Children need to use children's medicines, the 100 billion market is waiting to explode

In recent years, the serious shortage of children's medicines has attracted great attention from relevant government departments
.
According to the seventh census, there are 253 million children in China, accounting for 18% of the total population
.
With the country's comprehensive opening of second and third children, the demand for children's medical care has also continued to rise, and the number of consultations has increased by 4 million to 5 million per year, accounting for more than 10% of the total number of outpatient clinics in the country
.
The other end of the strong demand is the weak supply, which is mainly reflected in the shortage of children's medicines and the lack of research and development motivation of enterprises
.
According to a survey by the All-China Federation of Industry and Commerce Pharmaceutical Chamber of Commerce, as of September 2019, among the more than 6,000 pharmaceutical factories in China, only more than 10 specialized in the production of children's medicines, and only more than 30 companies had children's medicine production departments
.
In terms of varieties, among the conventional drugs, there are more than 3,600 kinds of prescription drugs for adults, and only more than 60 kinds of drugs for children
.
The research and development of children’s medicines is also very backward.
As of January 1, 2021, China has recorded a total of 228,448 approval numbers for marketed products, of which the total number of approval numbers for paediatric drugs (by children, paediatrics, pediatrics, etc.
) is 3,826, accounting for 3,826.
than only 1.
7%
.
In order to solve this problem, the state has plans to encourage the production and development of children's medicines.
The professional development of children's medicines has become a trend, and the 100 billion market is waiting to explode
.
The release of the dividends of the medical insurance policy gives priority to approval and innovation.
On October 25, 2018, the National Health Commission issued the "Notice on Printing and Distributing the National Essential Medicines List (2018 Edition)", and 685 varieties were shortlisted
.
The 2018 edition of the Essential Medicines List focuses on revisions to the categories, varieties, dosage forms and specifications of children's medicines, and adds 22 new types of medicines for children that are urgently needed in clinical practice, including 3 pediatric chemicals and 13 proprietary Chinese medicines
.
In terms of indications, it mainly includes anti-infective drugs, respiratory system drugs, and digestive and gastrointestinal drugs
.
Figure 1 Children's medicines added to the 2018 Essential Drug List The revision of the 2018 Essential Drug List closely fits clinical needs and the characteristics of childhood morbidity
.
The prevalence of respiratory diseases in children ranks first, among which cold, fever, cough, bronchitis, asthma, pneumonia and other diseases are the most common
.
The second type of disease is children's digestive system diseases, the most common types of diseases include indigestion, children's diarrhea, gastroenteritis and so on
.
These two categories are the main components of medicines for common diseases in children, and they also account for the largest proportion of prescription medicines for children
.
On August 27, 2019, the National Health and Medical Commission issued the "Third Batch of Drugs for Children Encouraged for R&D and Application".
There are 37 drugs in the list.
In addition to the above-mentioned drugs for common diseases in children, such as aspirin and metronidazole, this list contains more Cardiovascular system drugs, sexual development drugs, nervous system drugs and rare disease drugs
.
Figure 2 The third batch of children's drug list encouraged to develop tetrahydrobiopterin (BH4) for the treatment of phenylketonuria, patients with a congenital defect of an enzyme in the BH4 synthesis or metabolism pathway, resulting in phenylalanine metabolism disorders, appear Severe neuropsychiatric damage
.
On May 11, 2018, the disease was included in the "First List of Rare Diseases" jointly formulated by the National Health Commission
.
Merck's Kewang is a BH4 replacement therapy drug.
Domestic clinical studies have shown that for children with BH4 deficiency, the clinical symptoms of children with BH4 deficiency improved after corresponding oral treatment, especially for children who were treated in time within 3 months.
Intellectual development is close to that of normal children
.
Timolol is safe in the pediatric population and has been used by pediatric ophthalmologists as a first-line treatment for glaucoma for more than 30 years
.
Studies have confirmed that the topical use of timolol maleate eye drops in the treatment of infantile hemangioma is safe, effective and inexpensive.
lesser) one of the first-choice medications
.
At present, there is no commercial timolol gel preparation in China.
The commercially available timolol maleate eye drops are aqueous preparations, which are inconvenient to use and difficult to control the dosage.
Long-term use will cause dryness and peeling of the skin.

