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    Home > Medical News > Medical World News > Rational view of the "Nosina raw sodium" high price to solve the problem of the cost of rare diseases must adapt to the national conditions.

    Rational view of the "Nosina raw sodium" high price to solve the problem of the cost of rare diseases must adapt to the national conditions.

    • Last Update: 2020-08-23
    • Source: Internet
    • Author: User
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    Spinal muscular dystrophy (SMA) treatment "Nosina sodium" injection, because of "700,000 yuan and 41 Australian dollars" "rare" spread has become the focus of attention on the Internet in recent days.
    With the clarification of relevant facts, more and more rational voices, more netizens began to pay attention to the "Nosina sodium" represents the rare disease, orphan medicine problem, which is the whole society rational view, consider the reality of the national conditions to face the dedication of rare diseases, orphan medicine is undoubtedly a good thing.
    The author of this paper is a doctoral tutor in the Department of Pharmacy Management and Clinical Pharmacy of Peking University School of Pharmacy, and Shi Liwen, director of the International Research Center for Pharmaceutical Management of Peking University, said that the difference in domestic and foreign prices of nosina sodium is inaccurate In February 2019, Nosina sodium was approved for listing in China, and the company priced it at 700,000 yuan/branch.
    is not accurate because the Internet is talking about Northoxina's Australian dollar, which is a far cry from domestic prices.
    the official purchase price of Sodium Nosina in Australia is A$110,000, so-called A$41 is the level of personal out-of-the-money, not the actual price of the drug.
    it is understood that the drug is also quite expensive in other countries, such as Japan for 9.32 million yen about 610,000 yuan, the United Kingdom for 75,000 pounds about 680,000 yuan, the United States for 125,000 yuan about 870,000 yuan.
    the price of the drug in China is in the middle of the international price level, considering that the current sales of the drug in China is very limited, enterprises to set this price level is understandable.
    02 countries are facing the high cost of rare disease orphan drugs SMA is a rare disease.
    countries often refer to drugs such as nosinan sodium, which is mainly used for rare diseases, as orphan drugs, and treatments are often expensive.
    sodium is required for six injections per patient per year, at a medical cost of about $4.2 million, and requires lifelong injections.
    Novarte's Solgensma, another treatment for SMA that is said to cure SMA patients under the age of 2, is not yet available in China and is priced at $2.1 million in the U.S. market, nearly 20 times as much as Nosina's sodium.
    , which treats Gosser's disease, costs between $400,000 and $2 million a year depending on the weight.
    the beauty of the treatment of Pompe s disease, the annual cost of treatment is about 2 million yuan.
    treatment of type A haemophilia, the annual cost of about 3 million yuan.
    , which costs about $410,000 a year to treat bursts of sleeping hemoglobin urine, was named the world's most expensive drug when it went on sale in 2016.
    from a medical ethics point of view, the public is of course willing to orphan medicine prices are as low as cold medicine, headache medicine, every patient who needs it can be fairly accessed.
    but objectively, the high price of orphan drugs does have its reasonable factors.
    first of all, the market for orphan drugs for rare diseases is small.
    rare disease is a category scattered in the various disease systems of different diseases, refers to a single disease incidence is low, the total number of cases is small but serious diseases, mostly genetic diseases.
    The criteria for defining rare diseases vary from country to country, with common morbidity rates below a certain percentage (e.g. 5 per 10,000 in the European Union, one in 10,000 in Taiwan) or less than a certain number of people in total (e.g. 50,000 in Japan and 2,000 in Australia).
    SMA is an autosomal recessive genetic disease, and in May 2018 the National Health And Health Commission released the "First Rare Diseases" catalogue to include SMA.
    incidence of newborns is about 1/6000-1/10000.
    , it is estimated that china's mainland each year about 2000 new patients, the total number of patients about 3-5 million.
    low morbidity means low needs, meaning the same scale of research and development costs, in cold medicines and other common diseases of the drug pricing can be fully diluted, in rare disease orphan drugs pricing difficult to share.
