承認後の新薬や新器具に関する比較証拠の作成

Andrea Cipriani¹, John P A Ioannidis², Peter M Rothwell³

¹Department of Psychiatry, University of Oxford, Oxford, UK; Oxford Health NHS Foundation Trust, Warneford Hospital, Oxford, UK.

Lancet (London, England)

|March 23, 2020

まとめ

医薬品とデバイスの市場公開後の研究は,しばしば堅実な比較データがない. ガイドラインの実施は,メーカーはよりよい利益リスク評価のために,患者に関連した証拠を時宜に生成するよう促すことができます.

科学分野:

医薬品と医療機器
規制科学
臨床研究

背景:

医薬品とデバイスの証拠のギャップは,市場承認後も頻繁に存在します.
現在行われている市場投入後の研究は,しばしば断片化されており,研究には有効な比較手段,患者に関連する結果,または適時完了が欠けている.
市場投入後の研究に関する規制要件は,市場投入時に存在する制限を適切に解決できない場合があります.

研究の目的:

薬剤や器具の製造業者に対し,販売後の比較データを作成するための7つの指針を提案する.
薬剤と器具の利益リスクプロフィールに関する未解決の重要な問題の迅速な解決を奨励する.
販売後の証拠の質と妥当性を向上させる.

主な方法:

市場投入後の証拠の生成のための7つの主要な指針のセットの開発
規制機関,医療技術評価機関,支払業者に対して,カスタマイズされた証拠計画を作成することを強調する.
アクティブな比較器を用いたランダム化試験を優先する階層的な試験設計の提案

主要な成果:

原則は,事前承認データの制限に対処するカスタマイズされた証拠生成計画を提唱します.
純臨床効果を評価するために,活性比較薬を用いたランダム化試験の優先順位付け.
ランダム化されていない研究を制限し,革新的な設計を通じて試験効率を改善するための勧告
研究ネットワークとデータシステムへの政府投資の呼びかけ
データの生成を確実にするための金銭的インセンティブと罰則の提唱

結論:

提案された原則の実施は,製造業者に対し,高品質で比較可能な販売後のデータを提供するよう促すことができる.
薬剤と器具の利益リスクの正確な評価には,市場投入後の段階での証拠の欠陥を解決することが不可欠です.
規制当局,業界,患者間の共同努力は,適時かつ適切な承認後の研究に不可欠です.

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