title: 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.
abstract: BACKGROUND
  Multiple myeloma is an incurable B-cell malignancy with a natural history that involves alternating periods of remission and subsequent relapse. For relapsed and/or refractory multiple myeloma (RRMM), the typical patient currently receives more lines of therapy than has been feasible in the past, translating into longer progression-free survival (PFS). Consequently, cost issues have become more prominent because patients may be offered newer and more expensive therapies during a more prolonged overall treatment course.
  
  
  OBJECTIVE
  To estimate the economic impact of adding panobinostat to a U.S. health plan formulary as a treatment option with bortezomib and dexamethasone for patients with RRMM previously treated with a proteasome inhibitor (PI) and immunomodulatory drug (IMiD), using a budget impact and cost-benefit model.
  
  
  METHODS
  Total costs of commonly used salvage therapy regimens were combined with market share data and population prevalence estimates of RRMM to yield the total cost of treatment, from the perspective of a U.S. third-party payer (commercial or Medicare) with a time horizon of 1 year. Comparator treatment regimens included bortezomib-dexamethasone, lenalidomide-dexamethasone, lenalidomide-bortezomib-dexamethasone, carfilzomib monotherapy, carfilzomib-lenalidomide-dexamethasone, and pomalidomide-dexamethasone. Costs (2015 U.S. dollars) included drug costs for oral oncology agents, medical and administration costs for injectable oncology agents, costs of adverse event (AE) prophylaxis and monitoring, and costs of grade 3/4 AEs.
  
  
  RESULTS
  In a hypothetical health plan with 1 million members, the annual number of RRMM patients with previous PI and IMiD treatments was estimated at 16 and 118 for a commercial and Medicare plan, respectively. Introduction of panobinostat as part of the panobinostat-bortezomib-dexamethasone regimen was not expected to result in a substantial budget impact to either commercial or Medicare plans, with an incremental cost < $0.01 per member per month. Panobinostat-bortezomib-dexamethasone had a low cost per treated patient per month without progression, owing to the minimal increase in expenditure over existing bortezomib-based regimens and long median PFS, compared with median duration of treatment.
  
  
  CONCLUSIONS
  Adding panobinostat to a plan formulary as a treatment option is expected to be cost neutral (and potentially cost saving in the context of new and more expensive treatment regimens). With a low cost per month without progression, panobinostat-bortezomib-dexamethasone represents good value for the money.
  
  
  BACKGROUND
  Funding for this study was sponsored by Novartis, East Hanover, New Jersey. Bloudek and Kish are employees of Xcenda, a consulting company contracted by Novartis to conduct this analysis. Roy, Globe, and Kuriakose are employees of Novartis. Siegel is on the advisory boards and speaker's bureau of Celgene, Onyx/Amgen, Millennium/Takeda, and Novartis and is on the advisory boards of Merck. Jagannath is a consultant to Sanofi, Bristol-Meyers Squibb, and Celgene. Orloski is a contractor to Xcenda and provided medical writing support, which was funded by Novartis. Study design and concept were contributed by Bloudek, Roy, and Kish, assisted by Globe. Bloukek took the lead in data collection, along with Kish, and data interpretation was performed by Siegal, Jagannath, Globe, and Kuriakose. The manuscript was written primarily by Orloski, along with Roy and Kish, and revised by Roy, along with Siegal, Jagannath, Globe, Orloski, and Kuriakose.
authors.affiliation: 1 Xcenda, Palm Harbor, Florida.
authors.forename: Lisa
authors.initials: L
authors.lastname: Bloudek
authors.affiliation: 2 Novartis, East Hanover, New Jersey.
authors.forename: Anuja
authors.initials: A
authors.lastname: Roy
authors.affiliation: 1 Xcenda, Palm Harbor, Florida.
authors.forename: Jonathan K
authors.initials: JK
authors.lastname: Kish
authors.affiliation: 3 Myeloma Division, Hackensack University Medical Center, Hackensack, New Jersey.
authors.forename: David S
authors.initials: DS
authors.lastname: Siegel
authors.affiliation: 4 Multiple Myeloma Program and Hematology and Medical Oncology, The Tisch Cancer Institute, Mount Sinai Hospital, New York, New York.
authors.forename: Sundar
authors.initials: S
authors.lastname: Jagannath
authors.affiliation: 2 Novartis, East Hanover, New Jersey.
authors.forename: Denise
authors.initials: D
authors.lastname: Globe
authors.affiliation: 1 Xcenda, Palm Harbor, Florida.
authors.forename: Laurie
authors.initials: L
authors.lastname: Orloski
authors.affiliation: 2 Novartis, East Hanover, New Jersey.
authors.forename: Emil T
authors.initials: ET
authors.lastname: Kuriakose
mesh_terms: D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000191:economics; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000191:economics*; D003362:Cost-Benefit Analysis / Q000379:methods*; D006801:Humans; D006877:Hydroxamic Acids / Q000008:administration & dosage / Q000191:economics*; D007211:Indoles / Q000008:administration & dosage / Q000191:economics*; D007348:Insurance, Health / Q000191:economics; D006278:Medicare / Q000191:economics; D018803:Models, Economic; D009101:Multiple Myeloma / Q000188:drug therapy* / Q000191:economics* / Q000453:epidemiology; D000077767:Panobinostat; D010602:Pharmacopoeias as Topic; D016879:Salvage Therapy / Q000191:economics / Q000379:methods; D014481:United States / Q000453:epidemiology
chemical_list: D000970:Antineoplastic Agents; D006877:Hydroxamic Acids; D007211:Indoles; D000077767:Panobinostat