Oncology Specific Considerations in Conduct of Pragmatic Trials: Lessons Learnt From Clinicaltrials.Gov Registry Audit

Speaker(s)

Bajwa S1, Chhaya V2, Khambholja K3
1Catalyst Clinical Research, Panchkula, HR, India, 2Catalyst Clinical Research, Thiruvananthapuram, Kerala, India, 3Catalyst Clinical Research, Vadodara, India

Presentation Documents

OBJECTIVES: Pragmatic trials are essential in oncology to complement traditional randomised controlled trials (RCTs) with real-world data (RWD), providing a comprehensive understanding of treatment effects in diverse populations. Despite their importance, there is limited literature on the practical conduct of oncology pragmatic trials. This registry audit aims to assess the methodological aspects of these trials to inform future real-world evidence (RWE) methodology guidance in oncology.

METHODS: We analysed data from clinicaltrials.gov using "Cancer" and "Pragmatic Trial" as search terms. Descriptive analysis was performed on demographics, study design, primary and secondary outcomes, and geographical locations, presented via tables, bar graphs, and pie charts. Study designs were mapped against the European Medicines Agency (EMA's) RWE draft reflection paper (2024) to identify inter-regional methodological differences.

RESULTS: Out of 214 pragmatic trials identified, 76 were US-based. Only 13 trials had a sample size greater than 5000, highlighting the need for large-scale pragmatic trials. Most trials (192/214; 89.7%) targeted adults, with a female predominance (F: 34 trials vs. M: 11 trials). Thirteen (6.1%) trials included all age groups. Late-phase trials (56/214; 26.2%) outnumbered early-phase trials (10/214; 4.7%). Most studies (193/214; 90.2%) were interventional, with 10.7% (23/214) single-arm and 7.0% (15/214) non-randomised trials. Interventions included pharmacological (52/214; 24.3%), behavioural (63/214; 29.4%), and other (82/214; 38.3%) interventions, including medical technology, surgical, and health system interventions. Overall survival and progression-free survival were primary outcomes in 9 (4.2%) and 8 (3.7%) trials, respectively. Mapping against the EMA RWE reflection paper provided minimal additional insights due to limited methodology details.

CONCLUSIONS: The registry audit of oncology pragmatic trials based on RWE revealed significant evidence gaps such as missing demographic details, incomplete reporting of bias correction methods, and fewer large-scale trials. The analysis highlights the diversity and methodological challenges in oncology pragmatic trials, emphasising the need for detailed guidelines to optimise RWE generation.

Code

SA21

Topic

Study Approaches

Topic Subcategory

Registries

Disease

Oncology