{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,14]],"date-time":"2026-05-14T14:27:25Z","timestamp":1778768845211,"version":"3.51.4"},"reference-count":52,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2025,2,1]],"date-time":"2025-02-01T00:00:00Z","timestamp":1738368000000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["JRSM Open"],"published-print":{"date-parts":[[2025,2]]},"abstract":"<jats:sec><jats:title>Objectives<\/jats:title><jats:p> To investigate the drugs and indications that have passed through the UK's Early Access to Medicines Scheme (EAMS) to date, the type of evidence the regulator considers when accepting a drug into the EAMS, and potential risks to patients. <\/jats:p><\/jats:sec><jats:sec><jats:title>Design<\/jats:title><jats:p> Analysis of publicly available data: MHRA Public Assessment Reports; Electronic Medicines Compendium database; interactive Drug Analysis Profiles database; Eudravigilance database. <\/jats:p><\/jats:sec><jats:sec><jats:title>Setting<\/jats:title><jats:p> United Kingdom. <\/jats:p><\/jats:sec><jats:sec><jats:title>Participants<\/jats:title><jats:p> The 51 \u2018scientific opinions\u2019 available on the MHRA website in June 2024. <\/jats:p><\/jats:sec><jats:sec><jats:title>Main Outcome Measures<\/jats:title><jats:p> Public Assessment Reports, pharmacovigilance data. <\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p> After exclusions, there were 48 EAMS submissions, consisting of 48 indications and 32 drugs. 60% of indications were for cancer. Only 7% of EAMS submissions were based on double-blind, placebo-controlled randomised trials. The average sample size of studies conducted for the EAMS was 654. Most studies used surrogate (76%) and\/or survival (57%) outcomes. Only 17% used subjective outcomes. For 17% of the indications, no ongoing studies were being conducted. Animal studies were conducted preclinically for all drugs and 35% also conducted in vitro studies. 47% of the drugs had elevated rates of suspected adverse reaction reports according to pharmacovigilance data. <\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p> We recommend that the EAMS drugs with elevated reporting rates are reviewed, that future studies of EAMS drugs use patient-centred outcomes, that preclinical studies make greater use of human biology-based approaches, that post-approval trials are conducted, and that future reviews of the EAMS centre the experience of patients. <\/jats:p><\/jats:sec>","DOI":"10.1177\/20542704251317916","type":"journal-article","created":{"date-parts":[[2025,2,24]],"date-time":"2025-02-24T08:46:52Z","timestamp":1740386812000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":1,"title":["The UK's Early Access to Medicines Scheme 10 years on: an evaluation using publicly available data"],"prefix":"10.1177","volume":"16","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6885-6401","authenticated-orcid":false,"given":"Pandora","family":"Pound","sequence":"first","affiliation":[{"name":"Safer Medicines Trust, Kingsbridge, TQ7 9AX, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9269-3841","authenticated-orcid":false,"given":"Rebecca","family":"Ram","sequence":"additional","affiliation":[{"name":"Safer Medicines Trust, Kingsbridge, TQ7 9AX, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-0650-5985","authenticated-orcid":false,"given":"Kathy","family":"Archibald","sequence":"additional","affiliation":[{"name":"Safer Medicines Trust, Kingsbridge, TQ7 9AX, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2025,2,24]]},"reference":[{"key":"bibr1-20542704251317916","unstructured":"MHRA. 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