Suppr超能文献

为唐氏综合征成人患者调整淀粉样蛋白靶向抗体的处方标准。

Adapting prescribing criteria for amyloid-targeted antibodies for adults with Down syndrome.

机构信息

LuMind IDSC Foundation, Burlington, Massachusetts, USA.

Department of Disability and Human Development, University of Illinois Chicago, Chicago, Illinois, USA.

出版信息

Alzheimers Dement. 2024 May;20(5):3649-3656. doi: 10.1002/alz.13778. Epub 2024 Mar 13.

Abstract

Prior authorization criteria for Federal Drug Administration (FDA) approved immunotherapeutics, among the class of anti-amyloid monoclonal antibodies (mAbs), established by state drug formulary committees, are tailored for adults with late-onset Alzheimer's disease. This overlooks adults with Down syndrome (DS), who often experience dementia at a younger age and with different diagnostic assessment outcomes. This exclusion may deny DS adults access to potential disease-modifying treatments. To address this issue, an international expert panel convened to establish adaptations of prescribing criteria suitable for DS patients and parameters for access to Centers for Medicare & Medicaid Services (CMS) registries. The panel proposed mitigating disparities by modifying CMS and payer criteria to account for younger onset age, using alternative language and assessment instruments validated for cognitive decline in the DS population. The panel also recommended enhancing prescribing clinicians' diagnostic capabilities for DS and initiated awareness-raising activities within healthcare organizations. These efforts facilitated discussions with federal officials, aimed at achieving equity in access to anti-amyloid immunotherapeutics, with implications for national authorities worldwide evaluating these and other new disease-modifying therapeutics for Alzheimer's disease.

摘要

美国食品和药物管理局 (FDA) 批准的免疫疗法的预先授权标准,属于抗淀粉样蛋白单克隆抗体 (mAb) 这一类,由州药物处方委员会制定,适用于迟发性阿尔茨海默病的成年人。这忽视了患有唐氏综合征 (DS) 的成年人,他们通常在年轻时出现痴呆症,并且具有不同的诊断评估结果。这种排除可能会使 DS 成年人无法获得潜在的疾病修正治疗。为了解决这个问题,一个国际专家小组召开会议,制定了适合 DS 患者的处方标准的调整和获得医疗保险和医疗补助服务中心 (CMS) 登记处的参数。该小组提议通过修改 CMS 和支付方标准来减轻差异,以考虑到发病年龄较早,使用针对 DS 人群认知能力下降的经验证的替代语言和评估工具。该小组还建议提高为 DS 患者开具处方的临床医生的诊断能力,并在医疗机构内开展提高认识的活动。这些努力促进了与联邦官员的讨论,旨在实现获得抗淀粉样蛋白免疫疗法的公平性,这对全球评估这些和其他新的阿尔茨海默病疾病修正治疗方法的国家当局具有影响。

相似文献

1
Adapting prescribing criteria for amyloid-targeted antibodies for adults with Down syndrome.
Alzheimers Dement. 2024 May;20(5):3649-3656. doi: 10.1002/alz.13778. Epub 2024 Mar 13.
2
Amyloid age and tau PET timeline to symptomatic Alzheimer's disease in Down syndrome.
medRxiv. 2024 Aug 9:2024.08.08.24311702. doi: 10.1101/2024.08.08.24311702.
3
Assessing amyloid PET positivity and cognitive function in Down syndrome to guide clinical trials targeting amyloid.
Alzheimers Dement. 2024 Aug;20(8):5570-5577. doi: 10.1002/alz.14068. Epub 2024 Jun 28.
4
Alzheimer's drugs, APPlication for Down syndrome?
Ageing Res Rev. 2024 Apr;96:102281. doi: 10.1016/j.arr.2024.102281. Epub 2024 Mar 19.
6
Clinical and biomarker changes of Alzheimer's disease in adults with Down syndrome: a cross-sectional study.
Lancet. 2020 Jun 27;395(10242):1988-1997. doi: 10.1016/S0140-6736(20)30689-9.
7
Anti-Amyloid-β Monoclonal Antibodies for Alzheimer's Disease: Pitfalls and Promise.
Biol Psychiatry. 2018 Feb 15;83(4):311-319. doi: 10.1016/j.biopsych.2017.08.010. Epub 2017 Aug 24.
8
Novel anti-amyloid-beta (Aβ) monoclonal antibody lecanemab for Alzheimer's disease: A systematic review.
Int J Immunopathol Pharmacol. 2023 Jan-Dec;37:3946320231209839. doi: 10.1177/03946320231209839.
9
Alzheimer's polygenic risk scores are associated with cognitive phenotypes in Down syndrome.
Alzheimers Dement. 2024 Feb;20(2):1038-1049. doi: 10.1002/alz.13506. Epub 2023 Oct 19.
10
The direct health care cost to Medicare of Down syndrome dementia as compared with Alzheimer's disease among 2015 Californian beneficiaries.
Ann Phys Rehabil Med. 2021 Jan;64(1):101430. doi: 10.1016/j.rehab.2020.07.011. Epub 2020 Oct 15.

引用本文的文献

6
Biologically defined neuronal synuclein disease as a tool to advance drug development.
NPJ Parkinsons Dis. 2024 Dec 20;10(1):235. doi: 10.1038/s41531-024-00845-5.
9
Alzheimer Dementia Among Individuals With Down Syndrome.
JAMA Netw Open. 2024 Sep 3;7(9):e2435018. doi: 10.1001/jamanetworkopen.2024.35018.

本文引用的文献

1
Revised criteria for diagnosis and staging of Alzheimer's disease: Alzheimer's Association Workgroup.
Alzheimers Dement. 2024 Aug;20(8):5143-5169. doi: 10.1002/alz.13859. Epub 2024 Jun 27.
2
Plasma Biomarker Strategy for Selecting Patients With Alzheimer Disease for Antiamyloid Immunotherapies.
JAMA Neurol. 2024 Jan 1;81(1):69-78. doi: 10.1001/jamaneurol.2023.4596.
3
Down Syndrome in a New Era for Alzheimer Disease.
JAMA. 2023 Dec 12;330(22):2157-2158. doi: 10.1001/jama.2023.22924.
4
Disease-modifying drugs for Alzheimer disease: implications for people in Canada.
CMAJ. 2023 Oct 30;195(42):E1446-E1448. doi: 10.1503/cmaj.230595.
5
Who Should-and Can-Get Lecanemab, the New Alzheimer Disease Drug?
JAMA. 2023 Oct 17;330(15):1411-1415. doi: 10.1001/jama.2023.14443.
6
Role of Registries in Medicare Coverage of New Alzheimer Disease Drugs.
JAMA. 2023 Oct 10;330(14):1331-1332. doi: 10.1001/jama.2023.17131.
7
Eligibility for Anti-Amyloid Treatment in a Population-Based Study of Cognitive Aging.
Neurology. 2023 Nov 7;101(19):e1837-e1849. doi: 10.1212/WNL.0000000000207770. Epub 2023 Aug 16.
9
Lecanemab: Appropriate Use Recommendations.
J Prev Alzheimers Dis. 2023;10(3):362-377. doi: 10.14283/jpad.2023.30.
10
Predicting the Societal Value of Lecanemab in Early Alzheimer's Disease in Japan: A Patient-Level Simulation.
Neurol Ther. 2023 Aug;12(4):1133-1157. doi: 10.1007/s40120-023-00492-7. Epub 2023 May 15.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验