The families of several people who have rare illnesses yesterday urged the legislature to turn the success of the amyotrophic lateral sclerosis (ALS) Ice Bucket Challenge into a sustainable system that can guarantee lifetime care and support for people who have rare conditions.
They made the appeal at a press conference in Taipei yesterday held by Chinese Nationalist Party (KMT) Legislator Yang Yu-hsin (楊玉欣), called for the speedy passage of an amendment to the Rare Disease and Orphan Drug Act (罕見疾病防治及藥物法) at the next legislative session.
Yang has Miyoshi myopathy, a form of the rare genetic disorder distal muscular dystrophy, in which muscle fibers do not function properly, which can result in chronic weakness.
Photo: CNA
The amendment seeks to shorten the time and simplify the application for the inclusion of so-called “orphan drugs” into the National Health Insurance (NHI) program; provide psychological counseling services to patients affected by rare illnesses and their families; offer subsidies for medications, food and care necessary to help sustain the lives and assuage the discomfort of such patients.
Orphan drugs are treatments developed for rare conditions.
Among the attendees was the father of a five-year-old boy surnamed Su (蘇) who has atypical hemolytic uremic syndrome (HUS), a rare and life-threatening genetic disease that could damage vital organs such as the kidneys, heart and brain.
“My son was recently hospitalized after the disease made him unable to urinate for the fifth time in his life. He usually looks thin and healthy, but now his whole body is swollen and he is in desperate need for the drug [eculizumab],” Su said.
Eculizumab, sold as Soliris, is a less-invasive treatment administered as an intravenous infusion for patients with atypical HUS, compared with other common treatment options, such as dialysis and plasma exchange therapy.
However, the NHI Administration has yet to approve the drug as a viable treatment for the disease, meaning patients who require the medication, which costs nearly NT$210,000 (US$7,000) per vial, have to pay the full cost themselves.
Taiwan Foundation for Rare Disorders Founder Serena Chen (陳莉茵) said in a telephone interview that while the amendment is well-meant, the problem cannot be addressed unless the concerned government agencies work hard to improve their efficiency and effectiveness.
“Almost a year has passed since a six-year-old atypical HUS patient surnamed Yeh (葉) passed away in November last year after his parents could no longer afford Soliris. A little girl diagnosed with the disease also died because of the same reason in May this year, just 28 days after her birth. Yet the drug is still not covered by the NHI,” Chen said.
“We just hope that things will go differently for Su,” she added.
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