Call to ease Hong Kong patient’s ‘living hell’ with drug subsidy to treat rare disease
Mrs Ho, one of about 20-odd Hongkongers who suffer from a condition called familial amyloid polyneuropathy, would be helped by the medicine tafamidis – but the treatment costs up to HK$120,000 a month
Sufferers of a rare neurodegenerative disease are calling on Hong Kong’s health authorities to provide them with subsidised treatments and an escape route from a “living hell”.
The nightmare for Mrs Ho, one of roughly 20-odd Hongkongers who suffer from a condition called familial amyloid polyneuropathy (FAP), began about seven or eight years ago.
Swallowing was getting increasingly difficult and she was losing her appetite. Her hands and feet would swell and go numb and she would suffer bouts of coughing so severe she would start “expelling foam”. She shed 10 to 20 pounds (4.5kg to 9kg) in a year.
It wasn’t until two years ago that she learned she was suffering from FAP, a rare, hereditary disease, which sees the autonomic nervous system compromised by excess build-up of a protein called amyloid in the organs and tissues. Three of her brothers also have the disease.
Recently, the condition had been affecting her heart, causing it to pump less blood and leading to pleural effusion, the accumulation of fluid outside the lungs
“It is indescribable. It is a living hell,” wrote her daughter Iris Ho in a petition letter to Chief Executive Carrie Lam Cheng Yuet-ngor, which they handed to Undersecretary for Food and Health Chui Tak-yi at a symposium on Sunday to mark Rare Disease Day on Wednesday.
The mother can only take medication to relieve the symptoms. At 69, her age makes it too risky for a liver transplant.
“Medical treatment is supposed to give patients hope, but since most cannot afford it, we all continue to live in a state of helplessness,” Iris Ho wrote.
The drug FAP sufferers require is tafamidis, which was approved by European Union authorities in 2011. The drug is not yet listed under the Hospital Authority’s Drug Formulary – meaning it is not subsidised by the government.
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The drug changes amyloid deposition, which reduces the impact on the patient’s organs, such as the heart, and curbs the progression of the disease.
Treatment can cost up to HK$120,000 (US$15,400) a month, according to the Alliance of Rare Diseases, which organised the symposium at the University of Hong Kong.
Without treatment, the condition is usually fatal in 10 years on average.
Receiving the petition letter, Chui said the government would follow established mechanisms in deciding whether to introduce drugs, taking into account evidence from clinical trials, information from overseas and from pharmaceutical companies as well as studies from its own expert panel.
As to proposals for normalising funding for rare disease patients via the Community Care Fund, Chui said results of a study would be completed by the middle of this year.
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“Once the study is out we will announce a decision accordingly,” he said.
The alliance has also been lobbying the government on behalf of other rare disease sufferers, with mixed success.
In October last year, Lam met with alliance members, who were holding a rally outside her office, two days before her policy address, and pledged to “personally” ensure life-saving drugs for Hong Kong patients with spinal muscular atrophy.