Roche, 6 Falcon Way, Shire Park, Welwyn Garden City, AL7 1TW
Patient reported outcomes have become increasingly important in the development, approval and reimbursement of our products. The PSI Scientific Committee have put together this one day meeting to provide statisticians with introductions to PROs, how to include them in clinical trials, considerations about missing data, appropriate statistical methods to analyse and interpret the data and the perspective of an IQWiG assessor. Presenters will give their insight into discussions with colleagues, working groups and regulators and there will be plenty of opportunity to ask any questions you may have about PROs in your development program.
| Time | Topic |
| 9:00 – 9:30 | Registration |
| 9:30 – 9:45 | Welcome and Introduction to PSI and ISOQoL |
| 9:45 – 10:30 | Introduction to Patient Reported Outcome (PRO) Measurement
Tara Symonds (Clinical Outcome Solutions) |
| 10:30 – 10:45 | Break |
| 10:45 – 11:30 | Improving the design and reporting of PRO trial data: implementation of the SPIRIT-PRO and CONSORT-PRO Extensions
Melanie Calvert (Birmingham University) |
| 11:30 – 12:15 | Analysis of PROs in Oncology: A Perspective from Industry
Ingolf Griebsch (Boehringer-Ingelheim) |
| 12:15 – 13:15 | Lunch |
| 13:15 – 14:00 | Analysis of PRO data: SISAQOL recommendations and practical examples
Kim Cocks (Adelphi Values) |
| 14:00 – 14:45 | The Analysis & Interpretation of Patient Reported Outcomes (PROs)
Helen Doll (Clinical Outcome Solutions) |
| 14:45 – 15:00 | Break |
| 15:00 – 15:30 | A perspective from IQWiG when assessing treatment effects with PROs
Christoph Schürmann (IQWiG) |
| 15:30 – 16:00 | Q&A
Christoph Schürmann (IQWiG) |
| 16:00 | Close |
Registration
| Registration Fee | |
| PSI Member | £40+VAT |
| Non-Member | £135+VAT (price includes PSI membership for the year) |
Please click here to register.
 Tara Symonds (Clinical Outcome Solutions) |
Introduction to Patient Reported Outcome (PRO) Measurement
Abstract: 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.
Biography: 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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 Melanie Calvert (Birmingham University) |
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| Ingolf Griebsch (Boehringer-Ingelheim) |
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 Kim Cocks (Adelphi Values) |
SISAQoL guidance & analysis methods for missing data Abstract: 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. Biography: D r 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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 Helen Doll (Clinical Outcome Solutions) |
The Analysis & Interpretation of Patient Reported Outcomes (PROs) 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 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. Biography: 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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 Christoph Schürmann (IQWiG) |
A perspective from IQWiG when assessing treatment effects with PROs
Abstract: 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. Biography: 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 20019. 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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| All presenters | Q & A Session |
