Moderator: Molly Purser, PhD, Associate Director, Health Economics, RTI Health Solutions, Research Triangle Park, NC, USA
Speakers: Eric Faulkner, MPH, Vice President, Precision and Transformative Technology, Evidera, Bethesda, MD, USA; Clark Paramore, MSPH, Head of Value Demonstration, bluebird bio, Cambridge, MA, USA; Gregory Daniel, PHD, MPH, Deputy Director, Policy, Duke-Robert J. Margolis, MD, Center for Health Policy, Washington, DC, USA
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. This panel will debate how and to what extent RWE approaches can be used to address uncertainty across the cell and gene therapy lifecycle. After an overview of evidence uncertainties associated with transformative therapies where RWE can address gaps is presented, we will explore how manufacturers are leveraging RWE approaches to demonstrate value and discuss the policy levers needed to integrate RWE at the regulatory and payer level. Lastly, we will highlight lessons from ongoing US work on transformative and curative therapies and recent initiatives.
Conference/Value in Health Info
2019-05, ISPOR 2019, New Orleans, LA, USA