Purser M, Faulkner E, Paramore C, Daniel G. Leveraging real world evidence to address uncertainty for transformative and curative therapies. Presented at the ISPOR 2019; May 21, 2019. New Orleans, LA.

ISSUE: Uncertainty around magnitude and duration of therapeutic effect has posed the fundamental value challenge for advanced gene- and cell-based therapies that promise transformative or curative effect. Real world evidence (RWE) is proposed as a core solution to bridge evidentiary uncertainty at all stages, ranging from regulatory to reimbursement and risk sharing. While some models have long been applied to scenarios like single-arm oncology studies, others such as methodologies for demonstrating durability of effect over years or a lifetime are beginning to evolve and will be driven by RWE. However, are our RWE methods currently evolved or accepted as solutions to fill these gaps? Are our global health systems ready to embrace this shift? Do the issues differ when considering rare disease versus oncology examples, the two major areas of growth for these technology types?

OVERVIEW: This panel as an ISPOR Precision Medicine Special Interest Group Forum, will debate how and to what extent RWE approaches can be to address uncertainty across the cell and gene therapy lifecycle. Eric Faulkner will open with an overview of evidence uncertainties associated with transformative therapies where RWE can address gaps, highlighting results of a recent global landscape analysis and review of 100 health technology assessments spanning five global markets. Clark Paramore will offer a global manufacturer perspective and explore how manufacturers are leveraging RWE approaches to demonstrate value and support new therapy acceptance and uptake. Greg Daniel will discuss the policy levers needed to integrate RWE at the regulatory and payer level, highlighting lessons from ongoing US work on transformative and curative therapies and recent initiatives such as the FDA 21st Century Cures Act as tools expand use of RWE.

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