Talk about rare diseases: What can I cure patients' anxiety?

"Unforgettable three days, this is a conference held in a great uncertain factors ..."

On the evening of September 4th, Huang Rangfang summarized the meeting of the past three days with a circle of friends, and then fell asleep. As the founder of the rare disease center, Huang Rufang is a rare disease patient and a leading figure in the industry. In the past ten years, Huang Rufang led the rare disease summit forums, but in 2022 when the epidemic was repeated, he fell into the "rare disease anxiety" of how to run the meeting for a long time before the opening of the 11th forum.

This kind of anxiety is also permeated between the hearts of rare diseases and companies: the second batch of rare disease catalogs are waiting to be updated. My disease is not inside. What should I do? The financing environment of the first -level market is cold, and there is no funding for research and development. What should I do? What should I do if the price of high -value rare diseases is not reduced to the "basic" line of medical insurance negotiations?

At the summit forum from September 2nd to 4th, these people gathered together to find and create cure recipes together. "Fortunately." Huang Rangfang's second half of the concluding meeting was "unexpectedly good."

Rare disease anxiety in the post -epidemic era

Different from the tenth summit forums previously held, this year, more guests who chose to attend the conference online have changed.

At the opening ceremony of the Eleventh Rare Disease Summit Forum, the speech of the first 40 minutes appeared online.

In the opening ceremony of two and a half hours, the word "anxiety" appeared 20 times from the mouth of different speakers; in the first half of the year, any wind blowing of the new crown epidemic was touched the sensitive nerves of patients with rare diseases.

Huang Rufang made the theme speech in the opening ceremony. Photo Source: The organizer confession

According to Jia Yan, the person in charge of the information department of the Rou Dehan Disease Center, "there is medicine abroad, no medicine in the country", restrictions on medical prescriptions, crisis crisis of drug breaks, re -examination of home patients and participation in clinical trials, and incomplete hierarchical diagnosis and treatment policies. It is the five dilemma facing rare diseases in the public health incident.

Huang Rufang also said at the meeting: "I am grateful to the partners to continue supporting us, but can I continue to support it next year? This is a big question mark for me. (As one of the leaders of the conference), this question mark is also also Give me a strong sense of anxiety. "

As a colleague of Huang Rufang, Li Yangyang, director of the Rare Public Policy Center of Kou Dehan and chief business officer of Langyu Group, claimed to be an idealist, and rationalized his friend's anxiety. She believes that the background of the rare cause of the domestic disease to the "anxiety development stage" is the honeymoon period of the industry's policy before, and the industry has a foam illusion. Recognize the challenges in rare diseases.

At present, there are more than 7,000 rare diseases worldwide. Even in the European and American markets that are relatively mature in the rare disease industry, more than 90%of the diseases are still not treated, and less than 10%can be used.

In the question that can't be around, when will the rare disease catalog update?

2018 is the big year for the development of China's rare disease industry. At that time, the National Health and Health Commission and other five departments jointly released the first batch of rare disease catalogs in China. This list of 121 rare diseases is not only the first time that the official is defined under the "rare disease" in the form of a catalog, but also great Promote public communication, patient services, investigations, and medical insurance policies in the rare disease field in the future.

Shanghai girl Zhang Xue Shen was touched. "(After the first batch of catalogs are released) We can boldly say the name of our disease. If you don't know, we will tell you that the fifth one on the rare disease catalog is us."

As a mother of a child syndrome, Zhang Xue often faced the situation of non -understanding, misdiagnosis and refusal when she was exposed to rare diseases more than ten years ago. At that time, even children in similar dilemma had "information islands", which was difficult to find each other.

In the first half of this year, the National Health and Health Commission stated that it had launched the selection of the second batch of rare diseases in accordance with the regulations, and it was in the material review stage. This news added a fire to the discussion of the second batch of rare diseases in the circle.

"Rare disease directory impact assessment and prospect" Special picture source: The organizer confess to the picture

As a neurologist, Zhang Cheng, deputy director of the Department of Neurology, the First Affiliated Hospital of Sun Yat -sen University, believes that according to the current national conditions, the rare disease catalog cannot be included in all rare diseases. Essence Zhang Cheng suggested that rare disease directory can refer to medical insurance negotiations to make dynamic adjustments to accelerate the frequency of update; rehabilitation therapy generally needed for patients with rare diseases can also be included in the agenda.

For the rare types of diseases that are more likely to be included in the second batch of catalogs, Wang Yi, vice president of the China Antilateral Episodic Association: seriously affects the national health level and quality, major birth defects, important deaths and disability, diseases have some diseases. There may be means to intervene and prevent, more common or distinctive diseases in China, drug development prospects and can be transformed and innovative with modern medical research.

