AFAMS Antineoplastics and Immunomodulating Agents EO 003.01 Part 24.
2000- 2017 · 2015 . 2016 : 2017 . Total : Applications submitted . 548 : 686 . 1151 : 329 . 260 :...
Transcript of 2000- 2017 · 2015 . 2016 : 2017 . Total : Applications submitted . 548 : 686 . 1151 : 329 . 260 :...
An agency of the European Union
Orphan medicines figures 2000- 2018
Orphan Medicines - Product Development Scientific Support
1
Applications for orphan medicinal product designation
2000
2005
2006
2010
2011
2015
2016
2017 2018 Total
Applications submitted 548 686 1151 329 260 236 3210
Positive COMP
Opinions
348 500 759 220 144 163 2134
Negative COMP
Opinions
8 6 7 2 2 3 28
EC Designations 343 485 768 209 147 169 2121
Withdrawals after
submission
150 144 313 77 100 92 876
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
submitted positive opinions negative opinions withdrawals during assessment EC Designations
4
New orphan designated conditions
64 49 55
73 88
80
98
73
106
128
107
148 136
187 190
209
147
169
78% 51% 44% 42% 39% 33% 21% 23% 16% 26% 14% 28% 25% 18% 18% 18% 14% 9% 0
50
100
150
200
250
2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018
TOTAL DESIGNATIONS (2121) NEW CONDITIONS (524) - 25%
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Orphans designations based on significant benefit
50% 56%
69%
89%
70%
53%
70% 76% 77%
81%
47%
67% 66% 57%
64% 59% 59%
67% 67%
50% 44%
31%
11%
30%
47%
30% 24% 23%
19%
53%
33% 34% 43%
36% 41% 41%
33% 33%
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
Based on SB Non-SB
Total designations 2000 14 2001 64 2002 49 2003 55 2004 73 2005 88 2006 80 2007 98 2008 73 2009 106 2010 128 2011 107 2012 148 2013 136 2014 187 2015 190 2016 209 2017 147 2018 169
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Distribution of opinions on orphan designation by therapeutic area
Period 2000 – 2018 / Total opinions 2134
12%
8% 3%
2%
1%
2%
5% 34%
7%
14%
5% 5%
2%
A-alimentary tract & metabolism B - blood & blood forming organs C - cardiovascular system
D - dermatology G - genito urinary tract 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
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Distribution of orphan designations adult/paediatric use
Period 2000 – 2018/ Total designations 2121
Adult 31%
Paediatric 12%
Both 57%
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Distribution of orphan designations
adult/paediatric use Total designations 1952
2 10 4 5 8 6
13 3 6 3
13 15
62
19 25 22 20 12 12
9 30 28
17 22 39 33
49
43 76 61
51
44
70 113 113
155 114 132
3 24 17
33 43 43 34 46
24 27
54 41 42 47
49 55 34 21 25
2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018
Medical conditions affecting adults onlyMedical conditions affecting both children and adultsMedical conditions affecting children only
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Prevalence for designated orphan conditions
Period 2000 – 2018 / Total designations 2121
less than 1 in 10,000 40%
between 1 and 3 in 10,000 49%
more than 3 in 10,000 11%
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Authorised orphan medicinal products
0
3 4
5 6
4
9
13
6
9
4 5
10
7
15 14 14 14
22
11
7
12
8
16
21
15
28
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
Orphan marketing authorisations Orphan designations covered by marketing authorisations
11
Authorisations by type of product excluding extensions of indications
112 authorisations including 5 withdrawals from the register of medicinal products human use
15
1
5
2
2
20 1
7
6
3 4
5 1
3
1
1 1 1
1 7
1
1 1
3
3 13
1
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
12
Authorisations by type of product including extensions of indications
186 products including withdrawals from register of orphan medicinal products, register medicinal products human use and expired orphan status
22
6
10
3
2
56
1
10
7
3
3
5
1
3
1 1
1
1
1 13
1
3 1
2
3
4 1
17
1
1
A B C H L- immuno L-onco M N R S V J
Chemicals Cell therapyGenetherapy Biological, ProteinsBiological, coagulation factors Biological, monoclonal antibodies
13
164 initial marketing authorisations granted and 22 extensions of indication
Adopted opinions by CHMP awaiting EC decision: 1 ext of indication
On-going applications in review process: 19
Extensions of indication in review process: 3
Expired – end of market exclusivity: 40 initial MAA & 1 extension of indication
Withdrawals
• 68 during evaluation, including 2 extensions
• 5 from register human medicinal products; of these 2 from orphan register
• 18 from register orphan medicinal products after authorisation, including 6 extensions
• 36 from register orphan medicinal products before authorisation, including 7 extensions
Negative outcomes: 19 refusals, including 1 extension
Status of Orphan MAA
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Chart includes: 15 authorised extensions of indication 18 withdrawals from the register of orphan medicinal products (including 6 ext. of indication) 5 withdrawals from register medicinal products human use 40 removals of initial MAA from register after expire of the market exclusivity period & 1 ext of indication
164 initial orphan marketing authorisations and
22 extension of indication granted to date
Number of conditions: 124 Active orphan MA: 107 Active extension of indication: 15
A Alimentary tract and metabolism
B Haematology
C Cardiovascular system
H Systemic hormonal
J Anti-infectives for systemic use
L Immunology
L Antineoplastic
M Musculo-skeletal system
N Nervous system
R Respiratory system
S Sensory organs
V Various
A 19%
B 8%
C 5%
H 3%
L- immuno 3%
L-onco 41%
M 1%
N 8%
R 4%
S 2%
V 3%
J 3%
A B C H L- immuno L-onco M N R S V J
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Prevalence for orphan marketing authorisations period 2000-2018
< 1 44%
1-2 26%
2-3 14%
3-5 16%
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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