Table.1 Orphan drugs approved in 2014–2019 per ATC, their reimbursement status and descriptive statistics about the variables used in the analysis
From: Variables affecting pricing of orphan drugs: the Italian case
ATC | Total (89) | Reimbursed (58) | Not Reimbursed (11) | Ongoing negotiation/withdrawn (20) |
|---|---|---|---|---|
A—alimentary tract and metabolism | 16 | 9 | 2 | 5 |
B—blood and blood forming organs | 7 | 2 | 2 | 3 |
C—cardiovascular system | 4 | 2 | 0 | 2 |
D—dermatologicals | 2 | 1 | 0 | 1 |
J—anti-infective agents for systemic use | 7 | 5 | 1 | 1 |
L—antineoplastic and immunomodulating agents | 38 | 31 | 4 | 3 |
M—musculoskeletal system | 3 | 3 | 0 | 0 |
N—nervous system | 3 | 1 | 1 | 1 |
R—respiratory system | 3 | 1 | 1 | 1 |
S—sensory organs | 5 | 3 | 0 | 2 |
H—systemic hormonal preparations (excl. sex hormones and insulins) | 1 | 0 | 0 | 1 |
Year of P&R in Italy (GU) | 2014 | 2015 | 2016 | 2017 | 2018 | 2019 |
|---|---|---|---|---|---|---|
TOT num of products (negotiation concluded) | 5 | 8 | 14 | 19 | 13 | 10 |
Variable | Min | 1st Qu | Median | Mean | 3rd Qu | Max | NA’s |
|---|---|---|---|---|---|---|---|
Annual treatment cost (€) | 3934 | 44,959 | 66,601 | 140,606 | 165,465 | 1,100,066 | 0 |
Prevalence per 100 k population | 0.09 | 1.30 | 5.31 | 10.89 | 12.75 | 114.10 | 0 |
RCT | 0.00 | 0.00 | 1.00 | 0.65 | 1.00 | 1.00 | 3 |
ASMR | 1.00 | 3.00 | 4.00 | 3.89 | 4.50 | 5.00 | 8 |