Pediatric drug market and cross-border cooperation trends (International Part 1)

Children make up about 26% of the world's population, but the value of paediatric drug development and marketing has long been grossly underestimated.
china's "comprehensive two-child" policy, environmental pollution and other social problems, the paediatric drug market continued to climb, in 2020 is expected to be 22.5 billion U.S. dollars.
, however, the country's more than 7,000 pharmaceutical production enterprises, specializing in the production of children's medicine only a dozen districts, children's drug dosage forms accounted for less than 2% of the total pharmaceutical preparations, there is a serious imbalance between supply and demand.
stage, more than 50% of pediatric drugs do not have clinical data reference, but doctors adjust the dose according to experience for off-label administration, which may lead to ineffective or even serious side effects.
the global pediatric drug and vaccine market is estimated at about $122 billion in 2019 and is expected to reach $141 billion by 2025.
the past two decades, regulators have made great efforts to encourage pharmaceutical companies to develop pediatric drugs.
but designing, recruiting and executing paediatric clinical trials and determining the right clinical endpoint remains a challenge.
this report reviews the latest legislation and incentives to move the field forward in pediatric medicine, providing market trend forecasting, combing of differences in the distribution of Chinese and foreign treatments, and an analysis of potential challenges. The following part of
will focus on the analysis of the case of leading pediatric drug enterprises and the global trend of pediatric drug cooperation, and further provide insights into the opportunities of the pediatric drug industry, aiming to provide navigational reference for domestic enterprises in pediatric drug development, product introduction and sea.
. China and the U.S. lead the global market, with the Asia-Pacific region a major growth region with a global pediatric drug market of about $122 billion in 2019 and is expected to reach $141 billion in 2025, with a compound annual growth rate of 2.4%.
, North America remains the largest single regional market for pediatric drugs, followed by China.
in the pediatrics market is now largely driven by the Asia-Pacific region, led by China, and is expected to gain a larger market share in the coming years, according to BCC research data.
other fast-growing countries include India and South-East Asia.
Chart 1: Global Market Forecasts for Paediatric Drugs 2020 Public Data Collation II, Research in the Field of Infection Has a Decreasing Trend, Genetic Diseases and Cancers Are the Next Blast Point World Health Organization Data Show that pneumonia or other acute respiratory infections are the leading cause of death among children worldwide.
since 2013, FDA pediatric labeling has been distributed primarily in the field of infection.
existing treatments effectively and safely control many infectious diseases, antibiotic resistance is becoming more common and the next key challenge to be addressed.
traditional high-demand areas of inflammation, cardiovascular, respiratory tract, diabetes and so on also have a lot of drugs approved, is the major pharmaceutical companies have laid out the focus.
figure 2: FDA pediatric drug labeling disease field distribution data source: the FDA official website database in contrast, PhRMA clinical trial research data show that genetic diseases and cancer pediatric drug research accounted for 34%, even more than the number of infectious disease drugs research.
dermatology and psychotic drugs have also been the focus of research in recent years.
: The development of distributed genetic diseases in the field of clinical trial diseases of pediatric drugs and the development of gene therapy in recent years, as well as the incentive policy of rare diseases are inextricable.
12 types of childhood cancer and more than 100 subsypes, the most common of which include leukemia, lymphoma and brain tumors.
thanks to advances in cancer research and treatment, more than 80 percent of children with cancer survive for five years or more.
pediatric psychiatry drugs are currently the most approved drugs for the treatment of ADHD in children, other drugs such as antidepressants, anti-anxiety, personality schizophrenia and other drugs have not been approved much, but also can not meet market demand.
. Policy is good and sustained, the number of pediatric drug labeling in 2019 reached a record high pediatric drug labeling from 2013 on the growth trend, in 2019 a new record high, there are 68 pediatric drug labeling.
is closely related to regulatory support for pediatric drug development.
Figure 4: FDA Pediatric Drug Labeling Number (2013-2019) Data Source: FDA Official Database Paediatric Drug Concept developed relatively early in the West, the United States and the European Union and other developed countries have correspondingly introduced policies to encourage pediatric drug development, accumulated relatively mature policy and regulatory experience.
5: The United States, as one of the first countries to regulate pediatric drug legislation, enacted a bill in 1997 giving pediatric drugs a longer exclusive right.
