Core outcome measures in effectiveness trials

Non-responders will not be invited to participate in round two. Descriptive statistics will summarise the results of round one, including the percentage of participants scoring the outcome at each possible response from one to nine. Any additional outcomes identified by participants that are deemed to represent a new outcome by the researchers TL and SM will be included for round two, and any uncertainties will be discussed with the study advisory group.

All outcomes will be carried forward to round two. Participants will complete round two online. All participants will be reminded of their own round one scores. Participants will be randomised to one of three groups as follows:. Group one consisting of a third of the patient stakeholder group and a third of the healthcare professional stakeholder group, randomly allocated will be shown the proportion of people in their own stakeholder group choosing each score from one to nine.

Participants will be asked to re-score the outcomes. All outcomes from round one will be carried forward. The proportion of participants scoring the outcome at each possible response from one to nine will be used to summarise round two. All outcomes will be carried forward to round three. Round three will also be completed online.

All participants will be reminded of their round two scores. If response rates number at least 10 per stakeholder group per arm, participants will be retained in their randomised feedback groups one, two, and three, and will only have access to the feedback scores from others randomised to their group. If response rates are poor then the randomised groups one, two, and three will be shown feedback from all other participants, regardless of which group those participants were randomised to.

This will be done in order to maximise information. For each outcome presented in round three, the proportion of participants from each stakeholder group scoring one to three, four to six, and seven to nine on the nine-point Likert scale will be calculated for each item regardless of randomised group.

Research question: Can we derive a core outcome set from the two sets of key outcomes from the stakeholder groups? A consensus meeting with key stakeholders patients and healthcare professionals will be conducted at the end of the Delphi process. The participants for the consensus group meeting will be purposively sampled to ensure a range of views of men who have had the various treatments and the health professionals that administer them are represented.

The sample for both stakeholder groups will be drawn from those who completed all rounds of the Delphi study. The objective of the consensus meeting is to discuss outcomes about which there was disagreement in round three of the Delphi study, and to validate and agree on a final list of outcomes which will constitute the core outcome set. A secondary objective is to explore how treatment type may affect outcomes regarded as important by patients. There is no published core outcome set for localised prostate cancer effectiveness trials which has been developed in conjunction with key stakeholders using robust, standardised, and transparent methodology.

An outcome set for routine hospital data collection, focussed on comparability across institutions and individual clinicians, with a view toward lowering costs has been developed by ICHOM [ 38 , 39 ]. The present research will standardise core outcome definition, collection, measurement, analysis, and interpretation in effectiveness trials for localised prostate cancer. It will propose a core outcome set recommending what outcomes should be measured or at least considered in comparative clinical effectiveness trials.

However, importantly, trialists may supplement the core outcomes set with other outcomes, because the set represents a minimum. In addition, there may be situations where the core outcome set may not be relevant, especially for studies with certain specific or focused objectives for example, a trial designed to ascertain hernia rates via different surgical interventions, or different vesico-urethral anastomotic suturing techniques on duration of drainage.

Nevertheless, even under such circumstances, there is an argument for such researchers to consider if the interventions being assessed are likely to have any impact on core outcomes, and if so, then to consider measuring the relevant ones. This research will benefit all stakeholders involved in prostate cancer management in Scotland, the United Kingdom, and beyond.

Specifically, the study will have the following long-term benefits:. Clinicians will be better equipped to provide informed consent and facilitate decision-making by patients, and be able to foster improvements in clinical governance and in the design of decision aids. The NHS will be better equipped to prioritise funding of localised prostate cancer treatments that reflects the needs of patients.

Researchers will be able to design trials and synthesise evidence which address the most important outcomes to all stakeholders, hence encouraging participation of clinicians and patients in clinical trials. In addition, the output will serve as a platform to develop patient-reported core outcome measures for use in clinical trials of interventions for localised prostate cancer and in clinical practice.

The methodology developed will also serve as a model for the development of core outcome measures in other urological conditions and across other surgical specialities. The inclusion of key stakeholder groups in all processes of the research ensures that the core outcome set will be relevant to these groups and accepted as useful in future research.

The long-term objective is to develop and validate a universal set of core outcome measures relevant to all interventions for all urological cancers. Participant recruitment for the Delphi study started in December Additional file 1: 35K, pdf Localised prostate cancer care pathway. Competing interests. SM is the research fellow for the project and has been involved in all stages of study design, data collection, data synthesis, and is responsible for the management of the project.

