title: The value of specialty pharmaceuticals - a systematic review.
abstract: OBJECTIVE
  Novel specialty biopharmaceuticals hold promise for patients living with complex and chronic conditions. However, high research and development costs, special handling, and other necessary enhancements to patient support programs all contribute to frequently higher prices for these products. This study sought to assess the value of specialty pharmaceuticals through an examination of the clinical, functional, and economic benefits of these treatments for the top 3 disease areas by pharmaceutical spend: rheumatoid arthritis (RA), multiple sclerosis (MS), and breast cancer (BC).
  
  
  METHODS
  Systematic literature review.
  
  
  METHODS
  A systematic review of market research and cost-effectiveness articles was conducted for each disease area to assess clinical, functional, and economic outcomes associated with specialty medicine treatments versus the previous standard of care.
  
  
  RESULTS
  All RA clinical (American College of Rheumatology) and functional (Health Assessment Questionnaire) outcome articles were classified as positive. The median cost-effectiveness ratio was $38,900 per quality-adjusted life year (QALY). All MS clinical outcome (relapse rate) articles were positive. The MS functional outcome (Expanded Disability Status Scale) findings were less conclusive. The median cost-effectiveness ratio was $248,000 per QALY. The majority of BC articles yielded statistically inconclusive results for survival. All functional outcome (Quality of Life Questionnaire- Core 30) articles were positive. The median cost-effectiveness ratio was $51,900 per QALY.
  
  
  CONCLUSIONS
  Novel specialty therapies hold promise for arresting disease progression and improving quality of life for the 3 conditions associated with the highest specialty pharmaceutical spend. These findings demonstrate a strong value proposition for specialty pharmaceuticals, and suggest even greater potential individual patient benefit with consideration of patient heterogeneity.
authors.forename: Martin
authors.initials: M
authors.lastname: Zalesak
authors.forename: Joyce S
authors.initials: JS
authors.lastname: Greenbaum
authors.forename: Joshua T
authors.initials: JT
authors.lastname: Cohen
authors.affiliation: 230 Third Ave, Waltham, MA 02451. E-mail: fkokkotos@trinitypartners.com.
authors.forename: Fotios
authors.initials: F
authors.lastname: Kokkotos
authors.forename: Adam
authors.initials: A
authors.lastname: Lustig
authors.forename: Peter J
authors.initials: PJ
authors.lastname: Neumann
authors.forename: Daryl
authors.initials: D
authors.lastname: Pritchard
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Stewart
authors.forename: Robert W
authors.initials: RW
authors.lastname: Dubois
mesh_terms: D000893:Anti-Inflammatory Agents / Q000191:economics / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000191:economics / Q000627:therapeutic use*; D018501:Antirheumatic Agents / Q000191:economics / Q000627:therapeutic use*; D001172:Arthritis, Rheumatoid / Q000188:drug therapy* / Q000191:economics; D001943:Breast Neoplasms / Q000188:drug therapy* / Q000191:economics; D003362:Cost-Benefit Analysis; D016527:Drug Costs; D005260:Female; D006801:Humans; D009103:Multiple Sclerosis / Q000188:drug therapy* / Q000191:economics
chemical_list: D000893:Anti-Inflammatory Agents; D000970:Antineoplastic Agents; D018501:Antirheumatic Agents