A REVIEW ON THE APPLICATION OF PATIENT-REPORTED OUTCOMES IN THE LABELING OF NEW ONCOLOGY DRUGS
Author(s)
Dan Wang, Bachelor1, Ying Wang, None2, Yixuan Chen, None1, Shichao Jia, None1, Yingpei Zou, None1, Hanwen Cong, None1, Nan Yang, PhD1;
1West China School of Pharmacy, Sichuan University, Chengdu, China, 2School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, China
1West China School of Pharmacy, Sichuan University, Chengdu, China, 2School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, China
OBJECTIVES: To analyze the application of Patient-Reported Outcomes (PROs)in the labeling of new oncology drugs in the United States (US), European Union (EU), and China, and to compare presentation differences of PROs for drugs marketed across all three regions.
METHODS: A retrospective comparative study was conducted to analyze the labeling of new oncology drugs first approved in the US (since 2009), EU (since 2005), and China (since 2021), up to 2024. Study start dates were based on regulatory publications in each jurisdiction. We extracted and analyzed whether PROs were mentioned and, if so, the details of their presentation (e.g., scales, results).
RESULTS: The analysis included 165 (US), 153 (EU), and 104 (China) drug labels, among which 7.27%, 31.37%, and 10.58% contained explicit PRO information, respectively. Among these PRO-containing labels, detailed presentation was observed in 66.67% (US), 43.5% (EU), and 41.2% (China) of cases. Temporal analysis revealed a rise-and-fall pattern in the EU, peaking around 2015, whereas the US consistently maintained low levels. Among the 25 drugs approved in all three regions, PROs were included in 8 EU and 4 Chinese labels, but in none of the US labels. Within the subset of three drugs that contained PROs in both EU and Chinese labels, two presented data inconsistently. The most commonly used PRO measures include the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30), the Brief Pain Inventory Short Form (BPI-SF), and the European Quality of Life Five-Dimension Scale (EQ-5D). Non-small cell lung cancer, prostate cancer, and breast cancer were the indications most commonly associated with PROs.
CONCLUSIONS: PROs are important in oncology labeling but vary regionally. Inconsistencies in PRO-related information across labels for the same drug marketed in different regions are common. Future research should retrieve regulatory review reports to facilitate international harmonization of PRO presentation.
METHODS: A retrospective comparative study was conducted to analyze the labeling of new oncology drugs first approved in the US (since 2009), EU (since 2005), and China (since 2021), up to 2024. Study start dates were based on regulatory publications in each jurisdiction. We extracted and analyzed whether PROs were mentioned and, if so, the details of their presentation (e.g., scales, results).
RESULTS: The analysis included 165 (US), 153 (EU), and 104 (China) drug labels, among which 7.27%, 31.37%, and 10.58% contained explicit PRO information, respectively. Among these PRO-containing labels, detailed presentation was observed in 66.67% (US), 43.5% (EU), and 41.2% (China) of cases. Temporal analysis revealed a rise-and-fall pattern in the EU, peaking around 2015, whereas the US consistently maintained low levels. Among the 25 drugs approved in all three regions, PROs were included in 8 EU and 4 Chinese labels, but in none of the US labels. Within the subset of three drugs that contained PROs in both EU and Chinese labels, two presented data inconsistently. The most commonly used PRO measures include the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30), the Brief Pain Inventory Short Form (BPI-SF), and the European Quality of Life Five-Dimension Scale (EQ-5D). Non-small cell lung cancer, prostate cancer, and breast cancer were the indications most commonly associated with PROs.
CONCLUSIONS: PROs are important in oncology labeling but vary regionally. Inconsistencies in PRO-related information across labels for the same drug marketed in different regions are common. Future research should retrieve regulatory review reports to facilitate international harmonization of PRO presentation.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR113
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology