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Written by: Chen Shuxia
2021-01-04 10:20
Last update date: 2021-01-04 10:20
The government introduced the first drug for spinal muscular atrophy (SMA) two and a half years ago. The chairman of the Hong Kong Rare Disease Alliance, Zeng Jianping, said in an interview with Hong Kong and Taiwan’s "Millennium" today (4th), but so far there are only more than 10 children The patient and an adult patient took medication. Zhou Peishan, who had issued an open letter to Chief Executive Carrie Lam Cheng Yuet-ngor, was one of them. However, dozens of adult patients were temporarily unable to take the medication because the Hospital Authority refused to prescribe.
He mentioned that the authorities often refuse on the grounds of insufficient scientific research data and clinical evidence. However, international studies have shown that 70% of adult patients have markedly improved their mobility after 14 months of medication. He suggested that the government implement a "rare disease drug trial period." , The Hospital Authority, clinical experts, patient organizations, and pharmaceutical companies jointly negotiate and formulate clinical guidelines, trial periods and efficacy indicators for drugs to help patients use drugs early.
Zhou Peishan, a patient with spinal muscular atrophy (SMA), once issued an open letter to Lam Zheng, beginning the authorities to pay attention to the plight of patients with rare diseases.
(Photo by Yu Junliang)
Zeng Jianping said that he believed that the failure to use the drug came from the clinical guidelines of the Hospital Authority, or due to insufficient data on the use of drugs. He mentioned that last year, a European study showed that 70% of adult patients showed significant improvement in their mobility after 14 months of drug use. ) May not be sure, and worry about risks."
Advocate that the Hospital Authority, experts and family members jointly formulate clinical guidelines for trial drugs
He suggested that the government implement a "rare disease drug trial period" in which the Hospital Authority, clinical experts, patient organizations and pharmaceutical companies jointly negotiate and formulate clinical guidelines, trial periods and efficacy indicators for drugs, hoping to establish a joint risk-taking mechanism.
During this period, patients do not need to pay for the medicine. After the trial period is over, patients who fail to meet the curative effect will stop taking the medicine, and the patients who meet the standard will be funded by the current mechanism to continue to take the medicine.
He pointed out that the alliance had communicated plans with the same pharmaceutical company. Although the other party did not mention the free drug supply, the response was positive.
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