Faster rare disease drugs saving lives
Print Edition
Two-and-half-year-old Xuanxuan survived a rare kidney disease in early December, thanks to fast-track approval of the transplant.
The baby was diagnosed with Denys-Drash syndrome, a fatal disease with fewer than 500 cases reported worldwide. His symptoms worsened in November, but he was not eligible for a transplant at the time.
Finally, local pediatricians in southwest China鈥檚 Sichuan Province applied to the national transplant ethics commission and obtained special permission for the transplant.
This is part of China鈥檚 intensified efforts to rescue about 20 million patients with rare diseases.
More than 200,000 people in China are diagnosed with such diseases every year, often struggling due to limited treatment methods and sky-high medical expenses.
To speed up drug approvals, the National Medical Products Administration is designating rare disease medicines with a priority review. More than 60 rare-disease drugs have been approved for marketing, according to official data.
The country鈥檚 national drug regulator has pledged to greenlight rare disease medicines 鈥 with urgent clinical needs 鈥 cleared in overseas markets but which have not yet been marketed in China within 70 days.
Among the 81 overseas drugs with urgent clinical needs listed by the NMPA, more than half are for rare diseases, and 26 have already been approved, said Chen Shifei, deputy head of the NMPA.
Recently, a tear-jerking video showing how a national health insurance negotiator helped cut down the bidding price of Spinraza from over 53,680 yuan (US$8,422) per dose to 33,000 yuan went viral online and was lauded by netizens. Spinraza, a medicine for spinal muscular atrophy, was once priced at a whopping 700,000 yuan.
In the video, the female negotiator is seen fervently chasing a lower price.
鈥淗ave you given your best?鈥 鈥淎lready tried your utmost?鈥 鈥淚 hope you can do more!鈥 鈥 she said repeatedly.
Maomao, a three-year-old SMA patient in east China鈥檚 Jiangxi Province, will be benefiting from this deal.
鈥淚t took just two years for the medicine to be included in the reimbursement list once it was approved,鈥 said Maomao鈥檚 mother. 鈥淚t鈥檚 a miracle.鈥
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