Defense Date


Document Type


Degree Name

Doctor of Philosophy


Pharmaceutical Sciences

First Advisor

Norman V. Carroll


Introduction: Atypical Antipsychotics (AAPs) are approved by the Food and Drug Administration (FDA) for the treatment of schizophrenia and bipolar disorder. AAPs are commonly used off-label to treat depression, post-traumatic stress disorder and neuropsychiatric symptoms in dementia due to lack of alternative treatment options and treatment resistance. Concerns for off-label use arise since AAPs increase the risk of cardiovascular events and death. The objectives were 1) describe patterns of RU and costs among off-label AAPs users in a nationally representative population 2) identify prevalence of off-label use in the Medicare population 3) compare RU and costs between off-label AAPs users and non-users with mental health conditions in Medicare.

Methods: For the first objective, the Medical Expenditure Panel Survey (MEPS) datasets were used. AAPs users greater than 18 years were identified in this cross-sectional study. Generalized Linear Models (GLM) were used to estimate costs among users and non-users after controlling for age sex, gender, insurance type, marriage status, income and comorbidity index. For the second and third objective, Medicare datasets were used to identify prevalence, RU, and costs of off-label use in Medicare beneficiaries 18 years and older. RU and costs between propensity score matched AAPs user and non-user cohorts were compared in a retrospective cohort study.

Results: The adjusted odds of having an office-based outpatient (OR=2.47, 95%CI: 1.55-3.92) or inpatient (OR=1.63, 95%CI: 1.26-2.10) visit were significantly higher among off-label AAPs users. Adjusted office-based visit ($1,943 vs. $1,346), prescription ($4,153 vs. $1,252) and total ($10,694 vs. $4,823) costs were significantly higher among users (p<0.0001).

Among Medicare beneficiaries, approximately 37% of AAPs users had no FDA approved diagnosis. The typical off-label user was a white 70-year-old male. Common off-label uses were depression, anxiety and neurotic disorders and dementia. Off-label AAPs users had significantly higher mental health outpatient ($461 vs $297), prescription ($2,349 vs $282) and total ($3,665 vs $1,297) costs per beneficiary than non-users. About 30% of AAPs users had at least one mental health outpatient visit during the year versus 23% of non-users; no significant differences were found in inpatient visits. AAPs non-users had significantly higher all-cause inpatient costs ($6,945 vs. $4,841) per beneficiary (p

Conclusion: In a nationally representative population comprising a younger age group AAPs users had higher all-cause RU and total costs than non-users. Off-label prescribing of AAPs continued to be a prevalent practice affecting 37% of Medicare AAPs users. Off-label AAPs users had higher mental health costs but no significant differences in all-cause total health care costs in a Medicare population. Off-label use of AAPs can be a cost-effective option if future research shows off-label use is associated with increased effectiveness, which offsets any additional costs.


© Della Varghese

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