Stakeholders find that step therapy should be evidence-based, flexible, and transparent: assessing appropriateness using a consensus approach

BACKGROUND: Step therapy, one approach to utilization management, is used by health plans to ensure safe and clinically appropriate care while managing cost. Several patient and provider groups have each developed principles to guide the appropriate use of step therapy; however, no comprehensive multistakeholder informed set of criteria exist. OBJECTIVE: To assess multistakeholder consensus on criteria for the development and implementation of step therapy for pharmaceutical therapies. Stakeholders were asked to (a) assess the appropriateness of step therapy as a utilization management tool; (b) rate specific criteria across 5 domains (development, implementation, communication, appeals, and evaluation) of step therapy; and (c) categorize these criteria as standards or best practices. METHODS: We conducted a multiphase project culminating in a roundtable of experts representing patient, provider, plan, pharmacy, policy, and ethical perspectives. We first reviewed guiding principles, position statements, and legislative activity to draft criteria regarding step therapy protocol development, implementation, communication, and evaluation. To assess consensus across a convenience sample of experts, we employed an iterative 4-step modified Delphi method. Panelists were asked to (a) rate the overall appropriateness of step therapy, (b) rate the appropriateness of specific criteria, and (c) identify each as a standard or best practice. Appropriateness was rated from 1-9 and categorized in terciles (1-3: not appropriate, 4-6: neither, 7-9: appropriate) to assess quantitative agreement, disagreement, and indeterminate agreement. RESULTS: After the second round of voting, roundtable panelists (n = 16) disagreed on the appropriateness of step therapy for utilization management (50% appropriate, 31.25% neither, and 18.75% inappropriate). Agreement was achieved on 21 criteria across 5 themes (clinical criteria as the foundation for protocol development, implementation of protocols, transparency and communication of processes, navigation of the appeals process, and evaluation of health and administrative impact). Fourteen and seven criteria were categorized as standards and best practices, respectively. CONCLUSIONS: The stakeholders in this panel differed in their assessments of the appropriateness of step therapy but agreed regarding how these protocols should be developed, implemented, communicated, and evaluated. Most criteria were rated as standards that can be used by stakeholders when developing, implementing, and assessing step therapy processes today.