Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and...

Area of Science:

• Health research methodologies
• Regulatory science
• Data analytics

Background:

• Increasing availability of real-world data (RWD) and patient-level data (PLD) from randomized controlled trials (RCTs) enables large-scale secondary analysis.
• Novel analytical methods and study designs are emerging for analyzing and combining these diverse data sources.

Purpose of the Study:

• To outline the necessity for rigorous validation of novel analytical methods used with RWD and PLD.
• To propose a framework for ensuring the acceptability of these methods to regulators and decision-makers.

Main Methods:

• Emphasizes the need for prospective, well-controlled testing and validation of analytical methods, akin to drug evaluation.
• Highlights the European regulators' established methods qualification advice procedure as a suitable platform.
• Stresses the importance of active participation from patient groups and other stakeholders in the validation process.

Main Results:

• The established methods qualification advice procedure provides an efficient and transparent pathway.
• This procedure facilitates the development and validation of novel study designs for secondary data analysis.

Conclusions:

• Rigorous validation is crucial for the acceptance of novel analytical methods by regulators.
• The European methods qualification advice procedure, involving patient groups, offers a robust model for validating new data analysis approaches.