Banca de DEFESA: Ana Katheryne Miranda Kretzschmar

Uma banca de DEFESA de MESTRADO foi cadastrada pelo programa.
STUDENT : Ana Katheryne Miranda Kretzschmar
DATE: 18/12/2023
TIME: 14:00
LOCAL: PLATAFORMA TEAMS
TITLE:

Judicialization of onasemnogene abeparvoveque (Zolgensma®) for the treatment of spinal muscular atrophy (SMA) in the Ministry of Health: costs and clinical profile of patients. 

KEY WORDS:

 

spinal muscular atrophy, onasemnogene abeparvovec, judicialization, healthcare, cost analysis, healthcare financin

PAGES: 90
BIG AREA: Outra
AREA: Multidisciplinar
SUMMARY:

Objective: Investigate the costs and profile of patients who sued in court onasemnogene abeparvovec (Zolgensma®) for the treatment of spinal muscular atrophy (SMA) in the Ministry of Health. Method: Cross-sectional study, descriptive in nature and census design, based on records of legal actions filed against the Ministry of Health from January 2019 to September 2022. The data was requested from the Ministry of Health, via the Access to Information Law. Information was extracted on the epidemiological profile of the beneficiaries of the legal actions, as well as the expenses spent by the Ministry of Health in cases of granting the requests. Results: 136 legal cases were identified, all with a single beneficiary, of which 113 (83%) were favorable to patients at a cost of R$944.8 million (USD $192 M) in the period analyzed. Demographic (sex and age), clinical characteristics (SMA subtypes, use of ventilatory or nutritional support) and judicial process (type of legal service) were not associated with the granting of legal actions. Previous use of medication (nusinersen or ridisplam) was associated with the dismissal of legal proceedings. Of the 113 legal actions granted in favor of patients, only 6 (5.3%) would meet the criteria currently established by the National Commission for the Incorporation of Technologies – Conitec (children up to six months without ventilatory and nutritional support). There was an expenditure of R$ 146 million (USD $29,76 M) on the supply of onasemnogene abeparvoveque for children over 2 years of age, which is outside the recommendation contained in the drug leaflet. Conclusions: The Ministry of Health incurs a high cost with the judicialization of onasemnogene abeparvovec for AME, representing 3.06% of total spending on medicines in the SUS in 2019, including expenses from the three administrative spheres. Part of the legal demands have been granted in disagreement with the criteria established by health technology assessment agencies and recommendations from the drug manufacturer. 

COMMITTEE MEMBERS:
Interna - 1212574 - ANTONIA DE JESUS ANGULO TUESTA
Presidente - 1681340 - EVERTON NUNES DA SILVA
Externa à Instituição - MARTA DA CUNHA LOBO SOUTO MAIOR
Externa à Instituição - PATRICIA COELHO DE SOAREZ - USP