OBJECTIVES: A major challenge for Regulatory Agencies all around the word is to achieve a balance between the need of a rapid access to new therapies for patients and the assessment of benefit/risk. To speed market access of drugs with high unmet need, Early Access Programs (EAPs) have been activated in many States over the years. EAPs generally refer to patient access to drugs/indications before marketing authorization.

METHODS: This paper aims to compare EAPs in Australia, 4 European Countries (France, Germany, Italy, Spain) and UK. The analysis was conducted through a national and international literature review for each Country. Regulatory Agency websites were reviewed: PBAC for Australia, HAS for France, IQWiG and G-BA for Germany, AIFA for Italy, AEMPS for Spain, NICE for UK.

RESULTS: In Australia any unapproved drug can be supplied through the TGA Special Access Scheme (SAS). Early access to regulatory approved but unfunded therapies can be provided through Product Familiarization or Compassionate Use Programs. In France from 2020 there is a new Early Access Program (former ATU, former PEC-T), named or cohort depending on the program. In Germany, legislation allows compassionate use and enables treatment to a cohort of patients. In Italy AIFA defined different tools to grant EA to important drugs: Law 326, Law 648/96, Compassionate use (named/cohort). In Spain there is a program to make available drugs under special circumstances (name based). In UK, the Early Access to Medicines Scheme (EAMS) program was launched in 2014.

CONCLUSIONS: Our research highlighted that there are important differences in EAPs implemented the EU4, UK and Australia and this is a source of inequalities in access to medicines for patients. This research, being a comparison of all the existing early access scheme, will be the starting point to think about an harmonization, aimed to optimize them.