PSI One day Scientific meeting: The analysis and reporting of PROs in Clinical Trials

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)
Helen Doll (Clinical Outcome Solutions)
Kim Cocks (Adelphi Values)
Melanie Calvert (Birmingham University)
Ingolf Griebsch (Boehringer-Ingelheim)
Tara Symonds (Clinical Outcome Solutions)

 16:00 Close


   Registration Fee
PSI Member  £40+VAT 
Non-Member £135+VAT (price includes PSI membership for the year)

Please click here to register.

Please click here to download the flyer. 


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.


Melanie Calvert (Birmingham University)                                             

Improving the design and reporting of PRO trial data: implementation of the SPIRIT-PRO and CONSORT-PRO Extensions

Abstract: 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.

Biography: 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.

 Ingolf Griebsch (Boehringer-Ingelheim)  

Analysis of PROs in Oncology: A Perspective from Industry


Abstract: 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.


Biography: 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”.



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).


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.


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. 
 All presenters  Q & A Session

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