Global Pediatric Clinical Trials Market Outlook, 2031

The global pediatric clinical trials industry covers the design, conduct and management of clinical studies that test medicines, vaccines, biologics, gene and cell therapies, and medical devices in children, from newborns and infants through adolescents. For decades, most medicines were tested only in adults, and children were treated using doses and data extrapolated from adult studies, leading to errors and unknown risks. Regulations now require or encourage pediatric studies: in the United States, the Pediatric Research Equity Act requires pediatric assessments for new drugs unless waived, the Best Pharmaceuticals for Children Act grants six months of additional exclusivity for completing requested pediatric studies, and the Rare Pediatric Disease Priority Review Voucher Program rewards approvals for rare childhood diseases; in the European Union, the Paediatric Regulation requires paediatric investigation plans and offers rewards for compliance. The industry includes pharmaceutical and biotechnology sponsors, contract research organisations such as IQVIA, ICON, Parexel, Syneos Health, PPD within Thermo Fisher, Fortrea, Medpace and specialised pediatric CROs, children's hospitals and academic networks such as the Children's Oncology Group, the Institute for Advanced Clinical Trials for Children, conect4children in Europe and the Pediatric Trials Network funded by the US NIH. Vaccines, rare genetic diseases, oncology, infectious diseases, neurology, and neonatal conditions are the main areas of pediatric research.

Pediatric clinical research has grown because of regulatory requirements, the rise of treatments for rare genetic diseases that often begin in childhood, and large vaccine and infectious disease programmes. The pandemic delayed many pediatric trials in 2020, then produced major pediatric studies of COVID-19 vaccines. Respiratory syncytial virus prevention generated large infant trials, leading to approvals of nirsevimab in 2023 and Merck's clesrovimab, Enflonsia, in June 2025, and a maternal RSV vaccine. Gene therapies and genetic medicines for children, such as those for spinal muscular atrophy, Duchenne muscular dystrophy and sickle cell disease, have required specialised pediatric trials, and in May 2025 an infant became the first person treated with a personalised base-editing therapy. Regulatory frameworks have evolved. The ICH E11A guideline on pediatric extrapolation, finalised in 2024, supports using adult and other data to reduce the number of children needed in trials. The EU Clinical Trials Regulation became fully applicable for all trials in January 2025, and proposed EU pharmaceutical legislation would revise paediatric rewards and obligations. In the United States, the rare pediatric disease priority review voucher programme lapsed in December 2024 but was reauthorised through 2029 in February 2026 by the Mikaela Naylon Give Kids a Chance Act, which also strengthened FDA authority to require pediatric studies of cancer drug combinations.

Market Dynamics Market Drivers • Regulatory Requirements and IncentivesLaws in the United States, the European Union, the UK, Japan and other countries require companies to study new medicines in children or justify waivers, and they provide incentives such as extended exclusivity and priority review vouchers. The reauthorisation of the US rare pediatric disease voucher programme in 2026 and new FDA authority to require pediatric studies of cancer drug combinations will increase pediatric trial activity. Regulators increasingly expect pediatric plans early in development.
• Growth of Rare Disease, Gene Therapy and Vaccine PipelinesMany rare genetic diseases begin in childhood, and advances in gene therapy, antisense oligonucleotides, siRNA and gene editing have produced treatments that must be tested in children, sometimes in newborns or infants for best effect. Vaccine development for RSV, meningitis, pneumococcal disease and other infections requires large pediatric trials. Pediatric oncology, neurology and endocrinology also have growing pipelines, supporting demand for specialised pediatric trial capabilities.

Market Challenges • Recruitment Difficulties and Ethical ComplexityChildren are a vulnerable population, and trials require consent from parents and assent from children, careful risk-benefit assessment and ethics committee review. Many pediatric diseases are rare, making recruitment slow and requiring multinational sites. Parents may be reluctant to enrol children, particularly in placebo-controlled studies, and trials must minimise blood draws, procedures and visits. Recruitment challenges delay trials and increase costs.
• Operational and Scientific ChallengesPediatric trials require age-appropriate formulations, such as liquids and mini-tablets, weight-based dosing, specialised staff and child-friendly sites. Children's physiology changes with age, requiring studies across age groups and careful pharmacokinetic modelling. Endpoints validated in adults may not be suitable for children. Limited commercial returns for some pediatric indications and the high cost of trials can lead to delays or incomplete pediatric programmes.

