Title: Personalising Outcomes for Patients
Funder: Department of Health and Social Care

Abstract: Research Question How can we unify outcome assessment for randomised trial participants with different goals? Background Specification of a single 'primary' outcome is often a challenge given the multifactorial nature of most health conditions. In some conditions this challenge is exacerbated because the patient-centred outcomes are heterogeneous. Polycystic Ovarian Syndrome (PCOS) and Endometriosis are good examples of this. Women with these conditions will be diagnosed at different stages of their adult life and present with different symptoms. For the majority of these women, their treatment options will be the same, however, their desired outcome will be different. Previous qualitative work has suggested that patient and clinician opinion do not coincide regarding what outcomes from treatment are of importance to women. Additionally, in current gynaecological research, studies are generally small and underpowered, with substantial differences in the reporting and measuring of outcomes. This leads to research waste. In order to produce high-quality, thoughtful and efficient trial designs, it is important to consider how patients with the same diagnoses but differing symptoms, can be included in the same trial. This project will study whether a single, meaningful, patient-centred outcome of 'success' could be used in a clinical trial setting, and whether this results in improved statistical properties. Key Aims and Objectives The overall aim of this project is to develop and evaluate statistical methodology to combine different outcomes into a single, patient-centred definition of 'success of treatment'. Project Plan The programme of work has been divided into several discrete work packages and continuous workstreams: Work Package 1 (WP1) - Methodological systematic review. Work Package 2 (WP2) -h; Adapting ideas of basket trials for use in gynaecology trials. Work Package 3 (WP3) -h; Evaluation of methodology (using findings from WP1 and WP2).Continuous workstreams: Public Patient Involvement, personal development, write-up and dissemination. Anticipated Impact This project has the potential to deliver the following outputs and impact within 5 years of completion: Guidance produced to measure 'success of treatment' in a clinical trial setting which will be applicable to women's health and potentially adaptable to other clinical areas where recipients of clinical intervention seek very different goals. Adaptation and development of innovative clinical trial designs for use in gynaecology settings. Reduction of research waste in clinical trials saving the NHS and trial funders both money and time. Patients and the public more involved in methodological research projects, trial design and outcome selection. Raised awareness among clinicians and researchers of the importance of involving patients in research (including treatment evaluation and outcome selection) and the value they can add in the move towards personalised healthcare. Strengthening of the evidence base by ensuring trials are statistically efficient and report outcomes which can be used and understood. In turn, this benefits systematic reviewers and guideline developers.In the long term, with the use of the methods and guidance generated, this will change the way in which primary outcomes are chosen and measured. This will provide clinical decision-makers with accurate information on how treatments work for patients.

Lay abstract: There is increasing pressure to ensure that the research being done in and out of the healthcare setting is efficient and not wasteful. Wastage in research can come from five different areas: research question selection, study design, research conduct, publication, and reporting. In this project, the focus will be on the selection of the research question. Asking the wrong research questions (e.g. not collecting measurements that are of importance to patients) contributes immensely to research waste. Reducing this problem is the motivation for this project. In current practice, when selecting a research question, researchers generally have a measure of main importance (referred to as a primary outcome). Alongside this primary outcome, they also collect other measurements (secondary outcomes). However, specification of a single 'primary' outcome is often a challenge given the diverse nature of most health conditions. In some circumstances, this challenge is made worse because the patient-centred outcomes are diverse. A clinical area which is a good example of this is women's health/gynaecology. For this project, I will focus on the clinical area of gynaecology, using Polycystic Ovarian Syndrome (PCOS) and Endometriosis as exemplar conditions. Women may experience a variety of gynaecological conditions at different times in their adult life. The same condition will have different bothersome symptoms. For example, with PCOS, some women will receive their diagnosis as they are not having regular periods, whereas others may have male patterned hair growth. Often, these women will receive the same treatment, although the symptoms they want to be resolved are different. In a clinical trial, these women would not all be included. This is because although they have the same gynaecological condition and are being treated the same way, they will want different things (outcomes) from their treatment. The first woman wants a regular period, whilst the other wants a reduction in facial hair. For trial design the options are: Pick a primary outcome which is common to all patients; Analyse only in small groups, according to presenting symptoms; Combine essentially different outcomes into a single, patient-centred definition of 'success' of treatment.Option (1) allows all patients to be included in the trial. However, an outcome common to all patients may not be of primary importance to any. Option (2) is inefficient as it involves running multiple small studies rather than having a single definitive assessment of the treatment for the overall patient group. Option (3) will be the focus of this project. I will develop statistical methods to allow for different outcomes to be combined into a single, patient-centred definition of 'success' of treatment. The statistical methodology generated from this project will be applicable to other clinical areas in which patients with the same condition have differing symptoms. This project is of importance for NHS patients, as through improving the efficiency of trials, we increase the chance of finding the best treatments. Additionally, this project will increase the value for money of research. During the course of the project, I will also host meetings with patients to receive their ideas and actively include them in project decisions. Their advice will input into the research plan and ongoing circulation of findings. Methodological projects, like this one, can only benefit patients and health care if the new methods and the associated benefits are well communicated to researchers and trusted by clinicians and potential research participants. Methods developed during this project will be circulated through journal publication and attendance at national and international conferences. Social media (such as Twitter and Facebook) will also be used for dissemination purposes.

Research activity codes: 8.4 RESEARCH DESIGN AND METHODOLOGIES (HEALTH SERVICES)
Health categories: DISPUTED AETIOLOGY AND OTHER