Design of Trials, Meta-analyses and observational studies

Lead Research Organisation: University College London

Abstract

The CTU-pioneered multi-arm, multi-stage trial design (MAMS), allows efficient evaluation of multiple treatments and the flexibility to drop inactive treatment arms and incorporate new ones. This has been demonstrated in our ongoing STAMPEDE trial in prostate cancer, which added a new arms since 2012. We also began to design a new CTU MAMS trial of novel tuberculosis regimens, and worked with other groups to develop potential MAMS trials in osteosarcoma, hepatobiliary cancer, melanoma, dermatology and multiple sclerosis. We also ran a workshop on MAMS methodology, leading to further requests for help to develop MAMS trials in other diseases. There are many new agents demanding assessment, and a need to evaluate biomarkers that effectively identify more responsive patient subgroups. Our FOCUS4 trial (funded 2012) in colorectal cancer represents a new approach to trial design, linking the evaluation of five novel treatments to the assessment of potentially predictive biomarkers in a multi-staged framework. It offers the flexibility to refine and introduce new biomarkers and/or new therapies, and introduces a new paradigm in oncology. Already, others are considering similar trials in pancreatic and upper gastrointestinal cancer.

Technical Summary

There is an urgent need for new methods for many clinical studies. In particular, we need more rapid, efficient and better evaluation of therapies. This is partly because of the increase in numbers of new therapeutic approaches demanding evaluation, and the staggering failure rate to show that the ‘new’ is better than the ‘old’. Also, phase III trials often require large numbers of patients, take a long time, and cost many millions of pounds. One of the principal ways to improve this situation is through better trial designs.

Motivated by, and directly relevant to, the real challenges of clinical research, and very commonly those faced directly in studies conducted by the MRC Clinical Trial Unit (CTU) we focus on multi-arm, multi-stage platform trials; designing phase II (and III trials) based on an enhanced decision process at the end of phase II; improving the design of stratified medicine trials and biomarker validation studies; designing trials in uncommon diseases and cluster randomised and stepped wedge trials. Also, we want to establish a flexible framework for time-to-event designs, be better able to account for non-proportional hazards, missing data, repeated measures and recurrent events in trial design and explore the possibility of re-randomising participants into trials. In addition, we are investigating sample size estimation for developing and validating prognostic models.

Publications

10 25 50

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Blenkinsop A (2019) Multiarm, multistage randomized controlled trials with stopping boundaries for efficacy and lack of benefit: An update to nstage in The Stata Journal: Promoting communications on statistics and Stata

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Choodari-Oskooei B (2016) Quantifying the uptake of user-written commands over time. in The Stata journal

 
Description MRC HTMR Network award- Adaptive Design Outreach officer
Amount £136,000 (GBP)
Organisation Medical Research Council (MRC) 
Sector Public
Country United Kingdom
Start 03/2014 
End 02/2016
 
Description Adaptive Designs Working Group 
Organisation Medical Research Council (MRC)
Department Network of Hubs for Trials Methodology Research (HTMR)
Country United Kingdom 
Sector Academic/University 
PI Contribution The HTMR Network supported the Adaptive Designs Working Group (WG) to regularly meet and exchange ideas. This involves members of the methodology community and Hub members.
Collaborator Contribution Scope: The Adaptive Designs Working Group collaborates to increase uptake of methods, to improve knowledge and to link with key stakeholders such as regulators and industry in this important area for improving the speed and efficiency of trials. Future objectives: The Network plays a vital role in increasing the implementation of adaptive design methodology, with the main barriers to implementation already identified as a lack of software and a lack of expertise. The future plans for this group include continued annual meetings, strengthening the engagement with industry and the development of collaborative inter-Hub visits to develop novel adaptive designs. The group is focusing its efforts on preparing tutorial papers for applied journals and mainstream medical journals; presentations and lectures to increase uptake of methods among stakeholders; and the development of computer software to help researchers to undertake trials with adaptive designs.
Impact Adaptive designs meet regularly and host an annual meeting on their research. There is an adaptive design outreach officer - paid for by the Network- who is undertaking regular visits to CTUs and is working on new research methodologies. To date (2016 Feb) 7 CTUs have been visited, and they have collaborated with one group on developing a trial application
Start Year 2010
 
