01, a sky-high-priced medicine worth 700,000 yuan per shot, and brushed all 11 bank cards
Du Jia's second son Mili was only a few months old, and SMA was found to be found, that is, spinal muscular atrophy.
Du Jia remembers it very clearly. On the day of the diagnosis, she and her husband were carrying 11 bank cards and brushed more than 690,000 yuan, just for a shot of Nosina Senna injection . These 11 bank cards include the savings of the couple for many years, as well as the pensions of both parents and the money borrowed by relatives and friends...
is the world's first SMA precision targeted therapy drug. Since it was approved for domestic market in 2019, Nosinar Injection has set a new record for Chinese medicine at a price of 699,700 yuan per shot.
02. The sky-high price of medicine has dropped to 33,000, and the patient: Lucky!
What is the experience of reducing the sky-high-priced medicine from 700,000 yuan to 30,000 yuan?
"I am so lucky to be able to use this medicine on the first day of the New Year!" Mr. Du, a 29-year-old patient, experienced the dreamlike reality personally. At the same time, Mr. Du is full of longing for the future and hopes that more patients, especially children, can recover from their health in the future.
Starting from January 1, 2022, Nosinaren sodium injection has been officially included in the medical insurance drug catalog. This is undoubtedly great news for SMA patients.
From the absence of medicine, to the availability of medicine, to the enjoyment of medical insurance policies today, the medical burden of SMA has also been greatly reduced.
1. It is obviously a "life-saving medicine", why is it so expensive?
Whether it is a life-saving medicine of 700,000 yuan or an anti-cancer medicine of 1.2 million yuan, they can save lives, but the price is extremely high. Why is this?
In fact, the development of a new drug is not easy, and it requires going through a series of complex processes such as confirming the target of the drug action - determining the structure-activity relationship of the pilot compound - screening of active compounds - selection of candidate drugs. After the new drug of
is developed, also needs to go through preclinical research and clinical research, and finally apply for new drug , and can be officially launched after obtaining approval from the drug supervision department.
Among them, the pricing of drugs is affected by a variety of factors, such as R&D cost, market capacity, and profit recovery time , etc.
. The reason why the price of nucine sodium is so expensive is mainly because of the following two points:
1. There are few patients and high costs
SMA is an autosomal recessive disease caused by gene defects. It is relatively rare. The incidence rate of is around 1/10,000 .
Therefore, due to the relatively small number of patients, there are relatively few pharmaceutical companies willing to invest in research and development, which has affected the progress of drug research and development to a certain extent. In addition, the R&D costs are relatively high, and pharmaceutical companies will also have relatively high pricing in consideration of benefits.
2, high failure rate
In addition, during the research and development process, the failure rate of drugs is also relatively high. The relevant report of
shows that from 2011 to 2020, there were 9,704 drug clinical development projects, and it took an average of 10.5 years from Phase 1 clinical trial to the final FDA approval for marketing, and rare drugs take longer.
In addition, the clinical trial of may also face problems such as small sample size, large heterogeneity, and difficulty in recruiting . If any stage has not been completed, it will be difficult to continue to advance in the next step. Although
Nocinarenin sodium injection cannot cure SMA, clinical experimental results show that can significantly improve the patient's muscle strength and obtain a better prognosis, while not having serious side effects . Therefore, for SMA patients, nocinarin sodium injection is equivalent to a "life-saving drug".
2. What is SMA? The incidence rate is about one in ten thousand
At present, there are about 30,000 SMA patients in my country. The 2020 "Expert Consensus on Genetic Diagnosis of Spinal Muscular Atrophy" shows that the incidence of SMA is about 1/10,000 and the carrying rate of the population is about 1/50.
Although is a rare disease , SMA is not far from us. Research data shows that 1 out of every 35-50 people carry the SMA gene . If treatment is not received, 80% of SMA-I children live less than 2 years old on average. If you unfortunately get an SMA, what will your child behave?
1. Crying weakly, sucking and coughing, continuous fever, pneumonia cannot be cured for a long time;
2. When inhaling, the outline of the wall clock appears on the chest;
3. Can't sit firmly or stand on its own;
4. Limbs are loose, have poor strength, and lack of spontaneous activity.
reminds you that the earlier SMA occurs, it means that the worse the condition and the higher the risk of death. The older the onset, the less the condition. A few patients will develop the disease in adolescence or adulthood, and their life expectancy is similar to that of normal people, but the possibility of disability is relatively high.
3. The survey found that SMA patients have a heavy burden of disease
Poverty due to illness is the current situation of many ordinary Chinese families. The 2022 "SMA Quality of Life Research in China" shows that more than 80% of respondents are receiving treatment, and 14% of respondents have never been treated. The reason for not being treated is that the treatment costs are expensive and cannot afford .
In addition, the investigation also found that SMA patients have a heavier disease burden. In the past year, the median household income of all patients was , , , 84,000 yuan.
Of course, since the inclusion of Nosinaren sodium injection into medical insurance, the number of users has been increasing, and the medical expenses borne by patients have also been reduced. However, each family still has to pay more than 5,000 yuan in medical expenses per month, accounting for more than 76% of the family's monthly income .
In fact, SMA can be prevented. Although it is a recessive hereditary disease and has a high carrier rate of pathogenic genes, it is not completely impossible to detect in advance.
As long as prenatal examination is done and genetic intervention is carried out if necessary, the disease can be accurately diagnosed and treated as soon as possible to achieve the purpose of improving symptoms and improving quality of life.
Every year, millions of people in my country die of cancer, and countless patients are looking forward to the arrival of new drugs. The good news is that at present, new drug trials are underway for lung cancer, gastric cancer , liver cancer , breast cancer and other cancers. Patients are taking medication for free after enrollment. However, due to the limited quota, if necessary, can click on the author's avatar and send the message "clinical trial" four words to obtain the registration method.
Nocinarenin sodium injection is included in medical insurance, which is not only a great encouragement for SMA patients, but also brings hope for other rare diseases. I believe that with the advancement of medical technology, more and more rare drugs will be developed and the prices will become more and more reasonable, reducing the medical burden on patients.
References
[1] "700,000 per needle! In order to keep my son alive, my husband and I brushed all 11 bank cards in tears. Tencent Medical Document. 2021-03-02
[2] "From 700,000 per shot to 33,000, how can high-value rare disease drugs be included in medical insurance? 》.China News Weekly.2021-12-07
[3] "It is the "number one genetic disease killer" for babies under 2 years old. Parents must pay attention to these symptoms when children experience! 》.Health Times .2022-06-24
Reprinting of
is prohibited without authorization from the author-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-