Judicialization of Zolgensma in the Ministry of Health: costs and clinical profile of patients

Kretzschmar, Ana Katheryne Miranda; Teixeira, Ellen; Galato, Dayani; Silva, Everton Nunes da

doi:10.11606/s1518-8787.2024058005899

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Original Article • Rev. Saúde Pública 58 • 2024 • https://doi.org/10.11606/s1518-8787.2024058005899 copy

Judicialization of Zolgensma in the Ministry of Health: costs and clinical profile of patients

Authorship SCIMAGO INSTITUTIONS RANKINGS

ABSTRACT

OBJECTIVE

To investigate the costs and profile of patients who have filed a lawsuit against the Ministry of Health for the treatment of spinal muscular atrophy (SMA) with the onasemnogene abeparvovec (Zolgensma^®).

METHODS

This is a cross-sectional, descriptive study with a census design, based on records of lawsuits filed against the Ministry of Health between January 2019 and September 2022. Data was requested from the Ministry of Health via the Access to Information Act. Information was extracted on the epidemiological profile of the beneficiaries of the lawsuits, as well as the expenses spent by the Ministry of Health in cases where the requests were granted.

RESULTS

136 lawsuits were identified, of which 113 (83%) were favorable to patients at a cost of R$ 944.8 million in the period analyzed. Demographic (gender and age), clinical (SMA subtypes, use of ventilatory or nutritional support), and lawsuit (type of legal service) characteristics were not associated with the granting of lawsuits. Prior use of medication (nusinersena or ridisplam) was associated with the dismissal of lawsuits. Of the 113 lawsuits granted in favor of patients, only six (5.3%) would meet the criteria currently established by the National Committee for Health Technology Incorporation - Conitec (children up to six months without ventilatory and nutritional support). R$ 146 million was spent on supplying Zolgensma to children over the age of two, which is outside the recommendation contained in the drug’s package leaflet.

CONCLUSIONS

The Ministry of Health incurs a high cost with the judicialization of Zolgensma for SMA, representing 2.45% of total spending on medicines in the Unified Health System, including spending by the three administrative spheres. Some of the lawsuits have been granted in disagreement with the criteria established by health technology assessment agencies and the drug manufacturer’s recommendations.

Spinal Muscular Atrophy; Onasemnogene Abeparvovec; Health Judicialization; Cost Analysis

Evidence	Age	Type of SMA	Restrictions on use
Package leaflet (manufacturer Novartis)⁸	Under two years old	SMA type I or up to three copies of the SMN2 gene	The use of Zolgensma in patients with advanced SMA has not been evaluated, for example: total paralysis of the limbs and permanent dependence on ventilation.
Conitec¹³	Up to six months	AME5q type I	Children on invasive ventilation for more than 16 hours a day.
SMC¹⁶	Does not define age	SMA type I, or up to three copies of the SMN2 gene, in which patients are expected to develop SMA type I	No restrictions on use.
CADTH¹⁷	Up to six months	SMA patients who are symptomatic or pre-symptomatic with one to three copies of the SMN2 gene	Children who need permanent feeding or ventilatory support (invasive or non-invasive).
NICE¹⁸	Up to six months or age seven to 12 months, with treatment agreed by the multidisciplinary team	AME5q type I	Tracheostomized children and those on permanent ventilation for more than 16 hours a day. Use in children aged seven to 12 months is only indicated if the treatment will give them a 70% chance of sitting up independently.

Characteristics	Court order granted^a		Legal claim dismissed		p-value
Characteristics	No. of applicants	%	No. of applicants	%	p-value
Sex					0.590
Female	52	46	12	52.2
Male	61	54	11	47.8
Patient’s age on application					0.323
0-6 months	31	27.4	4	17.4
7-12 months	33	29.2	9	39.1
13-18 months	27	23.9	5	21.7
19-24 months	19	16.8	3	13.0
25 months or older	3	2.7	2	8.7
Patient’s age at judicial compliance^a
0-6 months	10	9.2
7-12 months	16	14.7
13-18 months	33	30.3
19-24 months	32	29.4
25 months or older	18	16.5
Type of SMA					0.438
I	99	87.6	19	82.6
II	13	11.5	3	13
III	1	0.9	1	4.3
Type of representation					0.161
Private	110	97.3	21	91.3
Public	3	2.7	2	8.7
Use of other medicines^b					0.025
Yes	45	39.8	15	65.2
No	68	60.2	8	34.8
Ventilatory support					0.701
Yes	54	47.8	12	52.2
No	59	52.2	11	47.8
Nutritional support					0.974
Yes	34	30.1	7	30.4
No	79	69.9	16	69.6

Type of SMA	Cost of medication		Additional costs		Total cost (R$)
Type of SMA	Quantity	Cost (R$)	Quantity	Cost (R$)	Total cost (R$)
I	99^a	833,386,445.27	37	2,950,690.29	836,337,135.56
II	13^b	99,377,369.67	6	271.240.14	99,648,609.81
III	1	8,812,225.00	0	R$ 0.00	8,812,225.00
Total cost					944,797,970.37

Group	SMA type I		SMA type II			SMA type III
Group	n	Cost (R$)	n	Cost (R$)	n	Cost (R$)
Children up to six months without ventilatory and nutritional support	6	53,782,491.83	0	0.00	0	0.00
Children up to six months with ventilatory and nutritional support	4	36,731,461.25	0	0.00	0	0.00
Children > six months and ≤ 24 months with ventilatory and nutritional support	42	384,831,441.21	0	0.00	0	0.00
Children > six months and ≤ 24 months without ventilatory and nutritional support	30	247,183,951.57	9	76,249,872.02	0	0.00
Children > 24 months	17^a	113,807,789.70	4^b	23,398,737.79	1	8,812,225.00
Total	99	836,337,135.56	13	99,648,609.81	1	8,812,225.00

Faculdade de Saúde Pública da Universidade de São Paulo Avenida Dr. Arnaldo, 715, 01246-904 São Paulo SP Brazil, Tel./Fax: +55 11 3061-7985 - São Paulo - SP - Brazil
E-mail: revsp@usp.br

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[1] Correspondence: Everton Nunes da Silva Universidade de Brasília 72220-275 Brasília, DF, Brasil E-mail: evertonsilva@unb.br