orphan medicines figures 2000-2019 - european medicines agency€¦ · classified as public by the...
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Classified as public by the European Medicines Agency
An agency of the European Union
Orphan Medicines Figures2000- 2019
Orphan Medicines - Product Development Scientific Support
Classified as public by the European Medicines Agency
1
Applications for orphan medicinal product designation
2000
2005
2006
2010
2011
2015
2016 2017 2018 2019 Total
Applications
submitted
548 686 1151 329 260 236 233 3443
Positive COMP
Opinions
348 500 759 220 144 163 113 2247
Negative COMP
Opinions
8 6 7 2 2 3 2 30
EC Designations 343 485 768 209 147 169 112 2233
Withdrawals after
submission
150 144 313 77 100 92 104 980
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2
Applications for orphan medicinal product designation
0
50
100
150
200
250
300
350
2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019
submitted positive opinions negative opinions withdrawals during assessment EC Designations
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New orphan designated conditions
14
50
25 2431 34
26 21 17 17
33
15
4134 33 35 37
21 16 1214
64
4955
73
8880
98
73
106
128
107
148136
187 190
209
147
169
112
0
50
100
150
200
250
2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019
Number of new conditions Number of total designations
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4
Orphan drug designations based on significant benefit
14
64
49
55
73
88
80
98
73
106
128
107
148
136
187
190
209
147
169
112
7
36
34
49
51
47
56
74
56
86
60
72
97
77
119
113
124
99
113
75
0 50 100 150 200 250
2000
2001
2002
2003
2004
2005
2006
2007
2008
2009
2010
2011
2012
2013
2014
2015
2016
2017
2018
2019
Based on SB Total ODD
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Distribution of opinions on orphan designationby therapeutic area
Period 2000 – 2019 / Total opinions 2247
12%
8%
3%3%
1%2%
5%
33%
7%
14%
5%5% 2%
A-alimentary tract & metabolism B - blood & blood forming organs C - cardiovascular systemD - dermatology G - genito urinary tract H - systemic hormonal preparationsJ & P - antiinfectives & antiparasitic L - antineoplastic agents L - immunomodulating agentsM & N - musculoskeletal & nervous system R - respiratory system S - sensory organsV - various
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6
Distribution of orphan designations adult/paediatric use
Period 2000 – 2019/ Total designations 2233
Adult31%
Paediatric12%
Both57%
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Distribution of orphan designations adult/paediatric use
Total designations 2233
2 104 5 8 6
133 6
313 15
62
19 25 22 20 12 12 6
9
30 28
17 2239
33
49
43 7661
51
44
70113 113
155 114 13285
3
24 17
33 4343
34 46
2427
54 4142
4749 55
34 21 25 21
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019
Medical conditions affecting children only Medical conditions affecting both children and adults Medical conditions affecting adults only
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Prevalence fordesignated orphan conditions
Period 2000 – 2019 / Total designations 2233
less than 1 in 10,00040%
between 1 and 3 in 10,00048%
more than 3 in 10,000
12%
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9
Authorised orphan medicinal products
0
34
56
4
9
13
6
9
45
10
7
15 14 1414
22
5
11
7
12
8
16
21
15
28
6
0
5
10
15
20
25
30
2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019
Orphan marketing authorisations Orphan designations covered by marketing authorisations
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10
Authorisations by type of product excluding extensions of indications
113 authorisations including 7 withdrawals from the register of medicinal productshuman use/orphan status expired and 1 revoked/orphan status expired
16
2
5
2
219 1
6
6
3 4
51
3
1
1
1
1 1
18
1
11
3
2
131
2
A B C H L-immuno
L-onco M N R S V J
Therapeutic area
Chemicals Cell therapy GenetherapyBiological, Proteins Biological, coagulation factors Biological, monoclonal antibodiesBiological, oligonucleotides
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11
Authorisations by type of product including extensions of indications
196 products including withdrawals from register of orphan medicinal products, register medicinal products human use and expired orphan status
22
6
11
3
2
59
1
11
7
3
3
5
1
3
11
11
1
114
1
3
1
2
3
4 2 19
11
A B C H L- immuno L-onco M N R S V J
Chemicals Cell therapy GenetherapyBiological, Proteins Biological, coagulation factors Biological, monoclonal antibodiesBiological, oligonucleotides
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Chart includes:105 active initial authorisations; 16 extensions of indication20 withdrawals from the register of orphan medicinal products (including 8 ext. of indication)7 withdrawals from register medicinal products human use/ orphan status expired1 revoked from register medicinal products human use/ orphan status expired44 removals of initial MAA from register after expire of the market exclusivity period3 removals of extensions of indication
169 initial orphan marketing authorisations and 27 extension of indication granted to date
Number of conditions: 127
A - Alimentary tract and metabolism
19%
B - Haematology8%
C - Cardiovascular system5%
H - Systemic hormonal3%
L - Immunology4%
L - Antineoplastic41%
M - Musculo-skeletal system
1%
N - Nervous system8%
R - Respiratory system4%
S - Sensory organs2% V -Various
3%
J - Anti-infectives for systemic use
2%
A - Alimentary tract and metabolism B - Haematology
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Prevalence for orphan marketing authorisationsperiod 2000-2019
< 144%
1-226%
2-314%
3-516%
Prevalence per 10,000 All orphan products Including extensions of indication, withdrawals and products which market
exclusivity has expired
< 1 1-2 2-3 3-5
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