Drug shortage for patients with rare condition

Pan Longfei has a masculine name. It means "a dragon flies in the sky." But his unwanted feminine appearance and voice make him a target for jokes.

Nicknamed "Old K", 26-year-old Pan was diagnosed in 2011 with Kallmann Syndrome, a rare condition characterized by delayed or absent puberty and an impaired sense of smell. The syndrome affects one in 10,000 to 86,000 people, with males suffering more than females.

For Kallmann Syndrome patients to maintain their sexual characteristics they need 2,000 IU human chorionic gonadotropin (HCG), a hormone.

Zhang Bin, a professor with the Third Affiliated Hospital of Sun Yat-Sen University in Guangzhou, Guangdong Province, said that the syndrome can only be helped with a weekly injection of HCG or human menopausal gonadotropin (HMG).

Treatment lasts between one and two years, after which, male patients can possibly restore fertility, he said.

But Zhang said his hospital has a shortage of 2,000 IU HCG.

Other hospitals face a similar situation. Since HCG is not an orphan drug that has been developed specifically to treat a rare medical condition, Kallmann Syndrome patients face more challenges as sufferers of other disorders or diseases can be prescribed the drug.

A doctor with the maternal and child care service center in Guangdong revealed that pharmaceutical companies have stopped producing HCG, leaving patients like Pan in a panic.

"I need HCG. I have a right to get the drug," Pan said on his microblog.

For pharmaceutical companies, it makes no sense to produce certain drugs.

"When we decide whether we should produce a certain drug, we consider market demand, profit and feasibility," said Wang Wenchu, deputy head of the Guangzhou Baiyunshan Pharmaceutical General Factory.

HCG's low profit dampens manufacturers' enthusiasm, and its raw material, pregnant women's urine, is difficult to collect, said Huang Rufang, director of the Chinese Organization for Rare Disorders.

"Owing to the raw material shortage, drug makers cannot make bricks with straw," Huang said.

Even for an orphan drug China still lags behind in terms of the legislation.

Globally, more than 30 countries have unveiled laws to offer preferential conditions to drug makers.

The United States promulgated the Orphan Drug Act in 1983, under which, companies that develop such a drug may sell it without competition for seven years, and may get clinical trial tax incentives. Since then, the output of orphan drugs has increased ten-fold.

In China, to have posterity and continue the family line are regarded as an essential part of filial piety. Many Kallmann Syndrome patients choose to conceal their disease, and bear the pain all alone.

"To guarantee the supply of the drug is what we can do in preventing orphan disease patients like Pan from becoming orphans of our society," Zhang Bin said.