Astellas Pharma, Northbrook, Illinois.
Xcenda, Palm Harbor, Florida.
J Manag Care Spec Pharm. 2020 Apr;26(4):538-549. doi: 10.18553/jmcp.2020.19329. Epub 2020 Feb 5.
Prostate cancer is the most common cancer and second-leading cause of cancer death among men in the United States. Prostate cancer poses a large economic burden, which increases with progression from localized to metastatic disease. Newly approved treatments for non-metastatic castration-resistant prostate cancer (nmCRPC) delay disease progression and reduce the risk of metastatic disease. Quantifying the potential budget impact of these new treatments is of interest to health care decision makers.
To estimate the budget impact of enzalutamide for the treatment of patients with nmCRPC in the United States over a 3-year time horizon.
An Excel-based model was developed to estimate the budget impact to a U.S. health plan of enzalutamide, a second-generation antiandrogen, as an add-on to androgen deprivation therapy (ADT) for the treatment of high-risk nmCRPC patients (prostate-specific antigen doubling time of ≤ 10 months). Comparators include apalutamide + ADT, bicalutamide + ADT, and ADT only. The analysis includes treatment costs for nmCRPC and for treatment after progression to metastatic castration-resistant prostate cancer (mCRPC). The treated population size was estimated from epidemiological data and literature. Dosing, duration of therapy, and adverse event rates were based on package inserts and pivotal studies. RED BOOK, Centers for Medicare & Medicaid Services fee schedules, and literature were used to obtain costs of drugs, adverse events, and health care visits. Market shares were estimated for each comparator before and after enzalutamide adoption. A 1-way sensitivity analysis was performed to quantify the impact of parameter uncertainty.
In a hypothetical 1-million-member plan with 3% annual growth, it was estimated that there would be approximately 19 eligible incident nmCRPC patients in year 1, increasing to 20 eligible incident patients in year 3. With an assumed market share of approximately 6% for enzalutamide in year 1, the budget impact would be $106,074 ($0.009 per member per month [PMPM]). With a 26% enzalutamide share in year 3, the budget impact would be $632,729 ($0.048 PMPM). Cumulative budget impact to the health plan over 3 years is estimated to be $1,082,095 ($0.028 PMPM). The increased cost of the treatment regimen is partly offset by reduced postprogression costs.
Treatment of nmCRPC patients with enzalutamide has a modest budget impact that is partly offset by delaying progression to mCRPC.
This research was sponsored by Astellas Pharma and Pfizer, the codevelopers of enzalutamide. All authors contributed to the development of the manuscript and maintained control over the final content. Schultz is employed by Astellas Pharma and owns stock in Gilead Sciences and Shire. O'Day and Sugarman are employees of Xcenda, which received consultancy fees from Astellas Pharma. Ramaswamy is employed by Pfizer. A synopsis of the current study was presented in poster format at the AMCP Managed Care & Specialty Pharmacy Annual Meeting 2019, in San Diego, CA, on March 25-28, 2019.
前列腺癌是美国男性中最常见的癌症和第二大癌症死亡原因。前列腺癌带来了巨大的经济负担，随着疾病从局部进展为转移性疾病，这种负担会增加。新批准的用于非转移性去势抵抗性前列腺癌（nmCRPC）的治疗方法可延迟疾病进展并降低转移性疾病的风险。了解这些新疗法的潜在预算影响对医疗保健决策者很有意义。
在 3 年的时间内，估算在美国使用恩扎卢胺治疗 nmCRPC 患者的预算影响。
开发了一个基于 Excel 的模型，以估算美国健康计划中恩扎卢胺的预算影响，恩扎卢胺是一种第二代抗雄激素药物，作为高风险 nmCRPC 患者（前列腺特异性抗原倍增时间≤10 个月）的雄激素剥夺疗法（ADT）的附加治疗。对照药物包括阿帕鲁胺+ADT、比卡鲁胺+ADT 和 ADT 单药治疗。分析包括 nmCRPC 治疗和进展为转移性去势抵抗性前列腺癌（mCRPC）后的治疗成本。治疗人群规模是根据流行病学数据和文献估算的。剂量、治疗持续时间和不良事件发生率基于产品说明书和关键研究。使用 RED BOOK、医疗保险和医疗补助服务中心费用表和文献获得药物、不良事件和医疗就诊的成本。在恩扎卢胺采用之前和之后，估计了每种对照药物的市场份额。进行了单因素敏感性分析以量化参数不确定性的影响。
在一个拥有 3%年增长率的 100 万成员计划中，预计在第 1 年会有大约 19 名新发病的 nmCRPC 患者，到第 3 年会增加到 20 名新发病的 nmCRPC 患者。假设恩扎卢胺在第 1 年的市场份额约为 6%，则预算影响将为 106,074 美元（每位成员每月 0.009 美元[PMPM]）。在第 3 年，恩扎卢胺的市场份额为 26%，则预算影响将为 632,729 美元（每位成员每月 0.048 美元）。在 3 年内，健康计划的累积预算影响估计为 1,082,095 美元（每位成员每月 0.028 美元）。治疗方案成本的增加部分被进展后成本的降低所抵消。
使用恩扎卢胺治疗 nmCRPC 患者的预算影响适度，部分被延迟进展为 mCRPC 所抵消。
这项研究由安斯泰来制药和辉瑞公司赞助，这两家公司共同开发了恩扎卢胺。所有作者都参与了手稿的编写，并对最终内容保持控制。舒尔茨受雇于安斯泰来制药，拥有吉利德科学和夏尔的股票。奥戴和萨格曼是 Xcenda 的员工，Xcenda 从安斯泰来制药获得咨询费。拉马钱德兰受雇于辉瑞公司。目前研究的概要在 2019 年 3 月 25 日至 28 日在加利福尼亚州圣地亚哥举行的 AMCP 管理式医疗和特种药房年会 2019 上以海报形式展示。
