Early Access to Medicines in the US and Europe

Overview
Faced with pressure from stakeholders – particularly patients – governments, regulators, and some countries have introduced new options and/or processes to speed up access to medicines. This includes access before marketing authorization, and speeding up the marketing authorization, or less often, the health technology assessment processes.

However, the approach of each country differs, leaving patients, clinicians, and companies navigating a myriad set of rules and processes. All approaches are focused on access for serious and/or life-threatening diseases where significant unmet clinical need remains, but each offers differences in terms of processes. They also differ according to whether companies must provide their products at no charge or are able to secure early reimbursement.
While the FDA’s early access programs are relatively well established, the EMA has introduced several more recent programs. These schemes emphasize early dialogue with multiple stakeholders, and enhanced opportunities for evidence generation during early access periods, including real-world studies to complement traditional randomized controlled trials.