Description Alan Turing Institute and AI 
Organisation Alan Turing Institute
Country United Kingdom 
Sector Academic/University 
PI Contribution Developed project idea which will be taken into Alan Turing Institute Hackathon week
Collaborator Contribution ATI will provide funding for project work for the week
Impact (none yet)
Start Year 2019
 
Description Alan Turing Institute and AI 
Organisation Health Data Research UK
Country United Kingdom 
Sector Private 
PI Contribution Developed project idea which will be taken into Alan Turing Institute Hackathon week
Collaborator Contribution ATI will provide funding for project work for the week
Impact (none yet)
Start Year 2019
 
Description B1- Big Idea- Adaptive Design Outreach officer 
Organisation Lancaster University
Country United Kingdom 
Sector Academic/University 
PI Contribution Network funding provided for this post
Collaborator Contribution This proposal is for an "Adaptive Design Outreach Officer" who would be associated with the Adaptive Designs Working Group (ADWG). The key aim would be to raise awareness and use of adaptive designs across a broad range of diseases and across the development and assessment spectrum. Adaptive methods in this context are defined in its broadest sense end will include Bayesian adaptive dose-finding and group-sequential methods, multi-arm trials, lack-of-benefit stopping rules and sample size reassessment methods. The main activity of the outreach officer would be to proactively engage with applied health researchers to discuss the merits and relative drawbacks of the currently available adaptive designs. In particular, the outreach officer would: 1. Develop training material on current adaptive designs 2. Visit each of the UKCRC registered Clinical Trials Units1 to present about the potential of adaptive designs 3. Facilitate the uptake and implementation of adaptive designs 4. Support the writing of tutorial papers on adaptive designs
Impact ongoing
Start Year 2014
 
Description B1- Big Idea- Adaptive Design Outreach officer 
Organisation Medical Research Council (MRC)
Department MRC Clinical Trials Unit
Country United Kingdom 
Sector Academic/University 
PI Contribution Network funding provided for this post
Collaborator Contribution This proposal is for an "Adaptive Design Outreach Officer" who would be associated with the Adaptive Designs Working Group (ADWG). The key aim would be to raise awareness and use of adaptive designs across a broad range of diseases and across the development and assessment spectrum. Adaptive methods in this context are defined in its broadest sense end will include Bayesian adaptive dose-finding and group-sequential methods, multi-arm trials, lack-of-benefit stopping rules and sample size reassessment methods. The main activity of the outreach officer would be to proactively engage with applied health researchers to discuss the merits and relative drawbacks of the currently available adaptive designs. In particular, the outreach officer would: 1. Develop training material on current adaptive designs 2. Visit each of the UKCRC registered Clinical Trials Units1 to present about the potential of adaptive designs 3. Facilitate the uptake and implementation of adaptive designs 4. Support the writing of tutorial papers on adaptive designs
Impact ongoing
Start Year 2014
 
Description B1- Big Idea- Adaptive Design Outreach officer 
Organisation University of Cambridge
Department MRC Biostatistics Unit
Country United Kingdom 
Sector Academic/University 
PI Contribution Network funding provided for this post
Collaborator Contribution This proposal is for an "Adaptive Design Outreach Officer" who would be associated with the Adaptive Designs Working Group (ADWG). The key aim would be to raise awareness and use of adaptive designs across a broad range of diseases and across the development and assessment spectrum. Adaptive methods in this context are defined in its broadest sense end will include Bayesian adaptive dose-finding and group-sequential methods, multi-arm trials, lack-of-benefit stopping rules and sample size reassessment methods. The main activity of the outreach officer would be to proactively engage with applied health researchers to discuss the merits and relative drawbacks of the currently available adaptive designs. In particular, the outreach officer would: 1. Develop training material on current adaptive designs 2. Visit each of the UKCRC registered Clinical Trials Units1 to present about the potential of adaptive designs 3. Facilitate the uptake and implementation of adaptive designs 4. Support the writing of tutorial papers on adaptive designs
Impact ongoing
Start Year 2014
 
