Alex's Lemonade Stand Foundation for Childhood Cancer

Clinical trials are a critical step in the research process, and for those kids for whom approved drugs have failed, they are a critical step towards survival. Despite their importance, design and access to clinical trials has presented a challenge because of the rarity of childhood cancer types and subtypes (see Section 3). As researchers study new drugs and interventions, they are also turning their attention to designing clinical trials that accelerate science and provide more access to children both upfront and with relapsed disease. Unlike adult cancer research which has heavy investment from the private sector and pharmaceutical companies, pediatric cancer trials are often led by academic labs.²³⁵ As research evolves and federal legislation like the RACE act (see Section 3) demand more industry involvement in childhood cancer research, the academic-industry partnership will also evolve.

Some other trends in clinical trial design include:

• Using master protocol trials to test many treatments under one big trial²³⁶
• Leveraging data from adult trials and using a flexible statistical technique called Bayesian to make trials smaller and faster but still reliable²³⁷
• Continuing to focus on tumor mutations versus where the tumor started in the body to guide trial eligibility²³⁸
• Designing trials that are more flexible, and adapting those trials as the data comes in²³⁹