Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals 115 6♦ interpreted as a lower bound of expanded access use in NICE appraisals. Lastly, we were unable to exactly quantify the added value of expanded access data. As we lack a counterfactual, we do not know what would have happened without the inclusion of expanded access data.Additionally, it is difficult to measure the impact of expanded access data, as it is not always clear how these data have exactly been used: the use of expanded access data – and the appraisal thereof – in HTA by the manufacturer, ERG or NICE committee are difficult to quantify due the complexity and extent of the discussions described in the documentation. Although we have provided the reader with both high-level quantitative statistics and with illustrative qualitative examples from our data set, future research could attempt to systematically analyze these topics. Conclusion Expanded access data are used in over one in five (21.1%) NICE appraisals, and this number appears to remain stable over time. In general, adding data from expanded access can yield more real-world information. Especially to estimate the resource use, pre-approval expanded access data can play a vital role informing post-approval real-world usage. In synthesis with evidence from well-controlled regulatory studies, data collected from expanded access programs may meaningfully inform NICE decision making. Further research is required to understand when expanded access data can and should be included in health technology assessments.
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