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冠動脈3枝病変患者に対するPCSK9阻害薬とスタチン併用療法の費用対効果評価

小寺, 聡 東京大学 DOI:10.15083/0002008316

2023.12.27

概要

【別紙2】

審査の結果の要旨

氏名 小寺 聡
本研究では、医療費が増加している日本で重要になっている費用対効果評価について検
討している。本研究の対象薬剤である PCSK9 阻害薬は LDL コレステロールを強力に低下
させる効果があり、虚血性心疾患に対して有効性が証明されているものの、高額な薬価が問
題となっている。PCSK9 阻害薬は費用対効果評価が必要な薬剤であるものの、日本での費
用対効果は不明であった。本研究では、冠動脈3枝病変の日本人患者において、マルコフモ
デルを用いて PCSK9 阻害薬とスタチン併用療法および標準治療(スタチン単剤)の費用お
よび効果を推定し、費用対効果評価を行った。費用対効果評価の結果は下記であった。
1.30 年の観察期間で、PCSK9 阻害薬とスタチン併用療法および標準治療の費用はそれぞ
れ 1,320 万円と 720 万円であった。PCSK9阻害薬併用療法の費用が標準治療より高い結果
であった。
2.30 年の観察期間で、PCSK9 阻害薬とスタチン併用療法および標準治療の質調整生存年
(QALY)は、それぞれ 8.95 および 8.47 であった。PCSK9 阻害薬併用療法の効果が標準
治療より大きい結果であった。
3.冠動脈3枝病変患者において、標準治療に対する PCSK9 阻害薬とスタチン併用療法の
増分費用効果比(ICER)は、QALY あたり 1350 万円(95%信頼区間 760 万〜2350 万円)
であった。PCSK9 阻害薬とスタチン併用療法の費用対効果が、閾値 500 万円を下回る可能
性は 0.0008%であった。標準治療と比較した際に、PCSK9 阻害薬併用療法の費用対効果が
低いことが示された。
4.ICER が QALY あたり 500 万円を下回るためには、PCSK9 阻害薬の年間価格を現在の
価格より 70%低い 184,000 円まで低下させる必要性が示された。
5.一元感度分析では、PCSK9 阻害薬の費用の変化が最も大きく ICER を変化させた。こ
のモデルでは、PCSK9 阻害薬の費用が費用対効果に最も影響することが示された。

6.シナリオ分析では、冠動脈3枝病変を合併した家族性高コレステロール血症患者におい
て、ICER は QALY あたり 340 万円であった。家族性高コレステロール血症患者に対して、
PCSK9 阻害薬併用療法は優れた費用対効果を示した。

以上、本論文は、日本の冠動脈3枝病変患者において、PCSK9 阻害薬併用療法の費用対
効果が低いことを明らかにした。本研究はこれまで検討が不十分であった日本の医療にお
ける費用対効果評価の発展に重要な貢献をなすと考えられる。
よって本論文は博士( 医学 )の学位請求論文として合格と認められる。

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