• PSI Toxicology SIG Webinar "Pig-A Assay":

    Dates: 18 – 18 Jun, 2019

    18 June 2019 14:00-15:00

    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).

    Speaker Biographies 

    Jim Whitwell
    Jim has a BSc (Hons) degree in Applied Biological Sciences from the University of Bristol. He has more than 25 years of experience in genetic toxicology and related fields and currently serves as Scientific Specialist and Subject Matter Expert for in vitro micronucleus assays within Genetic Toxicology.

    His areas of expertise include in vitro and in vivo chromosome aberration and micronucleus assays, peripheral blood and flow in vivo micronucleus studies and non-standard assay designs (e.g. sister chromatid exchange and mechanistic mode of action investigations).

    He is a member of several industry groups and has presented at industry conferences, as well authoring and co-authoring multiple publications in journals such as Mutation Research and Mutagenesis.

    Darren Kidd
    Darren has a BSc (Hons) degree in Biological Sciences from the University of Plymouth and a PhD entitled “In Vitro Gene Mutation and Apoptosis” from the University of York. He has more than 20 years of experience as scientist, toxicologist and study director in laboratory research and currently serves as Lead Scientist for in vitro toxicology assays within Genetic Toxicology.

    His areas of expertise include in vitro micronucleus, skin, ocular, phototoxicity and cytotoxicity assays, flow cytometric applications and mechanistic investigations.

    He is member of several industry groups and has authored or co-authored dozens of posters, presentations and publications in journals such as Mutation Research, Toxicology in vitro, Genetic Toxicology and Environmental Mutagenesis, Methods in Molecular Biology and Mutagenesis.

    Robert Smith 
    Robert has a MSc in Biology from the University of York. He has over 15 years of experience within genetic toxicology at Covance from working in the laboratory on standard and non-standard genotoxicity assays and currently serves as a Study Director within Genetic Toxicology with emphasis on screening as well as investigative work and method development.

    His areas of expertise include Ames, in vitro and in vivo chromosome aberration and micronucleus assays, in vivo Comet and MutaMouse, 3D skin micronucleus and fluorescence in situ hybridisation (FISH) assays.

    He is member of several industry groups and is the current secretary of UKEMS. He has authored or co-authored publications in Mutation Research and Mutagenesis.


    This webinar is free to attend, to register please click here.

  • PSI One day Scientific meeting, South West: Designing and Analysing Adaptive Trial Design Studies

    Dates: 24 – 24 Jun, 2019

    Date: 24th June 2019

    Location: Bath University, 10-4pm UK time 


     Time     Topic
     09:30 -10:00 Registration
     10:00-10:15 Welcome and Introduction to PSI Scientific Committee & South West Events
     10:15 -12:00 Group sequential and adaptive clinical trial designs

    Professor Chris Jennison (University of Bath)
     12:00 -12:45 Lunch
     12:45 -13:30  Facilitating Personalised Healthcare With Adaptive Designs

    Chris Harbron, Senior Principal Statistical Scientist, Roche
     13:30 -14:15 Introducing the Adaptive designs CONSORT Extension (ACE) Statement to improve reporting of randomised trials that use an adaptive design

    Munya Dimairo (ACE Steering Committee, University of Sheffield)

     14:15- 14:30  Break
     14:30 -15:15 Monitoring Outcomes in Adaptive Designs

    Sharon Barton, Associate Director Oncology & Early Clinical Development, AstraZeneca
     15:15 -16:00  A regulatory perspective of adaptive design trials

    Beatrice Panico, Senior Medical Assessor, (MHRA)
     16:00 -16:15  Close

    Chris Jennison (University of Bath), Munya Dimairo (University of Sheffield), Beatrice Panico (MHRA), Chris Harbron (Roche), Sharon Barton (Astrazeneca) 

    Adaptive designs are clinical trials that allow for prospectively planned modifications to one or more aspects of the design based on accumulating data from subjects in the trial and can provide a number of advantages over non-adaptive designs.  During this meeting we will hear about adaptive sample allocation for phase II/III designs, a new CONSORT extension reporting guideline for adaptive designs, regulatory aspects and case studies.
    Chris Jennison photo

    Chris Jennison (University of Bath),
    Christopher Jennison is Professor of Statistics at the University of Bath, UK. His PhD research at Cornell University concerned the sequential analysis of clinical trials and he has continued to work in this area for the past 35 years. His book with Professor Bruce Turnbull, "Group Sequential Methods with Applications to Clinical Trials", is a standard text on this topic and is widely used by practising statisticians. More recently, he has written with a variety of co-authors on adaptive trial design and over-arching optimisation of the drug development process.