HB was involved in abstract screening, full text screening and data extraction for the systematic review, and commented on drafts of the manuscript. PW is a member of the study advisory group and had input into most aspects of the study design, particularly the Delphi study and nested RCT, she also provided supervision and support for the development of the online Delphi questionnaire.

She commented on drafts of the manuscript. MC is a member of the study advisory group and had input into most aspects of the study design, particularly the Delphi study and RCT, she also commented on drafts of the manuscript.

FS carried out the searches and paper retrievals for the systematic review. SJM offered supervision and advice on the qualitative aspects of the study and commented on drafts of the manuscript. JND is a clinical content expert and offered advice on any clinical aspects of the study and assisted in recruitment of healthcare professionals for the Delphi study. TBLL is the principal investigator and grant holder for the study.

He conceived the idea for the project and supervises and is involved in and is ultimately responsible for all aspects of the design and execution of the research.

He commented on drafts of the manuscript. All authors read and approved the manuscript. Steven MacLennan, Email: ku. Hendrika J Bekema, Email: moc. Paula R Williamson, Email: ku. Marion K Campbell, Email: ku. Fiona Stewart, Email: ku. Sara J MacLennan, Email: ku. Thomas BL Lam, Email: ku. National Center for Biotechnology Information , U. Journal List Trials v. Published online Mar 4.

Author information Article notes Copyright and License information Disclaimer. Corresponding author. Received Nov 6; Accepted Feb This article has been cited by other articles in PMC. Abstract Background Prostate cancer is a growing health problem worldwide. Discussion This study will inform clinical practice and future trials of interventions of localised prostate cancer by standardising a core outcome set which should be considered in comparative effectiveness studies for localised prostate cancer.

Electronic supplementary material The online version of this article doi Keywords: Core outcome set, Systematic review, Semi-structured interviews, Delphi, Consensus methods, Treatment for localised prostate cancer.

Background Prostate cancer is the second most common male cancer worldwide an estimated 1. Aims and objectives Aim The overall aim of this project is to develop a core outcome set for localised prostate cancer effectiveness trials which recommends what outcomes should be measured, and reflects the interests of patients and healthcare professionals, in order to facilitate decision-making.

Objectives The specific objectives are: 1 to identify a list of outcomes from published studies reporting on any therapeutic intervention for localised prostate cancer; 2 to determine the heterogeneity of outcome definitions, and the number of different measuring instruments used and the specific ways in which they differ; 3 to identify a list of potentially important outcomes reported by men who have been treated for localised prostate cancer in order to augment the list generated from 1 ; 4 to prioritise and reach consensus regarding the most important outcomes from the perspective of patients and healthcare professionals into a core outcome set; and 5 to investigate how differences in Delphi study outcome scoring might be affected by the feedback participants have access to in order to assist the design of future Delphi studies.

Phase one: generation of a list containing all possible relevant outcomes Systematic review Research question: What are the outcomes reported in studies assessing the effectiveness of interventions for localised prostate cancer?

Study overview and method This study will consist of a systematic review of studies of the effects of surgery open, laparoscopic, and robotic-assisted radical prostatectomy , radiotherapy, brachytherapy, and active monitoring for localised prostate cancer.

Types of studies We intend to limit included studies to those that are likely to influence clinical practice. Types of interventions Interventions considered for this review are: surgery open, laparoscopic, or robotic, incorporating any approach and technique ; external beam radiotherapy EBRT , three-dimensional conformal radiotherapy 3D-CRT , or intensity modulated radiotherapy IMRT , incorporating any dose or schedule; brachytherapy permanent or temporary seed implantation, incorporating any dose or schedule ; cryotherapy; high intensity focussed ultrasound HIFU active surveillance or monitoring; and watchful waiting or observation.

Types of participants The participants are men of any age with clinically localised prostate cancer defined as cTc N0 M0, according to the TNM classification of malignant tumours [ 42 ]. Data analysis and presentation The data will be entered into Microsoft Excel in order to aid tabulation and analysis. Semi-structured interviews with individual patients Research question: What are the outcomes patients regard as potentially important following treatment for localised prostate cancer?

