The purpose of this programis to encourage investigators to use existing resources to conduct low-cost pragmatic patient-centered randomized controlled trials of interventions targeted at patients, families healthcare providers, communities or health care systems through the integration of RCTs into existing clinical practice settings. While key features of high-quality clinical trials must be maintained (standardized inclusion and exclusion criteria, randomization, study protocol with standardization where appropriate, masking to treatment assignment to the degree possible, and objective assessment of outcomes), separate research infrastructure and staff should be minimized.
To be responsive to this program, applicants must:
- Utilize existing electronic resources such as electronic health records, personal health records, or patient registries for key aspects of the trial such as identification, recruitment and consent of eligible patients; monitoring compliance or obtaining interim study data when necessary; and ascertainment of study outcomes.
- Conduct the trial in a real-world setting that includes a broad representation of patients who would have the condition under study including those with multiple morbidities, at high risk, and vulnerable.
- Utilize trial endpoints that are relevant to patients and clinicians (e.g., morbidity, hospitalizations, clinical events, and quality of life). Surrogate (such as blood biomarkers) or intermediate outcomes are discouraged; they may be acceptable when the proposed measures are valid and adequate rationale is provided. Secondary endpoints may include analyses focused on decreasing or maintaining utilization while improving health outcomes.
- Minimize separate infrastructure utilized only for the clinical trial. Investigators should strive to conduct all aspects of the trial including administration of the intervention within existing clinical resources and describe evidence of sustainability of successful interventions after the conclusion of the trial.
- Exploration of low-cost methods to collect, store and utilize biological specimens as part of the pragmatic trial structure is encouraged.