Are Existing Descriptions of Unmet Medical Need Fit for Purpose in the CMS Drug Negotiation Program Context?
Author(s)
Antal T. Zemplenyi, MSc, PhD1, Michael DiStefano, PhD1, David Ameyaw, MSc2, Harry Gyamfi, Masters1, Hanke Zheng, MS, PhD3, Omar A. Escontrias, MPH, DrPH4, Silke Schoch, BA, MA4, Jon Campbell, PhD3, Robert B. McQueen, BA, MA, PhD5;
1University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, Aurora, CO, USA, 2Syreon, Ghana, Ghana, 3National Pharmaceutical Council, Washington, DC, USA, 4National Health Council, Washington, DC, USA, 5University of Colorado Skaggs School of Pharmacy and Pharmaceutical Science, Assistant Professor, Aurora, CO, USA
1University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, Aurora, CO, USA, 2Syreon, Ghana, Ghana, 3National Pharmaceutical Council, Washington, DC, USA, 4National Health Council, Washington, DC, USA, 5University of Colorado Skaggs School of Pharmacy and Pharmaceutical Science, Assistant Professor, Aurora, CO, USA
OBJECTIVES: The extent to which a treatment addresses an unmet medical need (UMN) is one criterion that the Centers for Medicare and Medicaid Services (CMS) must consider when negotiating the price of a treatment. CMS refers to the Food and Drug Administration’s (FDA) description of filling an UMN. In this study, we 1) conducted a targeted literature review to identify and categorize potential elements of UMN, and 2) compared these elements to existing descriptions of UMN with the goal of informing CMS’ approach to determining the extent to which an UMN is addressed.
METHODS: A targeted literature review was conducted using PubMed, focusing on original research studies published after 2014 in English. Search terms were developed to identify studies that described elements broadly related to UMN in the context of pharmaceutical treatments. Following data extraction, we categorized identified elements according to commonly recognized domains. Elements not describing a need or barrier were excluded. The identified domains and elements were compared with descriptions of UMN from the FDA.
RESULTS: A total of 48 original research studies were identified. After standardization and cleaning, 55 elements were grouped into eight domains: quality of life, clinical effectiveness, economic burden on patient/caregiver, societal perspective priorities, economic burden on society, treatment administration attributes impacting treatment decisions, treatment availability, and health system functioning. While there was partial overlap between FDA’s UMN description and the clinical effectiveness domain, the FDA’s UMN description does not adequately address quality-of-life, patient administration attributes, and UMN domains and elements from caregiver and societal perspectives.
CONCLUSIONS: Relying on FDA guidance may lead CMS to overlook important elements of UMN in its price negotiations. Future work could prioritize specific elements of UMN from the perspectives of different stakeholders, including caregivers and patient organizations in order to better inform CMS price negotiations.
METHODS: A targeted literature review was conducted using PubMed, focusing on original research studies published after 2014 in English. Search terms were developed to identify studies that described elements broadly related to UMN in the context of pharmaceutical treatments. Following data extraction, we categorized identified elements according to commonly recognized domains. Elements not describing a need or barrier were excluded. The identified domains and elements were compared with descriptions of UMN from the FDA.
RESULTS: A total of 48 original research studies were identified. After standardization and cleaning, 55 elements were grouped into eight domains: quality of life, clinical effectiveness, economic burden on patient/caregiver, societal perspective priorities, economic burden on society, treatment administration attributes impacting treatment decisions, treatment availability, and health system functioning. While there was partial overlap between FDA’s UMN description and the clinical effectiveness domain, the FDA’s UMN description does not adequately address quality-of-life, patient administration attributes, and UMN domains and elements from caregiver and societal perspectives.
CONCLUSIONS: Relying on FDA guidance may lead CMS to overlook important elements of UMN in its price negotiations. Future work could prioritize specific elements of UMN from the perspectives of different stakeholders, including caregivers and patient organizations in order to better inform CMS price negotiations.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR26
Topic
Health Policy & Regulatory
Topic Subcategory
Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas