Study Design Trends in Registry-Based Oncology Studies: Analysis of HMA-EMA Real-World Data Catalogs
Author(s)
VATSAL CHHAYA, MSc1, Shaurya Deep Bajwa, MSc MBA2, KAPIL KHAMBHOLJA, PhD1.
1Catalyst Clinical Research, Baroda, India, 2Catalyst Clinical Research, Thiruvananthapuram, India.
1Catalyst Clinical Research, Baroda, India, 2Catalyst Clinical Research, Thiruvananthapuram, India.
OBJECTIVES: The EMA-HMA Real-World Data (RWD) catalogues provide structured information on patient registries and other data sources across Europe to support non-interventional study design. In alignment with EMA guidance on the use of registries for post-authorization evidence generation, this study aimed to characterize registry-based oncology studies captured in the EMA-HMA RWD catalogues, focusing on data source selection, design features, and regulatory alignment.
METHODS: A descriptive analysis of the EMA-HMA RWD catalogues was conducted. Eligible studies were non-interventional, oncology-focused, and based on patient registries. Study attributes were extracted, including cancer type, geographic scope, regulatory intent (e.g., inclusion in EU Risk Management Plan), data source type (secondary, linked), study design (cohort, case-control), objectives, and inclusion of special populations. Data extraction was conducted by a primary reviewer and independently verified. Study objectives were categorized into non-mutually exclusive domains. Descriptive statistics summarized key design elements; no inferential testing was performed.
RESULTS: Twenty-five registry-based oncology studies met inclusion criteria. Breast (24%), prostate (16%), and leukemia (12%) were the most studied cancer types. Most studies were conducted in Europe (85%), with fewer involving North America (12%) or Asia (3%). All studies used secondary (88%) or linked (12%) data sources; none collected primary data. Cohort designs predominated (84%), followed by case-control (12%). Reported objectives included epidemiology (21%), safety (21%), drug utilization (17%), and effectiveness (17%). Special populations included patients with organ impairment, immunocompromised status, pregnancy, or distinct subtypes. Over half (56%) supported an EU Risk Management Plan.
CONCLUSIONS: This study leveraged the HMA-EMA RWD catalogues to identify key design attributes and trends in registry-based oncology studies. Systematic use of these catalogues can guide future oncology RWE designs by helping sponsors identify fit-for-purpose sources, integrate linked data, and ensure regulatory alignment.
METHODS: A descriptive analysis of the EMA-HMA RWD catalogues was conducted. Eligible studies were non-interventional, oncology-focused, and based on patient registries. Study attributes were extracted, including cancer type, geographic scope, regulatory intent (e.g., inclusion in EU Risk Management Plan), data source type (secondary, linked), study design (cohort, case-control), objectives, and inclusion of special populations. Data extraction was conducted by a primary reviewer and independently verified. Study objectives were categorized into non-mutually exclusive domains. Descriptive statistics summarized key design elements; no inferential testing was performed.
RESULTS: Twenty-five registry-based oncology studies met inclusion criteria. Breast (24%), prostate (16%), and leukemia (12%) were the most studied cancer types. Most studies were conducted in Europe (85%), with fewer involving North America (12%) or Asia (3%). All studies used secondary (88%) or linked (12%) data sources; none collected primary data. Cohort designs predominated (84%), followed by case-control (12%). Reported objectives included epidemiology (21%), safety (21%), drug utilization (17%), and effectiveness (17%). Special populations included patients with organ impairment, immunocompromised status, pregnancy, or distinct subtypes. Over half (56%) supported an EU Risk Management Plan.
CONCLUSIONS: This study leveraged the HMA-EMA RWD catalogues to identify key design attributes and trends in registry-based oncology studies. Systematic use of these catalogues can guide future oncology RWE designs by helping sponsors identify fit-for-purpose sources, integrate linked data, and ensure regulatory alignment.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
RWD175
Topic
Health Policy & Regulatory, Real World Data & Information Systems, Study Approaches
Topic Subcategory
Distributed Data & Research Networks
Disease
Oncology