Doctors at the Veterans Affairs Boston Healthcare System are testing a new clinical trials method that allows researchers to compare the effectiveness of two standard diabetes treatments without spending the cost and time needed to conduct a traditional double-blind, placebo-controlled trial.

The new approach, called a point-of-care clinical trial, was developed by Stanford University biostatistician Philip Lavori, Ph.D., and a Boston-based team that includes Louis Fiore, M.D., director of the Department of Veterans Affairs Cooperative Studies Program Coordinating Center in Boston.

The method integrates standard clinical trial procedures, such as recruitment and randomization of subjects, into routine clinical care and allows researchers to switch study participants to the treatment that proves most effective before the trial is completed.

“The goal of point-of-care clinical trials is to deliver the best care to patients while learning from each experience and redefining that care,” said Lavori, a professor of health research and policy at Stanford’s School of Medicine and the senior author of an article on the method published online last month in Clinical Trials. “This learning-and-improving loop will enable healthcare institutions to more rapidly fold improvements into their medical practices.”

Interest has grown in comparative-effectiveness research (CER), which provides evidence of the benefits and harms of different treatment options, to help reduce medical care costs and allow physicians and patients to make informed decisions about health care. In 2009, the economic stimulus package included $1.1 billion for CER.

While clinical trials are widely considered the “gold standard” in comparative-effectiveness research, the high cost and complexity of traditional trials, along with low success rates in physician-adoption of CER results, has prompted researchers to look for alternative methods that could be integrated directly into a clinical care setting.

Point-of-care clinical trials combine the statistical validity of a traditional clinical trial, in which researchers randomize patient selection to minimize bias, and the real-world applicability of an observational study, in which researchers can draw on data from medical records and databases to compare drugs and procedures already in regular use.

The Boston Veterans’ Hospital, which began enrolling patients in October 2010, piloted the point-of-care clinical trial approach by comparing two standard methods of administering insulin to patients hospitalized for diabetes: sliding-scale insulin and the weight-based regimen. First, the hospital’s electronic medical record (EMR) system was programmed to randomize patients and search for patterns within clinical information. When a doctor ordered insulin for a patient, the EMR system offered the two usual treatment options plus a third choice labeled “no preference.” When the “no preference” option was chosen, the patient was asked to participate in the clinical trial; if the patient agreed, the EMR software randomly assigned one of the two treatment options and care was continued as usual.

Meanwhile, the EMR software tracked which of the two approaches resulted in the patients being released from the hospital more quickly. As the study advanced and new patients were enrolled, the software directed more patients to the treatment proving most successful at the time. The researchers will continue this process until the estimated probability that one treatment is better than the other is more than 99%.

“The idea of embedding research into clinical care has been around for quite a while, but to my knowledge this is the first time that a randomized trial has been fully integrated into a hospital’s informatics system,” said Fiore, the lead author of the Clinical Trials article and an associate professor of medicine at Boston University’s Schools of Medicine and Public Health. “It demonstrates an effective way to use electronic medical records to improve health care at a local level.”

Once integrated into a hospital’s EMR system, researchers said point-of-care trials can be conducted quickly for very little incremental cost. The method can’t be used for studies that need a control group or involve an unapproved drug, since neither is part of regular care. Yet the researchers say point-of-care trials can be useful for comparing efficacy of commonly used drugs, devices, treatments and interventions in which peer-reviewed evidence is lacking or inconclusive. For example, a point-of-care trial could be used to determine which FDA-approved stent produced fewer adverse side effects. In addition, a point-of-care trial could be used to look for correlations between genetic biomarkers and efficacy of drugs in the development of personalized medicine.

Fiore said so far the pilot study has been successful, and researchers plan to extend it to other Veterans Affairs hospitals nationwide during the coming months. “We have tested all the links in the clinical informatics chain and know that we are not interfering with clinical practice,” he said. “We also have demonstrated good acceptance from patients, learned how to inform doctors about the study and dealt with all the human subjects issues that arise from trying to do something new.”

--Karyn Korieth