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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ATC Total (89) Reimbursed (58) Not Reimbursed (11) Ongoing negotiation/withdrawn (20)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
  1. ASMR Amélioration du Service Médical Rendu, GU Gazzetta Ufficiale della Repubblica Italiana, NA's Not available, Qu Quartile, RCT Randomized Clinical Trial