Novel Methodology for Comparing Standard-of-Care Interventions in Patients With Cancer.

PURPOSE: The current clinical trials development and conduct process is cumbersome and expensive, with the majority of studies focusing on either the development of new agents or new indications for established agents. Unfortunately, research comparing standard-of-care interventions is rarely performed, leaving many important and practical patient-centered questions unanswered. Novel clinical trial methodologies and approaches are needed.

METHODS: We have identified simple key components that, when combined, enhance the ability to both perform and increase accrual for studies that compare standard-of-care interventions. These include selection of clinically relevant and practical questions, demonstration of clinical equipoise through surveys of knowledge users and completion of systematic reviews, appropriate study design and simply defined study end points, use of an integrated consent model incorporating oral consent, efficient research ethics board approval, Web-based randomization in the clinic, real-time electronic data capture and management, and regular formal team feedback.

RESULTS: We have demonstrated the feasibility of this model in a pragmatic trial comparing two standard-of-care interventions (growth factor support or ciprofloxacin) for the primary prophylaxis of febrile neutropenia in patients with breast cancer receiving adjuvant docetaxel with cyclophosphamide chemotherapy. Research ethics board approval took 3 months, and 110 (72%) of 153 potentially eligible patients have agreed to participate in the study. When surveyed, 81 (85%) of 95 patients were completely satisfied with the integrated consent model process.

CONCLUSION: Our proposed model contains elements that, when used alone or in combination, may allow efficient and cost-effective comparison of standard-of-care interventions.