Go Broad: The Application of Indirect Burden of Disease Evidence in US Value Assessment and Ex-US HTA Submissions

Rare diseases often require a significant amount of medical care which leads to a higher burden of direct medical costs. Additionally, indirect costs such as productivity loss, absenteeism, lifestyle disruptions, and family and societal impacts, contribute to the overall burden of patients with rare disease and their caregivers. However, these broad indirect impacts are less understood and not often measured and thus burden of disease estimates utilized in evidence for value assessment and ex-US HTA submissions are thought to be underestimating the total burden of disease.

Laura Housman will moderate the panel and provide a 5 outline of the issue including the difference between direct medical costs, non-medical costs, and indirect costs. Taylor Schwartz will provide a 10 minute overview of recently completed studies that utilized novel methodologies to measure non-medical costs and the broad indirect burden of disease as components of the overall burden of disease. He will also provide a comparative perspective as to how this information can be utilized by US value assessment and payers. Jan McKendrick will spend 10 minutes discussing providing a comparative perspective as to how measuring the indirect burden of disease can be useful as part of the evidence submissions in ex-US HTA submissions and how these evidence requirements may vary from country to country. Karl-Johan Myren will spend a 10 minutes sharing the perspective of the manufacturer on the pragmatic application of utilizing data on the indirect burden of diseases in EU HTA submissions. and the importance of indirect disease burden measurement for patient-centered drug development.