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

Abstract 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.

Medicine manipulation: An alternative to mitigate therapeutic gaps in the Brazilian Unified Health System?

Despite the various initiatives carried out in Brazil and in the world, the challenge of offering essential medicines in adequate presentations remains, especially to the public affected by diseases considered neglected and the pediatric population, for whom the therapeutic options remain limited. The main objective of this study was to evaluate the production of manipulated medicines as a strategy to mitigate therapeutic and access gaps to essential medicines within the Brazilian public health system, called the Unified Health System (SUS). The evaluation, carried out between 2020 and 2021, identified, among the medicines considered essential to the Brazilian health context, those unavailable, for which strategies were evaluated to mitigate the identified unavailability, which is conventionally called therapeutic gaps. For 57% (n = 235) of pharmaceutical presentations identified as therapeutic gaps in SUS, manipulation was identified as the best strategy to promote access. Of these presentations, 30% (n = 70) were identified as priorities in the context of patient care and were mainly related to the demands of the pediatric public and those affected by poverty-related diseases. Concerning poverty-related diseases, the absence of evidence on the development of a standard formula for drugs with indication for such diseases was demonstrated. The need for an annual investment of approximately US$74.75 per capita was estimated to offer treatments in adequate presentations to SUS users, which should reflect in the improvement of the quality of life of about 26 thousand people. It was observed that this investment amount corresponds to only 3% of the budget for the purchase of medicines financed exclusively by the Ministry of Health thorugh the Strategic Component of Pharmaceutical Assistance (CESAF) approved for 2021.