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Series: Pragmatic trials and real world evidence: Paper 3. Patient selection challenges and consequences
Katrien Oude Rengerink1, Shona Kalkman1, Susan Collier2
1Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, Utrecht 3584 CG, The Netherlands.
Abstract:
This paper addresses challenges of identifying, enrolling, and retaining participants in a trial conducted within a routine care setting. All patients who are potential candidates for the treatments in routine clinical practice should be considered eligible for a pragmatic trial. To ensure generalizability, the recruited sample should have a similar distribution of the treatment effect modifiers as the target population. In practice, this can be best achieved by including-within the selected sites-all patients without further selection. If relevant heterogeneity between subgroups is expected, increasing the relative proportion of the subgroup of patients in the heterogeneous trial could be considered (oversampling) or a separate trial in this subgroup can be planned. Selection will nevertheless occur. Low enrollment and loss to follow-up can introduce selection and can jeopardize validity as well as generalizability. Pragmatic trials are conducted in clinical practice rather than in a dedicated research setting, which could reduce recruitment rates. However, if a trial poses a minimal burden to the physician and the patient and routine clinical practice is maximally adhered to, the participation rate may be high and loss to follow-up will not be a specific problem for pragmatic trials.
Insights
Pragmatic trials in routine care settings face enrollment and retention challenges. Minimizing participant burden and adhering to clinical practice maximizes recruitment and generalizability for these crucial studies.
Area of Science:
- Clinical Trials
- Health Services Research
- Epidemiology
Background:
- Conducting clinical trials in routine care settings presents unique challenges for participant identification, enrollment, and retention.
- Ensuring the generalizability of pragmatic trial findings requires a recruited sample that mirrors the target population's distribution of treatment effect modifiers.
Purpose of the Study:
- To address the challenges of participant recruitment and retention in pragmatic trials conducted within routine clinical practice.
- To provide strategies for maximizing the validity and generalizability of pragmatic trial results.
Main Methods:
- Advocating for the inclusion of all potential candidates within selected sites to avoid further selection bias.
- Considering oversampling or separate trials for specific patient subgroups if heterogeneity is anticipated.
- Emphasizing minimal burden on physicians and patients to enhance participation rates.
Main Results:
- Selection of participants is unavoidable but can be minimized through inclusive enrollment strategies.
- Low enrollment and loss to follow-up can compromise the validity and generalizability of trial outcomes.
- Pragmatic trials adhering closely to routine clinical practice and minimizing participant burden can achieve high participation rates.
Conclusions:
- Pragmatic trials require careful consideration of enrollment and retention strategies to ensure representativeness.
- Minimizing participant burden and integrating trials into routine care are key to successful pragmatic trial execution.
- Adherence to clinical practice and inclusive recruitment enhance the validity and generalizability of findings from pragmatic trials.
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