Pharmacoeconomics and Pharmaceutical Economics Commons^™

Disentangling The Cost Of Orphan Drugs Marketed In The United States, Hana Althobaiti, Enrique Seoane-Vazquez, Lawrence B. Brown, Marc L. Fleming, Rosa Rodriguez-Monguio

Pharmacy Faculty Articles and Research

The increasing number and high prices of orphan drugs have triggered concern among patients, payers, and policymakers about the affordability of new drugs approved using the incentives set by the Orphan Drug Act (ODA) of 1983. This study evaluated the factors associated to the differences in the treatment cost of new orphan and non-orphan drugs approved by the FDA from 2017 to 2021. A generalized linear model (GLM) with the Gamma log-link analysis was used to ascertain the association of drug characteristics with the treatment costs of orphan and non-orphan drugs. The results of the study showed that the median …

Ethical Imperatives Of Timely Access To Orphan Drugs: Is Possible To Reconcile Economic Incentives And Patients’ Health Needs?, Rosa Rodriguez-Monguio, T. Spargo, Enrique Seoane-Vazquez

Pharmacy Faculty Articles and Research

Background

More than 6,800 rare diseases and conditions have been identified in the US, which affect 25–30 million Americans. In 1983, the US Congress enacted the Orphan Drug Act (ODA) to encourage the development and marketing of drugs to treat rare diseases and conditions. This study analyzed all orphan designations and FDA approvals since 1983 through 2015, discussed the effectiveness of incentives for the development of treatments for rare diseases, and reflected on the ethical imperatives for timely access to orphan drugs.

Methods

Study data were derived from the Food and Drug Administration (FDA) Orange Book and the Office of …