Results from expanded access programs: a review of academic literature 143 7♦ Eligibility criteria We will include publications where the main data source or the main theme is expanded access (data). These must have been published between 01-01-2000 and 01-01-2022. Methods The study will be conducted via Rayyan. All records will be assessed by two reviewers independently. If there is disagreement, a third reviewer and potentially fourth reviewer will assess the records. The researchers will be assigned random records. TBP is expected to review all records at least once. Search strategy We will search through PubMed for all articles with expanded access terms.2 We will exclude all articles published prior to 1st of January 2000 and after 1st of January 2022. PUBMED: ‘compassionate use trials’[MeSH Terms] OR ‘expanded access’[All Fields] OR ‘early access’[All Fields] OR ‘managed access’[All Fields] OR ‘special access’[All Fields] OR ‘named patient’[All Fields] OR ‘single patient IND’[All Fields] OR ‘compassionate use’[All Fields] OR ‘compassionate study’[All Fields] OR ‘pre approval access’[All Fields] Study/Source of Evidence selection Following the search, all identified citations will be collated and uploaded into EndNote Version 19 (Clarivate, London, UK) and duplicates removed. Following a pilot test with 50 records, titles and abstracts will then be screened by two or more independent reviewers for assessment against the inclusion criteria for the review. The full text of selected citations will be assessed in detail against the inclusion criteria by two or more independent reviewers. Reasons for exclusion of sources of evidence at full text that do not meet the inclusion criteria will be recorded and reported in the scoping review. Any disagreements that arise between the reviewers at each stage of the selection process will be resolved through discussion and an independent additional review of a third reviewer. We will also conduct a random sample of 100 articles that will be checked by an additional reviewer (e.g., third or fourth). Inclusion We will include all articles with compassionate use as main theme, such as disseminations of individual expanded access programs or named-patient cases, or overview of regulatory hurdles.
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