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 [email protected] European Medicines Agency

30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555 Send a question via our website www.ema.europa.eu/contact

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European Medicines Agency 2017 Reproduction and/or distribution of this document is possible for non-commercial purposes provided that EMEA is always acknowledged as the source in each copy. Citations may be made, provided the source is always acknowledged. 15

Orphan medicines figures 2000-2016 - European Medicines Agency

Orphan Medicines Figures. 2000- 2016. Orphan Medicines - Product Development Scientific Support. Page 2. 1. Orphan Applications Figures 2000-2016. 2000.

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