LCQ10: Treatment for uncommon disorders and cancers

Hong Kong (HKSAR) -      Following is a question by the Dr Hon Elizabeth Quat and a written reply by the Secretary for Food and Health, Professor Sophia Chan, in the Legislative Council today (November 8):


     It is learnt that there are more than 7000 known rare diseases worldwide, and most of them are hereditary diseases, autoimmune diseases and cancers.  While the European Union, the United States, Australia, Japan, South Korea and Taiwan have drawn up their official definitions for rare diseases and the relevant medical care policies, Hong Kong has not followed suit.  Some concern groups have pointed out that the existing support measures of the Government for patients of rare diseases and cancers also lag behind those of other advanced countries or regions.  In this connection, will the Government inform this Council:

(1) whether the authorities will expeditiously draw up an official definition for "rare diseases" and the relevant policies on supporting the patients; if so, of the details; if not, the reasons for that;

(2) given that the Policy Address just delivered has put forward the provision of more assistance for patients with uncommon diseases, whether the "uncommon diseases" mentioned include: (i) non-infectious non-anterior uveitis, (ii) acute lymphoblastic leukemia, (iii) familial amyloid polyneuropathy, (iv) cortical-basal ganglionic degeneration, (v) hereditary thrombocytopenia and (vi) fabry disease; if so, of the details; if not, the reasons for that;

(3) whether the authorities will establish a database on rare diseases for reference by the relevant personnel when making policy decisions on medical care, making clinical decisions and conducting medical researches; if so, of the details; if not, the reasons for that;

(4) whether the authorities will set up an one-stop centre for rare diseases at the Hong Kong Children's Hospital to be commissioned shortly, so that tasks such as screening, diagnosis, treatment, rehabilitation, research and training relating to rare diseases can be centrally handled there; if so, of the details and the timetable; if not, the reasons for that;

(5) as most rare diseases are hereditary diseases, whether the authorities will make improvements to the existing public prenatal check-up services, including extending the scope of such services to cover more non-invasive genetic tests relating to hereditary diseases, and include genetic tests relating to common hereditary diseases in public premarital or prenatal check-up services, with a view to reducing the incidence of newborn babies suffering from rare diseases; if so, of the details; if not, the reasons for that;

(6) of the top 10 deadliest cancers in Hong Kong at present and, in respect of each of them, (i) the number of new confirmed cases and (ii) the number and age distribution of deaths in each of the past 10 years (set out in a table);

(7) of the average length of time taken from a public hospital patient being suspected by a doctor of having cancer to that patient receiving first treatment, and the respective numbers of people queuing to receive the relevant treatments (with a tabulated breakdown by type of cancer);

(8) of the drugs newly developed for treating rare diseases and cancers which were registered in Hong Kong in the past 10 years, and the average time taken by the authorities to vet and approve the applications for registration of these drugs; whether the authorities will put in place a mechanism for the speedy registration of drugs for treating such diseases; if so, of the details; if not, the reasons for that;

(9) whether the authorities will make references to overseas practices and introduce a risk-sharing scheme on drug expenses for the treatment of rare diseases and cancers, under which the Hospital Authority (HA) is to enter into agreements with pharmaceutical companies on payments for drugs based on the efficacy of drug treatment achieving the target specified, or based on the duration of treatment specified in the agreement concerned, with a view to capping the drug expenses for HA and the patients concerned; if so, of the details; if not, the reasons for that; and

(10) whether it knows the existing number of patient groups which provide support services for patients with rare diseases or cancers and their carers; whether the authorities will enhance the cooperation with such patient groups so as to support these patients and their carers more effectively; if so, of the details; if not, the reasons for that?



     My reply to the question raised by the Dr Hon Elizabeth Quat relating to uncommon disorders and cancers is as follows:

(1) to (3) Currently, there is no internationally agreed definition of uncommon disorders.  The definition varies among countries/regions depending on their own healthcare system and situation.  The Government's policy is to strive to ensure appropriate treatment for all patients, including those with uncommon disorders.

     With the advancement of technology, new treatment options, including drugs, surgery and invasive treatment, are being developed.  In assessing the treatment options for various disorders, including uncommon disorders, the Hospital Authority (HA) would take into consideration the following factors:

i. Newly developed treatment options, having only preliminary medical evidence in safety and efficacy, may vary significantly in terms of treatment responses in different patients.

ii. Due to the small number of cases or patient groups for conducting scientific research at the initial stage, and the brief duration of research conducted, it is not easy to ensure the completeness of the clinical data.


The ultra-high cost of such options in the market is unaffordable for average patients.

     Under the current healthcare policy, we strive to ensure that all patients, whether they are patients with uncommon disorders or those suffering from other general illnesses, will not be denied appropriate treatment due to lack of means.  The healthcare support provided by the HA covers patients with uncommon disorders and those suffering from other diseases, and the mechanism in place also addresses the needs of all patients, including those with uncommon disorders.  The HA will continue to review and enhance its existing mechanisms and supporting arrangements to strengthen its services and support.  The HA maintains data on different types of disorders, which can facilitate research and policy formulation.

(4) The Hong Kong Children's Hospital (HKCH) is expected to commence operation by phases in late 2018.  HKCH will serve as a territory-wide tertiary referral centre for serious, complex and uncommon paediatric cases requiring multidisciplinary management.  While providing diagnosis, treatment and rehabilitation of higher quality to sick children from birth to 18 years of age who require such services, HKCH will also pool relevant expertise which will strengthen research and training regarding paediatric and genetic diseases.

