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Table 1 Characteristics of orphan drugs included in the analysis

From: Determinants of orphan drugs prices in France: a regression analysis

Variable Values Source
Total number 68  
Disease characteristics
Prevalence per 10,000, mean (sd) 1.19 (1.08) Public summary of opinion on orphan designation
Severity, n(%) Severe 13 (19%); Not severe 55 (81%) Public summary of opinion on orphan designation
Availability of alternative treatments, n(%) None 22 (32%); One 10 (15%); Several 30 (44%); Non-pharmacological 6 (9%)  
Drug characteristics
ATC class, n(%) A 14 (21%); B 3 (4%); C 4 (6%); G 1 (1%); H 3 (4%); J 3 (4%); L 32 (47%); N 5 (7%); R 1 (1%); V 2 (3%) European public assessment report
Treatment line, n(%) First 40 (59%); Subsequent 28 (41%) HTA report
Age of targeted population, n(%) Adults 34 (50%); Paediatric 15 (22%); Adults and paediatric 14 (21%); Elderly 5 (7%) European public assessment report
HTA details
AB score, n(%) Substantial 64 (94%); Moderate 2 (3%); Low 1 (1%); NR 1 (1%) HTA report
IAB score, n(%) I 6 (9%); II 25 (37%); III 14 (21%); IV 15 (22%); V 6 (9%); NR 2 (3%) HTA report
Commercialisation date See Fig. 2 Ameli database
Delay between HTA and commercialisation, mean (sd) months 6.68 (10.44) HTA report/ Ameli database
Study characteristics
Type of study, n(%) Phase II 14 (21%); Phase III 43 (63%); Other 11 (16%) HTA report
Comparator, n(%) None 20 (29%); Placebo 27 (40%); Active 18 (26%); NR 3 (4%) HTA report
Endpoint, n(%) Surrogate 45 (66%); Hard 12 (18%); NR 11 (16%) HTA report
Cost
Annual treatment cost, mean (sd) €96,518 (€169,942) Ameli database/European public assessment report
  1. ATC class: A Alimentary tract and metabolism; B Blood and blood forming organs; C Cardiovascular system; G Genito-urinary system and sex hormones; H Systemic hormonal preparations, excluding sex hormones and insulins; J Antiinfectives for systemic use; L Antineoplastic and immunomodulating agents; N Nervous system; R Respiratory system; V Various