K Thiran Jayasundera1, Rebhi O Abuzaitoun1, Gabrielle D Lacy1, Maria Fernanda Abalem2, Gregory M Saltzman3, Thomas A Ciulla4, Mark W Johnson5. 1. From the Kellogg Eye Center, Department of Ophthalmology and Visual Sciences (K.T.J., R.O.A.,G.D.L., M.F.A., M.W.J.). 2. From the Kellogg Eye Center, Department of Ophthalmology and Visual Sciences (K.T.J., R.O.A.,G.D.L., M.F.A., M.W.J.); University of Michigan Medical School, Ann Arbor, Michigan, USA; Department of Ophthalmology and Otolaryngology (M.F.A.). 3. University of São Paulo Medical School, São Paulo, São Paulo, Brazil; Department of Economics and Management (G.M.S.); Albion College, Albion, Michigan, USA; Department of Health Management and Policy (G.M.S.). 4. University of Michigan, Ann Arbor, Michigan, USA; Department of Ophthalmology (T.A.C.); Eugene and Marilyn Glick Eye Institute, Indiana University, Indianapolis, Indiana, USA; Clearside Biomedical, Inc (T.A.C.); Alpharetta, Georgia, USA; Retina Service, Midwest Eye Institute (T.A.C.), Carmel, Indiana, USA. 5. From the Kellogg Eye Center, Department of Ophthalmology and Visual Sciences (K.T.J., R.O.A.,G.D.L., M.F.A., M.W.J.). Electronic address: markwj@med.umich.edu.
Abstract
PURPOSE: To investigate the challenges and potential improvement strategies of cost-effectiveness analyses performed for therapeutics targeting inherited retinal diseases (IRDs). DESIGN: Perspective. METHODS: A literature review was conducted with discussion of current limitations and improvement recommendations. RESULTS: Cost-effectiveness analysis (CEA) performed for IRD therapeutics has multiple limitations. First, the available methods used to measure health-related quality of life and health utilities can be inaccurate when used in IRDs. Second, the financial burden to patients and society from vision impairment associated with IRDs has been inadequately studied and includes a variety of expenditures ranging from direct costs of IRD specialty health care to indirect expenses associated with daily living activities. Third, our collective understanding is limited in the areas of IRD natural history and health benefits gained from new IRD treatments (eg, gene therapies). In addition, the therapeutic effect from a patient perspective and its duration of action are not fully understood. Due to the scarcity of data, CEA for newly approved therapies has relied on assumptions and creations of predictive models for both costs and health benefits for these new therapeutics in order to calculate the incremental cost-effectiveness ratio. CONCLUSIONS: CEA studies performed for IRD therapeutics have been limited by the established health utilities in ophthalmology and the lack of disease-specific information. The assumptions and extrapolations in these studies create substantial uncertainty in incremental cost-effectiveness ratio results. An improved framework is required for CEA of IRD therapeutics in order to determine the cost-effectiveness of each therapy brought from clinical trials to clinical practice.
PURPOSE: To investigate the challenges and potential improvement strategies of cost-effectiveness analyses performed for therapeutics targeting inherited retinal diseases (IRDs). DESIGN: Perspective. METHODS: A literature review was conducted with discussion of current limitations and improvement recommendations. RESULTS: Cost-effectiveness analysis (CEA) performed for IRD therapeutics has multiple limitations. First, the available methods used to measure health-related quality of life and health utilities can be inaccurate when used in IRDs. Second, the financial burden to patients and society from vision impairment associated with IRDs has been inadequately studied and includes a variety of expenditures ranging from direct costs of IRD specialty health care to indirect expenses associated with daily living activities. Third, our collective understanding is limited in the areas of IRD natural history and health benefits gained from new IRD treatments (eg, gene therapies). In addition, the therapeutic effect from a patient perspective and its duration of action are not fully understood. Due to the scarcity of data, CEA for newly approved therapies has relied on assumptions and creations of predictive models for both costs and health benefits for these new therapeutics in order to calculate the incremental cost-effectiveness ratio. CONCLUSIONS: CEA studies performed for IRD therapeutics have been limited by the established health utilities in ophthalmology and the lack of disease-specific information. The assumptions and extrapolations in these studies create substantial uncertainty in incremental cost-effectiveness ratio results. An improved framework is required for CEA of IRD therapeutics in order to determine the cost-effectiveness of each therapy brought from clinical trials to clinical practice.
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