Replies to LegCo questions
LCQ21: Treatment of rare diseases
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
disease、Imiglucerase 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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