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 |