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

236 applications submitted for orphan designation in 2018

3

70, 30%

166, 70%

SME

Non-SME

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%

5

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

6

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

7

Distribution of orphan designations adult/paediatric use

Period 2000 – 2018/ Total designations 2121

Adult 31%

Paediatric 12%

Both 57%

8

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

9

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%

10

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

14

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

15

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

16

Thank you for your attention

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