Investigating and understanding the role of patient preferences in trial participation and outcome

Lead Research Organisation: University of Bristol
Department Name: Social Medicine

Abstract

Sometimes doctors do not know the best treatment to give a patient because there is not enough scientific evidence to say which is best. In this case medical trials are conducted to compare the different treatments. To compare treatments accurately in trials patients should have their treatment selected at random (i.e. by chance). Some patients, however, prefer to choose their own treatment but this causes problems for those who analyse the data. This study intends to find out more about patients who prefer to choose their treatment by seeing if they differ from those who have their treatment randomly allocated and if they achieve greater benefits from the treatment. The study will also explore why patients have preferences for treatment and if this stops them from agreeing to have their treatment selected at random. Data will be collected by asking patients to complete questionnaires, by talking to them and by tape recording their consultation with the doctor. By understanding more about the role of patient preferences in trial participation, scientists will be able to conduct and analyse trials more accurately and thus have a better idea of what treatment is best when there is doubt.

Technical Summary

Overall aim: To investigate and understand the role of patient preferences in trial participation and outcome through the use of qualitative and quantitative research methods.

Design: Stage I will involve a review of the relevant literature. Stage II will explore the basis upon which patient preferences for treatment are formed, whether they change over time, how they are presented and dealt with during trial recruitment, and what influence they have in facilitating or preventing recruitment to trials. Stage III will examine social and demographic differences between patients who choose a preferred treatment and those who agree to be randomised. Stage IV will compare symptomatic and quality of life measures between the two groups of patients to indicate the impact of patient preferences on outcome. Stage V will investigate how information from preference arms can be used in interpreting results in randomised trials.

Methods: The study will use data from a trial of treatments for localised prostate cancer (ProtecT study). Stage II will utilise audiotape recorded data from in-depth interviews with patients and from their consultation with the recruiting clinician. Methods of analysis will involve constant comparison and conversation analysis. Stage III and IV will extract data from patient completed questionnaires. Logistic regression will be used to examine social and demographic differences (Stage III), and to assess the impact of patient preferences on quality of life outcomes (Stage IV). Stage V will involve a review of statistical methods for analysing a preference group in a trial.
 
Description Qualitative research in trials national workshop
Geographic Reach National 
Policy Influence Type Influenced training of practitioners or researchers
 
Description Linbury Trust and Ashden Trust Project Grant - SMILE feasibility and main trial
Amount £164,000 (GBP)
Organisation The Sainsbury Family Charitable Trusts 
Department Linbury Trust
Sector Charity/Non Profit
Country United Kingdom
Start 09/2010 
 
Description MRC HTMR small study funding - Workshop on clinical trials in head and neck cancer.
Amount £5,472 (GBP)
Organisation Medical Research Council (MRC) 
Department Network of Hubs for Trials Methodology Research (HTMR)
Sector Academic/University
Country United Kingdom
Start 05/2013 
End 03/2014
 
Description MRC Network of Hubs for Trials Methodology Research (HTMR) funding- workshop on recruitment and retention in trials
Amount £16,052 (GBP)
Organisation Medical Research Council (MRC) 
Sector Academic/University
Country United Kingdom
Start 09/2012 
End 08/2013
 
Description NIHR HTA programme clinical evaluation and trials - ECHoES trial
Amount £381,646 (GBP)
Organisation National Institute for Health Research 
Sector Public
Country United Kingdom
Start 03/2013 
End 08/2014
 
Description MRC ConDuCT Hub - Hub in Trial Methodology Research 
Organisation University of Bristol
Department School of Social and Community Medicine
Country United Kingdom 
Sector Academic/University 
PI Contribution Findings from my MRC fellowship highlighted the importance of integrating qualitative research within trials, and especially the importance of using more novel methods of analysis (recordings of recruitment appointments) to investigate and overcome recruitment difficulties in RCTs. This work arising from my fellowship contributed to the set up of a formal collaboration with colleagues within and across departments and a proposal to set up a ConDuCT Hub to develop innovative research methods for the design and delivery of RCTs under the umbrella of the Hubs for Trials Methodology research (HTMR).
Collaborator Contribution Colleagues from the former Department of Community Based Medicine contributed their expertise to the ConDuCT Hub proposal. In particular, results from their research were combined with results from my research and those of my departmental colleagues to support the need for the ConDuCT Hub proposal and to clarify the aims, objectives and design of the research
Impact The ConDuCT Hub proposal to the MRC was successful and research is currently underway. My involvement has so far resulted in publications, funded research (SMILE feasibility trial, ECHoES trial), running a national workshop to highlight and discuss the importance of qualitative research within trials and giving presentations on findings from my fellowship research. Multi-disciplinary collaboration involving: qualitative research, statistics, health economics, health services research and epidemiology
Start Year 2009
 
