臨床成功に対する遺伝的証拠の影響の精製

Eric Vallabh Minikel¹, Jeffery L Painter^2,3, Coco Chengliang Dong⁴

¹Stanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA.

Nature

|April 17, 2024

まとめ

ヒトの遺伝的証拠は薬剤発見の成功率を大幅に高め遺伝子サポートによる薬剤メカニズムが承認される確率は2.6倍になりますより効果的な薬の開発には継続的な遺伝学的洞察が不可欠です

科学分野:

薬理学について
遺伝学
薬物開発

背景:

薬の発見と開発は高価で,失敗率は高い (臨床プログラムの約10%だけが承認される).
臨床開発の成功率を2倍にすることが示されています.
薬の開発の成功要因を理解する新たな機会を提供しています

研究の目的:

臨床薬の開発の成功と失敗を区別する特徴を特定するために,最近の遺伝的証拠を活用する.
薬剤メカニズムの成功確率に対する遺伝的サポートの影響を定量化する.

主な方法:

臨床開発プログラムの分析ヒトの遺伝的証拠の10年間の成長を利用する.
遺伝子サポートによる薬剤メカニズムの成功確率の推定
治療領域,開発段階,遺伝子信頼性,遺伝効果の大きさ,マイナーアレル頻度,発見年を含む相対的な成功に影響を与える要因の評価.

主要な成果:

遺伝子のサポートを受けた薬のメカニズムは遺伝子のサポートを受けていない薬のメカニズムの 2.6 倍もの成功確率を示しています
遺伝的サポートの成功率の利点は,異なる治療領域と開発段階によって異なります.
遺伝効果の大きさ,マイナーアレルの頻度,発見年数は最小限に左右されます.

結論:

薬の発見と開発の成功の強力な予測要因です
薬の開発の効率と効果を高めるために遺伝的洞察を利用する際には未開拓の大きな可能性が存在します
新しい標的を発見し,より効果的な治療法を開発するために,遺伝子データのさらなる探索は不可欠です.

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