OBJECTIVES: Real-World Evidence (RWE) is an increasing focus of discussion to address limitations in clinical trial data. However, generation of RWE for a new therapy can only typically be generated after the product has launched. Nevertheless, payers want to see product-specific RWE at launch to inform reimbursement decision-making. Early access / Compassionate Use programs (EAPs) offer the opportunity for patients to access therapies prior to marketing authorisation. This then offers the potential to generate RWE for a product in that market prior to its launch. This research evaluates the EAPs available in major European countries to inform a discussion on whether these can be leveraged for RWE collection pre-launch.

METHODS: Publicly-available information was screened for information on EAPs in France, Germany, Italy, Spain, the UK, and the Netherlands using their respective websites and key information extracted.

RESULTS: All 6 countries offer EAPs, many offer more than one (range: 1[Germany,Spain]–5[Italy]). Inclusion criteria is broadly aligned for most markets, in being for therapies in clinical development for severe/life-threatening diseases with no other treatment options available. Typically, applications can be made by either physicians or manufacturers although for some (e.g. Spain) it is physician-initiation only. Reimbursement/charging incentives vary between countries and within available country programs. In 2/6 (Germany, UK) manufacturers must provide their treatments at zero cost. Another 2/6 (Spain, Netherlands) offer reimbursement only in select cases. Only 2/6 (Italy and France) offer full-reimbursement (although in Italy this is only in select programs).

CONCLUSIONS: All major European markets offer EAPs, potentially offering an avenue to collect product-specific RWE outcome data prior to its launch for reimbursement stakeholders. However, there are additional challenges/barriers to this approach, including lack of reimbursement incentives, challenges in collecting outcomes data, and only severe patients being eligible who may experience worse clinical outcomes than the label population.