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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