PATIENTS DEFINING WHAT MATTERS: A CROSS-SECTIONAL SURVEY WITH A DISCRETE-CHOICE EXPERIMENT ON UNMET MEDICAL NEED
Author(s)
Joshua Coulter, MA1, LEO RUSSO, PhD2, Mark Cullen, MD3, Aida Habtezion, MD, MSc4, Leila Grace Lackey, DEnv, MHS1, Savanna Darnell, BS1, Gwyneth Mogg-Hall, MPH5, Corin Boysen, B.S., Biology2, Yaa Ababio, MPH1, Anika Gupta, B.S., Human Science1, Brett Hauber, PhD1.
1Pfizer, New York, NY, USA, 2Pfizer, Collegeville, PA, USA, 3Independent Researcher and Former Professor of Medicine, Biomedical Data Science and Epidemiology, Stanford University, Los Altos Hills, CA, USA, 4Department of Medicine, Division of Gastroenterology and Hepatology, Stanford University School of Medicine, Stanford, CA, USA, 5Pfizer, Cambridge, MA, USA.
1Pfizer, New York, NY, USA, 2Pfizer, Collegeville, PA, USA, 3Independent Researcher and Former Professor of Medicine, Biomedical Data Science and Epidemiology, Stanford University, Los Altos Hills, CA, USA, 4Department of Medicine, Division of Gastroenterology and Hepatology, Stanford University School of Medicine, Stanford, CA, USA, 5Pfizer, Cambridge, MA, USA.
OBJECTIVES: Characterizing unmet medical need (UMN) is critical to drug development, influencing research prioritization, regulatory approval, and reimbursement. Multiple frameworks assess UMN, but it is not clear whether the elements of these frameworks reflect patients’ views of UMN. We aimed to quantify the relative importance to patients of six dimensions that define UMN.
METHODS: A cross-sectional, web-based discrete-choice experiment (DCE) was administered to adult residents of the United States with a diagnosed medical condition. Each participant completed nine experimentally designed DCE tasks. Each task asked respondents to indicate which of two hypothetical medical conditions had the greater UMN. Conditions were characterized by different levels of UMN attributes: years of life lost (YLL), symptom burden, disease duration, treatment satisfaction, treatment access, and disease prevalence. Individual preference weights were estimated using hierarchical Bayes. Relative attribute importance (sum to 100%) was calculated as the maximum utility difference of each attribute (based on the mean of individual preference weights) divided by the sum of utility differences across all attributes.
RESULTS: Of 14,436 invited, 1,267 completed the survey. The sample was predominantly white, female, and over the age of 55. Differences in mean preference weights were statistically significant within all attributes except prevalence; respondents did not differentiate between rare and common diseases. Treatment access (very difficult vs very easy) was the attribute most associated with UMN (RAI=25.7%), followed by disease duration (22.5%, lifetime vs short), YLL (17.1%, 10 years vs 1 year), treatment satisfaction (16.6%, dissatisfied vs satisfied), symptom burden (15.5%, high vs low), and prevalence (2.5%, very common vs rare).
CONCLUSIONS: Patients associate treatment access and disease duration most with UMN. This study aimed to quantify the relative importance to patients of six dimensions, adapted from a published framework for the study context, that define UMN.
METHODS: A cross-sectional, web-based discrete-choice experiment (DCE) was administered to adult residents of the United States with a diagnosed medical condition. Each participant completed nine experimentally designed DCE tasks. Each task asked respondents to indicate which of two hypothetical medical conditions had the greater UMN. Conditions were characterized by different levels of UMN attributes: years of life lost (YLL), symptom burden, disease duration, treatment satisfaction, treatment access, and disease prevalence. Individual preference weights were estimated using hierarchical Bayes. Relative attribute importance (sum to 100%) was calculated as the maximum utility difference of each attribute (based on the mean of individual preference weights) divided by the sum of utility differences across all attributes.
RESULTS: Of 14,436 invited, 1,267 completed the survey. The sample was predominantly white, female, and over the age of 55. Differences in mean preference weights were statistically significant within all attributes except prevalence; respondents did not differentiate between rare and common diseases. Treatment access (very difficult vs very easy) was the attribute most associated with UMN (RAI=25.7%), followed by disease duration (22.5%, lifetime vs short), YLL (17.1%, 10 years vs 1 year), treatment satisfaction (16.6%, dissatisfied vs satisfied), symptom burden (15.5%, high vs low), and prevalence (2.5%, very common vs rare).
CONCLUSIONS: Patients associate treatment access and disease duration most with UMN. This study aimed to quantify the relative importance to patients of six dimensions, adapted from a published framework for the study context, that define UMN.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR36
Topic
Patient-Centered Research
Disease
No Additional Disease & Conditions/Specialized Treatment Areas