News

In 2023, under the guest editorship of Professor Victoria Cornelius and Dr Rachel Philips, Trials invited submissions that related to the collection, analysis and reporting of adverse events in clinical trials. Articles published included a range of topics from early to later phase trials, simple to complex interventions, different trial contexts, research inclusion and public involvement. They provide examples and recommendations that support much needed practical implementation advice for trialist. Collectively the articles indicate the scope of research needed for robust harm evaluation and inference, and how the whole pathway from collection to final inference needs careful attention to do this well. 

You can find the full series here: https://www.biomedcentral.com/collections/CART 

Read our editorial summarising the submissions & discussing the implications on applied clinical trials and future methodological research here:

Congratulations to Annie Wright for her presentation for which she won second prize for Best Presenter at last weeks Imperial, School of Public Health's PhD symposium. Annie's prize winning presentation was on the Global Landscape of Decentralized Clinical Trials, which included results from her Systematic Review.

On the 2nd and 3rd April 2025 ICTU and Imperial and partners NIHR RSS hub hosted their 2nd Contemporary Clinical Trials Methodology Meeting. This was a fantastic event bring together more than 130 trialists from the UK, US, Brazil and beyond!

On the statistical day 1 Dr Philip Pallmann started the first session by giving an overview of Multi-Arm-Multi-Stage (MAMS) trials and some of the statistical intricacies they involve. This was followed by Professor James Wason who presented a variety of real life MAMS trial he has worked on. Professor Max Parmar closed the MAMS session with an insightful and inspiring discussion on the advantages and future promise of MAMS trials.

The second session was on platform trials. Professor Peter Juni gave a summary of platform trials, followed by our statci’s Dr Ed Waddingham who presented the design of the PANTHER platform trial. Professor Richard Emsley wrapped up the platform session with a discussion on the challenges involved in platform trials for statisticians.

Target trial emulation and using real world data was the topic of the third session of the day. Dr Shaun Seaman gave an introduction to target trial emulation covering statistical methods for estimation. This was followed by him presenting a talk from Dr Li SU on sequential trial emulation. The final talk of the day was from Professor Stuart Pocock on present a critical appraisal of treatment-trial emulations and real-world evidence.

Full details of the talks and speakers bio’s are available in the programme link below. Including details of the talks from day 2 which covered operational and database and data management issues. Thanks to all the fantastic speakers!

Interested in patient and public involvement (PPI) to inform quantitative research?  You can now sign up to the mailing list for the new *PPI for Statistician Community of Practice*  which aims to:

-Share experiences for others to learn from

-Share resources

-Increase collaboration in this space.

This will be achieved through online seminar series, a dedicated online space to share resources, blogs and recorded seminars, and a mailing list to share relevant events/training courses. You can check out their LinkedIn space to find shared resources. 

The PPI for Statisticians CoP Organising Team are:

Jonathan Broomfield, University of Leicester,

Suzie Cro, Imperial College London

Laura Gray, University of Leicester

Beatriz Goulao, University of Aberdeen

Rachel Phillips, Imperial College London

Kara-Louise Royle, University of Leeds

James Wason, Newcastle University

We are pleased to announce our 2nd Contemporary Clinical Trials Methodology Meeting, happening on 2-3 April 2025. We will take time to explore practical aspects of clinical trial 'hot topics', diving into practical insights, including real-world examples and in-depth discussions.

🟣 Day 1: 2nd April, 10.00-4.30, South Kensington, Imperial College London

This day is primarily statistical and targeted to applied trial statisticians and statistically minded trialists.

We cover three topics: Multi-arm multi-stage trials, Platform trials and target trial emulation. Including when and how to statistically implement, examples of their use in practice and time for discussion of methodological challenges

🟣 Day 2: 3rd April, 10.00-4.00, South Kensington, Imperial college London

This day is primarily targeted to Trial Managers, Data Managers or staff interested in trial operations, data systems and data management.

Learn about: Platform trials and routine data-enabled trials Including real challenges faced for these types of trials, practical examples of operational solutions implemented, trial operations and data managers working together in complex settings.

Check out the exciting agendas and speakers in store at the event page by following the link below.

