Use of real-world evidence in the Medicare Drug Price Negotiation Program: A checklist for the Centers for Medicare and Medicaid Services and manufacturers.

Under the Inflation Reduction Act's (IRA's) - Medicare Drug Price Negotiation Program, the Centers for Medicare & Medicaid Services' (CMS's) "maximum fair price" must be informed by evidence on factors such as therapeutic advance of the selected drug compared with its alternative, comparative effectiveness across clinical and patient-reported outcomes, the impact on specific populations, and the ability to address unmet medical needs. This paper describes how real-world evidence could improve CMS decision-making and creates a best practices checklist to help CMS evaluate the quality of any manufacturer-submitted evidence. The checklist was developed in four steps: (i) identification of the IRA requirements for determining the maximum fair price through a review of official guidance from CMS, (ii) assessment of provisions that could be supported by real-world evidence (RWE) in addition to clinical trial evidence, (iii) literature review on existing best-practice guidelines relevant to RWE, and (iv) consolidation of these RWE guidelines into a checklist through a series of web conference discussions among experts. The checklist aims to improve the quality of the information available to CMS during the drug price negotiation process.