Rare Diseases: What Makes China A Rising Star?
Top corporate executives are increasingly turning their attention to rare diseases in China as changing regulatory policies provide a rare opening, and aided by more physicians advocating for orphan drug access. What does the first ever report on rare diseases in China have to say about the opportunities and challenges?
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Without automatic reimbursement in place, makers of orphan and rare disease drugs must look at innovative alternative ways to make their high-priced new therapies accessible to patients in China.
In a busy week for new drug approvals in China, the NMPA hands out nods for anticancers Elunate and Lenvima, rare disease treatment Soliris, and an additional indication for Humira, with Elunate being hailed by developer Chi-Med as the first fully "Made in China" novel oncology drug to receive unconditional approval.
Largely different from the US and EU, China is tackling rare diseases with a first-ever official list of 121 conditions for now, which could be a major milestone for orphan drug developers, some experts say.