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The release of the "36 Innovative Opinions" in October 2017 gives us hope for the development of orphan drugs in China, which is an important milestone
in the development of Chinese medicine.
In the past five years, the National Health Commission has released the first catalogue of rare diseases in China, including a total of 121 rare diseases, among which:
58 therapeutic drugs for 29 rare diseases have been included in the national medical insurance list
.
In addition, many rare disease drugs have been included in the priority review and approval process, from eight in 2016 to 28 in 2018 and 2019
.
More than two years ago, the State Administration for Market Regulation revised the Measures for the Administration of Drug Registration, which stipulates that when applying for marketing authorization for innovative drugs for rare diseases, it can apply for priority review and approval procedures and give policy support
to the review time limit.
In 2022, the NMPA issued four guiding principles related to "patient-centered" drug research and development (draft for comments), a major obstacle in the development of orphan drugs is slow patient recruitment, a large amount of data shows that under the "patient-centered" concept, the clinical trial patient recruitment cycle has been shortened from the previous average of 30 months to about 7 months, and the possibility
of phase II and III trial drugs being approved.
This concept has become the core guiding ideology of current drug research and development, and it is believed that this will actively promote the clinical trials of orphan drug research and development in China
.
of phase II and III trial drug approval has also increased.
After five years of development, what is the current situation of orphan drug research and development in China, what problems are still facing China's orphan drug research and development, whether we have a solution to the problem of accessibility and affordability, and my expectations for the future of China's orphan drug research and development are all explained
in this article.
More than 60 gene therapy companies
More than 60 gene therapy companiesIt is the main force in the research and development of orphan drugs
It is the main force of orphan drug research and developmentIn recent years, with the increasing pressure of medical insurance fee control policies such as medical insurance negotiations and volume procurement, innovative pharmaceutical companies are under increasing pressure, and they have planned to accelerate their overseas expansion to resolve it, and seeking orphan drug designation from the US Food and Drug Administration (FDA) has also become a hot spot
.
Domestic pharmaceutical companies are very optimistic about obtaining orphan drug designation from the US FDA, and many companies will obtain orphan drug designation in the United States as a strategy for orphan drugs to go global, and they have made full use of various support policies for orphan drugs in the United States to speed up the review and approval of
drugs in the United States.
CANbridge has the largest number of rare disease drug components involved in China's new drug development, with 10, Hengrui and Emmefei Biologics have seven, and Wuhan Minglai Technology and Shenzhen Institute of Immunogene Therapy have five
each.
According to reports, from 2014 to 2020, Chinese pharmaceutical companies won a total of 66 U.
S.
FDA ORPHAN DRUG DESIGNATIONS, of which 37 were won in 2020, more than in previous years combined
.
For example, Ascentage Centage, Renfu Pharmaceutical, Esheng Biologics, Hengrui, BeiGene, CP Tianqing and other enterprises have obtained orphan drug qualification recognition for research and development drugs in the United States, among which Ascentage is the Chinese pharmaceutical company
with the largest number of orphan drug qualification certifications in the United States.
each.
These pharmaceutical companies take full advantage of the difference in the prevalence of certain diseases between the two countries, make full use of the US support policy for orphan drugs and the vast Chinese patient market, and enhance the commercial value
of the drug itself.
There are many types of rare diseases in the world, about 8,000, and there are only about 600 orphan drugs on the market, basically small molecules, enzyme replacement and antibody drugs, and only six gene
therapy drugs.
therapy drugs.
Since the launch of Spark's Luxturna for the treatment of black blindness in 2017 and Zolgensma for the treatment of SMA by Novartis in the United States in 2019, there has also been a boom of investment in the gene therapy track in China, and more than 60 gene therapy companies have been established in the past three years, most of them are developing drugs for rare diseases, mainly focusing on ophthalmology, hemophilia, thalassemia, Pseudohypertrophic muscular dystrophy (DMD) and spinal muscular atrophy (SMA) and other diseases with relatively high prevalence and more than 10,000 patients, a total of 23 drugs under development have entered the new drug clinical research registration (IND) or clinical trial stage
.
Gene therapy is mainly for 80% of rare diseases of single-gene genetic diseases, there are about 6,000 kinds, especially more than 1,000 are inherited metabolic diseases, with very good application prospects
.
The therapy could theoretically be cured with a single shot and was more affordable
than patients with expensive enzyme replacement drugs.
At present, there are less than 100 domestic companies involved in the field of rare disease drugs, more than 60 are gene therapy companies, but basically concentrated in ophthalmology and some rare diseases with relatively high incidence, for other rare diseases with an incidence of 1 in 50,000 to 1 in 100,000 or even lower, drug research and development companies are very rare
.
