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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