The USC Leonard D. Schaeffer Center for Health Policy & Economics has featured a four-part series on precision medicine. On June 17th Dr. Kay Larholt, Director of Research for NEWDIGS, was one of the panelists for the session entitled Measuring Real-World Effectiveness and Value along with Dr. Megan O’Brien, Executive Director, Merck Center for Observational and Real-World Evidence.
Dr. Darius Lakdawalla, Schaeffer Center Director of Research, moderated the discussion that focused on the current state of Real-World Evidence generation for value assessment and how to incorporate RWE into decision-making at multiple levels – policy, value assessment and clinical.
A key question that was discussed in some detail was what role, if any, the government should play in RWE production for value assessment of biopharmaceuticals. If RWE in this context is considered a public good, then some might argue that government should fund its production. Along these lines, reference was made to the FDA’s Sentinel Initiative, launched in 2008 in response to the FDA Amendments Act (FDAA) passed by Congress, requiring the FDA to enhance post market safety surveillance for the U.S. The resulting national Sentinel System links and analyzes safety data from multiple sources. The question was raised whether a similar type of effort could be made in the realm of treatment effectiveness and value assessment. An additional consideration is that the government wears another key stakeholder hat related to value assessment – that of a major payer through CMS. While no conclusion was drawn, the perspectives offered good food for thought as we consider potential paths to sustainability for the platforms we are designing in LEAPS.
The discussion highlighted some of the key work of the LEAPS project, i.e. whether the incentives of the down-stream stakeholders (payers, providers, patients) could be aligned to produce real-world evidence that would be fit-for-purpose for the decisions that they need to make to ensure that patients get timely and targeted access to new and important biomedical innovations.