Carrie Lam

‘Best birthday gift’ for rare disease patient as Hong Kong leader Carrie Lam says new drug will reach city in two months

Josy Chow, 23, used two fingers to type 22-page proposal calling on authorities to import US-approved medication for spinal muscular atrophy

PUBLISHED : Thursday, 01 March, 2018, 3:06pm
UPDATED : Thursday, 01 March, 2018, 10:37pm

Chief Executive Carrie Lam Cheng Yuet-ngor brought good news to paralysed patient Josy Chow Pui-shan on Thursday, saying a new drug to treat her rare condition would arrive in the city in two months.

Chow, a 23-year-old University of Hong Kong student, earlier penned a proposal calling on the government to import the drug, which is not registered in the city but has been approved by the United States Food and Drug Administration.

“It is so surprising to see [Carrie Lam] and I am grateful for the good news,” Chow said. “I am hoping to host a party to celebrate.”

Paralysed woman who can only type with thumb and index finger says Hong Kong charity Operation Santa Claus has given her hope

Chow said she felt the administration cared about patients with rare diseases, and she hoped there would be new policies to help them.

During a surprise 10-minute visit to Chow at HKU on Thursday morning, Lam said the drug would be given to other patients who suffered from the same condition for free via an “expanded access programme”.

Chow has spinal muscular atrophy (SMA), a genetic disorder that gradually destroys physical abilities such as walking and breathing.

It has left her almost completely paralysed, and she typed her 22-page letter to Lam with only two fingers, taking almost four months.

The effort prompted the city’s leader to pledge last October to personally ensure that the drug would be made available quickly.

“Your bravery and perseverance have moved many, and it brought hope to SMA patients,” Lam wrote in a card for Chow on Thursday. “This is your contribution.”

With tears in her eyes, Chow described the news as the “best birthday gift”. She will turn 24 next Saturday.

The new US-manufactured medication, nusinersen (marketed as Spinraza), was the first drug for SMA approved by the US authority. It is not yet registered in the city as Lam said earlier that the pharmaceutical firm had no intention of introducing the drug in Hong Kong.

But Lam said the new arrangement would allow free access for local patients, adding that it should be available in about two months.

Secretary for Food and Health Professor Sophia Chan Siu-chee, who visited Chow with Lam, said all SMA type one patients could use the drug under the current arrangement, but further clinical work was needed to determine if type two patients could also benefit from it since their conditions were slightly different.

There are an estimated 85 SMA patients in the city, with 20 of them suffering from the most severe form of the disease and requiring frequent intensive care in hospital.

For regular use, the cost of the drug per patient is about HK$6 million (US$766,500) in the first year of treatment and HK$3 million annually in subsequent years.

Lam said the Hospital Authority and Department of Health would help the pharmaceutical company register the drug as well as include it on the list of expensive medications subsidised by the Community Care Fund.

Financial Secretary Paul Chan Mo-po said on Wednesday that the government would set aside HK$500 million for the fund’s programme, which subsidises rare disease patients in need of costly drugs.

“I never thought Mrs Lam would miss us so much, and it is so surprising that she came to visit us herself,” Chow’s mother said after the visit.