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    Home > Medical News > Latest Medical News > Focus on the "sky price" rare disease medicine: a drug 700,000 yuan why is so expensive?

    Focus on the "sky price" rare disease medicine: a drug 700,000 yuan why is so expensive?

    • Last Update: 2020-08-13
    • Source: Internet
    • Author: User
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    Pharmaceutical network August 7th, the network spread "the treatment of rare disease 'spinal muscular dystrophy' special effects drugs need '700,000 yuan a' sky price, and domestic prices are much higher than abroad", causing social concern. what is
    "spinal muscular dystrophy"? What is nosi's sodium injection? Why are drugs so expensive? Is the drug really selling at a wide price in the domestic and foreign markets? Is the price of medication expected to fall for domestic patients? Xinhua News Agency reporters launched an investigation.
    Focus 1: What is spinal muscular dystrophy? According to The National Children's Medical Center, Beijing Children's Hospital Neurology Director Dr. Lu Junlan, spinal muscular dystrophy (SMA) is a fatal disease in children, usually 80% of patients with type I can not live beyond 2 years; Wu Liwen, deputy director of neurology at Hunan Children's Hospital in
    , said the disease, characterized by degeneration and loss of motor neurons in the spinal cord and lower brain stem, is a rare disease of hereditary neuromuscular disease.
    experts said that the incidence of SMA in newborns is about 1/6000 to 1/10000, there are currently about 30,000 SMA patients, but the rate of pathogenic gene carrying in newborns is about 1/50, the risk is very high, so large-scale prenatal screening is very important.
    Focus II: What is nosinsssof injections? Experts said that the nosina sodium injection is an antisense nucleotide treatment drug for infants, children and adults with 5q type spinal muscular dystrophy, and the first imported drug approved for the treatment of spinal muscular dystrophy in China.
    doctors give the drug to the patient by injecting the drug into the body through the waist-piercing.
    Wu Liwen told reporters that children need to take medication as soon as possible after diagnosis: 6 injections in the first year, 4 injections in the first 2 months;
    Wu Liwen said that some children in the injecting drug use, the ability to exercise to a certain extent to recover.
    , a 2-year-old, 11-month-old child in Hunan, nono began injecting the drug in May this year and has been injected four times. After
    treatment, Nono's motor ability was better restored. "In terms of treatment, Nosina sodium injections are really useful, but they don't completely reverse the condition, "
    .
    ," Lu Junlan warned.
    Focus 3: Why are drugs so expensive? Reporters learned that Nosina sodium injection in 2019 was approved for import and listing, is not currently included in health insurance, by the patient's own expenses.
    in China, the price per unit of the drug is 6997 million yuan.
    under the new drug administration law in 2019, drug prices are no longer set by the government, but are dominated by market regulation, which will play a role through centralized bidding and procurement of health insurance. Song Hualin, vice president of the School of Law at Nankai University in
    and an expert in drug law, said that the number of rare patients is small and has clinical uncertainty, so drug development costs are high and drugs are expensive.
    individual pharmaceutical companies often get pricing advantage status because of technological leadership, which does not necessarily violate China's anti-monopoly law or drug regulatory laws and regulations.
    he believes that the deep reason that rare diseases into medical insurance has repeatedly aroused concern is that the current type of medical insurance can cover the limited types of rare diseases, and China's current commercial medical insurance still needs to be perfected, rare patients' medical needs are difficult to fully meet.
    Focus IV: Is the drug selling at a wide price in both domestic and foreign markets? Internet rumors that the drug "sells for only $41 in Australia, about 280 yuan" "almost free in Japan", and said that "our hospital windfall has reached such an appalling level" and so on.
    investigation found that according to the Australian Drug Welfare Scheme (PBS) network, the average patient pays $41 per unit, but the government procurement price of the drug is A$110,000 per unit.
    also understand that Japan recently included related drugs in the scope of medical care, patients only in accordance with the country's overall health care policy to bear a relatively low cost.
    but the drug itself is still a high-priced drug.
    also found that the global inclusion of the drug in public health insurance limited countries.
    in the United States, the drug sells for $125,000 each.
    in Canada, a local resident told reporters that after the company bought commercial insurance for him, it would also cost more than $5,700 per unit at its own expense.
    it is understood that most European countries do not include it in the scope of public health insurance.
    Focus 5: Is the price of medication expected to fall for domestic patients? Experts said that because of the low incidence of rare diseases, the use of drugs is very small, expensive, one into the volume of procurement to achieve price reduction is not small.
    how to reduce the pressure on the cost of medical care for such patients is still a challenge.
    reporter learned that in 2019, the China Primary Health Care Foundation has launched the "Spinal Newborn-Spinal Muscular Dystrophy SMA Patient Assistance Project" to provide assistance drugs to patients who meet the eligibility criteria for the project.
    family members of children can apply to participate in the assistance program, the first year will be about 1.4 million yuan price injection of six drugs at the original price of 700,000 yuan each.
    Lu Junlan, Wu Liwen and other experts said that they hope that the relevant departments can study ways to effectively reduce the medical burden of the patient through comprehensive means such as medical insurance.
    improving the treatment and survival of patients with rare diseases requires the joint efforts of the state, social organizations, medical institutions and patients.
    Song Hualin believes that for rare diseases with very low incidence and very high cost of diagnosis and treatment, multi-channel assistance should be taken, such as considering medical assistance and special assistance, and establishing special funds.
    he suggested that the provinces can determine the province's inclusion in the scope of medical protection of rare diseases, according to their own level of economic and social development, the balance of medical insurance funds and the diagnosis and treatment of rare diseases.
    Ms. Yang, who lives and works in Japan, told reporters that some local residents and experts have expressed concern and dissatisfaction with the inclusion of the highly priced drug in health care, saying it would threaten the balance and safety of security funds and crowd out other more acute and urgent diseases into the security system. More than
    experts believe that high-priced rare disease drugs have their particularity, whether it is through centralized procurement negotiations or the inclusion of health insurance, to promote its price reduction has practical difficulties.
    The relevant person in charge of the health department of Hunan Province said that some rare diseases have been successfully solved before, because China's large population base, some of the rare disease strains are not "rare", the health insurance sector can use the price-for-price negotiation means to promote enterprises to reduce prices, and the current security system to form a common interest.
    but in the long run, there are many rare diseases of new drugs clinical total is small, it is difficult to bargain.
    therefore, to really solve the problem of rare diseases drug difficulties, expensive drugs, need to actively develop commercial insurance, comprehensive use of charity, relief and other social resources for patients to reduce the burden, the relevant departments should also speed up the research and development of generic drugs and consistency evaluation, fundamentally improve the availability of related drugs.
    .
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