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Getting the questions right: A multi-stakeholder approach to designing cancer clinical trials

   Clinical Trials and Statistics Unit

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  Prof Christina Yap  No more applications being accepted  Competition Funded PhD Project (Students Worldwide)

About the Project

Estimands are used in clinical trials to ensure that we are asking (and answering) the right research questions. The estimand framework provides a structured approach for explicit specification of what we plan to estimate in order to address the questions that are important to different stakeholders(1). It aims to improve dialogue between multi-disciplinary groups developing trials, reduce misunderstandings, and ensure that all aspects of a trial (design, conduct, analysis and interpretation) are aligned with the trial objectives. However, routine adoption of the framework has been relatively slow and has largely been considered by statisticians and methodologists(2-4). There is a need to develop models of working with different stakeholders (including patients, clinicians, regulators and funders) to apply the framework and to produce exemplars of estimand approaches for different clinical settings, trial phases and interventions.

The student will be based within the Institute of Cancer Research Clinical Trials and Statistics Unit (ICR CTSU) where they will benefit from “real-world” experience, being embedded within trial teams working on cancer clinical trials.

The aim of the project will be to develop exemplars and guidance for application of the estimand framework in different types of trials. There will be a focus on early phase studies where application of the framework is particularly rare; but later phase trials of different types of intervention (for example, drug and non-drug treatments, palliative care interventions) will also be considered, dependent on the initial literature review. Specific objectives will be:

·      To review existing literature to gauge the extent to which estimands have been used, and how they have been implemented, in cancer clinical trials to date

·      To work with multi-disciplinary trial development groups to develop estimands relevant to different stakeholders (including patients) and consider their impact on trial design, conduct and analysis

·      Apply mixed methods to evaluate the experience of using the framework, considering the impact on the trial development process and identifying barriers to implementation 

·      To develop non-technical guidance/recommendations (and associated resources) for applying the estimand framework in cancer clinical trials

At least 3 peer-reviewed publications are anticipated in methodological/clinical trial journals.


The project will be supervised by Christina Yap (Professor of Clinical Trials Biostatistics) and Dr Fay Cafferty (Principal Statistician) at ICR-CTSU. Co-supervisors will include Dr Beatriz Goulao, an expert in patient and public involvement in numerical aspects of trials, and Dr Katie Gillies, an expert in mixed-methods research, both based at the Health Services Research Unit, University of Aberdeen. Collectively, the team has a highly relevant mix of expertise for the project, as well as previous experience in supervising PhD students.

As part of work in the early phase setting, the student will work closely with the team at the Drug Development Unit at the Royal Marsden Hospital. They will also spend some time with the team at the Health Services Research Unit, University of Aberdeen.

A key aspect will be working with patient advocates to better understand how the questions of interest to patients may differ to other stakeholders and how the estimand framework might be used to address these; and to identify barriers and approaches to engaging patients in estimand development. This will draw on methods for involving patients and the public in numerical aspects of trials(5).       


You are applying for a PhD studentship from the MRC TMRP DTP. A list of potential projects and the application form is available online at:

Please complete the form fully. Incomplete forms will not be considered. CVs will not be accepted for this scheme.

Please apply giving details for your first choice project. You can provide details of up to two other TMRP DTP projects you may be interested in at section B of the application form.

Before making an application, applicants should contact the project supervisor to find out more about the project and to discuss their interests in the research before 09 January 2023.

The deadline for applications is 4pm (GMT) 16 January 2023. Late applications will not be considered.

Completed application forms must be returned to: [Email Address Removed]

Informal enquiries may be made to Prof Christina Yap - [Email Address Removed] or Dr Fay Cafferty - [Email Address Removed]

Funding Notes

Studentships are funded by the Medical Research Council (MRC) for 3 years. Funding will cover tuition fees at the UK rate only, a Research Training and Support Grant (RTSG) and stipend. We aim to support the most outstanding applicants from outside the UK and are able to offer a limited number of bursaries that will enable full studentships to be awarded to international applicants. These full studentships will only be awarded to exceptional quality candidates, due to the competitive nature of this scheme.


1. European Medicines Agency. ICH E9 (R1) addendum on estimands and sensitivity analysis in clinical trials to the guideline on statistical principles for clinical trials 2020 19th August 2022. Available from:
2. Fletcher C, Hefting N, et al. Marking 2-Years of New Thinking in Clinical Trials: The Estimand Journey. Ther Innov Regul Sci. 2022;56(4):637-50. DOI: 10.1007/s43441-022-00402-3.
3. Kahan BC, Morris TP, et al. Estimands in published protocols of randomised trials: urgent improvement needed. Trials. 2021;22(1):686. DOI: 10.1186/s13063-021-05644-4.
4. Cro S, Kahan BC, et al. Evaluating how clear the questions being investigated in randomised trials are: systematic review of estimands. BMJ. 2022;378:e070146. DOI: 10.1136/bmj-2022-070146.
5. Goulao B, Poisson C, et al. Patient and public involvement in numerical aspects of trials: a mixed methods theory-informed survey of trialists' current practices, barriers and facilitators. BMJ Open. 2021;11(3):e046977. DOI: 10.1136/bmjopen-2020-046977.
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