September is Childhood Cancer Awareness Month, a time to recognize the children and families affected by childhood cancers, as well as emphasize the importance of research into these devastating conditions.
Childhood cancer remains the number one cause of death by disease for children in the United States. Each year, more than 15,000 children in the U.S. aged 0-19 are diagnosed, according to the American Childhood Cancer Organization.
Put another way, approximately 1 in 285 children in the U.S. will be diagnosed with cancer before their 20th birthday.
According to PhRMA’s 2020 report, there are 86 medicines currently in development for childhood cancer, including several cutting-edge medicines known as cell and gene therapies.
Pediatric diseases like childhood cancer are often clinically different than the adult version, requiring different approaches to disease management. Because children are not simply small adults, medicines may work differently. Understanding this, biopharmaceutical researchers are studying and developing medicines to meet the unique health needs of infants, children and adolescents.
Such research however poses several challenges, including difficulty enrolling young patients in clinical trials, as well as identifying the optimal dosage, preparation and delivery forms of medicines. It can take up to 15 years to complete a pediatric drug development program and nine years for a medicine to receive approval for use in pediatric patients after it has been approved for adults. A recent study found pediatric enrollment was about 20% in studies between 2004 and 2015. Patients with hematologic malignancies were more represented compared to solid and central nervous system (CNS) tumors.
Improvements to pediatric drug research and development can stem from enhanced communication, innovation and collaboration between all stakeholders involved, including patients, researchers and regulatory decision-makers.
Despite challenges, advances in science have led to incredible progress in the field of childhood cancer. For example, in 2017, the U.S. Food and Drug Administration (FDA) approved the first CAR-T therapy for pediatric and young adult patients diagnosed with a certain type of blood cancer. This revolutionary treatment provides a method of collecting a sample of a patient’s own white blood cells, genetically altering them to seek out and attack certain cancer cells, and injecting them back in the body to act as “smart bombs” that selectively target a patient’s cancer.
Over the last three years, several “tissue agnostic” therapies, meaning the therapy can be used for the treatment of any kind of tumor regardless of where in the body the tumor originated, were approved by FDA, including an oral medicine to treat certain pediatric patients whose metastatic solid tumors have a specific genetic biomarker.
Additional innovative treatments are currently under investigation, including immunotherapies and targeted medicines that seek out the specific genes driving a person’s cancer.
Thanks to new and improved treatment advances, 83% of children diagnosed with cancer from 2008 to 2014 will survive five years or longer, compared to 58% in the mid-1970s—meaning the death rate has declined by nearly two-thirds.
The Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) have further transformed the way medicines are developed and labeled for pediatric populations. Together, these laws spur pediatric research and development, through a careful balance of incentives and requirements, and have greatly advanced children’s medical care in the United States.
While we celebrate the continued success that biopharmaceutical companies have made in pediatric research and medicine development, more work remains to be done for patients with unmet medical needs. New knowledge and new medicines can offer hope that children today will recover from illnesses and grow into healthy adults.
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