Adaptive clinical trials
Lead Research Organisation:
MRC Biostatistics Unit
Abstract
Explain, in simple lay language, the outcomes and significance of the research. You should include what the research is trying to achieve and why this is important, and brief notes on the context of the research and the methods (see Guidance Notes).
Classically designing a clinical trial involves making assumptions about various attributes, such as the treatment effect, before any data are collected. The aim of our programme is to introduce novel designs that are more flexible. The rigidity of the classic design can lead to failed studies due to wrong assumptions such as using an optimistic treatment effect to limit the size of the study. Our novel designs will allow the use of accrued information in many ways; this includes stopping the study early for futility or efficacy, re-assessing the size of the study and recruiting more patients if need be altering dose choices during the study and identifying subgroups of patients who respond the best to treatment. Our overall goal is to minimise the number of people needed within a study whilst maintaining the statistical rigour needed to correctly answer the scientific questions.
Classically designing a clinical trial involves making assumptions about various attributes, such as the treatment effect, before any data are collected. The aim of our programme is to introduce novel designs that are more flexible. The rigidity of the classic design can lead to failed studies due to wrong assumptions such as using an optimistic treatment effect to limit the size of the study. Our novel designs will allow the use of accrued information in many ways; this includes stopping the study early for futility or efficacy, re-assessing the size of the study and recruiting more patients if need be altering dose choices during the study and identifying subgroups of patients who respond the best to treatment. Our overall goal is to minimise the number of people needed within a study whilst maintaining the statistical rigour needed to correctly answer the scientific questions.
Technical Summary
Given the increase in costs and high risk of developing new interventions smarter clinical designs are needed to make the best use of limited resources. The aim is that the leaner development plans will require fewer patients and shorter timelines. Our programme builds on that of the past where we have contributed to the design of numerous trials and continue to build collaborations in the area of adaptive designs. Most of the Hub Network for Trials Methodology Research stakeholders identified the need for more methodological research in the area of adaptive designs. This program can expand its research, training and software provision to meet this need and enable the translation of theory into future clinical trial practice. We aim to develop new trial designs to handle multiple treatments and multiple interim analyses that minimise the expected number of patients. There is an increasing need to consider diseases as multiple phenotypes and to find treatments that are effective for particular subgroups. Our methods aim to minimise the time of the clinical development and the amount of exposure time for patients whilst obtaining robust evidence for treatment safety and/or efficacy.
Each adaptive design is tailor-made for each clinical situation and the methodological problems are diverse. A clear example of this is the increasing need to design phase I trials for drug combination products. Currently phase I trial designs are very conservative using inefficient rule-based designs. We aim to not only develop better methods but to apply them in real trials, promote them in the clinical community and support clinical trials statisticians. We propose to develop more generalised methodology, such as methods that are robust to model assumptions, that can be applied across a broad range of trials to improve accessibility.
Of particular interest are study designs that can stop early and thus achieving a smaller expected sample size. Sample size re-estimation is another key area, where uncertain sample size parameters can be estimated part-way through a trial; we want to find optimal ways of how to do sample size re-estimation and how to use intermediate outcomes in these calculations.
Future clinical development will involve using more information from intermediate markers of efficacy and/or predictive/prognostic markers to help reduce the time and resources needed to get treatments to bedside. Many of our future proposals are looking to extend current methodology to optimally incorporate these new developments.
Each adaptive design is tailor-made for each clinical situation and the methodological problems are diverse. A clear example of this is the increasing need to design phase I trials for drug combination products. Currently phase I trial designs are very conservative using inefficient rule-based designs. We aim to not only develop better methods but to apply them in real trials, promote them in the clinical community and support clinical trials statisticians. We propose to develop more generalised methodology, such as methods that are robust to model assumptions, that can be applied across a broad range of trials to improve accessibility.
Of particular interest are study designs that can stop early and thus achieving a smaller expected sample size. Sample size re-estimation is another key area, where uncertain sample size parameters can be estimated part-way through a trial; we want to find optimal ways of how to do sample size re-estimation and how to use intermediate outcomes in these calculations.
