Methodological standards for comparative effectiveness research: considerations for evidentiary review for medicare drug price negotiation.

Abstract

Introduction: The 2022 Inflation Reduction Act (IRA) is expected to result in lower drug prices for Medicare beneficiaries in the United States (US). The Centers for Medicare & Medicaid Services (CMS) released the most recent draft guidance for the Medicare Drug Price Negotiation (DPN) program in May 2024.

Areas covered: In August 2023, the list of 10 drugs selected for the DPN were published and the first round of negotiations are now complete. While the latest CMS guidance highlights the importance of considering study limitations, bias, uncertainty, and generalizability, there were scarce methodological guidelines provided for the comparative effectiveness research (CER) studies. We conducted searches on PubMed and reviewed resources from websites of regulatory authorities.

Expert opinion: Broader domains of data quality, transparency and methodology including study design, outcomes assessment, bias, confounding and uncertainty should be considered when developing methodological guidelines for evidence submitted for DPN. There is opportunity to align with and leverage existing guidelines published by federal and non-federal organizations in the US and globally while customizing the new guidance based on the specific requirements of DPN, keeping in mind CMS's interest in specific populations as well as health equity.