Orphan Medicines Figures

Transcription

1 Orphan Medicines Figures Orphan Medicines - Product Development Scientific Support An agency of the European Union

2 Applications for orphan medicinal product designation Total Applications submitted Positive COMP Opinions Negative COMP Opinions EC Designations Withdrawals during assessment

3 Applications for orphan medicinal product designation submitted positive opinions negative opinions withdrawals during assessment EC Designations 2

4 260 applications submitted for orphan designation in 207 SME 2, 4% Non-SME 48, 57%

5 New orphan designated conditions % 5% 44% 42% 9% % 2% 2% 6% 26% 4% 28% 25% 8% 8% 8% 4% TOTAL DESIGNATIONS (952) NEW CONDITIONS (508) - 26% 4

6 250 Orphans designations based on significant benefit % 56% 69% 89% 70% 5% 70% 76% 77% 8% 47% 67% 66% 57% 64% 59% 59% 67% Designations Percentage based on SB 5

7 Distribution of opinions on orphan designation by therapeutic area Period / Total opinions 97 5% 5% 2% 2% 7% % 8% 5% % % % 2% 4% 6 A-alimentary tract & metabolism C - cardiovascular system G - genito urinary tract J & P - antiinfectives & antiparasitic L - immunomodulating agents R - respiratory system V - various B - blood & blood forming organs D - dermatology H - systemic hormonal preparations L - antineoplastic agents M & N - musculoskeletal & nervous system S - sensory organs

8 Distribution of orphan designations adult/paediatric use Period / Total designations 952 Adult 2% Both 55% Paediatric % 7

9 Distribution of orphan designations adult/paediatric use Total designations 952 Number of designations Medical conditions affecting children only Medical conditions affecting both children and adults Medical conditions affecting adults only 8

10 Prevalence for designated orphan conditions Period / Total designations 952 more than in 0,000 % less than in 0,000 40% between and in 0,000 49% 9

11 Authorised orphan medicinal products Orphan marketing authorisations Orphan designations covered by marketing authorisations 0

12 Authorisations by type of product excluding extensions of indications A B C H L- immuno L-onco M N R S V J Chemical Protein Cell therapy Genetherapy Biological, coagulation factor Biological, monoclonal 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 99 authorisations including 5 withdrawals from the register of medicinal products human use

13 Authorisations by type of product including extensions of indications A B C H L- immuno L-onco M N R S V J Chemical Protein Cell therapy Biological, coagulation factor Genetherapy Biological, monoclonal 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 2 62 products including withdrawals from register of orphan medicinal products, register medicinal products human use and expired orphan status

14 Status of Orphan MAA 42 initial marketing authorisations granted and 20 extensions of indication Adopted opinions by CHMP awaiting EC decision: 5 On-going applications in review process: 28 Extensions of indication in review process: 8 Expired end of market exclusivity: 6 initial MAA & extension of indication Withdrawals 65 during evaluation, including 2 extensions of indication from register human medicinal products 2 from register human and orphan medicinal products from register orphan medicinal products after authorisation, including 6 extensions 27 from register orphan medicinal products before authorisation, including 2 extensions Negative outcomes: 4 refusals

15 42 initial orphan marketing authorisations and 20 extension of indication granted to date 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 4 M % N 7% L antineoplastic 42% R % S % V 4% Number of conditions: Active orphan MA: 94 Chart includes: Active extension of indication: authorised extensions of indication withdrawals from the register of orphan medicinal products (including 6 ext. of indication) 5 withdrawals from register medicinal products human use 6 removals of initial MAA from register after expire of the market exclusivity period & ext of indication J 2% A 9% L immunology 4% B 7% C 6% H 4%

16 Prevalence for orphan marketing authorisations period Prevalence per 0,000 all orphan products Including extensions of indication, withdrawals and products which market exclusivity has expired -5 4% 2- % < 49% -2 24% <

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