Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals 107 6♦ 3 14 3 15 3 17 18 3 16 5 28 16 33 13 38 17 37 12 41 5 35 0 20 40 60 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 Year Number of technology appraisals Appraisal included EA data No Yes Technology appraisals using Expanded Access data Efficacy Resource Use Safety 21 3 9 20 8 8 11 A B Figure 6: Technology appraisals using expanded access (EA) data to support safety, efficacy and/or resource use. A: Venn-diagram displaying the overlap of safety, efficacy, and/or resource use labelling of technology appraisals. B: Bar chart of technology appraisals published between January 1st 2010, and January 1st 2021 that did (‘Yes’) or did not (‘No’) include data expanded access programs to support safety, efficacy and/or resource use. Although there is a significant increase over time in the absolute use of expanded access data by payers (ρ = 0.73 and p = 0.011; Figure 6B), there is no evidence of a significant increase in use of expanded access data over time relative to the total number of appraisals conducted (ρ = 0.32 and p = 0.332). Significant differences (χ2= 38.8, p = 0.001) exist in the disease areas that did versus those that did not include expanded access data. Oncology and hematology together account for 66% of the appraisals with expanded access data, whereas they make up 50% of the entire fraction of
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