Future clinical development will involve using more information from intermediate markers of efficacy and/or predictive/prognostic markers to help reduce the time and resources needed to get treatments to bedside. Many of our future proposals are looking to extend current methodology to optimally incorporate these new developments.
Organisations
- MRC Biostatistics Unit, United Kingdom (Lead Research Organisation)
- Cambridge University Hospitals NHS Foundation Trust, Cambridge (Collaboration)
- University of Cambridge (Collaboration)
- AstraZeneca plc (Collaboration)
- Roche Pharmaceuticals (Collaboration)
- Philips research cambridge (Collaboration)
- GlaxoSmithKline (GSK) (Collaboration)
- Cancer Research UK Cambridge Institute (Collaboration)
- Lancaster University (Collaboration)
- University of Birmingham, United Kingdom (Collaboration)
- Medical Research Council (Collaboration)
- Addenbrooke's Hospital (Collaboration)
Publications


Ahern AL
(2014)
Weight loss referrals for adults in primary care (WRAP): protocol for a multi-centre randomised controlled trial comparing the clinical and cost-effectiveness of primary care referral to a commercial weight loss provider for 12 weeks, referral for 52 weeks, and a brief self-help intervention [ISRCTN82857232].
in BMC public health

Anagnostou K
(2014)
Assessing the efficacy of oral immunotherapy for the desensitisation of peanut allergy in children (STOP II): a phase 2 randomised controlled trial.
in Lancet (London, England)

Bowden J
(2014)
Empirical Bayes estimation of the selected treatment mean for two-stage drop-the-loser trials: a meta-analytic approach.
in Statistics in medicine

Bowden J
(2016)
Gaining power and precision by using model-based weights in the analysis of late stage cancer trials with substantial treatment switching.
in Statistics in medicine

Bowden J
(2017)
Unbiased estimation for response adaptive clinical trials.
in Statistical methods in medical research

Bowden J
(2014)
A review and re-interpretation of a group-sequential approach to sample size re-estimation in two-stage trials.
in Pharmaceutical statistics

Bowden J
(2014)
Conditionally unbiased and near unbiased estimation of the selected treatment mean for multistage drop-the-losers trials.
in Biometrical journal. Biometrische Zeitschrift

Bowden J
(2016)
Assessing the suitability of summary data for two-sample Mendelian randomization analyses using MR-Egger regression: the role of the I2 statistic.
in International journal of epidemiology

Bowden J
(2016)
Weighing Evidence "Steampunk" Style via the Meta-Analyser.
in The American statistician
Description | New adaptive design mentioned as 'research highlight' in Nature Reviews Clinical Oncology |
Geographic Reach | National |
Policy Influence Type | Influenced training of practitioners or researchers |
URL | http://www.nature.com/nrclinonc/journal/vaop/ncurrent/full/nrclinonc.2015.138.html |
Description | Using Historical Data training course |
Geographic Reach | Local/Municipal/Regional |
Policy Influence Type | Influenced training of practitioners or researchers |
Impact | Adrian Mander was invited to create a course for GSK on the use of historical data to improve the design of clinical trials. This is a hugely important issue and has the ability to make efficiency gains for early phase clinical trials. GSK are already using the techniques in practice and this course was to enable a better understanding and wider usage of these techniques |
Description | AIM-HY |
Amount | £43,679 (GBP) |
Organisation | Medical Research Council (MRC) |
Sector | Public |
Country | United Kingdom |
Start | 01/2015 |
End | 12/2019 |
Description | EPAD- |
Amount | € 654,000 (EUR) |
Funding ID | 115736 |
Organisation | European Commission |
Department | Innovative Medicines Initiative (IMI) |
Sector | Public |
Country | Belgium |
Start | 04/2015 |
End | 03/2020 |
Description | HTMR Network Award |
Amount | £3,071 (GBP) |
Funding ID | N97 |
Organisation | Medical Research Council (MRC) |
Sector | Public |
Country | United Kingdom |
Start | 01/2018 |
End | 09/2018 |
Description | INNODIA |
Amount | € 500,000 (EUR) |
Funding ID | 115797 |
Organisation | European Commission |
Department | Innovative Medicines Initiative (IMI) |
Sector | Public |
Country | Belgium |
Start | 10/2015 |
End | 09/2022 |
Description | MBU grant |
Amount | £17,059 (GBP) |
Organisation | University of Edinburgh |
Department | MRC DPFS Resoure Centre |
Sector | Academic/University |
Country | United Kingdom |
Start | 11/2014 |
End | 02/2017 |
Description | NIHR HTA (Epilepsy Nurse) |
Amount | £52,826 (GBP) |
Organisation | National Institute for Health Research |
Sector | Public |
Country | United Kingdom |
Start | 04/2013 |
End | 11/2015 |