Description Clinical trials in small populations: methodological challenges and solutions (N68) 
Organisation Lancaster University
Country United Kingdom 
Sector Academic/University 
PI Contribution Co-applicants and part of the organising committee for a national 2-day workshop
Collaborator Contribution Co-applicants and part of the organising committee for a national 2-day workshop
Impact A two-day meeting bringing together methodologists, applied statisticians, patient representatives and regulators to disseminate state-of-the-art methods and set priorities for methodological research in trials in small populations
Start Year 2015
 
Description Clinical trials in small populations: methodological challenges and solutions (N68) 
Organisation University of Cambridge
Department MRC Biostatistics Unit
Country United Kingdom 
Sector Academic/University 
PI Contribution Co-applicants and part of the organising committee for a national 2-day workshop
Collaborator Contribution Co-applicants and part of the organising committee for a national 2-day workshop
Impact A two-day meeting bringing together methodologists, applied statisticians, patient representatives and regulators to disseminate state-of-the-art methods and set priorities for methodological research in trials in small populations
Start Year 2015
 
Description Clinical trials in small populations: methodological challenges and solutions (N68) 
Organisation University of Liverpool
Country United Kingdom 
Sector Academic/University 
PI Contribution Co-applicants and part of the organising committee for a national 2-day workshop
Collaborator Contribution Co-applicants and part of the organising committee for a national 2-day workshop
Impact A two-day meeting bringing together methodologists, applied statisticians, patient representatives and regulators to disseminate state-of-the-art methods and set priorities for methodological research in trials in small populations
Start Year 2015
 
Description Clinical trials in small populations: methodological challenges and solutions (N68) 
Organisation University of Warwick
Country United Kingdom 
Sector Academic/University 
PI Contribution Co-applicants and part of the organising committee for a national 2-day workshop
Collaborator Contribution Co-applicants and part of the organising committee for a national 2-day workshop
Impact A two-day meeting bringing together methodologists, applied statisticians, patient representatives and regulators to disseminate state-of-the-art methods and set priorities for methodological research in trials in small populations
Start Year 2015
 
Description Clustering and covariates in the design and analysis of RCTs 
Organisation Queen Mary University of London
Country United Kingdom 
Sector Academic/University 
PI Contribution Initiator of collaboration; provide expertise and intellectual input
Collaborator Contribution Leadership of the project
Impact A number of publications have resulted from this collaboration, for example PMID: 23825027, 24749914, 24755011, 24456267. More are forthcoming.
Start Year 2010
 
Description Collaboration with Cytel, manufacturers of East software 
Organisation Cytel
Country United Kingdom 
Sector Private 
PI Contribution We have formed a collaboration with Cytel, manufacturers of East software for designing adaptive trials, with the aim of getting our novel MAMS methods implemented in East and conversely using East to validate our software. Based on our experience with Stampede and other MAMS trials, we provide advice on how to develop a suitable user interface in East, as well as implementing our methods both analytically and via simulation.
Collaborator Contribution Based on our input and advice, Cytel will develop prototypes for us to try them out and gives us feed-back so that we know what does or does not work.
Impact This is multi-disciplinary collaboration which includes both the development of statistical methodology as well as implementing those in East.
Start Year 2019
 
Description Collaboration with WHO on complex adaptive trial designs 
Organisation World Health Organization (WHO)
Country Global 
Sector Public 
PI Contribution We designed mlti-arm multi-stage (MAMS) selection trial in postpartum haemorrhage. It is planned to be conducted in middlle and low income countries in Africa and South East Asia.
Collaborator Contribution WHO will conduct the trial with the technical/statistical support of the MRC CTU.
Impact Conference presentation, and a (working) design article.
Start Year 2020
 
