To give hope to rare disease patients, Hong Kong must revamp its drug policies

Last week was a remarkable one for rare disease patients in Hong Kong, with the government announcing that the long-awaited spinal muscular atrophy (SMA) drug would be available in the city very soon. Last October, Chief Executive Carrie Lam Cheng Yuet-ngor met SMA patients and their parents outside her office and promised that her administration would do all it couls to table a sound solution for them. The government has also committed to improving health care support and services, especially for rare disease patients, in the 2017 policy address. Unfortunately, as quickly as the bureaucracy has responded, this is a matter of saving lives and patients cannot wait. In fact, in her latest radio interview, Josy Chow Pui-shan, the SMA patient who earlier wrote to Lam, revealed that over the past five months the cruel reality is that her condition has worsened, with multiple admissions to the intensive care unit and deteriorating mobility. With abundant financial reserves and unprecedented determination to improve health care support, why is it so difficult for the government to give rare disease patients access to the needed resources? Hong Kong single mother with rare genetic disease dies following emotional appeal to lawmakers for drug subsidies Even though biomedical technology has been selected as one of Hong Kong’s strategic innovation areas, our regulations are far from up to date. For a new drug to be registered and sold in Hong Kong, the manufacturer has to first obtain market authorisation certificates from two “advanced” countries on our Department of Health’s list. Using only two fingers, Hong Kong girl types 22-page proposal calling for life-saving muscle drug In other words, even if an innovative drug has been reviewed and approved by the US Food and Drug Administration or Australia’s Therapeutic Goods Administration, it is not good enough for Hong Kong. This requirement effectively lengthens the introduction of a new drug by a year after it is first registered elsewhere. With such a high regulatory barrier, it is not surprising that drug manufacturers initially had no interest in introducing the SMA drug to Hong Kong. In the long run, this regulation must be reviewed if we are to help rare disease patients gain access to the latest drugs. This would also boost Hong Kong’s innovation push, which could also benefit patients. Given the current barriers, the government should also consider enabling manufacturers to introduce an “expanded access programme”. This aims to provide patients whose condition is rapidly worsening, and who have exhausted the best therapy available and cannot wait for a drug to go through the regulatory journey, with early access to investigational drugs. In fact, the early access programme is officially written into FDA policy and guidelines are available to help patients or physicians navigate the process. Rare diseases is one of the few areas where both the pan-democrat and pro-establishment parties share the same view In the UK, the manufacturer of the SMA treatment drug has promised to provide patients enrolling in the early access programme with a lifetime supply of the drug while they are waiting for the health authority’s reimbursement decision. Korea and Taiwan are already on the list of early access locations for the SMA treatment drug. This enables the most seriously ill patients to be treated as soon as possible, even before the drug is formally registered and costs are reimbursed by the governing body. It is a pity that SMA patients can access the drug in Taiwan, just a short flight away, whereas in Hong Kong they still face an agonising wait. Many more rare disease patients need urgent help. Rare diseases is one of the few areas where both the pan-democrat and pro-establishment parties share the same view. The government must take this chance to promptly review the drug registration policy, while the early access programme could be key to timely access to the SMA drug in the meantime. Sadly the lives of rare disease patients are in the balance. Now is the perfect moment for innovation in public administration and policy execution to benefit these people, and all those in similar need in Hong Kong. Raymond Mak is co-convener (Engagement) at the Path of Democracy and executive director of the Hong Kong Academy of Politics and Public Policy