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Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
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Preferred study designs to support a comparative therapeutic strategy question in oncology: a vignette study
Anna Pellat1, Isabelle Boutron2, Elodie Perrodeau2
1Gastroenterology, Endoscopy and Digestive Oncology Unit, Hôpital Cochin, Assistance Publique des Hôpitaux de Paris, 27 rue du Faubourg Saint Jacques, 75014, Paris, France; Université Paris Cité and Université Sorbonne Paris Nord, Inserm, INRAe, Centre for Research in Epidemiology and Statistics (CRESS), Hôpital Hôtel Dieu, 1 Place du Parvis Notre-Dame, 75004, Paris, France.
Journal of Clinical Epidemiology
|July 27, 2024
Summary
Oncologists prefer large observational studies or multicentric emulated trials over monocentric randomized controlled trials (RCTs) for comparative effectiveness research. Study design choices balance scientific rigor with feasibility, cost, and timeliness.
Area of Science:
- Clinical Epidemiology
- Oncology Research
- Health Services Research
Background:
- Comparative effectiveness questions in oncology often lack robust evidence.
- Randomized controlled trials (RCTs) are gold standard but may be infeasible.
- Target trial emulation offers a framework to apply RCT design principles to observational data.
Purpose of the Study:
- To determine oncologists' preferred study design for answering comparative effectiveness questions with limited evidence.
- To evaluate preferences between RCTs and target trial emulation in oncology research planning.
Main Methods:
- Online survey distributed to oncology specialists.
- Vignette-based inquiry presenting six study designs (RCTs vs. emulated trials) with varying characteristics.
- Pairwise comparisons using a Likert scale (-5 to 5) to assess preference for study design, feasibility, and timeliness.
Main Results:
- 213 oncologists participated, primarily from France.
- Monocentric RCTs were consistently least favored across comparisons.
- Multicentric emulated trials were strongly preferred over monocentric emulated trials; large European observational studies received the highest mean preference scores.
Conclusions:
- No single study design was overwhelmingly preferred, but monocentric RCTs were least favored.
- Oncologists' research planning involves a trade-off between scientific validity, feasibility, cost, and time to results.
- The optimal study design balances the need for timely, reliable answers to critical oncology questions.
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