China's FDA is offering to grant conditional OKs for orphan meds already approved abroad, even without in-China trial data. The new orphan drug move is one in\u00a0a series of new proposals posted online May 11. China\u2019s FDA is once again planning\u00a0major changes to its clinical trial and drug approval policies, further aligning them with its Western counterparts. A key feature is the new \u201cconditional marketing authorization\u201d for orphan meds.<\/p>\n
The CFDA is offering to grant conditional approval for meds that treat life-threatening conditions where significant unmet medical needs exist, if early- or mid-stage data can predict the drugs\u2019 clinical benefits. This policy resembles the EMA's \u201cconditional marketing authorization\u201d and the U.S. FDA\u2019s \u201cbreakthrough therapy\u201d program.<\/p>\n
The CFDA first listed the policy as part of its expedited review program rolled out last February, but the agency goes further this time by expanding it to cover orphan meds already approved in foreign countries, even those\u00a0without any trial data from China. As in the U.S. and Europe, drug-makers would be required to conduct confirmatory trials or follow-up studies as agreed with regulators.<\/p>\n
This is not entirely new territory for the CFDA. The agency conditionally approved Actelion\u2019s Zavesca (miglustat) for Niemann-Pick disease type C in 2013, based entirely on trial data obtained in Western countries. The Zavesca\u00a0data had been generated\u00a0for a European approval application, and the Chinese review included\u00a0post-marketing data as well. The CFDA asked for a follow-up trial on at least 15 patients in China to confirm its efficacy and safety. After that, the med gained official approval last year.<\/p>\n
But this is the first time CFDA has put the rare disease policy in writing. If implemented, it stands to increase new drug applications from\u00a0orphan drug-makers intent on gaining increased access to a pharma market that, despite economic woes, remains one of the world's fastest-growing.<\/p>\n
Other proposed policy shifts\u00a0include allowing foreign study data for Chinese NDAs, changing the clinical trial regulatory management system from certification to registration, and converting clinical trial approval to a 60-working-day response window. (Source: FiercePharma)<\/p>\n","protected":false},"excerpt":{"rendered":"
China's FDA is offering to grant conditional OKs for orphan meds already approved abroad, even without in-China trial data. The new orphan drug move is one in\u00a0a series of new proposals posted online May 11. China\u2019s FDA is once again planning\u00a0major changes to its clinical trial and drug approval policies, further aligning them with its Western counterparts. A key feature is the new \u201cconditional marketing authorization\u201d for orphan meds. The CFDA is offering to grant conditional approval for meds that treat life-threatening conditions where significant unmet medical needs exist, if early- or mid-stage data can predict the drugs\u2019 clinical benefits. This policy resembles the EMA's \u201cconditional marketing authorization\u201d and the... Read more »<\/a><\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-178","post","type-post","status-publish","format-standard","hentry","category-medicalnews"],"aioseo_notices":[],"_links":{"self":[{"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/posts\/178","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/comments?post=178"}],"version-history":[{"count":3,"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/posts\/178\/revisions"}],"predecessor-version":[{"id":181,"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/posts\/178\/revisions\/181"}],"wp:attachment":[{"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/media?parent=178"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/categories?post=178"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.ssri.com\/china\/e\/medicalnews\/wp-json\/wp\/v2\/tags?post=178"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}