Practice-based research networks: will their promise become reality?

Abstract

Patients often ask their provider “given my personal characteristics and health history, what are my treatment options and what are the potential benefits and risks of those options? And, if I don’t respond to the first therapy what are the next steps in treating my condition?” Unfortunately, the evidence to answer these questions is often unavailable, in part because we look to randomized controlled trials (RCTs) as the ‘gold standard’ for evidence. RCTs are conducted in ‘research grade’ environments with carefully selected patients and carefully selected treatment settings. These characteristics enhance their ability to determine whether a therapy can work (efficacy), but not whether the results can be generalized to routine care or routine patients (effectiveness). An alternative, practice-based research networks (PBRNs) can generate evidence with a more real-world view perspective. PBRNs typically consist of primary care clinicians in routine practice who also conduct studies on the patient populations that they serve. These networks have the patients and infrastructure to conduct pragmatic clinical trials, collect data for prospective registries or house information for retrospective analyses. In contrast to RCTs, pragmatic trials may enroll a broader more typical patient population, have active therapy comparisons (vs placebo in many RCTs), require patients to purchase and obtain their medications as is typical of routine care, may not have predefined follow-up testing or office visits (which resembles more usual patient experience), and may allow the clinician to modify the regimen if the patient’s condition does not improve or side effects occur. In addition to answering questions similar to those posed by the patient above, these pragmatic trial characteristics were viewed by payers in a recent survey as valuable [1]. The importance of PBRNs and pragmatic studies was also recognized by the Agency for Healthcare Research and Quality [2], the NIH [3] and the Patient Centered Outcomes Research Institute. The Patient Centered Outcomes Research has announced US$100 million in funding for the National Patient-Centered Clinical Research Network [4]. As we consider the development and use of PBRNs, it will be critical to carefully examine and shape the research questions to be addressed. It is not only the ability of these PBRNs to raise practical and important research questions, but also their ability to more rapidly and efficiently answer them that holds great promise. The PICOTS framework can help to identify the most pressing, practical and real-world questions that traditional RCTs may not address: ■ Patients: what types of patients have been studied in RCTs and which ones remain unstudied? Older ones? Those with comorbidities? Those with lesser disease severity?