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Following is a question by the Hon Dennis Kwok and a written reply by the Secretary for Food and Health, Dr Ko Wing-man, in the Legislative Council today (July 10):
Question:
Some members of the public have relayed that since the number of patients suffering from rare diseases (such as Mucopolysaccharidosis, myelofibrosis, cryopyrin-associated periodic syndromes and Pompe disease, etc.) is small, the Government has not attached importance to their treatment and nursing needs. These members of the public have pointed out that regarding applications for subsidies to cover the expenses on treatment and medications required by patients with rare diseases to slow down the progression of their diseases or even to sustain their lives, the Hospital Authority (HA) has been slow in vetting and approving such applications and the amounts of subsidies granted are insufficient. Moreover, the applications have to be vetted and approved on a case-by-case basis. In this connection, will the Government inform this Council:
(a) given that the European Union at present defines a rare disease as a disease which affects fewer than a two-thousandth of the population, whether the Government will consider making reference to such a practice and adopting a definition for the rare diseases in Hong Kong; if it will, of the implementation timetable and details; if not, the reasons for that;
(b) whether it knows the existing number of drugs for treating rare diseases that have been listed as drugs provided at standard fees and charges in the HA Drug Formulary (the Formulary); of the number of such drugs which had been considered to be listed as drugs provided at standard fees and charges in the past, and among them, the number of those which eventually had not been listed as such and the reasons for that;
(c) whether it will consider making reference to Taiwan's Rare Disease Control and Orphan Drug Act and putting in place a mechanism independent of the Formulary for vetting and approving patients' applications for subsidies to cover the expenses on drugs for treating rare diseases; if it will, of the details; if not, the reasons for that; and
(d) whether it will consider, by making reference to the practices of overseas places (e.g. the United States and Taiwan), (i) establishing an office of rare diseases research to coordinate researches on rare diseases, (ii) subsidising the diagnosis and treatment of and medications for patients with rare diseases, and (iii) formulating specific policies and initiatives to comprehensively cater for the treatment and nursing needs of patients with rare diseases?
Reply:
President,
For all patients attending public hospitals and clinics, doctors of the Hospital Authority (HA) will assess their conditions in accordance with established procedures. After diagnoses have been made, doctors will provide the appropriate healthcare treatment for patients based on their clinical conditions and the treatment guidelines.
To ensure equitable access to cost-effective drugs of proven efficacy and safety, HA has devised the Drug Formulary with a view to standardising the drug policy and drug utilisation, introducing new drugs and expanding the coverage of the Formulary systematically. Experts of HA evaluate new drugs and review the current list of drugs in the Formulary on a regular basis. In conducting the evaluation, the experts will have regard to such core values as evidence-based medical practice, rational use of public resources, targeted subsidy and opportunity cost. They will also take into account various factors, including the efficacy and safety of the drugs, international recommendations and practices, changes in technology, disease state, patient compliance, impact of the drugs on patients' quality of life, actual experience in the use of drugs, comparison with available alternatives, impact on healthcare costs and views of professionals and patients groups.
My reply to the various parts of the question is as follows:
(a) There is currently no universal definition of rare diseases in the international arena. The definition of rare diseases in different countries varies depending on their healthcare systems and situations. Given the difficulty to confirm the number of rare disease cases, the lack of reliable information or data on the causes of such diseases, and the relatively recent discovery of ways to treat some of the diseases, HA has not defined rare diseases.
(b) At present, there are about 1 300 drugs in HA Drug Formulary for treatment of various kinds of diseases. HA has received additional annual recurrent funding of $10 million since 2008-09 and extra funding of $35 million since 2010-11 (i.e. a total of $45 million each year) from the Government to provide enzyme replacement therapy (ERT) for patients suffering from six types of lysosomal storage diseases (LSD) (including Pompe disease, Fabry disease, Gaucher disease, and Mucopolysaccharidosis Type I, Type II and Type VI).
The six ERT drugs used to treat the aforementioned LSDs, namely Alglucosidasc alpha for Pompe disease, Algalsidase beta for Fabry diseaseˇBImiglucerase for Gaucher disease, Laronidase for Mucopolysaccharidosis Type I, Idursulfase for Mucopolysaccharidosis Type II and Glasulfase for Mucopolysaccharidosis Type VI, are all categorised as special drugs in the HA Drug Formulary. Patients who meet the specified clinical criteria will be provided treatment at standard fees and charges by HA as a highly subsidised service.
Since 2008-09, HA has provided ERT for 20 patients suffering from LSDs, and there are currently 16 patients receiving such therapy.
(c) and (d) Patients suffering from the six types of LSDs mentioned above have abnormal substances accumulating in their body tissues due to genetic changes, eventually causing permanent damage to various organs. Since the impact of LSDs on patients will become more profound with time, it is necessary to use drugs early in the course of illness when no irreversible harm has been caused to the body so as to maximise the efficacy of treatment. Drug treatment does not bring obvious benefit or is even totally ineffective to patients at a later stage of illness.
As the efficacy and safety of ERT vary according to the specific clinical conditions of patients, HA has set up an independent expert panel to assess how suitable ERT is for individual patients. Members of the expert panel include doctors specialised in medicine, paediatrics and clinical pharmacology and therapeutics, and pharmacists. The expert panel will consider and strike a balance between the efficacy and risks of ERT for the patients, and make reference to specific treatment guidelines so as to ensure that patients will receive safe and effective treatment. As for patients currently receiving ERT, the expert panel will monitor regularly their individual responses to the treatment and the overall clinical conditions so as to decide whether it is appropriate for them to continue receiving ERT.
In addition to ERT, HA also uses other conventional treatment options, including rehabilitation programme, pain-relief treatment, surgery and bone marrow transplant, for curing LSDs.
HA will continue monitoring the situation, and where necessary, make appropriate adjustment to the treatment options, so as to provide treatment and care for LSD patients in a holistic manner.
Ends/Wednesday, July 10, 2013
Issued at HKT 16:29
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