Abstract: Yang Qihui, head of the demyelinating project of the Pain Challenge Foundation, told the China Times reporter that there is no exact study and statistics on the incidence of multiple sclerosis in China. However, referring to the incidence in Japan, Taiwan and other places, the number of patients in Chinese mainland should be 30,000-50,000. "About one-tenth of them are reached by our patient organization, about 3,000-5,000 people."
China Times (www.chinatimes.net.cn) reporter Cui Xiaotian reported in Beijing
Rare disease patients usher in new hope. On July 30, the National Medical Insurance Bureau announced a list of drugs that passed the preliminary formal examination and are expected to be included in the 2021 national medical insurance directory.
The list contains 32 rare disease drugs, not only a high-value drug of hundreds of thousands of yuan, but also a relatively low-priced new drug for rare diseases, the latter of which has a greater chance of being included in the new version of the medical insurance directory, such as Bojian's recent domestic approval for the treatment of multiple sclerosis drugs - dimethyl fumarate enteric-coated capsules (Tefida) and aminopyridine extended-release tablets (compound two). These two products can be used in combination clinically, dimethyl fumarate focuses on reducing patient recurrence and delaying disease progression, and aminopyridine is mainly used to improve patients' lower limb motor dysfunction and improve walking ability in the short term.
Yang Qihui, head of the demyelinating project of the Pain Challenge Foundation, told the China Times reporter that there is no exact study and statistics on the current incidence of multiple sclerosis in China. However, referring to the incidence in Japan, Taiwan and other places, the number of patients in Chinese mainland should be 30,000-50,000. "About one-tenth of them are reached by our patient organization, about 3,000-5,000 people."
According to the reporter of this newspaper, these patients can now be prescribed to dimethyl fumarate in medical institutions, and can also participate in the patient assistance program of buying one free. A box of 56 dimethyl fumarate enteric-coated capsules (240 mg) is priced at about 11,000 yuan, slightly lower than the existing domestically listed drugs. Another product, ammydine, is expected to be available for prescription within the year.
There are 6 drugs listed in China, and a variety of medical insurance can cover them
"At one point I thought nothing could stop me. And now, I'm afraid that one day I'll lose even the most basic ability to walk. "This is the voice of a patient with multiple sclerosis.
Multiple sclerosis is an immune-mediated disease, the main feature is the central nervous system inflammatory demyelinating lesions, more often in 20-40 years of age in young and middle-aged people, the prevalence of women is about 2 times that of men, so this disease is also called "beauty disease". Early symptoms include blurred vision, visual field defects, weakness and numbness in the limbs, or persistent fatigue and ataxia.
Impaired walking ability is a key undesirable feature of the disease. If left untreated, patients will have multiple relapses, disabilities will progress, and life expectancy will be shortened by an average of 7-14 years compared to healthy people. According to the 2020 China Multiple Sclerosis Patients Comprehensive Social Survey Report, more than 43% of patients cannot take care of themselves completely and need to be cared for by others, and up to 88.5% of them are unemployed and out of school due to multiple sclerosis.
Fortunately, the treatment of multiple sclerosis is more abundant than other rare diseases, and most of them have been included in the national health insurance directory.
According to Yang Qihui, there is no specific drug that can cure multiple sclerosis, and the existing drugs are mainly used to reduce the frequency and severity of recurrence, requiring long-term medication. In addition to the two drugs recently listed by Bojian, there are also four drugs in China that belong to Novartis (Fingolimod, Sinimod), Sanofi (trifluramide), Bayer (recombinant human interferon β-1b) to choose from.
National health insurance has opened its doors to most multiple sclerosis drugs. Novartis' two drugs have been included in the national medical insurance in 2020, and Sanofi's terifluamine was included in the national medical insurance in 2019, and it needs to be renewed this year to compete with the two new drugs of Bojian. Bayer's drugs failed in 2019 to renew the national health insurance contract, and now they can only be purchased at their own expense.
"The domestic pricing of these drugs is similar, and the annual drug cost is about 100,000 yuan." At present, the degree of medical insurance reimbursement varies from place to place, for example, after reimbursement in Beijing, patients only need to pay less than 20,000 yuan, and some regions have a relatively low reimbursement ratio, and patient expenditure will be more. Yang Qihui said.
Rapid listing benefits from policy dividends
Focusing on bojian's two recently approved drugs in China, dimethyl fumarate was listed in the United States in 2013 and has been approved in 69 countries and regions so far, with more than 500,000 patients receiving treatment.
Professor Hu Xueqiang, chief physician of the Department of Neurology of the Third Affiliated Hospital of Sun Yat-sen University, told this reporter that the results of clinical trials and more than ten years of real-world evidence have fully proved that dimethyl fumarate can help patients significantly slow down the development of the disease process, reduce the occurrence of disabling lesions, and the earlier it is used, the more beneficial it is to patients.
Aminopyridine extended-release tablets are more suitable for combination drugs, the rest of the listed drugs focus on reducing patient recurrence, delaying disease progression, aminopyridine is mainly used to improve patients' lower limb motor dysfunction, short-term improvement of walking ability, is the first in China.
A number of rare disease drugs have been listed in China, thanks to the policy dividends of recent years in China. In 2018, the relevant ministries and commissions of the state jointly formulated and issued the First Catalogue of Rare Diseases, which included 121 diseases. This means that China's rare diseases have since been justified, and tens of millions of patients have stood in the sun.
Since then, many forces have participated in the solution of rare disease diagnosis and treatment, drugs, employment and other issues, and the most prominent manifestation is that more and more drugs listed in the United States have entered China.
In 2018, the State Food and Drug Administration, together with the National Health Commission, drafted the "Procedures for the Review and Approval of Overseas New Drugs urgently needed for clinical use" and the requirements for declaration materials. The document stipulates that for drugs treated with rare diseases, the drug review center shall complete the technical review within 3 months after acceptance, and for other new overseas drugs, the drug review center shall complete the technical review within 6 months after acceptance. The State Drug Administration makes an approval decision within 10 working days after receiving the review materials submitted by the Drug Review Center.
Wen Haoji, President of Bojian Asia Pacific, said: "We are very grateful to the State Food and Drug Administration for its priority review and approval process, so that the two innovative achievements of dimethyl fumarate enteric-coated capsules and aminopyridine extended-release tablets can quickly enter China. ”
Zhongtai Securities Research Report shows that as early as 2018, the global multiple sclerosis drug market has reached about 23 billion US dollars, the market space is large, of which Bojian occupies about 40% of the global multiple sclerosis drug market share (annual sales of about 9 billion US dollars). At present, there are about 20 related drugs listed worldwide, and Bojian accounts for a relatively large proportion. Recently, Bojian also purchased the exclusive global rights of the multiple sclerosis indications of opoutinib developed by the domestic innovative pharmaceutical company Nuocheng Jianhua, with a total transaction price of about 940 million US dollars, plus more than 10% of the sales share.