District 18 News
Written by: Lu Ningmin
2021-01-03 17:31
Last update date: 2021-01-03 17:32
Spinal Muscular Atrophy (SMA) is a disease caused by mutations in chromosomal genes. Due to the degeneration of motor neurons, patients gradually shrink their muscles and lose their mobility.
This disease was once incurable. In recent years, a new drug, Nusinersen, has been developed. However, the drug has been registered in Hong Kong for more than two and a half years, but only a dozen children and one adult patient have been arranged to take the drug.
The Hong Kong Alliance for Rare Diseases today (3rd) urged the government to take the lead in a commercial drug program involving government, business, and civilians to jointly implement the "Orphan Drug Trial Period" program, so that patients do not need to pay for the drug during the trial period. The medication is funded by the current safety net mechanism, and the new measures against rare diseases in the policy report have been implemented.
24-year-old Huang Jingru was diagnosed with type 2 spinal muscular atrophy when she was 1 year old. In recent years, her lung function has deteriorated. She hopes to try the medicine as soon as possible to maintain her physical strength and continue to work.
(Respondents provide pictures)
There are many patients with spinal muscular atrophy in Hong Kong who are self-reliant and strive to maintain their quality of life. 24-year-old Huang Jingru is one of them.
Suffering from type 2 spinal muscular atrophy, she is currently working as a customer service clerk in a government agency. She described her condition worsening in recent years, especially her lung function has deteriorated sharply. Everyone tells me that if you care about your relatives, you will cough and sputum."
Jingru said that she had to lie down all over to cough up the sputum, "but when I go back to work or go out of the street, I may suddenly cough."
Although she is ill, Jingru still insists on going to work to reduce the financial burden at home, so she hopes to try the medicine as soon as possible to maintain her physical strength and continue to work. It’s so difficult to return to work for a long time, and I have to stay at home to take care of me. There are many follow-up questions."
Patients want to test drugs to maintain their quality of life
Yan Zhaoming, 29, is also a type 2 spinal muscular atrophy. He works as a program developer and is the breadwinner of his family.
"I have poor mobility this year, even typing and writing are difficult. I hope that after taking the medicine, I will maintain my current physical condition." You Jiamin, 39, also a patient, works as a social worker and is currently a doctoral student. She is committed to Promote the rights and interests of patients with spinal muscular atrophy, especially support for adult patients. "Many adult patients have jobs, and their work ability is so high, so this class needs a trial period plan."
Nusinersen, a drug for the treatment of spinal muscular atrophy, has been registered in Hong Kong for more than two and a half years. Ou Yingzhi, a clinical consultant in the Department of Neurology of the Department of Internal Medicine and Drug Therapeutics of the Faculty of Medicine, CUHK, said that currently only one adult patient in Hong Kong has been arranged for medication, or Because the authorities did not have drug scientific research data and clinical guidelines to determine whether patients should be suitable for the drug, she cited two international research reports that nearly 70% of patients with spinal muscular atrophy have achieved significant results after 14 months of drug treatment, and no serious side effects were involved. It is recommended that the trial period of drugs used in Hong Kong can be set at 14 months.
Zeng Jianping, president of the Hong Kong Rare Disease Alliance, said that the drug has been registered in Hong Kong for more than two and a half years, but only more than a dozen children and one adult patient have been arranged for the drug. He questioned the authorities for the lack of scientific research data and clinical guidelines. Implement a drug trial plan for patients.
(Respondents provide pictures)
Lifetime treatment costs up to 4 million US dollars
Dr. Xue Li, an assistant professor in the Department of Pharmacology and Pharmacy of the Li Ka-shing School of Medicine of the University of Hong Kong, who specializes in orphan drug policy, said that the lifetime treatment cost of patients with spinal muscular atrophy can be up to 4 million US dollars, and the "orphan drug trial period" of such drugs There are precedents in foreign countries. The government, doctors, patient organizations and pharmaceutical companies jointly set the criteria for the trial period, including the trial time, number of people, and efficacy evaluation methods. The drug factory provides free or low-cost drugs for patients to use during the trial period. After the trial period is over, the government will subsidize patients with curative effects and financial needs to continue to take the medicine, so that patients can receive treatment as soon as possible.
Li estimates that there are currently no more than 200 adult and child patients in Hong Kong. Therefore, it is expected that the authorities will not need to use huge manpower to implement the orphan drug trial program.
Doubts that the authorities "sigh slow" for lack of scientific research data and clinical standards
Zeng Jianping, president of the Hong Kong Rare Disease Alliance, said that the drug has been registered in Hong Kong for more than two and a half years, but only more than a dozen children and one adult patient have been arranged for the drug. He questioned the authorities for the lack of scientific research data and clinical guidelines. Implement a drug trial plan for patients, "I believe that the frontline doctors are very important. The problem is only at the management level. It is only possible to treat this disease temporarily, but there will not be only one drug to be marketed in the future. If the government is willing To do so, subsidizing the price of rare drugs is even beneficial to the government." He urged the government to negotiate with the pharmaceutical companies as soon as possible to implement the details of the specific plan.
The orphan drug policy does not cover as much as the low-income country organization urges the policy report to implement the orphan drug mechanism
Rare diseases | Zeng Jianping urges Lam Cheng to implement the safety net mechanism of rare disease drugs and set up a steering committee to follow up
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Rare Disease Policy Address 2020