Pediatrics
Overview
America’s biopharmaceutical companies are advancing progress in pediatric treatments, supported by clinical research that has deepened our understanding of childhood illnesses. The Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) are landmark laws that have reshaped how medicines are developed and labeled for kids, combining incentives and requirements that have significantly improved children’s care. While this progress is strong, more work is needed to address unmet medical needs and ensure more children can access therapeutically appropriate medicines that help them grow into healthy adults.
Fact Sheet: Pediatric Drug Development
Overview
Industry Commitment to Pediatric Research
The innovative biopharmaceutical industry is committed to conducting pediatric research and is making great strides in combatting pediatric illnesses. Recent treatment advances are changing the treatment landscape across a range of serious and life-threatening diseases, including:
- Spinal Muscular Atrophy: New treatment options have transformed the outlook for children with spinal muscular atrophy, a disease that was once associated with limited options and progressive loss of motor function. Before disease-modifying therapies, infants with the most severe form often required permanent ventilation and frequently did not survive beyond age 2. Earlier screenings and interventions with innovative therapies are now offering hope to children and their families in transforming the trajectory of this devastating illness.
- Cystic Fibrosis: Therapies targeting the malfunctioning protein that results from mutations in the CFTR gene have helped shift care beyond managing symptoms toward therapies that address the underlying disease, dramatically improving and extending lives of CF patients. Today, children born with cystic fibrosis are predicted to live into their mid-60s on average, compared with life expectancies measured in the 30s only a decade ago.
A robust drug development pipeline offers tremendous hope for the future. Currently there are over 2,900 industry-sponsored pediatric clinical trials underway, involving nearly 1.5 million pediatric patients across a variety of therapeutic areas, including diseases where there is significant unmet need, such infectious diseases, neurologic conditions, genetic disorders and several forms of cancer.
Industry Commitment
Policies to Foster Pediatric Drug Development
Researching and developing innovative and therapeutically appropriate medicines for children is an important priority for America’s biopharmaceutical research companies. Developing medicines for children requires a tailored approach to clinical trials, including how studies are designed, how children participate, and how outcomes are measured. By design, this work is complex, iterative in nature and builds on prior scientific advances.
The Food and Drug Administration Modernization Act was enacted in 1997 and marked the first legislation that incentivized pediatric drug development. Permanently authorized in 2012, PREA and BPCA drive pediatric drug development by ensuring studies are conducted and by generating essential data on dosing, safety, efficacy and side effects.
- PREA authorizes FDA to require pediatric research for certain indications for which the sponsor is seeking approval in adults and to product formulations appropriate for children (e.g., liquid or chewable tablets).
- BPCA complements PREA and provides incentives (six months of added marketing exclusivity) to encourage manufacturers to conduct pediatric studies of medicines with the potential for use in children.
PREA and BPCA have been key drivers of pediatric research, generating important safety and efficacy information on the use of medicines in children. This balanced approach has driven research on innovative medicines in pediatric patients, resulted in more treatment options for children, and has greatly advanced children’s medical care. Learn more here.
Policies
1100+
PEDIATRIC STUDIES
completed since the first temporary reauthorization of BPCA and PREA in 2007
2900+
INDUSTRY-SPONSORED
pediatric clinical trials underway worldwide
Unique Research Challenges
Developing medicines for pediatric diseases poses unique scientific and operational challenges. Biopharmaceutical companies are committed to working with all stakeholders to combat the issues that prevent further research and results.
Research on children is conducted only after taking special ethical and medical considerations into account.
Microscope
Small patient populations make it challenging to recruit for and enroll patients in clinical trials.
Bar Chart
Diseases in children are often biologically different than those in adults, requiring additional assessments of medicine safety and efficacy.
Children respond differently to medicines than adults, requiring unique dosages and formulation considerations.
Related Resources
Why the hepatitis B vaccine matters
While hepatitis B is a serious disease, it is also preventable with early protection. One of the most effective ways to protect infants from serious illness is through the hepatitis B vaccine given soon after birth. Here’s what you should know:
What is hepatitis B and what are the long-term impacts of it?
Hepatitis B is a viral infection that attacks the liver. While some people recover, many who become infected—especially babies and young children—develop chronic infection and liver damage that can last a lifetime. Chronic hepatitis B can lead to cirrhosis (scarring of the liver), liver cancer and even liver failure.
How does someone get hepatitis B?
Hepatitis B is transmitted through blood and bodily fluids, and the virus can live outside the body (for example, on surfaces) for up to a week or more. Infants can be exposed to hepatitis B through everyday contact, and babies are especially vulnerable because an infected mother, family member or caregiver can unknowingly pass the virus on:
- During pregnancy or birth (from an infected mother to her baby)
- Through a bite from an infected person
- By touching open cuts or sores of an infected person
- By sharing, even incidental, personal items like toothbrushes, razors, or towels with an infected person
Why should people get the hepatitis B vaccine?