.
In contrast, it is not difficult to see that the newly added children's drugs in the 2018 Essential Drug List are to meet basic clinical needs.
On this basis, phenylketonuria, Duchenne muscular dystrophy, infant vascular Drugs for tumors, precocious puberty, epilepsy and other diseases are mainly based on checking and filling gaps
.
As the future and hope of the motherland, every small group of children cannot be ignored
.
The "Third Batch List" reflects the areas of children's diseases with urgent clinical needs.
The problem of drug accessibility needs to be improved urgently, and there is great potential for enterprises
.
Real-world evidence helps R&D accelerate the transformation of market value‍‍‍ It is included in the medical insurance catalogue and included in the priority review and approval
.
However, there is still a practical problem in the development of children's drugs.
Compared with adult trials, children's clinical trials are often difficult to carry out, resulting in insufficient evaluation of the efficacy and safety of drugs used in children
.
In response to the practical challenges faced by enterprises, the State Drug Administration issued the "Technical Guidelines for Real-World Research to Support the Development and Approval of Children's Drugs (Trial)" in August 2020
.
As a new research method, Real-World-Research/Study (RWR/RWS) has been gradually used to support the research and development and review of children's medicines, for the registration of new medicines, the expansion of children's indications, and the improvement of children's dosage regimens.
etc.
to provide support
.
Real-world evidence is not simply data collection, but reliable data obtained in real medical environments.
As the "Guiding Principles" point out, children's drug research and development should always focus on one core, which is to provide research evidence for children's clinical rational use of drug use.

.
Based on this, the "Guiding Principles" summarizes five situations in which real-world evidence is commonly used in children's drug research and development: (1) Long-term drug use risk monitoring, such as the impact of drugs on children's growth and development; (2) To meet children's diverse needs (3) A large amount of data has been accumulated in clinical practice, and it is expected to provide sufficient evidence for specific issues, such as safety and efficacy data on off-label doses, data on concomitant drugs, etc.
; (4) The pediatric dose determined by the extrapolation model, when the extrapolation reliability is high and the safety risk is small, can be used as the verification evidence for approval or conditional approval; (5) It is difficult to independently carry out RCT or a certain age group in a certain age group Populations unable to participate in key RCTs, such as preterm infants, neonates,
etc.
At present, providing safety evidence is the most common application mode of real-world evidence in children's drug development, in addition to supporting drug regimen optimization: such as expanding or narrowing the applicable population (such as expanding to younger children), optimizing the dose or frequency of administration ( Such as refining the dose according to the kg body weight), improving or modifying the administration operation or process (such as taking different types of fruit juices, jams, etc.
),
etc.
The "Guiding Principles" provide companies with the scope and timing of real-world research in children's drug research and development.
By organically combining real-world evidence with traditional clinical trials, the two "complement and support each other", and companies can effectively save money Children's research resources to improve data quality and research efficiency
.
As the shortage of children's medicines has received great attention and the orderly implementation of the two-child and three-child policies, the market demand for children's medicines in China has continued to increase
.
The industry predicts that China's children's drug market will exceed 200 billion yuan in 2022
.
At the same time, the supply-side reform and transformation and upgrading of the children's drug market will also usher in an outbreak
.
Postscript: Traditional clinical trials and real-world research are in a “complementary and supportive” relationship, and in new drug marketing applications, real-world evidence cannot replace traditional clinical trials
.
Therefore, for children's drug research and development, companies can only continue to innovate and develop sustainably if they do a good job of traditional clinical trials
.