Original Title: Citrato de tofacitinibe para o tratamento da retocolite ulcerativa moderada a grave em pacientes adultos com resposta inadequada, perda de resposta ou intolerantes ao tratamento prévio com medicamentos sintéticos convencionais ou biológicos

Authors' objectives: Is tofacitinib citrate effective and safe for the treatment of moderately to severely active UC in adult patients with inadequate response, loss of response or intolerance to aminosalicylates, corticosteroids, azathioprine, 6-mercaptopurine or anti-TNF biologic drugs?

Authors' results and conclusions: The Conitec’s members present at the 88th Ordinary Meeting, on July 7th, 2020, unanimously decided not to recommend the incorporation of tofacitinib for the treatment of moderate to severe ulcerative colitis in adult patients with inadequate response, loss of response or intolerance to previous treatment with synthetic drugs, in the scope of SUS. After analysing the contributions received in the public consultation, the members of Conitec’s plenary session considered the following aspects: although the cost of treatment with tofacitinib could lead to a significant reduction in administration and storage costs, the therapy with infliximab and vedolizumab showed better statistical results in the evaluated outcomes, or they were not considered different from tofacitinib for the outcome of clinical remission at week 8. Moreover, there was no additional scientific evidence that could change the preliminary recommendation, which was mainly based on the uncertainty about efficacy and safety of tofacitinib compared with other options available in SUS. Also, the type of economic evaluation submitted by the applicant proved to be inadequate because the comparator drugs do not have a similar efficacy that would justify the use of cost-minimization analysis. The Deliberation Record No. 530/2020 was signed.

Authors' recommendations: Scientific evidence: The applicant presented six scientific studies and one meta-analysis of indirect comparison (own elaboration). Conitec’s Executive Secretariat also conducted a search and included another indirect comparison study. In conclusion, the following studies were analysed: 1 study of 3 clinical trials (Sandborn, 2017), assessed with low risk of bias; 2 systematic reviews with indirect comparisons (Bonovas, 2018, and Trigo-Vicente, 2018); and 2 studies of post-hoc analysis of quality of life (Panés, 2017) and safety (Sandborn, 2018). These studies had a moderate quality score, according to a specific tool. In individuals with moderate to severe ulcerative colitis, therapy with tofacitinib at a dose of 10 mg twice daily for 8 weeks was associated with clinical remission in a higher, and statistically significant, proportion of patients compared with placebo, from 10% (95% Confidence Interval [CI] 4.3-16.3) to 13% (95% CI 8.1-17.9). In the maintenance phase (52 weeks), therapy with tofacitinib at a dose of 5 mg and 10 mg twice daily was also associated with a greater clinical remission compared with placebo (difference of 23.2% [95% CI 15.3-31.2%] and 29.5% [95% CI 21.4-37.6%], respectively). In relation to the mucosal healing outcome, therapy with tofacitinib at a dose of 5 mg and 10 mg for 8 weeks was associated with a higher percentage of cure compared with placebo (15.7%)...

Project Status: Completed

URL for project: http://conitec.gov.br/images/Consultas/Relatorios/EN2020/20210106_Report538_Tofacitinib_ModeratetoSevereUlcerativeColitis.pdf

Year Published: 2020

URL for published report: http://conitec.gov.br/images/Relatorios/2020/Relatorio_538_Tofacitinibe_RetocoliteUlcerativa_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