White Paper – Comparative Effectiveness in Motion: Guidance Development and the Growing Acceptance of Novel Methodologies in Health Technology Assessment

The question is no longer whether a treatment works, but how it performs relative to available alternatives.

Victor Laliman-Khara, Hoora Moradian, Nathalie Horowicz-Mehler

Regulators and payers are demanding clearer evidence of relative value and how treatments compare, often when direct head-to-head trials are unavailable. This evidence is critical to health technology assessment (HTA), market access, and reimbursement decisions.

In response, the field of comparative effectiveness has evolved significantly over the past two decades. While early methods were viewed with skepticism, methodological advances plus guidance from HTA bodies have assuaged some of these concerns. More recently, advancements like machine learning and generative AI have further expanded the field.

In this white paper, our experts explore how novel indirect treatment comparison (ITC) methods continue to evolve alongside the demand for complex evidence generation strategies to demonstrate the comparative effectiveness of new health technologies.

Key Topics:

• The expanding application of comparative effectiveness methods
• Growing acceptance of ITC methods by HTA bodies
• Treatment effect modifiers investigation and HTA
• The emerging role of artificial intelligence