The 2026 Childhood Cancer Report

From diagnosis to research: measuring momentum in the search for cures

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Home » The Childhood Cancer Report » Section 3: Critical Gaps: Challenges in Funding, Treatment and Access

Challenges in Drug Development

The key to curing pediatric cancer without lasting side effects is having a comprehensive library of effective drugs with minimal side effects. Between 2020 and 2025, the FDA approved 322 drugs for oncology indications204—only 53 of those drugs are approved for children.205 Children do benefit from adult approvals, as those approved therapies can be used, as appropriate, in pediatric studies without the need for researchers to develop a new drug. However, a significant gap still exists. Children wait 6.5 years longer for new cancer drugs than adults.206 A survey of the oncology clinical trials open in the United States shows the disparity in research options for kids: Only 17% of the 26,000 recruiting clinical trials are for children.207, 208

Without research, drugs cannot be studied, developed, and approved.

Money, both the small portion of the federal research budget (see Section 3) and the lack of pharmaceutical investment in pediatric cancer, is the main driver of the gap. The rarity of childhood cancer also creates small sample population sizes which further hinders data collection and clinical trial accruals. And other barriers exist for children. For example, the format of the study drug can limit access for younger children and babies, as they cannot swallow a pill and a liquid formulation of the drug may not be available. Language and cultural barriers can limit trial enrollment. And, of course, geography and access to pediatric oncology research centers can present a barrier.

However, oncology researchers are working to optimize clinical trial designs in a variety of ways to accelerate drug development. At the federal level, one piece of key legislation called the Research to Accelerate Cures and Equity (RACE) for Children Act (passed in 2017) mandates pediatric cancer drug testing for new adult cancer drugs that also have pediatric targets and has made a small difference in drug development (see Section 3).

Between 2017 and 2024, the FDA approved 61 adult cancer drugs relevant to children, and while most pre-2020 drugs were tested in kids only after approval, post-2020 drugs under the RACE Act were more often tested before approval and included formal pediatric testing requirements.209

From Early Stage Research to FDA Approval

Having a drug go from early stage research to clinical trials and eventual FDA approval takes a significant amount of time and study on behalf of the researchers and the clinicians. When the FDA considers approving a drug, it usually looks at three main things:

  • The condition and current treatments: They review the disease the drug is meant to treat and compare it to existing treatments to understand the potential benefits and risks.
  • Evidence from clinical trials: They examine the data from studies to see how well the drug works and whether it could help patients faster.
  • Managing risks: They check the known risks of the drug and how steps can be taken to reduce or manage those risks.

But before the FDA even considers an approval, the drug is thoroughly studied and tested in both the lab and in patients in clinical trials:

  1. Lab experiments: Early research on cells in a lab (not in animals or people) to see if a potential drug can affect cancer cells by slowing their growth or killing them.
  2. Model Development: New lab models are created for pediatric cancers to better predict how drugs might work in animals and humans.212
  3. Drug and Target Matching: Scientists screen for molecules or therapies (like small molecules, antibodies, or new technologies) that can affect the target.
  4. More Lab Experiments: Promising compounds are tested in the lab to make sure they specifically and effectively hit the target, and chemists work on improving potency and making the drug safe for animals.
  5. Animal Models: The compound is tested in animals to check if it reduces tumor growth, reaches the tumor, and is safe.
  6. Phase I clinical trial: If animal results are good, the drug moves to humans, starting with a small trial to find the safest and most effective dose.
  7. Phase II/III clinical trials: If Phase I is successful, larger trials test how well the drug works at slowing tumor growth in more patients.
  8. FDA approval: If trials show the drug is safe and effective, it can be approved for use in that cancer type.
Socioeconomic BarriersResearch Funding and Legislation