Solving the drug problem for patients with rare diseases: more than 40 kinds have been included in the national medical insurance drug list

In order to solve the problem of medication in patients with rare diseases, the relevant departments are in action. A reporter from the Beijing Youth Daily learned from the 2021 China Rare Disease Conference held in Beijing on the 18th that as of now, more than 60 rare disease drugs have been approved for marketing in China, of which more than 40 have been included in the national medical insurance drug catalog, involving 25 Kind of disease. Since its establishment in 2018, the National Medical Security Administration has dynamically adjusted the medical insurance drug catalog once a year for 4 consecutive years, and a total of 507 new drugs and good drugs have been included in the catalog.

After the implementation of the dynamic adjustment mechanism, the list of rare disease drugs included the most
According to Li Tao, deputy director of the National Medical Security Administration, a total of seven rare disease drugs were successfully negotiated in 2021, with an average price drop of 65%.
A reporter from the Beijing Youth Daily learned that there are as many as seven rare disease drugs in the new drugs this year. This is also the most rare disease drugs included in the catalog adjustment since the implementation of the dynamic adjustment mechanism of the national medical insurance catalog in 2018. It includes ampridine sustained-release tablets for the treatment of multiple sclerosis, agalase α injection concentrated solution for the treatment of bray disease, etabate acetate injection for the treatment of hereditary angioedema, nocinate sodium injection for the treatment of spinal muscular atrophy, human blood clotting silver IX for the treatment of hemophilia, chlorobenzazole soft capsule for the treatment of transthyretin amyloidosis cardiomyopathy, and iloeuzumab injection for the treatment of homozygote for the treatment of familosis.
Among them, there are two kinds of rare disease drugs previously known as "sky-high price", to achieve high-priced rare disease drugs into the medical insurance "0 breakthrough".
Fabre disease is a rare X-linked hereditary lysosomal storage disease. In April this year, the concentrated solution of Agalase α for injection by Takeda Pharmaceutical, a Japanese pharmaceutical company, was approved for marketing in China as a specific drug for patients with Fabre disease. However, because it is not included in medical insurance, the cost of treatment for one year is as high as millions of yuan. After the inclusion of health insurance, will greatly reduce the burden of patients. Another rare disease drug of Takeda Pharmaceuticals was newly included in the medical insurance in this negotiation, that is, the special drug Atibant Acetate Injection used to treat the acute attack of hereditary angioedema, which was only approved to be listed in China in April this year. Without this specific drug, Chinese patients can only rely on infusion of freeze-dried fresh plasma to subside edema.
Bojian Bio's two rare disease drugs Noxinasan sodium injection and ammonia pyridine sustained-release tablets successfully passed the health insurance negotiations, was included in the national health insurance catalog. Noxinasan sodium injection is the first drug used to treat spinal muscular atrophy in China. It was approved for listing in China in 2019, and the market price was once as high as 700000 yuan/needle. The initial dose of noxinacin sodium injection is 4 injections within 2 months, and the subsequent maintenance dose is 1 injection every 4 months, which requires lifelong medication. Even if only the initial dose is counted, 4 injections will cost 2.8 million yuan, which is a price that most patients' families cannot afford.
A reporter from the Beijing Youth Daily learned that the annual cost of drugs that have completed medical insurance negotiations is strictly limited to less than 300000 yuan. At the scene of the negotiation of Nuoxinasheng sodium injection, the health insurance negotiator's sentence "every small group should not be abandoned" touched countless netizens. After several rounds of negotiation, the drug was finally officially included in the medical insurance at a price of more than 33000 yuan. The company's drug Ampridine Sustained-release Tablets, which is used to improve walking dysfunction in patients with multiple sclerosis, was just approved for the market in May this year, and entered the medical insurance negotiations within half a year after the market.
Gradual freezing disease, hemophilia and other specific drugs have been put into medical insurance.
Since its establishment in 2018, the National Medical Security Administration has dynamically adjusted the list of medical insurance drugs once a year, and drugs for rare diseases are also included. At the same time, through the negotiation of access to rare disease drugs, the price of rare disease drugs has been greatly reduced.
In recent years, the adjustment of the medical insurance catalogue has also focused on the use of drugs for rare diseases. The number of drugs included in the catalogue has increased and the types of rare diseases covered have expanded. Compared with the 2009 version of the Medicare drug catalog, the number of drugs for rare diseases in the 2020 version of the catalog has increased by 105, and the number of rare diseases covered has increased by 69%.
These include nidanib ethanesulfonate soft capsules for the treatment of idiopathic pulmonary fibrosis and systemic sclerosis; fingolimod for the treatment of relapsing multiple sclerosis aged 10 years and above and sinimod for the treatment of adult relapsing multiple sclerosis patients; ambrisentan tablets for the treatment of patients with pulmonary hypertension with WHO grade II or III symptoms; deuteribenazine tablets are one of the first batch of new drugs urgently needed outside the clinic, approved in May 2020 for the treatment of Huntington's disease-related chorea and adult tardive dyskinesia, which entered health insurance that year; lanreotide acetate sustained-release injection for the treatment of acromegaly; edaravone sodium chloride and riluzole tablets for the treatment of amyotrophic lateral sclerosis (gradual freezing), as well as a number of hemophilia, Parkinson's disease treatment drugs and so on.
Up to now, more than 60 rare disease drugs have been approved for listing in China, of which more than 40 have been included in the national medical insurance catalog, involving 25 diseases.
Clinical urgent need, foreign rare disease drugs have been listed within 70 days of the conclusion.
According to estimates by relevant agencies, there are about 20 million patients with various rare diseases in my country, and more than 200000 new patients are added each year. In the face of an increasingly large group of patients with rare diseases, the pace of drug protection must be accelerated.
In recent years, China has implemented priority review and approval of rare disease treatment drugs. The State Drug Administration has included innovative drugs and improved new drugs with obvious clinical value for the prevention and treatment of rare diseases into the priority review and approval process.
Chen Shifei, deputy director of the State Drug Administration, said that the State Drug Administration and the State Health Commission jointly issued an announcement on matters related to the review and approval of clinically urgently needed overseas new drugs, selected and released a list of three batches of clinically urgently needed overseas new drugs, and encouraged enterprises to declare. Of the 81 drugs selected for release in the three batches, more than half were rare disease treatment drugs. At present, 26 rare disease drugs have been approved for listing through the special channel of clinically urgently needed overseas new drugs. "In all drug listing applications, rare disease drug review and approval time limit is the shortest." Chen Shifei said that in 2020, the newly revised drug registration management measures clearly will have obvious clinical value of the prevention and treatment of rare diseases of innovative drugs and improved new drugs into the priority review and approval process, for the clinical urgent need of overseas listed domestic unlisted rare disease drugs within 70 days of the trial. In 2021, the State Drug Administration newly approved 10 rare disease drugs, such as Lisipulan oral solution, for the treatment of rare diseases such as spinal muscular atrophy.
Chen Shifei said that these drugs fill the gap in the treatment of rare diseases in China, and bring hope for more rare disease patients to delay the development of the disease and improve the quality of life. (Beijing Youth Daily reporter Zhang Xin)

Source: Beijing Youth Daily