Therapeutic benefit of the most expensive drugs covered by Medicare and Medicaid.

Introduction: The United States began drug pricing negotiations in 2024 under the Affordable Care Act. Evaluating the therapeutic benefit of drugs will help guide pricing decisions and are an opportunity to improve healthcare affordability. Discussion: We conducted a cross-sectional study of the 50 most expensive drugs by dosage unit covered by Medicare Part B, Medicare Part D, and Medicaid in 2022. We reported their level of clinical benefit as evaluated by health technology assessment agencies in France and Germany, their level of innovation as rated by the Food and Drug Administration of the United States, and their safety and effectiveness ratings as measured by Prescrire International, an independent French organisation that evaluates medicines. Our study found that among the 50 most expensive drugs in the U.S. covered by Medicare and Medicaid in 2022, 28 (56%) were rated by French and German HTAs as having low therapeutic benefit and most (n = 20; 40%) were rated by Prescrire International as having poor effectiveness-safety ratings. Almost all (n = 48; 96%) drugs were ineligible under current exclusion criteria for price negotiations. Conclusion: Many of the most expensive drugs were rated as having low added clinical benefit. As the US begins price negotiations under the Inflation Reduction Act, understanding the clinical value of drugs may help inform debates over drug affordability.