Archive for category Medicare Part D

The Evidence Base for Step Therapy for Pharmaceuticals

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.


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Off-Label Use in Medicare Part D Protected Classes

Headlines recently reported on a study finding significant overuse of atypical antipsychotics in the U.S. population.  Using the IMS Health National Disease and Therapeutic Index, the authors reported that 60% of prescriptions written for atypical antipsychotics had no FDA-approved indication, with 90% of off-label uses lacking moderate or good evidence of effectiveness.  Among seniors, the rate of off-label use was even higher at 75%.  Given the adverse event profile for atypical antipsychotics, the lack of an efficacy advantage in recent comparative effectiveness studies, and their considerably greater expense, the high rate of off-label use of atypical antipsychotics is a real concern.

While once an “untouchable” therapy class, this study provides further impetus for commercial plan sponsors to more actively manage their atypical antipsychotic use to improve safety and cost-effectiveness.   These findings also stand in stark contrast to Medicare Part D’s continued inclusion of antipsychotics as a protected class, which requires plans to include “all or substantially all” drugs on the formulary.  Looking more broadly at the CNS-related protected classes, which also includes antidepressants and anticonvulsants, the magnitude of off-label use is equally alarming.  Consider the following data points:


  • 75% of use off-label in Medicaid in 2001 (Chen)
  • 61% of use off-label or unexplained in 2002 in marketscan data (Larson)


  • 84% of second generation use off-label in large MCO in 2005 (Patel)
  • 80% of use off-label in Medicaid in 2001 (Chen)


  • 60% of use off-label in VA in 2007 (Leslie)
  • 64% of use off-label in Medicaid in 2001 (Chen)
  • 86% of use off-label in nursing homes in 2004 (Kamble)

Ironically, these three protected classes under Part D have some of the highest rates of off-label use of all commonly used classes and have specifically been identified as classes of concern with a need for further research on the clinical and economic consequences of off-label use.

Yes, off-label use stimulates innovation when practiced on a smaller scale but it also brings with it safety and cost-effectiveness concerns.  Protected classes under Part D were mandated to ensure that the most vulnerable Medicaid recipients transferring into Medicare would have coverage for the exact same medications when transferred to Medicare, but the time has come to revisit this policy.  Section 176 of the Medicare Improvements for Patients and Providers Act of 2008 appears to have opened the door for this reconsideration, as CMS establishes formal criteria for classes of concern.

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