Compassionate drug uses in Italy. Analysis of regional and local diffusion

Aims. Compassionate drugs are provided to patients with a specific disease and no further treatment option, most frequently via Early (or Expanded) Access Programs. In Italy, it often occurs that compassionate uses concern medicines whose price has not been negotiated yet (and therefore unavailable on the market), although their use has been approved in Europe. Thus, compassionate drug uses turn out to be a way to expedite the access to new innovative drugs with demonstrated efficacy. This study aims to investigate how widespread is the use of compassionate drugs throughout the Country. Methods. We analyzed data from 20 early access programs implemented by 2 pharmaceutical companies in the last few years. Data were analyzed by the number of patients and centers in each Region and province, and a correlation was established between patients and centers in each Region and the resident population. A further analysis was carried out with the same criteria on the subpopulation of oncology patients, including more than 80% of total study population. Results. In our sample, 7529 patients received compassionate drug treatments in 348 centers throughout Italy. A significant correlation exists between the resident population in each Region and the number of requesting centers (r(2)=0.877) and patients treated (r(2)=0.844) in the Region. Taking the value of the linear regression slope as the expected one, certain Regions show a better "performance", in terms of more patients treated than expected, namely Umbria, Emilia-Romagna, Lazio, Lombardy, Tuscany, Liguria and Friuli Venezia-Giulia. Conclusions. In this study we showed that the use of compassionate drugs in Italy is diffused in a manner closely related to the population of each Region. A number of Regions - mostly but not exclusively from the South and Island areas - show a performance below the expectations, in terms of patients treated.