As a closer to the first batch of rare disease catalogs, Zhu Kun, deputy director of the Social Development Research Center of the Chinese Academy of Finance Sciences, is even more restrained. He believes that the second edition of the rare disease catalog must have improved in terms of principles or standards, but optimization also requires the process.

Enterprise: I hope to open the last mile on the payment side

The discussion of the discussion of medical insurance negotiations in 2021 is no less than that of the second batch of rare diseases.

Xu Yifan, vice chairman of the Fabarey Association, remembers that after the long solution of Agotase α injection of an annual treatment cost of over one million was successfully negotiated by the "official announcement", the WeChat group "opened the pot" in the group; but for the industry, in the industry For companies that have been in the inside, the mood is complicated: whether the negotiation line of "500,000 non -discussion (judgment), 300,000 (guarantee)" circulating in the industry is applicable to orphan drugs (that is, rare diseases) Intersection The case of "Dongpei Pharmaceutical" attracted corporate discussions.

Four years ago, East Pei Pharmaceuticals followed the footsteps of "The First Batch of New Medicine Listing Overseas and Urgent Domestic Clinical Domestic Pharmaceuticals". In China, nearly 100 employees were recruited. ) The successful listing in 2020 ended the suffering that the moderate or severe neurotic keratonitis (NK) patients had no medicine available.

However, with the failure of this eye -catching medical insurance negotiations with a total treatment cost of about 140,000 yuan, this small and medium -sized Italian pharmaceutical company has dissolved the Chinese team this year.

Why did this company come again? In the rare disease field with high value drugs, what are the "1+N" multi -party co -consensus? (Note: 1+N: The government as the main body, medical institutions, pharmaceutical enterprises, insurance institutions, social charity, and patients pay for innovative payment models for multi -party payment)

From the perspective of medical insurance researchers, Wu Jing, deputy dean of the School of Drug Science and Technology of Tianjin University, said that national medical insurance is a "value -based strategic purchase". Look at it "to pass the level; but in the second round of value assessment, the price threshold for orphan drugs has not yet been formed. Wu Jing suggested that in the future, the severity of the disease can be used as a direction to ensure the maximum value of orphan drugs selected into the insurance.

Jin Chunlin, director of the Shanghai Health and Healthy Development Research Center, proposed that the price red line of rare diseased drugs is radical. The medical insurance fund as "1" has a leading role in the later "N". In the future, you can explore more in innovative payment. For example, the government is worried about the number of patients, the efficacy and price of the drug, and the price of the gambling agreement with the enterprise to ensure the controlling the medical insurance fund expenditure.

As a representative of the enterprise, Yu Lei, the leader of the rare disease and rare hematological business of Sanofi (China), said that in practical exploration, Zhejiang and Jiangsu have passed the risk sharing model of innovative payment. By summing up these local experience, it can promote the national rare medical drug guarantee mechanism.

The existing medicines in Takeda China entered the medical insurance directory last year. Zhang Min, the vice president and the person in charge of the Excellence Department of the Excellence, said that with the help of multi -level protection, "dual channels", "Mentemen," and other policies, patients can Sexuality has developed rapidly; everyone needs to work hard to open up the "last mile" of entering the hospital. It is necessary to face the KPI thinking of some hospitals and the proportion of drugs to awaken the whole society's awareness of the diagnosis and treatment of rare diseases.

"National Talking about the Deep Winning of the Soul Guts" "Special Specialist Source: The organizer confession

From the perspective of the "Adjustment Plan for the Medical Insurance Catalog (Draft for Soliciting Opinions)" issued by the State Medical Insurance Bureau in June, it is clearly proposed at the national level that it is encouraged to be included in the new crown diagnosis and treatment schemes related drugs, children's medicines and rare diseases, and further relax the application conditions. In May, the "Drug Management Law (Draft for Revised Draft Consultation)" also mentioned that it can be given a 7 -year market exclusive period for rare diseases. All of the above reflect the sincerity of the country to retain innovative enterprises.

What can I cure the anxiety of patients with rare diseases? When this problem is floating in the air, love and sunlight have sprinkled in these melancholy corners, and the sick people also emit the energy of healing others.

Tomorrow of the rare disease industry in China, perhaps as Huang Rangfang said at the opening ceremony of the Eleventh Rare Disease Summit Forum: "When the cold is coming, the first frozen to death must be a weak grass. Of course, winter will pass, and it will pass in winter. The spring breeze blows again. "

Daily Economic News

- END -