Masaquel C, Miller-Wilson LA, D'Souza V, Schwinn J, Mina-Osorio P. Evidence gap analysis of the burden of disease in thyroid eye disease. Poster presented at the ISPOR 2024; May 7, 2024. Atlanta, GA.

OBJECTIVES: Thyroid eye disease (TED) is a debilitating, sight-threatening orbital inflammatory autoimmune disorder caused by the activation of pathogenic anti–thyroid-stimulating hormone receptor autoantibodies, leading to a range of ophthalmic manifestations. We assessed the scientific literature to identify evidence gaps related to disease burden and treatment patterns in TED.

METHODS: A structured literature review was conducted in PubMed, Embase, and the Cochrane Library using a predefined search strategy for articles published from May 5, 2013 to May 5, 2023 on the epidemiology; clinical, humanistic, and economic burden; and treatment patterns in TED. Online searches were also conducted for health technology assessments, prescribing information, treatment guidelines, and ongoing clinical trials.

RESULTS: We reviewed 209 articles. The lack of standardized diagnostic criteria for the classification of TED, as well as geographic and ethnic variations present challenges in diagnosis and epidemiological estimates. Studies utilizing questionnaires such as the Graves’ orbitopathy-specific quality of life (QOL) and TED-QOL provide insights into the multifaceted humanistic burden of TED; however, longitudinal data on health-related QOL are limited. While a significant economic burden related to TED has been reported, the long-term impact on healthcare costs has not been adequately studied. Globally, steroids are the most utilized treatment for active, moderate-to-severe TED. Intravenous glucocorticoids are considered standard first-line treatment. Monoclonal antibodies (e.g., teprotumumab, rituximab, and tocilizumab) are associated with numerous side effects, and studies on their efficacy in patients with TED reported conflicting results. More real-world evidence studies are needed to better understand the risk-benefit profiles as well as longitudinal outcomes of available TED therapies.

CONCLUSION: We identified several gaps in the literature including the lack of standardized diagnostic criteria for TED, the lack of robust long-term data on current TED therapies, and the need for additional clinical, humanistic, and economic burden studies in patients with TED.

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