ランダム化された臨床試験を非ランダム化された実世界のエビデンス研究で模倣する:RCT DUPLICATEイニシアチブの最初の結果
Jessica M Franklin1, Elisabetta Patorno1, Rishi J Desai1
1Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA (J.M.F., E.P., R.J.D., R.J.G., A.P., L.G.B., H.L., N.G., S.S.).
Circulation
|December 17, 2020
まとめ
リアルワールド・エビデンス (RWE) 研究はランダム化制御試験 (RCT) を真似ることができますが,合意は異なります. RWEエミュレーションは,心血管のアウトカムに関するRCTと良好な一致を示し,医療製品の評価におけるRWEの役割を支持しました.
科学分野:
- 流行病学
- 健康経済と成果研究
- 臨床試験の設計
背景:
- 規制機関は,医療製品の有効性評価のための非介入的実用研究 (RWE) を検討しています.
- RCT DUPLICATEイニシアチブは,結果を比較するために実際のデータを用いてランダム化制御試験 (RCT) を模倣します.
研究 の 目的:
- 現実のクレームデータを用いてRCTの真似の実現性と正確性を評価する.
- 抗糖尿病薬と抗血小板薬の心血管的アウトカムを,RWE研究と対応するRCTを比較することによって評価する.
主な方法:
- アメリカの商業およびメディケアの請求データを用いて,10件のRCT (3例はアクティブ対照,7例はプラセボ対照) を再現した.
- 実施されたRCTの含有/除外基準,エンドポイント,比較集団
- 120以上の事前に指定された混同因子に対する使用傾向スコアマッチング;将来的に定義された測定値と登録されたプロトコル.
主要な成果:
- 10件のエミュレーションのうち6件で規制上の結論が合意された.
- 10件のうち8件で,RWEによる危険比率はRCTの95%CIの範囲内であった.
- 10件の複製のうち9件は,規制または推定合意の成功基準を満たし,そのほとんどは効果の推定値にわずかな違いを示した.
結論:
- RCTとRWEの発見の一致は,使用されるメトリックに依存する.
- 類似の指示と使用パターンのアクティブ比較器を使用すると,RWEの有効性が向上します.
- RWE-RCT合意の一貫性と文脈を決定するために,さらなる試行エミュレーションが必要である.
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