Source: Hong Kong Government special administrative region
Following is a question by the Hon Elizabeth Quat and a written reply by the Acting Secretary for Health, Dr Cecilia Fan, in the Legislative Council today (October 15):
Question:
It is learnt that achondroplasia is a complex and rare hereditary skeletal disorder that affects patients’ growth and development, as well as multiple systemic functions. Some experts have pointed out that this disease has a prevalence of approximately 1 in 20 000 in Hong Kong, with patients having to face a long and painful treatment process alongside substantial medical expenses. In this connection, will the Government inform this Council:
(1) whether it has compiled statistics on the total number of achondroplasia patients currently in public hospitals, with a breakdown by age group (aged below 15, 15 to below 18, and 18 or above);
(2) whether it knows if the Hospital Authority (HA) has established an expert group or allocated dedicated resources for achondroplasia to support patients’ long-term treatment process; if HA has, of the details; if not, the reasons for that;
(3) whether the Government has estimated the lifetime medical costs for achondroplasia patients, including the provision of conventional bone-lengthening surgery treatment and related medical follow-up, such as hospitalisation, surgery, post-operative care and rehabilitation training;
(4) as some patient groups have pointed out that achondroplasia patients (particularly adult patients) are currently scattered across different public hospital clusters and receive follow-up services of varying standards, whether the Government knows if HA has developed unified and comprehensive treatment guidelines for this disease;
(5) as it is learnt that a new C-type natriuretic peptide (CNP) drug has been introduced in recent years which can promote cartilage growth in achondroplasia patients through subcutaneous injection and help mitigate the impact of severe complications on patients, and that several places have included this drug in their national insurance scheme or as a subsidised item, whether the Government will draw reference from the relevant experiences and study the inclusion of this drug in the Drug Formulary and the scope of the safety net subsidies;
(6) whether the Government will consider enhancing the transparency of the criteria for evaluating the cost-effectiveness of new drugs to expedite the process of including new drugs (especially those for treating rare diseases) in the safety net; and
(7) whether the Government will draw reference from the supplementary medical insurance for major diseases launched by the country (such as People-Benefit Insurance) in recent years, and study improving the Voluntary Health Insurance Scheme or establishing a separate insurance product for major diseases with the characteristics of “no age limit, no medical history restrictions and universal participation”, so as to encourage members of the public to take out insurance policies, thereby alleviating the financial pressure on medical institutions when introducing new drugs to treat rare diseases?
Reply:
President,
In consultation with the Hospital Authority (HA), the consolidated reply to the question raised by Hon Elizabeth Quat is as follows:
The Government and the HA attach high importance to providing optimal care for all patients (including those with rare diseases).
In tackling rare diseases, the HA has been adhering to the principle of “providing holistic care for patients” in providing holistic medical and rehabilitation services to patients with rare diseases through a multi-disciplinary and inter-departmental medical and rehabilitation team in an integrated service model. The relevant support includes not only drug treatments, but also multi-faceted services such as newborn screening, clinical diagnosis and assessment, surgery, interventional therapies, palliative care and rehabilitation services.
The HA has also been keeping in view the international clinical evidence and scientific research development on treatment options for rare diseases, and conducting exchanges with overseas and Mainland experts from time to time on issues of common concern so as to learn from each other’s experience and facilitate service development.
(1), (2) and (4) Achondroplasia as mentioned in the question is a rare hereditary skeletal disorder. Some of the common orthopaedic issues these patients encounter during childhood include lower limb malalignment and short stature. In addition to orthopaedic issues, patients may also develop other complex conditions such as sleep apnea, foramen magnum stenosis, hearing loss and malocclusion. As patients enter adulthood, they may also develop other orthopaedic conditions such as spinal stenosis, spinal deformity and osteoarthritis. According to the records of the HA, the total number of surviving cases of achondroplasia as of June 30, 2025 is 160 (Note 1). By age group (Note 2), 30 of these patients are aged 0 to 14, 10 are aged 15 to 18, and 130 are aged 19 or above.
The current treatment and support protocols of the HA for achondroplasia are based on the International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia published by international academic bodies in 2021. This document offers recommendations for healthcare professionals in addressing the multiple medical issues faced by patients.
Adhering to the principle of “providing holistic care for patients” and with reference to the relevant recommendations in the aforementioned document, the HA currently adopts a multi-disciplinary collaborative model in providing one-stop services for patients at Specialist Out-patient Clinics. A team comprising orthopaedic surgeons, paediatricians and relevant allied health professionals caters for patients’ medical, surgical and psychosocial needs across various stages of life. For paediatric patients with lower limb malalignment, the HA may arrange early surgical intervention. Cases which have been rigorously assessed as clinically suitable may also be arranged to undergo lower limb lengthening surgery, which is a highly complex procedure, to mitigate the impact of short stature on their quality of life. Furthermore, if paediatric patients develop life-threatening complications such as sleep apnea, hydrocephalus or foramen magnum stenosis, they will be arranged to receive related assessments and surgical treatments at tertiary specialist referral centres with relevant expertise (such as the Hong Kong Children’s Hospital). Their clinical needs will also be regularly monitored in accordance with international surveillance guidelines. As patients transition into adulthood, designated nurses within the team will co-ordinate their examinations, treatments and rehabilitation, while also providing support in terms of enhancing their quality of life and self-management skills, thereby facilitating their smooth integration into the community.
Note 1: The figures have been rounded to the nearest ten. Individual figures may not add up to the total due to rounding.
