Value-Based Pharmacy Initiatives, UPMC Center for High-Value Health Care, Pittsburgh, PA.
Pharmacy Services, UPMC Insurance Services Division, Pittsburgh, PA.
J Manag Care Spec Pharm. 2024 Nov;30(11):1211-1216. doi: 10.18553/jmcp.2024.30.11.1211.
Outcomes-based agreements (OBAs) are agreements between payers and manufacturers in which payment for medications is tied to patient outcomes. These contracts aim to measure the value of prescription medications on predefined clinical indicators in real-world patient populations. OBAs are gaining traction in the United States as the health care industry shifts from volume-based to value-based care. Multiple sclerosis (MS) is an appealing therapeutic area for OBAs because of its prevalence, high cost of medications, and multiple effective therapeutic options.
To describe findings from an OBA that was prospectively conducted in a large regional health system for patients with MS taking interferon β-1a or dimethyl fumarate.
In this prospective real-world analysis, commercial or health insurance exchange members were included based on the parameters of the OBA. Disability progression was assessed using a patient-reported outcome, patient-determined disease steps (PDDS). In the OBA, members aged 18 years or older with an MS diagnosis were included in the contract. A baseline score was collected for eligible members, with follow-up scores occurring between a 90-day and 180-day postbaseline score. If a follow-up score was greater than the baseline score, a subsequent PDDS score was collected between 90-days and 120-days to determine if the PDDS score remained elevated, indicating that the member had disability progression.
During the contract period, 410 patients were eligible for PDDS collection, with 241 and 169 patients in the dimethyl fumarate and interferon β-1a cohorts, respectively. There were 162 patients who were lost to follow-up, and 64 patients who were ineligible per contract parameters. Of the remaining 184 eligible patients (107 on dimethyl fumarate and 77 on interferon β-1a), 21 (11%) patients had confirmed disability progression (6 on dimethyl fumarate [5.6%] and 15 on interferon β-1a [19.5%]).
Our findings suggest that meaningful patient-reported outcomes, such as disability progression, can be operationalized in an innovative OBA.
基于结果的协议（OBAs）是支付方和制造商之间的协议，其中药物的支付与患者的结果挂钩。这些合同旨在根据预设的临床指标，在现实世界的患者群体中衡量处方药物的价值。随着医疗保健行业从基于数量的护理转向基于价值的护理，OBAs 在美国越来越受欢迎。多发性硬化症（MS）是 OBA 的一个有吸引力的治疗领域，因为它的发病率高，药物费用高，并且有多种有效的治疗选择。
描述在一个大型地区性卫生系统中对接受干扰素β-1a 或二甲基富马酸酯治疗的多发性硬化症患者进行的前瞻性 OBA 的结果。
在这项前瞻性真实世界分析中，根据 OBA 的参数纳入商业或健康保险交易所成员。残疾进展使用患者报告的结果、患者确定的疾病步骤（PDDS）进行评估。在 OBA 中，年龄在 18 岁或以上且患有 MS 诊断的成员被纳入合同。对符合条件的成员收集基线评分，并在基线评分后 90 至 180 天之间进行后续评分。如果后续评分大于基线评分，则在 90 至 120 天之间收集后续 PDDS 评分，以确定 PDDS 评分是否仍然升高，表明成员的残疾进展。
在合同期间，有 410 名患者有资格进行 PDDS 采集，二甲基富马酸酯和干扰素β-1a 队列分别有 241 名和 169 名患者。有 162 名患者失访，64 名患者因合同参数不合格。在其余 184 名符合条件的患者中（二甲基富马酸酯组 107 名，干扰素β-1a 组 77 名），有 21 名（11%）患者确认残疾进展（二甲基富马酸酯组 6 名[5.6%]，干扰素β-1a 组 15 名[19.5%]）。
我们的研究结果表明，可以在创新的 OBA 中对有意义的患者报告结果（如残疾进展）进行操作。
