Application of the target trial emulation framework to external comparator studies.

Area of Science:

• Health economics and outcomes research
• Pharmacoepidemiology
• Causal inference

Background:

• External comparator (EC) studies are crucial for evaluating emerging pharmacological treatments for regulatory and health technology assessment (HTA).
• The reliability of evidence from EC studies can be inconsistent, posing challenges for decision-making.

Purpose of the Study:

• To outline the application of the target trial emulation (TTE) framework to improve the design and analysis of EC studies.
• To enhance the clarity, transparency, and reliability of evidence generated from EC studies.

Main Methods:

• The study describes how to apply the existing TTE framework, a causal inference method, to EC studies.
• This involves specifying an ideal target trial protocol and emulating its key elements in real-world (RW) settings.
• Practical recommendations and special considerations for applying TTE to EC studies are provided.

Main Results:

• The TTE framework offers a structured approach to designing and analyzing EC studies.
• Application of TTE can lead to more robust and reliable real-world evidence.
• The paper details the adaptation of TTE components for EC study contexts.

Conclusions:

• The target trial emulation framework can significantly improve the quality of evidence from external comparator studies.
• Implementing TTE enhances the transparency and reliability of real-world evidence for treatment evaluations.
• This approach supports better regulatory and HTA decision-making regarding new pharmacological treatments.