画期的治療薬

画期的治療薬（英: 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（英語版）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。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、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（英語版）認めるということである。 FDAには有望な医薬品の審査および承認プロセスを迅速化するためのメカニズムを他にも有しており、これにはファスト・トラック（英語版）指定、迅速承認、および優先審査（英語版）が含まれる。 画期的治療薬の指定は、薬剤が「単独または他の薬剤と組み合わせて、重篤または生命を脅かす病気や状態を治療することを目的としている」ときに付与される。また、初期の臨床試験の結果から、臨床的に重要なエンドポイント（例えば、早期の臨床開発で観察される著しい治療効果）において既存の治療法よりも大幅な改善を示す可能性がある場合に付与される。 申請は、医薬品評価研究センター（英語版）（CDER）と生物製品評価研究センター（英語版）（CBER）によって審査される。CDERは年間約100件の画期的治療薬指定の申請を受けており、その約3分の1が承認されている。CBERは年間15～30件の申請を受けている。 画期的治療薬指定申請は、通常、第II相臨床試験終了前に、IND申請（新薬臨床試験開始申請）の修正として提出される。 ^ 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. http://www.nejm.org/doi/full/10.1056/NEJMhle1311493.  ^ Yandell, Kate (1 January 2016). “Picking Up the Pace”. The Scientist. http://www.the-scientist.com/?articles.view/articleNo/44773/title/Picking-Up-the-Pace/  ^ “CBS proclaims 'cancer breakthrough' – doesn't explain what FDA means by that term”. Health News Review. (13 May 2016). http://www.healthnewsreview.org/2016/05/cbs-proclaims-cancer-breakthrough-doesnt-explain-what-fda-means-by-that-term/ 9 October 2017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. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4326266/. "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 (10 December 2014). 9 October 2017時点のオリジナルよりアーカイブ。9 October 2017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.” ^ “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. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4326266/. "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"  ^ “Frequently Asked Questions: Breakthrough Therapies”. Food and Drug Administration. 21 January 2017時点のオリジナルよりアーカイブ。3 January 2017閲覧。 ^ “Fact Sheet: Breakthrough Therapies”. United States Food and Drug Administration (10 December 2014). 9 October 2017時点のオリジナルよりアーカイブ。9 October 2017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.” ^ Food and Drug Administration Safety and Innovation Act (FDASIA), Silver Spring, MD: Food and Drug Administration, (29 January 2015), オリジナルの18 July 2015時点におけるアーカイブ。, https://web.archive.org/web/20150718082406/https://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDASIA/ 20 July 2015閲覧。  ^ “Expedited Programs for Serious Conditions––Drugs and Biologics”. U.S. Food and Drug Administration (10 April 2019). 2024年9月19日閲覧。 画期的治療薬の指定を受けた医薬品の一覧（英語版）