{
  "abstract": "BACKGROUND: Tafamidis improves survival in patients with transthyretin amyloid cardiomyopathy (ATTR-CM); however, its cost raises concerns regarding healthcare sustainability. We evaluated the stage-specific cost-effectiveness of tafamidis in Japan. METHODS AND RESULTS: Individual patient-level data were reconstructed from published Kaplan-Meier curves from the ATTR-ACT long-term extension study. Time-dependent survival benefit between early (continuous tafamidis) and delayed (placebo-to-tafamidis) tafamidis initiation was quantified using restricted mean survival time (RMST). Stage-specific incremental cost-effectiveness ratios (ICERs) were estimated according to National Amyloidosis Centre (NAC) stage. Drug costs reflected the official Japanese reimbursement price, and quality-adjusted life years (QALYs) were derived from RMST gains. The primary endpoint was cardiovascular mortality. At maximum follow-up, RMST gains were 15.7, 13.1, and 1.2 months in NAC stage I, II, and III, respectively ICERs declined over time but remained above commonly accepted Japanese willingness-to-pay thresholds (5,000,000-15,000,000 yen/QALY). The lowest ICER was 47,000,000 yen/QALY in NAC stage I at 90 months. Sensitivity analyses showed that results were robust across utility assumptions, whereas drug cost was the principal determinant of cost-effectiveness. CONCLUSIONS: The survival benefit and economic value of tafamidis varied by disease stage. Earlier-stage patients derived the greatest benefit and economic value; however, ICERs remained above Japanese willingness-to-pay thresholds across all NAC stages. Earlier diagnosis may maximize clinical benefit, but substantial price reduction is likely required for sustainable healthcare in ATTR-CM.",
  "abstract_shown": true,
  "affiliations": [],
  "article_type": null,
  "arxiv": null,
  "author_total": 5,
  "authors": [
    {
      "name": "Yasufumi Nagata"
    },
    {
      "name": "Yosuke Nabeshima"
    },
    {
      "name": "Tetsuji Kitano"
    },
    {
      "name": "Masaaki Takeuchi"
    },
    {
      "name": "Masaharu Kataoka"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/7jt2m6a4h4n7p.bib",
  "cited_by": 0,
  "doi": "10.1253/circj.cj-26-0576",
  "fulltext_url": "https://www.jstage.jst.go.jp/article/circj/90/11/90_CJ-26-0576/_article",
  "id": "7jt2m6a4h4n7p",
  "issns": [
    "1347-4820",
    "1346-9843"
  ],
  "landing_url": "https://www.jstage.jst.go.jp/article/circj/90/11/90_CJ-26-0576/_article",
  "language": null,
  "licence": "CC-BY-NC-ND",
  "licence_label": "CC BY-NC-ND",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by-nc-nd/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "jstage-journal",
      "medicine/cardiovascular"
    ],
    [
      "openalex",
      "1312|Amyloidosis: Diagnosis, Treatment, Outcomes"
    ]
  ],
  "openalex": "W7214573421",
  "pdf_url": "https://www.jstage.jst.go.jp/article/circj/advpub/0/advpub_CJ-26-0576/_pdf",
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-23",
  "publisher": null,
  "ris": "https://research.draconitic.com/paper/7jt2m6a4h4n7p.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T12:17:24Z",
      "native_id": "10.1253/circj.cj-26-0576",
      "source": "jstage",
      "url": "https://www.jstage.jst.go.jp/article/circj/90/11/90_CJ-26-0576/_article"
    }
  ],
  "subjects": [
    {
      "field": "biochemistry",
      "origin": "openalex:1312|Amyloidosis: Diagnosis, Treatment, Outcomes",
      "subfield": "molecular-biology"
    }
  ],
  "title": "Stage-Specific Cost-Effectiveness of Early vs. Delayed Tafamidis for Transthyretin Amyloid Cardiomyopathy in Japan",
  "title_text": "Stage-Specific Cost-Effectiveness of Early vs. Delayed Tafamidis for Transthyretin Amyloid Cardiomyopathy in Japan",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T12:17:24Z",
  "url": "https://research.draconitic.com/paper/7jt2m6a4h4n7p",
  "venue": "Circulation Journal",
  "versions": []
}