Traditional Chinese Simplified Chinese Email this article
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):


     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?



     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


Print this page