Description | NIHR XP grant |
Amount | £10,597 (GBP) |
Organisation | National Institute for Health Research |
Sector | Public |
Country | United Kingdom |
Start | 09/2015 |
End | 03/2020 |
Description | NIHR- Jonathan Mant |
Amount | £85,434 (GBP) |
Organisation | National Institute for Health Research |
Sector | Public |
Country | United Kingdom |
Start | 04/2015 |
End | 03/2020 |
Description | AZ - Jodrell phase I |
Organisation | AstraZeneca |
Country | United Kingdom |
Sector | Private |
PI Contribution | We have only just initiated this collaboration so we have been shortlisting and trying to recruit |
Collaborator Contribution | Shortlisting and recruiting |
Impact | A trial called ATRIUM is being developed currently |
Start Year | 2014 |
Description | AZ - Jodrell phase I |
Organisation | Cancer Research UK Cambridge Institute |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | We have only just initiated this collaboration so we have been shortlisting and trying to recruit |
Collaborator Contribution | Shortlisting and recruiting |
Impact | A trial called ATRIUM is being developed currently |
Start Year | 2014 |
Description | AZ joint postdoc |
Organisation | AstraZeneca |
Country | United Kingdom |
Sector | Private |
PI Contribution | We have a joint position to look at Phase I dose escalation designs |
Collaborator Contribution | Provide advice and scientific directin. |
Impact | None yet. |
Start Year | 2017 |
Description | CRUK grant |
Organisation | University of Birmingham |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | I have provided expertise in efficient statistical analysis of phase II oncology trials using the RECIST criteria. |
Collaborator Contribution | Individuals at the University of Birmingham provided clinical and applied statistical expertise that has helped us jointly develop a programme of work for a grant application. |
Impact | With individuals at the University of Birmingham, I have successfully applied for a Cancer Research UK grant, to start in Jan 2015. |
Start Year | 2013 |
Description | CTU joint positin |
Organisation | Cambridge University Hospitals NHS Foundation Trust |
Department | Cambridge Clinical Trials Unit |
Country | United Kingdom |
Sector | Hospitals |
PI Contribution | We have a joint position to work on dual agent dose escalation studies. |
Collaborator Contribution | Scientific input |
Impact | None yet |
Start Year | 2017 |
Description | Cambridge CTU |
Organisation | Addenbrooke's Hospital |
Department | Cambridge Cancer Trials Centre |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | We collaborate on several trials and have a joint position. We also provide computing to Simon Bond |
Collaborator Contribution | They collaborate with us on clinical trial designs |
Impact | None so far |
Start Year | 2013 |
Description | Diabetes Research |
Organisation | University of Cambridge |
Department | Cambridge Institute for Medical Research (CIMR) |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Involved in multiple trials involving Il-2 dose/frequency finding |
Collaborator Contribution | They ran the trial and gave the scientific steer |
Impact | Publications and talks all completed/delivered |
Start Year | 2011 |
Description | MAMS grant |
Organisation | Lancaster University |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Provided input to multiple submitted papers, including two in 2013/2014, that covered multi-arm multi-stage trials and phase II oncology trials. I provided input into writing a grant that was funded by the MRC methodology research panel. I was a co-investigator on this grant. |
Collaborator Contribution | Dr Thomas Jaki has provided complementary expertise into the writing of papers and the grant application. |
Impact | Multiple papers published since 2012 (recorded under publications). Successful grant application. |
Start Year | 2011 |
Description | Maxine's PhD |
Organisation | GlaxoSmithKline (GSK) |
Country | Global |
Sector | Private |
PI Contribution | I am the academic supervisor of this CASE award, the industry partners are within GSK. I give the main methodology direction of the project |
Collaborator Contribution | Partners give scientific and practical guidance into the project |
Impact | Several workshops delivered to GSK and AZ |
Start Year | 2013 |
Description | Philips Research Cambridge collaboration |
Organisation | Philips Research Cambridge |
Country | United Kingdom |
Sector | Private |
PI Contribution | I am the academic supervisor for a Ph.D. student on the subject of 'Adaptive designs for telehealth trials' |
Collaborator Contribution | Individuals at Philips Research Cambridge have provided expertise on telehealth trials and have hosted the student for a research visit. They also provided a financial contribution to the EPSRC case award that funds the studentship. |
