PMID- 36833091
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20230228
IS  - 2227-9032 (Print)
IS  - 2227-9032 (Electronic)
IS  - 2227-9032 (Linking)
VI  - 11
IP  - 4
DP  - 2023 Feb 13
TI  - Disentangling the Cost of Orphan Drugs Marketed in the United States.
LID - 10.3390/healthcare11040558 [doi]
LID - 558
AB  - 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.
FAU - Althobaiti, Hana
AU  - Althobaiti H
AD  - Department of Clinical Pharmacy, College of Pharmacy, Umm Al-Qura University, 
      Makkah 24382, Saudi Arabia.
FAU - Seoane-Vazquez, Enrique
AU  - Seoane-Vazquez E
AD  - Department of Pharmaceutical Economics and Policy Department, Chapman University 
      School of Pharmacy, Irvine, CA 92618, USA.
AD  - Economic Science Institute, Argyros School of Business and Economics, Chapman 
      University, Orange, CA 92866, USA.
FAU - Brown, Lawrence M
AU  - Brown LM
AD  - Department of Pharmaceutical Economics and Policy Department, Chapman University 
      School of Pharmacy, Irvine, CA 92618, USA.
FAU - Fleming, Marc L
AU  - Fleming ML
AD  - Department of Pharmaceutical Economics and Policy Department, Chapman University 
      School of Pharmacy, Irvine, CA 92618, USA.
FAU - Rodriguez-Monguio, Rosa
AU  - Rodriguez-Monguio R
AD  - Department of Clinical Pharmacy, School of Pharmacy, University of California San 
      Francisco, San Francisco, CA 94143, USA.
AD  - Medication Outcomes Center, University of California San Francisco, San 
      Francisco, CA 94143, USA.
AD  - Philip R. Lee Institute for Health Policy Studies at the University of California 
      San Francisco, San Francisco, CA 94143, USA.
LA  - eng
PT  - Journal Article
DEP - 20230213
PL  - Switzerland
TA  - Healthcare (Basel)
JT  - Healthcare (Basel, Switzerland)
JID - 101666525
PMC - PMC9957503
OTO - NOTNLM
OT  - market entry
OT  - non-orphan drugs
OT  - orphan drugs
OT  - price
COIS- The authors declare that they have no competing interests.
EDAT- 2023/02/26 06:00
MHDA- 2023/02/26 06:01
PMCR- 2023/02/13
CRDT- 2023/02/25 02:09
PHST- 2022/12/30 00:00 [received]
PHST- 2023/02/05 00:00 [revised]
PHST- 2023/02/08 00:00 [accepted]
PHST- 2023/02/25 02:09 [entrez]
PHST- 2023/02/26 06:00 [pubmed]
PHST- 2023/02/26 06:01 [medline]
PHST- 2023/02/13 00:00 [pmc-release]
AID - healthcare11040558 [pii]
AID - healthcare-11-00558 [pii]
AID - 10.3390/healthcare11040558 [doi]
PST - epublish
SO  - Healthcare (Basel). 2023 Feb 13;11(4):558. doi: 10.3390/healthcare11040558.