Policy dividend release Breakthroughs have been made in the field of rare disease drugs

From September 14 to November 3, the food and drug public opinion monitoring system of China Health Media Group monitored mainstream media, national portals, Weibo, WeChat and other platforms and found that the topic of "rare disease drug clobazhan" received more attention from public opinion, and the relevant public opinion was hot
.

 

Event overview

 

In order to further improve the drug supply guarantee policy and meet the specific clinical needs of the people, on March 29, the National Health Commission publicly solicited opinions
on the "Work Plan for the Temporary Import of Urgently Needed Clinical Drugs" (Draft for Comments) and the "Work Plan for the Temporary Import of Clobazhan" (Draft for Comments).
On June 29, the National Health Commission and the State Food and Drug Administration officially jointly issued the above two plans
.
Among them, the "Work Plan for the Temporary Import of Chlorbazhan" clarifies that the National Health Commission shall organize the clinical demand for clobazam, determine the list of medical institutions to be used, select the medical institution leading the import, and the medical institution that leads the import will submit an application for temporary import to the State Food and Drug Administration, and directly go through the customs clearance procedures
with the import permit.

 

After that, the domestic pharmaceutical field also ushered in good news
.
On September 19, the State Food and Drug Administration approved the listing
of clobazam tablets declared by Yichang Renfu Pharmaceutical Co.
, Ltd.
(hereinafter referred to as Yichang Renfu Pharmaceutical).
In October, the drug was first listed on the Shandong Provincial Pharmaceutical Equipment Centralized Procurement Platform through the green channel
.
At the same time, the import and clinical application of original research drugs have also made progress
.
On September 22, the original drug clobazhan, which was temporarily imported as a clinically urgent drug, issued the first prescription
in the country at Peking Union Medical College Hospital.
Since mid-September, topics related to the price of clobazam, drug purchase channels and medical insurance policies have become the focus
of public attention.

 

Public opinion trends and key nodes

 

Taking September 14 to November 3 as the monitoring period, the attention of the media and netizens is shown in the figure, showing a "wave-like" characteristic
.

 

The first stage: Yichang Renfu Pharmaceutical's clobazam tablets were officially approved for marketing; Peking Union Medical College Hospital issued the country's first prescription for clobazam, which attracted attention

 

On September 19, the State Food and Drug Administration issued a drug approval certificate to receive information, showing that the clobazam tablets declared by Yichang Renfu Pharmaceutical were officially approved for marketing
.
It is reported that Yichang Renfu Pharmaceutical obtained the project approval for the development of clobazam tablets and began research and development in 2018, applied for clinical trials in November 2020, obtained clinical trial approval in February 2021, and obtained priority review and approval qualifications
in May 2022.

 

On September 20, the stock bar "Renfu Pharmaceutical Bar" published the article "600079: Renfu Pharmaceutical's Announcement on the Obtaining the Drug Registration Certificate of Clobazam Tablets", saying that Yichang Renfu Pharmaceutical, a holding subsidiary of Renfu Pharmaceutical Group Co.
, Ltd.
, recently received the drug registration certificate
of clobazam tablets approved and issued by the State Food and Drug Administration.
Clobazam tablets can be used for the treatment of refractory seizures in children, and are suitable for the combination treatment of seizures in patients with Lennox-Gastaut syndrome (LGS) aged 2 years and older, and there is currently no approval for marketing of this active ingredient in China
.
On the same day, the WeChat public account "Yichang Renfu Pharmaceutical" published the article "The first domestic company - Yichang Renfu Pharmaceutical Company clobazam approved for listing", saying that in response to the demands of parents of children, the company is ready to start applying for clobazam to enter the national medical insurance catalog in 2023, and will prepare to cooperate with third-party public welfare funds to provide drug assistance
for patients from economically disadvantaged families.

 

On September 22, CCTV news channel "Eastern Time and Space" broadcast "Temporary import policy of urgently needed clinical drugs landed, "life-saving drug" chlorbazan issued the country's first prescription today" said that with the joint efforts of the National Health Commission, the State Food and Drug Administration and other departments, as the original research drug temporarily imported for clinical urgent drugs, once known as the "life-saving drug", chlorbazan issued the first prescription
in the country in Peking Union Medical College Hospital.