Impact | The student (Lisa Law) started in October 2013 and has been upgraded to full Ph.D. student status in October 2014. During her time as a student, Lisa published two papers. Unfortunately Lisa chose to drop out of the PhD programme in 2016 and submitted what she had done up to that point for a MPhil degree, which she received successfully. |
Start Year | 2012 |
Description | Polypill |
Organisation | University of Cambridge |
Department | Cambridge Institute of Public Health |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Co-applicant on program grant, developing trial design with research team. |
Collaborator Contribution | Scientific direction and running trial |
Impact | Unfortunately this grant terminated as the trial was too difficult to implement in the time alloted |
Start Year | 2011 |
Description | Roche phase I trial design |
Organisation | Roche Pharmaceuticals |
Country | Global |
Sector | Private |
PI Contribution | We have engaged with industry to try and implement methods in practice. We have had some meetings and some presentations but also we have some joint work ongoing to help implement the PIPE design methodology |
Collaborator Contribution | The partners have listened and are committed to trying methods out in practice. Currently one of the their phase I trial designs is using the dual-agent design PIPE in one of their early phase trials (ClinicalTrials.gov Identifier: NCT02760797). |
Impact | Two trials were implemented and the results are in Roche. |
Start Year | 2014 |
Description | SMARTer trial collaboration |
Organisation | University of Cambridge |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | I have provided expertise on statistical design of adaptive clinical trials to develop a suitable clinical trial design for the needs of the collaborators. |
Collaborator Contribution | Input on the design and clinical expertise. They have also written a grant application to fund the trials. |
Impact | We have submitted a paper on the trial design and a grant application to the HTA efficient trial design call (currently at outline stage). |
Start Year | 2012 |
Description | WRAP study |
Organisation | Medical Research Council (MRC) |
Department | MRC Human Nutrition Research Group |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Trial design expertise and how to handle missing data |
Collaborator Contribution | Scientific direction and running the trial |
Impact | Paper accepted and work complete |
Start Year | 2012 |
Title | CRM |
Description | A stata module to design a continual reassessment trial |
Type Of Technology | Software |
Year Produced | 2013 |
Open Source License? | Yes |
Impact | There have been some downloads of this module but no references to it as of yet. |
Title | GROUPSEQ: Stata module providing functions to determine group sequential trial designs |
Description | A selection of functions to determine the boundaries and sample size required by several common group sequential designs, for two-arm trials with continuous outcome variables. |
Type Of Technology | Software |
Year Produced | 2017 |
Impact | Several trial statisticians and methodologists have subsequently been in contact to ask for further information around the design of group sequential clinical trials, particularly in Stata. |
URL | https://ideas.repec.org/c/boc/bocode/s458345.html |
Title | MVTNORM: Stata module to work with the multivariate normal and multivariate t distributions |
Description | A set of commands that allow users to compute the distribution function, density, equi-coordinate quantiles, and random vectors of the multivariate normal and multivariate t distributions. Any non-degenerate cases of the multivariate normal and multivariate t distributions can be worked with, along with a particular class of non-central multivariate t distributions. The commands are written in a combination of Stata and Mata for speed. |
Type Of Technology | Software |
Year Produced | 2017 |
Impact | MVTNORM has subsequently allowed the creation of the GROUPSEQ module for designing group sequential clinical trials. |
URL | https://ideas.repec.org/c/boc/bocode/s458043.html |
Title | MVTNorm |
Description | The Stata module calculates multivariate statistics. |
Type Of Technology | Software |
Year Produced | 2015 |
Open Source License? | Yes |
Impact | The stata module can be used in an group sequential /MAMS clinical trial and the companion stata journal article is in preparation. |
Title | Simon2stage |
Description | Stata module to design a simon two-stage design |
Type Of Technology | Software |
Year Produced | 2012 |
