title: Challenges in orphan drug development and regulatory policy in China.
abstract: While regulatory policy is well defined for orphan drug development in the United States and Europe, rare disease policy in China is still evolving. Many Chinese patients currently pay out of pocket for international treatments that are not yet approved in China. The lack of a clear definition and therefore regulatory approval process for rare diseases has, until now, de-incentivized pharmaceutical companies to pursue rare disease drug development in China. In turn, many grassroots movements have begun to support rare disease patients and facilitate drug discovery through research. Recently, the Chinese FDA set new regulatory guidelines for drugs being developed in China, including an expedited review process for life-saving treatments. In this review, we discuss the effects of these new policy changes on and suggest potential solutions to innovate orphan drug development in China.
authors.affiliation: Rare Genomics Institute, 2657 Annapolis Road, Hanover, MD, 21076, USA. alicec33@gmail.com.
authors.forename: Alice
authors.initials: A
authors.lastname: Cheng
authors.affiliation: Rare Genomics Institute, 2657 Annapolis Road, Hanover, MD, 21076, USA.|State Key Lab of Ophthalmology, Zhongshan Ophthalmic Center, Sun Yat-sen University, 54 Xianlienan Road, Guangzhou, 510040, Guangdong, China.
authors.forename: Zhi
authors.initials: Z
authors.lastname: Xie
mesh_terms: D002681:China; D002986:Clinical Trials as Topic; D017277:Drug Approval / Q000331:legislation & jurisprudence; D030342:Genetic Diseases, Inborn / Q000188:drug therapy; D006801:Humans; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*; D035583:Rare Diseases / Q000188:drug therapy
keywords: Chinese medicine; Clinical trial; Genetic disease; Orphan drugs; Rare disease; Regulatory policy