According to the WHO, there are 5,000 to 6,000 recognized rare diseases, accounting for about 10 percent 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 people account for 6%~8% of the total population of the European Union, i.e. 24~36 million people. In Australia, the number of people affected by rare diseases is about 1.2 million. The number of rare disease populations accumulated by thousands of small numbers is indeed also a large number. In China, the data on the types of diseases and the affected population have not been reported because rare diseases are not defined at present.
The purpose of granting rare drug status is to encourage the development of drugs that are needed, but whose prices are not normally unaffordable or unprofitable for developers. Medical research and drug development are not economically viable for the treatment of these diseases. Developers do this because they get tax breaks and extended exclusivity (7 years after approval). The idea behind the Rare Drug Act is that long-term exclusive sales will promote more companies to invest money in research. Many such drugs have been developed since the act was implemented, including treatments for glioma, multiple myeloma, bladder fibrosis, and phenylketonuria.
A similar situation exists in the European Union, where rare drugs are regulated by the Committee on Orphan Medicinal Products of the European Medicines Agency (EMEA), and an agreement was reached between the FDA and EMEA in late 2007. In late 2007, an agreement was reached between the FDA and the EMEA whereby the same application can be made to both bodies, thus reducing the time and money required for companies to apply for rare drug status. However, the two agencies still maintain separate approval processes.
Development of Rare Drugs
Rare drugs, like other drugs, usually follow a defined development pathway. Trials focus on characterization of the drug molecule, stability, safety and efficacy. But the statistical burden can be simplified. For example, when there are fewer than 1,000 patients with a rare disease, 1,000 cases may be excluded from a phase III clinical study. Since the market for these products is inherently small, as the cost of developing, researching, and producing these drugs is likely to exceed the revenues, government intervention is needed, usually to create such a market or organization to produce these products. Critics of free market economics often cite this as market failure in a free market economic system. Advocates of free markets often respond that in the form of expanded safety and efficacy testing, development costs would be considerably lower without government intervention.
In a market economy, government incentives are essential to improving the availability and accessibility of medications for patients with rare diseases.
Overseas, some have outlined government involvement, which can take a variety of forms:
● Taxes to support companies that produce or research these drugs.
● Granting additional rights and licenses over and above those under regular patent law.
● Subsidies and grants to universities and corporate sponsors for the development of medicinal products (including drugs,
biologics, medical devices, and medicinal foods) for use in rare diseases.
● Creating government-run companies to research and produce these medicines (for an example of this type, see Crown corporation)
The introduction of incentives for rare medicines in foreign countries has resulted in the creation of treatment programs for many rare diseases from scratch, with excellent results. In the United States, fewer than 10 rare disease drugs were on the market before the implementation of the Rare Drug Act of 1983, while by February 2007, 1,697 rare drugs were registered with the FDA, 302 received marketing approval, and about 110 pharmaceutical companies already had FDA marketing approval for rare drugs. While most companies have only one rare drug, about 40% have two or more rare drugs in development. As of April 2007, 447 rare drugs were registered and 34 were on the market in the European Union; 98 rare drugs were on the market in Japan between 1993 and 2004***.
China mentioned rare diseases and new drugs for rare diseases as early as the 1999 edition of the Measures for the Administration of Drug Registration, but so far they have not been clearly defined and the incentive policies adopted have not been obvious. The current Administrative Measures for Drug Registration (2005 edition) also does not provide further incentives for the research and development of rare drugs. In March 2007, the State Food and Drug Administration published a draft of the Administrative Measures for Drug Registration for public comment, mentioning that "new drugs for the treatment of AIDS, new drugs superior to those already on the market for the treatment of malignant tumors, rare diseases and other diseases will be included in the 'green channel' for special approval, and the approval process will be simplified". "However, there is no special definition of rare diseases and medicines for rare diseases. It should be said that China's rare diseases and rare drugs related policies are still relatively lagging behind.
Borrowing from foreign experience in defining rare diseases and incentivizing policies for R&D of rare medicines can be a driving force in defining the concepts of rare diseases and rare medicines in China and in the implementation of policies.