How Does Real-World Evidence Support Regulatory and HTA Agency Assessments and Beyond?

OBJECTIVES: To explore whether regulatory and health technology assessment (HTA) agencies across Europe and the US are aligned in their considerations for the use of real-world evidence (RWE) in new drug submissions and how that supports patient access strategies.

METHODS: The most recent guidance documents published by the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), the National Institute for Health and Care Excellence (NICE), Haute Autorité de Santé (HAS), and the Federal Joint Committee or Gemeinsamer Bundesausschuss (G-BA) were reviewed to capture perspectives on the value of RWE with their assessment processes and beyond.

RESULTS: Historically, the FDA and the EMA used RWE to provided external comparator data for single-arm trials, and to collect post-marketing data; however, advances in availability of data and analytic methods mean a potentially more prominent role for RWE within future assessments. HTA guidance suggests RWE can fill evidence gaps (eg, disease natural history, treatment patterns, outcomes for existing treatments), and validate assumptions around long-term clinical and economic value. However, acceptance of RWE differs between HTA agencies, with more limited acceptance where the assessment paradigm is grounded in comparative trial evidence. Uncertainties in RWE (eg, low data quality. prevalence of bias/confounding) and lack of trust were cited as barriers to meeting payer expectations. Aside from HTA reassessment processes in some markets, little was mentioned regarding value of RWE beyond the initial HTA.

CONCLUSIONS: As innovative treatments launch and payers and regulators provide more comprehensive guidance, perspectives on the use of RWE in evidence submissions will likely evolve rapidly, providing opportunity for more comprehensive data analysis and evidence generation in a timely manner. This will support launch and patient access strategies where RWE can provide evidence for regulatory submissions and national HTA as well as the emerging needs for JCA and broader patient access.