Orphan drugs will account for 1/3 of the global drug pipeline value, how will domestic research and development go?

Previously, Evaluate released the "2022 Orphan Drug Report" report, showing that the compound annual growth rate of the orphan drug market from 2021 to 2026 is 12%, and the growth rate is twice that of the non-orphan drug mark.

Image source: Evaluate "2022 Orphan Drug Report"

According to Frost & Sullivan's analysis, the market size of orphan drugs in China in 2020 is approximately US$3 billion, accounting for approximately 1% of the global orphan drug market si.

Figure 1 Market size of rare disease drugs in China (2016-2030E, USD 100 million)

(Data source: Frost & Sullivan analysis report)

According to the "2022 China Rare Disease Industry Trend Observation Report" jointly released by Frost & Sullivan and Beijing Pain Challenge Public Welfare Foundation, there are about 20 million rare disease patients in China, and the number is growing rapid.

Favorable policies work together to promote the domestic orphan drug market

Therefore, in recent years, China has successively introduced various types of supporting policies related to rare disease diagnosis, drug development and registration, medical security, taxation, e.

In October 2017, the General Office of the Central Committee of the Communist Party of China and the General Office of the State Council issued the "Opinions on Deepening the Reform of the Review and Approval System to Encourage the Innovation of Drugs and Medical Devices", proposing to support the research and development of drugs and medical devices for the treatment of rare diseas.

In May 2018, five departments including the National Health and Health Commission, the Ministry of Science and Technology, the Ministry of Industry and Information Technology, the State Food and Drug Administration, and the State Administration of Traditional Chinese Medicine jointly released the first batch of rare disease lists including 121 rare diseas.

In 2020, the newly revised "Measures for the Administration of Drug Registration" clarified that the review time limit for rare disease drugs that are urgently needed in clinical practice is 70 da.

In 2020, the "Opinions on Deepening the Reform of the Medical Security System" issued by the Central Committee of the Communist Party of China and the State Council clearly proposed to improve a fair and appropriate treatment security mechanism and explore a drug security mechanism for rare diseas.

In January 2022, the Center for Drug Evaluation of the State Food and Drug Administration issued the "Technical Guidelines for Clinical Research and Development of Drugs for Rare Disease.

The "Regulations on the Implementation of the Drug Administration Law of the People's Republic of China (Draft for Comments)" issued in May 2022 clearly stated that "for new rare disease drugs approved for marketing, if the drug marketing license holder promises to ensure the supply of drugs, the maximum A market exclusivity period of no more than 7 years, during which the same varieties are no longer approved for listi.

In addition, the national fiscal and taxation policies are also supporting the research and development of rare disease dru.

Domestic innovative drug companies focusing on orphan drugs have emerged

The difference between the patient population base and the market size proves that the domestic orphan drug market still has huge development space and opportuniti.

According to statistics, from the perspective of the whole cycle from R&D to marketing, the success rate of rare disease drugs from clinical phase I to marketing approval is 10%, while the success rate of R&D for all diseases is 9%, which is only half of rare disease dru.

Top 10 best-selling orphan drugs in the world in 2026

Image source: Evaluate "2022 Orphan Drug Report"

In 2012, Sanofi China R&D Center and China Pharmaceutical Industry Research Institute signed a memorandum of cooperation to jointly conduct the first joint research on diagnosis and treatment of rare diseases in Chi.

Image source: Evaluate "2022 Orphan Drug Report"

At the same time, many domestic innovative drug companies have also entered the field of innovative drug research and development for rare diseases, such as Weisheng Pharmaceutic.

In addition, biopharmaceutical companies focusing on the field of rare diseases have also emerg.

Where is the future of the domestic rare disease diagnosis and treatment market?

With the continuous improvement of domestic orphan drug policies and the continuous influx of capital, the following problems still exist:

The patient population is large, but the diagnosis rate is low

       Rare diseases generally have complex symptoms and low morbidity/prevalence, resulting in a low level of understanding of rare diseases among clinicia.

In addition, most rare diseases are caused by genetic mutations, and the diagnosis of rare diseases generally requires genetic testi.

Medical institutions with rare disease diagnosis technology and capabilities are mainly concentrated in the top three hospitals in cities such as Beijing, Shanghai, Guangzhou and Shenzh.

It is difficult for patients in tier cities to be diagnosed due to the lack of relevant clinical knowledge reserves and diagnosis and treatment techniques in medical institutio.

According to data from the China Rare Disease Alliance, more than 42% of rare disease patients have been misdiagnosed, and it takes an average of 26 years to be diagnos.

The lack of clinical experience of rare diseases by clinicians, uneven distribution of medical resources, high misdiagnosis rate and long diagnosis cycle have all hindered the development of domestic orphan dru.

       Strong import dependence

       According to statistics, of the 121 rare diseases in the "First Batch of Rare Diseases List", 86 rare diseases have therapeutic drugs in the world, of which 77 rare diseases have therapeutic drugs in China, and 9 rare diseases still face drugs available overseas and domestic Drugless predicame.

The research and development of orphan drugs often requires more capital investment, technical support and supporting policies than ordinary innovative dru.

At present, the supporting policies for rare diseases in China are constantly improving, but the research and development of technology and capital investment still need to be improv.

Therefore, from drugs for rare diseases From the perspective of the field, there is still the problem of strong import dependen.

       Access to medication remains low for patients

       In the past two years, China has continuously negotiated medical insurance to reduce the price of high-value drugs for rare diseases and improve the availability of drugs for patien.

As mentioned at the beginning of the article, the annual treatment cost of Nosinagen Sodium Injection has dropped to 99,000-198,000 yuan per ye.

But this is still not enou.

There are still 9 high-value drugs with an annual treatment cost of more than 500,000 that have not been included in the medical insurance li.

Although these drugs have been approved for marketing in China due to lack of medical insurance, there are still no drugs available for patien.

Sexuality remains unsatisfi.

       To sum up, the domestic rare disease diagnosis and treatment market has entered a period of rapid developme.

We should strengthen relevant education for clinical researchers as soon as possible, establish and improve a service system for the needs of patients with rare diseases throughout the life cycle, explore multiple payment mechanisms for medical insurance, and further promote domestic rare diseas.

The industry is developing rapid.