FEATURE/Chen Chun-han death highlights appeal for full SMA drug coverage

By Sunny Lai, CNA staff reporter

When Chen Chun-han (陳俊翰) died on Feb. 11, he left behind a legacy of advocacy for the rights of people with disabilities.

Within weeks after his death, it appears that action might finally be taken on one of Chen's main appeals: providing drug coverage to all spinal muscular atrophy (SMA) patients in Taiwan.

Chen was one of about 400 people in Taiwan afflicted with SMA, a rare neuromuscular genetic disease that affects a person's central and peripheral nervous systems and leads to the degeneration of muscles and respiratory organs.

By the time he died at age 40, Chen could only move his eyes, mouth and a pinkie finger, yet he, and many like him, have not been eligible to have Taiwan's national health insurance (NHI) system fully cover the costs of incredibly expensive SMA drugs.

He campaigned to change that, and Taiwan's National Health Insurance Administration (NHIA) announced, just four days after Chen died, that it would consider the possibility.

At what cost life?

Three kinds of drug therapies for SMA are currently available and partially covered under the NHI system: a gene therapy drug marketed as Zolgensma, an intrathecal injection (into the spinal canal) known as Spinraza, and a liquid oral medication called Evrysdi.

Zolgensma is a one-time-only drug targeted exclusively at children up to 2 years old. The NHI currently covers the drug's NT$49 million (US$1.55 million) cost for infants aged six months and younger, when the drug is supposed to be most effective.

Spinraza and Evrysdi, which can cost NT$6 million and NT$5 million a year, respectively, are for older patients, but the NHI only covers the cost for those who score a 15 or higher on the Revised Upper Limb Module (RULM) assessment, signifying some range of arm mobility.

The threshold was set, according to Lee Wei-te (李威德), secretary-general of Taiwan Muscular Dystrophy Association (TMDA), to get treatment to those patients likely to benefit, especially given the high price of the medicines.

That threshold has remained in place despite the efforts of Chen and others in previous years, putting the medications beyond the financial reach of some who need them.

According to data provided by the NHIA, 158 SMA patients had applied for Spinraza or Evrysdi treatments by the end of 2023, well short of the 400 in Taiwan because of concerns about the drugs' side effects and procedures as well as RULM threshold, Lee said.

Of the 158, 145 had passed the RULM assessment.

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One of them was Yang Po-yu (楊博宇), a postdoctoral researcher at Academia Sinica, the same organization where Chen used to work.

The 36-year-old told CNA that he could feel the decline in physical abilities becoming more significant with age, but started using the drug when his RULM score was 16.

Since mid-2023, he has received five intrathecal injections, each costing NT$2 million (US$63,300). He will continue to be hospitalized one day every four months for the rest of his life to get a Spinraza shot.

Asked how the therapy was working, Yang, sitting in a wheelchair while using his mobile phone adeptly with both hands, said he and other SMA patients have seen slowdowns in their physical declines.

"In fact, I may be seeing some improvement, and others have experienced more obvious improvement," Yang said.

That is important, Yang said, because "the people at the hospital see those of us in the first 100 (SMA patients) receiving treatment as paving the way for the remaining 300."

Though he made the cut, Yang said the existing eligibility system is "entirely unreasonable."

"Some have suggested that someone as accomplished and capable as Chen, if treated earlier, might not have been so vulnerable as to be taken away by a simple cold," Yang said, referring to the cold-related complications that are suspected of causing Chen's death.

A 'cruel' eligibility system

Unlike Yang, Hsu Chan-jung (許展容) fell short on the RULM assessment with a score of 8, and the lack of access to the medication has left her grasping for hope, she told CNA.

"In the early stages, my hope was to walk. Later, it was standing, then simply hoping to sit well. As my physical abilities declined, I had a glimmer of hope when the new medication became available," said Hsu, who struggles a bit when sending text messages on her phone.

Hsu called the eligibility criteria "very cruel" because they minimize the value of movements SMA patients can make and deprive them of hope.

"I don't aspire to be normal like everyone else. I simply pray that my condition doesn't get worse," said the 50-year-old pursuing a master's degree in social enterprise at Fu Jen Catholic University.

If she has drawn inspiration, it has been from Chen. She said that at a press conference attended by both Chen and her, he told her he could have stayed in the United States to get treatment but decided to return to Taiwan in 2022 to advocate for the drug rights of SMA patients.

"He may not even have had two points [in the RULM assessment], but he was willing to come back. This is a very strong and powerful encouragement for us. Even in his condition, he didn't give up, so why should we give up?" Hsu said.

Hope on the way?

On Feb. 15, Huang Yu-wen (黃育文), director of the NHIA's Medical Review and Pharmaceutical Benefits Division, said the manufacturers of Spinraza and Evrysdi have applied to expand NHI coverage to include all SMA patients.

A panel of experts will discuss the issue in March, and then meet with the NHIA, and approval for full coverage could come as early as in the first half of 2024, Huang said.

Lee believes a positive outcome is possible.

"I think there is already a consensus...I don't think there's any reason for the Ministry of Health and Welfare to impose further restrictions," Lee said, confident that funding should be adequate to cover the new expenses.

Lee, who has witnessed the passing of many SMA patients due to a breath going wrong or a bit of phlegm getting stuck, sees the SMA drug issue as about far more than just dollars and cents.

"The value of Chun-han's life cannot be measured in terms of money, right?" Lee said.

"Comprehensive medication coverage for all SMA patients will empower more individuals with SMA to showcase their potential and value in life -- that is what Chun-han aimed to achieve," Lee said.

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