Comparative effectiveness research in the “real” world: lessons learned in a study of treatment-resistant hypertension

Abstract

Comparative effectiveness research (CER) is vital to translate new efficacious diagnostic and therapeutic approaches into effectiveness in usual clinical practice settings. Studying the practice environment in which effectiveness protocols are implemented is necessary to identify the complex challenges that can limit translation of evidence. These issues were addressed in our National Heart, Lung, and Blood Institute-funded R34, “Controlling Blood Pressure in Treatment-Resistant Hypertension (TRH): A Pilot Study.” Qualitative methods were used in this cluster (clinic)-randomized, four-arm pilot study of TRH in eight diverse, community-based practices including: (i) focus group discussions with practice staff and physicians; (ii) conference calls with physicians; and (iii) discussions with research coordinators. Sources were summarized and analyzed by content analysis. Results include data segregated into categories representing facilitators of and barriers to research. Key facilitators included: (i) early success in controlling challenging TRH patients (ii) improved management of TRH, and (iii) reimbursement for study time and expenses. Barriers included: (i) time-consuming regulatory requirements; (ii) limited training and research experience of some study coordinators; and (iii) reluctance of some physicians to refer to Hypertension Specialists. Qualitative assessment is valuable for identifying facilitators and barriers to CER. This information is important in designing and implementing CER to accelerate translation of clinical efficacy into effectiveness.

Introduction

Comparative effectiveness research (CER) and practical clinical trials (PCT) are vital tools to translate new drugs, devices, and other treatment approaches from proven efficacy to effectiveness.1, 2, 3 Efficacy studies establish that a treatment produces the desired effect in the controlled environment of one or a few experienced, research-intensive sites. Subjects are typically recruited through a variety of approaches, both from within and outside the entity conducting the trial, and subjects tend to be more demographically homogeneous. The research often involves a placebo. Effectiveness studies are conducted in the “real world” of a variety of busy clinical practices, with heterogeneous subjects that are more representative of the general population and may (PCT) or may not (CER) include a placebo.

If we are to translate efficacy into effectiveness and widespread adoption, we must understand the process by which the evidence produced in controlled research sites may vary from the desired effect in clinical practice. We must also appreciate the “adjustments” necessary to tailor efficacious interventions to specific populations or practice types to improve their rate of adoption. Published work in CER and PTC is relatively new, but a general consensus has formed that it must be representative of sites that provide care and the diverse spectrum of patients that can benefit.³ This requires that we view this research as a collaborative effort, which includes input from practices in the design of the research and includes them in the presentation and publication of results. Specifically, it is important to involve staff from representative practice types, locations, and with varying degrees of resource-intensity in the design and implementation of the approach and interpretation of results.4, 5 Studying the practice environment in which effectiveness protocols are conducted adds a layer of insight into the complexity of issues and challenges that can limit translation of evidence. This requires understanding the culture, resources, and capacity of primary care practice in a variety of community settings.⁶ For this reason, we included several qualitative methods to study the process of implementing a recent National Heart, Lung, and Blood Institute-funded CER R34, “Controlling Blood Pressure in Treatment-Resistant Hypertension (TRH): A Pilot Study.”⁷ The lessons we learned are important to consider in designing future research conducted in community-based practices.