Date of Award
The purpose of this study is to evaluate the Orphan Drug Act of 1983 by subtracting the cost of incentives for drug development from the estimated value created by each drug in the form of quality-adjusted life-year gains. Data regarding costs of incentives is retrieved from the FDA as well as a report released by the National Organization for Rare Disorders. Data regarding value created by each drug is retrieved from a Freedom of Information Act request to the FDA, the CEA registry, and a report released by the US Department of Health and Human Sciences. Sufficient data was available for a total of 16 drugs. The net average value created for the data set was $44.04 billion assuming 100% treatment scenario and $4.336 billion assuming 10% treatment scenario. It is unclear whether the calculations performed in this study are indicative of the Orphan Drug Designation program as a whole as the criteria for inclusion of data in this study was nonrandom. More research is needed to determine a representative data sample for orphan drugs in order to evaluate the Orphan Drug Act more fully.
Castellanos, Nicholas, "Indie Illness: An Evaluation of the Orphan Drug Act" (2022). Honors Theses. 2738.