Use of rhDNase Therapy and Costs of Respiratory-Related Care in Patients with Cystic Fibrosis

Ollendorf, Daniel A.; McGarry, Lisa; Watrous, Marc; Oster, Gerry

doi:10.1345/1542-6270(2000)034<0304:uortac>2.3.co;2

Cited by 1 publication

(1 citation statement)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The study raises awareness about the lack of research on the socio-economic impact of rare diseases, which can be substantial, as well as methodological issues related to the comparability of the available evidence across borders which need to be addressed in future research. Prevalence 1:2,500 -3,600 newborns (13)(14) 1:3,600 male infants (18) 1:3,600 males; 1:4,000 -6,000 females (20) A: 1:5,000 -10,000 males B: 1:20,000 -34,000 males (21) 8-150:100,00 children (22) Type I 1:100,000; Type II 1:250,000; Type III 1:50,000 to 1:280,000; Type IV 1:75,000; Type VI 1:250,000 (23)(24)(25)(26)(27) 74:100,000 women; 13:100,000 men (30) 1:22,000 newborns (32) 1:150,000 (NMD) (35) 5:100,000 (37) Prevalence (per 100,000 population) (16) A c c e p t e d M a n u s c r i p t 33 A c c e p t e d M a n u s c r i p t 34 9-57% (14)(15)(50)(51)(52)(53)(54)(55)(56)(57)(58)(59)(60)(61) n/a 15-81% (15,(50)(51)(52)(53)(54)(55)(56)(57)(58)(59)(60)(61) DMD 541,593 (65) n/a n/a 1...…”

Section: Discussionmentioning

confidence: 99%

Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

2015

View full text Add to dashboard Cite

A c c e p t e d M a n u s c r i p t 2 Highlights  We systematically reviewed the socioeconomic cost literature for 10 rare diseases. Direct and indirect costs incurred by patients, carers and society were searched. 77 studies were included with an unequal distribution of studies over disease types. Level of existing evidence is highest for diseases with available drug treatments. Indirect costs are in most cases of similar or higher magnitude than direct costs. found to range between 1-2 per 100,000 population (PWS, a sub-type of Histiocytosis, and EB) up to 42 per 100,000 population (Scleroderma). Overall, cost evidence on rare diseases appears to be very scarce (a total of 77 studies were identified across all diseases), with CF (n=29) and Haemophilia (n=22) being relatively well studied, compared to the other conditions, where very limited cost of illness information was available. In terms of data availability, total lifetime cost figures were found only across four diseases, and total annual costs (including indirect costs) across five diseases.Overall, data availability was found to correlate with the existence of a pharmaceutical treatment and indirect costs tended to account for a significant proportion of total costs.Although methodological variations prevent any detailed comparison between conditions, most of the rare diseases examined are associated with significant economic burden, both direct and indirect.

show abstract