Pediatrics
Overview
America’s biopharmaceutical companies are advancing progress in pediatric treatments, supported by major growth in clinical research that has deepened our understanding of childhood illnesses. Landmark laws like the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) have reshaped how medicines are developed and labeled for kids, combining incentives and requirements that have significantly improved children’s care. While progress is strong, more work is needed to address unmet medical needs and ensure more children can access treatments 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 research has provided important new dosing, safety and efficacy information in pediatric populations that is changing the treatment landscape across a range of serious and life-threatening diseases, including:
- Asthma: Pediatric patients age 6 to 11 now have a therapy option that addresses severe eosinophilic asthma, a complex and challenging condition to treat, which was previously only approved for use in children age 12 and older. This breakthrough is a significant step forward, as asthma is the third-ranking cause of hospitalization among children younger than 15.
- Type 2 Diabetes: The first non-insulin drug approved to treat type 2 diabetes in pediatric patients is now available. The medicine enhances the incretin system, a natural body system that helps to regulate glucose, and improves blood sugar control.
- Peanut Allergy: A new oral immunotherapy designed to reduce the incidence and severity of allergic reactions due to accidental peanut exposure is now available for children with peanut allergy. The therapy delivers a controlled daily dose of peanut protein that is gradually increased over months to build tolerance in the immune system’s overreaction to peanuts.
- Rare Genetic Brain Tumor: Children with a rare genetic disorder, tuberous sclerosis complex, now have a treatment option for tumors that occur in the brain. Affecting about 1 in 6,000 newborns, this disease primarily occurs in childhood and this new approval offers a dissolvable dosage form that is easier to take for pediatric patients.
- Chronic Myelogeneous Leukemia: Additional research revealed that a breakthrough targeted therapy for this rare blood cancer is safe and effective in very young children, offering an important treatment option for physicians to use in treating this rare blood cancer.
- HIV/AIDS: New combination therapies are now available for pediatric patients, reducing the complexity of treatment for this devastating disease by offering options that can be taken once a day in a fixed-dose pill.
A robust drug development pipeline offers tremendous hope for the future. Currently there are over 2,100 industry-sponsored pediatric clinical trials underway, involving 1.2 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
Before the 1997 Food and Drug Administration Modernization Act (FDAMA), many FDA-approved medicines had never been clinically tested in children, leaving critical gaps in dosing, safety, efficacy and side-effect information. Since children often respond differently than adults, studying medicines specifically in pediatric populations is essential.
The lack of pediatric-specific drug information spurred two key laws: the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). Together, they drive pediatric drug development by ensuring studies are conducted and by generating essential data on dosing, safety, efficacy and side effects.
- PREA gives the FDA the authority to require pediatric studies requires pediatric studies for certain new medicines approved for use in adults where the indication for use in children would be comparable to that for adults, and produce formulations appropriate for children (e.g., liquid or chewable tablets).
- BPCA complements PREA requirements by providing sponsors with an incentive (6 months additional marketing exclusivity) to undertake voluntary pediatric studies at the request of the FDA, many of which may not be required under PREA. BPCA has proven to be an effective incentive for stimulating pediatric drug development, especially in cases where PREA requirements do not apply (for example, for orphan diseases, including rare cancers).
Made permanent with large bipartisan support in 2012, 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
Pediatrics Chart
770+
PEDIATRIC STUDIES
completed since the first temporary reauthorization of BPCA and PREA in 2007
800+
LABELING UPDATES
reflecting pediatric information made since 2007
2,630+
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.
TAILORED APPROACHES ARE NEEDED TO DEVELOP INNOVATIVE MEDICINES FOR CHILDREN
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.
Advancing Solutions Together
America’s biopharmaceutical companies are committed to advancing innovative treatments for pediatric patients. Collaboration is essential to overcoming the scientific and operational challenges of pediatric research, and pre-competitive partnerships play a key role in improving pediatric oncology medicine development.
- In October 2016, the Children’s Cause for Cancer Advocacy (CCCA), PhRMA, and BIO convened the first workshop that brought together all major pediatric oncology stakeholders, including 15 biopharmaceutical companies, patient groups, FDA, NIH and leading pediatric oncologists, to address scientific and operational challenges in developing new treatments. Participants agreed to collaborate on creating a pre-competitive public-private partnership to advance solutions.
- Additionally, in 2017, the Institute for Advanced Clinical Trials for Children (I-ACT for Children) was launched to address challenges in bringing new therapies to children. I-ACT for Children, an independent nonprofit, seeks to foster public private collaboration in finding solutions to barriers to pediatric medicine development, as well as improving the pediatric clinical trial process.
Advancing Solutions
Related Resources
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
FDA - Pediatrics
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