Medical experts call for greater access to rare disease treatments

Some of China's leading medical experts have suggested establishing a "1+4" multiparty payment mechanism to make exorbitant treatments for rare diseases available to more patients.

The "one" refers to the basic national medical insurance, meaning that drugs for rare diseases shall be included in the country's list of drugs that can be partially reimbursed, they said.

There have been such pilot programs in some provinces in recent years but the practices throughout all the provincial-level regions should be uniform, said Ding Jie, a national political advisor who was among those who made the suggestion.

"The 'four' refers to: the setting up of special relief programs at both national and provincial governments, integrating social resources, introducing commercial insurance and having individuals pay for part of the medical bills," said Ding during a live broadcast on Wednesday about increasing the accessibility and affordability of rare disease therapies for patients.

She said the suggestion was a result of extensive discussions with experts in medicine, public health, medical insurance, law and economics.

Wang Lin, a member of the expert committee of diagnosis, treatment and security of rare diseases with the National Health Commission and vice director of the rare disease branch of the Beijing Medical Association, said that Zhejiang province has already set up a special relief program at the provincial level to "help patients with the payment of expensive therapies as much as possible while relieving the pressure of medical insurance funds".

Each person in the province paid 2 yuan ($0.28) every year to the relief program, according to Wang.

She added that the country has made progress over the past two years in terms of accelerating the introduction of innovative rare disease drugs to the domestic market to increase drug availability.

The latest examples include the first effective drug for Fabry, a lethal rare malady which is caused by a deficiency of a particular enzyme. The drug was made available to Chinese patients last month.

Another drug for mucopolysaccharidosis, a rare metabolic disorder caused by the absence or malfunction of lysosomal enzymes, was approved by the National Medical Products Administration earlier this week.

Experts have also pointed out that families with rare disease patients accounted for a large number of those who were dragged into poverty due to illness.

"Helping such families through various means also contributes to the country's efforts in poverty alleviation," Ding said.