Opportunities for CMS to further specify and broaden its approach to assessing unmet medical need in the context of the Medicare drug price negotiation program

The United States Inflation Reduction Act of 2022 established the Medicare Drug Price Negotiation Program, directing the Centers for Medicare and Medicaid Services (CMS) to establish "maximum fair prices" for select drugs. In arriving at maximum fair prices, CMS is required to consider several criteria, including the extent to which a selected drug addresses unmet medical need (UMN). Through a targeted literature review of 48 original research studies, we identified and categorized 40 potential elements of UMN as they relate to pharmaceuticals, as well as treatment-related barriers to addressing this UMN, from the patient, caregiver, and societal perspectives. We synthesized these elements of UMN into seven domains: (1) traditional clinical effectiveness, (2) impacts on patient, caregiver, or family quality of life, (3) economic burden on the patient, caregiver, or family, (4) economic burden on society, (5) societal perspective elements, (6) elements of treatment administration, and (7) availability of other treatments. Comparing these elements with the United States Food and Drug Administration guidance regarding UMN reveals potential key gaps in the current CMS approach to assessing UMN in the Medicare Drug Price Negotiation Program, particularly regarding domains 2 through 6, including quality of life, economic burdens, societal elements, and treatment administration.