画期的治療薬
画期的治療薬（Breakthrough therapy）は、アメリカ合衆国の2012年7月9日の法律FDA安全及びイノベーション法の902項を根拠として整備された、アメリカ食品医薬品局 (FDA) の医薬品開発迅速化制度のことである[1][2]。この「画期的」（Breakthrough）とは、臨床試験において特定の疾患に対する顕著な治療効果が確認できたというわけではなく[3][4]、重篤あるいは致命的な病気のために、既存の治療選択肢よりも治療上の利益が大きいことを示す予備試験の臨床試験がある場合に、FDAは優先審査認めるということである[4][5]。
出典[編集]
- ^ Darrow, JJ; Avorn, J; Kesselheim, AS (27 March 2014). “New FDA breakthrough-drug category—implications for patients.”. The New England Journal of Medicine 370 (13): 1252–8. doi:10.1056/nejmhle1311493. PMID 24670173.
- ^ Yandell, Kate (2016年1月1日). “Picking Up the Pace”. The Scientist
- ^ “CBS proclaims 'cancer breakthrough' – doesn't explain what FDA means by that term”. Health News Review. (2016年5月13日) 2017年10月9日閲覧。
- ^ a b “FDA's Expedited Approval Mechanisms for New Drug Products”. Biotechnol Law Rep 34 (1): 15–37. (February 2015). doi:10.1089/blr.2015.9999. PMC 4326266. PMID 25713472. "The fourth expedited approval mechanism—the Breakthrough Therapy designation—applies to a new drug product if it "is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints."172 In its Final Guidance, FDA has interpreted "preliminary clinical evidence" to mean evidence "sufficient to indicate that the drug may demonstrate substantial improvement in effectiveness or safety over available therapies, but in most cases is not sufficient to establish safety and effectiveness for purposes of approval."173 This generally will require more than data from in vitro studies or animal models.174 FDA expects preliminary clinical evidence to come from Phase 1 or 2 clinical trials.175"
- ^ “Fact Sheet: Breakthrough Therapies”. United States Food and Drug Administration (2014年12月10日). 2017年10月9日時点のオリジナルよりアーカイブ。2017年10月9日閲覧。 “A breakthrough therapy is a drug:
・intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and
・preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.”