Guidelines for clinical evaluation of anti-cancer drugs.

{"article_title":"Guidelines for clinical evaluation of anti-cancer drugs.","author":"Minami H, Kiyota N, Kimbara S, Ando Y, Shimokata T, Ohtsu A, Fuse N, Kuboki Y, Shimizu T, Yamamoto N, Nishio K, Kawakami Y, Nihira SI, Sase K, Nonaka T, Takahashi H, Komori Y, Kiyohara K","journal_title":"Cancer science","issn":"1349-7006","isbn":"","publication_date":"2021-07-01","volume":"112","issue":"7","first_page":"2563","page_count":"","accession_number":"33990993","doi":"","publisher":"Wiley Publishing on behalf of the Japanese Cancer Association","doctype":"Guideline","subjects":"Japan; Antineoplastic Agents therapeutic use; Clinical Studies as Topic standards; Antineoplastic Agents pharmacology; Drug Development organization & administration; Drug Development standards; Humans; Japan; Neoplasms drug therapy; Rare Diseases drug therapy; Treatment Outcome","interest_area":["Oncology"],"abstract":"Clinical studies intended for regulatory approval must demonstrate the clinical benefits of the drug in a target population. Clinical development of a drug proceeds by stepwise clinical studies; after safety and pharmacokinetics are evaluated and the recommended dosage and administration are determined, efficacy and safety are evaluated in an exploratory manner, and finally clinical benefits are compared with conventional standard therapies. Guidelines for the clinical evaluation of anti-cancer drugs in Japan were established in 1991 and amended in 2006 after molecular-targeted drugs were introduced. Recent progress in the development of drugs acting on the immune system and cancer genomic medicine targeting rare but important molecular subtypes have altered the strategy for development of anti-cancer drugs. It is often difficult to conduct a confirmatory randomized controlled study using overall survival as the primary endpoint in rare molecular subtypes, and the primary evaluation of the efficacy of some drugs and subsequent approval is based on the tumor response. As conducting clinical studies for rare subtypes solely within Japan is difficult, drug development needs to be conducted within a global study. However, this requires robust monitoring to detect possible ethnic differences in pharmacokinetics and drug efficacy. Development using the conditional approval system for drugs enforced in 2020 may be considered, when clinical utility is evaluated based on surrogate endpoints. Because of these changes, we have revised the guidelines for the clinical evaluation of anti-cancer drugs in Japan. To promote global development of anti-cancer drugs involving Japan, the guidelines have been translated into English. \u00a9 2021 The Authors. Cancer Science published by John Wiley & Sons Australia, Ltd on behalf of Japanese Cancer Association.","url":"https:\/\/search.ebscohost.com\/login.aspx?direct=true&db=mdl&AN=33990993","isPdfLink":true,"isSAML":false,"an":"33990993","number_other":"","type_pub":"","issn_electronic":"1349-7006","languages":"English","language":"eng","date_entry":"","date_update":"","titleSource":"Cancer science [Cancer Sci] 2021 Jul; Vol. 112 (7), pp. 2563-2577. Date of Electronic Publication: 2021 Jun 08.","date_pub_cy":"","type_document":"","contract_publisher":"","authored_on":"2021-07-01","description":"Clinical studies intended for regulatory approval must demonstrate the clinical benefits of the drug in a target population. Clinical development of a drug proceeds by stepwise clinical studies; after safety and pharmacokinetics are evaluated and the recommended dosage and administration are determined, efficacy and safety are evaluated in an exploratory manner, and finally clinical benefits are compared with conventional standard therapies. Guidelines for the clinical evaluation of anti-cancer drugs in Japan were established in 1991 and amended in 2006 after molecular-targeted drugs were introduced. Recent progress in the development of drugs acting on the immune system and cancer genomic medicine targeting rare but important molecular subtypes have altered the strategy for development of anti-cancer drugs. It is often difficult to conduct a confirmatory randomized controlled study using overall survival as the primary endpoint in rare molecular subtypes, and the primary evaluation of the efficacy of some drugs and subsequent approval is based on the tumor response. As conducting clinical studies for rare subtypes solely within Japan is difficult, drug development needs to be conducted within a global study. However, this requires robust monitoring to detect possible ethnic differences in pharmacokinetics and drug efficacy. Development using the conditional approval system for drugs enforced in 2020 may be considered, when clinical utility is evaluated based on surrogate endpoints. Because of these changes, we have revised the guidelines for the clinical evaluation of anti-cancer drugs in Japan. To promote global development of anti-cancer drugs involving Japan, the guidelines have been translated into English.<br \/> (© 2021 The Authors. Cancer Science published by John Wiley & Sons Australia, Ltd on behalf of Japanese Cancer Association.)","upload_link":"https:\/\/search.ebscohost.com\/login.aspx?direct=true&site=eds-live&db=mdl&AN=33990993&authtype=shib&custid=ns346513&group=main&profile=eds","no_of_pages":"","authored_by":"Minami H, Kiyota N, Kimbara S, Ando Y, Shimokata T, Ohtsu A, Fuse N, Kuboki Y, Shimizu T, Yamamoto N, Nishio K, Kawakami Y, Nihira SI, Sase K, Nonaka T, Takahashi H, Komori Y, Kiyohara K","header":{"DbId":"mdl","DbLabel":"MEDLINE Ultimate","An":"33990993","RelevancyScore":"892","PubType":"Academic Journal","PubTypeId":"academicJournal","PreciseRelevancyScore":"891.576171875"},"plink":"https:\/\/search.ebscohost.com\/login.aspx?direct=true&site=eds-live&db=mdl&AN=33990993&authtype=shib&custid=ns346513&group=main&profile=eds","physicalDescription":{"Pagination":{"StartPage":"2563"}},"additionalInfo":{"Authored_By":"Minami H, Kiyota N, Kimbara S, Ando Y, Shimokata T, Ohtsu A, Fuse N, Kuboki Y, Shimizu T, Yamamoto N, Nishio K, Kawakami Y, Nihira SI, Sase K, Nonaka T, Takahashi H, Komori Y, Kiyohara K","Journal_Info":"Publisher: Wiley Publishing on behalf of the Japanese Cancer Association Country of Publication: England NLM ID: 101168776 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1349-7006 (Electronic) Linking ISSN: 13479032 NLM ISO Abbreviation: Cancer Sci Subsets: MEDLINE","Publication_Type":"Guideline; Journal Article","Published_Date":"2021-07-01","Source":"Cancer science [Cancer Sci] 2021 Jul; Vol. 112 (7), pp. 2563-2577. Date of Electronic Publication: 2021 Jun 08.","Languages":"English","Electronic_ISSN":"1349-7006","MeSH_Terms":"Antineoplastic Agents\/*therapeutic use , Clinical Studies as Topic\/*standards, Antineoplastic Agents\/pharmacology ; Drug Development\/organization & administration ; Drug Development\/standards ; Humans ; Japan ; Neoplasms\/drug therapy ; Rare Diseases\/drug therapy ; Treatment Outcome","Subjects":"Antineoplastic Agents pharmacology, Drug Development organization & administration, Drug Development standards, Humans, Japan, Neoplasms drug therapy, Rare Diseases drug therapy, Treatment Outcome, Antineoplastic Agents therapeutic use, Clinical Studies as Topic standards","Title_Abbreviations":"Cancer science","Volume":"112"}}