OBJECTIVES To identify, map and analyse current policies and practices of national accelerated access schemes from an international and comparative perspective and explore their implications for coverage and access.

METHODS An extensive mapping exercise was carried out to map early access pathways across 23 countries globally using both primary and secondary data. First, a comprehensive literature review was undertaken to identify national early access policies relating to the unauthorised use of medicines. Medline®, Web of Science, SCOPUS, and Google Scholar were searched for peer-reviewed literature. National health organisation websites and Google were searched for grey literature. Results were then validated with key stakeholders from Health Technology Assessment (HTA)/insurance organisations. Stakeholders were asked to provide feedback on eligibility criteria, evidence requirements, funding arrangements, and monitoring requirements.

RESULTS Across N=23 countries, 88% offer access to medicines prior to marketing authorisation (MA): a) Most countries have access pathways for both individually named-patients and for defined cohorts of patients; b) The majority of countries require that a drug treats, diagnoses or prevents a serious condition; c) Many countries require completion of phase II trials at a minimum. Variations in eligibility criteria, evidence requirements, and funding arrangements result in differences in both the availability and time to availability for medicines across settings prior to marketing authorisation. While national early access programmes may reduce evidence thresholds for access to small cohorts of patients prior to MA, HTA agencies still require robust clinical and economic evidence to approve products post-MA.

CONCLUSIONS While the majority of countries have early access schemes for unauthorised medicines, the eligibility criteria, evidence thresholds, and funding arrangements vary across settings. While these mechanisms enable faster market access to small cohorts of patients, this does not necessarily translate to faster access at population level post-MA, which is dependent on HTA or reimbursement committee decisions.