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    Home > Medical News > Medical World News > FDA Development and New Drug Development (2): Orphan Drug Act

    FDA Development and New Drug Development (2): Orphan Drug Act

    • Last Update: 2021-02-03
    • Source: Internet
    • Author: User
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    Orphan medicine is not related to orphans who have lost their parents, but is the medicine and treatment provided to groups with rare diseases. The World Health Organization defines rare diseases as diseases with a total population of 0.65 to 1, commonly leukemia, thalassemia, haemophilia, phenylatonuria, albutonism, Fabre disease, Goshe disease, etc. The FDA currently defines rare diseases as those with fewer than 200,000 people worldwide, and only a few hundred people in some rare diseases. The FDA identified 6,000 rare diseases and the European Medicines Agency EMA identified 8,000 rare diseases. The incidence of rare diseases is closely related to ethnicity, genetic background, geography, etc. Since the U.S. Congress passed the Orphan Drugs Act in 1983, the FDA has approved about 350 orphan drugs, and even in the United States, the vast majority of rare diseases remain incurable.
    to develop any drug, input and output are the first consideration. New drugs developed by pharmaceutical companies are used to treat serious diseases with large numbers of diseases, such as diabetes, cancer, and so on, which are rare diseases with low numbers, are expensive to develop and have little incentive to develop because of low demand and low expected benefits.
    After the introduction of the Orphan Drugs Act, the U.S. federal government has great incentives for pharmaceutical companies to develop and support research funding for orphan drugs, give the orphan drug market exclusive for 7 years, enter the fast-track approval channel, clinical trials require fewer people, and some orphan drugs even in the phase 3 clinical phase only dozens of patients have been approved for the market, usually new drug phase 3 clinical requirements often require thousands of patients. The above measures significantly reduce the risk of research and development of orphan drugs.
    1980, Representative Henry Wexman of California began investigating the case of people with rare diseases in Congress. But his work didn't attract much attention until actor Jack Klugman appeared. The younger brother of actor Jack Klugman has died of a rare disease. In his popular TV show Forensic Quincy (Quincy, M.E.) In the middle, Klugman told the public about the treatment needs of patients with rare diseases and the difficulties they face. His program, which attracted the attention of millions of Americans as soon as it aired, played an important role in pushing Congress through the bill. On January 4, 1983, President Ronald Reagan signed the Orphan Drugs Act into law to encourage the development of drugs and treatments for rare diseases. The Orphan Drug Act affects the lives of nearly 30 million Americans, one in 10 of whom is a rare disease. In 2012, the FDA approved a total of 39 new drugs, of which 13, accounting for 33%, in recent years, the FDA approved about 35% of orphan drugs.
    more than 10 million people with all kinds of rare diseases in China. We remain blank in the development of laws and policies related to orphan medicine. The research and development of "orphan medicine" is also in a blank, the treatment of rare patients can only rely on foreign imports, in the face of hundreds of thousands of millions of treatment costs, many rare disease patients can only choose expensive imported drugs or no drugs available. China's CFDA does not have a clear definition of rare diseases and orphan drugs, although there are references to rare diseases in drug registration management practices, which can reduce the number of clinical trial cases but have no practical guidelines.
    2012, the FDA approved Kalydeco, a drug that treats cystic fibrosis. The drug is used to treat patients with rare cystic fibrosis with mutations in the gene of the transfilm regulator. Patients who use the drug spend $300,000 a year and need to take it for life. Orphan drugs are expensive in the United States, costing between $200,000 and $500,000 per patient. The introduction of the Orphan Medicine Act has been a surprising trend: some orphan drugs for the treatment of tumors and blood disease sub-types have been successfully developed. The top 10 best-selling orphan drugs are mainly used to treat diseases such as cancer and blood diseases that do not appear to be rare. For example, five of the eight orphan drugs approved in 2013 were subtypes for cancer treatment, and six of the 13 orphan drugs approved in 2012 were subtypes for cancer treatment.
    Genzyme, which was paid $20 billion by Sanofi in 2011, is a company that specializes in the development and production of orphan drugs. There are a variety of orphan drugs for rare diseases in Jianzan's product line, the most famous of which is the orphan drug Ceredase/Cerezyme, which treats Goshe's disease, which is Kenzan's most profitable drug, accounting for 30 percent of its annual sales and $1 billion in annual revenue. Guacher Desise is a genetic disease caused by an enzyme defect in lysoszyme, in which glucose cerebrosine accumulates in the mesh endothoste cells and lipids are deposited in organs and tissues, affecting bone marrow, liver, spleen, bones and nervous system. Dr. Pitt of France first reported the disease in 1882. In 1964, scientists discovered that the amassing of glucosin glycosides was caused by a lack of β-glucosinase-glycoglycerase (GBA), which provided a theoretical basis for the diagnosis and treatment of Goshe disease. It is estimated that the incidence of Goshe disease in China is between 200,000 and 500,000.
    the drug went on sale in China at the end of 2008. Initially Ceredase was made from the human placenta, so the drug cost about $150,000 a year to "water up." In 1994 Ceredase introduced an upgraded version of Cerezyme. Cerezyme is a biopharmaceus produced by genetic engineering using hamster cells, but the average price increased to $200,000 a year after the upgrade. Cerezyme's success has also been controversial, with Ernest Beutler, an expert in goshey disease treatment at the Scripps Institute, noting that the health care system spends more than $200,000 a year on each Goshe disease patient because most patients need only a quarter of the standard dose of the drug. At present, the dose of this drug is much higher than the dose it actually needs, which is an improper profiteering behavior of Jianzan. Other experts point out that Genzyme's development costs on Cerezyme are very low because the early development of the drug was done by the National Institutes of Health (NIH), which licensed the production of the drug to Genzyme.
    because the face of orphan drugs patient group is very small, Europe, the United States, Japan and other governments have different degrees of preferential support policies. Orphan drugs are mostly caused by single-gene mutations, and the success rate of research and development for these targets is high. And drug regulators will reduce the number of clinical trials of orphan drugs, speeding up the time to market. In the 10 years from 2000 to 2010, the compound annual growth rate of orphan drugs was 26%, higher than 20% of that of ordinary drugs, and global sales of orphan drugs are now over $50 billion. (Biological Exploration)
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