Hepatitis B is extremely contagious—up to 100 times more transmissible than HIV. Vaccination offers the best protection before exposure. Since introducing the birth dose in the U.S., cases in kids and teens have dropped by 99%. That’s critical, especially since nearly half of those infected don’t know it and may unintentionally transmit the virus to their loved ones—putting babies and children at risk.
Why is it so important that a newborn gets vaccinated against hepatitis B at birth?
If a newborn contracts hepatitis B at birth or an infant is infected in their first year of life, the chances of developing a serious, lifelong condition are extremely high.
Before universal vaccination at birth in the U.S., up to 20,000 infants each year were infected. Today, that number is close to zero annual cases thanks to vaccination.
Why is the hepatitis B vaccine recommended for all newborns, not just those whose mothers test positive?
The hepatitis B vaccine is recommended for all newborns—not just those whose mothers test positive—because everyday contact, not just childbirth, can transmit the virus. Maternal testing also isn’t foolproof: some mothers aren’t tested and some become infected later in pregnancy. Vaccinating at birth ensures all babies are protected, even if maternal infection is missed or someone is infected in the baby’s household.
This universal vaccination approach ensures no baby is left vulnerable.
How was the recommendation to vaccinate against hepatitis B at birth developed?
The recommendation to vaccinate all newborns against hepatitis B at birth was developed after decades of research, real-world data and ongoing safety monitoring of FDA-approved vaccines. Experts found that infants are especially vulnerable to serious, chronic illness if infected early, and that vaccinating at birth is safe and highly effective.
Initially, the vaccine was only given to at-risk infants, but hepatitis B rates remained stubbornly high due to both maternal transmission and casual contact. This led to the shift toward a universal birth dose, which protects all babies regardless of maternal status and has resulted in a dramatic drop in hepatitis B infections. That strong evidence is why leading pediatric and infectious disease experts continue to recommend the birth dose for every newborn.
The bottom line: Ensuring babies receive the hepatitis B vaccine at birth is a simple, proven way to give them a healthy start. It’s a small step today that provides protection for a lifetime.
Carolyn Ha
Support for continued progress is critical for pediatric drug development
Researching and developing new medicines for children is a priority for the biopharmaceutical industry. Despite the scientific complexity and challenges inherent to developing treatments for our youngest patients, there has never been more progress in pediatric health outcomes.
The progress we are seeing in pediatric health outcomes is in part due to the advancement in medicines that have been specifically studied in and approved for use in children. To help advance the development of pediatric medicines, Congress, in a bipartisan fashion, passed provisions that were eventually permanently reauthorized in two laws: the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). These policies work together to foster pediatric drug development, enabling biopharmaceutical companies to continue making significant investments in critical research areas for children.
The BPCA has been successful in advancing pediatric research. For example, ibrutinib was awarded BPCA pediatric exclusivity based on the sponsor’s research into use of the drug in pediatric chronic graft-versus-host disease (cGVHD) patients. After ibrutinib was initially approved in 2017 for adult patients with cGVHD who have tried and failed on at least one previous medicine, the U.S. Food and Drug Administration (FDA) issued a BPCA written request to the sponsor for studies in pediatric cGVHD patients. In August of 2022, the FDA approved the use of this medicine for the treatment of pediatric patients one year and older with cGVHD who have tried and failed on one previous medicine. This is the first approved treatment option for children under 12 with cGVHD and the only drug class of its kind (Brutons tyrosine kinase inhibitor or BTKi) for a pediatric population. This approval also includes a specific oral formulation designed for children, providing a helpful alternative and underscoring the importance of the BPCA in advancing pediatric research.
Unfortunately, the recent passage of the Inflation Reduction Act (IRA) puts the progress we’ve made towards treatments for children in the U.S. at risk, by forcing companies to make hard decisions in the disease areas to invest in. The IRA allows selection of certain medicines for price setting after only seven years of FDA approval and imposition of a set price beginning at year nine, substantially earlier than the average time of 13-14 years that companies currently have until they face generic competition for marketed products. The IRA also undermines long-standing incentives, including BPCA pediatric exclusivity, by allowing government price-setting notwithstanding any unexpired exclusivity.
In short, the IRA undermines the provisions of the BPCA that have proven so successful in advancing pediatric research. The IRA’s impact on innovation could jeopardize progress in pediatric drug development, putting at risk some of the nearly 600 medicines in development for children.
America’s biopharmaceutical research companies are proud of our work to bring new medicines to pediatric patients. Let’s continue to support a policy framework that makes that innovation possible. Our children, and their families, are counting on it.
Learn more here.
Abigail Lore
Support for continued progress is critical for pediatric drug development
PREA and BPCA: Spurring Pediatric Drug Development
The Pediatric Research Equity Act (PREA) and Best Pharmaceuticals for Children Act (BPCA), permanently reauthorized in 2012, work together to foster pediatric drug development, creating a balanced approach that generates important safety and efficacy information on use of medicines in children and enables biopharmaceutical companies to continue to make significant investments in pediatric drug research.
Fact Sheet