Disentangling the Cost of Orphan Drugs Marketed in the United States
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 tre...
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| Format: | Article |
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MDPI AG
2023-02-01
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| Series: | Healthcare |
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| Online Access: | https://www.mdpi.com/2227-9032/11/4/558 |
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| author | Hana Althobaiti Enrique Seoane-Vazquez Lawrence M. Brown Marc L. Fleming Rosa Rodriguez-Monguio |
| author_facet | Hana Althobaiti Enrique Seoane-Vazquez Lawrence M. Brown Marc L. Fleming Rosa Rodriguez-Monguio |
| author_sort | Hana Althobaiti |
| collection | DOAJ |
| description | 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 (<i>p</i> < 0.001). Higher market entry prices were associated with biologics (108%; <i>p</i> < 0.001), orphan status (177%; <i>p</i> < 0.001), US sponsor companies (48%; <i>p</i> = 0.035), chronic use (1083%; <i>p</i> < 0.001), treatment intent (163%; <i>p</i> = 0.004), and indications for oncology (624%; <i>p</i> < 0.001) or genetic disorders (624%; <i>p</i> < 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. |
| first_indexed | 2024-03-11T08:46:27Z |
| format | Article |
| id | doaj.art-f60d7acdbe7d455d968f016d52848ee8 |
| institution | Directory Open Access Journal |
| issn | 2227-9032 |
| language | English |
| last_indexed | 2024-03-11T08:46:27Z |
| publishDate | 2023-02-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Healthcare |
| spelling | doaj.art-f60d7acdbe7d455d968f016d52848ee82023-11-16T20:46:53ZengMDPI AGHealthcare2227-90322023-02-0111455810.3390/healthcare11040558Disentangling the Cost of Orphan Drugs Marketed in the United StatesHana Althobaiti0Enrique Seoane-Vazquez1Lawrence M. Brown2Marc L. Fleming3Rosa Rodriguez-Monguio4Department of Clinical Pharmacy, College of Pharmacy, Umm Al-Qura University, Makkah 24382, Saudi ArabiaDepartment of Pharmaceutical Economics and Policy Department, Chapman University School of Pharmacy, Irvine, CA 92618, USADepartment of Pharmaceutical Economics and Policy Department, Chapman University School of Pharmacy, Irvine, CA 92618, USADepartment of Pharmaceutical Economics and Policy Department, Chapman University School of Pharmacy, Irvine, CA 92618, USADepartment of Clinical Pharmacy, School of Pharmacy, University of California San Francisco, San Francisco, CA 94143, USAThe 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 (<i>p</i> < 0.001). Higher market entry prices were associated with biologics (108%; <i>p</i> < 0.001), orphan status (177%; <i>p</i> < 0.001), US sponsor companies (48%; <i>p</i> = 0.035), chronic use (1083%; <i>p</i> < 0.001), treatment intent (163%; <i>p</i> = 0.004), and indications for oncology (624%; <i>p</i> < 0.001) or genetic disorders (624%; <i>p</i> < 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.https://www.mdpi.com/2227-9032/11/4/558orphan drugsnon-orphan drugspricemarket entry |
| spellingShingle | Hana Althobaiti Enrique Seoane-Vazquez Lawrence M. Brown Marc L. Fleming Rosa Rodriguez-Monguio Disentangling the Cost of Orphan Drugs Marketed in the United States Healthcare orphan drugs non-orphan drugs price market entry |
| title | Disentangling the Cost of Orphan Drugs Marketed in the United States |
| title_full | Disentangling the Cost of Orphan Drugs Marketed in the United States |
| title_fullStr | Disentangling the Cost of Orphan Drugs Marketed in the United States |
| title_full_unstemmed | Disentangling the Cost of Orphan Drugs Marketed in the United States |
| title_short | Disentangling the Cost of Orphan Drugs Marketed in the United States |
| title_sort | disentangling the cost of orphan drugs marketed in the united states |
| topic | orphan drugs non-orphan drugs price market entry |
| url | https://www.mdpi.com/2227-9032/11/4/558 |
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