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Rare disease is a disease with a low incidence.
At present, there are about 20 million patients with various rare diseases in China, and more than 200,000 new patients are added every year
.
Due to the low investment in scientific research and the low diagnosis rate, rare disease drugs have the characteristics of low market demand and high R&D costs, most of the rare disease drugs have always been very expensive
.
In order to get rid of the dilemma of “no medicines available” and “cannot use medicines” for patients with rare diseases, relevant state departments have continued to make efforts to continue to increase favorable policies.
Among them, medical insurance negotiations are an important measure
.
Since the establishment of the National Medical Security Administration in 2018, it will dynamically adjust the list of medical insurance drugs every year, which includes many oncology drugs and rare disease drugs, and the number of drugs included in the list continues to increase, and the coverage of rare diseases continues Expand
.
Through medical insurance negotiations, the burden of medication for patients with rare diseases has been greatly reduced
.
On December 18, the 2021 China Rare Diseases Conference was held.
At the meeting, it was revealed 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 List, involving 25 types disease, including Amyotrophic Lateral Sclerosis disease, hemophilia
.
In 2021, the new round of medical insurance catalogue adjustments will include more drugs for rare diseases.
A total of 7 drugs for rare diseases have been successfully negotiated, with an average price drop of 65%
.
Among them, Bojian’s widely-concerned "700,000-a-shot" Nosinagen Sodium Injection is also on the list of medical insurance catalogues.
The original domestic price of this product was 699,700 yuan a shot, but it dropped to 550,000 yuan at the beginning of this year.
Now that it is included in medical insurance, it is estimated that the drug will not exceed 33,000 yuan per injection
.
Such a substantial reduction will bring good news to the majority of patients with spinal muscular atrophy in China
.
It is reported that spinal muscular atrophy is a rare disease, an autosomal recessive inherited disease, mainly due to the genetic neuromuscular disease caused by the defect of SMN protein function due to the mutation of motor neuron survival gene 1
.
At present, the number of SMA patients in China is about 30,000
.
Takeda's million-dollar drug agalsidase alpha injection concentrated solution for Fabry treatment is also newly added to medical insurance this time, which will reduce the burden of drug costs for patients
.
Data shows that the product is a human-derived enzyme replacement therapy, which can bring long-term heart and kidney protection and delay the disease process, and improve the quality of life of Fabry disease patients and their families
.
Fabry disease is an X-linked genetic disease.
Due to the mutation of the GLA gene encoding α-galactosidase A (α-Gal A) in the middle of the long arm of the X chromosome, the structure and function of α-Gal A are abnormal and metabolized A clinical syndrome caused by the accumulation of the substrate trihexosylceramide (GL-3) and related glycosphingolipids in multiple organs throughout the body
.
The indication range of the concentrated solution for agalsidase alpha injection in the medical insurance catalogue this time is: it is used for long-term enzyme replacement therapy for patients diagnosed with Fabry disease, and is suitable for adults, children and adolescents
.
In addition to the two rare disease drugs mentioned above, Bojian is also on the list of fampridine sustained-release tablets for improving walking dysfunction in patients with multiple sclerosis (MS), and Feizeyou, which is used by Takeda for the treatment of hereditary angioedema
.
In addition, human coagulation factor IX for the treatment of hemophilia, clofenac soft capsules for the treatment of adult wild-type or genetic transthyretin amyloid cardiomyopathy, and injection of a new long-acting lipid-lowering drug iloyuumab Liquid has also successfully entered the adjusted medical insurance drug list, which will bring the gospel of cost reduction for patients with related rare diseases and improve the quality of life
.
At present, there are about 20 million patients with various rare diseases in China, and more than 200,000 new patients are added every year
.
Due to the low investment in scientific research and the low diagnosis rate, rare disease drugs have the characteristics of low market demand and high R&D costs, most of the rare disease drugs have always been very expensive
.
In order to get rid of the dilemma of “no medicines available” and “cannot use medicines” for patients with rare diseases, relevant state departments have continued to make efforts to continue to increase favorable policies.
Among them, medical insurance negotiations are an important measure
.
Since the establishment of the National Medical Security Administration in 2018, it will dynamically adjust the list of medical insurance drugs every year, which includes many oncology drugs and rare disease drugs, and the number of drugs included in the list continues to increase, and the coverage of rare diseases continues Expand
.
Through medical insurance negotiations, the burden of medication for patients with rare diseases has been greatly reduced
.
On December 18, the 2021 China Rare Diseases Conference was held.
At the meeting, it was revealed 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 List, involving 25 types disease, including Amyotrophic Lateral Sclerosis disease, hemophilia
.
In 2021, the new round of medical insurance catalogue adjustments will include more drugs for rare diseases.
A total of 7 drugs for rare diseases have been successfully negotiated, with an average price drop of 65%
.
Among them, Bojian’s widely-concerned "700,000-a-shot" Nosinagen Sodium Injection is also on the list of medical insurance catalogues.
The original domestic price of this product was 699,700 yuan a shot, but it dropped to 550,000 yuan at the beginning of this year.
Now that it is included in medical insurance, it is estimated that the drug will not exceed 33,000 yuan per injection
.
Such a substantial reduction will bring good news to the majority of patients with spinal muscular atrophy in China
.
It is reported that spinal muscular atrophy is a rare disease, an autosomal recessive inherited disease, mainly due to the genetic neuromuscular disease caused by the defect of SMN protein function due to the mutation of motor neuron survival gene 1
.
At present, the number of SMA patients in China is about 30,000
.
Takeda's million-dollar drug agalsidase alpha injection concentrated solution for Fabry treatment is also newly added to medical insurance this time, which will reduce the burden of drug costs for patients
.
Data shows that the product is a human-derived enzyme replacement therapy, which can bring long-term heart and kidney protection and delay the disease process, and improve the quality of life of Fabry disease patients and their families
.
Fabry disease is an X-linked genetic disease.
Due to the mutation of the GLA gene encoding α-galactosidase A (α-Gal A) in the middle of the long arm of the X chromosome, the structure and function of α-Gal A are abnormal and metabolized A clinical syndrome caused by the accumulation of the substrate trihexosylceramide (GL-3) and related glycosphingolipids in multiple organs throughout the body
.
The indication range of the concentrated solution for agalsidase alpha injection in the medical insurance catalogue this time is: it is used for long-term enzyme replacement therapy for patients diagnosed with Fabry disease, and is suitable for adults, children and adolescents
.
In addition to the two rare disease drugs mentioned above, Bojian is also on the list of fampridine sustained-release tablets for improving walking dysfunction in patients with multiple sclerosis (MS), and Feizeyou, which is used by Takeda for the treatment of hereditary angioedema
.
In addition, human coagulation factor IX for the treatment of hemophilia, clofenac soft capsules for the treatment of adult wild-type or genetic transthyretin amyloid cardiomyopathy, and injection of a new long-acting lipid-lowering drug iloyuumab Liquid has also successfully entered the adjusted medical insurance drug list, which will bring the gospel of cost reduction for patients with related rare diseases and improve the quality of life
.
