A clinical trial methodology combining elements of randomised studies and observational trials can reduce the time and costs involved in comparative effective research (CER) while allowing optimal treatment to be incorporated quickly into medical practice, US researchers say.

The point-of-care approach was developed by a team working under Philip Lavori, a professor of health research and policy at Stanford University’s School of Medicine in California. A pilot study of the methodology, described in the journal Clinical Trials, is ongoing at the Veterans Affairs Boston Healthcare System, which started enrolling patients in October 2010.

According to Lavori, the aim of a point-of-care trial is to “deliver the best care to patients while learning from each experience and redefining that care”. Funding for the Boston study came from the Department of Veterans Affairs Cooperative Studies Program and the US National Institutes of Health.

Blended approach

The approach, the researchers explain, blends the statistical validity of a traditional clinical trial, which requires “time-consuming and costly steps” to randomise patient selection and minimise bias; and the ‘real-world’ applicability of an observational study, where researchers can draw on data from medical records but without eliminating the potential bias associated with certain patients being allocated particular treatments.

Point-of-care trials will not work for trials needing a control group or involving an unapproved drug, as neither is part of regular care. But they are useful – and cost-effective – for comparing the efficacy of commonly used medicines, devices, treatments and interventions where peer-reviewed evidence is lacking or inconclusive, the researchers say.

“Using evidence to decide what treatments to use seems like a good idea — but as soon as it involves questions of coverage it becomes highly political,” Lavori comments.

“Our idea is that if systems of care like the VA can integrate implementation of research results directly into care, we will keep the decision-making where it belongs. It brings medical decision-making back down to expert physicians and their patients, and out of the political realm.”

The pilot study is comparing two standard methods for treating hospitalised patients with diabetes: a sliding-scale regimen of short-acting insulin, guided by an algorithm that factors in blood sugar levels, sugar consumption, etc; and a weight-based protocol whereby patients are given longer-acting insulin at doses linked to their weight.

According to Dr Louis Fiore, lead author of the Clinical Trials article and director of the Department of Veterans Affairs Cooperative Studies Program Coordinating Center in Boston, the pilot “has been successful so far, and we plan on rolling it out to other VA hospitals nationwide over the coming months”.

EMR role

A key component of the point-of-care approach is a flexible electronic medical record (EMR) system that can be programmed to randomise patients and search for patterns within clinical information, the researchers note.

For the Boston VA study, informatics specialists programmed the EMR system to incorporate recruitment and data collection as part of everyday care. When a doctor inputs an order for insulin, the system offers the two standard protocols plus a third, labelled “no preference”.

If the choice is “no preference”, a nurse explains the trial to the patient. If the patient then agrees to participate, the EMR software randomly assigns one of the two protocols and care continues as usual, with the doctors entering the patient’s clinical details into the system.

At the same time, the EMR software is tracking which of the two approaches is associated with the best outcome (i.e., in this case, getting out of hospital quicker).

As the study advances and new patients are enrolled, the software will preferentially direct more patients to the treatment proving most successful at the time. This process will continue until the estimated probability that one treatment is better than the other is more than 99%.

Quicker, more tailored

An important advantage of point-of-care trials is that they allow researchers quickly to compare treatments on a local patient population, then immediately to implement the best alternative into the clinic’s physician ordering system, Lavori et al say.

That eliminates delays for peer-review or physician adoption. Moreover, resulting decisions are tailored to specific populations, which may vary widely in genetic, geographical or socio-economic composition, they add.