Table 3.

All-cause health care costs among patients with SMA who were adherent vs. not adherent to risdiplam

All-cause health care costs per patient per year (2023 USD), mean ± SD [median]2	SMA type 1	SMA type 2		SMA type 3		SMA type 4
	Adherenta (n = 1)	Adherenta (n = 15)	Non-adherent (n = 3)	Adherenta (n = 40)	Non-adherent (n = 6)	Adherenta (n = 15)	Nonadherent (n = 5)
Total all-cause health care costsb	91,754.4 ± NA	49,061.8 ± 61,414.4 [21,475.4]	378,126.1 ± 172,674 [356,524.6]	34,249.2 ± 67,075.8 [14,909.1]	64,075.6 ± 55,904.2 [56,113]	15,583.4 ± 31,353.9 [4,594.1]	46,498.7 ± 60,638.7 [16,817]
Medical service costsb	91,204.6 ± NA	45,882.9 ± 60,925.1 [21,402.8]	376,187.5 ± 170,627.2 [354,854.5]	30,514.8 ± 66,468.8 [10,188.7]	62,668.9 ± 54,824 [54,218.8]	11,221.4 ± 29,712.2 [1,328.5]	32,651.7 ± 61,018.6 [4,206.9]
Mechanical ventilation support	12,389.7 ± NA	10,637.1 ± 24,242.5 [0]	159,882.9 ± 266,441.2 [12,186.4]	2,209.1 ± 7,594.2 [0]	27,106.3 ± 53,260.2 [0]	0 ± 0 [0]	0 ± 0 [0]
Nutritional support	34,984.1 ± NA	11,850.8 ± 31,034.4 [116.6]	162,689.6 ± 281,294.1 [568.8]	1,414.3 ± 3,614.7 [0]	8,372.6 ± 19,541 [0]	58.2 ± 201 [0]	0 ± 0 [0]
Pharmacy costsc	549.8 ± NA	3,178.9 ± 7,039.4 [1,132.7]	1,938.7 ± 2,046.9 [1,670.1]	3,734.4 ± 8,649.5 [535.3]	1,406.7 ± 1,539.2 [864.6]	4,362.1 ± 13,761.8 [195.7]	13,846.9 ± 30,460.1 [206.2]

IQR, interquartile range; NA, not applicable; PDC, proportion of days covered; SD, standard deviation; SMA, spinal muscular atrophy; USD, United States dollar

^aAdherence was defined as PDC ≥ 0.80

^bOne outlier patient with excessive health care costs (>$4 million) was excluded from the analysis. This patient underwent multiple procedures that were unrelated to SMA within a relatively short timeframe

^cTreatment costs of nusinersen and onasemnogene abeparvovec recorded in the medical claims and treatment costs of risdiplam recorded in pharmacy claims were excluded