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Research in Review

Addition of Panobinostat to Multiple Myeloma Care Has Little Economic Impact

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JCP Editors


J Clin Pathways. 2016;2(7):16-19.

Adding panobinostat to treatment regimens for multiple myeloma would have a minimal economic impact and could even save the health care system money, claims a study published in the Journal of Managed Care & Specialty Pharmacy

Thanks to the addition of new therapies and multiple treatment lines, patients with multiple myeloma are living longer than ever. However, as these patients have continued to be offered newer and more expensive therapies during prolonged treatment courses, costs to both them and the system have also increased significantly. 
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In February 2015, panobinostat was granted accelerated approval by the US Food and Drug Administration for the treatment of multiple myeloma after it demonstrated improved progression-free survival when used in combination with bortezomib and dexamethasone in patients who received up to 2 lines of previous treatment. Since then, guidelines from the National Comprehensive Cancer Network recommend panobinostat as a category 1 option for this same patient population, but, as with most new drugs under consideration for formulary placement, the addition of panobinostat is expected to also increase costs.

Therefore, researchers led by Lisa Bloudek, PharmD, MS, Xcenda (Palm Harbor, FL), used a Microsoft Excel-based budget-impact and cost-effectiveness model to estimate the economic impact of adding panobinostat to the formulary as a treatment option for these patients. 

The target population for the model used in the study included adults aged 18 years and older who initiated standard therapy for relapsed or refractory multiple myeloma and had received treatment with at least 2 other regimens. A hypothetical health plan of 1,000,000 members was created using default values derived from 2012 US Census data, Medicare demographic data, information from the Surveillance, Epidemiology, and End Results (SEER) database, and published literature.

In a hypothetical commercial plan, the introduction of panobinostat to treatment plans of bortezomib plus dexamethasone did not have a significant budgetary impact and was actually associated with net savings of $46,450. Additionally, panobinostat plus bortezomib and dexamethasone was found to be highly cost effective, with a cost per month without progression under $10,000. 

Similar findings were reported in a hypothetical Medicare plan, which had cost savings of $342,169 after the introduction of panobinostat. Cost per month without progression for panobinostat plus bortezomib and dexamethasone was $8993, which was lower than lenalidomide and dexamethasone—another common regimen for multiple myeloma—but higher than bortezomib and dexamethasone ($6106). 

Researchers concluded that adding panobinostat to bortezomib and dexamethasone treatment for multiple myeloma would not be associated with a significant budgetary impact for a health care plan, due in large part to its progression-free survival benefit, comparatively low incidence of costly adverse events, and proportionate reduction in market share for costly alternative regimens. 

However, along with limitations inherent to modeling studies, the researchers did acknowledge that their focus on only third-party payers may have influenced their findings. Additionally, they also did not consider out-of-pocket expenses or indirect costs, such as lost productivity, in their analysis. —Sean McGuire


Bloudek L, Roy A, Kish JK. Estimating the economic impact of adding panobinostat to a U.S. formulary for relapsed and/or refractory multiple myeloma: A budget impact and cost-benefit model. J Manag Care Specialty Pharm. 2016;22(8):991-1002. doi: 10.18553/jmcp.2016.22.8.991.

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