challenges in trial design and repo rting\, share examples of good practice\, and discuss approaches to implem entation and uptake of SPIRIT-PRO and CONSORT-PRO guidance. \nBiography: 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 cli nical trials and routine care\, to improve service delivery\, enhance pati ent care and outcomes and ensure that the patient perspective is at the he art of health research and healthcare decision-making. She is the cross-cu tting theme lead for PROs research within National Institute for Health Re search 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 (I SOQOL) Board of Directors she currently Chairs the ISOQOL Standards and Be st Practice and Committee and is a member of the Best Practice for PROs in Clinical Trials Task force. She has >\;150 peer reviewed publications i n journals including the NEJM\, BMJ\, JAMA and the Lancet including: the S PIRIT-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). He r 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 committ ees leading national and international strategy for PROs research/implemen tation including collaborative research with the EORTC led SISAQOL initiat ive. \n | \n
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| \n Analysis of PROs in Oncol ogy: A Perspective from Industry \n\; \nAbstract: In my t alk\, 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 e videntiary standards employed by regulatory and health technology assessme nt agencies to evaluate these data and highlight the need to derive consen sus recommendation for the analysis of these data in Oncology to increasin gly use these data in decision making. \n&nb sp\; \nBiography: 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 20 12\, I have been leading the Global market access team for Oncology at Boe hringer Ingelheim. Lead of the industry consortium for the IMI 2 applicati on (Call 18) &ldquo\;Establishing international standards in the analysis of patient reported outcomes and health-related quality of life data in ca ncer clinical trials&rdquo\;. \n\; \ n | \n
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\n Kim Cocks (Adelphi Values)\n
SISAQoL guidance &\; analy sis methods for missing data
\nAbstract : This session will cover two topics\, firstly an overview of new guidelines aimed at standardizing the analysis of PROs in oncology clinic al trials and\, secondly\, presenting some practical examples of longitudi nal analyses dealing with informative missing data.
\nBiography: D r Kim Cocks is a Director in the Patient-Cente
red Outcomes team at Adelphi Values\, heading up their statistics and prog
ramming team in the UK. Kim has over 20 years&rsquo\; experience as a medi
cal 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 r eported outcome data in oncology trials (SISAQOL).
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\n Helen Doll (Clinica l Outcome Solutions)
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Abstract: 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 t o flag the main issues that should be considered in these analyses\, such as multiplicity of endpoints\, repeated assessment timepoints\, between-gr oup or within-subject analysis\, and treatment of missing data. Further di scussion of assessment and application of existing thresholds for evaluati ng meaningful change\, such as the use of PDFs and CDFs\, and detailed tab ular representation of outcome by anchor status\, will also be included. T he need to ensure that sufficient analyses for the relevant stakeholders a re captured in the clinical trial/additional HTA SAP will also be covered\ , so that these analyses\, and their interpretation\, take account of loca l and regional review needs to support HTA submissions.
\n < p>Biography: Helen Doll is the Strategic Lead of Quantita tive Sciences at Clinical Outcomes Solutions. Helen is a Medical Statistic ian and Psychometrician who has completed over 25 years of clinical resear ch experience\, both in academia and in the pharmaceutical industry. Prior to joining COS in the February of 2018\, Helen had led the quantitative t eam within ICON&rsquo\;s Patient Centred Outcomes group for more than five years. She has also held Senior Medical Statistician and Lecturer positio ns at the Universities of Oxford and East Anglia. Helen has co-authored mo re than 130 peer-reviewed papers\, is a Fellow of the Royal Statistical So ciety\, and provides statistical reviews for journals such as the British Medical Journal and Quality of Life Research. Helen holds a BSc in Biologi cal Sciences from the University of Warwick\, and a Diploma in Applied Sta tistics\, MSc and a DPhil in Medical Statistics from the University of Oxf ord.\n\n
Christoph Schü\;rmann (IQWiG)
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\n Abstract: Assessing patient reported outcom es is part of the (added) benefit assessment of medical interventions. PRO s usually are considered to analyse aspects of morbidity and health-relate d quality of life. For valid conclusions on PRO assessments\, suitable and validated instruments are required. This talk gives an overview on PRO as sessment criteria applied by IQWiG and commonly experienced challenges whe n interpreting aggregate data. In this context relevant matters are pre-pl anned periods of evaluation and cut-off choices along with strategies for handling missing data. Some examples from past assessments are presented t o illustrate these issues and their impact on the final assessment. \;\n
\n Biography: Christ oph Schü\;rmann graduated from the University of Dortmund with a diplo ma \;in Statistics in 2002 and with a doctoral degree in 20018. He's b een a research associate at the department of Medical Biometry at the Inst itute \;for Quality and Efficiency in Health Care (IQWiG) since 20019. His research interests are systematic reviews\, meta-analysis\, network m eta-analysis\, surrogate endpoint validation and health-related quality of life assessments. \;
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