Description Collaborative research with MRC BSU 
Organisation University of Cambridge
Department MRC Biostatistics Unit
Country United Kingdom 
Sector Academic/University 
PI Contribution Joint collaborative research in design and analysis of clinical trials
Collaborator Contribution Joint collaborative research in design and analysis of clinical trials
Impact Several papers including PUBMED id: 19153970; 19452569; 21225900
 
Description Jane Walker - University of Oxford 
Organisation University of Oxford
Department Blavatnik School of Government
Country United Kingdom 
Sector Academic/University 
PI Contribution Contribute to the design, conduct and analysis of a pragmatic multicentre randomised controlled trial to compare the effectiveness and cost-effectiveness of Proactive Liaison Psychiatry with usual care.
Collaborator Contribution Lead on the design, conduct and analysis of a pragmatic multicentre randomised controlled trial to compare the effectiveness and cost-effectiveness of Proactive Liaison Psychiatry with usual care.
Impact None as yet
Start Year 2016
 
Description MAMS design for erosive lichen planus 
Organisation University of Nottingham
Department Centre of Evidence Based Dermatology
Country United Kingdom 
Sector Academic/University 
PI Contribution Group keen to develop a methodologically efficient design to assess new treatments for women with vulval erosive lichen planus, a rare but debilitating condition. We have worked to help develop study designs, including updates to the multi-arm multi-stage approach, which will be covered in a PhD (Dan B). Eventually calculated that a multi-stage approach could not be used with the particular outcome measures required for the condition. Funding was successfully obtained for the trial which will be run from Nottingham CTU. MRC CTU's Scientific Strategy Group chose not to proceed with this. The unit has developed the first draft of the Statistical Analysis Plan and will engage in an advisory capacity. One member of the unit will serve on the IDMC.
Collaborator Contribution Treatment of patients, design and discussion and leadership of research.
Impact Grant applications submitted to NIHR and RfPB. Lead researcher has successfully won an NIHR fellowship during this time to contribute further to the research.
Start Year 2012
 
Description Making decisions at the end of a Phase 2 clinical trial 
Organisation Amgen Inc
Country United States 
Sector Private 
PI Contribution Methodological development of an approach to the decision as to whether to proceed to a phase three trial at the end of phase two that synthesizes the available data. An exploration as to how this can also be used to design trials at phase two and three.
Collaborator Contribution Methodological development and simulation work as part of a PhD research project
Impact 2 research papers published and a further paper has been submitted; has had implications for how trial design decisions are made within Amgen
Start Year 2012
 
Description Methodology Research Collaboration with industry 
Organisation AstraZeneca
Country United Kingdom 
Sector Private 
PI Contribution Commitment to developing research activity in to design, conduct or analysis methodology in areas of mutual interest. Detailed discussions ongoing
Collaborator Contribution Commitment to developing research activity in to design, conduct or analysis methodology in areas of mutual interest. Detailed discussions ongoing
Impact None yet
Start Year 2014
 
Description NIHR & MRC Trials Methodology Research Parternship Executive Group 
Organisation Association of the British Pharmaceutical Industry
Country United Kingdom 
Sector Charity/Non Profit 
PI Contribution Intellectual input and plans for further collaboration on future projects Member of Executive Group (Coordinated from University of Liverpool) Co-chair of Health Informatics Working Group (Co-chaired from University of Leeds) Co-chair of Statistical Analysis Working Group (Co-chaired from Kings College London)
Collaborator Contribution 25 partner organisations around UK (not all listed at this stage) Intellectual input and plans for further collaboration on future projects
Impact (None yet)
Start Year 2019
 
Description NIHR & MRC Trials Methodology Research Parternship Executive Group 
Organisation King's College London
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual input and plans for further collaboration on future projects Member of Executive Group (Coordinated from University of Liverpool) Co-chair of Health Informatics Working Group (Co-chaired from University of Leeds) Co-chair of Statistical Analysis Working Group (Co-chaired from Kings College London)
Collaborator Contribution 25 partner organisations around UK (not all listed at this stage) Intellectual input and plans for further collaboration on future projects
Impact (None yet)
Start Year 2019
 
