A preliminary model-based assessment of the cost-utility of a screening programme for early age-related macular...

To estimate the cost-effectiveness of screening for age-related macular degeneration (AMD) by developing a decision analytic model that incorporated and assessed all of the National Screening Committee criteria. A further objective was to identify the major areas of uncertainty in the model, and so inform future research priorities in this disease area.

Major databases were searched in March 2004 and updated in January 2005.

Systematic literature reviews covered the epidemiology and natural history of AMD, the screening and treatment effectiveness and health-related quality of life relating to AMD. A hybrid cohort-individual sampling model was implemented to describe the range of pathways between the incidence of age-related maculopathy (ARM) and death via clinical presentation and treatment at different stages of the disease. As significant shortfalls in the data available from the literature were apparent, so a range of primary data sources were also used to populate the model. To obtain estimates for the value of parameters deemed to be within an expert's remit, data describing some parameters were elicited from relevant experts. The data identified informed probability distributions describing the uncertainty around the model parameters. To incorporate joint parameter uncertainty (i.e. correlations between parameters), the AMD natural history model was calibrated probabilistically. Randomly sampled sets of input parameters were assigned weights representing the accuracy of their predictions of a set of observed model outputs. The analysis of the AMD screening model estimated the costs, numbers of quality-adjusted life-years (QALYs) and cases of blindness in a general population sample of 50-year-olds over the remainder of their lifetime, for 16 alternative screening options (including no screening). The reference case analysis incorporated current treatment options of laser photocoagulation and photodynamic therapy. Sensitivity analyses describing six alternative sets of intervention strategies, based on horizon scanning of potential future treatments for AMD, were also undertaken.

There remains significant uncertainty about whether any form of screening for AMD is cost-effective. However, annual screening from age 60 years seems to provide the highest mean net benefits, but this is based on a cost-effectiveness estimate that has very poor precision (high levels of uncertainty). The probabilistic sensitivity analysis shows that the 95% credible interval for annual screening from age 60 years ranges from this option dominating the previous option to an incremental cost per QALY of over 0.5 million pounds sterling. Plotting a cost-effectiveness acceptability frontier shows that although annual screening from age 60 years has the highest net benefits at a value of QALY of 30,000 pounds sterling, the associated probability of this option being the most cost-effective option is only around 20%. The sensitivity analyses around potential future treatment options indicate that screening may become more cost-effective with the new treatments.

The conclusions focus on the interpretation of the results from the perspective of defining the major areas of uncertainty, which were defined as disease progression, rates of clinical presentation, screening test and optician effectiveness, treatment effectiveness, and costs of blindness. Future research may be best targeted at assessing how routine data may be used to describe clinical presentation rates of ARM. Other potential studies include a pilot study of the effectiveness of screening and opticians' referral patterns for AMD and a costing study of blindness as a continuum of association with deterioration in vision.