7 new drugs for rare diseases included in medical insurance
Press the deceleration button for the passing life
In the 2022 national medical insurance negotiations, "soul bargaining" was staged again.
The listed price of Lisporan Oral Solution Powder (60 mg per bottle) added to the "Guotan" this time is 63,800 yuan. After several rounds of "bargaining" by the Medical Insurance Bureau, the price was reduced to 3,780 yuan, a drop of about 94.08%. .
"Roche's risperan oral solution powder (trade name: Amanxin) has entered the national medical insurance, and the transaction price is 3,780 yuan per bottle." I heard this news not long ago. The rare disease spinal muscular atrophy (SMA) Patient Wang Yan felt excited and happy like many other patients.
What makes Wang Yan and other SMA patients sigh is not only the lower drug price, but also more medication choices.
Previously, the drug Nosinagen Sodium Injection, which was also used to treat SMA, entered the medical insurance catalog at a price of about 33,000 yuan/needle after the 2021 "National Talks" from the initial market price of 700,000 yuan/needle.
Because Nosinagen Sodium Injection requires intrathecal administration, and many SMA patients suffer from severe scoliosis, so it will be more painful when using Nosinagen Sodium, and the administration is more difficult, and it is newly included in the national medical insurance drug Catalog's Risperan solves these pain points.
On January 18, the list released by the National Medical Insurance Bureau showed that 7 drugs for rare diseases were added to the 2022 National Medical Insurance Catalogue, involving multiple sclerosis, pulmonary hypertension, hereditary angioedema, amyotrophic lateral sclerosis, spinal muscular Atrophy and neuromyelitis optica spectrum disorders.
Previously, 45 drugs for rare diseases had been included in the medical insurance catalogue, covering 26 rare diseases, which greatly reduced the burden on patients with rare diseases.
The "Research Report on the Definition of Rare Diseases in China 2021" pointed out that diseases with a newborn incidence rate of less than 1/10,000, a prevalence rate of less than 1/10,000, and a patient population of less than 140,000 are defined as rare diseases.
According to previous data released by the National Health and Medical Commission and other institutions, there are more than 20 million patients with various rare diseases in my country, and more than 200,000 new patients are newly diagnosed every year.
"Soul Bargaining" is staged again, and "medicine is guaranteed" for rare diseases
Hearing the news that SMA treatment drugs were included in the national medical insurance again, to Wang Yan, "it was like a ray of sunshine appeared in the dark, and someone pressed the slowdown button on my rapidly passing life."
When Wang Yan was 1 year old, he couldn't walk and couldn't stand still. Later, he went to the Affiliated Hospital of Fuzhou Medical University and Capital Children's Hospital for examinations before being diagnosed with the rare disease of SMA.
In 2022, the drug Nocinogen Sodium Injection for the treatment of SMA will enter the national medical insurance list. Wang Yan hurriedly signed up for the drug and injected 5 injections of Nocinogen Sodium. Although his physical strength still did not improve after taking the medicine, his physical fitness and weight have improved. .
The doctor told him that this rare disease drug needs 6 injections in the first year, and only 3 injections per year thereafter.
The "Study on the Quality of Life of SMA Patients in China" previously released by the Meier SMA Care Center and other institutions showed that since Nosinagen Sodium entered the National Medical Insurance List, the number of families of patients who started using the drug has increased significantly.
76.4% of the patients who were using Noxinargen Sodium Injection at the time of the survey started taking the drug after being covered by medical insurance.
Overall, no matter whether it is using Nosinagen Sodium or Risparam, compared with patients who do not take drugs, the autonomy of daily activities is significantly improved, and the average score of quality of life is also higher in patients who take drugs, especially those who take drugs for a long time. higher.
The drug burden of most patients' families is also greatly reduced.
In the 2022 national medical insurance negotiations, "soul bargaining" was staged again.
The listed price of Lisporan Oral Solution Powder (60 mg per bottle) added to the "Guotan" this time is 63,800 yuan. After several rounds of "bargaining" by the Medical Insurance Bureau, the price was reduced to 3,780 yuan, a drop of about 94.08%. .
