Access to Modulating Drugs for Cystic Fibrosis

OBJECTIVES: Ten years ago, the first CFTR protein modulator was approved in the United States, which brought about a real revolution in the treatment of cystic fibrosis in the world. The cost of the drug is a huge challenge for countries health systems. This type of medicine can generate a cost of 3 PIB per capita (approximately R$ 122,000.00 per year, per patient) and this is absolutely unfavorable. The present work seeks to mitigate this budgetary impact with a different analysis bias.

METHODS: Analysis of the Brazilian Federal Constitution of 1988; Declaration of Rights of 1689, Declaration of the Rights of Man and Citizen of 1789, Universal Declaration of Human Rights of 1948, Declaration of Doha of 2001. Economic Evaluation Budget Impact Analysis Empirical analysis of patients registered at the Associação Carioca de Mucoviscidosis of the State of Rio de Janeiro, RJ.

RESULTS: In Brazil, around 1,500 people will be able to use this medication, provided they are eligible and aged 6 years or older. On the other hand, the new recently approved cost-effectiveness threshold is being implemented. It is a parameter to better understand the impact on the efficiency of a health system generated by the incorporation of a new technology in relation to existing ones.

CONCLUSIONS: To provide free treatment to those patients with the most severe stage of the disease, regardless of where they live; availability of CFTR modulators in all countries with appropriate regulatory approval, without delay; pricing of all CFTR modulators at affordable levels proportionate to each country's situation. Patients who currently use the triple combination in Brazil were only able to access it via court order, but this is not a medium and long-term solution.