Development and validation of a follow-up methodology for a randomised controlled trial, utilising routine clinical data as an alternative to traditional designs: a pilot study to assess the feasibility of use for the BladderPath trial

Pilot and Feasibility Studies

Ana Hughes¹  Richard T. Bryan¹  Ann M. Pope¹  Nicholas D. James²  Alicia Jakeman³  Prashant Patel⁴  Amandeep Dosanjh⁴  Helen M. Parsons⁵  Harriet P. Mintz⁶ 

[1] Institute of Cancer and Genomic Sciences, University of Birmingham, Edgbaston, B15 2TT, Birmingham, UK;The Institute of Cancer Research, 237 Fulham Road, SW3 6JB, London, UK;The Royal Marsden NHS foundation Trust, Fulham Road, Chelsea, SW3 6JJ, London, UK;University Hospitals Birmingham NHS Foundation Trust, Edgbaston, B15 2GW, Birmingham, UK;University Hospitals Birmingham NHS Foundation Trust, Edgbaston, B15 2GW, Birmingham, UK;Institute of Cancer and Genomic Sciences, University of Birmingham, Edgbaston, B15 2TT, Birmingham, UK;Warwick Medical School, University of Warwick, CV4 7AL, Coventry, UK;Warwick Medical School, University of Warwick, CV4 7AL, Coventry, UK;University Hospitals Birmingham NHS Foundation Trust, Edgbaston, B15 2GW, Birmingham, UK;

关键词: Bladder cancer;    Routine;    Administrative;    Data;    Outcomes;    Events;    Follow-up;    Randomised controlled trial (RCT);    BladderPath;    Hospital episode statistics (HES);   

DOI  :  10.1186/s40814-020-00713-y

来源: Springer

【 摘 要 】

BackgroundBladder cancer outcomes have not changed significantly in 30 years; the BladderPath trial (Image Directed Redesign of Bladder Cancer Treatment Pathway, ISRCTN35296862) proposes to evaluate a modified pathway for diagnosis and treatment ensuring appropriate pathways are undertaken earlier to improve outcomes. We are piloting a novel data collection technique based on routine National Health Service (NHS) data, with no traditional patient-Health Care Professional contact after recruitment, where trial data are traditionally collected on case report forms. Data will be collected from routine administrative sources and validated via data queries to sites. We report here the feasibility and pre-trial methodological development and validation of the schema proposed for BladderPath.MethodsLocally treated patient cohorts were utilised for routine data validation (hospital interactions data (HID) and administrative radiotherapy department data (RTD)). Single site events of interest were algorithmically extracted from the 2008–2018 HID and validated against reference datasets to determine detection sensitivity. Survival analysis was performed using RTD and HID data. Hazard ratios and survival statistics were calculated estimating treatment effects and further validating and assessing the scope of routine data.ResultsOverall, 829/1042 (sensitivity 0.80) events of interest were identified in the HID, with varying levels of sensitivity; identifying, 202/206 (sensitivity 0.98; PPV 0.96) surgical events but only 391/568 (sensitivity 0.69; PPV 0.95) radiotherapy regimens. An overall temporal quality improvement trend was present: detecting 41/117 events (35%) in 2011 to 104/109 (95%) in 2017 (all event types). Using the RTD, 5-year survival rates were 43% (95% CI 25–59%) in the chemoradiotherapy group and 30% (95% CI 23–36%) in the radiotherapy group; using the HID, the 5-year radical cystectomy survival rate was 57% (95% CI 50–63%).ConclusionsRoutine data are a feasible method for trial data collection. As long as events of interest are pre-validated, very high sensitivities for trial conduct can be achieved and further improved with targeted data queries. Outcomes can also be produced comparable to clinical trial and national dataset results. Given the real-time, obligatory nature of the HID, which forms the Hospital Episode Statistics (HES) data, alongside other datasets, we believe routine data extraction and validation is a robust way of rapidly collecting datasets for trials.

【 授权许可】

CC BY