Stakeholders Find Step Therapy Should Be Evidence-Based, Flexible and Transparent: Assessing Appropriateness Using a Consensus Approach

Stakeholders disagree on when step therapy is appropriate, but agree on a set of criteria about how to develop, implement, communicate, safeguard and evaluate step therapy protocols.

Authors: Karmarkar T, Dubois RW, Graff JS.
Publication: Journal of Managed Care & Specialty Pharmacy. 2021; 27(2):268-275.
January 28, 2021

Step therapy is a common utilization management tool used by health plans to encourage the appropriate use of first-line drugs in order to ensure safe and cost-effective patient care. When multiple treatments exist, patients are required to try a clinically recognized first-line therapy before payment approval of more complex or expensive treatment options.

Research published in the Journal of Managed Care & Specialty Pharmacy shows that multiple stakeholders, including payer, provider and patient groups, disagree on when step therapy is appropriate but agree on a set of criteria about how to develop, implement, communicate, safeguard and evaluate step therapy protocols. The majority of these criteria can be achieved today.

Why it matters:

Step therapy varies in the frequency of use and the number of steps required in the protocols across health plans. Several patient and provider groups promote guiding principles on how to appropriately use step therapy to manage patient care. Over 30 states have enacted legislation to ensure patient protections when step therapy protocols are used. However, no comprehensive multi-stakeholder informed set of criteria for step therapy protocols exist.

The results of this work can serve as a foundation for step therapy protocol development and implementation. The study also can inform voluntary approaches of oversight such as plan or benefit manager accreditation or monitoring of these practices and policies.


The researchers aimed to 1) assess the appropriateness of step therapy, 2) assess consensus regarding the development, implementation, communication, safeguards and evaluation criteria for step therapy and 3) categorize these criteria as standards or best practices.

To achieve these three goals, the researchers used a multi-phased approach, culminating in an expert roundtable, to assess consensus on a checklist of criteria about the use of step therapy.


While stakeholders disagree on when step therapy is appropriate, researchers found they agree on 21 criteria that all stakeholders can use to guide the implementation of step therapy protocols, outlined below:

  • Clinical evidence before costs: Step therapy protocols should be based on up-to-date and comprehensive clinical evidence, patient perspectives, clinical practice guidelines and be reviewed by an objective, unbiased, evidence-based group of experts.
  • Patients should face no more steps than are necessary: Implementation practices, such as lengthy trials of treatments or interruptions in patient care, are not appropriate.
  • Transparency and communication are critical: Communicating information on coverage decisions, the evidence that led to these coverage decisions and how to navigate the processes can empower physicians, pharmacists and patients.
  • Patient safeguards are needed: The development of both exceptions criteria and an appeals process is critical to avoiding a “one-size-fits-all” approach to care that may leave patients with unique circumstances behind. Plans should respond in a timely manner to exception requests and appeals.
  • Evaluate the results: Any policy or protocol implemented that can impact patient care should be evaluated to ensure it is achieving the intended goals.

The majority of these agreed-upon criteria (14) were considered standards or goals that could be achieved today. The remaining seven criteria were considered best practices or aspirational goals and require further policy or infrastructure changes.