 

At this stage, public opinion mainly focuses on the following four aspects
.

 

The first is to pay attention to the price and purchase channels
of imported chlorbazam and domestic chlorbazhan.
According to the article "Epilepsy drug clobazam officially approved for admission to the hospital at 317 yuan per box" published by the Economic Observation Network, on September 22, the relevant doctors of Peking Union Medical College Hospital confirmed that the imported clobazam had officially entered the hospital on the same day, the price per box was 317 yuan, and the drug specification was 10 mg × 100 tablets / box
.

 

According to the article "Domestic clobazam approved: parents of children have been waiting for drugs for a long time, pharmaceutical companies estimate that they can buy them after October" published by The Paper said that Yichang Renfu Pharmaceutical sales staff introduced that it is expected that domestic clobazam tablets
can be purchased in designated hospitals after October.
On September 21, a parent of a group of children with rare epilepsy said that for parents, the approval of domestic clobazam is good news, but it may take some time from the approval of the drug to the prescription of the hospital, and everyone is waiting
.

 

According to the article "Chlorbazam Original Research Drug Goes on the Market, First Generic Approved, Rare Disease Drugs Will Welcome Policy Dividends" published by the Beijing News said that from the list of medical institutions that plan to take the lead in importing and using clobazam, there are 50 hospitals, including 6 in Beijing, including Peking Union Medical College Hospital of the Chinese Academy of Medical Sciences, Peking University First Hospital, Peking University People's Hospital, Beijing Children's Hospital affiliated to Capital Medical University, Beijing Tiantan Hospital affiliated to Capital Medical University, and General Hospital of the People's Liberation Army Chinese People's Liberation Army
.

 

The second is to pay attention to the dilemma
of rare disease medication.
For example, the article "Alleviating the difficulty of medication for children with epilepsy, the first domestic imitation cloba has been approved for marketing, rare diseases have few drugs and the dilemma of drug efficiency is still to be solved" said that the dilemma faced by rare disease drugs mainly includes three aspects
.
First of all, at the access level, there are still some rare disease drugs that have not been marketed
in China.
Secondly, in terms of payment, compared with common diseases, rare disease drugs have less medical insurance coverage, lower medical insurance reimbursement ratio, heavier economic burden on patients, and different regional protection levels
.
Finally, in terms of diagnosis and treatment, the prognosis and treatment effect of different individuals vary greatly, and it is more difficult
to establish disease-specific, sensitive, and stable clinical and patient outcome studies compared with common diseases.
Some industry insiders suggested that China can learn from the experience of foreign laws and regulations to systematically regulate the research and development, introduction, production, supply, guarantee, market supervision of rare disease drugs, and further promote the innovative research and development and introduction and marketing
of rare disease orphan drugs.

 

Third, it is believed that policy support can effectively improve the accessibility of
drugs for rare diseases.
For example, the article "Domestic clobazam tablets launched in October: consistency evaluation improves the accessibility of rare disease drugs" published by 21 Finance and Economics said that after the implementation of China's consistency evaluation policy for the quality and efficacy of generic drugs, domestic enterprises accelerated the research and development and consistency evaluation of generic drugs, and quickly promoted the rapid entry of drugs that have been proven to be effective and safe in the world into the domestic market
.
Due to the low incidence and small number of patients, many companies in the field of rare disease treatment have insufficient motivation to invest in the research and development of new drugs, resulting in low frequency of drug replacement, and many patients even face the dilemma
of no drugs available.
The implementation of relevant policies for consistency evaluation is conducive to promoting the research and development of drugs for the treatment
of rare diseases.

