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T-11: Pragmatic Randomized Clinical Trials: Key Insights from Investigators in COPD and Diabetes

Poster Presenter

Priscilla Velentgas

Senior Director, Science at Aetion
Aetion
United States

Objectives

Feasibility surveys prior to new to market treatment compared with standard of care (SOC) pragmatic randomized clinical trials (pRCTs) provide valuable considerations for planning. Key insights from respondents to surveys conducted in Europe and the US during 2016 are presented.

Method

E-mail and telephone survey of GP and specialist physicians. Questions included willingness to randomize patients to SOC, routine use of spirometry and PRO instruments in COPD, and impact of providing study treatment at no cost for diabetes, as well as perceived challenges for implementing a pRCT.

Results

Respondents included 82 physicians treating COPD patients representing 15 European countries, Canada, and the US, and 10 physicians treating diabetes in the US. Eighty-nine percent of physicians treating COPD patients, and 80% of physicians treating diabetes expressed interest in participation in a pRCT. Most physicians treating COPD patients (86%) were comfortable with randomizing patients to a SOC treatment consistent with treatment guidelines in COPD (not asked on diabetes survey). Physicians indicated that spirometry, while routinely performed on their COPD patients, is conducted with variable frequency at their sites (range 40-100% in previous 12 months, mean 84%), and commented that requirement for spirometry as inclusion criteria for the study was a departure from the “real world” aspect of a pRCT trial. Eighty-five percent of COPD physicians reported use of the COPD Assessment Test (CAT) and/or Modified Medical Research Council (MMRC) dyspnea scale in routine care of their patients. In response to a question asked of diabetes physicians in the US about utilizing patient insurance for coverage of SOC treatments in a pRCT, several commented that their institutions or local IRB required that the cost of medications studied in a pRCT be covered through the study rather than through insurance or health plan reimbursement.

Conclusion

Among 92 responding sites with some prior experience in conducting observational studies, clinical trials, or both, there was a high degree of interest in participation in a pRCT, and overall high comfort level with the underlying premise of randomizing patients to a marketed SOC treatment consistent with existing treatment guidelines. The selected sample of sites and their response may not be representative of sites with no research experience or in different therapeutic areas. Tradeoffs between the value of additional clinical assessments such as spirometry or HbA1c beyond what is routinely performed to achieving study aims versus increased site and patient burden must be considered by researchers and sponsors. A cautionary note that a pRCT must provide the treatments of interest free of charge was raised by several respondents as policy by their institution and/or local EC/IRB. Eligible lower income patients without medical insurance in the US may not be able to afford the cost of treatments if not provided through the study.