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ABSTRACT: Background
Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing access to medicines before marketing approval to patients not recruited in trials. The economic impact of clinical trials has previously been investigated. No evidence on the net economic benefit of CUP exists. This research aims to address this information gap by estimating the economic consequences of 11 CUP in Italy conducted between March 2015 and December 2020 from the perspective of public health care system in Italy (National Health Service). Eight programs concern cancer treatments, two refer to spinal muscular atrophy, and one is indicated for multiple sclerosis.Methods
Since CUP medicines are covered by the industry, the net economic benefit includes: (i) avoid
SUBMITTER: Jommi C
PROVIDER: S-EPMC8645125 | biostudies-literature | 2021 Dec
REPOSITORIES: biostudies-literature