Concerned about rare diseases, domestic hemophilia patients urgently need to get rid of the new dilemma of "inaccessible medicine"

Rare diseases are diseases with a low incidence and a small number of patients, but rare diseases are actually not uncommon.
There are nearly 7,000 kinds of internationally recognized rare diseases, accounting for about 10% of human diseases
.
In China, the number of rare disease patients is expected to exceed 20 million
.
At present, the majority of rare disease groups are generally faced with the dilemma of no drugs available or inaccessible, and all parties are also working hard to improve the quality of life of domestic rare disease patients
.
In January 2021, a new round of national medical insurance negotiations included drugs for the treatment of rare disease spinal muscular atrophy, which originally required 700,000 yuan per drug, but now only costs about 30,000 yuan, which greatly reduces the cost of related patients.
The burden of drug costs has also attracted the attention of other rare disease patient groups
.
Among them, hemophilia is a hereditary blood coagulation disorder characterized by positive family history, juvenile onset, persistent bleeding after spontaneous or mild trauma, hematoma formation, and joint hemorrhage
.
At present, there are about 140,000 hemophilia patients in China, and hemophilia A accounts for more than 80% of the total.
Such patients are recessive hereditary bleeding caused by X-chromosome-linked coagulation factor VIII deficiency and abnormal coagulation function.
sexually transmitted diseases
.
It is understood that, in the past, domestic patients with hemophilia A generally lacked new treatments, but now, "medication is difficult to obtain" is becoming a new dilemma faced by such patients
.
The reason behind this is that despite the availability of new treatments, most patients’ families still cannot afford the medicines
.
It is understood that Roche's emecizumab injection is a blockbuster drug approved in China for the treatment of hemophilia A.
It has clinically outstanding efficacy and high safety.
It is well tolerated and can be administered Convenient
.
As a recombinant humanized, bispecific monoclonal antibody, this product can replace the cofactor activity of activated coagulation factor VIII, promote the activation of FIXa by FIXa, and then lead to a significant increase in the generation of thrombin, making FVIII dysfunctional or completely The bleeding site of hemophilia A patients lacking FVIII achieves hemostasis
.
Emicizumab entered the Chinese market in November 2018 for the routine prophylactic treatment of hemophilia A patients with factor VIII inhibitors (congenital factor VIII deficiency) to prevent or reduce bleeding Frequency of
.
The product has been closely watched by patients as soon as it was launched, but the high cost has also discouraged many patients' families
.
According to a recent heated discussion in the industry called "An Open Letter from Chinese Hemophilia Patients to Roche Pharma", the letter mentioned that since the domestic launch, Roche Pharma has never conducted any adjustment to the sales price of emilacizumab.
adjust
.
As a life-long drug, the dosage of the drug is directly linked to the patient's weight.
According to the selling price of 8,100 yuan/30 mg, the annual treatment cost for a patient weighing 60 kg will reach 1.
2 million yuan.
The cost also reached hundreds of thousands of yuan
.
In addition, judging from the participation in the medical insurance negotiation, in the three years since the launch of the drug, Emicelizumab has repeatedly declared and passed the preliminary drug review, but the declaration has not been brought to the negotiating table for three years
.
In this regard, the majority of hemophilia patients through an open letter called on Roche Pharmaceuticals to put patients first and reduce the sales price of Emicizumab injection, in order to better benefit rare disease patients and enter the national medical insurance catalogue as soon as possible.
one step
.
The industry pointed out that for rare disease patients, the availability of drugs is a very important step, but the problems of drug supply and payment security are also the next problems to be solved urgently
.
We also expect that with the efforts of relevant departments and enterprises in the future, the majority of rare disease patients will be able to have medicines available and affordable
.
Disclaimer: Under no circumstances does the information or opinions expressed in this article constitute investment advice to anyone
.