Description NIHR & MRC Trials Methodology Research Parternship Executive Group 
Organisation Medical Research Council (MRC)
Country United Kingdom 
Sector Public 
PI Contribution Intellectual input and plans for further collaboration on future projects Member of Executive Group (Coordinated from University of Liverpool) Co-chair of Health Informatics Working Group (Co-chaired from University of Leeds) Co-chair of Statistical Analysis Working Group (Co-chaired from Kings College London)
Collaborator Contribution 25 partner organisations around UK (not all listed at this stage) Intellectual input and plans for further collaboration on future projects
Impact (None yet)
Start Year 2019
 
Description NIHR & MRC Trials Methodology Research Parternship Executive Group 
Organisation National Institute for Health Research
Country United Kingdom 
Sector Public 
PI Contribution Intellectual input and plans for further collaboration on future projects Member of Executive Group (Coordinated from University of Liverpool) Co-chair of Health Informatics Working Group (Co-chaired from University of Leeds) Co-chair of Statistical Analysis Working Group (Co-chaired from Kings College London)
Collaborator Contribution 25 partner organisations around UK (not all listed at this stage) Intellectual input and plans for further collaboration on future projects
Impact (None yet)
Start Year 2019
 
Description NIHR & MRC Trials Methodology Research Parternship Executive Group 
Organisation University of Leeds
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual input and plans for further collaboration on future projects Member of Executive Group (Coordinated from University of Liverpool) Co-chair of Health Informatics Working Group (Co-chaired from University of Leeds) Co-chair of Statistical Analysis Working Group (Co-chaired from Kings College London)
Collaborator Contribution 25 partner organisations around UK (not all listed at this stage) Intellectual input and plans for further collaboration on future projects
Impact (None yet)
Start Year 2019
 
Description NIHR & MRC Trials Methodology Research Parternship Executive Group 
Organisation University of Liverpool
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual input and plans for further collaboration on future projects Member of Executive Group (Coordinated from University of Liverpool) Co-chair of Health Informatics Working Group (Co-chaired from University of Leeds) Co-chair of Statistical Analysis Working Group (Co-chaired from Kings College London)
Collaborator Contribution 25 partner organisations around UK (not all listed at this stage) Intellectual input and plans for further collaboration on future projects
Impact (None yet)
Start Year 2019
 
Description NIHR & MRC Trials Methodology Research Parternship Executive Group 
Organisation University of Oxford
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual input and plans for further collaboration on future projects Member of Executive Group (Coordinated from University of Liverpool) Co-chair of Health Informatics Working Group (Co-chaired from University of Leeds) Co-chair of Statistical Analysis Working Group (Co-chaired from Kings College London)
Collaborator Contribution 25 partner organisations around UK (not all listed at this stage) Intellectual input and plans for further collaboration on future projects
Impact (None yet)
Start Year 2019
 
Description Novel clinical trial design in a surgical setting (ROSSINI II) 
Organisation Birmingham Women's and Children's NHS Foundation Trust
Country United Kingdom 
Sector Public 
PI Contribution Key role in the trial design and sample size calculation. Developed a proposal for the design of the trial, using novel multi-arm multi-stage design, with binary outcome (total: eight arms).
Collaborator Contribution Responsible for submitting the funding application and running the clinical trial
Impact None yet.
Start Year 2014
 
Description Re-randomisation of patients to a clinical trial 
Organisation Monash University
Country Australia 
Sector Academic/University 
PI Contribution Intellectual input to original design and resulting paper; currently working on follow up projects to demonstrate strengths and weaknesses and to understand practicalities of the design
Collaborator Contribution Intellectual input to original design and resulting paper; currently working on follow up projects to demonstrate strengths and weaknesses and to understand practicalities of the design.
Impact Several invited seminars (Leeds, Leicester, LSHTM), conference papers, one published paper.
Start Year 2014
 
