The economic impact of precision medicine in rheumatology: targeting the use of methotrexate in early rheumatoid arthritis

Stratified (personalised/precision) medicine is promising from an economic perspective since in principle only those patients likely to benefit will receive a treatment. A recurring methodological challenge in economic evaluation of stratified medicine is a result of gaps in the data and evidence. A particular challenge occurs when evaluating a strategy to predict the risk of a rare adverse drug reaction (ADR) and effectiveness of a medicine. There are limited randomised clinical trial (RCT) evidence for such an intervention on the basis that the required sample size to achieve adequate power makes the design unfeasible. Furthermore, the data required to evaluate how the precision medicine strategy can be moved from the research to clinical environment, taking account of the model of service delivery, cannot be readily identified from a RCT.

Hypothesis: observational data can provide a source of data to design and populate model-based
economic evaluations of a companion diagnostic to identify patients at risk of an adverse drug reaction from a treatment for patients with rheumatoid arthritis (RA).

Objectives:
(1) structure a model-based economic evaluation of a precision medicine strategy to stratify patient populations with RA at risk of an ADR and/or poor response from anti-rheumatic treatment
(2) structure the model using observational data
(3) populate the model with observational data to identify the incremental costs and benefits of the precision medicine strategy

Methods to be used during the project are:
(1) systematic reviews of the clinical and economic literature
(2) microcosting
(3) model-based cost effectiveness analysis

Methods to use existing datasets to generate economic evidence to move precision medicine from the research to clinical environment.

This PhD involves the development of quantitative methods but also offers a student the opportunity to work with practising clinicians and produce outputs with practical relevance to inform the more effective use of the NHS budget. Importantly, this PhD focuses on a core cross-cutting research theme stratified (precision) medicine both through the selection of the clinical area for the methodological research but also via the involvement of three academics with core roles in current research programmes across the University.


The student will further develop core skills in quantitative research methods including: systematic review; model-based cost effectiveness analysis; microcosting. The student will need to become proficient in the use of statistical software packages (such as R and/or Stata).

Both Centres (health economics, musculoskeletal research) run internal and external seminars providing an excellent means of knowledge exchange between peers within economics but also practising clinicians. The student will have opportunities to attend relevant modules from the University of Manchester's MSc in the Economics of Health, MSc in Statistics, MSc in Health Data Science and MSc in Rheumatology.