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| American orphan drug discovery accelerated, China's orphan drug development trend development |
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| Author：中國銘鉉 企劃部 Release Time：2017-8-23 11:16:59 Number Browse：715 |
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An orphan drug is used to diagnose, treat and prevent rare diseases on August 23.According to the world health organization (WHO) : the definition of a rare disease with patients of total population of 0.65 ‰ ~ 1 ‰ of the disease, according to every country around the world, the standard of identifying a rare diseases exist certain differences, mostly in prevalence or the number of cases as the standard definition of rare diseases.
Since the 1980 s, the development of the orphan drug problems begin to cause the attention of the society, there are a lot of countries and regions have established the rare diseases related institutions or organizations, special laws and regulations has been formulated to promote the development of orphan drugs.With the increasing attention of countries around the world, the research and development of drugs for rare diseases is becoming the focus of large pharmaceutical companies.
In 2016, sales of orphan drugs in the world amounted to $114 billion, an increase of 12.2% over 2015.According to Evaluate Pharma, expects global prescription drug market in 2022 to $1.06 trillion, among them, 32% of its sales growth will come from orphan drug products, compound annual growth rate will reach 11% in the next five years, than the growth rate of prescription drugs (5.3%) more than doubled, the importance of orphan drug already.
America: the proportion of new drugs for orphan drugs,
The first batch of the indications is over 60%
Since 1983, when the American orphan drug act was introduced, orphan drugs account for a large proportion of the new drugs approved by the FDA each year.The FDA has adopted an "orphan drug identification, post-ipo approval" method for orphan drug registration.
Since 1983, the FDA has approved a 625 items listed orphan drug, qualification for orphan drug of nearly 4200, of which the first half of 2017 (as of June 30), approved by FDA orphan drug 37 (indications according to the statistics), 256 granted orphan drug qualifications.
In the past five years, the number of orphan drugs approved by the FDA has increased rapidly, and the first half of 2017 has exceeded the number of approvals for all of 2016.
In recent years, the research focus in the pharmaceutical industry is concentrated in the anti-tumor, anti-infection, nervous system disease, and blood system diseases, etc., and the orphan drug has a place in all areas, including antitumor orphan drug approved 19, more than 50% of the total number of approvals.Of the 37 indications approved, 23 were approved for the first time, more than 60 percent of the total, and the absolute strength of the American orphan drug research and development field.
China: gradual integration of international,
Local companies have begun to dabble in research and development
There is no official definition of rare diseases and the definition of orphan drug identification standard. There is no clear policy regulation and special examination and approval department.At present, China's orphan drug development market is almost blank, relying mainly on imports.From 1983 to 2011 in the United States in 360 kinds of orphan drug to market, only nearly 60 in the Chinese market, breed mostly before 2002 in the United States with orphan drug qualification listed anti-aids drugs, anti-tb drugs, falciparum malaria, leprosy, cysticercosis, cryptococcal meningitis, intestinal amebiasis and other drugs.In addition, due to the lack of selection mechanism for orphan drugs, the coverage of orphan drugs in the reimbursement directory is low, the reimbursement rate is not high, but it can be low.
With the society's emphasis on rare diseases, rare diseases and orphan drugs gradually come into the view of China's policy law, and some policies have been introduced to stimulate the development of orphan drugs.2009 CFDA formulated the special examination and approval of the new drug registration management regulations, make clear a regulation for treating diseases such as AIDS, malignant tumor, a rare disease and clinical treatment has obvious advantages of new drugs to carry out special examination and approval.In the 12th five-year plan of the state council put forward to encourage drug research and development of rare disease, then the CFDA introduced a series of new drug approval reform policies on rare diseases medicine for special groups such as separate review of channels, multi-channel dynamic additional information as well as the examination and approval of special policy, such as shorter time.
With the support of state incentives, local companies have also been involved in the development of orphan drugs.According to statistics released by CDE in the first half of 2017, 12 rare diseases have been given priority reviews.Compared with the United States, the development of orphan drugs in China still has a great distance.
American orphan drug research and development boom is inseparable from America's generous incentives.The United States, the European Union and other countries set up special orphan drug certification and management, and with the orphan drug act as a starting point, established perfect orphan drug policy and law system, including: research and development funding plan;New drug application fee waivers, clinical trials reporting acceleration mechanism of examination and approval, review, priority review vouchers, up to 70% preferential tax policy and market monopoly, the development of drugs for rare disease has injected vigor and vitality.
Relatively speaking, the research and development of Chinese orphan drugs is still in its infancy, but based on the large population base of China, the medical needs of orphan drugs in China will have great space.At the same time, China is facing the critical period of the upgrading of the pharmaceutical industry, and encouraging enterprises to develop the orphan drugs with high availability and high sex will have important social significance and economic value.According to the data, the number of patients with stomach cancer, liver cancer and esophageal cancer in China in 2016 was 6791 million, 4661 thousand and 479,000, which were classified as rare diseases in the United States.Therefore, developing these orphan drugs could become a fast way for Chinese pharmaceutical companies to seize a place in the us market under the boom of the global strategy of the pharmaceutical industry.
Conclusion the < < <
Government intervention is an effective measure to protect the rights and interests of rare patients. There are still a lot of things in China to encourage the development of orphan drugs in China and improve the availability of specialized drugs in the country.Looking forward to our country to draw lessons from the experience of the leading countries such as America, formulated in accordance with China's specific national conditions orphan drug r&d incentive policies, enlarge the coverage of orphan drug, improve accessibility, burning local enterprises r&d enthusiasm, promote the development of Chinese orphan drug, the benefit of a rare disease.(author's unit: dongguan jinan university research institute international drug registration platform)
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