Trend of expenditure for orphan drugs in Italy 2013-2023

The level of expenditure on orphan drugs in Italy increased by more than 1.6 billion euros between 2013 and 2023. Orphan drugs are used to treat rare diseases. Their use is so limited that their development is considered not profitable by pharmaceutical companies. Consequently, they may be defined as drugs which are not marketed by the pharmaceutical industry due to business reasons, even though they address a public healthcare need.   

Orphan drugs consumption and expenditure

In 2017, the therapeutic category named antineoplastic and immunomodulating agents accounted for 56.9 percent of the orphan drugs consumption and for 58.7 percent of the expenditure. The expression antineoplastic and immunomodulating agents refers to preparations intended for treating malignant tumors.    

Clinical trials

The drugs pipeline for clinical trials consists of four phases: the pre-clinical testing involves laboratory and animal testing, the second one refers to the trials on humans and the other two involve an increasing number of individuals. In 2019, in Italy most of the clinical trials aiming at treating rare diseases were in the phases two and three of the clinical pipeline. More specifically, 97 drugs were in phase two and 88 were in phase three, whereas only two products were in the final stage of the pipeline.