    Professor Jennison's research is informed by experience of clinical trial analysis at the Dana Farber Cancer Institute, Boston and a broad range of consultancy with Medical Research institutes and Pharmaceutical companies.

    Group sequential and adaptive clinical trial designs

    We shall describe group sequential methods for monitoring a clinical trial that compares a new treatment against a control. This methodology is applicable across a range of response distributions. When the primary endpoint is a time-to-event outcome, tests constructed using the error spending approach are able to accommodate the unpredictable numbers of events at each analysis. We shall see how group sequential testing can lead to an earlier conclusion of the trial and fewer patients recruited.

    In some Phase III clinical trials, more than one new treatment is compared to the control. We shall consider an adaptive clinical trial in which two versions of a new treatment are to be compared with a control when the primary endpoint is overall survival. At an interim analysis, one of the two treatments will be selected based on observed progression free survival. Then, in the remainder of the trial new patients will be randomised between the selected treatment and the control.

    This three arm trial requires an adaptive design. A key element of such a design is a closed testing procedure which protects the familywise type I error rate when two different null hypotheses may be tested. Another crucial component of the design is a combination test that can merge data from before and after the interim analysis. We shall discuss closed testing procedures and combination tests in general before applying these methods to our three arm trial. With this methodology in place, we then assess the potential benefits of treatment selection in this adaptive trial design.

    Munya Dimairo (University of Sheffield)
    Munya is a Research Fellow in Medical Statistics within the Sheffield Clinical Trials Research Unit at the University of Sheffield. He is involved in the design, conduct, analysis, and reporting of clinical trials. He is the lead Trial Statistician of an ongoing adaptive multi-arm multi-stage adaptive trial and IDMC Statistician on several trials. Munya is interested in the use of innovative trial designs and is collaborating on a number of initiatives to bridge gaps in the practical application of adaptive designs. For example, he is leading the development of the CONSORT Extension for randomised adaptive trials and the creation of an online platform to educate researchers across disciplines on the practical application of adaptive designs in randomised trials.

    Introducing the Adaptive designs CONSORT Extension (ACE) Statement to improve reporting of randomised trials that use an adaptive design

    Munya Dimairo on behalf of the ACE Steering Committee (m.dimairo@sheffield.ac.uk; mdimairo@gmail.com)

    ACE Steering Committee: Munya Dimairo; Philip Pallmann; James Wason; Susan Todd; Thomas Jaki; Steven A. Julious; Adrian P. Mander; Christopher J. Weir; Franz Koenig; Marc K. Walton; Jon P. Nicholl; Elizabeth Coates; Katie Biggs; Toshimitsu Hamasaki; Michael A. Proschan; John A. Scott; Yuki Ando; Daniel Hind; and Douglas G. Altman

    The reporting of adaptive designs (ADs) in randomised trials is inconsistent and needs improving 1–4. Incompletely reported AD randomised trials are difficult to reproduce and are hard to interpret and synthesise. This consequently hampers their ability to inform practice as well as future research and contributes to research waste. Better transparency and adequate reporting will enable the potential benefits of ADs to be realised.

    We developed an Adaptive designs CONSORT Extension (ACE) guideline through a two-stage Delphi process with input from multidisciplinary key stakeholders in clinical trials research in the public and private sectors from 21 countries, followed by a consensus meeting 1. Delphi survey response rates were 94/143 (66%), 114/156 (73%), and 79/143 (55%) in round one, two and across both rounds, respectively. Members of the CONSORT Group were involved during the development process.