 

According to the article "More "life-saving drugs" on the way, the dilemma of rare disease drugs will be effectively alleviated" published by the China Times, industry insiders said that in recent years, the global rare disease drug market has developed rapidly, and China has also successively introduced policies such as reducing import tariffs for rare disease drugs, accelerating the review and approval of rare disease drugs, and including rare disease drugs into medical insurance, constantly stimulating the research and development momentum of domestic pharmaceutical companies to solve the drug problems
of rare disease patients.
With policy support, more and more domestic pharmaceutical companies have begun to accurately deploy pipeline research and development
related to rare disease drugs.
At the same time, in order to improve the accessibility of rare disease drugs, it is recommended that relevant departments accelerate the establishment and improvement of the payment system for rare disease drugs, include relevant drugs in the scope of medical insurance reimbursement, and combine commercial medical insurance to alleviate the economic pressure
of rare disease treatment.

 

The fourth is to pay attention to the research and development of generic drugs of
clobazam in China.
For example, the article "The first domestic generic anti-epileptic drug clobazam approved" published by "Time Weekly", Renfu Pharmaceutical responded: it is expected to be listed within this year, and it is expected to enter medical insurance in 2023" said that at the end of 2021, 8 companies have been approved to develop clobazam
in accordance with relevant regulations.
In addition to Yichang Renfu Pharmaceutical, the research and development speed of Jinan Kehui Pharmaceutical Technology Co.
, Ltd.
is also relatively high
.

 

On the Weibo platform, topics such as "China's first orphan drug against rare and refractory epilepsy approved" and "life-saving drug clobazan issued the first prescription in the country" have aroused heated discussions among netizens, with nearly 6 million views
.

 

The second stage: domestic clobazan took the lead in listing on the centralized procurement platform of drugs and equipment in Shandong Province, and public opinion fluctuated slightly

 

On October 9, domestic clobazan took the lead in listing the net through the green channel on the centralized procurement platform of drugs and devices in Shandong Province, with a drug specification of 10 mg × 28 tablets / box, and the price of the net was 84 yuan
per box.
The news has aroused widespread media attention
in Shandong Province.

 

The article """Life-saving Drug" Domestic Clobazan Takes the Lead in Selling on the Net in Our Province" published in the "Qilu Evening News" said that domestic Chlorbazan was listed on the provincial centralized drug procurement platform to promote the drug dilemma of children with refractory epilepsy to be further alleviated
.
At present, all medical institutions in Shandong Province can purchase the drug
through the centralized procurement platform of drugs and devices in Shandong Province.

 

The headline "Shandong Business Daily Subap News" published an article entitled ""Life-saving drug" domestic chlorbazam is sold on the Internet in Shandong, local pharmaceutical companies' research and development has come to an end, and patients' families cry with joy: finally see the light" said that the listing of domestic chlorbazam will greatly reduce the burden
of family medication for children.
The research and development of clobazan of Shandong pharmaceutical company Jinan Kehui Pharmaceutical Technology Co.
, Ltd.
has also entered the end, and the company's staff said that it has overcome technical difficulties and entered the stage
of submitting review and approval materials.

 

The third stage: domestic chlorbazam was listed nationwide, which attracted great attention

 

On October 22, Yichang Renfu Pharmaceutical held a national listing conference
for clobazam tablets.
The article ""Life-saving drug" domestic chlorbazam officially listed, priced at 84 yuan a box" published by Qilu Evening News Qilu One Point said that this means that domestic chlorbazan will officially enter hospital pharmacies
.
At the press conference, the president of Yichang Renfu Pharmaceutical also said that in the future, the company will actively promote clobazam tablets to enter the national medical insurance catalog, and seek cooperation with public welfare funds to reduce the economic burden
of patients.

 

At this stage, public opinion mainly focuses on the following four aspects
.

 

First, it is believed that the rapid launch of clobazam generic drugs is the result of the joint promotion of
multiple departments.

 

The article "From appeal to landing, the year when the problem of cloba occupancy drug was solved" said that from appeal to landing, chlorba took less than a year
.
Kang Zhen, a professor at the School of Basic Medicine and Clinical Pharmacy of China Pharmaceutical University, said that the approval of domestic generic drugs and the listing of imported clobazam are almost synchronous, which shows that the relevant departments have responded faster and better to the voice of the public, and the National Health Commission, the State Food and Drug Administration and other departments have worked together to ultimately benefit patients
.