Note 2: Based on the patient’s age as of June 30, 2025.
(3) Generally speaking, patients with achondroplasia who undergo conventional bone-lengthening surgery will also receive follow-up treatment and medical procedures for this condition at healthcare facilities under the HA. That being said, patients may also be required to attend HA facilities for treatment of other conditions, making it difficult to ascertain whether each attendance is attributable to the treatment of achondroplasia. Moreover, given each patient’s life expectancy, future clinical attendance needs and categories of medical services required vary and are difficult to predict, the HA does not have an estimate for the lifetime medical costs for such patients.
(5) and (6) Drug treatment is an important part of the medical services provided to patients with rare diseases. Currently, the HA provides drug subsidies to patients with rare diseases through recurrent funding from the Government and the safety net, such as the Community Care Fund Medical Assistance Programmes.
The HA has put in place an established mechanism for regular evaluation of registered new drugs and review of the existing drugs listed in the HA Drug Formulary (HADF) and the coverage of the safety net. The review process is evidence-based, taking into account the safety, efficacy, cost-effectiveness and other relevant considerations, including international recommendations and practices, as well as professional views, so as to ensure that public resources are utilised fairly and efficiently to enable patients to receive appropriate treatment. In view of the continuous rapid advancement in medical technology and the ever-increasing public expectations, the HA has, since the implementation of the HADF, continued to refine its review mechanism through incorporating additional stakeholders into the review process, thereby increasing the transparency of the decision-making process regarding the HADF’s coverage.
To speed up the introduction of suitable new drugs to the HADF, the HA has simplified the application process for inclusion of new drugs in the HADF since the end of 2024. Clinicians and pharmaceutical companies can submit new drug applications directly to the Drug Advisory Committee. The frequency of prioritisation exercise for including new drugs in the safety net will also increase from twice a year to four times a year. With the implementation of the above new mechanisms, the HA has been actively optimising the procedures for introducing new drugs into the HADF, with the objective of reducing the time required for introducing new drugs with proven efficacy into the HADF or the coverage of the safety net by half, from the original 10 months to five months; and from 18 months to nine months respectively, so as to enable patients to have access to new drugs as soon as possible, and to obtain the subsidies under the safety net to alleviate the burden of drug expenses.
Meanwhile, the Government and the HA will also press ahead with the implementation of the fees and charges reform for public healthcare announced at the end of March this year, which aims to strengthen healthcare protection for “poor, acute, serious, critical” patients. Relevant measures include enhancing the protection in terms of drugs and medical devices for critically ill patients through accelerating the introduction of more effective innovative drugs and medical devices to the HADF and the coverage of the safety net, with a view to ensuring that the limited healthcare resources can be directed in a more targeted manner to assist those patients most in need. This will thereby improve the sustainability of the healthcare system and better serve as a safety net for all.
Nevertheless, drugs for the treatment of rare diseases can be very expensive and their efficacy may only be marginal when compared with other available treatment alternatives. Therefore, in considering the introduction of individual drugs for rare diseases, apart from reviewing the efficacy and safety of the drugs through clinical evidence and making reference to the guidelines on the use of the drugs for the relevant rare diseases in different regions, as well as the arrangements of the disease management and the drug reimbursement schemes thereat, the HA has to take into account the cost-effectiveness and opportunity cost considerations of the drugs, with a view to ensuring rational and effective use of the limited public healthcare resources.
It is understood that the C-type natriuretic peptide (CNP) drug introduced in recent years is Vosoritide (Voxzogo), which is a registered drug in Hong Kong. It is noted that this drug has not yet been registered in the Chinese Mainland and therefore should not have been formally included in any national insurance scheme or subsidy programmes of the Chinese Mainland. Some overseas regions, such as the United Kingdom and Singapore, have also not yet included this drug in their national insurance or subsidy programmes.
The HA will continue to pay close attention to the latest scientific research and clinical evidence of drugs suitable for the treatment of achondroplasia, with a view to ensuring that patients are provided with cost-effective drugs of proven safety and efficacy as well as continuous optimal care.
(7) It is understood that different commercial supplementary medical insurance plans (such as Huiminbao) have been rolled out in various provinces and municipalities of the Mainland. They belong to voluntary additional medical insurance on top of the Mainland’s prevailing basic medical insurance system premised on universal contribution.
A medical insurance system similar to the Mainland’s one premised on universal contribution has not been implemented in Hong Kong. After extensive public consultations, as well as discussions with stakeholders such as the healthcare sector, insurance industry and patient groups, in Hong Kong, the Government has been fully implementing the Voluntary Health Insurance Scheme (VHIS) since April 2019 to establish standards and improve market transparency for individual indemnity hospital insurance products, providing Hong Kong citizens with greater confidence in using private healthcare services through purchasing medical insurance. Insurance companies can design different VHIS products to cater for citizens’ needs, and such products must fulfill requirements on consumer protection in respect of age, health condition/medical history, etc. The requirements include guaranteed renewal up to the age of 100 regardless of any change in the health conditions of the insured persons, and covering unknown pre-existing conditions.
At the same time, the Government will continue to offer a medical safety net for all Hong Kong citizens through public healthcare services, including enhancing healthcare protection for “poor, acute, serious, critical” patients on all fronts. In fact, the majority of individuals with costly and complex medical needs would opt for receiving treatments in the public healthcare system. The Government will continue to prudently study how to make the best use of public funds to meet the medical needs of the individuals concerned in the most cost-effective manner.