NLM Gateway
A service of the U.S. National Institutes of Health
Your Entrance to
Resources from the
National Library of Medicine
    Home      Term Finder      Limits/Settings      Search Details      History      My Locker        About      Help      FAQ    
Skip Navigation Side Barintended for web crawlers only

Quality of life of a genetic disease, mucopolysaccharidosis and anticipation of efficacy and efficiency of its screening in Japan.

Hisashige A, Katayama T, Mikasa H; International Society of Technology Assessment in Health Care. Meeting.

Annu Meet Int Soc Technol Assess Health Care Int Soc Technol Assess Health Care Meet. 1996; 12: 53.

Department of Preventive Medicine, University of Tokushima, Japan.

OBJECTIVE: To evaluate quality of life of a genetic disease (mucopolysaccharidosis, MPS) and anticipate efficacy and efficiency of its neonatal mass screening in Japan. METHODS: Quality of life of MPS was evaluated by utility measurement (rating scale, standard gamble, and time trade-off). Subjects are 6 medical professionals and 93 medical students. Effectiveness of neonatal screening program for MPS was critically evaluated according to the standard of the U.S. Preventive Services Task Force. In addition, the efficiency of its screening program was evaluated by cost-effectiveness and cost-utility analyses based on the model of the results of critical appraisal. RESULTS: Utility values (by standard gamble) of MPS varied widely from 0.24 to 0.76 among its subtypes. High correlation coefficient of utility values was observed between medical professionals and medical students. Efficacy of a screening program were graded III for quality of evidence. If the effectiveness of this screening system was taken for granted, costs per life years gained and QALY gained of this program were anticipated to be $9,077 and $9,270, respectively (discount rate of costs: 5%). If both cost and health outcome were discounted at 5%, these CE ratios were $49,119 and $29,588. Sensitivity analysis for efficacy and discount rate confirmed these resutls. CONCLUSION: The quality of life for MPS was evaluated to be extremely low. On the assumption of efficacy of its screening which could improve its QOL, it was anticipated that the screening was relatively efficient. Therefore, before introduction of a nationwide mass screening for MPS, the effectiveness of a screening program for MPS must be established through a feasible method under the situation of its low incidence rate.

Publication Types:
  • Meeting Abstracts
Keywords:
  • Cost-Benefit Analysis
  • Incidence
  • Infant, Newborn
  • Japan
  • Mass Screening
  • Neonatal Screening
  • Quality-Adjusted Life Years
  • Sensitivity and Specificity
  • United States
  • diagnosis
  • economics
  • hsrmtgs
Other ID:
  • HTX/97607335
UI: 102222647

From Meeting Abstracts




Contact Us
U.S. National Library of Medicine |  National Institutes of Health |  Health & Human Services
Privacy |  Copyright |  Accessibility |  Freedom of Information Act |  USA.gov