An alternative method for determining the best drug therapies for managing chronic disease could reduce treatment costs or give patients more immediate relief than other common approaches typically approved for use by managed care and health insurance companies, a study by researchers at UC Davis School of Medicine has concluded.

The May 26 online edition of the Archives of Internal Medicine includes the research study, titled "N-of-1 Trials of Expensive Biological Therapies -- A Third Way?

For their study, investigators developed an economic model to evaluate the costs of three clinical strategies that doctors use to treat rheumatoid arthritis: stepped care, open access and an "n-of-1 trial." They wanted to explore whether n-of-1 trials represented a way to balance the interests of patients with those of payers when establishing policies regarding the use of expensive drugs.

An n-of-1 trial is a single-patient, randomized, double-blind, multiple crossover comparison of an active drug against an alternative drug or placebo. Such trials are feasible when a condition being studied is chronic or recurrent, and the costs of treatment very high.

During an n-of-1 experiment, a patient is given one treatment and then another, in random sequence, with all symptoms and side effects carefully noted. At the end of the trial period, both patient and doctor are able to determine, with a high degree of confidence, which medicine works better for that particular individual.

For both clinical and economic reasons, managed care organizations typically require stepped-care experiments for expensive drug therapies used in chronic illnesses such as rheumatoid arthritis. Physicians and patients must first try more established and less expensive drugs before stepping up to more costly biopharmaceuticals.

Immediate, or open, access is another option, whereby patients have the ability to choose any appropriate therapies regardless of cost considerations.

"A stepped-care policy can be appropriate in many circumstances," said Richard Kravitz, professor of internal medicine and lead author of the study. "However, it also prompts questions from patients about why they can't have open access to a particular medicine right away. Our model suggests an alternative approach that can help patients and their physicians identify a beneficial therapy in a timely and cost-effective manner."

Charles Casey is a senior public information officer for the UC Davis Health System.