Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism

• Published in: Brazilian Journal of Pharmaceutical Sciences 2023-01, Vol.59
• Main Authors: Fachini, Adrielle da Silva, Malaquias, Lorena Freitas Barros, Nogueira, Luma de Lira, Giacone, Ludmila Alvim Gomes Pinho, Tavares, Noemia Urruth Leão, Cardoso, Maria Teresinha de Oliveira, Gelfuso, Guilherme Martins, Silva, Dayde Lane Mendonça da, Santana, Rafael Santos
• Format: Article
• Language: English
• Subjects: Clinical trials; Drug dosages; Enzymes; Genomics; Literature reviews; Metabolism; Patients; Pediatrics; Pharmaceutical industry; PHARMACOLOGY & PHARMACY
• ISSN: 2175-9790; 1984-8250; 2175-9790
• DOI: 10.1590/s2175-97902023e21109

Inborn errors of metabolism are rare disorders with few therapeutic options for their treatments, which can make patients suffer with complications. Therefore, compounded drugs might be a promising option given that they have the ability of meeting the patient’s specific needs, (i) identification of the main drugs described in the literature; (ii) proposal of compounding systems and (iii) calculation of the budgetary addition for the inclusion of these drugs into the Brazilian Unified Health System. The research conducted a literature review and used management data as well as data obtained from official Federal District government websites. The study identified 31 drugs for the treatment of inborn errors of metabolism. Fifty eight percent (58%) (18) of the medicines had their current demand identified, which are currently unmet by the local Health System. The estimated budget for the production of compounded drugs was of R$363,16.98 per year for approximately 300 patients. This estimated cost represents a budgetary addition of only 0.17% from the total of expenditures planned for drug acquirement. There is a therapeutic gap for inborn errors of metabolism and compounding pharmacies show potential in ensuring access to medicine therapy with a low-cost investment.