orphan medicines figures 2000- 2020 · classified as public by the european medicines agency an...

Classified as public by the European Medicines Agency

An agency of the European Union

Orphan Medicines Figures2000- 2020

Orphan Medicines – Scientific Evidence Generation


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


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


3

235 applications submitted for orphan designation in 2020

53, 23%

182, 77%

SME

Non-SME


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


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


6

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


7

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


8

Distribution of orphan designations adult/paediatric use

Period 2000 – 2020/ Total designations 2382

Adult30%

Paediatric12%

Both58%


9

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


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%


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


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


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


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%


15

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


16

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