Description MRC Hubs for Trials Methodology Research Trial Recruitment Working Group 
Organisation Medical Research Council (MRC)
Department MRC All-Ireland Hub for Trials Methodology Research
Country United Kingdom 
Sector Public 
PI Contribution I lead and contribute to the working group
Collaborator Contribution Contributed to all outputs to date
Impact Document on "Tips for optimising recruitment of patients to randomised clinical trials"; national cross hub workshop on recruitment into surgical trials; national cross hub workshop on the value of integrating qualitative research in randomised trials; session on trial recruitment at the National HTMR annual meeting; preparation for a forthcoming national cross hub workshop with clinical trial unit directors on recruitment and retention in randomised trials.
Start Year 2011
 
Description MRC Hubs for Trials Methodology Research Trial Recruitment Working Group 
Organisation Medical Research Council (MRC)
Department MRC North West Hub for Trials Methodology Research
Country United Kingdom 
Sector Public 
PI Contribution I lead and contribute to the working group
Collaborator Contribution Contributed to all outputs to date
Impact Document on "Tips for optimising recruitment of patients to randomised clinical trials"; national cross hub workshop on recruitment into surgical trials; national cross hub workshop on the value of integrating qualitative research in randomised trials; session on trial recruitment at the National HTMR annual meeting; preparation for a forthcoming national cross hub workshop with clinical trial unit directors on recruitment and retention in randomised trials.
Start Year 2011
 
Description MRC Hubs for Trials Methodology Research Trial Recruitment Working Group 
Organisation Medical Research Council (MRC)
Department Network of Hubs for Trials Methodology Research (HTMR)
Country United Kingdom 
Sector Academic/University 
PI Contribution I lead and contribute to the working group
Collaborator Contribution Contributed to all outputs to date
Impact Document on "Tips for optimising recruitment of patients to randomised clinical trials"; national cross hub workshop on recruitment into surgical trials; national cross hub workshop on the value of integrating qualitative research in randomised trials; session on trial recruitment at the National HTMR annual meeting; preparation for a forthcoming national cross hub workshop with clinical trial unit directors on recruitment and retention in randomised trials.
Start Year 2011
 
Description MRC Hubs for Trials Methodology Research Trial Recruitment Working Group 
Organisation University of Edinburgh
Department Edinburgh Hub for Trials Methodology Research
Country United Kingdom 
Sector Academic/University 
PI Contribution I lead and contribute to the working group
Collaborator Contribution Contributed to all outputs to date
Impact Document on "Tips for optimising recruitment of patients to randomised clinical trials"; national cross hub workshop on recruitment into surgical trials; national cross hub workshop on the value of integrating qualitative research in randomised trials; session on trial recruitment at the National HTMR annual meeting; preparation for a forthcoming national cross hub workshop with clinical trial unit directors on recruitment and retention in randomised trials.
Start Year 2011
 
Description MRC Hubs for Trials Methodology Research Trial Recruitment Working Group 
Organisation University of Oxford
Department Oxford Hub
Country United Kingdom 
Sector Academic/University 
PI Contribution I lead and contribute to the working group
Collaborator Contribution Contributed to all outputs to date
Impact Document on "Tips for optimising recruitment of patients to randomised clinical trials"; national cross hub workshop on recruitment into surgical trials; national cross hub workshop on the value of integrating qualitative research in randomised trials; session on trial recruitment at the National HTMR annual meeting; preparation for a forthcoming national cross hub workshop with clinical trial unit directors on recruitment and retention in randomised trials.
Start Year 2011
 
