Some time ago, the hotly discussed "1.2 million a-shot sky-high god needle" CAR-T immunotherapy heat receded slightly, followed by a shot of 2.125 million US dollars (equivalent to about 13.48 million yuan), refreshing the "most expensive drug in history" Zolgensma is about to enter China.
Recently, the Drug Evaluation Center (CDE) of the State Food and Drug Administration of China announced that the clinical trial application submitted by Novartis' AAV gene therapy drug Zolgensma (OAV101 injection) for the treatment of spinal muscular atrophy (SMA) has been tacitly licensed for clinical trials in China. Priced at $2.125 million (about 13.58 million yuan) abroad, it is also known as "the most expensive drug in history."
Novartis replied that "Zolgensma only needs to be injected once", even if only one injection is the price of "1 suite" in Kitakami, Shanghai and Guangzhou...
01
What is spinal muscular atrophy (SMA)
Speaking of ice bucket challenges, many people can think of the phrase "frozen people (amyotrophic lateral sclerosis, also known as ALS)." But when it comes to its "brother disease" - SMA, many people are confused. In fact, spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disorder characterized by progressive, debilitating muscle atrophy and weakness. Children with SMA are predominantly infants and may present with hypotonia, poor head control, "frog leg" posture, dyspnea, developmental delay, scoliosis, or joint contractures. As the disease progresses, motor function gradually decreases.
SMA is the leading genetic factor in the death of infants under two years of age, severely affecting the quality of life of patients and caregivers, and patients have previously faced the dilemma of not having access to drugs.
In May 2018, SMA was included in the First Catalogue of Rare Diseases jointly formulated by the National Health Commission and other departments.
Based on the patient's onset time and the maximum motor function that can be achieved, SMA is mainly divided into four types:
Type I is the most common and severe, and children often face greater physical challenges, with significant differences in their life cycle based on a number of factors; patients with types II, III, and VI tend to have a normal or near-normal life cycle, but their physical functioning changes over time.
As cruel as "frostbite people", SMA patients will continue to lose the corresponding motor function, but "gradual frost people" are generally adults, and SMA is generally infancy. And SMA is the earlier the onset of the disease, the more serious the disease, "gradually freezing people" is just the opposite.
On May 24, 2019, the U.S. FDA approved Novartis' first gene therapy for the treatment of SMA patients under 2 years of age, Zolgensma. Patients only need to receive one intravenous administration to express smN proteins in cells for a long time, achieving long-term remission and even cure.
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Can spinal muscular atrophy (SMA) be covered by Medicare?
Peng Jing, chief physician of the Department of Pediatrics at Xiangya Hospital of Central South University, previously said in an interview with the media that the incidence of SMA is about one in 6,000 to 10,000. Based on this estimate, there may be between 20,000 and 30,000 patients in China.
"The misdiagnosis rate of this rare disease is extremely high. Newborn children may be misdiagnosed as ischemic hypoxic encephalopathy due to lack of muscle strength, or for cerebral palsy or movement disorders due to lack of strength throughout the body. In addition, the rare disease may be diagnosed with severe pneumonia until death due to often lung disease and lack of strength in the respiratory muscles. But the disease also has its own characteristics, although the child is weak, but the eyes are very good, if you think of this disease, diagnosis is not difficult. Knocking on the knee tendon reflex can not lead out, and doing an electromyography, or genetic testing, can quickly help the patient make a definitive diagnosis. Professor Peng Jing said.
At present, 3 SMA therapies have been approved worldwide, of which Zolgensma has been approved in nearly 40 countries and regions around the world. In 2020, Zolgensma was included in Japan's medical insurance, and patients only need to pay 30% of the fee; then in March 2021, it was included in the national medical service system by the United Kingdom; in 2022, Zolgensma entered China, in addition, in addition to Zolgensma, it also includes Bohain Spinraza and Roche's Evrysdi, the latter two of which have been approved in China.
Spinraza (Northinacine sodium injection) was approved by the State Food and Drug Administration in February 2019 for the treatment of 5q spinal muscular atrophy (accounting for about 95% of all SMA cases), which is also the first drug approved to treat SMA in China.
Previously, Spinraza was priced at nearly 700,000 bottles in China, requiring repeated injections every year and being a completely self-funded drug.
In the 2021 National Medicare Drug List negotiations, 7 drugs for rare diseases were included in the Medical Insurance List, including spinraza, which is highly concerned for the treatment of spinal muscular atrophy.
January 1, 2022, is the day when the 2021 version of the National Medical Insurance Drug List will be implemented. The price of Spinraza single needle has dropped from 700,000 yuan to more than 30,000 yuan. According to data from the Mei'er SMA Care Center, on January 1 this year alone, more than 20 SMA patients in 11 provinces have used Spinraza.
Although Spinraza's sharp price reduction has given hope to many rare disease patients, an industry insider admitted that Spinraza's price reduction is "(price) unexpected, (insured) reasonable".
"Spinraza is not a new drug, it has been basically profitable in the global market, the number of SMA children in China is large, and the drug market is very large. Bojian is actually answering the question of whether to seize the Chinese market in the face of the price reduction of medical insurance negotiations. According to the analysis of the above-mentioned industry insiders, the entry of rare disease drugs into the medical insurance list is essentially under the established medical insurance negotiation framework, due to the breakthrough of individual cases reached by enterprise price reductions, and Bojian has voted for China in the strategic choice.
In recent years, the number of rare disease drugs included in the national medical insurance list has increased year by year. According to the statistics of the Beijing Pain Challenge Public Welfare Foundation, after the negotiation of the national medical insurance list in 2021, 58 drugs with 28 rare diseases have been included in the national medical insurance directory. Based on the statistics of the First Rare Disease Catalogue, the medical insurance catalogue covers more than 60% of the 87 rare disease indications in the mainland. The 2021 medical insurance directory negotiations have achieved a "zero breakthrough" in the national medical insurance catalogue of high-value rare disease drugs.
But on the other hand, it is also the industry consensus that medical insurance cannot support all high-value drugs. Zheng Jie, head of the National Medical Insurance Negotiation Drug Fund Calculation Expert Group, has publicly stated that "at present, the annual treatment cost of all drugs in the national medical insurance catalogue has not exceeded 300,000 yuan", and said that in the future, the National Medical Insurance Bureau will "resolutely eliminate the entry of sky-high drugs into medical insurance". The national medical insurance catalogue, which bears the functional positioning of "guaranteeing the basics", determines that the drugs that can be included must have the basic condition of "reasonable price".
The good news is that car-T immunotherapy like "1.2 million a shot" has not entered the medical insurance negotiations, but it has been included in the scope of medical insurance protection of many insurance companies, so that more families can see the hope of rehabilitation, I believe that in the near future, more sky-high magic needles can enter the homes of ordinary people through insurance protection.