Chapter 6 102 ABSTRACT Objectives To quantify and characterize the usage of expanded access data in National Institute for Health and Care Excellence (NICE) technology appraisals (TAs). Expanded access offers patients that are ineligible for clinical trials or registered treatment options access to investigational therapies. Although expanded access programs are increasingly used to collect real-world data (RWD), it is unknown if and how these data are used in NICE health technology assessments. Design Cross-sectional study of NICE appraisals (2010-2020). We automatically downloaded and screened all available appraisal documentation on NICE website (over 8,500 documents), searching for EA-related terms. Two reviewers independently labelled the expanded access usage by disease area, and whether it was used to inform safety, efficacy, and/or resource use. We qualitatively describe the five appraisals with the most occurrences of EA-related terms. Primary outcome measure Number of technology appraisals that used expanded access data to inform safety, efficacy and/or resource use analyses. Results In 54.2% (206/380 appraisals) at least one reference to expanded access was made. 21.1% (80/380) of the TAs used expanded access data to inform safety (n=43), efficacy (n=47) and/ or resource use (n=52). The number of TAs that utilize expanded access data remained stable over time, and the extent of expanded access data utilization varied by disease area (p=0.001). Conclusion NICE uses expanded access data in over one in five appraisals. In synthesis with evidence from well-controlled trials, data collected from expanded access programs may meaningfully inform cost-effectiveness modelling.
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