Market Trends • Extrapolation, Modelling and Innovative Trial DesignsRegulators increasingly accept extrapolation of efficacy from adult or older pediatric populations, supported by modelling and simulation, pharmacokinetic studies and real-world data, as described in the ICH E11A guideline. Bayesian designs, master protocols that test several drugs in one trial, and external or synthetic control arms using natural history data help reduce the number of children needed. These approaches shorten timelines and reduce burden on patients.
• Decentralised Trials and Pediatric Research NetworksDecentralised trial elements, including home visits, telemedicine, electronic consent, wearable devices and home delivery of study medicines, reduce the burden on families and help reach children far from specialist centres. Pediatric research networks, such as conect4children in Europe, the Institute for Advanced Clinical Trials for Children in the United States and national networks in the UK, Canada and Japan, provide trained sites, standard processes and patient advocacy input to speed trial start-up.



Segment Analysis Phase II and phase III trials account for the largest share of pediatric clinical trial spending, while phase I and pharmacokinetic studies are important for establishing safe doses in children. Phase I pediatric studies, often combined with pharmacokinetic and safety assessments, determine how drugs are absorbed, distributed, metabolised and eliminated in children of different ages and establish appropriate doses, and they are often conducted after adult data are available. Phase II studies assess efficacy and safety in pediatric patients with the target condition and refine dosing, and many pediatric programmes in rare diseases combine phase II and III elements. Phase III trials, including large vaccine studies with thousands of infants and children and confirmatory studies of new medicines, account for the largest share of spending because of their size and duration. Phase IV and post-marketing studies monitor long-term safety, growth and development in children after approval, and they are particularly important for gene therapies, vaccines and chronic treatments. Pediatric formulation development and bioequivalence studies test age-appropriate forms, such as liquids, granules and mini-tablets. Observational studies and registries collect natural history and real-world data for rare diseases and support extrapolation. Long-term extension studies follow children in trials for years to assess durability of benefit.

Vaccines, infectious diseases and rare genetic diseases account for large shares of pediatric trial activity, while oncology, neurology and respiratory diseases are major and growing therapeutic areas. Vaccines and infectious diseases represent a large share of pediatric trials, including studies of vaccines against pneumococcal disease, meningococcal disease, RSV, influenza, rotavirus and COVID-19, and of long-acting antibodies such as nirsevimab and clesrovimab for infant RSV prevention, often involving thousands of participants. Rare genetic diseases, including spinal muscular atrophy, Duchenne muscular dystrophy, cystic fibrosis, sickle cell disease, lysosomal storage disorders and rare metabolic diseases, account for many pediatric programmes, particularly for gene therapies, antisense drugs and enzyme replacement therapies. Pediatric oncology, including leukaemia, brain tumours, neuroblastoma and sarcomas, is studied through cooperative groups such as the Children's Oncology Group and industry trials, and new US requirements for pediatric study of cancer drug combinations will expand this area. Neurology, including epilepsy, migraine, autism-related conditions and neuromuscular disease, is a growing area. Respiratory diseases, including asthma and cystic fibrosis, and immunology, including juvenile arthritis, atopic dermatitis and inflammatory bowel disease, have many pediatric trials for biologics. Endocrinology, including growth disorders, type 1 diabetes and obesity, psychiatry, including ADHD and depression, and neonatology form further areas.

Children aged 2 to 11 and adolescents account for the largest number of pediatric trial participants, while neonates and infants represent the most specialised and challenging group. Neonates, from birth to 28 days, including premature babies, are the most vulnerable pediatric group, and trials in neonatal intensive care units study treatments for conditions such as sepsis, respiratory distress, bronchopulmonary dysplasia and neonatal seizures, as well as preventive antibodies given at birth. Neonatal trials require specialised sites and very careful dosing. Infants and toddlers, from 1 month to under 2 years, participate in vaccine trials, RSV prevention studies and treatments for early-onset genetic diseases such as spinal muscular atrophy, where early treatment gives the best results. Children aged 2 to 11 years participate in trials across many therapeutic areas, including asthma, epilepsy, infections, rare diseases and vaccines, and they require age-appropriate formulations and child-friendly procedures. Adolescents aged 12 to 17 years are often included in adult trials or studied separately for conditions such as psoriasis, atopic dermatitis, inflammatory bowel disease, migraine, depression, obesity and type 1 diabetes, and regulators increasingly encourage including adolescents in adult trials. Young adults transitioning from pediatric care are sometimes studied in extension programmes. Each age group has different physiology, consent requirements and practical considerations.

