How do patients with spinal muscular atrophy survive beyond "550,000 in 4 days"?

Recently, a piece of news that “costed 550,000 yuan for 4 days in the hospital” circulated on the Internet, causing people to question the hospital's arbitrary charges

Although this is an "Oolong Incident", it reflects the pain of rare disease treatment

Most of the most expensive drugs in the world are rare disease drugs

SMA is a chromosomal recessive genetic disease, the incidence of newborns is about 1/6000-1/10000

According to the White Paper on the Survival Status of SMA Patients in China, there are currently about 30,000 SMA patients in China

Nosinagen Sodium is the world's first SMA precision targeted therapy developed by Biogen.

The price of Nosinagenic Sodium in the United States is US$125,000 per dose, which is approximately RMB 870,000.

Why are rare disease drugs so expensive?

According to data published by the US drug price tracking website GoodRx, most of the 10 most expensive drugs in the world are drugs for rare diseases

In the field of rare diseases, why has the annual treatment cost exceeded one million yuan become a norm?

According to statistics, during the ten years from 2011 to 2020, the average success rate of rare disease (excluding tumor indications) drugs from phase 1 clinical trials to the approval of the US FDA for marketing is 17.

In addition, according to the Frost & Sullivan report, from 2020 to 2030, the global rare disease drug market will increase from 135.

However, the problem is that pharmaceutical companies need to maintain a balance between investment and revenue in R&D

Patients with rare diseases only account for 0.

Is it feasible for rare disease drugs to enter medical insurance?

As far as rare diseases are concerned, the inclusion of corresponding products in medical insurance within an appropriate scope is an important measure to benefit more patients.

According to the "Statistical Bulletin of Medical Security Development in 2020", as of the end of 2020, the number of people participating in full-caliber basic medical insurance was 1.

SMA treatment drugs are expensive.

At present, China has included a number of rare disease drugs in the "National Medical Insurance Drug List 2020", and medical insurance covers 40 rare disease drugs, although most of them are for orphan disease indications and reuse common drugs, and a total of 16 rare diseases are included.

Multiple measures to help rare disease drug research and development

In addition to the high price of medicines, for patients with rare diseases, the dilemma faced is the helplessness of no medicines available

According to China's "First List of Rare Diseases", a total of 162 therapeutic drugs for the treatment of 74 rare diseases are currently on the market globally, of which only 83 drugs for 53 rare diseases have entered China
.
However, there are only 55 drugs that are clearly registered as rare disease indications in China, involving 31 rare diseases
.
For the majority of patients with rare diseases, the existing treatment drugs are obviously insufficient
.

In response to the research and development of drugs for rare diseases, in recent years, China has issued a number of policy documents leaning towards drugs for rare diseases
.

In 2016, the "Opinions on Implementing Priority Review and Approval for Resolving the Backlog of Drug Registration Applications" pointed out that rare disease drug registration applications can be reviewed and approved first
.
In 2017, the "Opinions on Encouraging Drug Innovation and Implementing Priority Review and Approval" also pointed out that rare diseases can apply for reduction or exemption of clinical trials when applying for clinical trials
.
In the newly revised Drug Administration Law in 2019, the priority review and approval of new drugs for rare diseases was once again emphasized
.

According to the "2018 Drug Evaluation Report", in 2018, the Center for Drug Evaluation included a total of 313 registration applications into the priority review process, of which 63 were for children's drugs and drugs for rare diseases
.

Many candidate products have entered Phase 2 clinical stage

In the field of SMA treatment, currently only three drugs have been approved for marketing in the world.
In addition to Biogen's Noxinagen sodium, the other two drugs are Novartis' Zolgensma and Roche's Evrysdi
.

Among them, Zolgensma was approved by the FDA in May 2019 with a price of US$2.
125 million.
Patients only need one injection to complete the treatment.
In 2020, this product will bring Novartis's sales revenue of US$920 million
.

Evrysdi was approved in August 2020.
It is the first oral therapy approved by the FDA for the treatment of SMA and is priced at a maximum of US$340,000 per year
.
In June 2021, this product was approved for listing in China
.
Patients need 5-6 injections in the first year, and 3 injections a year for maintenance treatment thereafter
.

In addition to the marketed products, there are several drugs under development in the SMA field.
The candidate products such as branaplam (LMI070) developed by Novartis, SRK-015 developed by Scholar Rock, and Reldesemtiv jointly developed by Cytokinetics and Astellas have all entered phase 2 clinical trials.
Stage
.

In China, the research and development of rare disease treatment drugs by local pharmaceutical companies is still in the exploratory stage
.
The rare disease drug research and development enterprise matrix composed of Beihai Kangcheng, Inno Pharmaceuticals, Shufang Pharmaceuticals, and Beijing Kexin Bicheng, has begun to take shape
.
In the future, as the research and development of rare diseases continues to advance, it is believed that patients with rare diseases including SMA will usher in new treatment options and experiences
.

Reference materials:

[1]https://med.
sina.
com/article_detail_103_2_87035.
html

[2]https://xw.
qq.
com/amphtml/20210906A066CS00

[3]https://