Estimating the clinical cost of drug development for orphan versus non-orphan drugs.
Cost of drug development
Orphan drugs
Rare diseases
Journal
Orphanet journal of rare diseases
ISSN: 1750-1172
Titre abrégé: Orphanet J Rare Dis
Pays: England
ID NLM: 101266602
Informations de publication
Date de publication:
10 01 2019
10 01 2019
Historique:
received:
05
06
2018
accepted:
28
12
2018
entrez:
12
1
2019
pubmed:
12
1
2019
medline:
7
5
2019
Statut:
epublish
Résumé
High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool. However, estimates of the cost of orphan drug development are sparse. Using publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs. We found that the out-of-pocket clinical costs per approved orphan drug to be $166 million and $291 million (2013 USD) per non-orphan drug. The capitalized clinical costs per approved orphan drug and non-orphan drug were estimated to be $291 million and $412 million respectively. When focusing on new molecular entities only, we found that the capitalized clinical cost per approved orphan drug was half that of a non-orphan drug. More discussion is needed to better align on which cost components should be included in research and development costs for pharmaceuticals.
Sections du résumé
BACKGROUND
High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool. However, estimates of the cost of orphan drug development are sparse.
METHODS
Using publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs.
RESULTS
We found that the out-of-pocket clinical costs per approved orphan drug to be $166 million and $291 million (2013 USD) per non-orphan drug. The capitalized clinical costs per approved orphan drug and non-orphan drug were estimated to be $291 million and $412 million respectively. When focusing on new molecular entities only, we found that the capitalized clinical cost per approved orphan drug was half that of a non-orphan drug.
CONCLUSIONS
More discussion is needed to better align on which cost components should be included in research and development costs for pharmaceuticals.
Identifiants
pubmed: 30630499
doi: 10.1186/s13023-018-0990-4
pii: 10.1186/s13023-018-0990-4
pmc: PMC6327525
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
12Références
Orphanet J Rare Dis. 2011 Jun 17;6:42
pubmed: 21682893
Orphanet J Rare Dis. 2014 Nov 26;9:170
pubmed: 25427578
J Health Econ. 2016 May;47:20-33
pubmed: 26928437
JAMA. 2012 May 2;307(17):1838-47
pubmed: 22550198
J Pharm Bioallied Sci. 2010 Oct;2(4):290-9
pubmed: 21180460
Nat Rev Drug Discov. 2010 Dec;9(12):921-9
pubmed: 21060315
PLoS One. 2014 Nov 25;9(11):e113894
pubmed: 25422889
Drug Discov Today. 2012 Jul;17(13-14):660-4
pubmed: 22366309
JAMA. 2013 Oct 2;310(13):1343-4
pubmed: 24084916
Ann Neurol. 2009 Aug;66(2):184-90
pubmed: 19743448
JAMA Intern Med. 2017 Nov 1;177(11):1569-1575
pubmed: 28892524
Lancet. 2008 Jun 14;371(9629):2041-4
pubmed: 18555916
Drug Dev Res. 2014 Jun;75(4):231-4
pubmed: 24829189
Nat Biotechnol. 2014 Jan;32(1):40-51
pubmed: 24406927