According to the WHO report, there are 5000~6000 rare diseases confirmed at present, accounting for about 10% of human diseases. In the United States, about 25 million people suffer from more than 6,000 rare diseases. In Europe, there are about 5,000-8,000 rare diseases, and the affected population accounts for 6%-8% of the total population of the EU, that is, 24-36 million people. The number of patients with rare diseases in Australia is about 6.5438+0.2 million. The number of patients with rare diseases accumulated by thousands of small numbers is indeed a large number. At present, there is no definition of rare diseases in China, so the data of disease types and incidence population have not been reported.
The purpose of granting the qualification of rare drugs is to encourage the research and development of drugs that are really needed, but the price is not unaffordable or unprofitable for developers under normal circumstances. Due to the treatment of these diseases, the economic benefits of medical research and drug development are not high. Developers do this because they can get tax breaks and extend the exclusive market operation time (7 years after approval). The idea behind the Rare Drugs Act is that long-term exclusive sales will encourage more companies to invest in research. After the implementation of the bill, many such drugs were developed, including the treatment of glioma, multiple myeloma, bladder fibrosis, phenylketonuria and so on.
A similar situation exists in the European Union, where rare drugs are managed by the Rare Drugs Committee of the European Medicines Agency (EMEA). At the end of 2007, FDA reached an agreement with EMEA. Therefore, you can apply for the same application to two institutions, thus reducing the time and funds required for companies to apply for the status of rare drugs. However, the two institutions still maintain separate approval procedures.
Development of rare drugs
Rare drugs, like other drugs, usually follow the prescribed development path. This experiment focuses on the characterization, stability, safety and effectiveness of drug molecules. But the statistical burden can be simplified. For example, when the number of patients with a rare disease is less than 1000, 1000 cases may not be tested in phase III clinical research. Because the market of these products is very small, the cost of developing, researching and producing these drugs may exceed the income, which requires government intervention. Usually, such a market or organization should be established to produce these products. Critics of the free market economy often refer to this as market failure in the free market economy system. Advocates of free market often answer that the development cost will be greatly reduced without government intervention in the form of expanding safety and effectiveness testing.
Under the market economy system, it is very important for the government to formulate relevant stimulus policies to improve the accessibility and accessibility of drug treatment for patients with rare diseases.
Some people abroad have concluded that government intervention can take the following forms:
● Use taxes to support companies that produce or research these drugs.
● Grant others more rights than the traditional patent law allows.
● Provide medical products (including medicines) developed by universities and commercial sponsors for rare diseases.
Biological products, medical devices and medical foods).
Create a government-run company to research and produce these drugs (see this type of example of Crown corporation).
The introduction of foreign drug incentive policy for rare diseases has made many treatment schemes for rare diseases grow from scratch and achieved good results. In the United States, before the implementation of the 1983 Rare Drugs Act, there were less than 10 rare drugs on the market. By February 2007, there were 1697 kinds of rare drugs registered by FDA, of which 302 kinds had been listed. About 1 10 pharmaceutical companies have obtained FDA approval for the rare drug market. Although most companies only have one rare drug, about 40% companies have two or more rare drugs under study. As of April 2007, 447 rare drugs have been registered in the EU, and 34 have been listed. During the period from1993 to 2004, 98 kinds of rare drugs were listed in Japan.
Rare diseases and new drugs for the treatment of rare diseases were mentioned as early as the Administrative Measures for Drug Registration 1999 in China, but there is no clear stipulation so far, and the incentive policy adopted is not obvious. The current Measures for the Administration of Drug Registration (2005 edition) has no further incentive policy for the research and development of rare drugs. In March, 2007, the draft of "Measures for the Administration of Drug Registration" published by the US Food and Drug Administration mentioned that "new AIDS drugs with better efficacy than those already on the market for the treatment of malignant tumors and rare diseases will be included in the' green channel' for special examination and approval to simplify the examination and approval procedures", but there is no special definition of rare diseases and rare diseases. It should be said that China's policies on rare diseases and rare drugs are still relatively backward.
Learning from foreign experience in defining rare diseases and encouraging the research and development of rare drugs can promote the definition and policy implementation of rare diseases and rare drugs in China.