Pediatric Cancer Dataset Gaps, FDA ‘High Bar’ Make Use Of External Control Arms Difficult

European data privacy regulations could further complicate efforts to build external control arms using real-world data that are housed outside the US, experts said at advisory committee meeting on use of real-world evidence in pediatric cancer drug development.

Boy with teddy bear
For pediatric cancer drug sponsors, it may be difficult to find a comparator arm outside their study. • Source: Alamy

The ability to leverage real-world evidence as an external control in pediatric cancer drug trials currently is limited by the quality of the underlying datasets, including a lack of genomic and molecular marker information, and the US Food and Drug Administration’s high bar for use of such data to support regulatory decision-making.

In addition, European data privacy regulations could further complicate efforts to build external control arms using real-world data that are...

More from Real-World Evidence

EMA Sets New Priorities As Number Of RWE Studies Climbs 48%

 

Real-world data studies conducted by the European Medicines Agency increased by 48% according to its latest report on the topic, prompting the agency to consider how it can better integrate this type of evidence into regulatory decision-making.

AI Will Bolster, Not Squelch, Patient Voice In Clinical Research, US FDA Says

 
• By 

Artificial intelligence tools used for clinical research should be developed with input from patients and focus on their needs and abilities, FDA experts said.

Post-Approval, Real-Time Monitoring Will Help Approval Standards, US FDA’s Makary Says

 
• By 

The FDA will use electronic health records to “get eyes” on drugs immediately after approval, Commissioner Martin Makary told the DIA 2025 Global Annual Meeting. He also wants to reduce development time with better communication and new approval pathways.

US FDA’s Prasad: ‘We Will Always Embrace Surrogate Endpoints’

 

The new CBER director, once best known in the pharma world for criticizing accelerated approval, committed to expediting cancer drugs with surrogate endpoints.

More from Clinical Trials