More Than One Way to Measure? A Casuistic Approach to Cancer Clinical Trials

Area of Science:

• Oncology
• Regulatory Science
• Personalized Medicine

Background:

• Personalized medicine presents challenges to traditional oncological treatment testing.
• Randomized controlled trials (RCTs) are currently inadequate for evaluating targeted therapies.
• The Food and Drug Administration (FDA) still mandates RCTs for market approval.

Purpose of the Study:

• To question the reliance on statistical inferences in regulatory decision-making.
• To discuss the impact of tumor heterogeneity on drug assessment.
• To propose an alternative approach for regulatory evaluation.

Main Methods:

• Critical analysis of the current regulatory paradigm.
• Examination of the implications of tumor heterogeneity.
• Literature review on regulatory challenges and decision-making.

Main Results:

• Current regulatory frameworks struggle with targeted therapies due to tumor heterogeneity.
• The paradigm's resistance to change stems from various criticisms.
• Statistical inferences alone are insufficient for regulatory decisions.

Conclusions:

• The casuistic method offers a potential solution for regulatory decision-making.
• Integrating case-based reasoning can improve assessment of new oncological treatments.
• Adapting regulatory approaches is crucial for personalized medicine advancements.