The reason for this phenomenon is mainly that the number of patients with known rare diseases in China is very small (no epidemiological data), and the investment in drug research and development is relatively high, and the price of these drugs after the market is too high to enter medical insurance, how to get the corresponding return is a problem
.
Drugs can be afforded to patients and rewarded by companies, which is really difficult to achieve
in the field of rare diseases in China.
.
The R&D expenses required to enter this field in China mainly come from investors, and investors need a return on investment
.
Enterprises should assume some social responsibility, but the biggest social responsibility should be to be able to survive and make more innovative drugs
sustainably.
How to achieve this balance, find a balance, solve the problem of access to rare disease drugs, and benefit all parties is worth all of us to explore
.
We believe that the internationalization and diversified payment system and the state's support for rare disease drug research and development enterprises in some policies and tax exemptions will promote the development of
this industry.
At this stage, China lacks professional talents in rare disease diagnosis, screening, basic research and drug research and development, and epidemiological investigation and natural history research also require a large number of talents to be deeply involved
.
We hope to have a "torch" that will light up the industry, so that patients, patients' families, doctors, R&D personnel and investors can be closely connected to form a "I have you, you have me" pattern
.
.
Orphan drug payments, behind the policy
Orphan drug payments, behind the policyWho pays for it remains a central issue
in China's orphan drug development.
The biggest problem facing China's innovative drug development, especially orphan drug research and development, is payment
.
Orphan drugs themselves are aimed at a niche population, and if the price is too low, companies and investment institutions will not enter the field
.
However, if the price is too high and the patient cannot afford to pay the high cost of the drug for a long time, if the payment problem is not solved, either the patient is faced with the situation of no drug available, or there is a drug but cannot afford it, that is, the problems of accessibility and affordability cannot be solved
.
.
China's orphan drug research and development is not short of talents, technology, and experts, in fact, it is necessary to have a good policy support, which is the core of
rare disease drug research and development and industrial development.
Innovation in the field of drug research and development is inseparable
from the government's policy promotion of rare diseases.
From a global perspective, the United States has more than 600 orphan drugs, which is the most mature orphan drug market in the world, which is undoubtedly related
to preferential policies and perfect incentive systems.
In 1983 and 2002, the United States successively introduced the "Orphan Drug Act" and the "Rare Disease Act", from the tax credit for the clinical research stage enjoyed by enterprises, the exemption of almost $2.
3 million in application fees for new drugs to be marketed, as well as various early R&D funding and FDA communication channels, to the market exclusivity enjoyed by 7 years after marketing, these are the main reasons for
the rapid development of orphan drug research and development in the United States.
from the government's policy promotion of rare diseases.
From a global perspective, the United States has more than 600 orphan drugs, which is the most mature orphan drug market in the world, which is undoubtedly related
to preferential policies and perfect incentive systems.
Many new drug technologies start from rare diseases, such as gene therapy, gene editing and mRNA technology, which will further enhance the innovation ability and level of drug research and development in China and bring benefits
to the majority of domestic and global patients with rare diseases.
In the past three years, more than 60 newly established biotechnology companies in China have been engaged in the research and development of gene therapy drugs, and the companies providing R&D OEM services for new technology platforms such as cell therapy, gene therapy and mRNA have grown, and the cooperation between the two sides has accelerated the research and development
of new drugs.
China has also successively introduced a number of policy measures to support the research and development of orphan drugs, promoting the development of rare diseases in terms of drug research and development
, registration and review, medical insurance reimbursement, patient registration and screening, and taxation.
In April 2018, the Implementation Measures for Data Protection of Drug Trials (Interim (Draft for Comments) was released, listing drugs for the treatment of rare diseases as data protection objects for the first time, and granting a data protection period of 6 years from the date of the indication first approval in China; In May of the same year, China's "First Batch of Rare Disease Catalogue" was released, involving a total of 121 rare diseases, which was the first time that China officially defined rare diseases in the form of a catalogue, which was regarded as the initial and most milestone policy node of rare diseases in China, indicating that the national level has made substantial progress
in promoting the prevention and control of rare diseases.
Two years ago, the updated Measures for the Administration of Drug Registration were issued, clarifying that the review time limit for overseas listed and unmarketed rare disease drugs that are urgently needed for clinical practice is 70 working days
.
In May this year, the Regulations for the Implementation of the Drug Administration Law (Draft Amendments for Solicitation of Comments) proposed that new drugs for rare diseases can have market exclusivity for no more than seven years under the condition of continuous supply
.
This is the first time that China has written the market exclusivity period for rare disease drugs into its
regulations.