"In the past, the 700,000 yuan/needle (patient) could not be used at all, and now it can be used, and there are new options. This in itself is actually a manifestation of the improvement and progress of the entire medical insurance system." Information from Beijing Pain Challenge Public Welfare Foundation Guo Jinchuan, research director, said that in recent years, more and more patients with rare diseases have ushered in the hope of "care for their diseases, medicine for their diseases, and protection for their medicines".
In fact, pharmaceutical companies that develop drugs for rare diseases also realize "small profits but quick turnover" by being included in medical insurance.
Take Nosinagen Sodium for the treatment of SMA as an example. According to data from China Science and Technology CHIS Kaisi, after entering the 2021 version of the medical insurance drug list, the sales of this drug in the national level hospitals in the first half of 2022 will be 379 million yuan, an increase of 5% year-on-year. times, which is 2.5 times the annual sales in 2021.
Both supply and demand are working hard to make rare diseases "good medicines are easy to sell"
In the 2022 version of the national medical insurance catalog released before the Spring Festival, a total of 19 rare disease drugs entered the preliminary review, and only 7 rare disease drugs were successfully included in the catalog.
Compared with the overall negotiation success rate of 82.3% in the "National Discussion" drugs, the uncertainty of the "National Discussion" of rare disease drugs is still relatively high.
According to the "negotiation rules" previously circulated in the industry, only drugs with an annual treatment cost of less than 500,000 yuan can be eligible for negotiation, and only drugs with an annual treatment cost of no more than 300,000 yuan can finally enter the catalog.
This means that there are still many high-value rare disease drugs that are difficult to enter the medical insurance.
The "China Rare Disease Industry Trend Observation Report" pointed out that among the rare diseases included in the first batch of catalogs in my country, 29 drugs for 14 rare diseases were not covered by medical insurance.
In the eyes of industry insiders, the success rate of the "national talk" of rare disease drugs is not high because the main principle of the national medical insurance is to "protect the basics", and the high-value rare disease drugs that cost hundreds of thousands or millions of dollars occupy the medical insurance fund higher share.
The National Medical Insurance Administration also stated in its reply to the National People’s Congress’s proposal that after 4 consecutive years of access negotiations on exclusive drugs, drugs for rare diseases such as Nosinagen Sodium, Miglustat, and Deuterium Tetrabenazine have been included in the catalog after price reductions. The average reduction rate exceeds 50%, which greatly reduces the burden of medication for patients with rare diseases.
However, particularly expensive rare disease drugs cannot be included in medical insurance for the time being, and local governments are not supported to independently explore the protection mechanism for high-value rare disease drugs.
Guaranteeing drugs for rare diseases cannot rely solely on medical insurance negotiations, but also requires the intervention of multiple forces to jointly build a "1+X" (medical insurance + others) security system.
Liao Zangyi, an associate professor at the School of Politics and Public Administration, China University of Political Science and Law, said that policies such as "Benefiting the People's Insurance" that are generally introduced in various places basically include drugs for rare diseases, and the reimbursement rate for non-previous indications can be as high as 80%. The reimbursement ratio of basic medical insurance remains the same, which is also a better implementation path for "X".
Liao Zangyi believes that from the market point of view, due to the small number of patients with rare diseases, in the process of drug registration and approval, encouraging policies such as reducing the number of patients enrolled and being approved in the second phase of a single-arm (single-group clinical trial) can be used to speed up drug development. Approval for the listing of the company effectively solves policy obstacles for enterprises and ensures the rapid benefit of patients after listing.
In recent years, my country has further optimized the market access, review and approval of rare disease drugs to speed up market supply.
For example, in April 2021, Dimethyl Fumarate Enteric-Coated Capsules (trade name: Tefida), a drug for the treatment of the rare disease multiple sclerosis (MS), was granted priority review by the State Drug Administration as an urgently needed overseas new drug. The approval process is approved to enter China.
Fampridine Sustained-release Tablets, also produced by Biogen, are used to improve the walking ability of adult patients with multiple sclerosis (MS). It took half a year to create the "China Speed" in the field of rare disease drugs.
