Opportunities for pharmaceutical companies to introduce heavy policies

The third child is here, is the spring of children's medicine far away?

01 Low R&D willingness of manufacturers

The General Office of the National Health Commission, the General Office of the Ministry of Industry and Information Technology, and the General Department of the National Food and Drug Administration issued three batches of lists of children's drugs that encourage R&D and application in May 2016, May 2017, and July 2019, respectively

Now, halfway through 2021, CDE's report in June this year mentioned that 12 products that encourage R&D and application for children's drugs have completed technical review and have been approved for marketing, and 11 applications for marketing applications that encourage R&D and application for children's drugs are under review.

This means that only 28 of the 106 children's drug lists that encourage research and development have started research.

Among the currently available products on the market, there are mainly two domestically approved products

One is an improved new drug: the second-class improved new drug chloral hydrate/syrup combination package approved for marketing at the end of March 2021 is the first batch of varieties in the list of children's drugs to encourage research and development.

For many years, China has only this variety of medical institution preparations, and lack of marketed drugs.

The other is a generic drug: Levetiracetam concentrated solution for injection is in the first batch of products that are in the first batch of children's medicines that are encouraged to develop and declare and has been collected.

More approved drugs are imported rare disease drugs

Bosentan tablets approved in 2017 are used to treat pulmonary hypertension in patients with WHO stage III and IV primary pulmonary hypertension, or pulmonary hypertension caused by scleroderma

The import registration application for eculizumab injection approved in 2018 is used to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children with rare diseases

In August 2020, Astellas China officially approved tacrolimus granules for marketing.

On April 30, 2021, Takeda velaglucerase alpha for injection was approved by the National Medical Products Administration of China for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease

In June 2021, Aucta's vigabatrin powder for oral solution was approved.

02 Children's Drug Development Challenge

The new national regulations have been encouraging the marketing of children's drugs: since the implementation of the newly revised "Drug Registration Management Measures" on July 1, 2020, review resources have further returned to clinical urgent needs, in accordance with the "Priority of Drug Marketing Authorization" issued by the State Food and Drug Administration.

To this end, the Center for Drug Evaluation has opened up an independent review channel for children's drugs, established a special label for "children's drug", and proactively served in communication, acceptance, review and other links to improve the efficiency of variety processing and ensure that high-quality children's drugs are listed as soon as possible

However, the phenomenon of long R&D cycle, high cost, low profit, and insufficient R&D motivation of enterprises for children’s drugs has not yet been completely changed

In the final analysis, there are the following considerations:

1.
R&D is difficult

Compared with adult preparations, the selection of excipients and their safety, young animal experiments, and breakthroughs in key technologies for children's drug research are faced with considerable challenges in the research and development of children's drugs
.
The return on investment of enterprises is not high, which objectively hinders the research and development of children's medicines for a long time
.
Moreover, clinical research on children's drugs is very difficult, and the recruitment, safety and benefit protection of children are far more difficult than adults
.

There are many preparations in pediatric clinics that have been used by medical institutions for many years, with excellent curative effects and widely praised.
However, due to the high cost of application and registration, it has been difficult to achieve conversion
.
After the Drug Approval Center releases technical documents supporting the development of children's drugs with real-world evidence, hospitals can collect past clinical application data and use real-world data research results to bring medical institution preparations to the market
.
Many companies want to be exempt from clinical research when developing children's drugs.
At present, at least real-world data research needs to be completed.
Then the development cycle will be at least 3 years longer.
Even with policy support, it is impossible for so many companies to declare at once
.
After all, real-world research-related policies have only officially begun to land in 2020, and the supporting documents have not been able to support them
.

2.
The birth rate of children has fallen

The birth rate of children has fallen sharply in recent years.
Even if the state encourages three births, it is impossible for companies to see whether the birth rate of newborns will increase in the short term.
Under the general trend of market size decline, there is no evidence to support parents willing to spend more.
Money is on children's drugs, and companies have concerns about the growth of the market for children's drugs
.

Three, children's medicine will also enter the collection

The encouraged drugs are mainly oral solutions.
At present, the medical insurance department is studying the linkage of centralized procurement of drugs with the same medication route
.
If the price of oral solution and ordinary tablets are linked, and they are not grouped separately, basically the price will not look good
.
Unless products with new indications for children can be bid separately in groups
.
However, if generic drugs are on the market, generic drugs will also receive new indications for children, and centralized procurement will still face price cuts
.

Under such an unclear bidding policy, companies are even less confident to invest in research and development
.

summary

If you wait for the implementation of the policy and then grab the project, it may be over-invested.
Companies that want to invest in children's medicine can make arrangements in advance.
After all, if R&D is accelerated to the market, it will take at least 4 years for a project
.

Attachment: List of three batches of children's drugs to encourage research and development

Note: Oral liquid preparations include: oral solutions, oral suspensions, oral emulsions, and oral drops
.