Replies to LegCo questions
LCQ9: Medical needs of patients with rare genetic disease
Following is a question by the Hon Wong Yuk-man and a written reply by the
Secretary for Food and Health, Dr York Chow, in the Legislative Council today
(May 6):
Question:
According to the information provided by the Hong Kong Mucopolysaccharidoses and
Rare Genetic Diseases Mutual Aid Group, there are more than 30
Mucopolysaccharidoses patients in Hong Kong at present. They consider that as
the number of Mucopolysaccharidoses patients is small, their needs have all
along been neglected by the Government. Most patients can live up to 20 odd
years only, and several of them are already in critical condition at present.
Although the effective treatments for Mucopolysaccharidoses recently found in
foreign countries have brought patients a hope for survival, the medical fee of
over HK$4 million a year has rendered the local patients helpless. In this
connection, will the Government inform this Council:
(a) of the existing specific policies for assisting Mucopolysaccharidoses
patients, and whether emergency measures are in place to support individual
patients who are in critical condition; and
(b) given that at present, many countries and places (including Macao) provide
Mucopolysaccharidoses patients with subsidies to pay for their treatments, why
Hong Kong is not doing the same?
Reply:
President,
(a) There are over 50 types of rare genetic lysosomal diseases, which include
Mucopolysaccharidoses, Fabry disease, Pompe disease and Gaucher disease. At
present, there is no effective medical treatment for curing rare genetic
diseases like Mucopolysaccharidoses. The Administration has been looking after
the medical needs of patients with rare genetic disease. With a view to
improving the quality of life and prolonging the lifespan of these patients, the
Hospital Authority (HA) currently seek to alleviate patients' discomfort and
treat the complications arising from the disease through the collaboration of
healthcare staff from various specialties, such as paediatrics, orthopaedics,
otorhinolaryngology, ophthalmology and respiratory medicine, along with genetic
counseling, as well as the provision of appropriate drugs, surgery and
rehabilitation programme. These services are covered by the standard fees of HA.
Since 2008-09, the Administration has provided additional annual recurrent
funding of $10 million to HA to provide specific subsidised enzyme replacement
therapy (ERT) to patients with rare genetic lysosomal diseases. Most of these
drugs have only been on the market for a relatively short period and their costs
are extremely high. Also, the efficacy of these drugs has yet to be proved by
sufficient scientific research data. As such, in regard to these drugs which
have only preliminary medical evidence, the Administration has to carefully
examine their efficacy as well as other possible effects on individual patients
so as to assess whether it is suitable for the patient to be prescribed with the
drugs and whether it is cost-effective and fair use of public money to subsidise
their drug costs by public funding. HA has set up an Expert Panel on ERT for
Rare Genetic Lysosomal Diseases (Expert Panel) to formulate treatment guidelines
for specific drugs and the assessment criteria for the use of these drugs on
patients. The Expert Panel also makes assessment on the clinical conditions of
individual patients, including Mucopolysaccharidoses patients, to determine on a
case-by-case basis whether it is suitable for them to undergo the respective ERT
in accordance with the criteria.
(b) The Expert Panel under HA will continue to assess applications to receive
ERT from patients with rare genetic lysosomal diseases, including
Mucopolysaccharidoses patients, so as to consider whether to provide individual
patients with subsidised treatment. Meanwhile, HA will provide timely support to
patients according to their conditions and medical needs.
We need to point out that different countries adopt different practices on
scientific evaluation, introduction of drug formularies, registration
requirements, assessment of patients' applications for subsidies and the mode of
subsidy of drugs for rare genetic diseases. As regards the provision of
subsidised ERT to Mucopolysaccharidoses patients, there is no standardised
approach among different countries. Due to differences in healthcare systems
among different countries, it is not appropriate to make comparison among them.
Ends/Wednesday, May 6, 2009
Issued at HKT 12:46
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