Challenges in orphan drug development and regulatory policy in China.

摘要: While regulatory policy is well defined for orphan drug development in the United States and Europe, rare disease China still evolving. Many Chinese patients currently pay out of pocket international treatments that are not yet approved China. The lack a clear definition therefore approval process diseases has, until now, de-incentivized pharmaceutical companies to pursue In turn, many grassroots movements have begun support facilitate discovery through research. Recently, FDA set new guidelines drugs being developed China, including an expedited review life-saving treatments. this review, we discuss effects these changes on suggest potential solutions innovate