Description SMILE feasibility and main trial (intervention for CFS/ME) 
Organisation University of Bristol
Department School of Social and Community Medicine
Country United Kingdom 
Sector Academic/University 
PI Contribution Work arising from my MRC funded fellowship resulted in the formal collaboration with colleagues from the Centre for Child and Adoloescent Health and a proposal to assess the feasibility and acceptability of an RCT comparing interventions for children with chronic fatigue syndrome or myalgic encephalopathy. My fellowship work in particular highlighted the importance of analysing recruitment appointments to maximise trial recruitment and this method was integrated into the research proposal resulting from the collaboration. I am responsible for designing and overseeing the qualitative component of this feasibility trial.
Collaborator Contribution Colleagues from the Centre for Child and Adolescent Health, University of Bristol, designed and undertook a feasibility study to assess the feasibility and acceptability of an RCT comparing interventions for children with chronic fatigue syndrome or myalgic encephalopathy.
Impact The research proposal was successful and research commenced in September 2010. The feasibility study converted to a main trial from October 2012. Multi-disciplinary research comprising paediatrics, CFS/ME clinical research, qualitative research, Health economics, health services research and epidemiology
Start Year 2010
 
Description The ECHoES trial 
Organisation Moorfields Eye Hospital NHS Foundation Trust
Department NIHR Moorfields Biomedical Research Centre
Country United Kingdom 
Sector Academic/University 
PI Contribution An NIHR HTA Programme Clinical Evaluation and Trials proposal has been funded to assess the effectiveness of shared care for patients with neovascular age related macular degeneration. I devised a qualitative component as part of this proposal to determine the views of health professionals and patients on the devolving of monitoring from hospital based ophthalmologists to community based optometrists to assess the need for re-treatment.
Collaborator Contribution Collectively the partners put together an HTA proposal to assess the effectiveness of community versus hospital eye service follow-up for patients with neovascular age related macular degeneration.
Impact An NIHR HTA Programme Clinical Evaluation and Trials proposal was accepted for funding and is currently being undertaken. Multidisciplinary collaboration involving: ophthalomology, health services research, health economics, epidemiology, vision and vascular science, qualitative research
Start Year 2012
 
Description The ECHoES trial 
Organisation Queen's University Belfast
Department Centre for Experimental Medicine
Country United Kingdom 
Sector Academic/University 
PI Contribution An NIHR HTA Programme Clinical Evaluation and Trials proposal has been funded to assess the effectiveness of shared care for patients with neovascular age related macular degeneration. I devised a qualitative component as part of this proposal to determine the views of health professionals and patients on the devolving of monitoring from hospital based ophthalmologists to community based optometrists to assess the need for re-treatment.
Collaborator Contribution Collectively the partners put together an HTA proposal to assess the effectiveness of community versus hospital eye service follow-up for patients with neovascular age related macular degeneration.
Impact An NIHR HTA Programme Clinical Evaluation and Trials proposal was accepted for funding and is currently being undertaken. Multidisciplinary collaboration involving: ophthalomology, health services research, health economics, epidemiology, vision and vascular science, qualitative research
Start Year 2012
 
Description The ECHoES trial 
Organisation Queen's University Belfast
Department Centre for Public Health (CPH)
Country United Kingdom 
Sector Academic/University 
PI Contribution An NIHR HTA Programme Clinical Evaluation and Trials proposal has been funded to assess the effectiveness of shared care for patients with neovascular age related macular degeneration. I devised a qualitative component as part of this proposal to determine the views of health professionals and patients on the devolving of monitoring from hospital based ophthalmologists to community based optometrists to assess the need for re-treatment.
Collaborator Contribution Collectively the partners put together an HTA proposal to assess the effectiveness of community versus hospital eye service follow-up for patients with neovascular age related macular degeneration.
Impact An NIHR HTA Programme Clinical Evaluation and Trials proposal was accepted for funding and is currently being undertaken. Multidisciplinary collaboration involving: ophthalomology, health services research, health economics, epidemiology, vision and vascular science, qualitative research
Start Year 2012
 
