REAL WORLD EVIDENCE FOR LABEL EXPANSION: IDENTIFYING OFF-LABEL USE TRENDS IN THE HOSPITAL SETTING
Author(s)
Josine Kuiper, PhD1, Jetty Overbeek, PhD2, Nikita Jeswani, BA, MBA3;
1PHARMO Institute, Senior Business Development Manager, Utrecht, Netherlands, 2PHARMO Institute, Utrecht, Netherlands, 3Lumanity, London, United Kingdom
1PHARMO Institute, Senior Business Development Manager, Utrecht, Netherlands, 2PHARMO Institute, Utrecht, Netherlands, 3Lumanity, London, United Kingdom
OBJECTIVES: To explore patterns of off-label use among high-cost drugs dispensed within the hospital setting using data from the PHARMO Data Network and identify candidate drugs and disease areas for further investigation.
METHODS: A retrospective analysis was conducted using data from the high-cost medicine dataset within hospital data from the PHARMO Data Network using the last five years of data availability (2020-2024). Off-label use was defined as prescribing outside the EMA-approved product label as registered by prescribing specialists in line with Dutch national agreements on appropriate use, pricing, and data collection. Descriptive analyses were used to identify off-label trends over time and therapeutic areas with off-label drug use.
RESULTS: From 2020 to 2024, a total of 10,977,262 drug dispensations were recorded. Of these, 3,129,976 (29%) were prescribed for off‑label indications and this proportion slightly increases over time. Off‑label use was most frequent for oncolytic drugs (51%), followed by immunosuppressants (19%), ophthalmological agents (7%), and antianemic preparations (5%). At the individual drug level, the highest proportions of off‑label prescribing were observed for bevacizumab (9%), aflibercept (6%), paclitaxel (5%), adalimumab (5%), and infliximab (4%).
CONCLUSIONS: Nearly one‑third of all prescriptions in this period occurred outside approved indications and may be candidates for label expansion. These patterns may highlight areas where further evaluation using RWE could support evidence generation for potential label expansions or optimisation of existing indications.
METHODS: A retrospective analysis was conducted using data from the high-cost medicine dataset within hospital data from the PHARMO Data Network using the last five years of data availability (2020-2024). Off-label use was defined as prescribing outside the EMA-approved product label as registered by prescribing specialists in line with Dutch national agreements on appropriate use, pricing, and data collection. Descriptive analyses were used to identify off-label trends over time and therapeutic areas with off-label drug use.
RESULTS: From 2020 to 2024, a total of 10,977,262 drug dispensations were recorded. Of these, 3,129,976 (29%) were prescribed for off‑label indications and this proportion slightly increases over time. Off‑label use was most frequent for oncolytic drugs (51%), followed by immunosuppressants (19%), ophthalmological agents (7%), and antianemic preparations (5%). At the individual drug level, the highest proportions of off‑label prescribing were observed for bevacizumab (9%), aflibercept (6%), paclitaxel (5%), adalimumab (5%), and infliximab (4%).
CONCLUSIONS: Nearly one‑third of all prescriptions in this period occurred outside approved indications and may be candidates for label expansion. These patterns may highlight areas where further evaluation using RWE could support evidence generation for potential label expansions or optimisation of existing indications.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
RWD136
Topic
Real World Data & Information Systems
Disease
No Additional Disease & Conditions/Specialized Treatment Areas