The National Health Insurance Administration (NHIA) has decided to cover an expensive, but effective gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, sparking debate over fairness and treatment access for patients with other rare conditions.
The one-time therapy, which costs about NT$100 million (US$3.28 million) per injection, would be covered on a temporary basis, the NHIA said on Tuesday.
Its effectiveness would be reassessed three years after treatment begins, and coverage terms may be adjusted as needed.
Photo: Taipei Times
AADC deficiency affects infants and young children, causing severe developmental delays and autonomic dysfunction.
Without treatment, many patients die between the ages of two and five, but the therapy can significantly improve motor function and quality of life.
The treatment was developed by National Taiwan University Hospital (NTUH) in 2007, with clinical protocols established in 2010 before being licensed to a US company.
The NHIA estimates that 13 patients would receive the therapy in its first year at a cost of about NT$1.3 billion.
From the second through fifth years, five additional patients are expected to be treated annually, costing about NT$500 million per year.
The decision has sparked criticism from physicians, including pulmonologist Su Yi-fong (蘇一峰), who said that drugs for other rare diseases — such as lymphangioleiomyomatosis (LAM or "bubble lung") — treat dozens of patients for less than NT$100 million, yet are not fully covered.
Supporters countered that the AADC therapy offers life-saving benefits and meaningful developmental gains.
Physician Chiang Kuan-yu (姜冠宇) said the investment could help position Taiwan as a key site for Asian genetic data, supporting future research collaborations and negotiations with international pharmaceutical companies.
NTUH Department of Medical Genetics director Chien Yin-hsiu (簡穎秀) defended the technology transfer to the US, saying few believed in the feasibility of AADC gene therapy two decades ago.
There had been no effective treatment for AADC deficiency in the past, and affected children could only lie immobile in bed with no motor development, Chien said.
Among the 31 patients who received the new therapy in a trial, 80 percent were able to sit up, and among those treated early with intensive rehabilitation, 40 percent were able to walk independently, she said.
"Just being able to sit up instead of only lying down makes an enormous difference," she said. "For parents providing daily care, the improvement is like night and day."
Regarding LAM coverage, NHIA Director-General Chen Lian-yu (陳亮妤) said the disease is not currently classified as rare, although applications have been submitted.
Coverage would follow once the ministry completes its review, she said.
LAM drugs have been covered under NHI since 2001, with 26 patients reported last year.
NHIA said it would assist families facing difficulties in the application process.
Actor Darren Wang (王大陸) was sentenced to six months in prison, commutable to a fine, by the New Taipei District Court today for contravening the Personal Data Protection Act (個人資料保護法) in a case linked to an alleged draft-dodging scheme. Wang allegedly paid NT$3.6 million (US$114,380) to an illegal group to help him evade mandatory military service through falsified medical documents, prosecutors said. He transferred the funds to Chen Chih-ming (陳志明), the alleged mastermind of a draft-evasion ring, although he lost contact with him as he was already in detention on fraud charges, they said. Chen is accused of helping a
SECURITY: Starlink owner Elon Musk has taken pro-Beijing positions, and allowing pro-China companies to control Taiwan’s critical infrastructure is risky, a legislator said Starlink was reluctant to offer services in Taiwan because of the nation’s extremely high penetration rates in 4G and 5G services, the Ministry of Digital Affairs said yesterday. The ministry made the comments at a meeting of the legislature’s Transportation Committee, which reviewed amendments to Article 36 of the Telecommunications Management Act (電信管理法). Article 36 bans foreigners from holding more than 49 percent of shares in public telecommunications networks, while shares foreigners directly and indirectly hold are also capped at 60 percent of the total, unless specified otherwise by law. The amendments, sponsored by Chinese Nationalist Party (KMT) Legislator Ko
UNREASONABLE SURVEILLANCE: A camera targeted on an road by a neighbor captured a man’s habitual unsignaled turn into home, netting him dozens of tickets The Taichung High Administrative Court has canceled all 45 tickets given to a man for failing to use a turn signal while driving, as it considered long-term surveillance of his privacy more problematic than the traffic violations. The man, surnamed Tseng (曾), lives in Changhua County and was reported 45 times within a month for failing to signal while driving when he turned into the alley where his residence is. The reports were filed by his neighbor, who set up security cameras that constantly monitored not only the alley but also the door and yard of Tseng’s house. The surveillance occurred from July
‘SAME OLD TRICK’: Even if Beijing resumes individual travel to Taiwan, it would only benefit Chinese tourism companies, the Economic Democracy Union convener said China’s 10 new “incentives” are “sugar-coated poison,” an official said yesterday, adding that Taiwanese businesses see them clearly for what they are, but that Beijing would inevitably find some local collaborators to try to drums up support. The official, speaking on condition of anonymity, made the remark ahead of a news conference the General Chamber of Commerce is to hold today. The event, titled “Industry Perspectives on China’s Recent Pro-Taiwan Policies,” is expected to include representatives from industry associations — such as those in travel, hotels, food and agriculture — to request the government cooperate with China’s new measures, people familiar with
RSS