Reporter of the Economic Business: Chen Xing Reporter of the Economic Business Business: Dong Xingsheng
Rare diseases drugs are ushering in a high-profile "era". Recently, Bi Jingquan, deputy director of the Economic Committee of the National Committee of the Chinese People's Political Consultative Conference, proposed that to encourage the development of drugs for rare diseases, special policies that comply with the laws of rare diseases need to be formulated. Encouraging enterprises to develop drugs for treating rare diseases is the key to solving the problem that no drugs are available for rare diseases.
At the recent CIIE, multinational pharmaceutical companies such as Roche Pharmaceutical , Sanofi , AstraZeneca and other multinational pharmaceutical companies have appeared with their rare disease drugs, and many of them are expected to be launched in China, benefiting patients with rare disease in China.
has few patients, high R&D costs, and heavy drug burden, which has made the research and development of rare disease drugs once a "hard to crack". However, the small number of patients should not be a reason to be ignored.
Wang Lei, global executive vice president, international business and Chinese president of AstraZeneca, said in an interview with the reporter of " Daily Economic News " that from diagnosis to medication, patients with rare diseases still have many needs waiting to be met. "Rare diseases are an important theme in countries around the world and are also a headache. There are thousands of rare diseases, many of which do not have drugs, and there are huge and unmet medical needs. Also, most of the patients with rare diseases are children, so all countries regard rare diseases as important things to face."
Image source: Photo Network-500668961
AstraZeneca will launch its first rare disease drug in China
From policy makers to pharmaceutical companies, the popularity of rare disease drugs has been rising again and again.
During this year's CIIE, many multinational pharmaceutical companies appeared with rare disease drugs, including AstraZeneca. On November 11, AstraZeneca announced that it will launch its first rare disease innovative drug, Shulizumab, in China, which was approved in China for the treatment of paroxysmal sleep hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children.
Eculizumab is the world's first approved C5 complement inhibitor, which works by selectively inhibiting the activation of terminal complement C5 proteins. At present, markets such as the United States, , EU, , and Japan have approved it for the treatment of adult patients with systemic myasthenia gravis (gMG) who are positive for paroxysmal sleep hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR) antibodies. In 2018, aHUS and PNH were included in my country's "First Batch of Rare Diseases Catalog". Although
has been included in the "First Rare Disease Catalog", there is currently a lack of effective treatment methods for domestic patients with aHUS and PNH.
Fu Rong, deputy director of , Tianjin Medical University General Hospital, said that paroxysmal sleep hemoglobinuria (PNH) is a life-threatening blood system rare disease. Patients will experience symptoms such as anemia , bone marrow failure, hemoglobinuria . Previously, the treatment methods of PNH were mainly symptomatic treatment, including blood transfusion, glucocorticoids, immunosuppression, etc. However, traditional therapies face many difficulties in controlling hemolysis and preventing renal damage.
and aHUS is a persistent complement -mediated systemic thrombotic microvascular disease, which has a acute onset and a much higher incidence in children than in adults. . Mao Jianhua, deputy director of , a Children's Hospital Affiliated to Zhejiang University School of Medicine, said: "Attypic hemolytic uremic syndrome (aHUS) is a rare and fatal thrombotic microvascular disease. Plasma treatment is the initial method of aHUS management, but the efficacy is not good. Some even require dialysis and kidney transplantation. In recent years, with in-depth understanding of the pathogenesis of aHUS, C5 complement inhibitors have also become one of the therapeutic weapons."
reporter learned from AstraZeneca that the drug will be implemented in cities such as Suzhou , Qingdao , Beijing, Shanghai, Tianjin, Hangzhou and other cities as soon as possible. After Shu Lirui, AstraZeneca is also promoting the clinical development of more than 20 rare disease drugs, and laying out six major diseases in the blood, kidney, , central nervous system, , metabolism, cardiovascular and ophthalmology. In the future, a number of new drugs will continue to be introduced, involving complement and non-complement systems, and gradually achieve synchronous research and development with the world.
At this year's CIIE, AstraZeneca also announced two actions in the field of rare diseases. They reached a strategic cooperation with CanSino to sign a contract with to explore development opportunities in the field of diagnosis and treatment of rare diseases; they also made progress with the Qingdao Municipal Government in the construction of Qingdao regional headquarters with the theme of rare diseases and the innovation ecosystem. The two sides further deepened their cooperation in the introduction of innovative drugs for rare diseases and diagnosis and treatment services.
contract signing site Image source: Image provided by the company
Rare Disease directory needs to be updated, payment problems need to be broken together
From no one is concerned to many multinational pharmaceutical companies unveiled with rare disease drugs at the CIIE. How did the rare disease drug market attract the attention of pharmaceutical companies?
In September 2021, AstraZeneca established a rare disease business unit in China to promote innovative progress of multiple rare disease drugs in China. Wang Lei said that from diagnosis to medication, patients with rare diseases still have many needs waiting to be met. "Rare diseases are an important theme in countries around the world and are also a headache. There are thousands of rare diseases, many of which do not have drugs, and there are huge and unmet medical needs. Also, most of the patients with rare diseases are children, so all countries regard rare diseases as important things to face." Wang Lei said.
Frost Sullivan and Beijing Disease Challenge Charity Foundation released the "2022 China Rare Disease Industry Trend Observation Report" shows that there are currently more than 7,000 rare diseases known worldwide, and more than 300 million rare diseases worldwide, of which nearly 20 million patients in my country are fighting against rare diseases.
Although rare diseases have attracted unprecedented attention since the formulation of the "First Rare Disease Catalog", as of now, there are still many gaps in the rare diseases field waiting to be filled.
Wang Lei believes that, first, we must speed up the approval of rare disease drugs. At present, the approval of many rare disease drugs has greatly accelerated, and this speed must be persisted; second, we must introduce industrial policies to encourage research and development, because most rare diseases lack medical treatment and medicine. Currently, there are thousands of rare diseases, and there are only a few hundred corresponding drugs. Social forces, scientific research forces, and corporate forces should be concentrated to encourage the development of rare disease drugs; third, we should solve the problem of patient payment, and the number of patients with rare diseases is small. Unlike developing a drug that uses millions or tens of millions of people, the unit price of rare diseases is often higher, and the individual payment burden of patients is heavy. "So can we set up a special project for rare diseases in the medical insurance fund to negotiate rare diseases separately"; fourth, there are fewer doctors who can see rare diseases than patients with rare diseases, so we should consider establishing a rare disease registration, diagnosis and treatment network, and establish a special disease center or medical center for rare diseases. It is possible to refer patients with rare diseases to special rare disease centers through registration, diagnosis and consultation, so as to obtain the fastest diagnosis and best treatment.
has many problems with rare diseases that need to be broken, but it has attracted consensus that the types of rare diseases in the rare diseases directory should be enriched first.
Wang Lei said that the types of rare diseases included in the catalog only account for a very small part of the currently known rare diseases. Rare diseases need to be updated, so that relevant therapeutic drugs can be developed and promoted in a targeted manner, so as to introduce more relevant industrial policies, medical insurance policies, and tiered diagnosis and treatment methods.
In terms of payment, Wang Lei also said that he will strive to enable Shu Lirui to achieve the lowest price in the world in China, "if there is an opportunity for medical insurance negotiations, we will also actively participate."
Daily Economic News
