OBJECTIVES : Health plans employ utilization management tools, such as step therapy (ST), to encourage the cost-effective use of pharmaceuticals. Further, recent policy changes expand ST to Medicare Advantage plans, emphasizing the need for comprehensive best practices. We conducted a review of best practices and state legislation for ST development and implementation processes to inform efforts to evaluate health care policies.

METHODS : We reviewed the gray literature to identify principles and legislative tracking tools (AMCP, National Psoriasis Foundation) to identify state legislation discussing ST practices. We identified nine themes associated with ST protocol development, implementation and evaluation. We then assessed agreement, (all principles include) silence (some principles, but others are silent), or disagreement (principles include but disagree on specifics) across the sets of principles and legislation.

RESULTS : Six sets of principles from diverse stakeholders shared several themes: 1) robust clinical evidence to support ST, 2) flexibility allowing for physician input, 3) opportunities to appeal health plan restrictions, 4) preventing interruptions in patient care and 5) ensuring formulary and appeals processes are transparent. There was silence regarding financial considerations, evaluation of policy impact on outcomes or protocol updates. In the evaluation of state legislation, we identified 24 enacted pieces of legislation across 28 states, discussing ST. These policies reflected all five shared themes from the principles. Differences across policies were based on patient populations included (i.e. oncologic or pediatric conditions) and time limits on appeals (i.e. 48 vs. 72 hours). State legislation was silent on administrative efficiency, financial implications of risk-sharing agreements, standards for e-prior authorization, timing of updates of evidence and protocols and the maximum number of steps allowed.

CONCLUSIONS : This review highlights critical components to ensuring ST protocols. Agreement on best practices can facilitate the evaluation of the development, implementation, and outcomes associated with efficiency of care delivery, costs and patient outcomes.