Limitations
DISEASE, LENS
We caution that these results and the limited model horizon should not be extrapolated beyond the lens of the clinical trial period, as they only provide early estimates using the preliminary data to support the underlying clinical trial hypothesis, and the demonstrated need of the clinical trial to provide true efficacy and mortality estimates. By incorporating this decision analytic structure prior to enrollment, the same approach can be updated during any pause for interim analysis to update prior parameter estimates. The final decision analytic model can be updated following the completion of the clinical trial using data from the trial itself. Specifically, follow-up decision analysis can then be based on both clinical trial data and real-world epidemiological estimates with a lengthened time horizon (a lifetime horizon with long-term follow up data that includes QALY utility adjustments over a longer period of time) that truly captures the disease burden and trajectory, at the societal- or country-level perspectives. These estimates can also provide the basis for finalized cost estimates, such as those discounted at a 3% annual rate per the guidelines of the Public Health Service Panel on Cost Effectiveness in Health and Medicine or other projections of market pressure and resource allocation constraints [