Expanded Access Programme: looking for a common definition

Table 3 CASE MODEL 1: Example of protocols of EAP that have the purpose of providing the treatment to patients who are not eligible for enrolment into a clinical trials.

Case model 1 Therapeutic area: Neoplasia
Clinical study design	Target	Endpoints	Italian or international	Phase
Multicentre, open-label, single-arm, expanded-access	Advanced stage carcinoma (inoperable or metastatic)	Primary: assess the safety and tolerability of long-term treatment	Italy and other European countries	Phase 3
	Progressive disease after treatment with marketed drugs or intolerance	Secondary: assess progression-free and overall survival
	Patients not previously treated with the experimental drug (who has not participated in a clinical trial)	Secondary: assess progression-free and overall survival

In this case-model, patients included in the protocol have no comparable or satisfactory therapeutic alternatives but the protocol has safety and tolerability primary endpoints
Considerations: this protocol (on the basis of the target), can come under Ministerial Decree 8 May 8 2003, as a CUP and EAP, because it ensures access to investigational drugs for patients who have no comparable or satisfactory therapeutic alternatives. However, the primary endpoint is typical of a clinical trial

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