This january we welcome a new PhD student to the Statci team in ICTU. Mingxin Jiang joins us to conduct a PhD to evaluate novel multiple imputation methods for handling missing data in clinical trials, working with Dr Suzie Cro and Dr Leila Janani. Mingxin has an MSc in Epidemiology obained from Imperial College London in 2024. Welcome Mingxin!

Carrots or Mince pie..what fuels Santa's reindeer best? Find out and understand the importance of using estimands

to clearly specify trial research questions in the festive article, All I want for Christmas…is a precisely defined research question by Suzie Cro and Rachel Phillips now published in Trials. This light hearted article includes an explanation of the five components of an estimand five years on from the original publication of the final estimands framework in the ICHE9(R1). If you are still unsure about estimands this festive piece will help to unravel them for you. Read the article following the link below.

Why is it important to measure exposure to digital health interventions and how to tackle the challenge? Jack Elkes talks about his current PhD research to answer this question. Digital health interventions are transforming healthcare, offering exciting opportunities and posing new challenges for statisticians and researchers alike. From defining engagement to tackling complex data with innovative methods, this field has much to explore.

In November, the Isaac Newton Institute hosted a one-day workshop to explore evidence for effectively communicating research in mathematical and data science to non-experts such as policy makers, mainstream media and the wider public. The event aimed to explore the following three themes: What evidence is there about what kind of communication works? What can we learn from other disciplines? How can we embed communication and evaluation of its impact into research? During the event our own Rachel Phillips shared her experiences of conducting public involvement activities in clinical trials. She covered what public involvement is, why and how we do it and shared a case study on public involvment in trials methodology research. This was a great event for cross-industry learning! Check out the full programme and stream the whole programme via the link below.

Suzie Cro, Rachel Phillips and Oswald Dallimore joined the PSI Medical Statistics Careers Event on 20th November and hosted a stand to discuss and help students explore the exciting opportunities available at in medical statistics within an acamdeic trials unit. We invited expressions of interest from individuals who would like to apply for a NIHR Pre-Doctoral or Doctoral Fellowship in medical statistics or clinical trial design with us.  Do get in touch if you are interested in either of these schemes! This was a great event, where we in fact first met Oswald two years ago and which lead to his successful current NIHR pre-doctoral fellowship with us.

This autumn we welcome three new pre-doctoral fellows to Statci. Our new members include: Oswald Dallimore who is currently studying the MSc in Statistics for Clinical Trials at UCL during his first year of his fellowship, and in the second year of his fellowship will be conducting a systematic review of Bayesian clinical trial design and analysis in phase II/proof-of-concept studies, Xiao Xuan Tan who is studying the application of machine learning in RCTs, and Spyros Bissas who plans to explore the use of machine learning to optimize myopia control strategies in clinical trials, with a focus on interventions for managing and slowing myopia progression. More information on our three new fellows can be found at our team page by following the below link. Welcome to the team!

Annie Wright, a statistician and NIHR BRC PhD student, takes us behind the scenes at ICTMC 2024 in Edinburgh through two short videos. It's fantastic to see the diverse activities our team members engage in, and this is a creative, engaging way to showcase that. Stay tuned for a blog post highlighting our key moments from ICTMC!

The 7th International Clinical Trials Methodology Conference will be held in Edinburgh 30th September to 3rd October. Make sure not to miss ICTU’s statistical team who will be presenting at the conference at the following times:

TUESDAY 1st October

>13.25 - 13.45, Suzie Cro, Lightning Talk (Poster session PS.1B), Introducing retrieved dropout reference-based centred multiple imputation for estimation of treatment policy strategies with missing data

>14:45-15:05, Zhangyi He, Poster, How to improve the efficiency of group sequential designs by optimising the timing of interim analyses

>14:45-15:05, Ed Waddingham, Poster, SepTIC: A multi-factorial trial   with an information-rich outcome to efficiently evaluate three interventions in sepsis clinical care

>14:45-15:05, Emanuela Falaschetti, Poster, Importance of control group definition to address the research question. Lesson learned from Endobarrier trial

>15:10 - 16:30, Rachel Phillips, Talk, Public  perspective on potential treatment intervention harm in clinical trials –   terminology and communication

WEDNESDAY 2nd October

>8:30 AM -   9:50 AM, Victoria Cornelius, Talk, Design,   analysis and inference for follow-up trials where sample size is fixed and has inadequate power for hypothesis testing

