Orphan Medicines Figures. 2000- 2016. Orphan Medicines - Product Development Scientific Support. Page 2. 1. Orphan Appli
Orphan Medicines Figures 2000- 2016
Orphan Medicines - Product Development Scientific Support
An agency of the European Union
Orphan Applications Figures 2000-2016 2016
Total
1151
329
2714
500
759
220
1827
8
6
7
3
24
EC Designations
343
485
768
209
1805
Withdrawals during
150
144
313
77
684
2000
2006
2011
2005
2010
2015
Applications submitted
548
686
Positive COMP Opinions
348
Negative COMP Opinions
assessment 1
Orphan Applications Figures 2000-2016 350 300 250 200 150 100 50 0 2001
2002
submitted
2
2003
2004
2005
positive opinions
2006
2007
2008
negative opinions
2009
2010
2011
2012
2013
withdrawals during assessment
2014
2015
2016
EC Designations
329 Applications Submitted for Orphan Designation (2016)
147, 45%
182, 55%
SME
3
Non-SME
New Orphan Designated Conditions 2016
18%
2014
18%
2012
28%
18%
2015 2013
25%
2011
14%
2009
16%
2007
21%
2005
39%
2003
44%
2001
78%
190
136 107
26%
2010
23%
2008 2006
33%
2004
42%
2002
51%
0
106
73 80
49 50
73
55 64
187 148
128
98 88
100
NEW CONDITIONS (487) - 27% 4
209
150
TOTAL DESIGNATIONS (1805)
200
250
Distribution of Opinions by Therapeutic Area Period 2000 – 2016 / Total opinions 1827
ATC Codes
2%
A-alimentary tract & metabolism
6% 12%
C - cardiovascular system
G - genito urinary tract
11% 8%
B - blood & blood forming organs
D - dermatology
5%
3%
0% 3%
7% 5%
H - systemic hormonal preparations J & P - antiinfectives & antiparasitic L - antineoplastic agents L - immunomodulating agents M & N - musculoskeletal & nervous system R - respiratory system S - sensory organs V - various
5
2%
36%
Distribution of Designations Adult/Paediatric Use Period 2000 – 2016/ Total Designations 1805
Adult 34%
Both 53%
Paediatric 13%
6
Distribution of Designations Adult/Paediatric Use Period 2000 – 2016 / Total Designations 1805 3 24
17 33
43
43
34
24
27 54
46
41
42
47
49
34 55
44 9 30
2 2000
10 2001
17
22
4
5
8
2002
2003
2004
39
76
61
70
51
113
155
49 62
6 2005
Medical conditions affecting children only Medical conditions affecting adults only
7
43
33
28
113
13 3 2006
2007
6 2008
3 2009
13
15
2010
2011
2012
19
25
22
20
2013
2014
2015
2016
Medical conditions affecting both children and adults
Prevalence for Designated Orphan Conditions Period 2000 – 2016 / Total designations 1805 more than 3 in 10,000 11%
between 1 and 3 in 10,000 49%
8
less than 1 in 10,000 40%
Authorised Orphan Products 25
21
20
16
15
14
13
12
11
10
9
5 4
3
0
0 0 2000
3 2001
4 2002
6
5
5 2003
6 2004
4 2005
5
4
4 9 2006
Orphan products
9
8
7
13 2007
6 2008
9 2009
4 2010
5 2011
10 2012
Orphan indications authorised
7 2013
15
14
14
2014
2015
2016
Authorisations by type of product excluding extensions of indications 1
1
1
Number of products
5
1 1
3
7
5 10
1
1
1 1
1 2
3
8
5
1
B
1 C
Therapeutic area A-alimentary tract & metabolism
H
Chemical
L immuno
Protein
L onco
M
Cell therapy
B - blood & blood forming organs
N
R
Genetherapy
S
V
Biological
J
Antibody
C - cardiovascular system
H - systemic hormonal preparations
L - antineoplastic agents
L - immunomodulating agents
M & N - musculoskeletal & nervous system
R - respiratory system
S – Sensory Organs
V – various
J & P - antiinfectives & antiparasitic
95 authorisations including 4 withdrawals from the register of medicinal products human use 10
4
27
2 A
2
Authorisations by type of product including extensions of indications 1
Number of products
12
2
1 2
3 1
1
1
9 1
1
2
1 1
9
10
5
1
4
50 14
6
A
B
Chemical
3
C
Protein
H
3
L immuno
2
L onco
Cell therapy
M
Biological
N
R
S
Genetherapy
V
Antibody
Therapeutic area A-alimentary tract & metabolism
B - blood & blood forming organs
C - cardiovascular system
H - systemic hormonal preparations
L - antineoplastic agents
L - immunomodulating agents
M & N - musculoskeletal & nervous system
R - respiratory system
S – Sensory Organs
V – various
J & P - antiinfectives & antiparasitic
146 products including withdrawals from register of orphan medicinal products, register medicinal products human use and expired orphan status 11
J
Status of Orphan Marketing Authorisation Applications: 128 granted
Adopted opinion by CHMP awaiting EC decision: 1 On-going applications in review process: 36 Extensions of indication in review process: 4 Expired – end of market exclusivity: 26 Negative outcomes for orphan MAA
12
55 withdrawn during evaluation, including 2 extensions 4 withdrawn register human medicinal products 13 withdrawn register orphan medicinal products after authorisation, including 6 extensions 23 withdrawn register orphan medicinal products before authorisation, including 2 extension 14 refusals
128 Orphan Marketing Authorisations granted to date*
A Alimentary tract and metabolism
M 1%
R 3%
N 7%
S 1%
V 3%
J 3%
A 19%
B Haematology
B 8%
C Cardiovascular system H Systemic hormonal J Anti-infectives for systemic use
C 6%
L Immunology H 3%
L Antineoplastic M Musculo-skeletal system N Nervous system
L onco 42% L immuno 4%
R Respiratory system S Sensory organs V Various
A
B
C
H
L immuno
L onco
M
N
R
S
Number of conditions: 101
13
V
J
* Including: 12 authorised extensions of indication 13 withdrawals from the register of orphan medicinal products (including 6 ext. of indication) 4 withdrawals from register medicinal products human use 26 removals from register after expire of the market exclusivity period
Prevalence for Orphan Marketing Authorisations period 2000-2016 Prevalence per 10,000 All orphan products
Including products later withdrawn by the sponsor and extensions of indication
3-5 14%
2-3 13%
less than 1 48%
1-2 25%
less than 1
14
1-2
2-3
3-5
Thank you for your attention Further information
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