China is to publish its first national list of rare diseases to guide policymakers as part of a broader overhaul to improve diagnoses and speed up drug approvals in the world’s second-largest drug market.
Rare illnesses, by definition affecting only a small group of people, are often genetic and costly to treat or control.
However, they are an increasingly significant segment of the market, and of big pharmaceutical firms’ profits.
Global sales of so-called “orphan” drugs to treat rare diseases are set to increase from US$124 billion this year to US$209 billion in the next five years.
That growth — an important driver for companies such as Celgene, Bristol-Myers Squibb, Novartis, Johnson & Johnson and Shire — is double that of the wider pharmaceuticals market, according to consultancy Evaluate.
“China, with the largest population in the world, should also have the largest population of rare diseases,” said Peter Fang, head of Asia-Pacific for Shire, which has a focus on rare illnesses and a portfolio of specialist therapies.
Shire sells immunology and hemophilia drugs in China.
However, Fang estimated that for some rare illnesses — such as Fabry disease, caused by the buildup of fat-like substances — less than 5 percent of patients in China are diagnosed.
In China’s broad, but shallow healthcare system, rare illnesses have been largely ignored, leaving patients outside the safety net. Drugs they need are hard to get hold of or are expensive, with no reimbursement under public insurance policies.
One such medical “orphan” is eight-year-old Hu Yizhuo from Nanjing, who has tuberous sclerosis complex, a rare genetic disorder that causes benign tumors to grow in the brain and throughout the body.
To control his symptoms, including frequent seizures, he takes daily doses of Sabril, made by Sanofi, and Pfizer’s Rapamune, but there is a catch. The two drugs are not easily available in China, where a focus on cost control and long approval backlogs means many specialist drugs are often out of reach.
Instead, Hu’s parents get the drugs smuggled from Taiwan, Turkey or Hong Kong via agents, or from local doctors prescribing them for off-label use.
“My son needs his medicine, without it he could die,” said his mother, Fang Liuyan, 39, a former accountant, adding that there was no way to buy them via regular, approved channels in China. “We don’t care [about the legality], any risks are secondary to being able to control his condition.”
Pfizer said Rapamune was available in China through a joint venture firm, although not with preferential “orphan” drug status.
Sanofi did not respond to requests for comment.
The Chinese Ministry of Health declined to comment, although the Chinese Food and Drug Administration last week said that the ministry would look to increase support for developing drugs and medical equipment to treat rare diseases.
Hu is one of an estimated 16 million Chinese with rare diseases, although there is scant data and even less financial support. Only a fraction receive treatment.
Campaigners and pharmaceutical companies hope the new list can begin to change that, as the Orphan Drug Act of 1983 did in the US, leading to the approval of more than 600 “orphan” drugs and incentives for companies to develop more.
Li Dingguo (李定國), chairman of the Shanghai Rare Disease Prevention and Treatment Fund, said China’s draft list covers more than 100 diseases: a basis for policy and the starting point for a debate on better diagnostics, primary care and financial support.
The ministry has been seeking feedback, and could publish the list this year.
“Here in China, the state has good intentions, but there is no detailed regulation, no preferential policies and no tax breaks” for orphan drugs, Li said. “Because China lacks these medical safeguards, we see lots of patients with rare diseases just waiting to die.”
China is slowly overhauling its healthcare system: It expanded its national reimbursable drug list this year for the first time since 2009, although it is still dominated by cheaper, essential medicines.
It also flagged moves this month that lay the groundwork to speed up “orphan” drug approvals — key given the huge regulatory backlog — and even clinical trial waivers.
However, Beijing is also looking to trim a potential healthcare bill of US$1 trillion by 2020 and is on a major drive to reduce drug prices — out of kilter with often high-priced speciality treatments for rare diseases that have a limited market.
“They need to consider a lot of people’s interests, so it’s quite tough,” said Huang Rufang (黃如方), director of the Chinese Organization for Rare Disorders.
More local firms, too, are weighing up the “orphan” drug market, although most remain wary.
Zheng Weiyi, chairman of Nanjing Yingnuo Pharma & Technology, said his company was among those developing “orphan” drugs for the Chinese and global markets.
“There are no subsidies and the only supportive policy has been getting orphan drugs on the fast-track approval list,” he said. “We think, though, that even without supportive policies we can still make money from getting products to market.”
Some highlight the changes in China could encourage more research into diseases that are more prevalent in the country, such as certain sub-types of Fabry disease.
“There’s a possibility of looking at Chinese specific rare diseases or mutations,” Fang said.
Meanwhile, Hu’s mother can only wait.
“This disease can mean a lifetime of taking medicines, which we now pay for ourselves. Emotionally and financially, it’s a huge strain,” she said. “I hope in future the drugs could go on the insurance list and help relieve some of the pressure on us.”
Additional reporting by Anita Li and Clara Ferreira Marques
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