Rare disease activists call for formal programmes, not ad hoc efforts

There is an area in which Hong Kong lags well behind mainland China in the healthcare: its approach to rare diseases. This week, calls have risen to close a gap in legislation to support patients with rare diseases. Those calls have been met with lukewarm response from health officials.

On Tuesday, Labour Party lawmaker Dr Fernando Cheung submitted a 39-page draft bill to the Legislative Council (LegCo) to push for a definition of rare diseases, the registration of rare disease drugs, a subsidy scheme for rare diseases, a commission on rare diseases policy and a database for rare diseases.

The lack of an official definition of these diseases, according to Dr Cheung, has resulted in a delay in diagnosis, a limited number of patients getting subsidized, an absence of a patient registry and insufficient social care services for these patients.

FHB: Case by case

But health officials deem legislation unnecessary, citing existing measures for patients with rare diseases.

“The statutory regime as proposed for rare diseases would introduce an unnecessary legal divide between rare and non-rare disease patients and would complicate clinical treatment processes,” the Food and Health Bureau and the Hospital Authority said. “The proposed bill will also have policy and resource implications on the government.”

Mr Perry Lai, business development officer of the Hong Kong Alliance for Rare Diseases, tells Harbour Times why legislation is essential.

War for drugs

A separate branch in the public healthcare system for rare diseases could help expedite drug registration and get more funding for buying orphan drugs, he says.

“The current mechanism places great emphasis on cost-effectiveness. When it comes to allocating resources, priority is given to drugs that can treat more patients. The low number of incidence has placed patients with rare diseases at a disadvantage,” Mr Lai explains.

“Some policy support could attract foreign pharmaceutical companies to bring their drugs to the Hong Kong market,” he adds.

Currently, it is no easy task for patients with rare diseases to get the medication they need. Orphan drugs, already scarce and expensive, need to be introduced to the local market by the foreign drugmakers and go through a myriad of registration procedures to finally reach patients.

Some are not rare enough

These drugs could be too expensive for many. Dr Cheung said many patients are outside the current safety net, the Samaritan Fund, or denied access to publicly-funded medication due to a lack of an official definition in Hong Kong.

One in every 67 Hong Kongers, or 1.5 percent of the population, suffers from a rare disease. That means there are an estimated 111,000 patients in the city.

Mr Lai says rare diseases are complex and patients need multidisciplinary care.

“Patients with rare diseases suffer from misdiagnosis and it normally takes five years after consulting seven or eight doctors to get the right diagnosis. They also need to see doctors from different departments, which is time-consuming and inefficient,” he explains.

Currently, the Hong Kong government does not put rare diseases in a category so there is no guideline for helping these patients.

“Besides registration or funding in the healthcare system, these patients also need support in terms of diagnosis, rehabilitation, employment, family planning. It is a lifelong strategy,” Mr Lai says.

There have been global examples where specific laws for rare diseases are formulated and implemented.

America, Japan, EU, Australia, Taiwan, South Korea, China …not Hong Kong

The U.S. was the first to pass the Orphan Drug Act, which includes incentives and assistance to encourage drugmakers to develop orphan drugs for small groups of patients. Japan, the EU, Australia, Taiwan and South Korea have also passed legislation to enable similar orphan drug designation systems and provide incentives.

The latest example is China, which published last year its first national list of rare diseases with 121 indications. The move is set to encourage research and development of orphan drugs and make them more affordable.

“If such legislation has been in place in other countries for decades, and even in countries less developed than Hong Kong, then why can’t the Hong Kong government follow suit?” Mr Lai argues.

Last year, chief executive Mrs Carrie Lam personally reached out to a pharmaceutical company to bring Spinraza, a drug to treat spinal muscular atrophy, to Hong Kong. A few patients eventually got the medication because of her intervention.

“You cannot always have the chief executive to speak for a particular group of patients. We welcome administrative measures to solve issues faced by the patients, but this is not a long-term approach. Legislation is the only way,” Mr Lai says.