Field Test of New Clinical Trial Method Underway

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A field test has begun of a new type of clinical trial at the Veterans Affairs Boston (Mass.) Healthcare System, which proponents say is less costly and can also guide doctors to switch to the best treatment before the trial is completed. The new approach — called a point-of-care clinical trial — is described in the online issue of the journal Clinical Trials (paid subscription required).

Stanford University biostatistician Philip Lavori, along with the Boston clinicians and researchers, developed the point-of-care clinical trial as an alternative to expensive, lengthy, double-blind, placebo-controlled clinical trials to compare drugs and procedures that are already in regular use. “The goal of point-of-care clinical trials,” says Lavori, “is to deliver the best care to patients while learning from each experience and redefining that care.”

This new kind of trial can provide a useful tool for comparative effectiveness research, where systematic inquiries can identify more costly and less effective medical care, by pitting one treatment against another. Point of care trials can combine the statistical validity of the traditional clinical trial, with the real-world applicability of an observational study, where researchers can draw on data from medical records and databases.  The 2009 Recovery Act (stimulus bill) contains $1.1 billion for comparative effectiveness research, while the Affordable Care Act of 2010 creates a a non-profit Patient-Centered Outcomes Research Institute to identify research priorities and conduct research that compares the clinical effectiveness of medical treatments.

The journal article describes the test in Boston that compares two standard methods of treating hospitalized — but non-critical care — patients for diabetes: sliding-scale insulin and the weight-based regimen. The sliding-scale regimen gives the patient short-acting insulin three to four times using an algorithm based on blood sugar levels, planned activities, and sugar consumption. In the weight-based protocol, patients receive longer-acting insulin throughout the day in doses based on their weight. Patients began enrolling in the study in October 2010.

A key requirement of the point-of-care trial is the availability of electronic patient records in a database that allows for the trial managers to randomize patients to the different test groups and search for patterns in their clinical data. IT specialists at the Boston Veterans Affairs facility programmed its electronic records to incorporate recruitment and data collection as part of everyday care giving.

Now, when a doctor orders insulin, the system offers not only the sliding-scale and weight-based protocols, but also a third choice: no preference. If no preference is the choice, a nurse explains the trial to the patient. If the patient agrees to participate, the 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.

The software at the same time tracks which of the two approaches is associated with the best outcome, defined in this case as getting out of the hospital quicker. As the study progresses and new batches of patients enter the trial, the software directs more patients to the most successful treatment at that time. The process continues with more enrolled patients until the estimated probability that one treatment works better than the other exceeds 99 percent.

One advantage of the point-of-care clinical trial is that it allows researchers to compare treatments on a local patient population, then allows facilities to implement the best alternative into their physician ordering systems. Doctors’ decisions can also be tailored to specific populations, which can vary in genetic, geographical, and socioeconomic factors.

The authors caution that point-of-care clinical trials cannot replace studies requiring control groups. Nor can they be used in trials of new drugs that have not yet been approved by the FDA, since the point-of-care trial involves day-to-day patient care. Plus, the requirement for sophisticated, flexible electronic records databases and systems will limit the ability of some hospitals and clinics to adopt the new methods.

Read more: FDA, NIH Award Grant to Study Adaptive Clinical Trial Design

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