Inconsistent application of protocols. Health plans' inconsistent application of step therapy protocols has an important implication for current and potential enrollees. Patients enrolled in different health plans have different access to the same drugs. Some plans require patients to first try and experience treatment failure with a larger number of prior therapies than other plans. Inconsistency across plans also may negatively affect a patient who moves from one health plan to another if that person is taking a drug for which the two plans employ different protocols.

Consistency with labeling. It is unsurprising that health plans' step therapy protocols are more stringent than FDA label indications. FDA-approved drugs are deemed safe and efficacious. By contrast, health plans cover drugs that are deemed medically necessary, which accounts for various additional factors, such as how a drug performs in real-world settings, comparative effectiveness and budget impact.

Consistency with clinical guidelines. Many step therapy protocols are inconsistent with leading clinical organizations' recommended treatment pathways. Although a step therapy protocol that is inconsistent with a treatment guideline is not necessarily clinically inappropriate, the difference does raise questions about its appropriateness.

Future research. Researchers found a need to examine the possible association between greater stringency and excessive requirements to obtain specialty drugs.

Therapy legislation. Considerable variation in health plans' handling of step therapy supports the need for legislated consumer protections and provides important context for the current policy conversation.

"We found the use and content of step therapy protocols employed by 17 major U.S. health plans to determine enrollees' access to specialty drugs for 10 major clinical conditions to be highly variable, both within and among plans," researchers concluded. "Moreover, roughly one-half of plans' step therapy protocols were more stringent than the treatment guidelines recommended by national clinical organizations.

"These differences raise questions and concerns about potentially overly restrictive step therapy protocols; the burden protocols place on patients; potential delays in provision of and discontinuities in effective drug therapy; and potential differences in patients' access to specialty drugs, across plans, for the treatment of the same clinical conditions. Our findings support the development of legislative initiatives to help ensure appropriate use."