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LCQ9: Medical needs of patients with rare genetic disease
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     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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