Replies to LegCo questions
LCQ20: Drugs for Thalassaemia treatment
Following is a question by the Hon Albert Chan Wai-yip and a written reply by
the Secretary for Food and Health, Dr York Chow, in the Legislative Council
today (November 3):
Question:
Recently, quite a number of patients suffering from Thalassaemia major have
relayed to me that for years, they are only able to receive Desferrioxamine
injection drug therapy because of financial problems; yet, most of them do not
receive injections of this drug on time because of work. Quite a number of
patients have also stated that they suffer from serious side effects after
receiving injections of this drug, while the same therapeutic effect can
actually be achieved just by receiving Deferasirox oral drug therapy, which has
a minimal effect on their quality of life. Nevertheless, since Deferasirox has
not yet been listed as General Drugs of the Hospital Authority (HA), they have
to pay a lot of money to buy Deferasirox. In this connection, will the
Government inform this Council whether it knows:
(a) the number of cases received by HA in each of the past three years regarding
patients with Thalassaemia major requesting for the listing of Deferasirox as
General Drugs;
(b) the reasons why HA has still not listed Deferasirox as General Drugs; and
(c) whether HA will consider listing Deferasirox as General Drugs in the near
future to enable more patients with Thalassaemia major to receive Deferasirox
therapy, thus reducing their pain in the treatment process; if it will, of the
details; if not, the reasons for that?
Reply:
President,
The standard drugs in the Hospital Authority (HA) Drug Formulary (the Formulary)
can be classified into two categories, namely general drugs and special drugs.
General drugs have well-established clinical indications and effectiveness and
are available for general use, while special drugs are to be used under
specified clinical conditions with specific specialist authorisation. Under the
existing mechanism, all general drugs and special drugs prescribed under
specified clinical conditions are provided to patients at standard fees and
charges by HA.
At present, there are three drugs in the Formulary which are used in iron
chelation treatment for Thalassaemia patients, including the injection drug
Desferrioxamine, the oral drug Deferiprone and the other oral drug Deferasirox.
Desferrioxamine is classified as a general drug in the Formulary, while
Deferiprone and Deferasirox are classified as special drugs. All three drugs are
covered by standard fees and charges which are highly subsidised by the
Government. Patients who meet the specific clinical conditions and require the
drug for treatment will be prescribed with the drug by doctors and provided with
the drug at standard fees and charges by HA.
The reply to various parts of the question is as follows:
(a) In the past three years, HA has not received any request for assistance from
individual patients with Thalassaemia major demanding for Deferasirox to be
classified as a general drug. However, the Food and Health Bureau and HA
received in October 2009 a letter from the Thalassaemia Association of Hong Kong
with a request for Deferasirox to be classified as a first-line drug for the
treatment of Thalassaemia. In December 2009 and January 2010, the Association
also referred to us via the Legislative Council Secretariat 20 cases of request
for assistance from Thalassaemia patients and demanded for Deferasirox to be
used for treatment of these patients. HA's specialists in haematology had
assessed each of their cases and we gave a reply in February 2010.
(b) At present, there is different scientific evidence on the efficacy and side
effects of the three iron chelating drugs. The injection drug Desferrioxamine is
recognised by the medical profession as an efficacious, safe and reliable iron
chelating drug which is proven to have long-term efficacy on patients' survival.
For the Thalassaemia patients in general, serious side effects caused by the use
of Desferrioxamine on patients (such as thrombocytopenia) are not common. From
the perspective of the medical profession, Desferrioxamine is the "gold
standard" drug for iron chelation treatment, and is therefore classified as a
general drug in the Formulary.
The oral drug Deferiprone is a second-line drug after the injection drug
Desferrioxamine. It has similar efficacy as Desferrioxamine, and is effective in
removing the iron accumulated in the heart and protecting the cardiac function
of patients and hence enhancing their survival rate. However, it may also cause
neutropenia. Hence, Deferiprone is classified as a special drug in the Formulary
and prescription by specialist doctors according to patients' clinical
conditions is required for its use.
On the other hand, Deferasirox is not superior to Desferrioxamine and
Deferiprone in terms of its efficacy, side effects and cost-effectiveness in the
treatment of Thalassaemia in general. Also, its long-term efficacy and safety
require the accumulation of more data to prove. As compared with Deferiprone
which has the function of protecting the heart, there is not yet sufficient
scientific evidence to show that Deferasirox can effectively remove the iron
accumulated in the heart and improve the cardiac function of patients.
Therefore, doctors do not recommend the use of Deferasirox on patients with
heavy cardiac iron load. Research findings also show that Deferasirox may cause
side effects such as neutropenia and acute renal failure. For these reasons, it
is appropriate for HA to classify Deferasirox as a special drug which requires
prescription by specialist doctors for its use according to the clinical
conditions of individual patients. If the clinical conditions of individual
patient are not suitable for use of Desferrioxamine and Deferiprone, doctors
will arrange for the patient to use Deferasirox on a trial basis in order to
provide additional treatment alternative.
Besides, if patients cannot adapt to injection or are not suitable for treatment
with Desferrioxamine because of other clinical reasons, or if excessive
accumulation of cardiac iron is found in patients and enhanced efficacy for
cardiac iron removal is needed, doctors will arrange for the use of the
injection drug Desferrioxamine in combination with the oral Deferiprone.
However, there is not yet sufficient scientific evidence in the medical
profession to show that Deferasirox can be used in combination with the
injection drug Desferrioxamine. To conclude, Deferasirox is considered
unsuitable for classification as a general drug in the Formulary at present.
(c) In prescribing iron chelating drugs to Thalassaemia patients, doctors take
into consideration the overall safety, efficacy and cost-effectiveness of the
drugs. They also carefully assess the clinical conditions and treatment needs of
each patient as well as the actual efficacy and possible effects of the drugs on
individual patients in order to make suitable treatment decisions. Doctors also
take into account the needs of patients for their work and social life, other
needs in their daily life as well as the impact of the treatment on their
quality of life.
For the treatment of Thalassaemia, HA has put in place an established mechanism
under which an expert panel comprising specialist doctors will from time to time
review the latest clinical and scientific data on iron chelation treatment.
After study, the expert panel considers that Deferasirox is not suitable for
general use and is not appropriate to be classified as a general drug at
present. HA will continue to review the guidelines of drug treatment from time
to time in the light of the development of the drugs and scientific evidence.
Ends/Wednesday, November 3, 2010
Issued at HKT 16:30
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