orphan medicines figures 2000- 2020 · classified as public by the european medicines agency an...
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Classified as public by the European Medicines Agency
An agency of the European Union
Orphan Medicines Figures2000- 2020
Orphan Medicines – Scientific Evidence Generation
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 2020 Total
Applications
submitted
548 686 1151 329 260 236 233 235 3678
Positive COMP
Opinions
348 500 759 220 144 163 113 150 2397
Negative COMP
Opinions
8 6 7 2 2 3 2 2 32
EC Designations 343 485 768 209 147 169 112 149 2382
Withdrawals after
submission
150 144 313 77 100 92 105 82 1063
Classified as public by the European Medicines Agency
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 2020
submitted positive opinions negative opinions withdrawals during assessment EC Designations
Classified as public by the European Medicines Agency
3
235 applications submitted for orphan designation in 2020
53, 23%
182, 77%
SME
Non-SME
Classified as public by the European Medicines Agency
New orphan designated conditions
4
14
50
25 2431 34
26 21 17 17
33
15
4134 33 35 37
21 16 1221
14
64
4955
73
8880
98
73
106
128
107
148136
187 190
209
147
169
112
149
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 2020
Number of new conditions
Number of total designations
Classified as public by the European Medicines Agency
5
Orphan drug designations based on significant benefit
Total ODD
Based on SB
20202019
149112
64%67%
2018 169 67%2017 147 67%2016 209 59%2015 190 59%2014 187 64%2013 136 57%2012 148 66%2011 107 67%2010 128 47%2009 106 81%2008 73 77%2007 98 76%2006 80 70%2005 88 53%2004 73 70%2003 55 89%2002 49 69%2001 64 56%2000 14 50%
14
64
49
55
73
88
80
98
73
106
128
107
148
136
187
190
209
147
169
112
149
7
36
34
49
51
47
56
74
56
86
60
72
97
77
119
113
124
99
113
75
96
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
2020
Based on SB Total ODD
Classified as public by the European Medicines Agency
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Distribution of opinions on orphan designationby therapeutic area
Period 2000 – 2020 / Total opinions 2397
13%
8%
2%3%
1%
2%5%
32%
7%
15%
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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0 10 20 30 40 50 60
Blood and lymphatic system disorders
Respiratory, thoracic and mediastinal disorders
Congenital, familial and genetic disorders
Nervous system disorders
Eye disorders
skin and subcutaneous tissue disorders
Cardiac diseases
Endocrine disorders
Gastrointestinal disorders
Hepatobiliary disorders
Immune systems disorders
Infections and infestations
Muskuloskeletal and Connective tissue disorders
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Renal and urinary disorders
Surgical and medical procedures
Vascular disorders
Distribution of opinions on orphan designationMedDRA Classification
Period 2020 / Total opinions 150
Classified as public by the European Medicines Agency
8
Distribution of orphan designations adult/paediatric use
Period 2000 – 2020/ Total designations 2382
Adult30%
Paediatric12%
Both58%
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Distribution of orphan designations adult/paediatric use
Total designations 2382
2 104 5 8 6
133 6 3
13 15
62
19 25 22 20 12 12 6 13
9
30 28
17 2239
33
49
43 7661
51
44
70113 113
155 114 13285 96
3
24 17
33 4343
34 46
2427
54 4142
4749 55
34 21 25 2140
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 2020
Medical conditions affecting children only Medical conditions affecting both children and adults
Medical conditions affecting adults only
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10
Prevalence fordesignated orphan conditions
Period 2000 – 2020 / Total designations 2382
less than 1 in 10,00040%
between 1 and 3 in 10,00048%
more than 3 in 10,000
12%
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11
Authorised orphan medicinal products
0
34
56
4
9
13
6
9
45
10
7
15 14 1414
22
5
21
11
7
12
8
16
21
15
28
6
22
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 2020
Orphan marketing authorisations Orphan designations covered by marketing authorisations
Classified as public by the European Medicines Agency
12
Authorisations by type of product excluding extensions of indications
125 authorisations including 7 withdrawals from the register of medicinal productshuman use/orphan status expired and 1 revoked/orphan status expired
15
2
5
3
219
1
6
6
35
81
3
1
1
1
1
1
1
2
1
8
4
11
0
314 1
11
12
A B C H L -immuno
L -onco M N R S V J
Therapeutic area
Chemicals Cell therapyGenetherapy Biological, ProteinsBiological, coagulation factors Biological, monoclonal antibodies
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13
Authorisations by type of product including extensions of indications
224 products including withdrawals from register of orphan medicinal products, register medicinal products human use and expired orphan status
23
7
11
4
2
62
1
13
10
36 8
1
3
1
1
1
1
1
1
2
1
14
2
31
1
3
5
124
1 2
A B C H L -immuno
L -onco M N R S V J
Therapeutic area
Chemicals Cell therapyGenetherapy Biological, ProteinsBiological, coagulation factors Biological, monoclonal antibodiesBiological, oligonucleotides
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14
Chart includes 224 products:117 active initial authorisations; 21 active extensions of indication23 withdrawals from the register of orphan medicinal products (including 9 ext. of indication)7 withdrawals from register medicinal products human use/ orphan status expired1 revoked from register medicinal products human use/ orphan status expired51 removals of initial MAA from register after expire of the market exclusivity period4 removals of extensions of indication
190 initial orphan marketing authorisations and 34 extension of indication granted to date
Number of conditions: 133
A - Alimentary tract and metabolism
17%
B - Haematology9%
C - Cardiovascular system5%
H - Systemic hormonal3%
L - Immunology3%L - Antineoplastic
40%
M - Musculo-skeletal system
1%
N - Nervous system9%
R - Respiratory system4%
S - Sensory organs2%
V -Various3%
J - Anti-infectives for systemic use
4%
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Prevalence for orphan marketing authorisationsperiod 2000-2020
< 145%
1-225%
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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