Chapter 7 128 METHODS We conducted a review of all publications indexed in MEDLINE through PubMed that report original results of expanded access usage. We included all peer-reviewed literature that was published between the 1st of January 2000 and the 1st of January 2022.All articles that included any term related to expanded access (e.g., compassionate use, pre-approval access, managed access, special access) were considered.2 We relied on the self-assessed classification of expanded access by the authors and removed all instances with an erroneous expanded access classification, e.g., off-label use and clinical trials, where possible. The detailed search protocol is available in the Supplementary Material. Citation and review management All citations were exported from PubMed in EndNote Version 19 (Clarivate, London, United Kingdom), where duplicates and publications without full text were detected and removed. Citations were subsequently uploaded in Rayyan, an online systematic review platform.68 TBP, DGJC, NA, and SSA independently conducted the review – all records were reviewed at least twice. A random sample of 100 articles was additionally assessed by a third independent reviewer. Eligibility, screening, and labeling Based on the titles and abstract, we labelled articles for ‘inclusion’, ‘exclusion’, or further investigation (‘unknown’). Articles labelled ‘unknown’ or where reviewers disagreed on inclusion/ exclusion were further assessed by reading the full text. If the third reviewer was unsure, remaining disagreement was solved through discussion of the full text with a fourth author. Exclusion reasons included: 1. Non-English literature 2. Not relevant (topic is not expanded access, e.g., off-label use or formal clinical trials) 3. Not primary research • Errata, editorials, replies • News articles • Meta-analyses, guidelines, systematic reviews 4. Not disseminating investigational results of pharmaceutical therapeutics • Devices, procedures • Other research topics (e.g., legal/ethical/policy issues)
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