Table 4.

CASE MODEL 2: Example of protocols of EAP that follow a clinical trial and include patients who wish to continue the treatmentᅟ

Case model 2 Therapeutic area: Urogenital diseases
Clinical study design	Target	Endpoints	Italian or international	Phase
Multicentre, open-label, single-arm, early-access	Patients who have participated in the clinical trial and need prompt access to the results	Primary: assess the safety and tolerability in long-term treatment	Italy and other European countries	Phase 3

In this case-model, patients included in the protocol have no comparable or satisfactory therapeutic alternatives but they have participated in the clinical trial and need to continue the treatment

Considerations: despite the protocol target being coherent in Italy and in Europe with the EAP, the primary endpoint is typical of a clinical trial and the sponsor manages the protocol as if it were a clinical trial. In the USA this protocol would have be configured as an Open-label Extension study