Table 3

Costs and QALYs per newborn by four screening and treatment strategies over 5 and 60 years, and ICERs for pairwise comparisons from societal perspective, discounted 3% per annum

StrategyCostQALY
5 years$ (95% CI)(95% CI) 
 No NBS and supportive care$35
(13 to 69)		0.00003
(0.00002 to 0.00006)		 
 No NBS and nusinersen Tx$150
(107 to 221)		0.00009
(0.00005 to 0.00013)		 
 NBS and gene Tx$198
(116 to 310)		0.00015
(0.00008 to 0.00025)		 
 NBS and nusinersen Tx$184
(109 to 285)		0.00015
(0.00008 to 0.00025)		 
Incremental analysisIncr costIncr QALYICER
 $ (95% CI)(95% CI)$/QALY (95% CI)‡
 NBS and nusinersen Tx vs NBS and gene Tx−$15
(−29 to 3)		–Dominant*
 NBS and nusinersen Tx vs no NBS and nusinersen Tx$33
(−22 to 135)		0.00007
(−0.00001 to 0.00016)		$494 000
(dominant to 5 321 000)
 NBS and nusinersen Tx vs no NBS and supportive care$149
(74 to 247)		0.00012
(0.00005 to 0.00022)		$1 237 000
(920 000 to 1 916 000)
 NBS and gene Tx vs no NBS and nusinersen Tx$48
(−45 to 161)		0.00007
(−0.00001 to 0.00016)		714 000
(dominant to 4 611 000)
 NBS and gene Tx vs no NBS and supportive care$164
(81 to 271)		0.00012
(0.00005 to 0.00022)		$1 360 000
(1 035 000 to 2 093 000)
 No NBS and nusinersen Tx vs supportive care$116
(81 to 164)		0.00005
(0.00003 to 0.00008)		$2 179 000
(1 402 000 to 3 864 000)
60 yearsCostQALY 
 $ (95% CI)(95% CI) 
 No NBS and supportive care$131
(47 to 273)		0.00018
(0.00011 to 0.00031)		 
 No NBS and nusinersen Tx$432
(311 to 594)		0.00061
(0.00040 to 0.00084)		 
 NBS and gene Tx$407
(202 to 787)		0.00146
(0.00076 to 0.00247)		 
 NBS and nusinersen Tx$867
(484 to 1 414)		0.00146
(0.00076 to 0.00247)		 
Incremental analysisIncr costIncr QALYICER
 $ (95% CI)(95% CI)$/QALY (95% CI)‡
 NBS and nusinersen Tx vs NBS and gene Tx$460
(257 to 734)		–Dominated†
 NBS and nusinersen Tx vs no NBS and nusinersen Tx$436
(65 to 985)		0.00085
(0.00020 to 0.00181)		$513 000
(274 000 to 847 000)
 NBS and nusinersen Tx vs no NBS and supportive care$736
(369 to 1 286)		0.00128
(0.00058 to 0.00227)		$577 000
(378 000 to 958 000)
 NBS and gene Tx vs no NBS and nusinersen Tx−$24
(−228 to 290)		0.00085
(0.00020 to 0.00181)		Dominant
(dominant to 239 000)
 NBS and gene Tx vs no NBS and supportive care$276
(71 to 619)		0.00128
(0.00058 to 0.00227)		$216 000
(70 000 to 464 000)
 No NBS and nusinersen Tx vs supportive care$300
(197 to 415)		0.00043
(0.00025 to 0.00061)		$706 000
(438 000 to 1 196 000)

  • *Nusinersen was less costly but equivalently effective.

  • †Nusinersen was more costly but equivalently effective.

  • ‡Rounding to nearest 1000.

  • ICER, incremental cost-effectiveness ratio; NBS, newborn screening; QALY, quality-adjusted life-year.