>13.25 – 13.45, Zohra Zenasni, Poster, Methods to investigate contemporary trial practices in research involving preterm and full-term neonates, a systematic review

>13.25 – 13.45, Jack Elkes, Poster, Analysing the impact of engagement on efficacy for Digital Mental Health Interventions: An optimal strategy to identify user groups for analysis using unsupervised clustering methods

>13.25 – 13.45, Consuelo Nohpal de la Rosa, Poster, Causal inference methods to account for compliance in trials of device interventions

>13:45 - 14:05, Suzie Cro, Poster, Improving the Analysis and Interpretation of Studies Within A Trial using Bayesian Methods and ACCEPT Analyses

Earlier this year Dr Brennan Kahan from MRC CTU @ UCL, along with Dr Suzie Cro, Dr Richard Emsley (KCL) and Dr Ian White (MRC CTU @ UCL) taught a MRC-NIHR TMRP funded workshop covering the statistical methods that can be used to estimate different estimands. The taught materials from the workshop are now freely available to all online following the link below and includes: (i) slides for each lecture; (ii) computer practical’s, which include statistical code in Stata and R to implement different estimators, along with datasets and instructions for their use. These materials can be used to learn different methods that can be used to estimate each intercurrent event strategy including treatment policy, composite, hypothetical, principal stratum and while-on-treatment and the statistical code necessary to implement these estimators.

We have recently advertising our survey to statisticians, clinical investigators and trial operational staff who have been involved in clinical trials for digital interventions. We are looking to answer the question: Are efficient methodological designs being implemented in RCTs of DHIs and what are the barriers and facilitators to their use?

If you think you are elgible please take our survey using the link below. If you have any questions please email Jack Elkes (j.elkes@imperial.ac.uk).

Congratulations to Jack Elkes for winning the judges award for best poster at the Imperial Mental Health Research Network 

summer event. He presented his latest research using unsupervised cluster methods to identify groups of users from engagement data in a self-guided digital mental health intervention.

In June 2024, Laetitia de Abreu Nunes attended the Statisticians in the Pharmaceutical Industry (PSI) conference in Amsterdam, Netherlands. Laetitia presented a poster on her methodological review of statistical methods leveraging the natural structure of adverse events for signal detection in randomised controlled trials, which she had the chance to introduce during the lightning poster session. This review was conducted as part of her PhD at Queen Mary University of London. The PSI conference was a great opportunity to discuss current approaches to safety signal detection and how to move towards better practices with other researchers and industry members. Laetitia also won one of the best poster prizes for her poster.

On the 25th April Imperial College Clinical Trials Unit and the NIHR RSS Imperial & Partners Hub hosted the 1st Contemporary Clinical Trials Methodology Meeting.

The day focussed on the theme of adaptive designs. Dr Leila Janani, Professor Stuart Pocock and Professor Laurent Billot covered Group Sequential Designs and Interim Analysis with some examples in action!

Mr Lukas Pin, Dr Sofia Villar and Professor John Norrie talked about Response Adaptive Randomisation including a demonstration of an Rshiny application.

The day rounded off with Sample Size Re-estimation from Professor Alan Montgomery, Dr Dominique-Laurent Couturier and Ruth Goodall. A massive thank you to all the speakers and the organising comittee Suzie Cro (lead), Victoria Cornelius, Rachel Phillips, Rani Rayat, Matenyeh Kaba, Anneliese Levy, Sara Gazi, Gareth O’Brien, Lauren Noto.

We are all looking forward the next one!

The programme for the first ICTU & NIHR RSS Imperial and partners hubs' contemporary clinical trials meeting is now available by clicking the below link. Inside find the abstracts and biographies for the speakers who will be talking about group sequential trial designs and interim analysis, response adaptive randomisation, or sample size re-estimation. Registration for the meeting is now closed. We look forward to seeing all those who have registered to attend shortly on 25th April.

We are delighted to announce registration is now open to attend Imperial Clinical Trials Unit (ICTU) and the NIHR RSS Imperial and partners Hub's 1st Contemporary Clinical Trials Methodology Meeting, 25th April 2024.

