OBJECTIVES: Early access programs (EAPs) are an innovative approach that manufacturers can use to provide patients with potentially life-changing therapeutics pre-EMA approval. To qualify for an EAP the therapeutic must treat a disease or specific patient population with a high clinical unmet need. The aim of this study was to assess the current perception of EAPs in the EU5 and how inclusion in an EAP may correlate or potentially lead to reimbursement challenges.

METHODS: Qualitative research with physicians and payers in Germany, France, U.K., Italy and Spain was performed to understand EAP dynamics in each individual market.

RESULTS: Our research identified insightful nuisances and variation of interest across the EU5. In France, an EAP is known as a ATU and it is the most established EAP. Inclusion in an ATU can correlate with reimbursement. In Germany stakeholders, do not recommend EAPs but an investigator led clinical trial to collect data for NUB submission. In the UK EAPs are perceived positively and they can lead to fast track NICE appraisal and they ensure a constant engagement with the regulatory and reimbursement agencies. In Italy and Spain, inclusion in an EAP can be in a narrower patient population than the anticipated label, which can affect reimbursement upon approval.

CONCLUSIONS: EAPs are, most importantly, an innovative mechanism for improving access to therapeutics for patients with a high clinical need but they can also provide added value to manufacturers in certain markets. Patients can get access to a therapy pre-approval and manufacturers can increase physician comfort and use in Europe of their product.