CMS Issues Proposed Guidance Document on Study Protocols That Use Real-World Data

The Centers for Medicare and Medicaid Services (CMS) released a proposed guidance document on study protocols that use real-world data (RWD).  The proposed guidance focuses on studies with RWD sources in the context of Medicare National Coverage Determinations (NCDs) using CMS’s Coverage with Evidence Development (CED) paradigm. The proposed guidance document includes a standardized template for manufacturers or other sponsors to use when developing CED study protocols using RWD. 

CMS states that this proposed standardized template “is part of a broader CMS modernization initiative to provide a more transparent and predictable evidence-generation framework to facilitate Medicare coverage.”  This is connected to the CED guidance finalized by CMS in August 2024. The American College of Radiology® (ACR®) submitted comments on that guidance when it was first proposed in 2023. 

The proposed standardized RWD study protocol template is called the HARPER+. CMS states that the HARPER+ framework “is based on the HARmonized Protocol Template to Enhance Reproducibility (HARPER) but adapted for medical devices and Medicare coverage criteria.” The template has been enhanced with sections for study sponsors to provide additional information to confirm that all CED study standards are met. The standardized format is meant to help CMS facilitate an efficient review of the protocol.

CMS is seeking public comments on the proposed guidance document and template and will respond to comments in the final version of the guidance. Once finalized, the RWD study protocol template will be available to download on the CMS website. CMS strongly encourages prospective study sponsors to use the template once finalized to avoid delays in Medicare coverage.  Written comments should be submitted via email to CAGInquiries@cms.hhs.gov by March 18. We encourage our members to carefully review this proposed guidance and to consider providing input through public comments.

If you have questions regarding NCDs, contact Alicia Blakey, ACR Principal Economic Policy Analyst.