ISSUE: The growing number of highly specialized products (cell-based and gene therapies) and the focus on personalized medicine are rapidly expanding trends in the drug development landscape. These products often achieve regulatory approval based on single-arm trials due to ethical considerations or difficulty of recruiting patients, thus imposing a challenge to HTA bodies to conduct comparative effectiveness research. Few HTA submissions based on single-arm trials have been successful in convincingly establishing comparative effectiveness. During this assessment process, decision makers have shown preference for local data reflecting clinical practice. However, this preference can bring methodological challenges depending on the data local availability and quality and present operational difficulties when industry is planning for multiple submissions across different countries. Further, firm guidelines on what is considered convincing evidence is lacking, and the quality assessment criteria of a “good” external control arm differ substantially across HTA bodies.

OVERVIEW

: Several manufacturers have already supported their regulatory and HTA filings using single-arm trials while creating an external real-world arm (historical trial data and/or using real-world-evidence [RWE] sources) to establish comparative effectiveness. However, selecting the most appropriate source for this control arm remains challenging. When a RWE control arm is used, HTA authorities show a preference for local data. For manufacturers, it is challenging to ensure patient representation in a specific location while using “good” quality data and making efficiencies in resources. This panel will debate, from an industry, academic and HTA perspective (15 min each presentation) if a control arm based on a single RWE data source can be achievable and meet the requirements of different HTA bodies; whether quality or locality of RWE data is the main driver in HTA acceptance and whether current HTA guidance can inform decisions in this area. 10min discussion will follow. Stakeholders (industry, researchers, payers) will benefit from this panel.