Correction to: Economic Evaluations Alongside Efficient Study Designs Using Large Observational Datasets: the PLEASANT Trial Case Study.

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Economic Evaluations Alongside Efficient Study Designs Using Large Observational Datasets: the PLEASANT Trial Case Study.

BACKGROUND: Large observational datasets such as Clinical Practice Research Datalink (CPRD) provide opportunities to conduct clinical studies and economic evaluations with efficient designs. OBJECTIVES: Our objectives were to report the economic evaluation methodology for a cluster randomised controlled trial (RCT) of a UK NHS-delivered public health intervention for children with asthma that was evaluated using CPRD and describe the impact of this methodology on results. METHODS: CPRD identified eligible patients using predefined asthma diagnostic codes and captured 1-year pre- and post-intervention healthcare contacts (August 2012 to July 2014). Quality-adjusted life-years (QALYs) 4 months post-intervention were estimated by assigning utility values to exacerbation-related contacts; a systematic review identified these utility values because preference-based outcome measures were not collected. Bootstrapped costs were evaluated 12 months post-intervention, both with 1-year regression-based baseline adjustment (BA) and without BA (observed). RESULTS: Of 12,179 patients recruited, 8190 (intervention 3641; control 4549) were evaluated in the primary analysis, which included patients who received the protocol-defined intervention and for whom CPRD data were available. The intervention's per-patient incremental QALY loss was 0.00017 (bias-corrected and accelerated 95% confidence intervals [BCa 95% CI] -0.00051 to 0.00018) and cost savings were £14.74 (observed; BCa 95% CI -75.86 to 45.19) or £36.07 (BA; BCa 95% CI -77.11 to 9.67), respectively. The probability of cost savings was much higher when accounting for BA versus observed costs due to baseline cost differences between trial arms (96.3 vs. 67.3%, respectively). CONCLUSION: Economic evaluations using data from a large observational database without any primary data collection is feasible, informative and potentially efficient. Clinical Trials Registration Number: ISRCTN03000938.

PLEASANT: Preventing and Lessening Exacerbations of Asthma in School-age children Associated with a New Term - a cluster randomised controlled trial and economic evaluation.

BACKGROUND: Asthma episodes and deaths are known to be seasonal. A number of reports have shown peaks in asthma episodes in school-aged children associated with the return to school following the summer vacation. A fall in prescription collection in the month of August has been observed, and was associated with an increase in the number of unscheduled contacts after the return to school in September. OBJECTIVE: The primary objective of the study was to assess whether or not a NHS-delivered public health intervention reduces the September peak in unscheduled medical contacts. DESIGN: Cluster randomised trial, with the unit of randomisation being 142 NHS general practices, and trial-based economic evaluation. SETTING: Primary care. INTERVENTION: A letter sent (n = 70 practices) in July from their general practitioner (GP) to parents/carers of school-aged children with asthma to remind them of the importance of taking their medication, and to ensure that they have sufficient medication prior to the start of the new school year in September. The control group received usual care. MAIN OUTCOME MEASURES: The primary outcome measure was the proportion of children aged 5-16 years who had an unscheduled medical contact in September 2013. Supporting end points included the proportion of children who collected prescriptions in August 2013 and unscheduled contacts through the following 12 months. Economic end points were quality-adjusted life-years (QALYs) gained and costs from an NHS and Personal Social Services perspective. RESULTS: There is no evidence of effect in terms of unscheduled contacts in September. Among children aged 5-16 years, the odds ratio (OR) was 1.09 [95% confidence interval (CI) 0.96 to 1.25] against the intervention. The intervention did increase the proportion of children collecting a prescription in August (OR 1.43, 95% CI 1.24 to 1.64) as well as scheduled contacts in the same month (OR 1.13, 95% CI 0.84 to 1.52). For the wider time intervals (September-December 2013 and September-August 2014), there is weak evidence of the intervention reducing unscheduled contacts. The intervention did not reduce unscheduled care in September, although it succeeded in increasing the proportion of children collecting prescriptions in August as well as having scheduled contacts in the same month. These unscheduled contacts in September could be a result of the intervention, as GPs may have wanted to see patients before issuing a prescription. The economic analysis estimated a high probability that the intervention was cost-saving, for baseline-adjusted costs, across both base-case and sensitivity analyses. There was no increase in QALYs. LIMITATION: The use of routine data led to uncertainty in the coding of medical contacts. The uncertainty was mitigated by advice from a GP adjudication panel. CONCLUSIONS: The intervention did not reduce unscheduled care in September, although it succeeded in increasing the proportion of children both collecting prescriptions and having scheduled contacts in August. After September there is weak evidence in favour of the intervention. The intervention had a favourable impact on costs but did not demonstrate any impact on QALYs. The results of the trial indicate that further work is required on assessing and understanding adherence, both in terms of using routine data to make quantitative assessments, and through additional qualitative interviews with key stakeholders such as practice nurses, GPs and a wider group of children with asthma. TRIAL REGISTRATION: Current Controlled Trials ISRCTN03000938. FUNDING DETAILS: This project was funded by the NIHR Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 20, No. 93. See the HTA programme website for further project information.