If you are an applied trial statistician or a statistically minded trialist/clinician and want to know more about adaptive clinical trials this trial methodology meeting is for you.

Learn about Group Sequential Trial Designs, Response-Adaptive Randomisation and Sample Size Re-estimation across three main sessions. Including when and how to implement, examples of their use in practise and time for discussion of methodological challenges.

Speakers include:

Group Sequential Designs and interim analysis - Stuart Pocock, Laurent Billiot, Leila Janani (Chair: Victoria Cornelius) Response-Adaptive Randomisation: Sofia Villar, Lukas Pin, John Norrie (Chair: Suzie Cro) Sample size re-estimation: Domique-Laurent Couturier, Alan Montgomery, Ruth Goodall (Chair: Rachel Phillips)

Register to attend at the below link!

Annie Wright, formerly a clinical trials statistician at the unit, has embarked on her NIHR Biomedical Research Centre Digital Health PhD. Under the supervision of Professor Victoria Cornelius and in collaboration with Professor David Sharp of the UK DRI Care and Technology Centre at Imperial College London, she brings her expertise and enthusiasm for clinical trials and routine data to her new role.

Her research will delve into the practical implementation of decentralised clinical trials (DCT), aiming to enhance their trial design for seamless integration of routine and remote data collection. DCTs, which leverage digital technology with a patient-centric approach, depart from the traditional centralised site model by enabling trial activities to be conducted partially or entirely from patients' homes. DCTs have numerous perceived benefits and offer a unique way of thinking about running clinical trials.

Annie's project will analyse the current landscape of DCTs, optimise the integration of longitudinal data, and engage with the public to gauge their perspectives on this approach to clinical trials.

On February 26th an NIHR Public and Patient Involvement and Engagement (PPIE) for Statistical Methodology workshop, was held at the University of Leicester. Dr Suzie Cro presented on her surprises and learnings from co-chairing the HEALTHY STATS public involvement group with her public partner co-hair Ania Henley. Other researchers and public partners presented their experiences of conducting PPIE for Statistical Methodology research and Nifty Fox presented a workshop on engaging story telling. Click the below link to see all the presentation slides from the day.

Dr Suzie Cro explores public partners perspectives on the importance of discussing estimands when designing a trial in a Health Research Board Trial Methodology Research Network (HRB TMRN) webinar, 18th January. She also presents a co-developed practical tool that helps explains what an estimand is and what impact it may have in trial results. The recording is available at the following link.

Registrations are open to attend a free webinar on a guide to pre-doctoral and doctoral NIHR fellowships for aspiring and early career statisticians. Hear from Statci's Ellie Van Vogt who will be talking about their pre-doctoral experiences and Victoria Cornelius who will be giving tips about applying to the doctoral fellowship.  The webinar will be held on 23rd January, 12.45-3 and registration is at the below link.

Registrations are now open to attend a free workshop to be held December 1st 2023 (Bristol),  funded by the NIHR, to learn practical methods for conducting missing data sensitivity analyses in randomised controlled trials using Stata or R.

In this workshop, facilitated by Daisy Gaunt (UoBristol, previous NIHR Doctoral Fellow), Baptise Leurent (University College, previous NIHR Doctoral Fellow), and Suzie Cro (Imperial College London, current NIHR advanced fellowship) available accessible methods to conduct such sensitivity analysis will be de-mystified and practically demonstrated. Register following the link below.

This year, at the Australian Trials Methodology Conference, Ellie Van Vogt was invited to present their research virtually on causal machine learning for the detection of heterogeneous treatment effects.  They presented work that was part of an ongoing project with the Alan Turing Institute and the Medical Research Council Clinical Trials Unit at University College London. Ellie presented in the Trial Analysis session, chaired by Rory Wolfe. Other presenters in the session were Jonathan Bartlett (London School of Hygiene and Tropical Medicine) and Thomas Jaki (Cambridge University). Ellie’s presentation highlighted the benefits of incorporating a machine learning approach into the subgroup analysis of trials, to identify previously not-considered subgroups and investigate high-level interactions between baseline covariates. Recordings of the presentation are available to attendees and a publication of this work will be available soon.