In 2012, the U.S. Congress passed the U.S. Food and Drug Administration's Safety and Innovation Act (FDASIA), which made the impact of the Pediatric Research Equality Act and the Best Children's Drug Act (BPCA) on pediatric drug development permanent, using a carrot (BPCA) soft-and-hard strategy to promote pediatric drug development and encourage the inclusion of pediatric populations in clinical trials.
PREA requires that all new drug applications be submitted to the Pediatric Research Program 60 days after the end of The Adult Clinical Phase II meeting, unless the FDA agrees to an exemption or delay.
BPCA provides that bidders who conduct pediatric population clinical trials as required by the FDA can still get six months of market access even if clinical results are not conceded, such as Lilly's Cymbalta do Lothian.
addition, the FDASIA Act also introduced priority review vouchers for developers of drugs for rare pediatric diseases. PRV) program, PRV holders can obtain priority approval for any new drug after that.
since its adoption, the program has attracted a lot of interest from drug companies seeking treatment for rare pediatric diseases, with BioMarin getting its first PRV in 2012 and selling it five months later for $6.75 billion.
, more than a dozen pharmaceutical companies have received PRV.
Chart 6: The U.S. Pediatric Drug Laws and Regulations Show Intent The European Union established the Paediatric Drug Administration Regulations on January 27, 2007, which require all new drug applications to be submitted to the Children's Drug Research and Development Program as of July 26, 2008, and from January 26, 2009, new adaptations, new prescriptions, and new dosage forms must also include the Child Drug Development Program.
China, as the world's second largest market for pediatric drugs, is also drawing on the experience of the United States and the European Union and other regions, issued the "Technical Guidelines for Clinical Trials of Paediatric Population Drugs", "China's National Prescription Collection (Children's Edition)" and other guidance documents, to further explore the path of pediatric laws and regulations, to provide additional value for pediatric drug development.
4, large pharmaceutical companies dominate the market, Gilead pediatric labeling numbers ranked first in the international pediatric drug market competition, basically led by large pharmaceutical companies, Gilead, GlaxoSmithKline, Mercado, Eljian, Johnson and Johnson and other companies from 2013 to date completed a number of drug pediatric clinical trials.
areas of the layout are infectious diseases, respiratory diseases (asthma), digestive tract diseases, painkillers, anti-cancer drugs and vaccines.
Chart 7: FDA Pediatric Drug Labeling Number by Company Source: FDA Official Website Database A look at 68 FDA-approved pediatric labels in 2019, antiviral drugs top the list, cardiovascular disease drugs and pediatric ADHD drugs are hot areas.
10 pediatric drug market exclusives granted by the FDA in 2019 are all included in the large pharmaceutical companies.
can be seen that due to high research and development funding, technical difficulties, and clinical trial target recruitment difficulties, start-ups into the field of pediatric drugs is more difficult.
Chart 8: FDA Pediatric Drug Market Exclusive Grant (2019) Data Source: FDA official website database from the clinical pipeline, the top 10 are also large pharmaceutical companies, Johnson and Johnson jumped to the top of the list.
chart 9: Global pediatric drug clinical number (by company) V, clinical research orphan drugs accounted for about 30%, large enterprises to increase the research and development of pediatric-specific drugs PhRMA published data show that the global pediatric drug clinical research a total of 597, including 317 drugs for adults and pediatrics, 280 pediatric-specific drugs;
for this analysis, we define companies with more than two pediatric drugs in research as Class A enterprises (289), mainly large and medium-sized enterprises, and others as Category B enterprises (308).
Orphan Drug policy benefits include preferential approval, seven-year exclusive rights to the U.S. market after listing, PDUFA fee waivers (approximately $2 million), and a 50 percent tax subsidy for clinical trials in the U.S., which have greatly stimulated the enthusiasm of pharmaceutical companies to develop orphan drugs.
the U.S., the number of people with the disease is less than 200,000, mainly in the field of genetic diseases and cancer.
The number of patients with orphan drugs is small and tends to be priced higher than other drugs, and when the two are balanced, there is optimism in terms of sales of products already on the market, such as Spinraza, a pediatric drug used to treat spinal muscular dystrophy, which had sales of $2,097 million in 2019, up 22% from 2018.
Figure 10: Comparing category A and B enterprises in the global clinical study of paediatric drugs (by adaptive score), it can be seen that category A enterprises belong to large and medium-sized enterprises, generally have more resources and experience, and clinical research is more biased towards pediatric-specific (more than 50%), to achieve precision treatment.
