LuMind IDSC Foundation, Burlington, Massachusetts, USA.
Department of Disability and Human Development, University of Illinois Chicago, Chicago, Illinois, USA.
Alzheimers Dement. 2024 May;20(5):3649-3656. doi: 10.1002/alz.13778. Epub 2024 Mar 13.
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.
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。