This WFH video podcast features a conversation about Health Technology Assessment (HTA) and Comparative Effectiveness Research (CER). Hosting the discussion is Brian O'Mahony, past president of the WFH. He is joined by Dr. Michael Lauer of the National Heart, Blood and Lung Institute (USA), and health economist Adam Hutchings (UK).
Over the past 20 years, many developed countries have been able to provide an ample supply of factor concentrates for hemophilia treatment, with prophylaxis available for children and many adults. However, with the global economic recession, this environment is changing. HTA and CER measures are gaining in use as governments evaluate healthcare policies and make decisions about healthcare spending, presenting both challenges and opportunities to patient organizations and clinicians.
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 2 months ago