延迟支付假设性充血性心力衰竭基因治疗的潜在益处：成本-后果分析。
The Potential Benefits of Deferred Payment for a Hypothetical Gene Therapy for Congestive Heart Failure: A Cost-Consequence Analysis.
机构信息
Leonard D. Schaeffer Center for Health Policy and Economics, University of Southern California, 635 Downey Way, Los Angeles, CA, 90089, USA.
Center for Improving Chronic Illness Care, University of Southern California, 635 Downey Way, Los Angeles, CA, 90089, USA.
出版信息
Appl Health Econ Health Policy. 2020 Oct;18(5):669-677. doi: 10.1007/s40258-020-00563-y.
BACKGROUND
The emergence of potentially curative pharmacologic treatments that deliver long-term clinical benefits with a limited number of doses may create short-term budget challenges for payers as their unit price can be high.
OBJECTIVE
This paper tests the clinical and financial properties of a deferred payment model (DPM) in hypothetical therapy for congestive heart failure (CHF) from the perspective of payers, manufacturers, and patients.
METHODS
We present an empirical analysis of longitudinal data for cardiovascular admissions and mortality using a Markov transition model for patient progression under different payment scenarios. The model calculates life-years gained and avoided cardiovascular admissions under the status quo and deferred payment and a hypothetical budget constraint. We tracked over 91,000 Medicare fee-for-service beneficiaries over a period of 5 years (2009-2014) using MedPAR 5% data files.
RESULTS
We find that a DPM is associated with earlier treatment and a consequent improvement in clinical outcomes. A 25% down-payment is associated with the highest relative improvement and reduces hospital admissions by 0.52% (by 2611 vs. 2071 cases) and mortality by 0.29% (by 799 vs. 648 cases), both relative to the status quo payment. Deferred payment results in limited financial gains for payers or manufacturers, primarily because of the small share of expected cost savings on the total cost of therapy. Our results are robust to changes in relative risk for cardiovascular admissions and a change in the cost of therapy.
CONCLUSIONS
A DPM may result in faster access to CHF gene therapy and may thus reduce hospital admissions and mortality in contrast to a status quo payment with the same budget constraint. Although the financial benefits of a DPM in CHF gene therapy are limited, it is possible that deferred payments will show greater promise for treatments with higher cost offsets.
背景
具有有限剂量但能提供长期临床获益的潜在治愈性药物治疗方法的出现，可能会给支付方带来短期预算挑战，因为其单位价格可能很高。
目的
本文从支付方、制造商和患者的角度，测试心力衰竭（CHF）假设治疗中递延支付模式（DPM）的临床和财务属性。
方法
我们使用患者在不同支付方案下进展的马尔可夫转移模型，对心血管入院和死亡率的纵向数据进行实证分析。该模型根据现状和递延支付以及假设的预算限制，计算获得和避免心血管入院的生命年。我们使用 MedPAR 5%数据文件，对超过 91000 名 Medicare 按服务收费受益人的数据进行了为期 5 年（2009-2014 年）的跟踪。
结果
我们发现 DPM 与更早的治疗和随后的临床结果改善有关。25%的预付款与相对改善程度最高相关，与现状支付相比，可减少 0.52%的住院（减少 2611 例与 2071 例）和 0.29%的死亡率（减少 799 例与 648 例）。递延支付对支付方或制造商的财务收益有限，主要是因为预期节省的总成本中仅占一小部分治疗总成本。我们的结果对心血管入院相对风险和治疗成本的变化具有稳健性。
结论
与具有相同预算限制的现状支付相比，DPM 可能会更快地获得 CHF 基因治疗，并因此减少住院和死亡率。尽管 DPM 在 CHF 基因治疗中的财务收益有限，但对于具有更高成本抵消的治疗方法，递延支付可能会更有希望。
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