MedStar Georgetown University Hospital, Washington, DC, USA.
AbbVie, North Chicago, Illinois, USA.
Headache. 2024 Nov-Dec;64(10):1253-1263. doi: 10.1111/head.14824. Epub 2024 Sep 9.
To estimate the number needed to treat and cost per additional responder for atogepant and rimegepant versus placebo for the preventive treatment of episodic migraine (EM) in the United States.
Migraine has an enormous impact on a person's daily activities and quality of life, and results in significant clinical and economic burden to both individuals and society. It is important to understand the comparative efficacy and economic value of oral calcitonin gene-related peptide receptor antagonists (gepants) for preventive treatment of EM. Currently, atogepant and rimegepant are US Food and Drug Administration approved for preventive treatment of migraine (rimegepant for EM and atogepant for EM and for chronic migraine). In the absence of head-to-head trials, we utilized an indirect treatment comparison on efficacy data from clinical trials conducted for the preventive treatment of EM. We estimated number needed to treat, a valuable metric used in clinical practice to compare treatment efficacy, and cost per additional responder, which can be used to establish the cost effectiveness of a treatment.
An indirect treatment comparison was conducted to compare the efficacy of atogepant 60 mg once daily and rimegepant 75 mg once every other day as preventive treatments for EM using published data from the registrational trials of atogepant (ADVANCE) and rimegepant (BHV3000-305). The efficacy outcome of interest was ≥50% reduction from baseline in mean monthly migraine/headache days (≥50% responder rate), which was variably defined for a base case and two scenario analyses. Number needed to treat and cost per additional responder versus placebo were calculated and compared between both treatments (weeks 9-12 in the base case analysis; weeks 1-12 and 9-12 for atogepant and during weeks 9-12 for rimegepant in the scenario analyses).
In the base case analysis, ≥50% responder rates were 64.9% (95% confidence interval [CI], 53.9-74.5) for atogepant and 51.8% (95% CI, 42.9-60.6) for rimegepant, compared to 44.1% (95% CI, 39.4-49.0) for placebo. The median number needed to treat versus placebo in the base case scenario was 4.8 (95% CI, 3.1-9.0) for atogepant compared to 13.0 (95% CI, 5.9-75.1) for rimegepant. The cost per additional responder versus placebo in the base case scenario was estimated to be $15,823 (95% CI, $11,079-$29,516) for atogepant compared to $73,029 (95% CI, $32,901-$422,104) for rimegepant. Results of the two scenario analyses were consistent with the base case analysis.
Atogepant had substantially lower numbers needed to treat and costs per additional responder versus placebo than rimegepant for the preventive treatment of EM across all evaluated scenarios. These analyses suggest that atogepant may be more cost effective than rimegepant for the preventive treatment of EM. Limitations include differences in inclusion/exclusion criteria and in reporting of the ≥50% responder rates between trials.
估计依替巴肽和利马昔坦相对于安慰剂在预防发作性偏头痛（EM）中的治疗人数和每个额外应答者的成本，用于美国。
偏头痛对个人的日常活动和生活质量有巨大影响，给个人和社会带来了重大的临床和经济负担。了解口服降钙素基因相关肽受体拮抗剂（ gepants）用于预防 EM 的相对疗效和经济价值非常重要。目前，依替巴肽和利马昔坦已获得美国食品和药物管理局批准，用于预防偏头痛（利马昔坦用于 EM，依替巴坦用于 EM 和慢性偏头痛）。由于没有头对头试验，我们利用临床试验的间接治疗比较，对 EM 的预防性治疗进行了疗效数据的间接治疗比较。我们估计治疗人数，这是一种在临床实践中用于比较治疗效果的有用指标，以及每个额外应答者的成本，这可以用于确定治疗的成本效益。
利用依替巴肽（ ADVANCE）和利马昔坦（ BHV3000-305）注册试验中公布的数据，进行间接治疗比较，以比较依替巴肽 60mg 每日一次和利马昔坦 75mg 每两天一次作为 EM 的预防治疗。关注的疗效结果是与基线相比，每月偏头痛/头痛天数至少减少 50%（≥50%应答率），这在基线分析和两种方案分析中定义各不相同。在基线分析中，依替巴肽的≥50%应答率为 64.9%（95%置信区间[CI]，53.9-74.5），利马昔坦为 51.8%（95%CI，42.9-60.6），安慰剂为 44.1%（95%CI，39.4-49.0）。在基线方案分析中，与安慰剂相比，依替巴坦的中位数治疗人数为 4.8（95%CI，3.1-9.0），而利马昔坦为 13.0（95%CI，5.9-75.1）。在基线方案分析中，与安慰剂相比，依替巴坦的每个额外应答者的成本估计为 15823 美元（95%CI，11079-29516），而利马昔坦为 73029 美元（95%CI，32901-422104）。两种方案分析的结果与基线分析一致。
在所有评估的方案中，依替巴坦的治疗人数和每个额外应答者的成本均显著低于利马昔坦，用于预防 EM。这些分析表明，依替巴坦可能比利马昔坦更具成本效益，用于预防 EM。局限性包括试验之间的纳入/排除标准和≥50%应答率报告存在差异。
