Tsang Kin-ping, the executive committee member of the Hong Kong Alliance of Patients’ Organisations, says the contract system is inhumane. “It’s impossible to say the drug is here, but [the panel] still has to observe the patients,” says Tsang. “There’re hundreds of billions of dollars in the Hong Kong treasury. How can this be handled in this way?”
Although there are no official figures, it has been estimated less than 1 per cent of the population suffers from a rare disease. As ERT is only for patients with specific lysosomal storage disorders, the total cost of providing all these patients with the treatment should be less than HK$100 million, which is around 0.25 per cent of the annual recurrent allocation of more than HK$40 billion for the HA.
Patients’ rights advocate Tsang Kin-ping says the HA only considers the cost of medical resources, whereas he thinks cost-effectiveness should be calculated in a broader way. For instance, he says, patients and their caregivers can contribute to the labour force and rely less on social welfare if the patients can recover or control their symptoms after receiving treatment.
Tsang is also critical of the expert panel that assesses whether a patient should receive ERT. He says the decisions involve the use of public funding, but the panel’s member list and agenda are not disclosed. Their decisions are not made public and the panel has no responsibility to explain its rationale for rejecting an application or not renewing a contract. Neither is there an appeals mechanism. Tsang has suggested that the HA invite patients onto the panel but this was rejected.
In a written response, an HA spokesperson said that involving patients in the panel could lead to potential conflicts of interest. As for the lack of an appeals mechanism, the reply said that any application that is rejected can be resubmitted and that the panel would consider any new evidence.
While there are critical voices about the way patients are assessed for ERT, some rare disease patients do not even have any specific drugs to relieve their symptoms. Karen Chau Hiu-yin is a 21-year-old MPS Type IV patient. The symptoms include shortened limbs and trunk, cloudy cornea and ear infection. A drug for MPS Type IV is now available in the United States, but the new drug is not listed in the HA Drug Formulary
Chau’s father, Chau Po-yuen, is the chairperson of Hong Kong Mucopolysaccharidoses and Rare Genetic Diseases Mutual Aid Group, and he has written letters to the HA asking for the inclusion of the MPS Type IV drug in the HA Drug Formulary.
Doctors were unable to correctly diagnose Karen’s problem until she was five years old and attending a special school. Her father noticed she had the same symptoms as a classmate with MPS. He later managed to get a referral to the clinical genetic service and, after a DNA specimen was sent for testing overseas, his daughter was diagnosed as a MPS Type IV patient.
Lam Ching-wan, a professor at the Li Ka Shing Faculty of Medicine, Department of Pathology at the University of Hong Kong, says clinical genetics specialists are important to rare disease patients. They can conduct genetic diagnoses and identify the exact rare disease a patient has, so that a comprehensive treatment plan can be drawn up.
Lam says patients cannot contact genetics experts directly, because the Medical Council of Hong Kong does not recognise clinical genetics as a specialty. The council does not provide any examinations for those who want to be clinical genetics specialists.
Not only are they hard to find, but Lam says there are only a few clinical genetics experts in Hong Kong. He says a formal system to recognise the specialty and a local genetics college to train specialists may help make up the shortfall. But he says genetics is difficult to study and not many doctors are interested. “On average, a specialty involves the study of a hundred diseases, but for this, [genetics], 6,000 diseases have to be studied. It’s sixty times,” Lam explains.