In September, Dr Rachel Phillips presented at the 6th Conference of the Central European Network (CEN), Basel, Switzerland. This was a joint conference of: the Austro-Swiss Region (ROeS), the German Region (DR), and the Polish Region of the International Biometric Society (IBS) and supported by the Basel Biometric society (BBS). Rachel was invited to join the session on best practices for Data Monitoring Committees and how to get there. In this session she presented her collaborative work on recommendations for visualising harm outcomes that aim to improve reporting practice in journal articles and extended these ideas to applications for Data Monitoring Committee (DMC) reports.

In August this year, Ellie Van Vogt attended the International Society for Biostatistics Conference (ISCB) in Milan, Italy. Ellie presented their poster from work with the Alan Turing institute and the MRC clinical trials unit on the use of causal machine learning for the detection of heterogeneous treatment effects in clinical trials. This conference was a wonderful opportunity to network with other researchers across different medical disciplines and discuss the implementation of causal machine learning methods into clinical trials. Follow the link below to view their poster!

Congratulations to Bao Khanh Nguyen who has been awarded a pre-doctoral fellowship by the NIHR. Bao has an MSc in Mathematics and will be joining us at ICTU to gain experience in medical statistics and clinical trials. She will be undertaking research to evaluate novel multiple imputation methods for handling missing data in clinical trials, working with Dr Suzie Cro and Dr Matteo Quartagno (MRC CTU at UCL).  

NIHR Pre-doctoral Fellowships are designed to support people who are looking to start or advance their career within a health and social care research methodology and we welcome you to get in touch with us if this is an opportuntiy you'd be interested in exploring with us. Follow the link below to find out more about the NIHR pre-doctoral fellowship scheme.

The HEALTHY STATS public involvement group provides advice on an NIHR funded project to develop accessible statistical methods to determine treatments effects that matter to patients and prescribing physicians in randomised controlled trials (Suzie Cro’s Advanced fellowship NIHR300593). The group have developed a practical tool for public partners involved in trial design that describes what an estimand is and what impact it may have in trial results interpretation. The aim of the tool is to break down the barriers of scientific jargon and explain what an estimand is to public partners, to facilitate involvement of public partners in estimand discussions. The tool and more information about this work is now published in Trials, which can be accessed via the below link.

At this years PSI conference Dr Suzie Cro gave a presentation of her work on ‘Reference-based Multiple Imputation for longitudinal binary outcomes’. This included presentation of a simulation study exploring two algorithms for implementing reference-based Multiple Imputationin this setting: (i) joint modelling with the multivariate normal distribution and an adaptive rounding algorithm and (ii) joint modelling with a latent multivariate normal model. A recording of the talk will soon be available via PSI on demand for PSI members.

The CONSORT group includes many initiatives to improve reporting of results from randomised controlled trials (RCTs). The latest output from the group demonstrate how items related to harm outcomes could be integrated into the main CONSORT checklist. Thirteen items from the main CONSORT were modified to improve harms reporting and three new items were added. The CONSORT group recommend until future work produces an updated checklist, trial authors, journal reviewers, and editors should use the integrated CONSORT Harms 2022 checklist presented in this paper. This publication is the culmination of a large international research project that began in 2017 and which our own Dr Rachel Phillips became involved in 2019. You can read more about this in this 2019 blog post.  Alongside this latest CONSORT update, Rachel and Professor Victoria Cornelius have published an opinion piece on the future direction of research into harms in RCTs. They call for clear advice on best analysis practice for harm outcomes that is endorsed by key stakeholders and can be easily implemented. Arguing that better reporting encouraged by the new CONSORT harm extension along with guidance for analysis would enable us to better assess the true benefit-risk of interventions. You can read more on their thoughts about the future direction of harms reseach here and how their ongoing DoRAH project hopes to address this.

On June 22nd we will be at the NIHR statistics conference. This is a fantastic event including talks and presentations focused on, Achieving Best Statistical Practice in Health and Care Research. Along with Dr Pavel Mozgunov (MRC Biostatistics unit), StatsCI's Dr Rachel Philips, Dr Suzie Cro and Ms Ellie Van Vogt will be holding a workshop on “Lessons learned from implementing an adaptive design in an early phase dose-finding clinical trial“ . Additionally Mr Jack Elkes has a poster on the results of his systematic review of reporting and analysis of user engagement in trials of digital mental health interventions.  For more details including other sessions and how to register for the event follow the below link.