The market exclusivity period guarantees the return on R&D investment of enterprises to a certain extent, which is equivalent to the patent compensation system, which is also in line
with the incentive system of developed countries such as the United States, Europe and Japan to support the research and development of orphan drugs.
with the incentive system of developed countries such as the United States, Europe and Japan to support the research and development of orphan drugs.
It can be seen that China's policy in the field of rare diseases has indeed made a very big breakthrough, but the most critical payment problem has not been properly solved
.
Patients with rare diseases cannot afford to face the high cost of
drugs.
Recently, the "sky-high drug" noxinaxen sodium has been talked about in the medical insurance directory, but this is only one of many rare disease drugs, and the high cost of most orphan drugs is still unbearable for many patient families, and it is also an important reason for
many families to be poor due to illness.
Even, orphan drugs from multinational pharmaceutical companies are facing withdrawal from the Chinese market because of high prices and few patients
.
For example, saphotrexate hydrochloride tablets (trade name "Kewang") is considering withdrawing from the Chinese market, which is an indispensable drug for some patients with hyperphenylalaninemia (HPA), and there is no generic drug on the market in the Chinese mainland market, once the drug is withdrawn, it means that these patients will have no drug available, which is a fatal blow
to patients and their families.
.
Faced with the problem of payment, Ding Jie of Peking University First Hospital proposed medical insurance for rare diseases at the "two sessions" for many years
The "1+4" model is to establish a multi-party payment and multi-level medical security mechanism (including commercial insurance) to completely solve the problem
of payment for high-value drugs for rare diseases.
Ding Jie believes that the construction of commercial insurance for high-value drugs for rare diseases can learn from some successful local experiences, such as the construction of commercial insurance under the guidance of the government for long-term, unlisted medical insurance, focusing on serious diseases or value-added orphan drugs, and strengthening the public welfare and affordability of such insurance, so as to reduce the economic burden of patients with rare diseases and reduce the pressure
on medical insurance expenditure.
At the same time, do a good job in the connection
between social security and commercial insurance.
Similar to Ding Jie's suggestion, during the "Two Sessions" in 2021, Lei Dongzhu, president of the First People's Hospital of Chenzhou City, Hunan Province, proposed a "1+X" multi-level medical security system
for rare diseases.
1, refers to the basic medical insurance, X, refers to serious illness insurance, public charity, social mutual aid, commercial health insurance, individual self-payment and other supplementary medical insurance to provide multi-level protection, to achieve the diagnosis and treatment of rare disease patients
.
The specific operation method proposed by Lei Dongzhu is to encourage policy-based commercial insurance and implement policies
such as insurance with illness and secondary reimbursement.
Realize individual self-payment capping, and the personal burden of rare disease patients is capped
at 80,000 yuan per year.
As an important part of the multi-level medical security system for rare diseases, commercial health insurance will have great prospects
in the future.
such as sickness insurance and secondary reimbursement.
In the absence of a unified national rare disease security system in the country, Ding Jie suggested that all provinces explore the drug security mechanism for rare diseases according to the situation of rare disease patients and medical insurance funding conditions in their provinces, pilot the provincial medical insurance coordination, and gradually promote the realization of national medical insurance coordination after the provincial medical insurance coordination experience is mature
.
.
With the continuous attention of the society, the continuous blessing of policies, and the continuous improvement of the system, it is believed that China's orphan drugs will usher in a rapid development in the future, and will also become a key area for
Chinese pharmaceutical companies to seek innovation and development.
It is hoped that the recommendations of the two experts will be realized as soon as possible and the problem
of orphan drug payment will be completely resolved.
China's orphan drug research and development is in the ascendant, and in recent years, the state has successively introduced many policies and regulations to support the research and development of orphan drugs, from the central to the local level, are trying to explore ways to solve the payment of
high-value drugs.
With the continuous development and commercialization success of cell therapy, gene therapy and mRNA technology, we have more and better ways to do rare disease drug development
.
If gene therapy can be cured with one shot, patients do not need long-term medication, which can reduce the burden
on society and family.
China's orphan drug research and development must take the road of internationalization, first of all, choose the latest technical means to develop drugs for rare diseases or indications for which there are no therapeutic drugs in the world; In addition, patient-centered, the establishment of a rare disease drug research and development ecology is very important for orphan drug research and development, through the creation of ecology, we can better carry out investigator-initiated clinical trials, can also better carry out patient-centered clinical trials, improve the success rate of drug research and development, reduce the cost of drug research and development, and ultimately make effective drugs benefit rare disease patients
as soon as possible.
"The road is long and long, I will seek from top to bottom", China's orphan drug research and development is very difficult, need strong support from all walks of life, not to insist on it when there is hope, but to persist to have hope, let us work together to make our due contribution
to China's rare diseases and orphan drug cause.