 

The article "Times Commentary "Life-saving Drug" Clobazam Marketed, Diseases Can Be Rare, Life First Will Not Be Rare" published by Qianjiang Evening News Hourly News said that the listing of domestic clobazam tablets has solved the medication problems of rare epilepsy patients
.
In order to build a higher quality and higher level of security of the national medical security system, the state has been working hard, rare disease "life-saving drug" domestic cloba accounted for the market, a series of "niche drugs" into medical insurance, reflecting respect for life, embodying the concept
of people first and life first.

 

Second, it is believed that relevant departments still need to increase their efforts in accelerating the research and development, production and marketing of rare disease drugs
.
For example, the article "Domestic clobazhan on the market, rare disease drugs must have "rare strength" released by the Beijing News, in addition to the obstacles in market transactions and drug circulation, the most fundamental reason is the scarcity of special drugs and the slow progress of research and development
.
Therefore, it is suggested to encourage the R&D and production of rare disease drugs by encouraging the transformation of achievements and issuing R&D subsidies, so as to deliver more high-quality and low-cost special drugs and life-saving drugs to patients with rare diseases
.

 

The article "Onion Quick ReviewAlleviating the difficulty of drug purchase for patients with rare epilepsy, Shandong has set an example" published by Qilu Evening News Qilu One Point said that it is necessary for relevant departments to increase their efforts in accelerating the research and development and marketing of rare disease targeted drugs
.
On the one hand, expand the types of rare disease targeted drugs, allow more rare disease targeted drugs to enter the market, and solve the problem of drug purchase for rare disease groups; On the other hand, it is necessary to increase policy output, not only to make drugs affordable for patients, but also to let pharmaceutical companies and R&D institutions see development opportunities, and prevent the situation of delisting relevant drugs after they are on the market
.

 

The third is to pay attention to the R&D layout
of rare disease drugs by domestic pharmaceutical companies.
For example, the article "Domestic clobazan listed pharmaceutical companies accelerate the layout of rare diseases" published in Shanghai Securities News said that the approval of clobazam is only one of the latest achievements of local enterprises in the field of
rare diseases.
With the support of policies, with the continuous influx of capital, many local enterprises have set foot in the field of rare diseases and made positive progress
.
For example, many pharmaceutical companies such as Remegen Biologics, Junshi Biologics, Hansen Pharmaceutical, Hengrui Pharmaceutical, CANbridge, Gan&Lee Pharmaceutical, Maibo Pharmaceutical, and Chia Tai Tianqing have made layouts
in the field of rare disease drugs.

 

The fourth is to pay attention to the prescription
of clobazam in hospitals in many places.
For example, the client of Yangtze River Daily and Polar News said in an article on October 28 that the temporary import of chlorbazam was prescribed in Wuhan Children's Hospital and the Children's Hospital Affiliated to Shandong University (Jinan Children's Hospital
).

 

On the Weibo platform, topics such as "the first domestic epilepsy life-saving drug clobazhan" and "the voices of families of children with domestic life-saving drug clobazhan" have aroused heated discussions among netizens, with more than 5 million views
.

 

Public opinion commentary

 

The launch of domestic drugs, the import of original drugs, the implementation of relevant policies and measures have won praise, and the clinical needs of patients with refractory epilepsy have been met
to a certain extent.
From foreign drugs that cannot be legally used in China, to the compliant circulation of foreign drugs in China, and then to the listing of domestic drugs, this series of changes has benefited from the promotion and escort
of policies.
The issuance of the "Work Plan for the Temporary Import of Urgently Needed Clinical Drugs" has opened up a green channel for the clinical use of drugs in similar situations in China, and also brought hope and convenience
to patients with rare diseases in similar situations.

 

In the future, it is believed that with the joint participation and efforts of relevant departments, industry-university-research institutions, and patient organizations, laws and regulations related to the use of drugs for rare diseases will be further improved, and relevant mechanisms will be further improved, so as to better meet the treatment needs
of rare disease groups.
(Zhang Miaomiao, Public Opinion Monitoring Center, China Health Media Group)