Centralized Investigator Review of Radiological and Functional Imaging Reports in Real-World Oncology Studies: The...

Area of Science:

• Oncology
• Clinical Trials
• Pediatric Cancer Research

Background:

• Novel anticancer therapies are increasingly used off-label or via compassionate use for pediatric and young adult patients (<25 years) with solid tumors or lymphomas.
• Real-world data collection is crucial for assessing the safety and efficacy of these treatments in this specific population.

Purpose of the Study:

• To collect prospective, real-world clinical safety and efficacy data for novel anticancer therapies in patients under 25 years old.
• To evaluate the impact of centralized medical monitoring on the accuracy of objective tumor response assessments.

Main Methods:

• The SACHA-France study (NCT04477681) is a prospective, real-world data collection initiative.
• Included 640 patients (<25 years) with solid tumors or lymphomas from March 2020 to February 2024.
• Employed centralized medical monitoring of radiological/functional imaging reports by a coordinating investigator.

Main Results:

• 176 out of 640 patients (28%) demonstrated objective tumor responses.
• Centralized review modified response assessments in 45 of 176 cases (26%).
• This highlights the value of expert medical review in interpreting study data.

Conclusions:

• Centralized medical monitoring significantly impacts the accuracy of objective tumor response evaluation in real-world oncology studies.
• This pragmatic approach is recommended for enhancing clinical trial data quality, especially when formal centralized radiological review is absent.
• The findings support the improved assessment of novel anticancer therapies in pediatric and young adult oncology.