Pediatric Gene Therapy Launches

enorman@mit.eduFoCUS, Research Briefs

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Gene therapy cures for hereditary genetic conditions are likely to reach the United States market in the coming decades. Several disease areas with potential cures are orphan conditions, which affect fewer than 200,000 patients in the US.1 To understand the current cost burden associated with managing orphan conditions, we analyzed 55,188 pediatric inpatient stays across disease areas including select hemoglobinopathies, bleeding disorders, lysosomal storage diseases, cystic fibrosis, and other enzymatic deficiencies. We find national pediatric inpatient cost estimates of nearly $1 billion in 2016, attributable to our eleven orphan diseases of interest—despite accounting for less than one percent of pediatric inpatient stays that year.

Download FoCUS Research Brief 2019F205v038

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