While step therapy has been around for a decade or more, it still represents one of the lowest hanging fruit for plan sponsors who want to improve the value received from their pharmacy benefit with minimal member disruption. Today more than 50 therapy classes have the opportunity for step therapy, including several specialty medication classes.
When I recently returned to pharmaceutical policy work, I was surprised to see how few evaluations of step therapy had been conducted in the last decade, particularly considering step therapy’s high degree of popularity among plan sponsors in both private and public settings. I was also surprised to see that most of the evaluations of step therapy that had actually examined clinical and economic outcomes were funded by pharmaceutical manufacturers rather than managed care organizations that can provide thought leadership on this benefit tool.
In a paper published today in the Journal of Managed Care Pharmacy , I review the literature on step therapy and highlight important areas for future research. Clearly, evaluations of step therapy are needed for numerous therapy classes of clinical significance, such as statins and specialty medications. This is important because one would fully expect patient response and the clinical implications of patient choices to vary by therapy class and by the underlying indication. The lack of evaluations of step therapy in the Medicare Part D population is a particularly notable gap.
Most of the research to date has focused on the drug cost savings of step therapy, a necessary condition for step therapy’s uptake but certainly not the only outcome of interest. While savings from step therapy are widely known within healthcare organizations, a better understanding the clinical profile of patients who receive prior authorization for brand medications or receive no medication following a step edit is an important area of inquiry as it has the potential to affect not only economic outcomes, but clinical outcomes and member satisfaction. Perhaps the second most notable gap is the lack of evaluation of alternative forms that are growing in popularity, such as removal of grandfathering and integration of medical claims into the real-time step edits.
In the paper, I also discuss some of the key methodological concerns with the publications to date and highlight examples of potential bias. Based on this review, here are a few methodological tactics you should watch for when considering step therapy evaluations:
- Reporting of non-significant findings as if they were statistically significant
- Evaluation of all-cause medical expenses rather than disease-related expenses (all-cause medical costs are highly variable are have a greater chance of showing random differences across groups for reasons that have nothing to do with the program being evaluated)
- Inclusion of patients who were unaffected by the program to calculate drug savings, which will reduce the magnitude of apparent savings
- Examination of a small subpopulation of patients affected with extrapolations to the entire program
As I point out in the paper, the popularity of step therapy among commercial, Medicaid, and Medicare plans is no doubt due to the wide availability of generic alternatives that offer significant savings, the strong clinical evidence that typically underlies these programs, and their ability to affect only new users, thereby minimizing member disruption. It is important that evaluations of step therapy keep pace with their growing use in order to optimize program design and patient outcomes.