    This talk will summarise the development process and introduce the ACE reporting guideline focusing on new and modified reporting items. The ACE checklist is comprised of seven new items, nine modified items, six unchanged items for which additional explanatory text clarifies further considerations for ADs, and 20 unchanged items not requiring further explanatory text. The ACE abstract checklist has one new item, one modified item, one unchanged item with additional explanatory text for ADs, and 15 unchanged items not requiring further explanatory text.

    The intention is to enhance transparency and improve reporting of AD randomised trials to improve the interpretability of their results and reproducibility of their methods, results and inference. We also hope indirectly to facilitate the much-needed knowledge transfer of innovative trial designs to maximise their potential benefits.


    1.        Dimairo M, Coates E, Pallmann P, et al. Development process of a consensus-driven CONSORT extension for randomised trials using an adaptive design. BMC Med. 2018;16(1):210.

    2.        Stevely A, Dimairo M, Todd S, et al. An Investigation of the Shortcomings of the CONSORT 2010 Statement for the Reporting of Group Sequential Randomised Controlled Trials: A Methodological Systematic Review. PLoS One. 2015;10(11):e0141104.

    3.        Hatfield I, Allison A, Flight L, Julious SA, Dimairo M. Adaptive designs undertaken in clinical research: a review of registered clinical trials. Trials. 2016;17(1):150.

    4.        Yang X, Thompson L, Chu J, et al. Adaptive Design Practice at the Center for Devices and Radiological Health (CDRH), January 2007 to May 2013. Ther Innov Regul Sci. 2016;50(6):710-717.

    MBeatrice Panico

    Beatrice Panico (MHRA)
    Maria Beatrice Panico is currently a Senior Medical Assessor in the Clinical Trials Unit at the Medicines and Healthcare products Regulatory Agency (MHRA).

    She is a medical doctor, fully qualified in Neurology with a PhD in Neuroscience. She has extensive experience in pharmacovigilance in the pharmaceutical industry.

    MHRA Presentation abstract

    The MHRA supports innovation and several trials with innovative designs are already ongoing in the UK. Some innovative trials are ‘adaptive design trials’: modifying the conduct of ongoing trials increases the chance of the trial formally being a success (i.e. that the null hypothesis can be rejected). A central tenet of adaptive design protocols is that the adaptations are pre-specified in the protocol and are not made on an ad-hoc basis. Trials have to be safe and scientifically sound. It is therefore crucial that Sponsors of adaptive design trials provide the regulators with a strong scientific rationale why an innovative design is the best solution to address the trial objectives rather than a more traditional approach. The rationale should also discuss how the trial integrity will be maintained despite continuous adaptations.

    Adaptations that can prove challenging in the current regulatory scenarios are addition of new Investigational Medicinal Products, new trial populations and some seamless Phase 2-3 trials.

    Such changes can be introduced via substantial amendments.

    However, if the proposed changes are so extensive that they change the nature of the initially approved trial (for example, they are not in line with the original research hypothesis, they make the data obtained up to the point of the amendment inadmissible or make the sponsor lose control of Type 1 error) then a new clinical trial application would probably be necessary. The decision is always on a case by case both for initials and amendments.

    In conclusion:  adaptations can be acceptable if safe and scientifically justified. Early engagement with regulators is strongly recommended in order to address potential issues of concerns.

     Chris Harbron (Roche) Personalised HealthCare (PHC), targeting therapies to those patients most likely to benefit is becoming an increasingly key strategy within drug development. However during development a challenge is that there can be uncertainty on the optimal PHC strategy, both on the need for a selected population and the exact definition of a subpopulation, for example which assay to use to measure a biomarker and with what cutoff. Adaptive designs provide an efficient way of mitigating this uncertainty whilst still maintaining the overall rigour and operating characteristics of the trial.  I will review several published adaptive designs for biomarkers and describe in more detail a method for adapting a biomarker threshold at interim analyses to balance the power of the study and the precision in estimating the threshold.
     Sharon Barton (Astrazeneca)  Abstract: Within early clinical trials we aim to make robust decisions as early as possible, typically at a planned interim or final analysis using pre-defined decision criteria.  An alternative approach would be to include continuous monitoring on a key efficacy or safety endpoint in addition to these planned analyses.  This approach uses a predictive power calculation to assess the chance of observing a given rate or better.  The predictive power can be recalculated after each patient’s outcome is available and if the predictive power falls below a pre-agreed value then the arm/study may be stopped.  Simulation methods are used to evaluate the operating characteristics of the design and a monitoring plan is created detailing the decision rule after each patient.  An example will be shared outlining how this is planned to be incorporated into a trial using discontinuation rate due to adverse events in the first 4 weeks, but the approach can equally apply to efficacy endpoints.  Using continuous monitoring within a trial may result in quicker decisions that still have robust statistical characteristics. 

