Progress in rare disease drug research in China

Due to the low number of rare disease patients, the cost of research and development is high, and from an economic point of view alone, the research and development of rare disease drugs (also known as "orphan drugs") is not cost-effective

On May 22, 2018, the "First Batch of Rare Diseases" jointly released by the National Health Commission, the Ministry of Science and Technology and other five ministries and commissions, involving a total of 121 rare diseases

China has approved the listing of 74 rare disease drugs, accounting for 26% of the world's 282 listed rare disease drugs, China is in clinical phase III and I period of 49 and 41 respectively, clinical phase I, phase II, phase III rare disease drugs in research 41, 36 and 49, accounting for 10%, 6% and 20%

1.

The company with the largest number of rare disease drugs in the world is Novartis, Switzerland, with a total of 138 rare disease drugs under research, accounting for 65% of the total number of 213 new drug research and development, followed by Squibb and Pfizer, with 108 and 98 rare disease drugs developed respectively, accounting for 64% and 58% of the total number of drugs developed by it, and Roche and Sanofi each have 96, accounting for 48% and 64%

Among the top 20 companies, the smallest proportion is Eli Lilly, which develops less than one-third (28%) of the total number of new drugs developed and is developed for a rare disease, in stark contrast to Novartis's huge high proportion of 65%, followed by Merck and Otsuka, Japan, accounting for only 35% and 41%,

China also has a company among the top 20, that is, BeiGene, with a total of 36 rare disease drugs in research, ranking 19th, accounting for 55%

Although the economic return is not the most cost-effective, the proportion of rare disease drug research and development in the world's leading pharmaceutical companies is relatively high

2.

If divided by the drug ingredients involved, there are 4 companies in the world that have developed more than 70 rare disease drug ingredients, namely Pfizer, Sanofi, Roche and Takeda, involving 77, 77, 76 and 74 rare disease drug ingredients

The largest number of rare disease drug components involved in China's new drug research and development is Beihai Kangcheng, with 10, Hengrui and Aimefei Bio have 7, and Wuhan Minglai Technology and Shenzhen Immunogene Therapy Research Institute have 5 each

1.

Obtaining orphan drug certification qualification is an important channel

From 2014 to 2020, China's pharmaceutical companies have won a total of 66 FDA orphan drug qualification certifications, of which 37 have been awarded in 2020, which is more
than the sum of previous years.

In recent years, with the increasing pressure of medical insurance control policies such as medical insurance negotiations and volume procurement, the pressure on innovative pharmaceutical companies has also increased, and they intend to resolve by accelerating the going to sea, and it has become popular
to strive for orphan drug qualification from the US FDA.
(See Figure 4 for details)

2.
Top 20 enterprises

In 2020, more than 20 enterprises in China have obtained the FDA orphan drug certification qualification in the United States, of which Yasheng Pharmaceutical, led by Dr.
Yang Dajun, has won 8 orphan drug qualification certifications with 4 drug sacs and has become the biggest winner, and BeiGene Shenzhou and Junshi Bio have ranked second
with 4 and 3 certifications respectively.
CStone Pharmaceuticals and Corning Jereh each received 2 certifications
.
(See Figure 5 for details)

.
PART

Priority review and approval

In December 2017, the former State Food and Drug Administration issued the Opinions on Resolving the Backlog of Drug Registration Applications and Implementing Priority Review and Approval, which clarified that drug registration applications for the treatment of rare diseases will be given priority review and approval
.

In May 2018, the Announcement on Matters Related to the Optimization of Drug Registration Review and Approval issued by the State Food and Drug Administration and the Health and Construction Commission pointed out that for rare disease drugs that have been listed overseas, if the applicant believes that there is no racial difference after research, it can submit the clinical trial data obtained abroad and directly apply for listing
.

In July 2020, the State Administration for Market Regulation issued the newly revised Measures for the Administration of Drug Registration, which stipulates that when applying for marketing authorization for innovative drugs for rare diseases, it may apply for preferential review and approval procedures and give policy support
for review time limits.

Many rare disease drugs have greatly accelerated the speed
of review and approval by including them in the priority review and approval process.
The number of rare disease drugs included in the priority review and approval procedure increased from 8 in 2016 to 28 in 2018 and 2019, an increase of 2.
5 times, and decreased slightly in 2020 to 21, but the proportion of the total number of drugs included in the priority review and approval increased year after year, from 4.
1% in 2016 to 14.
6% in 2021, an increase of 10 percentage points in 5 years
.
(See Figure 6 for details)

In addition to speeding up the review and approval, the annual national medical insurance negotiations also care for rare disease drugs, and China's rare disease drug research and development will also accelerate and maintain rapid development
.