ISSUE: The implementation of advanced methods in real-world evidence (RWE) generation presents opportunities and challenges from the perspectives of HTA/regulatory submissions and industry stakeholders, with potentially substantial resource requirements, uncertainties, and risks.

From an HTA and regulatory standpoint, RWE faces hurdles stemming from data quality concerns, including bias, small sample sizes, and interpretability. HTA/regulatory bodies demand robust evidence to support decision-making, necessitating transparency, reproducibility, and alignment with established guidelines. To address these challenges, innovative methodologies such as quantitative bias analysis (QBA), target trial emulation, Bayesian borrowing, and transportability have all been proposed, even being cited in official guidelines. However, why has the implementation of these methods in-practice been limited?

This issue panel sets the stage for a comprehensive discussion, where experts from HTA/regulatory bodies, industry, and academia will share insights, experiences, and best-practices in navigating the evolving landscape of RWE methodologies. Through these multiple perspectives and collaborative solutions, stakeholders can overcome barriers to the uptake of advanced methods in RWE generation, paving the way for more robust, transparent, and impactful evidence to inform healthcare decision-making.

OVERVIEW: Dr. Duffield begins by describing the current landscape of submissions regarding advanced methods, and the challenges NICE faces in their evaluations (15m). Dr. Dixon will prove an industry perspective on the obstacles related to resource constraints, and challenges with organizational inertia in embracing novel approaches. Prof. Dickerman will highlight the foundational elements needed and hurdles in executing advanced methods such as target trial emulation (20m). Ms. Hsu will discuss the time and resource implications of implementing advanced methods through the medium of past case studies (15m).

By delving into the critical challenges of uptake barriers and potential solutions in leveraging advanced methodologies for RWE through multiple perspectives, this issue panel aims to foster collaboration and innovation in evidence generation.