Description Re-randomisation of patients to a clinical trial 
Organisation Queen Mary University of London
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual input to original design and resulting paper; currently working on follow up projects to demonstrate strengths and weaknesses and to understand practicalities of the design
Collaborator Contribution Intellectual input to original design and resulting paper; currently working on follow up projects to demonstrate strengths and weaknesses and to understand practicalities of the design.
Impact Several invited seminars (Leeds, Leicester, LSHTM), conference papers, one published paper.
Start Year 2014
 
Description Sample size calculations for cluster randomised trials 
Organisation Queen Mary University of London
Country United Kingdom 
Sector Academic/University 
PI Contribution Review of how sample size calculations are made and reported in cluster randomised trials, critical review of the published sample formulae with practical guidance. Further work will include development of guidance for calculating sample size for trials with an ordinal outcome
Collaborator Contribution Joint supervision of PhD student
Impact Two research papers have been published and a presentation has been made at a conference. One further article is in development
Start Year 2011
 
Description Stepped wedge trial design 
Organisation London School of Hygiene and Tropical Medicine (LSHTM)
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual contribution to all activities, lead authorship on one of 6 publications
Collaborator Contribution Intellectual contributions
Impact Special issue of Trials (6 articles), workshop to present the findings. Further collaboration with other parts of UCL as well as with LSHTM. Work is already being cited.
Start Year 2014
 
Description Stratified Medicine WG 
Organisation Medical Research Council (MRC)
Department Network of Hubs for Trials Methodology Research (HTMR)
Country United Kingdom 
Sector Academic/University 
PI Contribution The HTMR Network supports the Stratified Medicine Working Group (WG) to regularly meet and exchange ideas. This involves members of the methodology community and Hub members.
Collaborator Contribution Scope- Stratified Medicine is about tailoring treatments to specific patients, helping to ensure the highest chance of benefit and minimising the potential for harm or unnecessary treatment The Stratified Medicine Working Group collaborates to look at novel designs for biomarker-stratified trials, and to bring the stratified medicine approach into a wider range of disease areas, complementing work already done in the widely studied examples of cancer and heart disease. Future Objectives- The Working Group is preparing a guidance paper to help people reading a stratified medicine research paper, and has started to explore means for delivering advice to applicants to the MRC Research Panel on Stratified Medicine. Members of the Working Group are also planning to develop prognostic models that help in the prediction of harms and benefits following the use of thrombolysis to treat stroke, drawing on individual participant data meta-analysis of clinical trials.
Impact ongoing collaborations.
Start Year 2012
 
Title A combined test for a generalized treatment effect in clinical trials with a time-to-event outcome 
Description Performs a combined test for a generalized treatment effect in clinical trials with a time-to-event outcome 
Type Of Technology Software 
Year Produced 2017 
Impact None yet - just released 
 
Title R package for the design of Non-inferiority and durations trials 
Description Gives utility functions to help the design and analysis of non-inferiority trials. These include functions for sample size calculations on various scales and for testing for non-inferiority. 
Type Of Technology Webtool/Application 
Year Produced 2020 
Open Source License? Yes  
Impact None yet - just released 
 
Title nstage 
Description nstage is a Stata add-on to aid the design of a multi-arm multi-stage (MAMS) trial by recommending the duration of each trial stage and decision procedures at the end of each stage. It was updated in 2016 to calculate the familywise type 1 error rate and in 2019 to allow early stopping for efficacy. 
Type Of Technology Software 
Year Produced 2016 
Impact Used in the design of a number of MAMS trials including STAMPEDE, FOCUS4, and RAMPART. 
URL https://ideas.repec.org/c/boc/bocode/s457931.html
 
Description An Interview with Professor Andrew Nunn 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Andrew Nunn joined the MRC's Tuberculosis & Chest Diseases Unit as a statistician in 1966. During the next 20 years he was directly involved in the design, conduct and analysis of the programme of trials conducted under the leadership of Professors Wallace Fox and Denny Mitchison in East Africa, Hong Kong and Singapore which led to the worldwide adoption of short course chemotherapy for tuberculosis.

