Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals 105 6♦ Records identi ed through the NICE database (N=496) ‘Expanded Access term’ present in documentation (N=206) No ‘Expanded Access term’ present in documentation (N=174) EA data not used for safety, e cacy and/or resource use in appraisal (N=126) Records screened (N=380) - STA (n=334) - MTA (n=33) - HST (n=13) EA data used to support appraisal (N=79) - Safety (n=43) - E cacy (n=47) - Resource use (n=52) Records excluded (N=116) - Replaced (n=45) - Terminated (n=57) - Withdrawn (n=14) Figure 5: Screening and selection of technology appraisals from NICE. STA, single technology appraisal. MTA, multiple technology appraisals. HST, highly specialized technology. EA, expanded access. NICE, National Institute for Health and Care Excellence We wrote a computer script (i.e., a web scraper)50 to automatically list and download all documentation (e.g., manufacturer submissions, ERG report, final appraisal determination) available through NICE’s website. Subsequently, the script extracted the text from these documents and automatically screened whether the text contained ‘expanded access (EA) terms’, like ‘Compassionate Use’,‘Expanded Access’‘Early Access’, etcetera, as well as all possible spellings thereof. A detailed protocol, including all search terms, is available in the Supplementary Material. The data and code from the paper are available on the GitHub from the first author, https:// github.com/TobiasPolak. When at least one of these ‘expanded access terms’ was present, two authors (T.B.P. and D.G.J.C.) independently and manually, reviewed the context of the term. We primarily labelled the data usage with one or more of the following categories: 1. Safety: expanded access data were used to evaluate the safety profile 2. Efficacy: expanded access data were used to evaluate the efficacy profile
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