B companies will prioritize a two-pronged strategy for children and adults to reduce risk and leverage limited resources to achieve the expected benefits.
Chart 11: The proportion of pediatric-specific drugs in all stages of the two types of enterprises is compared to six, the number of clinical declarations registered by the FDA of Chinese and foreign enterprises in China is flat, and the clinical data of Clinicaltrials.gov, which are mainly in the field of infection, show that there are currently 501 pediatric drug research in China, of which 62.5% are for adults and pediatrics, 313 are for pediatric-specific drug research, and 188 are conducted for pediatric drugs.
of this report is the clinical study of pediatric-specific drugs, of which 93 are Chinese enterprises and 95 are foreign enterprises, basically flat.
from the field of disease, 42% of clinical studies on the field of infection, covering the commonly used pediatric white break, influenza, pneumonia, meningitis and so on.
It is worth noting that compared with clinical trials conducted overseas, the number of clinical trials in China on infection, hand, foot and mouth disease treatment, developmental diseases and eye diseases is relatively large, but cancer drugs, skin diseases drug research is relatively small, genetic diseases and other orphan diseases are still in a blank period.
Chart 12: Comparison of clinical research distribution in China and abroad It is worth noting that the clinical trials conducted by foreign enterprises in China, and the clinical focus carried out overseas are quite different, and do not actively carry out the layout of genetic diseases and tumors (compared to Chart III), but pay more attention to infection, nervous system (anti-epileptic), cardiovascular drugs (heart failure, blood pressure reduction) and other research in line with the Chinese market demand, in preparation for the later entry into China.
domestic enterprises occupied the high points of hand, foot and mouth disease, ophthalmology disease, respiratory diseases and so on, the development of infectious diseases drugs is also close to foreign enterprises.
Chart 13: Comparison of clinical research distribution carried out by Chinese and foreign enterprises in China (by disease field) from the research and development stage distribution, although the clinical phase III research is mainly overseas enterprises, the number of early clinical led by domestic enterprises are higher than that of foreign pharmaceutical companies, which coincides with the rapid development of research and development innovation capabilities of domestic enterprises in recent years and the trend of active internationalization.
Chart XII: Comparison of clinical research distribution (by clinical stage) carried out by Chinese and foreign enterprises in China, the clinical design of pediatrics is complex, and patient recruitment is facing great challenges Despite the gratifying results achieved by the implementation of the laws and regulations on the development of paediatric drugs, the market demand for paediatric drugs is far from being met.
resource-poor areas have been exacerbated by limited economic conditions and fragmented health-care systems.
In the absence of paediatric dosage forms, about 50% of children's medications are given by doctors through off-label (off-label use) to adjust adult doses for administration, which puts both safety and effectiveness at risk.
drug adverse reaction monitoring issued by the State Drug Administration, the most common adverse reactions in children are significantly higher than in the general population.
, especially anti-infective drugs, the adverse reactions of the general population were 42.3%, and the rate of children was 73%.
, it can be seen that the clinical development of pediatric drugs is urgent.
, however, the high cost and complexity of pediatric clinical trials (e.g. changes in recruitment of children, clinical trial logistics, ethics, pharmacodynamics due to rapid growth in children, etc.) are the biggest challenges facing developers.
countries with small populations, or some niche diseases, do not have sufficient paediatric populations to participate in clinical trials, nor do they have the basic support of recruitment networks and platforms.
coupled with the fact that guardians tend to be concerned about the risk of clinical trials, or are less involved, taking into account factors such as the impact on patient enrollment.
design of pediatric clinical trials is also an important challenge.
the development of systems such as liver and kidney, there are great differences in pharmacodynamic parameters between different age groups in pediatric patients.
, specific studies are needed for groups of different ages, and the requirements for preparations are relatively high.
ideal way to develop a variety of formulations for a product for different age groups, such as suspensions, oral solutions, particles, etc., and conduct corresponding pharmacodynamic experiments.
, pediatric clinical placebo use was more restricted than in adults.
placebo is used, the candidate must have a clear deactivation criteria to reduce the frequency and duration of placebo use.
, appropriate clinical endpoints are required to collect physiological indicators such as weight, height, bone growth, and sexual maturity for regulatory filing.