Do Patient-Reported Outcomes Influence Drug Prices? Evidence From the Italian Pricing and Reimbursement System
Author(s)
Vittoria Ardito, MSc, Francesco Benito Malandrini, PharmD, Oriana Ciani, BSc, MSc, PhD.
Center for Research on Health and Social Care Management (CERGAS), SDA Bocconi School of Management, Milano, Italy.
Center for Research on Health and Social Care Management (CERGAS), SDA Bocconi School of Management, Milano, Italy.
OBJECTIVES: Patient-reported outcome measures (PROMs) are increasingly used in clinical trials and regulatory processes to capture patients' perspectives on symptoms and Health-Related Quality of Life. However, their role in pricing/reimbursement decisions remains underexplored. This study investigates whether the presence and characteristics of PROMs in regulatory submissions are associated with drugs’ negotiated prices, focusing on Italy as a case study.
METHODS: All drugs authorized by the European Medicines Agency between 2017 and 2023 were initially considered. We collected data on clinical and regulatory features, presence, type, and role of PROMs, and negotiated prices. Drugs’ characteristics and PROM-related data were extracted from European Public Assessment Reports. Drug prices were extracted from Farmadati, and standardized using two approaches: price per Defined Daily Dose (DDD; N=279); estimated cost per full treatment cycle/year (for drugs lacking a DDD; N=114). Treatment durations were based on posology indications, body weight assumptions, and median progression-free survival. Logarithmic regressions were performed to examine the association between price and PROMs.
RESULTS: Overall, of the 393 drugs considered (21% orphan, 16% generic, 10% biosimilar), 54% included PROMs (6% generic measures, 16% disease-specific measures, 30% both). PROMs were most frequently reported in drugs classified under antineoplastic and immunomodulating agents (ATC L, 51%), followed by nervous system (ATC N, 10%) and alimentary tract and metabolism (ATC A, 9%). Multivariable regression analyses showed no statistically significant association between PROMs’ presence and negotiated prices.
CONCLUSIONS: Despite increasing focus on integrating patients’ perspectives in clinical development and regulatory assessments, our findings suggest that PROMs do not currently influence pricing negotiations in Italy. As clinical endpoints show convergence across new therapies (particularly in oncology), PROMs could discriminate products in terms of their added value. However, the lack of impact on pricing may discourage manufacturers from investing in the systematic collection of PROMs, unless clearer incentives mechanisms are established within reimbursement frameworks.
METHODS: All drugs authorized by the European Medicines Agency between 2017 and 2023 were initially considered. We collected data on clinical and regulatory features, presence, type, and role of PROMs, and negotiated prices. Drugs’ characteristics and PROM-related data were extracted from European Public Assessment Reports. Drug prices were extracted from Farmadati, and standardized using two approaches: price per Defined Daily Dose (DDD; N=279); estimated cost per full treatment cycle/year (for drugs lacking a DDD; N=114). Treatment durations were based on posology indications, body weight assumptions, and median progression-free survival. Logarithmic regressions were performed to examine the association between price and PROMs.
RESULTS: Overall, of the 393 drugs considered (21% orphan, 16% generic, 10% biosimilar), 54% included PROMs (6% generic measures, 16% disease-specific measures, 30% both). PROMs were most frequently reported in drugs classified under antineoplastic and immunomodulating agents (ATC L, 51%), followed by nervous system (ATC N, 10%) and alimentary tract and metabolism (ATC A, 9%). Multivariable regression analyses showed no statistically significant association between PROMs’ presence and negotiated prices.
CONCLUSIONS: Despite increasing focus on integrating patients’ perspectives in clinical development and regulatory assessments, our findings suggest that PROMs do not currently influence pricing negotiations in Italy. As clinical endpoints show convergence across new therapies (particularly in oncology), PROMs could discriminate products in terms of their added value. However, the lack of impact on pricing may discourage manufacturers from investing in the systematic collection of PROMs, unless clearer incentives mechanisms are established within reimbursement frameworks.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR62
Topic
Health Policy & Regulatory
Topic Subcategory
Pricing Policy & Schemes, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas