The High "Cost" of Experimental Drugs Obtained Through Health Litigation in Brazil.

Ricardo Eccard Da Silva,Maria Rita C G Novaes,Elisangela Da Costa Lima,Claudia G S Osorio-De-Castro

doi:10.3389/fphar.2020.00752

Ricardo Eccard Da Silva, Maria Rita C G Novaes + Show 2 more

Open Access

https://doi.org/10.3389/fphar.2020.00752

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Abstract

BackgroundBrazilian patients have legal right to access unlicensed medicines undergoing clinical research, if there is evidence of efficacy and safety. This study investigated the occurrence of serious adverse events related to very high-cost medicines from clinical studies, expanded access and compassionate use programs, obtained by patients though health litigation.MethodsA descriptive study using secondary data investigated unlicensed medicines obtained through lawsuits from 2010 to 2017, costing more than 1 million Brazilian reais (BRL), adjusted by the Brazilian Consumer Index to July 2017. Data sources were the Brazilian Health Surveillance Agency Registry (DATAVISA) and Adverse Events in Clinical Studies (NotivisaEC) Databases. Medicines were categorized by the Anatomical Therapeutic Chemical classification to level 03 and events by the WHO Adverse Drug Reaction Terminology. The study received ethical approval by the University of Brasilia Institutional Research Board.ResultsIn the period, 812 drugs were obtained through litigation, and of these, 78 exceeded cost of 1 million BRL; 44 of them presented reports of 1,248 serious adverse events. Total Brazilian Government expenditure with these drugs was 3.2 billion BRL. Class L04A (n=7) showed greater expenditures (over 1.8 billion BRL). One hundred ninety-six deaths occurred and L01X was the most involved category (49.5%). Most other serious events (n=419) and sequelae (n=10) were related to L01X.ConclusionVery high-cost drugs paid for by the government and obtained through health litigation presented deaths and serious adverse events in expanded access and compassionate use programs in Brazil.

Highlights

Serious and/or rare conditions with high social impact and risk of death are usually treated with high-cost drugs either recently brought to market or still in trial for later licensing (OPAS, 2009)
We proceeded to identify which purchases involved drugs demanded through litigation but which had been used in clinical trials, expanded access or compassionate use programs, made at any point during the study period with a contract value equal to or greater than 1 million BRL
The resulting list of costly medicines obtained through litigation was screened for their safety profile during use in clinical trials, expanded and compassionate use programs in Brazil through both the Sistema de Controle de Pesquisa Clıń ica (Clinical Trial Control System, SCPC) and the Sistema de Notificacão de Eventos Adversos Graves em Ensaios Clıń icos (Adverse Events in Clinical Trials Notification System, NotivisaEC) to verify existence of adverse events reports (Anvisa, 2018)

Summary

Introduction

Serious and/or rare conditions with high social impact and risk of death are usually treated with high-cost drugs either recently brought to market or still in trial for later licensing (OPAS, 2009). These products may worsen a patient's condition if their efficacy and safety are not known or acceptable. Authorizations for the expanded access program are given to groups of patients and the drugs must necessarily be in a phase III clinical trial (Brasil, 2013a). This study investigated the occurrence of serious adverse events related to very high-cost medicines from clinical studies, expanded access and compassionate use programs, obtained by patients though health litigation

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