Systematic review for pharmacoeconomics evaluation in spinal muscular atrophy
- VernacularTitle:脊髓性肌萎缩药物经济学评价的系统综述
- Author:
Xiaohong ZHU
1
,
2
,
3
;
Shixian LIU
1
,
2
,
3
;
Shunping LI
1
,
2
,
3
;
Lei DOU
1
,
2
,
3
;
Ruixue WANG
1
,
2
,
3
;
Zehua SONG
1
,
2
,
3
;
Hao CHEN
1
,
2
Author Information
1. Centre for Health Management and Policy Research，School of Public Health，Cheeloo College of Medicine，Shandong University，Jinan 250012，China
2. NHC Key Lab of Health Economics and Policy Research （Shandong University），Jinan 250012，China
3. Center for Health Preference Research，Shandong University，Jinan 250012，China
- Publication Type:Journal Article
- Keywords:
spinal muscular atrophy;
pharmacoeconomics;
systematic review;
nusinersen sodium;
sovaprevir;
risperidone
- From:
China Pharmacy
2024;35(15):1868-1875
- CountryChina
- Language:Chinese
-
Abstract:
OBJECTIVE To review the current research progress on pharmacoeconomics evaluation related to spinal muscular atrophy （SMA）， in order to provide valuable insights for clinical treatment， screening and medical insurance payment decision- making. METHODS A computerized search was conducted across multiple databases including PubMed， Web of Science， Embase， Scopus， Cochrane Library， EBSCOhost， CNKI， VIP， CBM and Wanfang database as well as other important health technology assessment （HTA） websites， such as National Institute for Health and Care Research，International Society of Technology Assessment in Health Care， Agency for Healthcare Research and Quality， etc. The pharmacoeconomics evaluation studies related to SMA were collected from the inception to December 31st， 2023. The literature/reports were rigorously screened based on predefined inclusion and exclusion criteria by two researchers， and the essential information from the included literature/ reports was extracted using Excel 2019. The quality of the included literature/reports was evaluated by Consolidated Health Economic Evaluation Reporting Standards 2022. RESULTS Finally， 9 articles and 15 HTA reports were included， with overall good quality of literature， but poor quality of HTA reports. There were a total of 24 studies on the pharmacoeconomics evaluation of SMA， including treatment options such as nusinersen sodium， sovaprevir， risperidone， and best supportive therapy.The review results showed that nusinersen sodium was not cost-effective in the treatment of SMA； there was no consensus on the economic viability of treatment options such as risperidone and sovaprevir； newborn/prenatal screening combined withmedication therapy was cost-effective. CONCLUSIONS newborn/prenatal screening combined with SMA medication therapy demonstrates economic advantages. It is suggested to further investigate the cost-effectiveness of new SMA drugs and SMA screening in China， taking localization parameters and medical insurance prices into account， and gradually incorporate SMA screening into the scope of neonatal genetic disease detection， in order to alleviate the financial burden of patients’ families and healthcare systems.
