Democratic Progressive Party Legislator Chen Man-li (陳曼麗) and family members of spinal muscular atrophy (SMA) patients yesterday urged the government to include SMA medication in the National Health Insurance (NHI) system.
More than a dozen children with SMA and their family members — holding signs with slogans such “I want to live on, please give me medicine,” “I don’t want a distorted life” and “please save SMA children, time is not our friend” — gathered at the Legislative Yuan to call for government action.
“President Tsai, please give use medicine to save us. We want to live on,” they chanted together.
Photo: CNA
SMA, a rare genetic neuromuscular disorder that most often affects infants and children, is characterized by weakness and atrophy in skeletal muscles, and is caused by a loss of specialized nerve cells called motor neurons that control muscle movement.
The severity of symptoms and the age of onset varies by type, and it is a leading genetic cause of death in infants.
Previously, there were no effective medicines for the disease and only treatments that might help manage the condition, until a new medication, nusinersen, marketed as Spinraza, was developed in the US in December of 2016, Chen said.
After US Food and Drug Administration approved nusinersen as treatment for SMA, 39 countries followed in funding the medication, and it has also been approved by the Food and Drug Administration (FDA) in Taiwan, she said.
While there are approximately 400 people with SMA in Taiwan, they urged the National Health Insurance Administration (NHIA) to include the new drug in its discussions for deciding NHI-funded treatments, Chen said.
A mother of a child with SMA said her nearly two-year-old son was diagnosed with SMA type 1 before he was one month old, and he was not able to lift his neck and barely able to turn over his body at five months.
Her son currently receives medicine funded by a pharmaceutical company until he is two years old, as he is eligible for a compassionate use program, she said, adding that he can now run and walk normally for his age.
However, the mother said she hopes the treatment could be funded by the NHI system, as he would need to continue its use.
Infants with serious SMA have an average life expectancy of less than two years, but as parents see children able to run after receiving treatment in the compassionate use program, they now have hope, said Lee Yi-chie (李怡潔), convener of the Alliance for Striving for Rare Disease SMA Medication.
Many patients cannot afford to wait, as muscle atrophy can be irreversible, so they hope the NHI system can fund Spinraza as soon as possible, she said.
Currently, the FDA license for Spinraza is for SMA patients under two years old or patients with two copies of the motor neuron 2 gene, and it is not available for patients using continuous positive airway pressure for more than 12 hours per day and exceeding 30 days, so eligibility should be the same if NHI funds the medicine, the NHIA said.
Moreover, the agency said it has been negotiating with the pharmaceutical company on the price of the drug, in an effort to include it in the NHI-funded treatment items discussion meeting next month.
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