We had another fantastic research away day with a focus on Adaptive Designs on the 27th April organised by Suzie Cro and Leila Janani. There was a great variety of talks from both internal and external speakers in the morning. Professor Yin’s talk about ‘Control of Type 1 Error Rates in Bayesian Sequential Designs’ sparked a lot of interest in the group. We also enjoyed Professor Billot’s talk on ‘Group Sequential Designs with Unblinded Sample Size Reassessment’. In the afternoon we used these talks as well as thinking about our own problems we face in designing trials in an idea generating exercise to identify issues and research areas we wanted to focus on both personally and as a unit. This exercise was a great success with Zhangyi He conducting research on optimising the timing of interim analyses off the back of discussions; we all look forward to seeing his results!

We are very pleased to announce that Mrs Emanuela Falaschetti is our new Deputy Head of Statistics, and Dr Leila Janani is our new Deputy Head of Trial Methodology.

They will closely work with Dr Rachel Phillips and Dr Suzie Cro to improve the design, analysis and delivering trials at ICTU and to build on trial methodology work.

Congratulations to you both!

We are delighted to welcome back Dr Rachel Phillips to the ICTU statistics team. Dr Rachel Phillips and Dr Suzie Cro now head up the statistics team for ICTU. They bring with them extensive experience in clinical trials, CTUs, and a passion to improve the way we design and deliver trials through research.

Our previous pre-doctoral fellow, Ellie Van Vogt, has successfully been awarded a 3-year doctoral fellowship by the NIHR to use causal machine learning to improve the detection of heterogeneous treatment effects in complex RCT settings.

They will be undertaking research to develop causal machine learning techniques for the estimation of heterogeneous treatment effects in complex settings with competing risks, with a particular focus on critical care settings. Ellie aims to develop sound guidance on the use of causal machine learning methods for determining subgroups of heterogeneous treatment effects, how to communicate results effectively and investigate methods for the incorporation of subgroups into ongoing trials.

On 7th December PSI held their annual medical statistics careers event which we attended. The event included a live presentation from our Ellie Van Vogt on her experiences as a pre-doctoral NIHR research fellow in medical statistics with us. Ellie summarised how her fellowship enabled her to conduct an MSc in health data analytics and machine learning at Imperial, followed by a year conducting a research project exploring causal machine learning methods to detect differences in treatment effects by patient characteristics in trials (known as treatment heterogeneity). The event also included a panel discussion with statisticians from academia and industry, followed by an exhibition by various companies (including us) giving individuals the opportunity to find out more about job opportunities and life as a medical statistician. We invited expressions of interest from individuals who would like to apply for a NIHR Pre-Doctoral or Doctoral Fellowship in medical statistics or clinical trial design with us.  Get in touch with us if you are interested in either of these schemes!

Ellie Van Vogt and Jack Elkes represented ICTU at the School of Public Health showcase of research activities to the Imperial College President - Hugh Brady. Both Jack and Ellie spoke with the president about their research, talking him through each of their posters. The event was a great opportunity to share more of our methods research and a chance to meet the president in person for the first time.

StatsCI had our second research away day of the year on the South Kensington campus of Imperial College at the end of October 2022. We started the day off with some games and looked back at the past few months to celebrate what the team has achieved. Ellie, Suzie and Victoria gave us some insight into their research networks and collaborations that they have made and nurtured through their careers as statisticians.

Our theme for this research away day was ‘Adaptive Trial Designs’. We were lucky enough to have Dr Munya Dimairo from the University of Sheffield with us for the day. Munya is the lead on a collaborative initiative that has set up PANDA; a platform that brings together a wealth of information and resources about adaptive designs and presents them in an accessible manner to anyone involved in the design and implementation of trials using adaptive designs. We were very excited to hear him talk about the platform before exploring it ourselves and then utilising PANDA to answer a mock trial proposal based on real world examples of requests from investigators. This was such a valuable experience, and we are grateful for Munya travelling to us to help deliver such a great away day. We all highly recommend using the PANDA platform to learn more about adaptive designs.

We finished off the day with a scavenger hunt in the Science Museum and some drinks together as a team. Follow the link to our twitter page where you can find a video round up of the day