Profile of Drug Utilization in Patients with Rare Diseases in Tuscany, Italy: A Population-Based Study

Francesca Gorini (), Michele Santoro, Anna Pierini, Lorena Mezzasalma, Silvia Baldacci and Alessio Coi
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Francesca Gorini: Institute of Clinical Physiology, National Research Council, 56124 Pisa, Italy
Michele Santoro: Institute of Clinical Physiology, National Research Council, 56124 Pisa, Italy
Anna Pierini: Institute of Clinical Physiology, National Research Council, 56124 Pisa, Italy
Lorena Mezzasalma: Institute of Clinical Physiology, National Research Council, 56124 Pisa, Italy
Silvia Baldacci: Institute of Clinical Physiology, National Research Council, 56124 Pisa, Italy
Alessio Coi: Institute of Clinical Physiology, National Research Council, 56124 Pisa, Italy

IJERPH, 2023, vol. 20, issue 2, 1-14

Abstract: Patients with rare diseases (RDs) generally have delayed diagnosis and misdiagnosis, which lead to inappropriate care or the need to modify treatment during the course of the disease. The medical care of RD patients can be further complicated by the presence of comorbidities. In this population-based study, we evaluated the prevalence, intensity of use, and consumption of drugs prescribed to RD patients residing in Tuscany (Italy) in the years 2008–2018. Data from the Registry of Rare Diseases of Tuscany were integrated with information retrieved from regional pharmaceutical prescription databases. The overall prevalence of drug use in the RD patients was 85.4%. Drugs for the alimentary tract and metabolism and antiinfectives for systemic use showed the highest prevalence of use, while drugs for the nervous system had the highest intensity of use only in the pediatric patients. The adults exhibited a female preponderance in terms of the prevalence of use and drug consumption in almost all the age groups and therapeutic categories. Conversely, a higher prevalence of use was observed in the male children. These results provide relevant insights into drug profiles in RD patients, representing a first step for future analyses to monitor changes in drug utilization in patients with RDs over time.

Keywords: rare disease; drug; prevalence of use; intensity of use; consumption; population-based (search for similar items in EconPapers)
JEL-codes: I I1 I3 Q Q5 (search for similar items in EconPapers)
Date: 2023
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