Addressing RWE Transportability Concerns in Health Care Decision-Making. How to Do It Right?

Background: A key step in assessing the value of new technologies in health care decision making is establishing the comparative efficacy and safety versus standard of care. The potential of real-world evidence (RWE) to supplement trial findings is well documented, as RWE studies potentially better reflect the target population in the decision problem. While RWE studies may increase external validity by providing context on the generalisability of trial findings, variation in availability and quality of real-world data turns decision makers to consider “transferring” data from other countries and settings. Whether and how RWE can be reliably extended to target populations outside of the studied population and geography, becomes a key question.

Objectives: This symposium brings together a global panel representing the industry, academia, and stakeholder perspectives, to introduce methods of transportability analysis, and illustrate their application through presentation of real-life case studies. Attendees can expect to learn how well-designed transportability analyses could be used in decision making to address concerns about external validity in RWE studies.

Description:
Traditional methods to control for confounding can be used to address transportability concerns, but how these methods may be applied in decision-making is not well understood. Alessandria Strübing will present the industry challenges in identifying suitable local real-world data that are acceptable by decision makers (15min). Evie Merinopoulou will follow with a presentation of a transportability analysis framework that could potentially address uncertainties in decision making stemming from the lack of local data (15min).
Panellists will draw from real-life case studies and experiences to illustrate how transportability methods can be applied in practice. Alind Gupta will present a successful case that used adjustment for known effect modifiers to evaluate the transferability of overall survival estimates from a US population to Canadian patients (15min). Blythe Adamson will present case studies from US to different geographies and draw learnings on the applicability and validity of those methods in transferring US RWE to other European target populations (15min).
Panellists will discuss the need for a multidisciplinary team and domain expertise to validate assumptions about comparability of relevant populations that cannot be verified empirically.
Stephen Duffield will discuss key considerations for the acceptability of these approaches from a decision maker perspective and how transportability methods fit in the updated NICE RWE framework (15min). The symposium will end with a 15min Q&A session.