OBJECTIVES:

BACKGROUND

Since 1994 the temporary authorisation for use (ATU) scheme allows the exceptional and temporary use of unlicensed medicines outside of a clinical trial to a single or a group of patients to treat serious or rare diseases when no appropriate treatment exists. ATU drugs can only be prescribed in hospitals after approval of the French drug regulatory body (ANSM). Their prices are freely set by the manufacturer and fully funded outside the DRG system through a specific budgetary allocation within the budget envelope of Missions of General Interest and Contracting Support (MIGAC) for inpatient use, or through the reassigned list (liste de rétrocession) for outpatient dispensation.

If this programme was implemented to improve early access to promising drugs, the recent increase of ATU drug spending raises the question of its sustainability.

OBJECTIVE

This study aims to describe the evolution of the inpatient use ATU drug spending from 2012 to 2016.

METHODS:

We included 2012-2016 ATUs data for inpatient use (unit price, volume of prescriptions) extracted from the French DRG based information system (PMSI). For each year the numbers of hospitals prescribing ATUs and medicinal products funded were studied. Therapeutic area repartition was assessed for 2016.

RESULTS:

From 2012 to 2016 inpatient use ATU drug spending increased more than 10-fold from €35.4 to €471.4 million. The number of hospitals prescribing these medicines rose by more than 2-fold (190 to 481). The number of ATU drugs increased by 40% (105 to 148). In 2016, the expenditure was mainly due to hemato and/or oncology drugs (85%) followed by gastroenterology- hepatology drugs (11%).

CONCLUSIONS:

From 2012 to 2016 the expenditure of inpatient use ATU drug drastically increased as well as the numbers of medicinal products covered and prescribing hospitals. In 2016, hemato and/or oncology drugs represented the major portion of the expenditure.