Open Source License? | Yes |
Impact | None so far but this was linked to a publication and those methods have been cited so this software is being used. |
Title | Simpute |
Description | A stata module to due simple imputaton |
Type Of Technology | Software |
Year Produced | 2013 |
Open Source License? | Yes |
Impact | It has been downloaded but no citations as of yet. |
Description | Adaptive Designs Talk at PSI conference |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | This talk raised the awareness of the Hub Network activities in adaptive trial designs. There was some discussion of the methods and discussion about being available for consultative purposes. No notable impacts |
Year(s) Of Engagement Activity | 2013 |
Description | Adaptive Designs symposium at the Institute of Psychiatry |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Professional Practitioners |
Results and Impact | There was some interesting debates about adaptive designs Raised our visibility in Psychiatry |
Year(s) Of Engagement Activity | 2013 |
Description | Adaptive designs training course at Pavia University, Italy |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | We gave a 3 day training course on adaptive trial designs with practicals in R. |
Year(s) Of Engagement Activity | 2017 |
Description | Armitage Lectures |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Annual workshop and lecture created and hosted by the MRC Biostatistics Unit, to honour the immense contributions of Professor Peter Armitage who was at the unit from 1947 to 1961, and whose work is recognised throughout the world as achieving a successful balance between methodological rigour and applied commonsense, to which all statisticians aspire. An eminent medical statistician visits for a week and works with members of the unit. The highlight is the Armitage Lecture, where more than 100 delegates attend. This event raises the unit research profile and creates new collaborations. |
Year(s) Of Engagement Activity | 2012,2013,2014,2015,2016,2017 |
URL | https://www.mrc-bsu.cam.ac.uk/news-and-events/armitage-lectureships-and-workshops/ |
Description | BSU Open Day |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Schools |
Results and Impact | Unit held open day as part of MRC Festival of Medical Research. The aim of the open day was to welcome secondary school students and members of the general public to come to the unit, find out about the research the unit does, and to take part in activities that illustrate BSU research, with the overall theme being 'Fun with statistics'. An open day of this format was a first for the unit and overall it was a very successful event. There were 40 attendees over a 4 hour event. All attendees pre-booked and were split into 4 groups for a 1 hour session comprising of an introduction, participation in hands-on activities, and a brief careers talk. The small groups and length of session allowed for quality engagement between the scientists and the audience. Feedback from the attendees was very positive, and the wider MRC Festival activities that took place in Cambridge demonstrated the benefits in delivering these types of events. |
Year(s) Of Engagement Activity | 2016 |
URL | http://www.mrc-bsu.cam.ac.uk/bsu-open-day-2016-why-are-statistics-important/ |
Description | Cambridge Science Festival |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Public/other audiences |
Results and Impact | Each year BSU participate in Cambridge Science Festival - members of the general public explore and discuss issues of scientific interest and concern, through a series of different events. The festival also aims to raise aspirations by encouraging young people to consider a career in science, technology, engineering or mathematics. BSU take part over two full days - 'Science Saturday' and the 'Cambridge Biomedical Campus' day. The unit presents a stand with 4 - 5 interactive activities that each communicate a basic statistical method or idea, representing one of the four research themes in the unit. Each year a new activity is developed and delivered requiring scientific input from staff and students across the unit. Over the two days, BSU engage with approximately 500 adults and children who visit the festival. |
Year(s) Of Engagement Activity | 2012,2013,2014,2015,2016,2017 |
URL | http://www.sciencefestival.cam.ac.uk/ |
Description | Contributed talks at the 18th INFORMS Applied Probability Conference |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | SV gave contributed talks at the 18th INFORMS Applied Probability Conference and the 7th Greek Stochastics meeting. Talk entitled "Novel bandit-based solutions for practical stochastic scheduling problems". |
Year(s) Of Engagement Activity | 2015 |
