China's Latest Western-Style FDA Tweak Puts Orphan Drug Approvals

China's FDA, long criticized for its cumbersome and slow drug-review process, 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 a series of new proposals posted online May 11. China’s FDA is once again planning major changes to its clinical trial and drug approval policies, further aligning them with its Western counterparts. A key feature is the new “conditional marketing authorization” for orphan meds.

The CFDA is offering (Chinese) 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’ clinical benefits.

This policy resembles the EMA's “conditional marketing authorization” and the U.S. FDA’s “breakthrough therapy” program, which itself is just short of 5 years old. The “breakthrough therapy” designation is granted to a med where preliminary clinical evidence can already indicate the drug's substantial treatment effects. Since the program's initiation in 2012, 38 new breakthrough drugs have been approved in the U.S., including five so far this year. 

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 without any trial data from China.

Just as they do in the U.S. and Europe, drugmakers would be required to conduct confirmatory trials or followup studies as agreed with regulators.

This is not entirely new territory for the CFDA. The agency conditionally approved Actelion’s Zavesca (miglustat) for Niemann-Pick disease type C in 2013, based entirely on trial data obtained in Western countries.

The Zavesca data had been generated for a European approval application, and the Chinese review included postmarketing 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.

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 orphan drugmakers intent on gaining increased access to a pharma market that, despite economic woes, remains one of the world's fastest-growing.

Other proposed policy shifts include 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.