     Meanwhile, the paediatric departments in the regional hospitals will mainly provide secondary, emergency and community paediatric care.  Under this hub-and-spoke model, HKCH and the regional hospitals will form a coordinated and coherent paediatric service network, enhancing the overall healthcare quality of the public paediatric service.

(5) In examining whether to introduce expanded carrier screening, the HA needs to carefully consider various factors, such as the types of genetic or hereditary diseases with testing available in Hong Kong, interfacing between genetic testing services and genetic counselling, as well as the accuracy and safety of the screening tests.  The HA has discussed the development and effectiveness of safeT21(T21) tests at the relevant Coordinating Committee.  The HA is now exploring the facilities required for the introduction of T21 testing in HKCH as a second tier screening test for Down's syndrome and preparing for manpower training in this respect.

(6) In 2015, the top 10 causes of cancer deaths were cancers of the lung, colorectum, liver, pancreas, stomach, breast, prostate, non-Hodgkin lymphoma, leukaemia and nasopharynx.  Of these cancers, the number of new cases and registered deaths with a breakdown by age group from 2006 to 2015 recorded by the Hong Kong Cancer Registry of the HA are set out at Annex.

(7) As the diagnosis and treatment process of individual cancer patients vary depending on the types of cancer and their clinical symptoms, the HA does not have a standardised approach to assess the waiting time for different types of cancer.

     The HA reviews on a regular basis the waiting time for patients with colorectal cancer, breast cancer and nasopharyngeal cancer to receive their first treatment after diagnosis.  In the period between January and December 2016, the waiting time at the 90th percentile (note) for patients with colorectal cancer, breast cancer and nasopharyngeal cancer to receive their first treatment after diagnosis were 76 days, 64 days and 54 days respectively.

(8) According to the Pharmacy and Poisons Ordinance and its subsidiary legislation, "pharmaceutical products" must satisfy the criteria of safety, efficacy and quality, and must be registered with the Pharmacy and Poisons Board (the Board) before they can be sold in Hong Kong.

     In order to facilitate timely registration of new pharmaceutical products for the treatment of patients in Hong Kong, the above legislation was amended in February 2015 to enable legislative amendments relating to new pharmaceutical products could be made via negative vetting procedures to expedite registration in Hong Kong.

     In the past 10 years (i.e. between 2007 and October 31, 2017), the Product Registration Committee established under the Board has considered and approved the registration of 171 new pharmaceutical products containing 100 new pharmaceutical substances for the treatment of cancer, or for the treatment of uncommon disorders as defined by the United State or European Union drug regulatory authorities.  These 171 new products included 99 new pharmaceutical products (containing 63 new pharmaceutical substances) were approved for registration between 2007 and 2014 before the above legislative amendments, with the average processing time of 5.4 months per application (i.e. between the date upon submission of complete registration documents by the applicant and the date when the Department of Health (DH) issued the letter to inform the applicant that the Board has approved its application); and 72 new pharmaceutical products (containing 37 new pharmaceutical substances) were approved for registration between 2015 and October 31, 2017 after the above legislative amendments, with the average processing time of 2.7 months per application.

     The DH conducts regular monthly seminars to explain the registration requirements of pharmaceutical products.  If necessary, applicants can contact the DH via hotline, and the DH will provide assistance to the applicants as appropriate.

(9) The HA holds discussions with pharmaceutical companies from time to time on setting up risk sharing or capping programmes for individual expensive drug treatments where both HA and pharmaceutical companies would contribute to the drug costs in specific proportions in a defined period or cap the drug treatment costs to be borne by patients, so as to provide the concerned patients with sustainable, affordable and optimal drug treatments in the long term.  Moreover, the HA would liaise with pharmaceutical companies on providing special or compassionate drug programmes, having regard to the exceptional circumstances of specific individual patients, so as to facilitate early access to drug treatments.

(10) Promoting partnership with patients is one of the key strategic directions of the HA in delivering patient-centred care.  The HA regularly organises sharing sessions with patient groups.  All patient groups listed on the Smart Patient Website (SPW) are invited.  During these sharing sessions, the HA discusses patient-related topics and imminent issues with patient representatives, and updates them on the latest information on healthcare services, such as HA Drug Formulary, waiting time for new specialist out-patient cases, and Patient Experience and Satisfaction Surveys, etc.  Representatives of patient groups may also provide feedback on service enhancement based on patients' needs.  As at July 2017, more than 200 patient groups were listed on the HA's SPW, including the Hong Kong Alliance for Rare Diseases and relevant organisations, as well as cancer-related patient groups.

     In parallel, the HA has set up Patient Resource Centres (PRCs) in cluster hospitals to provide support for patients and their families/carers, including facilitating the connection with patients groups and self-help groups for mutual support.  In particular, the HA leverages on the patient groups in promoting self-care and sharing of experience among patients.  At present, ward or home visits are conducted by many patient groups to show care and concern for patients.  Community networks are also established to support patients for early reintegration into community.  PRCs will also engage patient groups as volunteers to participate in various projects in hospitals, such as setting up of Patient Support Stations and Information Kiosks, as well as Scheme on Collaborative Projects of Patient Groups and PRCs, etc, to strengthen the psycho-social support to patients.  Such partnership can also enhance the mutual trust between patient groups and healthcare staff.

Note: The 90th percentile waiting time refers to the number of days between the date when a case is diagnosed with cancer after pathological examination and the date when the patient receives the first treatment.  The waiting time of 90 per cent of such cases is shorter than the value indicated.

Published on: 2017-11-08

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