In May 2022, the State Drug Administration issued the "Regulations for the Implementation of the Drug Administration Law of the People's Republic of China (Draft for Comments)", giving a special policy of "market exclusivity period" for rare disease drugs, and stipulating that new drugs for rare diseases approved for marketing Up to 7 years of market exclusivity can be obtained.
In November 2022, four departments including the Ministry of Finance issued the "Announcement on the Release of the Third Batch of Anticancer Drugs and Rare Disease Drugs List Applicable to Value-Added Tax Policy". Tax cuts for retail sales.
These policies are conveying the signal that "good medicines will not worry about selling" and encouraging the enthusiasm of original research pharmaceutical companies.
Make drugs for rare diseases more accessible in the "last mile"
Although various policies targeting rare disease populations are working, there is still the problem of "drugs are included in medical insurance, but not in hospitals" in actual implementation.
At the end of 2021, agalsidase-α, a special drug for the treatment of Fabry disease, entered the national medical insurance drug list through medical insurance negotiations. The price of a single injection dropped from 12,000 yuan to 3,100 yuan, but there are still many patients who cannot use it for various reasons This medicine has been included in the medical insurance.
A similar situation existed in previous years.
In 2017, recombinant human interferon β-1b (Betalon) for injection, a drug for the treatment of multiple sclerosis, was included in the medical insurance. After negotiation, the price was reduced by 28.9%, and the medical insurance payment standard was 590 yuan (0.3mg/bottle).
However, after entering the medical insurance, Betalon's sales performance was not satisfactory, and it failed to renew the contract in the 2019 medical insurance negotiations.
"The reason for this drug was that the hospital couldn't prescribe it at all, and the patients couldn't use it." Guo Jinchuan believes that there are two ways to solve the problem of "country talk about medicine" landing. One is "dual channels", that is, from the hospital 1, pharmacy two channels to solve the patient's medication problem; the second is from the "proportion of drugs" (that is, the proportion of drugs to the total medical expenses), the average cost per time (the average amount of expenses incurred by all patients in a medical institution within a certain period of time) ), medical institutions' drug list restrictions and other assessment mechanisms.
In 2021, the National Medical Insurance Administration and the National Health and Medical Commission formulated and introduced a "dual-channel" management policy. On the basis of supporting designated medical institutions to prepare drugs, they will guide designated retail pharmacies to rationally allocate drugs in the medical insurance catalogue, especially For new drugs that have been negotiated and admitted, a unified reimbursement policy with designated medical institutions shall be implemented.
According to data from the National Health and Medical Commission, as of December 2022, about 210,000 key medical institutions across the country have been equipped with 275 negotiated drugs in the National Medical Insurance Catalog during the agreement period, including 54,000 designated medical institutions and designated retail pharmacies. 155,000.
In addition, in September 2022, a reply letter from the National Medical Insurance Administration to the Central Committee of the Peasants and Labor Party mentioned that the negotiated drugs for rational use will be listed separately, and will not be included in the scope of assessment indicators that affect their implementation, such as the proportion of drugs in medical institutions and the average cost per time. , medical institutions shall not use the control of the total amount of medical insurance, the limitation of the number of medical institutions' drug lists, and the proportion of drugs to affect the negotiation of drug allocation and use.
"These are equivalent to having a clear policy statement for equipping hospitals with 'national medicines', and there will be a certain degree of flexibility for hospitals." Guo Jinchuan said that there may be time differences in the specific implementation, so rare diseases are also needed Patients actively express their need for medication.
Liao Zangyi pointed out that at present, rare diseases can only be detected in authoritative tertiary hospitals in first-tier cities such as Beijing, Shanghai, Guangzhou and Shenzhen, and a large number of grassroots hospitals lack corresponding diagnosis and treatment capabilities.
Therefore, he suggested continuing to strengthen drug allocation, such as ensuring that each prefecture-level city has at least one pharmacy supplying rare disease drugs. For hospitals equipped with rare disease drugs, relevant enterprises and local governments can provide appropriate subsidies, so as to promote rare disease drugs. More accessible in the "last mile".
(At the request of the interviewee, Wang Yan is a pseudonym)
China Youth Daily China Youth Daily reporter Wang Lin trainee reporter Yuan Jie Wang Xueer Source: China Youth Daily