    Short bio: Sharon Barton is an Associate Director, Statistics Team Leader within Oncology Biometrics at AstraZeneca in Cambridge, UK.  She joined AstraZeneca in 2017 and currently leads a team of statisticians supporting early clinical development.  Prior to joining AstraZeneca, Sharon worked at GlaxoSmithKline for 14 years within both early and late phase clinical development across a broad range of disease areas.  Prior to joining GlaxoSmithKline, Sharon worked as a statistician for the contract research organisation PPD. 


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

    To register for this event please click here.
  • Introduction to Bayesian Statistics

    Dates: 25 – 26 Jun, 2019

    Presented by: Ros Walley, Foteini Strimenopoulou & Phyllis Smetana

    At the end of the course attendees will:
    • Understand the motivation for Bayesian designs and the meaning of the Bayesian terminology including the differences between the frequentist and Bayesian methods 
    • Learn the basics of constructing a prior distribution
      Know the impact of priors on the results and how to summarise the posterior distribution
    • Understand how Bayes methods can be used to improve decision making.
    • Know some of the practical aspects (including software choices) of Bayesian design implementation.
    The course will focus on the practical implementation of the background and practicalities of Bayesian design, analysis and reporting and will not go into deep statistical methodology or software coding. Examples and practical exercises are used throughout to aid understanding and interaction. References will be to give the attendee links to the statistical formulae and other reading.

    Although not essential, it would be useful for attendees to bring a laptop with either SAS or R installed, so that they can join in with the practical workshops rather than just watch.

    The following topics will be covered:

    • Informative vs Vague Priors
    • Construction of Priors
    • Documentation of Priors
    • Posteriors
    • Credible Intervals
    • Prior-data conflict
    • Decision rules
    • Simulation and MCMC
    • Checks for convergence
    • Types of software to run Bayesian analyses
    Course runs from: 10:00 – 17:30 (registration from 9:30) on Day 1 and 
                                     09:00 – 16:45 on Day 2.
    Click here to download the flyer.


      Early Bird (Up To & Including 24th May)   After 24th May
    PSI Member  £495 + VAT £595 + VAT
    Non-Member £590 + VAT (This includes PSI membership for 2019) £690 +  VAT (This includes PSI membership for 2019)
    Click here to register.
  • PSI Webinar: Adaptive design: updated draft FDA guidance and its implications

    Dates: 04 – 04 Jul, 2019

    14.00-15.30 UK Time

    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 will be to provide an overview of its main contents, which will be presented by Jürgen Hummel (PPD). In addition, Kaspar Rufibach (Roche) will introduce 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 will also comment on the guidance from a European Regulatory perspective, which will be followed by a brief panel discussion.


    This Webinar is free to attend, to register please click here.

     Photo Jurgen Hummel (2019)
    Jürgen Hummel joined PPD near Glasgow (Scotland) in 2006, where he is Senior Director, Statistical Science. He heads up the statistical science track within PPD Biostatistics and provides statistical consultancy both internally and externally. Specifically, he has been leading PPD’s cross-departmental Adaptive Design Working Group since it was set up 8 year ago.  In his career he held a variety of technical and managerial positions within the biostatistics department of different organizations, predominantly within the CRO industry.  Jürgen earned a Diplom (German equivalent of a master’s degree) in mathematics and economics at Augsburg University, Germany, and has been a chartered statistician with the Royal Statistical Society since 2006.          

