Disentangling the Cost of Orphan Drugs Marketed in the United States

Hana Althobaiti; Enrique Seoane-Vazquez; Lawrence M Brown; Marc L Fleming; Rosa Rodriguez-Monguio

doi:10.3390/healthcare11040558

Disentangling the Cost of Orphan Drugs Marketed in the United States

Healthcare (Basel). 2023 Feb 13;11(4):558. doi: 10.3390/healthcare11040558.

Authors

Hana Althobaiti¹, Enrique Seoane-Vazquez^{2

3}, Lawrence M Brown², Marc L Fleming², Rosa Rodriguez-Monguio^{4

5

6}

Affiliations

¹ Department of Clinical Pharmacy, College of Pharmacy, Umm Al-Qura University, Makkah 24382, Saudi Arabia.
² Department of Pharmaceutical Economics and Policy Department, Chapman University School of Pharmacy, Irvine, CA 92618, USA.
³ Economic Science Institute, Argyros School of Business and Economics, Chapman University, Orange, CA 92866, USA.
⁴ Department of Clinical Pharmacy, School of Pharmacy, University of California San Francisco, San Francisco, CA 94143, USA.
⁵ Medication Outcomes Center, University of California San Francisco, San Francisco, CA 94143, USA.
⁶ Philip R. Lee Institute for Health Policy Studies at the University of California San Francisco, San Francisco, CA 94143, USA.

Abstract

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 and interquartile range (IQR) drug cost was USD 218,872 (IQR = USD 23,105) for orphan drugs and USD 12,798 (IQR = USD 57,940) for non-orphan drugs (p < 0.001). Higher market entry prices were associated with biologics (108%; p < 0.001), orphan status (177%; p < 0.001), US sponsor companies (48%; p = 0.035), chronic use (1083%; p < 0.001), treatment intent (163%; p = 0.004), and indications for oncology (624%; p < 0.001) or genetic disorders (624%; p < 0.001). Higher market entry treatment cost for newly approved drugs were associated with biologics, orphan status, US sponsor companies, chronic use, therapeutic intent, and indications for oncology or genetic disorders.

Keywords: market entry; non-orphan drugs; orphan drugs; price.

Grants and funding

The work of Enrique Seoane-Vazquez and Rosa Rodriguez-Monguio was funded by a grant from Arnold Ventures. Arnold Ventures had no role in the design and conduct of the study, approval of the manuscript, and decision to submit the manuscript for publication.