Chapter 6 106 3. Resource use: expanded access data were used to inform cost parameters 4. Trivial: expanded access data were not used or trivially mentioned in the appraisal Patients and physicians also share their treatment experience. As the impact of these accounts is harder to quantify, we did not include them in our main analysis but secondarily labelled: 1. Treatment experience: When patients or physicians cited experience within the expanded access program. Discordance was resolved by discussion between the two reviewers. To give the reader a sense of these different types of usage, examples are provided in the Results section. Additionally, we provide a narrative summary of the five appraisals that contain the most occurrences of the search terms to illustrate the use of expanded access data qualitatively. Lastly, TAs were classified as single technology appraisal (STA), multiple technology appraisal (MTA), or highly specialized technology (HST). All TAs were categorized according to their area of disease. Statistics The Spearman rank correlation test was used to detect time trends in the yearly number of appraisals using expanded access data. We performed a Pearson chi-square test to assess whether the proportion of appraisals that included expanded access data differed by disease area. For all significance testing, we set the 2-sided significance level at 0.05. RESULTS We screened all 496 TAs conducted between January 1st, 2010 and January 1st, 2021. This ranged from Technology Appraisal 185 (TA185) to TA667 and from Highly Specialized Technology 1 (HST1) to HST13. N=116 appraisals were excluded (for details, see Figure 5). The remaining 380 appraisals had 8,925 documents that were downloaded and screened. In 54.2% (206 of 380 appraisals) at least one reference to expanded access was made. In total, 80 out of 380 (21.1%) of the TAs used expanded access data to inform safety (n=43), efficacy (n=47) or resource use (n=52). As a single TA could have multiple labels, there is overlap between safety, efficacy and resource use. This is depicted in Figure 6A. Additionally, in 54 appraisals (14.5%) the expanded access program was cited by patients or physicians as treatment experience.
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