Original Title: Mirabegrona para o tratamento da disfunção de armazenamento em pacientes com bexiga neurogênica

Authors' objectives: What is the efficacy and safety of mirabegron for storage dysfunction in adult patients with neurogenic bladder?

Authors' results and conclusions: The CONITEC’s members present at the 85th Ordinary Meeting, on February 4th, 2020, unanimously decided not to recommend the incorporation of mirabegron in the scope of SUS, for the treatment of storage dysfunction in patients with neurogenic bladder.

Authors' recommendations: Scientific evidence: In the search for evidence, 121 references were retrieved, of which three studies were selected, one randomized controlled trial (RCT) and two case series. Concerning the methodological quality, the case series presented a high risk of bias, while the RCT presented an unclear risk of bias since it did not report how allocation and blinding of the participants and assessors had been carried out. The main efficacy outcomes were cystometric volume, residual volume, urgency episodes, incontinence episodes, number of pads, Incontinence Quality of Life (I-QoL) and Patient Perception of Bladder Condition (PPBC) scores. Moreover, these studies also reported adverse events. In regard to the cystometric volume, mirabegron 50 mg/day led to an increase from 183.50 (standard deviation [SD]=121.60) mL at baseline to 238.81 (SD=150.56) mL after four weeks of treatment; in the group of patients receiving placebo, there was a reduction of 210.44 (SD=135.34) mL at baseline to 167.56 (SD=102.96) mL after four weeks of treatment (p=0.016). The two case series reported this outcome without a comparison group, and there were no significant differences before and after the intervention. In regard to the residual volume, only one case series evaluated this outcome: the residual volume was 83.4 (SD=92.2) mL at baseline; 56.8 (SD=61.4) mL in four weeks; and 78.8 (SD=113.3) mL in 12 weeks. There were no differences between follow-up and baseline values. Compared with placebo, mirabegron yielded significant improvements in quality of life and functionality, after four weeks of study (p = 0.0013 and p = 0.0001, respectively, for I-QoL and PPBC scores). Adverse events were more frequent in the group receiving mirabegron, but the studies did not provide detailed information on the difference between the groups. Budget impact analysis: The budget impact analysis was conducted from the perspective of the Brazilian Public Health System (SUS), over a five-year time horizon (2020-2024). The treatment cost was limited to the purchase price of medicines according to the Health Price Database. Given the absence of specific data on individuals with neurogenic bladder, the four main causes of neurogenic bladder were considered: Parkinson's disease, multiple sclerosis, stroke and spinal damage. In the baseline scenario, it was considered the incorporation of mirabegron, and its budget impact over five years was estimated to be BRL 11,273,255,511.53. An alternative scenario was developed taking into consideration the percentages of use of antimuscarinic agents obtained from a publication of the National Health Service (NHS) of the United Kingdom (UK). Thus, the budget impact of incorporating both antimuscarinics and mirabegron was estimated to be BRL 2,095,249,966.02, in the first year, and after five years, this amount would be BRL 10,679,375,762.42. Considerations: The studies have shown the efficacy of mirabegron in the treatment of incontinence symptoms, especially increase in the cystometric volume, quality of life (I-QoL) and functionality (PPBC) in patients with permanent spinal damage and multiple sclerosis. However, it should be noted that the two case series reported the outcome without a comparison group, and the RCT had a small sample size and an unclear risk of bias. Moreover, the estimated budget impact was over ten billion reais after five years of incorporation. Therefore, there is still insufficient evidence to allow the recommendation of mirabegron for patients with neurogenic bladder.

Project Status: Completed

URL for project: http://conitec.gov.br/images/Consultas/Relatorios/EN2020/20200915_Report509_Mirabegron_NeurogenicBladder.pdf

Year Published: 2020

URL for published report: http://conitec.gov.br/images/Relatorios/2020/Relatorio_mirabegrona_bexiga_neurogenica_509_2020_FINAL.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