    It is generally believed that the cost of developing new drugs is typically $8-1 billion, and the cost of capitalization can be as high as $2 billion, and these costs do not fall because they are orphan drugs, but are often higher in fact - here we do not discuss the failures behind the success stories of research and development and the sunken costs that result from them.
    case, according to a small amount of drug scale to share, high prices and even sky-high prices are a natural result.
    same reason, there is the question of profit.
    the gross profit margin in the price of orphan drugs alone may be a very striking figure, but I'm afraid we can't simply use profit margin indicators to determine whether there is a "windfall".
    the total amount of returns is expected, regardless of what drugs they operate, which is the basis for supporting their research and development innovation and ongoing operations.
    the smaller the market, the higher the profit margin allocated to the price, which is actually a simple economic question.
    followed by a lack of competition in the market for orphan drugs for rare diseases.
    Although the prevalence of rare diseases is low, but a wide variety, has been found up to about 7000 kinds, so the total number of patients with various types of rare diseases is not many, a rough estimate of the total number of patients with various types of rare diseases in China about 20 million people.
    80 per cent of rare diseases are hereditary, 50 per cent in childhood and 30 per cent die before the age of five.
    Because of the small number of patients, the development of therapeutic drugs is difficult, so at present, less than 1% of rare diseases have effective treatment drugs, most rare diseases are in a state of no medicine available, for this reason, people are known as "orphan drugs" for the treatment of rare diseases, there is basically no competitive alternative drugs.
    take SMA as an example, this is a type of spinal frontal motor neuron degeneration caused by muscle weakness, muscular dystrophy disease, type 1 SMA children generally within 6 months of onset, died of respiratory failure within 2 years of age.
    that the disease is an "incurable disease" until a clinically available drug appears.
    In 2016, the FDA approved the launch of the "Nosina Sodium" injection from Yanjian, and clinical data showed that patients had a 100 percent survival rate after 4.8 years of continuous use of Nosina sodium injections and that 96 percent of patients were able to walk with assistance.
    if a child receives medication before symptoms appear, their motor function develops almost the same as in a normal child.
    case, pharmaceutical manufacturers can fully grasp the pricing dominance.
    government in order to encourage pharmaceutical companies to carry out research and development, to achieve "drug availability", it is difficult to kill chickens to collect eggs way to carry out price control.
    03 to solve the problem of rare disease orphan medicine must consider the national conditions of rare disease orphan medicine problem, with the economic and social development and gradually revealed, must also be gradually resolved in the economic and social development.
    , only economic and social development to a certain level can have the conditions to completely solve the problem of rare disease orphan medicine.
    the United States, Japan, the United Kingdom and Taiwan formally introduced the rare disease protection policy, its per capita GDP is more than 20,000 U.S. dollars.
    At present, China's per capita GDP has just reached 10,000 U.S. dollars, urban and rural residents per capita financing level is only 800 yuan, from the point of view of economic affordability is still difficult to provide universal and adequate protection for rare disease drugs.
    Secondly, we must promote the establishment of basic medical security, medical insurance for major diseases, medical assistance, commercial health insurance, social assistance, charitable donations and corporate support and other multi-channel multi-channel financing and security mechanism to accelerate the exploration of China's national conditions in line with the innovative model of medical security for rare diseases.
    work together to share the cost of expensive drugs for orphans with rare diseases, address economic risks and ensure access to medicines.
    is of particular concern that in recent years, our charities have played an increasingly important role in helping the difficult groups of patients with rare diseases and in solving social problems such as poverty and return to poverty among patients with rare diseases.
    As of 2015, there are more than 20 public welfare funds for rare diseases in China, more than 70 social organizations related to rare diseases, and some public welfare funds for rare diseases have been incorporated into the social security mechanism for multi-paying rare diseases of local governments, which is a more realistic method in line with the experience of various countries.
    , encourage support for the research and development of orphan drugs for rare diseases, and explore the establishment of incentive systems for the development of orphan drugs.
    Government departments should work together to introduce a series of preferential support policies to encourage pharmaceutical companies to pay attention to research and development of orphan drugs for rare diseases and solve the problem of "available medicines" by relaxing clinical trials, speeding up approval, extending market exclusive periods, granting patent protection, increasing investment in research and development, reducing related taxes and implementing cross-subsidies.
    this is a more fundamental and fundamental problem than the price problem.
    , because of the huge population base, China encourages the research and development of orphan drugs has certain advantages.
    .
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