On May 27th, the approval of foreign drugs for rare diseases in China accelerated. It is reported, by May 25, "about to encourage pharmaceutical medical equipment innovation to speed up the new medical devices to appear on the market for examination and approval of the review of relevant policy" (draft) of social opinion has feedback to state food drug safety administration (CFDA). 

 

Take the recent listing of the beauty of Pompeii, for example. Wu qingyi, director of the China special medicines department of sanofi, told the economic report in the 21st century that the us and the Chinese people have entered the fast-track approval channel through a three-stage clinical trial process. Since listing in the United States in 2007, the drug has treated more than 2,000 Pompeii patients in more than 60 countries. 

 

However, in addition to speeding up the approval of drugs for rare diseases, high prices remain to be resolved. Shanghai children's medical center in cardiology, director of medical master standing army, points out that many patients will have economic problems, such as a 15 kg of Pompeii disease in children with cost about 500000 yuan. 

 

Going public for 10 years 

 

Pompey's disease is one of a handful of rare diseases exist in effective treatment methods, the incidence of neonatal about 1/40000, at present our country diagnosed with only hundreds, more patients failed to timely diagnosis. 

 

The number of rare cases in China has reached 20 million. First rare disease diagnosis and treatment center of Shanghai children's medical center was founded in Shanghai in 2015, a total of more than 1500 cases of diagnosis and treatment of, confirmed nearly 400 cases of rare diseases, first reported in China has more than 30 kinds of rare disease. 

 

Rare diseases prevention and control of Shanghai, chairman of the foundation, the national health development planning commission of diagnosis and treatment of rare diseases schuster mm with security expert committee, deputy director of the committee to the 21st century economic reporter revealed that the health law draft have been identified, which has its own part involves the rare diseases, China will also be published for the first time the "rare diseases" list of countries. 

 

CFDA institutions such as for rare diseases is becoming more and more attention, successively introduced including rare diseases drugs for drug registration "priority review approval" and other policies, proposals, cover has clinical value extremely, urgent clinical needs of drugs. The U.S. and the United States have been approved by the CFDA for a three-stage clinical trial process. 

 

Issued by the CFDA on May 11, 2017 "about encourage pharmaceutical medical equipment innovation to speed up the new medical devices to appear on the market for examination and approval of the review of relevant policy" of the draft, emphasized support for rare diseases treatment and medical equipment research and development. 

 

The economy is in a bind 

 

China has only a handful of hospitals with a proven ability to detect and verify the quality of testing equipment and inspectors. Therefore, enhancing the use of the diagnosis of Pompeii disease is the key to saving the patients of Pompeii. 

 

In li-jun fu, Pompeii disease the most effective treatment is prevention: "Pompeii disease early diagnosis and early treatment is the key to obtain the best curative effect, otherwise will lead to serious irreversible consequences. Clinical studies have shown that in Taiwan through newborn screening to early diagnosis and early treatment of infantile Pompeii disease, can obtain the best therapeutic effect." 

 

Therefore, fu suggested that the government should step up screening efforts for a small number of preventable, treatable genetic diseases, such as Pompeii. For those who are diagnosed, the registration system for rare cases should be established to provide financial assistance to patients through the model of "health insurance + business insurance + social charity". 

 

Before the approval of the Chinese mainland, Pompeii patients had to rely on palliative care to relieve symptoms. It is reported, Shanghai children's medical center from 2005 since the first case of Pompeii disease diagnosis, li-jun fu exploded near 50 cases of infantile Pompeii patients, 42 children have died, including 40 children die before the age of 1. 

 

But despite the approval, the high cost of treatment remains a serious challenge. Pompeii patients require lifelong treatment, and the cost of treatment is closely related to the dosage, must use 20 mg per kilogram of body weight, a 15 kg child treatment costs about 500000 yuan a year, and with weight gain and cost of treatment is rising year by year. 

 

Thanks to a large population base, international drug companies such as sanofi, novartis and Merck are betting on China's "orphan drugs" market. But in the case of Mr Li, the mere drug delivery is unrealistic, because the cost of developing drugs for rare diseases is extremely high. 

 

"It takes government, social groups, medical institutions, patient organizations and businesses to work together to get patients with rare diseases to use the drugs and use them." The Shanghai rare disease prevention foundation has tried to establish a payment model for some rare cases, Mr. Li said.