Early Access Programmes in Europe Versus Key Markets in Other Regions: An In-Depth Comparison

OBJECTIVES: To compare characteristics, regulations and usage of Early Access Programmes (EAPs) for medicines in Europe with those in Australia, Brazil, Canada, China, Japan, Saudi Arabia and US.

METHODS: Information on EAPs and drug availability prior to access and coverage decisions was obtained from national regulatory authorities’ websites in 51 countries in June 2024. We analysed 10 criteria including transparency, maturity, monitoring requirements, funding options, eligibility and exit criteria, number of applications and success rates in the past five years.

RESULTS: EAPs exhibit significant variation across the 10 criteria analysed, both within and outside of Europe.

In Europe, 26 out of 44 countries have EAPs in place, of which half are well-established and widely used with clear application criteria. Nine countries offer reimbursed options. Ireland stands out as the most notable example that does not offer any opportunity for early access. Overall, EAP maturity and transparency is lowest in Eastern European countries. No evidence of EAP regulations streamlining for Joint Clinical Assessment.

Among the key markets outside of Europe, all seven offer early access. Three have well-established EAPs in place (Australia, Canada, US), albeit without reimbursement. Australia and US provide multiple pathways, accommodating different levels of urgency based on patient numbers, and which have seen high usage.

Recently established EAPs in Brazil, China, Japan and Saudi Arabia have less detailed regulations, resulting in ambiguity (Brazil, Japan, Saudi Arabia), lower acceptance rates (e.g. Japan), or EAP coverage restricted to specific drugs and locations (China).

CONCLUSIONS: High variation exists in EAPs across countries evaluated, with more mature programmes in key markets. High transparency, clear eligibility and exit criteria provide greater incentives for manufacturers seeking early access for their products. In contrast, insufficient detail on monitoring requirements, lack of data collection infrastructure, and ambiguous regulations pose challenges for manufacturers and could impact use of early access pathways.