Original Title: Ampliação de uso do sirolimo para tratamento de indivíduos adultos com linfangioleiomiomatose

Authors' objectives: Is sirolimus more effective, safe or cost-effective for the treatment of patients with LAM when compared with the options available in the Brazilian Public Health System (SUS)?

Authors' results and conclusions: Conitec’s plenary session discussed and considered the need to incorporate in the scope of SUS a drug treatment that acts directly on the disease. They also took into account the clinical benefits of sirolimus in LAM, such as reduction in the progression of the disease, and the difficulty in accessing the medication due to its high cost. Therefore, the Conitec’s members present at the 88th Ordinary Meeting, on July 8th, 2020, unanimously decided to recommend the expansion of the use of sirolimus for the treatment of adult patients with LAM, in the scope of SUS.

Authors' recommendations: Scientific evidence: The evidence supporting the use of sirolimus for the treatment of patients with LAM was based on three cohort studies, four clinical trials, and one sub-analysis of data. These studies were of low methodological quality and high risk of bias. The results of the outcomes were reported with different forms and units of measurement, making interpretation, summarization and analysis difficult. Six studies reported that changes in Forced Expiratory Volume in 1 second (FEV1) were lower in patients using sirolimus, compared with placebo or before using it. After 12 months of treatment with sirolimus, there was an improvement in FEV1, and a significant reduction in patients not using it. Regarding Forced Vital Capacity (FVC), four studies reported an improvement in FVC values in patients using sirolimus (p ≤ 0.001). A quality of life outcome was reported by three studies, and in two of them there was an improvement in the quality of life of patients using sirolimus (p0.05). The most common adverse events were gastrointestinal, infectious, pulmonary or upper respiratory tract, dermatological, and neurological events. Budget impact analysis: The incremental value of incorporating sirolimus was estimated to range from BRL 896.2 thousand in the first year to BRL 1.22 million after five years. The incremental budget impact was estimated to be approximately BRL 5.3 million after five years. The analysis was considered to be underestimated due to the assumptions used to estimate the population.

Project Status: Completed

URL for project: http://conitec.gov.br/images/Consultas/Relatorios/EN2020/20210106_Report539_ExpansionofSirolimus_Lymphangioleiomyomatosis.pdf

Year Published: 2020

URL for published report: http://conitec.gov.br/images/Relatorios/2020/Relatorio_539_Sirolimo_Linfangioleiomiomatose_Final_2020.pdf

URL for additional information: http://conitec.gov.br/recomendation-reports

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Brazil

Organisation Name: National Committee for Technology Incorporation (CONITEC)

Contact Address: Esplanada dos Ministérios, Bl. G, Ed. Sede, 8º andar, CEP: 70058-900

Contact Name: Clarice Moreira Portugal

Contact Email: clarice.portugal@saude.gov.br

Copyright: 2021 Conitec