Orphan drugs: encourage local imitation, promote cross-border cooperation and development
Recently, the "Pharmaceutical Economics" reporter learned at the 4th China Rare Disease Summit Forum in 2015 that there are 7,000 species of rare diseases known worldwide, and 5,78 species have been found in China, but 378 species are available worldwide. Among the medications, there are less than 100 species in China (92 species of indications).
According to the research progress of the related research topics of Peking University Medical Management International Research Center, the diagnosis technology for rare diseases in China has been relatively mature. It is urgent to establish an emergency registration channel for rare diseases, adopt a mature one, incorporate one method, and then gradually adopt legislation. The level is perfect.
Encourage local imitation and promote cross-border cooperation and development
Experts at the meeting pointed out that due to China's limited research and development capabilities, the production of rare disease generic drugs could be relieved or the clinical needs were alleviated. Professor Duo Tao, the dean of the First Maternal and Child Health Hospital affiliated to Tongji University, pointed out that the urgent need for clinical rare drugs The degree is imminent, and the production of generic drugs takes 3 to 5 years. This has lost the most precious treatment time for rare patients.
The shortage of rare disease drugs does not exist only in China, and less than 20 drugs in the United States are approved through special development of rare diseases. Judging from the regulations and policies introduced by China in recent years, it is indeed encouraging local enterprises to speed up the rare pathogens of imitation patents, but there are not many companies that tend to copy them.
"Although the laws and regulations of many countries clearly stipulate the extension of the patent period for the development of rare diseases, at the same time, WHO's treatments for rare diseases, AIDS and other patents that are not expired in principle also encourage enterprises to carry out imitation research and development in order to benefit the rarer. Patient," Duan Tao said.
With the rapid advancement of China's international economic status, the price of imported drugs is also high. Take a clinically used rare disease drug as an example. The price of 30 tablets is 8800 yuan in China, 5500 yuan in Taiwan in China, and 5900 yuan in the United States. In addition, from the perspective of reimbursement, most rare diseases in China have not been covered.
Duan Tao suggested that under the current situation, for the treatment of rare diseases, Chinese pharmaceutical companies can fully explore the model of cooperation with India to develop drugs that have been copied in India for clinical patients in China, and better avoid international drugs. A barrier to patent protection. He believes that this practice is not limited to rare diseases.
“Whether it is a generic drug or a original drug, since about 80% of the rare diseases found are caused by genetic defects, from the clinical use of drugs, although there are the best drugs designed for Chinese patients, those without There is not much racial differentiation in drugs that have been tested in Chinese drug clinical trials,” said the expert.
Diagnostic technology is relatively mature
Huang Rufang, director of the Center for Rare Disease Development, admitted that about 50% of rare diseases worldwide can develop at birth or during childhood, and the condition often progresses rapidly. Therefore, early detection and diagnosis are extremely important. Genetic diagnosis is one of the most effective and promising diagnostic methods for rare diseases.
Gene detection technology can not only verify the clinical diagnosis of the disease, provide accurate genetic counseling services for patients, but also be used for the detection and prenatal diagnosis of pathogenic gene carriers. "In combination with the current clinical application, China's genetic testing technology has been at the forefront of the world, and the cost of testing has been declining, even down to 1,000 yuan / person." Yin Wei, former chief operating officer of Huada Gene, pointed out.
Shi Luwen said that although China has been able to detect rare diseases more quickly in the clinic, unfortunately, due to the extremely low incidence of rare diseases, the use of drugs lacks commercial profits. So far, there is almost no orphan developed independently in China. The drug is on the market.
Since the 1980s, the United States, the European Union, Australia, Japan, South Korea and other countries and regions have successively enacted a rare disease-related bill, which has opened the way for orphan drug research and development in terms of approval channels, tax cuts, and market exclusivity. At least 40 countries and regions around the world have passed orphan drug related bills. “Although China has mentioned the registration, clinical trials and approval management of rare disease drugs in some relevant regulations since 1999, there are still no specific operational details. The clinical trial of rare diseases in the 2007 edition of the Drug Registration Management Measures. There are two preferential measures for approval and approval, but the specific definition or scope of rare diseases is still not given. It is difficult for Chinese medical experts, research institutions and government scientific research departments to carry out systematic research, which makes us medical research and drug development for rare diseases. The level is still at a low level, many patients still can not get a clear diagnosis, long-term misdiagnosis or missed diagnosis, drug accessibility is low." Shi Luwen pointed out.
In response to the current situation of research on rare diseases in China, experts suggested that the first is to implement the definition of the "list system" of rare diseases as soon as possible, bypass the test of the incidence rate, and find that one type is included, and a unified rescue mechanism and reimbursement are established. Policy, etc. The second is to establish an emergency channel for orphan drugs. At this stage, China conducts special evaluations on some drugs that are urgently needed in clinical practice. However, it may be suitable for a few patients with rare diseases. The state should establish an emergency channel to allow appropriate drugs to enter China more quickly in order to treat patients. The third is to set up a special management department, which is coordinated by a special department, and multi-sectoral coordination to solve the problem of rare diseases and orphan drugs, and play the role of more social non-governmental organizations. The fourth is to actively encourage basic research on rare diseases and related innovative drug research. Once the root cause of rare diseases is found, it will bring greater benefits to the health of all human beings. (Jilin Qijian Biotechnology Co., Ltd., www.qjbio.com.cn)
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