The UK's Early Access to Medicines Scheme 10 years on: an evaluation using publicly available data

ObjectivesTo 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.DesignAnalysis of publicly available data: MHRA Public Assessment Reports; Electronic Medicines Compendium database; interactive Drug Analysis Profiles database; Eudravigilance database.SettingUnited Kingdom.ParticipantsThe 51 ‘scientific opinions’ available on the MHRA website in June 2024.Main Outcome MeasuresPublic Assessment Reports, pharmacovigilance data.ResultsAfter 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.ConclusionsWe 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.