This presentation makes a very important point about rare diseases and how small sample sizes can inhibit a robust assessment of the uncertainties surrounding the parameters of a cost-effectiveness analysis. It concerns me that decision makers may deny reimbursement for a technology because of some flawed methdology in correctly estimating effectiveness for rare diseases in small subgroups like children. It behooves us to take a look at the limitations of the statistics before denying coverage.
This presentation makes a very important point about rare diseases and how small sample sizes can inhibit a robust assessment of the uncertainties surrounding the parameters of a cost-effectiveness analysis. It concerns me that decision makers may deny reimbursement for a technology because of some flawed methdology in correctly estimating effectiveness for rare diseases in small subgroups like children. It behooves us to take a look at the limitations of the statistics before denying coverage.
TheGrayIntellectual 1 month ago