Following the closure of that unit he joined the MRC's Uganda AIDS Programme which researched the dynamics of the HIV epidemic in a rural African environment. On his return to the UK he became head of the Division Without Portfolio within the newly formed MRC Clinical Trials Unit with responsibility for developing trials in neglected areas.

Andrew is Co-Chief Investigator of STREAM, the first randomised controlled-trial of a treatment regimen for multi-drug resistant TB. In the context of treatment guidelines based on 'very low quality' evidence, Andrew designed STREAM to generate gold-standard evidence on the safety and efficacy of the promising 9-month short-course regimen previously evaluated only in observational studies. The trial has subsequently been adapted to test a bedaquiline-containing oral regimen.
Year(s) Of Engagement Activity 2018
URL https://soundcloud.com/user-110325996-105034477/andrew-nunn-talks-medical-statistics-tb-and-algerian...
 
Description An Interview with Professor Jayne Tierney. 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact Jayne Tierney has been a Research Scientist at the Unit since it was formed in 1999, and prior to that, in the Cancer Trials Office in Cambridge. For more than 20 years, she has been responsible for designing and conducting systematic reviews and meta-analyses to rigorously re-evaluate the effectiveness of therapies; projects that have influenced practice guidelines and the treatment of patients worldwide.

Today we discuss Jayne's career in depth, improving systematic reviews, and how to stand out as an individual when the work you do is mostly collaborative.
Year(s) Of Engagement Activity 2018
URL https://soundcloud.com/user-110325996-105034477/jayne-tierney-talks-systematic-reviews-impact-and-fi...
 
Description An interview with Patrick Royston for the first podcast in a series 'MRCCTU: The Ideas Behind the Trials' 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact Professor Patrick Royston was awarded the Stata Journal Editors' Prize 2016, for his significant contributions to Stata and the Stata community over the last 25 years, and for three papers he published in the Stata Journal during the previous two years. Today, Patrick will officially receive his award at 2017 London Stata Users Group meeting in London.

To commemorate this occasion, we have interviewed Patrick for the first podcast in our new series 'MRCCTU: The Ideas Behind the Trials'. The interview explores Patrick's career in more depth, and provides an insight into his work with Stata, including fractional polynomials, and survival analysis "beyond the Cox model". There is also some excellent advice for younger statisticians just starting out in the field. Click the link below to listen to the podcast.
Year(s) Of Engagement Activity 2017
URL https://soundcloud.com/user-110325996-105034477/patrick-royston-receives-the-stata-journal-editors-p...
 
Description Marie Curie workshop 
Form Of Engagement Activity A formal working group, expert panel or dialogue
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact 40 people from a range of backgrounds attended a workshop "Missing Data in Palliative Care Studies" at which Ian White spoke. This led to a draft document for use by the Marie Curie charity and others.
Year(s) Of Engagement Activity 2017
 
Description Member of trial steering committee, POSNOC trial 
Form Of Engagement Activity A formal working group, expert panel or dialogue
Part Of Official Scheme? Yes
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Appointed member of trial steering committee, POSNOC trial (2014-2024): POSNOC - A randomised trial of armpit (axilla) treatment for women with early stage breast cancer.

The POSNOC trial will provide evidence relevant to patients and to the NHS. The protocol has been designed to integrate into current NHS
practice.
The hypothesis of the POSNOC trial is that low axillary tumour burden patients (clinically and ultrasound negative) with macrometastases in 1 or 2 SNs, receiving systemic therapy, would have non-inferior outcomes whether they are randomised to adjuvant therapy alone or adjuvant therapy plusaxillary treatment (ANC or ART)
Year(s) Of Engagement Activity 2014,2015,2016,2017,2018,2019,2020,2021
URL http://www.nets.nihr.ac.uk/__data/assets/pdf_file/0004/111469/PRO-12-35-17.pdf