A framework to guide the optimal development and use of real-world evidence for drug coverage and formulary decisions

Summary

It is now widely acknowledged that real-world evidence (RWE) is transforming the process through which healthcare payers make coverage and formulary decisions for pharmaceuticals. However, there remains an uneven match between the hopes for RWE and the reality; payers cite needs for greater relevance, timeliness and transparency in order for RWE to be more useful to them. Increasingly, efforts to design the standards for prospective RWE analyses have convened manufacturers, payers and patient groups to ensure that evidence generated is both rigorous and fully informed by what matters most to patients.

This White Paper, by authors from the Institute for Clinical & Economic Review (MA, USA) and Office of Health Economics (London, UK), presents a new conceptual framework to address three elements largely missing from these earlier efforts focused on defining ‘best practices’ or ‘standards’ for RWE: (1) how to understand the role that contextual factors play in determining how high the evidentiary standard, or ‘bar’ will be in each situation, (2) how to tailor key process and methodological approaches to the height of that evidentiary bar and (3) how to ensure that broader process principles that support transparency are integrated successfully throughout the course of any RWE initiative.