Page 84 - 《中国药房》2024年15期

spinal muscular atrophy （SMA）[J]. Pharmacoeconomics， [22] National Centre for Pharmacoeconomics. Cost-effective-
2022，40（Suppl. 1）：69-89. ness of nusinersen （Spinraza） for the treatment of 5q spi‐
[12] HUSEREAU D，DRUMMOND M，AUGUSTOVSKI F， nal muscular atrophy （SMA）[EB/OL].（2017-07-11）
et al. Consolidated health economic evaluation reporting [2023-09-27]. https：//www.ncpe.ie/nusinersin-spinraza/.
standards （CHEERS） 2022 explanation and elaboration：a [23] Canadian Agency for Drugs and Technologies in Health.
report of the ISPOR CHEERS Ⅱ good practices task force Pharmacoeconomic review report：nusinersen （Spinraza）：
[J]. Value Health，2022，25（1）：10-31. （Biogen Canada Inc.）：indication：treatment of patients
[13] MALONE D C，DEAN R，ARJUNJI R，et al. Cost- with 5q SMA [EB/OL]. [2023-09-27]. https：//pubmed.
effectiveness analysis of using onasemnogene abeparvo‐ ncbi.nlm.nih.gov/30480926/.
cec （AVXS-101） in spinal muscular atrophy type 1 pa‐ [24] National Institute for Health and Care Excellence. Single
tients[J]. J Mark Access Health Policy，2019，7（1）： technology appraisal nusinersen for treating spinal muscu‐
1601484. lar atrophy [ID1069] [EB/OL].（2018-01-15）[2023-09-
[14] ZULUAGA-SANCHEZ S，TEYNOR M，KNIGHT C， 27]. https：//www.nice.org.uk/guidance/ta740/evidence/
et al. Cost effectiveness of nusinersen in the treatment of appraisal-consultation-committee-papers-pdf-9266538637.
patients with infantile-onset and later-onset spinal muscu‐ [25] ZULUAGA-SANCHEZ S，PURSER M，MADER G，
lar atrophy in Sweden[J]. Pharmacoeconomics，2019，37 et al. Improved quality of life and life-years in patients
（6）：845-865. with infantile-onset spinal muscular atrophy following
[15] JALALI A，ROTHWELL E，BOTKIN J R，et al. Cost- treatment with nusinersen[EB/OL]. （2019-05-18）[2023-
effectiveness of nusinersen and universal newborn scree- 09-27]. https：//www.ispor.org/heor-resources/presentations-
ning for spinal muscular atrophy[J]. J Pediatr，2020，227： database/presentation/intl2019-1846/91949.
274-280.e2. [26] ZULUAGA-SANCHEZ S，PURSER M，MADER G，
[16] THOKALA P，STEVENSON M，KUMAR V M，et al. et al. Improved quality of life for patients and caregivers
Cost effectiveness of nusinersen for patients with infanti- among patients with later-onset spinal muscular atrophy
le-onset spinal muscular atrophy in US[J]. Cost Eff Re‐ following treatment with nusinersen[EB/OL]. （2019-05-
sour Alloc，2020，18：41. 18）[2023-09-27]. https：//www. ispor. org/heor-resources/
[17] BROEKHOFF T F，SWEEGERS C C G，KRIJKAMP E M， presentations-database/presentation/intl2019-1846/92064.
et al. Early cost-effectiveness of onasemnogene abepar- [27] National Institute for Health and Care Excellence. Single
vovec-xioi （Zolgensma） and nusinersen （Spinraza） treat‐ technology appraisal risdiplam for treating spinal muscu‐
ment for spinal muscular atrophy Ⅰ in the Netherlands lar atrophy in children and adults [ID1631] [EB/OL].
with relapse scenarios[J]. Value Health，2021，24（6）： （2020-10-01） [2023-09-27]. https：//www.nice.org.uk/
759-769. guidance/gid-ta10612/documents/committee-papers.
[18] SHIH S T，FARRAR M A，WILEY V，et al. Newborn [28] ARJUNJI R，ZHOU J，PATEL A，et al. PND5 cost-
screening for spinal muscular atrophy with disease- effectiveness analysis of newborn screening for spinal
modifying therapies：a cost-effectiveness analysis[J]. J muscular atrophy in the United States[J]. Value Health
Neurol Neurosurg Psychiatry，2021，92（12）：1296-1304. Reg News，2020，22：S75.
[19] WANG T J，SCUFFHAM P，BYRNES J，et al. Cost- [29] DEAN R，ARJUNJI R，AWANO H，et al. Cost-
effectiveness analysis of gene-based therapies for patients effectiveness of onasemnogene abeparvovec for spinal
with spinal muscular atrophy type Ⅰ in Australia[J]. J muscular atrophy type 1 （SMA 1） against nusinersen in
Neurol，2022，269（12）：6544-6554. Japan[EB/OL]. （2020-10-23） [2023-09-27]. https：//www.
[20] SHIH S T F，KELLER E，WILEY V，et al. Modelling the ispor. org/docs/default-source/intl2020/1140691avexisispor-
cost-effectiveness and budget impact of a newborn scree- japan-cea-bim-poster5-15-20cfinal-pdf. pdf? sfvrsn=9e4fe-
ning program for spinal muscular atrophy and severe com‐ 5bf_0.
bined immunodeficiency[J]. Int J Neonatal Screen，2022， [30] Scottish Medicines Consortium. Onasemnogene abepar-
8（3）：45. vovec 2×10 vector genomes/mL solution for infusion
13
[21] KHORSHID A，BOYD A L H，BEHR B，et al. Cost- （Zolgensma ） [EB/OL]. （2021-02-05） [2023-09-27].
®
effectiveness of IVF with PGT-M/A to prevent transmis‐ https：//www. scottishmedicines. org. uk/media/5813/onase-
sion of spinal muscular atrophy in offspring of carrier mnogene-abeparvovec-zolgensma-final-feb-2021-amended-
couples[J]. J Assist Reprod Genet，2023，40（4）：793-801. 010321docx-for-website.pdf.

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