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

Objectives: 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.

Design: Analysis of publicly available data: MHRA Public Assessment Reports; Electronic Medicines Compendium database; interactive Drug Analysis Profiles database; Eudravigilance database.

Setting: United Kingdom.

Participants: The 51 'scientific opinions' available on the MHRA website in June 2024.

Main outcome measures: Public Assessment Reports, pharmacovigilance data.

Results: 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.

Conclusions: 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.