Pharmaceutical and biotechnology sponsors fund most pediatric clinical trials, while contract research organisations conduct a large share of the work and academic networks lead many investigator-initiated and oncology studies. Pharmaceutical and biotechnology companies sponsor most pediatric trials, driven by regulatory requirements, exclusivity incentives, priority review vouchers and commercial opportunities in pediatric diseases, and large vaccine makers such as Pfizer, Merck, GSK and Sanofi run some of the largest pediatric studies. Contract research organisations, including IQVIA, ICON, Parexel, Syneos Health, PPD, Fortrea and Medpace, and specialised pediatric research companies manage trial design, site selection, recruitment, monitoring and data management, and many have dedicated pediatric units. Children's hospitals and academic medical centres serve as trial sites and conduct investigator-initiated research, and cooperative groups such as the Children's Oncology Group run large pediatric cancer trials. Government agencies, including the US National Institutes of Health's Pediatric Trials Network and the Eunice Kennedy Shriver National Institute of Child Health and Human Development, fund studies of off-patent drugs used in children. Non-profit organisations and patient foundations fund research into rare pediatric diseases and help recruit participants. Networks such as conect4children and the Institute for Advanced Clinical Trials for Children connect sponsors, sites and families.

Regional Analysis North America leads the pediatric clinical trials market because of US regulatory requirements and incentives, a large biotechnology sector and extensive children's hospital research networks, while Europe is a close second and Asia-Pacific is the fastest-growing region. North America, led by the United States, accounts for the largest share of pediatric clinical trial activity. The Pediatric Research Equity Act, the Best Pharmaceuticals for Children Act and the rare pediatric disease priority review voucher programme, reauthorised through 2029 in February 2026, drive pediatric studies, and the new authority to require pediatric studies of cancer drug combinations will add oncology trials. The United States has leading children's hospitals, the Children's Oncology Group, the Institute for Advanced Clinical Trials for Children and NIH-funded networks, as well as many biotechnology companies developing therapies for rare pediatric diseases. Canada contributes through children's hospitals and the Maternal Infant Child and Youth Research Network. Europe is the second largest region, driven by the EU Paediatric Regulation, which requires paediatric investigation plans, the EU Clinical Trials Regulation, fully applicable from January 2025, and networks such as conect4children and national pediatric research networks in the UK, Germany, France, Italy and Spain. Proposed EU pharmaceutical legislation would change paediatric obligations and rewards. Asia-Pacific is the fastest-growing region, with large pediatric populations, faster recruitment and growing clinical research capacity in China, Japan, South Korea, India, Australia and Southeast Asia. Japan has incentives for pediatric development, and China has encouraged pediatric drug development and trials. Latin America, led by Brazil, Mexico and Argentina, hosts many vaccine and infectious disease trials, and Africa is important for pediatric vaccine, malaria, tuberculosis and HIV trials.

Key Developments February 2026: The Mikaela Naylon Give Kids a Chance Act reauthorised the voucher programme through 2029 and strengthened FDA authority to require pediatric studies of new cancer drug combinations.
June 2025: Merck's long-acting antibody clesrovimab was approved to protect newborns and infants from RSV based on large pediatric trials.
May 2025: Doctors in Philadelphia reported treating an infant with CPS1 deficiency using a base-editing therapy designed for his mutation, opening new possibilities for bespoke pediatric treatments.
January 2025: All ongoing clinical trials in the EU, including pediatric studies, were required to transition to the Clinical Trials Regulation and the Clinical Trials Information System.
December 2024: Authority to award rare pediatric disease priority review vouchers for newly designated products expired, creating uncertainty for rare pediatric drug developers until its 2026 reauthorisation.
August 2024: The International Council for Harmonisation adopted guidance on using extrapolation to support pediatric drug development, reducing the need for large efficacy trials in children.

Considered in this report
• Historic Year: 2020
• Base Year: 2025
• Estimated Year: 2026
• Forecast Year: 2031

Aspects covered in this report
• Global Pediatric Clinical Trials Market with its value and forecast along with its segments
• Various drivers and challenges
• Ongoing trends and developments
• Top profiled companies
• Strategic recommendation

By Phase
• Phase I
• Phase II
• Phase III
• Phase IV

By Therapeutic Area
• Vaccines and Infectious Diseases
• Rare Genetic Diseases
• Oncology
• Neurology
• Respiratory Diseases
• Immunology
• Endocrinology and Metabolic Diseases

By Age Group
• Neonates
• Infants and Toddlers
• Children
• Adolescents

By Sponsor
• Pharmaceutical Companies
• Biotechnology Companies
• Academic and Government Institutions
• Non-Profit Organisations