URL | http://www.stochastics.gr/meetings/eta/ |
Description | Dose finding talk (Charleston) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Talk sparked a discussion on various dose-ranging aspects No immediate impacts |
Year(s) Of Engagement Activity | 2014 |
Description | Dose ranging talk (Cambridge) invited by the Cambridge Discussion Group |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Professional Practitioners |
Results and Impact | Talk sparked a debate about these methods. Made some industry links within Roche and Astra Zeneca |
Year(s) Of Engagement Activity | 2014 |
Description | Invited presentation at an early phase clinical conference (Paris) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | I presented a novel clinical trial design on dual-agent dose escalation, there was a lot of interest from academics and industry. Since then I have worked closely with a member of Roche pharmaceuticals on how to implement these methods in practice. |
Year(s) Of Engagement Activity | 2015 |
Description | Invited talk at CLDAG 2017 conference |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | I was an invited speaker to this international statistical conference. I spoke about adaptive dose ranging trial designs. |
Year(s) Of Engagement Activity | 2017 |
Description | Invited talk at the PSI meeting on Extrapolation |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | I was invited to give a talk on using historical data in trial inference and design. |
Year(s) Of Engagement Activity | 2017 |
Description | Invited talk for the Breathlessness conference (Cambridge) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Adrian Mander gave a presentation on the various ways to adapt clinical trials to the area of palliative and end of life care. There has been numerous discussions since the talk by email with plans to produce a paper introducing this area to the wider audience undertaking these challenging trials. |
Year(s) Of Engagement Activity | 2015 |
Description | Invited talk on early phase clinical trial designs (London) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | 80 members of clinical trials units attended a workshop run by the NCRI, the talk on early phase designs was aimed at changing practice and how we design phase I trials. |
Year(s) Of Engagement Activity | 2015 |
Description | Invited to give a departmental seminar on adaptive designs (Norwich) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Policymakers/politicians |
Results and Impact | Around 30 members of the clinical trials unit attended my talk on adaptive clinical trials, there was a great deal of interest and promises of future collaboration as there was a recognition that these methods will be needed in the near future |
Year(s) Of Engagement Activity | 2015 |
Description | NCRI talk on phase I trials (Liverpool) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | There was some debate and discussion. None so far but this was the start of raising the awareness of better trials in oncology |
Year(s) Of Engagement Activity | 2012 |
Description | Oral presentation at the 23rd London Stata Users Group Meeting |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Presented a talk entitled "Group sequential clinical trial designs for normally distributed outcome variables". |
Year(s) Of Engagement Activity | 2017 |
URL | http://www.timberlake.co.uk/2017-stata-user-group-abstracts |
Description | Oral presentation at the Adaptive Designs and Multiple Testing Procedures Workshop |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Presented a talk entitled "A two-stage Fisher exact-test for multi-arm trials with binary endpoints". |
Year(s) Of Engagement Activity | 2017 |
URL | https://www.mrc-bsu.cam.ac.uk/event/adaptive-designs-multiple-testing-procedures-workshop-2017/ |
Description | Oral presentation at the CEN-ISBS 2017 Joint Conference on Biometrics and Biopharmaceutical Statistics |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Presented a talk, in a contributed session, entitled "Do single-arm trials have a role in drug development plans incorporating randomised trials?". |
Year(s) Of Engagement Activity | 2017 |
URL | http://cenisbs2017.org/ |
Description | Phase I course in dose-escalation adaptive designs at UCL |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | This was a two day training course aimed at statisticians in early phase designs. There were also regulators at this meeting. |
Year(s) Of Engagement Activity | 2017 |
Description | Poster presentation at the 38th Annual Conference of the International Society of Clinical Biostatistics |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Presented a poster entitled "Admissible multi-arm stepped-wedge cluster randomized trial designs". |
