"A drug 700,000 yuan" "foreign cheap domestic expensive"? Focus on "sky-high" rare disease drugs.

A few days ago, the network spread "the treatment of rare disease 'spinal muscular dystrophy' special effects drugs need '700,000 yuan a' sky price, and the domestic price is much higher than abroad", causing social concern.
disease of spinal muscular dystrophy? What is the drug for nosina raw sodium injections? Why is medicine so expensive? Is the drug really selling at a very different price in both domestic and foreign markets? Is the price of drug use expected to fall in domestic patients? The reporter launched an investigation into this.
focus one: What is spinal muscular dystrophy? Spinal muscular dystrophy (SMA) is a fatal disease in children, with 80% of type I patients not able to live past 2 years of age, according to Dr. Lu Junlan, director of neurology at the National Children's Medical Center and Beijing Children's Hospital.
Wu Liwen, deputy director of neurology at Hunan Children's Hospital in Hunan Province, said the disease, characterized by the degeneration and loss of motor neurons in the spinal cord and lower brain dry, is a rare disease of hereditary neuromuscular disease.
experts said that china's SMA incidence in newborns is about 1/6000 to 1/10000, there are currently about 30,000 SMA patients, but the rate of gene carrying in newborns is about 1/50, the risk is very high, so large-scale prenatal screening is very important.
focus two: What is the drug for nosina sodium injections? Experts said that Nosina sodium injection is infants, children and adults 5q spinal muscular dystrophy antisympathetic nucleotide treatment drugs, but also the first approved treatment of spinal muscular dystrophy imported drugs.
the patient by injecting the drug into the body through a waist-worn injection.
Wu Liwen told reporters that children need to take medication as early as possible after diagnosis: the first year need to inject 6 drugs, the first 2 months need to inject 4 injections;
Wu Liwen said that some children in the injection of drugs, exercise ability has a certain degree of recovery.
2-year-old and 11-month-old child in Hunan province began injecting the drug in May this year and has now been injected four times.
treatment, Nono's athletic ability was better restored.
" is therapeutically useful, but it doesn't completely reverse the condition.
," Lu Junlan warned.
: Why are drugs so expensive? Reporters learned that Nosina raw sodium injections in 2019 was approved for import and market, is not currently included in health insurance, by the patient at their own expense.
, the price per unit of the drug is 699.7 million yuan.
under the newly revised Drug Administration Act of 2019, drug prices are no longer set by the government, but are market-regulated and play a role through centralized bidding and procurement of health insurance.
Hualin, associate dean of Nanke University School of Law and an expert in drug law, said the small number of patients with rare diseases has clinical uncertainty, so drug research and development costs are high and drugs are expensive.
pharmaceutical companies often get pricing advantage 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 why rare diseases are often concerned about medical insurance is that the current type of medical insurance can cover a limited variety of rare diseases, and China's current commercial medical insurance still needs to be perfected, rare disease patients' medical needs are difficult to fully meet.
focus four: is the drug sold at a wide price in domestic and foreign markets? Internet rumors that the drug "sold in Australia for only $41, about 280 yuan" "almost free in Japan", and said that "our hospital profits have reached such an appalling level" and so on.
the Australian Drug Benefits Program (PBS) network, the average patient pays $41 per unit for medication, but the government-purchased price for the drug is A$110,000 per unit, according to the Australian Drug Benefits Program (PBS).
reporters understand that Japan recently included related drugs in the scope of health care, patients only need to bear the country's overall health care policy relatively low costs.
but the drug itself is still a high-priced drug.
reporters also found that the world's inclusion of the drug in public health insurance is limited.
U.S., the drug sells for $125,000 each.
, a local resident told reporters that after the company bought commercial insurance for it, it was not cheap to use the drug at its own expense of more than $5,700 per unit.
that most European countries do not include it in publicly-paying health insurance.
5: Is the price of drug use expected to fall in domestic patients? Experts said that because of the low incidence of rare diseases, the use of drugs is very small, expensive, one by one into the purchase of belt volume to achieve price reduction is not small.
how to reduce the pressure on the medical costs of such patients is still a challenge.
reporters learned that in 2019, China Primary Health Care Foundation launched the "Chiroprotein-Spinal Muscular Dystrophy SMA Patient Assistance Program" to provide assistance to patients eligible for the project application.
families of children can apply to participate in aid projects, the first year can be about 1.4 million yuan price injection of six original price of 700,000 yuan each drug.
, Wu Liwen and other experts expressed the hope that the relevant departments can study ways to effectively reduce the medical burden of patients with the disease through comprehensive means such as medical insurance.
improving the treatment and survival of patients with rare diseases requires the joint efforts of states, 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, the establishment of special funds.
he suggested that provinces could determine the types of rare diseases covered by medical care in the province based on their level of economic and social development, the balance of health 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 expensive drug in health care, saying it would threaten the balance and safety of funding and crowd out opportunities for other diseases with more widespread and pressing needs to be included in the system. more than
experts believe that high-priced rare disease drugs have their particularity, whether with centralized procurement negotiations or into the health insurance approach, to promote its price reduction have practical difficulties.
Hunan Province health department officials said that some rare disease drug problems have been successfully resolved, because China's population base is large, some kinds of rare disease patients are not "rare", medical insurance departments can use the volume of price-for-price negotiations to promote enterprise price reduction, and the current security system to form a common interest.
, however, in the long run, there are many new drugs for rare diseases that have a small clinical total and are difficult to bargain with.
Therefore, to really solve the problem of difficult and expensive drug use of rare diseases, we need to actively develop commercial insurance, comprehensive use of charitable, relief and other social resources to reduce the burden on patients, the relevant departments should also speed up the development of generic drugs and consistent evaluation, fundamentally improve the access to related drugs."
source: Xinhua News Agency, !-- the end of the content display -- the !-- determine whether to log in to the end.