OBJECTIVES: Clinical trial design is an important part of robust evidence generation for pharmaceutical manufacturers submitting a health technology assessment (HTA). Factors such as randomisation, blinding, and comparator selection are typically considered fundamental, yet clinical trial site locations are not known to drive HTA decision making, despite being discussed anecdotally. The UK is widely considered a hub of clinical research, with UK-based clinical trial sites allowing early patient access to medical innovation. Considering the regulatory uncertainties arising from BREXIT on the operation of UK trial sites, and because the National Institute for Health and Care Excellence (NICE) does not explicitly call for mandatory UK-based research, this study was conducted to investigate the benefit of UK-based trial sites in securing a positive NICE HTA decision.

METHODS: This study was executed through statistical and machine learning analysis of 380 historical NICE appraisals dating back to 2011 summarised in IQVIA proprietary data. The rate of NICE HTA decisions (positive, optimised, negative) was first analysed descriptively for appraisals with and without UK-based trial sites, and then by machine learning analysis for the importance and direction of influence of UK-based trial sites on the prediction of NICE decision.

RESULTS: Analysis identified a higher rate of positive decision making for technologies that among other factors submitted to NICE with UK-based clinical research (34.4 vs 26.7%, p=0.012). Machine learning analysis confirmed these results, with UK-based clinical research shown to have a small but consistently positive influence on the prediction of positive NICE HTA decision in a Random Forest model.

CONCLUSIONS: Despite not being a driving factor, these findings demonstrate the correlation of UK clinical trial sites to NICE HTA decision making, and in doing so highlight the importance of ensuring that UK sites continue to be selected for pivotal clinical trials in future.