Study overview and method Semi-structured interviews with patients who have had treatment for localised prostate cancer. Analysis of semi-structured interviews Audio recordings of the semi-structured interviews will be fully transcribed, stored, and analysed using NVivo10 software QSR International, Burlington, Massachusetts, United States and themes will be derived from issues raised by participants.

Consultation exercise Research question: From the list of outcomes identified from the systematic review and semi-structured interviews with patients, what are the outcomes that should be entered into phase two for further study? Study overview and method Consultation exercise with patients. Phase two: prioritisation of important outcomes from each stakeholder group and integration of outcomes into a core outcome set Research question: What are the most important outcomes for each key stakeholder group?

Study overview A survey of key stakeholder opinions using Delphi methodology will be conducted. Study method Delphi study round one Email addresses for potential participants will be collected by the research team via contacting prostate cancer specific patient-led support groups and charities throughout the United Kingdom for the patient group , and national and international professional bodies such as BAUS and the European Association of Urologists EAU for the healthcare professionals group.

Analysis of Delphi study round one Descriptive statistics will summarise the results of round one, including the percentage of participants scoring the outcome at each possible response from one to nine. Delphi study round two Participants will complete round two online. Participants will be randomised to one of three groups as follows: Group one consisting of a third of the patient stakeholder group and a third of the healthcare professional stakeholder group, randomly allocated will be shown the proportion of people in their own stakeholder group choosing each score from one to nine.

Analysis of Delphi study round two The proportion of participants scoring the outcome at each possible response from one to nine will be used to summarise round two. Delphi study round three Round three will also be completed online.

Analysis of Delphi study round three For each outcome presented in round three, the proportion of participants from each stakeholder group scoring one to three, four to six, and seven to nine on the nine-point Likert scale will be calculated for each item regardless of randomised group.

Consensus group meeting Research question: Can we derive a core outcome set from the two sets of key outcomes from the stakeholder groups? Study overview A consensus meeting with key stakeholders patients and healthcare professionals will be conducted at the end of the Delphi process. Discussion There is no published core outcome set for localised prostate cancer effectiveness trials which has been developed in conjunction with key stakeholders using robust, standardised, and transparent methodology.

Trial status Participant recruitment for the Delphi study started in December Additional file Additional file 1: 35K, pdf Localised prostate cancer care pathway. Footnotes Competing interests The authors declare they have no competing interests. References 1. The existence or use of a core outcome set does not imply that outcomes in a particular study should be restricted to those in the relevant core outcome set.

Rather, there is an expectation that the core outcomes will be collected and reported, making it easier for the results of studies to be compared, contrasted and combined as appropriate; while researchers continue to explore other outcomes as well. COMET aims to collate and stimulate relevant resources, both applied and methodological, to facilitate exchange of ideas and information, and to foster methodological research in this area.

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Correspondence to Cecilia A C Prinsen. CT is the principle investigator. CP is the coordinating investigator. CT and CP are responsible for the design of the study and the study protocol. SKJ is responsible for the search of the literature review. CP is responsible for drafting the protocol manuscript. All authors have read and approved the final protocol manuscript. This article is published under license to BioMed Central Ltd. Reprints and Permissions. Prinsen, C. Trials 15, Download citation.

Received : 05 March Accepted : 02 June Published : 25 June Anyone you share the following link with will be able to read this content:. Sorry, a shareable link is not currently available for this article. Provided by the Springer Nature SharedIt content-sharing initiative. Skip to main content.

Search all BMC articles Search. Download PDF. Discussion Since the Delphi method allows a large group of international experts to participate, we consider it to be the preferred consensus-based method for our study.

Background There is a lack of consensus with regard to the selection of outcomes and outcome measurement instruments for clinical trials. Figure 1. Overview of the Delphi method. Full size image. Table 1 Search strategy Full size table. Discussion When insufficient or contradictory information is available on a certain topic, consensus-based methods such as a Delphi method are generally considered to be appropriate methods to determine the extent to which experts agree on that topic.

Trial status The Delphi study is currently ongoing. References 1. Article PubMed Google Scholar 9. Article PubMed Google Scholar Article Google Scholar Article Google Scholar Download references. View author publications. Additional information Competing interests The authors declare that they have no competing interests. About this article. Cite this article Prinsen, C. Copy to clipboard. Trials ISSN: Contact us Submission enquiries: Access here and click Contact Us General enquiries: info biomedcentral.