Informing efficient randomised controlled trials: Exploration of challenges in developing progression criteria for internal pilot studies

Objectives: Designing studies with an internal pilot phase may optimise the use of pilot work to inform more efficient randomised controlled trials (RCTs). Careful selection of preagreed decision or 'progression' criteria at the juncture between the internal pilot and main trial phases provides a valuable opportunity to evaluate the likely success of the main trial and optimise its design or, if necessary, to make the decision not to proceed with the main trial. Guidance on the appropriate selection and application of progression criteria is, however, lacking. This paper outlines the key issues to consider in the optimal development and review of operational progression criteria for RCTs with an internal pilot phase. Design: A structured literature review and exploration of stakeholders' opinions at a Medical Research Council (MRC) Hubs for Trials Methodology Research workshop. Key stakeholders included triallists, methodologists, statisticians and funders. Results: There is considerable variation in the use of progression criteria for RCTs with an internal pilot phase, although 3 common issues predominate: trial recruitment, protocol adherence and outcome data. Detailed and systematic reporting around the decisionmaking process for stopping, amending or proceeding to a main trial is uncommon, which may hamper understanding in the research community about the appropriate and optimal use of RCTs with an internal pilot phase. 10 top tips for the development, use and reporting of progression criteria for internal pilot studies are presented. Conclusions: Systematic and transparent reporting of the design, results and evaluation of internal pilot trials in the literature should be encouraged in order to facilitate understanding in the research community and to inform future trials

Video clinics versus standard face-to-face appointments for liver transplant patients in routine hospital outpatient care: study protocol for a pragmatic randomised evaluation of myVideoClinic.

BACKGROUND Video clinics, where patients can have a hospital appointment with their clinician from home, are emerging in practice, but their effectiveness is unclear. This study will evaluate whether a video clinic implemented at the University Hospitals Birmingham (UHB) NHS Foundation Trust improves patient satisfaction compared to standard face-to-face appointments for liver transplant patients. METHODS This will be a parallel, two-arm, statistician-blinded, randomised evaluation. Clinically stable liver patients at 1 to 5 years post-transplant (n = 180) will be randomised in equal numbers to video clinic appointments (intervention) or standard face-to-face appointments (control). The intervention group will have outpatient appointments from home via a secure video link accessed through the UHB patient portal. All patients will complete baseline questionnaires before randomisation and electronic follow-up questionnaires after each follow-up appointment during the subsequent 12 months. The primary outcome is the difference in scores between groups for three domains of patient satisfaction, namely 'convenience of location', 'getting through to the office by phone' and 'length of time waiting' (modified Visit-Specific Satisfaction Instrument). Secondary outcomes include quality of life (EQ-5D-5 L), costs, clinical contacts and user experience. Statistical analysis will be descriptive and performed on an intention-to-treat basis. The primary outcome will be analysed using baseline and 3-, 6-, 9- and 12-month questionnaires (according to patient follow-up appointment frequency) and comparisons made between study arms. A within-trial cost consequences analysis will be undertaken on the economic data. Patients (n = 8), carers/family members (n = 6) and health professionals (n = 14) will be interviewed about the experience of video clinics and the interviews will be analysed using thematic analysis. DISCUSSION This study will allow an in depth understanding of whether video clinics can improve patient satisfaction with their care. In addition, the intervention could save patients time and costs, removing the need to travel long distances for outpatient appointments. Video clinics may be applicable to a wide range of other clinical settings and health conditions. The study has been approved by the NHS Health Research Authority and a National Research Ethics Committee (Ref: 17/WM/0338) and research governance approval has been obtained from UHB (Ref: RRK6080). TRIAL REGISTRATION ISRCTN: 14093266 (25/03/2018; retrospectively registered)