Description The ECHoES trial 
Organisation University of Bristol
Department School of Clinical Sciences
Country United Kingdom 
Sector Academic/University 
PI Contribution An NIHR HTA Programme Clinical Evaluation and Trials proposal has been funded to assess the effectiveness of shared care for patients with neovascular age related macular degeneration. I devised a qualitative component as part of this proposal to determine the views of health professionals and patients on the devolving of monitoring from hospital based ophthalmologists to community based optometrists to assess the need for re-treatment.
Collaborator Contribution Collectively the partners put together an HTA proposal to assess the effectiveness of community versus hospital eye service follow-up for patients with neovascular age related macular degeneration.
Impact An NIHR HTA Programme Clinical Evaluation and Trials proposal was accepted for funding and is currently being undertaken. Multidisciplinary collaboration involving: ophthalomology, health services research, health economics, epidemiology, vision and vascular science, qualitative research
Start Year 2012
 
Description The ECHoES trial 
Organisation University of Liverpool
Department Department of Eye and Vision Science
Country United Kingdom 
Sector Academic/University 
PI Contribution An NIHR HTA Programme Clinical Evaluation and Trials proposal has been funded to assess the effectiveness of shared care for patients with neovascular age related macular degeneration. I devised a qualitative component as part of this proposal to determine the views of health professionals and patients on the devolving of monitoring from hospital based ophthalmologists to community based optometrists to assess the need for re-treatment.
Collaborator Contribution Collectively the partners put together an HTA proposal to assess the effectiveness of community versus hospital eye service follow-up for patients with neovascular age related macular degeneration.
Impact An NIHR HTA Programme Clinical Evaluation and Trials proposal was accepted for funding and is currently being undertaken. Multidisciplinary collaboration involving: ophthalomology, health services research, health economics, epidemiology, vision and vascular science, qualitative research
Start Year 2012
 
Description The ECHoES trial 
Organisation University of Oxford
Department Health Economics Research Centre
Country United Kingdom 
Sector Academic/University 
PI Contribution An NIHR HTA Programme Clinical Evaluation and Trials proposal has been funded to assess the effectiveness of shared care for patients with neovascular age related macular degeneration. I devised a qualitative component as part of this proposal to determine the views of health professionals and patients on the devolving of monitoring from hospital based ophthalmologists to community based optometrists to assess the need for re-treatment.
Collaborator Contribution Collectively the partners put together an HTA proposal to assess the effectiveness of community versus hospital eye service follow-up for patients with neovascular age related macular degeneration.
Impact An NIHR HTA Programme Clinical Evaluation and Trials proposal was accepted for funding and is currently being undertaken. Multidisciplinary collaboration involving: ophthalomology, health services research, health economics, epidemiology, vision and vascular science, qualitative research
Start Year 2012
 
Description Oral presentation at conference 
Form Of Engagement Activity Scientific meeting (conference/symposium etc.)
Part Of Official Scheme? No
Type Of Presentation Paper Presentation
Geographic Reach International
Primary Audience Other academic audiences (collaborators, peers etc.)
Results and Impact Mills N. Oral presentation at Society for Clinical Trials conference in Boston in May 2013 "An introduction to qualitative research methods in clinical trials" as part of an invited speaker session - "Can Qualitative Research Methods Revolutionize the Design and Conduct of Randomized Clinical Trials?"

Education impact at present with plans for a paper
Year(s) Of Engagement Activity 2013
 
Description Oral presentation at conference 
Form Of Engagement Activity Scientific meeting (conference/symposium etc.)
Part Of Official Scheme? No
Type Of Presentation Keynote/Invited Speaker
Geographic Reach International
Primary Audience Health professionals
Results and Impact Mills N. "The role of patient treatment preferences in improving trial recruitment: evidence from the ProtecT trial". British Thoracic Oncology Group; Dublin. Attended by over 400 delegates

Educational output at present
Year(s) Of Engagement Activity 2012
 
Description Workshop 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Type Of Presentation Workshop Facilitator
Geographic Reach National
Primary Audience Other academic audiences (collaborators, peers etc.)
Results and Impact Mills H, Heawood A, Donovan J. Organised and ran the course "How qualitative methods can contribute to the design and conduct of randomised trials - A University of Bristol School of Social and Community Medicine course in conjunction with ConDuCT Hub (20 March 2012 and 18 June 2013).

Educational impact at present
Year(s) Of Engagement Activity 2012,2013