Pediatrics rising costs, new therapies and key drug approvals

This page summarizes pediatric therapeutic trends for spring 2025, including rising medication costs, novel therapies, key immunization updates, significant changes in drug spending and anticipated drug approvals.

Advancements in pediatric medicine

Pediatric medicine has seen significant advances, including cellular immunotherapies and gene therapies, updated immunization schedules, improved neurological disorder treatments and increased use of bispecific agents like Blincyto for pediatric B-ALL. In fact, the FDA approval expanding Blincyto’s use in pediatric B-ALL significantly increased utilization, posing logistical challenges for hospitals implementing its 28-day treatment cycles.

Additionally, neurological care advanced through new pharmacological and neuromodulation approaches for epilepsy, autism spectrum disorder and cerebral palsy. Pediatric mental health treatment has also improved, with new medications and digital tools addressing anxiety, depression and ADHD.

In infectious diseases, advancements in antiviral and monoclonal antibody therapies have improved management of RSV and influenza, despite reduced vaccination uptake for the 2024–25 season.

The recent approval of Alyftrek for cystic fibrosis patients aged six and older with certain genetic mutations introduces competition for Vertex’s Trikafta.

Development and approval of therapies

Several cellular immunotherapies and gene therapies have recently been approved:

• Remestemcel-L-rknd (Ryoncil): In December 2024, the FDA approved remestemcel-L for treating children with steroid-refractory acute graft-versus-host disease (SR-aGVHD). This therapy uses mesenchymal stromal cells from the bone marrow to stop the inflammation that causes SR-aGVHD. This is a new treatment option for children as young as two months old.
• Eladocagene exuparvovec (Kebilidi, Upstaza): This therapy was approved in the U.S. in November 2024. It treats aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder that affects neurotransmitter production by replacing a defective gene that codes for the AADC enzyme in the brain. This product is approved for pediatric patients at least 16 months of age and is marketed as Upstaza in the United Kingdom and European Union.
• Mirdametinib (Gomekli): In February 2025, the FDA approved mirdametinib for treating neurofibromatosis type 1 (NF1) with symptomatic plexiform neurofibromas, a rare genetic disorder causing nerve tumors. This oral MEK inhibitor is available for both adults and children aged two and older. It offers a new way to treat this condition.

Bispecific agents for pediatric patients with B-ALL

Blincyto (blinatumomab) is approved for adults and pediatric patients (≥1 month old) with B-cell precursor acute lymphoblastic leukemia (B-ALL) in specific scenarios. Expanded indications doubled pediatric utilization, challenging hospitals to manage the 28-day continuous infusion cycles. Hospitalization is recommended for the first nine days of cycle 1 and the first two days of subsequent cycles to monitor infusion reactions.

Pediatric immunization schedules

The Centers for Disease Control and Prevention (CDC) has updated the 2025 immunization schedules for children and adolescents. In 2024, the pediatric immunization schedule underwent several updates to improve disease prevention among children and adolescents. In 2025, changes to the immunization schedule are expected including crucial changes:

• COVID-19: The CDC recommends an updated COVID-19 vaccine for everyone 6 months of age and older for the 2024–2025 season. Additional doses are recommended for immunocompromised children and adolescents.
• Influenza: Despite a decrease in pediatric vaccination rates, the CDC and ACIP recommend a new influenza vaccine for the 2024–2025 season. The vaccine now protects against influenza A (H1N1 and H3N2) and B (Victoria lineage). Note: It is safe to co-administer influenza and COVID-19 vaccines.
• Nirsevimab (Beyfortus): Recommended for infants 8 months and younger before or during their first RSV season, and for children aged 8 to 19 months at high risk for severe RSV during their second RSV season.
• DTaP-IPV-Hib-HepB (Vaxelis): This combination vaccine reduces the number of injections needed for the pediatric immunization series. It is now recommended for American Indian and Alaska Native infants, as its efficacy and safety are comparable to PedvaxHIB.
• Measles, Mumps and Rubella (MMR)/Measles, Mumps, Rubella and Varicella (MMRV): A minimum interval of 3 months between MMRV doses is recommended. Children aged 12 months and older planning international travel should receive a second dose at least four weeks after the first.
• Meningococcal B: A two-dose series of the MenB-FHbp vaccine (Bexsero) is recommended for adolescents aged 16 to 23 years, with doses administered at 0 and 6 months. A three-dose series is advised for those at increased risk.
• Meningococcal: Menactra (MenACWY-D) has been removed from the schedule, and Penbraya, a pentavalent meningococcal vaccine, is now recommended for individuals aged 10 through 25 years.

Top 15 medication spend and changes

The top 15 medications purchased by Children’s Hospital Association facilities are provided in Table 1 and include a detailed breakdown by indication and portion of purchases.

Table 1. Top 15 medication spend for Children’s Hospital Association

Source: Children’s Hospital Association data, Oct. 2023 – Sept. 2024
*Portion of spend for the NDCs making up the top 85% of CHA spend; top 10 medications comprise 37.7% of overall spend for that period
Abbreviations: aHUS = atypical hemolytic uremic syndrome; ALL = acute lymphoblastic leukemia; HLH = hemophagocytic lymphohistiocytosis; HSCT = hematopoietic stem-cell transplantation; PNH = paroxysmal nocturnal hemoglobinuria; VOD = veno-occlusive disease

Table 2 shows the top 10 medications with the greatest change in spend for Children’s Hospital Association facilities.

Table 2. Top 10 medications with greatest change in spend

Source: Children’s Hospital Association data, calendar year 2024 vs. calendar year 2023

New and noteworthy novel drug approvals

Abbreviations: AML = acute myeloid leukemia; HSCT = hematopoietic stem cell transplantation; MDS = myelodysplastic syndrome; RNA = ribonucleic acid; SC = subcutaneous

Pipeline: Anticipated high-impact approvals

Abbreviations: CNS = central nervous system; Inj = injection; IV = intravenous; SC = subcutaneous; IM = intramuscular

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