Rivolo Simone, Loftus Jane, Peter Balazs, Fahey Marion, Kongnakorn Thitima
Evidera, Inc. Milan, Italy.
Value & Evidence, Pfizer, Walton Oaks, UK.
J Med Econ. 2023 Jan-Dec;26(1):963-972. doi: 10.1080/13696998.2023.2228167.
Paediatric growth hormone deficiency (pGHD) manifests as growth failure associated with inadequate growth hormone (GH) production. Daily injections of recombinant human GH (dGH) [somatropin] is the current standard of care, which has been shown to be well tolerated and effective, but associated with suboptimal adherence, leading to reduced effectiveness. Somatrogon, a once-weekly injectable long-acting human GH, has demonstrated clinical non-inferiority and significantly lower life interference (i.e. treatment burden) vs. somatropin in two Phase 3 studies. This work evaluated cost-effectiveness and cost-utility of somatrogon vs dGHs from an Irish payer perspective.
A Markov model was developed for patients starting somatrogon or dGHs treatment at 3-12 years and continuing up to achievement of near adult height (NAH), with growth driven by trial-based height velocity (HV) and treatment-specific adherence. Patients could discontinue treatment at the end of Year 1 (4%). DGH adherence (95.3%-65% over treatment duration) and adherence-growth relationship were based on published evidence. Higher Year 1 adherence of 4%, tapering over time, for somatrogon vs. dGHs was based on clinical consultation. Treatment costs, monitoring costs and costs due to different wastage types (device setting and adherence) were sourced from local data. Health utilities based on height and injection frequency were derived from published literature. Scenario analysis, deterministic and probabilistic sensitivity analysis were performed.
Somatrogon treatment led to 1.87-3.66 cm greater NAH gain and 0.21-0.50 higher quality adjusted life years (QALYs) vs. dGHs, across the base case and scenarios evaluated. Somatrogon treatment was associated with cost savings of €5,699-€21,974 and lower cost per cm gained vs. dGHs (€197-€527), per patient. Somatrogon was cost-effective vs. dGHs, with the result consistent across the sensitivity analyses conducted.
Somatrogon weekly injections were estimated to result in higher NAH, higher QALYs, lower overall costs and lower costs per cm gained than dGHs, in pGHD.
儿童生长激素缺乏症(pGHD)表现为与生长激素(GH)分泌不足相关的生长迟缓。每日注射重组人生长激素(dGH)[生长激素]是目前的标准治疗方法,已证明其耐受性良好且有效,但依从性欠佳,导致疗效降低。在两项3期研究中,每周注射一次的长效人生长激素索马戈,与生长激素相比,已证明临床非劣效且对生活的干扰(即治疗负担)显著更低。这项研究从爱尔兰医保支付方的角度评估了索马戈与dGH的成本效益和成本效用。
针对3至12岁开始接受索马戈或dGH治疗并持续至接近成人身高(NAH)的患者,建立了一个马尔可夫模型,生长情况由基于试验的身高增长速度(HV)和特定治疗的依从性驱动。患者可在第1年末停药(4%)。DGH的依从性(治疗期间为95.3%-65%)和依从性与生长的关系基于已发表的证据。索马戈与dGH相比,第1年依从性更高,为4%,并随时间逐渐降低,这基于临床会诊。治疗成本、监测成本以及因不同浪费类型(设备设置和依从性)产生的成本均来自本地数据。基于身高和注射频率的健康效用值来自已发表的文献。进行了情景分析、确定性和概率敏感性分析。
在评估的基础病例和情景中,与dGH相比,索马戈治疗使NAH增加了1.87-3.66厘米,质量调整生命年(QALY)提高了0.21-0.50。与dGH相比,索马戈治疗使每位患者节省成本5699-21974欧元,每增加1厘米身高的成本更低(197-527欧元)。与dGH相比,索马戈具有成本效益性,在进行的敏感性分析中结果一致。
在pGHD中,估计每周注射索马戈比dGH能带来更高的NAH、更高的QALY、更低的总体成本以及每增加1厘米身高更低的成本。