时间：2022-10-16 09:20:13
In China， most of rare diseases are still incurable， while drugs for those curable rare diseases， called as orphan drugs， are highly priced. Moreover， there has more than 130 orphan drugs marketed in the country but only 60 of them are included in the medical insurance system. Therefore， most of rare disease patients can’t afford those expensive orphan drugs.
According to a survey conducted in 2012， it cost Chinese rare disease patients 6，700 yuanon average for orphan drugs in 2011 and 71% of their families said they cannot afford the bill.
Unfortunately， Chinese medical insurance system makes very slow progress to alleviate the tremendous burden on those desperate families who have to pay tens of thousands or even millions of yuan for orphan drugs per year to cure a patient with rare disease.
Profitable but Unavailable
In 2014，with the ice bucket challenge going viral， the disease Amyotrophic Lateral Sclerosis （ALS），also known as Lou Gehrig’s disease or Charcot disease， came to wild public notice. Since then， the voice of rare disease patients has been gradually heard by the public.
Rare diseases affect only a small proportion of the population， most often at birth or in infancy. According to the World Health Organization （WHO）， rare diseases， also referred to as orphan diseases， are defined as diseases or conditions that affect 0.65%-1% of the whole population， Based on the definition， there are about 7，000 kinds of orphan disease across the world currently with patients totaling 350 million.
Due to the low morbidity of rare diseases in China， most of hospitals lack corresponding medical technologies and staff to correctly diagnose such diseases， leading to the frequent incidents of missed diagnoses and misdiagnoses. In some cases， it took a patient with rare disease three to five years to have a definite diagnosis. But what’s even worse is that those patients find those orphan drugs unaffordable and unavailable.
It is reported that there is only 130 orphan drugs marketed in the country， equal to less than 4% of the total number of orphan drugs in the US. Over the past decade， in fact， many traditional pharmaceutical giants have been stepping up efforts in the field of the research and development of orphan drugs， in a bid to take a bite of the lucrative market.
In 2009， U.S. pharmaceutical giant Pfizer entered an agreement with Israeli Protalix Biotherapeutics for the research and development of drugs for Gaucher disease， marking the two drug makers’ first foray into the orphan drug market. Later， Roche， Sanofi-Aventis and Novartis also took a role in the orphan drug field.
Data from Reuter shows that during the period from 2001 to 2010， the global orphan drug market saw an annual growth of 25.8%， higher compared with other drugs’ annual growth of 20.1% over the period.
The global orphan drug market is expected to reach 127 billion that by 2018， implying a compound annual growth of 7.4% during the period from 2012 to 2018， twice that of global Rx drug market.
“Despite of the small population of patients with rare disease， most of orphan drug makers we know made a considerable profit，” said John Crowley， CEO of Amicus Therapeutic， a firm engaged in the research and development of orphan drugs.
Crowley’s two daughters are also affected by rare disease. In 1998， 15-month-old Megan and 4-month-old Patrick were diagnosed with Pompe disease， a rare and fatal neuromuscular disorder that affects only a few thousand children worldwide， usually leaving them with little to no muscle function， enlarged hearts， and severe difficulty breathing. Crowley was told to take their children home and “enjoy their short time together...there is nothing that can be done.”
Determined to find a treatment to save his children’s lives but frustrated with the pace of Pompe research， Crowley leaved his previous firm at the age of 31 to help co-found a start-up biotech firm Novazym， focused exclusively on producing a lifesaving medicine.
In 2001， Genzymeis Corporation， the world’s largest biotechnology firm engaged in the development and delivery of transformative therapies for patients affected by rare diseases， bought out by Genzyme for a consideration of 137.5 million dollars.
Crowley said that his family spent 1 million dollars for the medical treatment， which is unaffordable for ordinary families. According to a report， when Genzyme marketed drugs for Pompe disease in the Chinese market， there were no more than ten patients who can afford the high prices. It cost patients affected by Pompe disease nearly 1 million dollars a year to buy those drugs and few family can afford.
“Foreign orphan drug makers can hardly make a profit in China， as the country adopts a medical insurance reimburse- ment system in which majorities of orphan drugs are not listed，” said Crowley.
According to Crowley， Genzyme spent more than 20 million yuan on entering into Chinese orphan drug market， which is one of main reasons that caused the high prices of orphan drugs.
The tremendous contrast between the actual sales and entry costs in the country has been holding majorities of orphan drug makers back for years. Some orphan drugs have been withdrawn from the market for economic or therapeutic reason.
Currently， China has more than 130 orphan drugs marketed in but only 60 of them are included in the medical insurance system. Therefore， most of rare disease patients can’t afford those expensive orphan drugs.
According to a survey on the survival situation of Chinese patients with rare disease in 2012， the average cost of medical treatment for Chinese rare disease patients is about 6，700 yuan， much higher than the average income of a family. Data showed that 71% of families said they cannot afford the bill and only half of patients jointed social security program which determined their survival rate of most of patients to some extent.
According to the 2009 version of the National Essential Medicine List， among 307 kinds of essential medicines， only 3 kinds of medicines are used to cure rare diseases. Besides， only 57 out of 130 kinds of orphan drugs are listed in the national medical insurance system. What’s more， expenses in only 10 of those orphan drugs can be full reimbursement.
Qingdao Mode
It is impossible for the medical insurance system to pay all the bills for the small population of rare disease patients for now. On the other hand， orphan drugs belong to biological medicines， which require years-long complicated research and development， and most of orphan drug researchers are top-notch international scientists.
In fact， generic drugs can be used as substitutes to lower the orphan drug prices. However， domestic pharmaceutical firms are unwilling and incapable to make generic drugs.
“In view of the unaffordable prices of orphan drugs， it is a top priority to increase the accessibility of orphan drugs，”said Huang Rufang， director of the Center for Orphan Dis- ease Development. “China’s city Qingdao allows expenses in 20 high-priced drugs， be reimbursed through various ways， most of which are orphan drugs， performing best in tackling the problem of inaccessibility of orphan drugs.”
Under the Qingdao model， the government， pharmaceutical firms and patients share the expenses in orphan drugs， which is the new weather vane of the medical aid system for serious diseases.
According to the official data， the Financial Department of Qingdao City spent 300 million yuan for the program， while pharmaceutical lowered the prices of medicines. Patients only need to pay a small portion of medical treatment expenses. The Qingdao government also encouraged social organization and charities to donate some funds.
In such circumstances， patients with rare disease only need to pay 10，000 yuan to 20，000 yuan for orphan drugs， according to Huang.
With high-priced medicines hardly being included in the medical insurance list， Qingdao model is viewed as the best market entry opportunity by special medicine providers.
“Of course， strengthening the research and development of domestic orphan drug as soon as possible is the fundamental way to lower unattainable orphan drug prices，” said Huang.