Year(s) Of Engagement Activity | 2017 |
URL | http://iscb2017.info/ |
Description | Poster presentation at the 4th International Clinical Trials Methodology Conference (ICTMC) and the 38th Annual Meeting of the Society for Clinical Trials |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Presented a poster entitled "Blinded and unblinded sample size re-estimation procedures for stepped wedge cluster randomised trials". |
Year(s) Of Engagement Activity | 2017 |
Description | Poster presentation at the Current Developments in Cluster Randomised Trials and Stepped Wedge Design workshop |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Presented a poster entitled "Design of an adaptive cluster randomised crossover trial, with application to the STRIVE trial". |
Year(s) Of Engagement Activity | 2017 |
Description | Research Design Service (LONDON) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Professional Practitioners |
Results and Impact | I was invited to talk about adaptive clinical trials as part of a workshop of talks. There is a call for novel designs and my talk covered various methods that can be used to make trials more efficient |
Year(s) Of Engagement Activity | 2015 |
Description | Talk about adaptive enrichment designs (Institute of Cancer Research |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Professional Practitioners |
Results and Impact | We had a day long discussion about various potential collaborations There were no massive impacts, a similar study was planned in their institute but it was too late to help improve it. |
Year(s) Of Engagement Activity | 2014 |
Description | Talk at Clinical Trials in Small Populations workshop |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | SV was co-organiser of worksop, gave a tutorial on `Response-adaptive randomisation' during Day 1 and also contributed to one of the talks during Day 2 entitled 'Bringing patient population size into clinical trial design using response-adaptive randomisation'. |
Year(s) Of Engagement Activity | 2015 |
Description | Talk at Lancaster University - Methodological work to make bandit models suitable to design (and improve) multi-armed clinical trial |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | SV invited speaker at workshop that gathered worldwide experts on ''bandit problems" (reinforcement learning bandits, restless bandits, non-parametric bandits, Bayesian bandits, etc., ) to discuss recent methodological advances and applications. SV spoke about recent methodological work to make bandit models suitable to design (and improve) multi-armed clinical trials. |
Year(s) Of Engagement Activity | 2016 |
Description | Talk at the local RSS NW group |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Professional Practitioners |
Results and Impact | A review talk on the multiple bandit-based designs publications of Sofia Villar |
Year(s) Of Engagement Activity | 2016 |
Description | Talk on adaptive designs to Astra Zeneca |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | There was much interest in the methods and raised awareness of our group to the company Since this talk we have secured a data sharing agreement and collaboration. There are also 3 AZ post-docs that we have been linked with |
Year(s) Of Engagement Activity | 2013 |
Description | Workshop on Recent and Future Trends in Biostatistics |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Postgraduate students |
Results and Impact | One-off, unique workshop for early career statisticians on the theme of recent and future trends in biostatistics. Workshop included three plenary speakers and six contributed presentations, as well as interactive presentations and a panel discussion. Many attendees reported that they found the workshop very useful. Delegate feedback included:"A great mix of sessions", "A very enjoyable workshop", "Workshop opened itself to initiate conversations with other like minded individuals", "I have come away with a lot of information on new and exciting statistical techniques". One delegate has used information from panel discussion debate on the topic of biostatistics training, to quote in their career development review. |
Year(s) Of Engagement Activity | 2017 |
Description | Workshop provision at the Society for Social Medicine 61st Annual Scientific Meeting |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Assisted in the organisation and delivery of a workshop entitled "Novel trial designs and analytical methods for evaluating complex public health interventions: a discussion", in collaboration with members of the MRC Lifecourse Epidemiology Unit. |
Year(s) Of Engagement Activity | 2017 |