title: A Value-Based Medicine cost-utility analysis of genetic testing for neovascular macular degeneration.
abstract: BACKGROUND
  There is a dearth of patient, preference-based cost-effectiveness analyses evaluating genetic testing for neovascular age-related macular degeneration (NVAMD).
  
  
  METHODS
  A Value-Based Medicine, 12-year, combined-eye model, cost-utility analysis evaluated genetic testing of Category 3 AMD patients at age 65 for progression to NVAMD. The benefit of genetic testing was predicated upon the fact that early-treatment ranibizumab therapy (baseline vision 20/40-20/80) for NVAMD confers greater patient value than late-treatment (baseline vision ≤20/160). Published genetic data and MARINA Study ranibizumab therapy data were utilized in the analysis. Patient value (quality-of-life gain) and financial value (2012 US real dollar) outcomes were discounted at 3 % annually.
  
  
  RESULTS
  Genetic testing-enabled, early-treatment ranibizumab therapy per patient conferred mean 20/40-1vision, a 0.845 QALY gain and 14.1 % quality-of-life gain over sham therapy. Late-treatment ranibizumab therapy conferred mean 20/160+2vision, a 0.250 QALY gain and 4.2 % quality-of-life gain over sham therapy. The gain from early-treatment over late-treatment was 0.595 QALY (10.0 % quality-of-life gain). The per-patient cost for genetic testing/closer monitoring was $2205 per screened person, $2.082 billion for the 944,000 estimated new Category 3 AMD patients annually. Genetic testing/monitoring costs per early-treatment patient totaled $66,180. Costs per early-treatment patient included: genetic testing costs: $66,180 + direct non-ophthalmic medical costs: -$40,914 + caregiver costs: -$172,443 + employment costs: -$14,098 = a net societal cost saving of $160,582 per early treatment patient. When genetic screening facilitated an incremental 12,965 (8.0 %) of the 161,754, new annual NVAMD patients aged ≥65 in the US to undergo early-treatment ranibizumab therapy, each additional patient treated accrued an overall, net financial gain for society of $160,582. Genetic screening was cost-effective, using World Health Organization criteria, when it enabled an incremental 4.1 % (6634) of 161,754 annual NVAMD patients ≥65 years to receive early-treatment ranibizumab therapy.
  
  
  CONCLUSIONS
  Genetic screening-enabled, early-treatment ranibizumab therapy for NVAMD is cost-effective if it enables an incremental 4.1 % of the annual US cohort of new-onset NVAMD patients ≥65 to undergo early-treatment with ranibizumab.
authors.affiliation: Center for Value-Based Medicine®, 6010 West Mill Road, Flourtown, PA 19031 USA.|The Eye Research Institute, Philadelphia, PA USA.|The Retina Service, Wills Eye Hospital, Jefferson Medical University, Philadelphia, PA USA.
authors.forename: Gary C
authors.initials: GC
authors.lastname: Brown
authors.affiliation: Center for Value-Based Medicine®, 6010 West Mill Road, Flourtown, PA 19031 USA.|The Eye Research Institute, Philadelphia, PA USA.|The Research Department, Wills Eye Hospital, Jefferson Medical University, Philadelphia, PA USA.
authors.forename: Melissa M
authors.initials: MM
authors.lastname: Brown
authors.affiliation: Center for Value-Based Medicine®, 6010 West Mill Road, Flourtown, PA 19031 USA.
authors.forename: Heidi B
authors.initials: HB
authors.lastname: Lieske
authors.affiliation: Center for Value-Based Medicine®, 6010 West Mill Road, Flourtown, PA 19031 USA.
authors.forename: Philip A
authors.initials: PA
authors.lastname: Lieske
authors.affiliation: Center for Value-Based Medicine®, 6010 West Mill Road, Flourtown, PA 19031 USA.
authors.forename: Kathryn S
authors.initials: KS
authors.lastname: Brown
keywords: Comparative effectiveness; Cost-effectiveness; Genetic testing; Neovascular macular degeneration