Starting in August, the National Health Insurance (NHI) system would cover a drug for spinal muscular atrophy (SMA) for infants under six months old, National Health Insurance Administration (NHIA) Deputy Director-General Tsai Shu-ling (蔡淑鈴) said.
SMA is a rare neuromuscular genetic disease that affects the central nervous system, peripheral nervous system and voluntary muscle movements in patients, which results in degeneration of muscles in limbs and respiratory organs.
The one-time gene therapy drug, sold under the brand name Zolgensma, has a list price of US$2.1 million in the US.
However, the NHIA has negotiated the price down to NT$49 million (US$1.58 million) per dose, Tsai said.
It would be the first time the NHI has covered a gene therapy, Tsai said.
About eight children in Taiwan would benefit from the new policy every year, she said.
While the drug has proven effective among patients over a broader age range and has lifelong effects, its prohibitive cost has led the NHIA to initially make it only available for infants aged up to six months old, who have shown the most promising response to treatment, Tsai said.
However, she said it is difficult to guarantee that the medicine would have “lifelong” effects, as it is a new drug and follow-up checks with patients have been conducted for only the past eight years.
The NHIA would track the progress of Taiwanese who receive the drug and review cases from other countries to determine whether coverage should be expanded to other age groups, Tsai said.
Other SMA treatments include a spinal injection called Spinraza and a liquid oral medication called Evrysdi, both of which are covered by the NHI for children aged up to three years old, she said.
Patients older than three have to undergo revised upper limb module assessment, a test designed to determine the upper limb functions in SMA patients to see whether they are eligible for therapies covered by the NHI system, she added.
Tsai said she hopes the NHI’s coverage of therapies for the condition would benefit at least half of the 400 people with SMA in Taiwan.