     Photo Kaspar Rufibach
    Kaspar Rufibach is a member of Roche's Methods, Collaboration, and Outreach group and located in Basel. He does methodological research, provides consulting to Roche statisticians and broader project teams, gives biostatistics trainings for statisticians and non-statisticians in- and externally, mentors students, and interacts with external partners in industry and the academic community in various working groups. He has co-founded and co-leads the Oncology estimand Working group that currently has 31 members from 19 companies and works on various topics around estimands in oncology. Research interests are methods to optimize study designs, advanced survival analysis, probability of success, estimands, estimation of treatment effects in subgroups, and general nonparametric statistics. Before joining Roche, Kaspar received training and worked as a statistician at the Universities of Bern, Stanford, and Zurich.

    Kit Roes is Professor of Clinical Trial Methodology at the Julius Center of the University Medical Center Utrecht. His research focus is on design and analysis of clinical trials, with an emphasis of innovative designs, rare diseases and bridging the gap between clinical trials and real world evidence. He participates in the Regulatory Science Network Netherlands, is chair of Methodology at the Dutch Medicine Evaluation Board and member of the Biostatistics Working Party of the EMA. He serves on multiple Data and Safety Monitoring Boards. His experience includes over 20 years in clinical research and development in the pharmaceutical industry and academic life sciences, serving clinical research and drug development as expert as well as in different (international) senior management positions.

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

    Dates: 17 – 17 Jul, 2019


    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 TBC

    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)  

    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

  • A PSI Training Course on Improving Influence and Increasing Impact: Communication Skills for Industry Statisticians

    Dates: 16 – 16 Sep, 2019

    UCB Pharma
    Allée de la Recherche 60, 1070 Anderlecht, Belgium. Brussels
    16th September 2019
    Course runs from: 10:00 – 17:00 (registration from 9:30)

    The landscape is changing across the pharma industry and as statisticians, in order to continually add value, we must make sure we adapt. This course will focus on what this means for statisticians outside the technical aspects of their role. Of critical importance is self-awareness during our interactions, working effectively in teams, influencing, being customer focussed and understanding our own consulting and leadership styles.

    Now more than ever we need to be creative and influential thinkers with business acumen who can work with our colleagues from other disciplines, not just be technical experts - we need to be proactive partners with strong communication skills

    This 1-day course will lead participants, in a non-threatening and encouraging atmosphere through steps to focus on improving the key skills of making impactful verbal and written communications.

    The first half of the session will cover topics such as poster preparation, written communication documents such as reports and briefing documents and also verbal communication in presentations. The second half of the session will focus on communication in teams and identifying key roles that need to be taken on.

    It is designed to be interactive and lively, with a focus on the statistician rather than the statistics and will include workshops to practise the skills and behaviours discussed. 

    This course is aimed at statisticians who want to improve their consultancy style interactions within their internal project teams and/or with external customers and understand the impact of their own behaviours and interaction preferences. 

    The following topics will be covered: 

    - Preparing poster layout and content
    - Skills and tips for preparing written reports and abstracts
    - Preparing and delivering presentations
    - Effective team communication and key roles
    - Delivering negative messages to teams with positive impact


    Registration costs (includes lunch and refreshments).


    Registration Fee before 16th August 2019

     Registration Fee after 16th August 2019

    PSI Member     

     £345 + VAT

     £395 + VAT


     £440 + VAT (includes PSI membership for 2019)

      £490 + VAT (includes PSI membership for 2019)

    Please click here to register. 

  • Introduction To Industry Training Course 2019

    Dates: 01 Oct, 2019

    Are you a PSI member with approx. 1-3 years' experience as a Statistician or a Statistical Programmer within the industry?


    Final dates to be confirmed

    PSI Member: £1,050 + VAT
    Non-Member: £1,145 + VAT

    AIM: To describe the drug development process from research right through to research, toxicology, data management & role of the CRO, clinical trials, product development & manufacture and marketing.



    For further information contact:

    Alex Godwood & Zelie Bailes

    Sosei Heptares 
    Steinmetz Building
    Granta Park
    CB21 6DG
    United Kingdom


       Email: Alex.Godwood@SoseiHeptares.com ; zelie.a.bailes@gsk.com