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 Rare illnesses, by definition affecting only a small group of
people, are often genetic and costly to treat or control.
But 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 to $209
billion in the next five years from $124 billion this year.
That growth - an important driver for companies like 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. It sells immunology and
hemophilia drugs in China.
He estimated, however, that for some rare illnesses, like Fabry
disease, caused by the build-up of fat-like substances, fewer than
five 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 8-year-old Hu Yizhuo from the eastern
city of Nanjing, who has tuberous sclerosis complex (TSC), a rare
genetic disorder that causes benign tumors to grow in the brain and
around the body.
To control his symptoms, including frequent seizures, he takes daily
doses of Sabril, made by Sanofi SA, and Pfizer Inc's Rapamune. But
there's 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 Turkey, Taiwan and 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 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, though not with preferential 'orphan' drug status. Sanofi did
not respond to requests for comment.
China's health ministry declined to comment, though the China Food
and Drug Administration said last week the ministry would look to
increase support for developing drugs and medical equipment to treat
rare diseases.
MILLION DOLLAR DRUGS
Hu is one of an estimated 16 million Chinese with rare diseases,
though 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 United
States, leading to the approval of more than 600 'orphan' drugs and
incentives for firms to develop more.
Li Dingguo, chairman of the Shanghai Rare Disease Prevention and
Treatment Fund, said China's draft list covers over 100 diseases: a
basis for policy and the starting point for a debate on better
diagnostics, primary care and financial support. The health ministry
has been seeking feedback, and could publish the list this year.
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"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."
RARE CURES
China is slowly overhauling its healthcare system: it expanded its
national reimbursable drug list (NRDL) this year for the first time
since 2009, though it is still dominated by cheaper, essential
medicines. [nL4N1G824C]
It also flagged moves this month which lay the groundwork to speed
up 'orphan' drug approvals - key given the huge regulatory backlog -
and even clinical trial waivers.
But Beijing is also looking to trim a potential healthcare bill of
$1 trillion by 2020 and is on a major drive to reduce drug prices -
out of kilter with often high-priced specialty 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, director of the Chinese Organization for
Rare Disorders.
More local firms, too, are weighing up the 'orphan' drug market,
though most remain wary.
Zheng Weiyi, chairman of Nanjing Yingnuo Pharma & Technology, said
his company was among those developing 'orphan' drugs for both 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 told Reuters.
"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," said Shire's Fang.
Hu's mother, meanwhile, 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."
(Reporting by Jackie Cai, Anita Li and Adam Jourdan; Additional
reporting by Clara Ferreira Marques; Editing by Clara Ferreira
Marques and Ian Geoghegan)
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