Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study.

ABSTRACT: Background: Despite calls for the use of real-world data, the rare diseases (RD) treatment landscape suffers from a scarcity of data referred to orphan medicinal products (OMP) use at the population level. Objectives: We aim to describe the characteristics and patterns of utilization of OMP in a sizable group of RD patients globally monitored by an area-based rare diseases registry located in the Veneto region, Italy, during a 3-year period (1 January 2019 to 31 December 2021). Methods: A list of OMP (n = 60) was assembled for study purposes, according to extensive criteria with regard to the status of orphan designation and of national reimbursement decisions. Results: OMP prescriptions involved 1,010 patients, corresponding to the 2.3% of all the patients moni

SUBMITTER: Mazzucato M 

PROVIDER: S-EPMC9563616 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature