1 0 years of the Orphan Regulation in Europe conference - The experience up to date 03 May 2010 S. Aarum MD PhD / S.Tsigkos MD PhD An agency of the European Union
Outcom es of applications for ODD 180 � 1113 applications 160 number of applications submitted 140 120 � positive outcome in 2/3 100 of the cases, high 80 60 success rate 40 � few negative opinions 20 0 per year 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 � withdrawals may submitted positive opinions negative opinions withdrawals reapply at a later stage
W ithdraw als per therapeutic area � 269 withdrawals Cardiovascul Anti- ar and � most withdrawals within infectious Respiratory Other 5% 11% oncology (such as 29% Metabolism malignant melanoma, 4% Musculoskel Immunology pancreatic cancer) etal and 5% nervous system Oncology 13% 33%
Opinions per therapeutic area immunology other 10% 10% musculoskeletal and nervous system 12% metabolism 10% oncology antiinfectious 46% 3% cardiovascular and respiratory 9% immunology oncology cardiovascular and respiratory antiinfectious metabolism musculoskeletal and nervous system other
Prevalence of designated applications designations up to 10/2009 � More than a third affect less than 1 in 10,000 in the EU 12% less than 1 in 10,000 36% � Would attract little between 1 and 3 in interest per se without 10,000 more than 3 in 10,000 OD incentives 52%
Target populations ( age)
The story so far… � A simple framework that works � 1113 applications submitted � More than two thirds positive opinions � 724 designated orphan products
Protocol Assistance � Protocol assistance is free 2009 311 77 scientific advice 2008 264 56 2007 215 73 2006 201 58 � Yearly increase in the 2005 136 55 2004 87 22 2003 72 25 number of SA/ PA procedures 2002 71 17 2001 69 2000 � Links to success in 58 0 50 100 150 200 250 300 350 400 Scientific Advice Protocol Assistance marketing authorisation
Use of EU special contribution for OD Use of EU special contribution for orphan medicines ( 2 0 0 9 ) � Protocol assistance is one of the most utilized Post- a uthorisa tion proce dure s I nspe ctions 4 % incentives Ma rk e ting 4 % a uthorisa tions 2 9 % � Contrast to post- authorization procedures and inspections Protocol a ssista nce 6 3 %
OD Marketing Authorisations Fabrazyme for Fabry disease 2001 Replagal for Fabry disease Glivec for chronic myeloid leukaemia Tracleer for pulmonary arterial hypertension Trisenox for acute promyelocytic leukaemia 2002 Somavert for acromegaly Zavesca for Gaucher disease Carbaglu for hyperammonaemia Aldurazyme for Mucopolysaccharidosis 2003 Busilvex for haematopoietic progenitor cell transplantation Ventavis for pulmonary arterial hypertension Onsenal for Familial Adenomatous Polyposis Litak for Hairy cell leukaemia Lysodren for adrenal cortical carcinoma Pedea for Patent Ductus Arteriosus 2004 Photobarr for Barret’s oesophagus Wilzin for Wilson's disease Xagrid for Thrombocythaemia
OD Marketing Authorisations Orfadin for Hereditary tyrosinemia type 1 Prialt for chronic pain requiring intrathecal (IT) analgesia 2005 Xyrem for cataplexy in patients with narcolepsy Revatio for pulmonary arterial hypertension Naglazyme for replacement therapy in patients with mucopolysaccharidosis VI Myozyme for Glycogen Storage Disease type II (Pompe’s disease) Evoltra for acute lymphoblastic leukaemia Nexavar for advanced renal cell carcinoma Sutent for gastrointestinal stromal tumour and metastatic renall cell carcinoma Savene for anthracycline extravasation 2006 Thelin for idiopathic pulmonary arterial hypertension or pulmonary arterial hypertension Exjade for chronic iron overload due to blood transfusions Sprycel for acute lymphoblastic leukaemia and chronic myeloid leukaemia Diacomit for severe myoclonic epilepsy in infancy Elaprase for mucopolysaccharidosis type II (Hunter syndrome) Inovelon for Lennox-Gastaut syndrome Cystadane for homocystinuria
OD Marketing Authorisations Revlimid for multiple myeloma Soliris for paroxysmal nocturnal haemoglobinuria Siklos for sickle cell syndrome Atriance for acute lymphoblastic leukaemia 2007 Increlex for primary insulin-like growth factor-1 deficiency due to molecular or genetic defects Gliolan for Intra-operative photodynamic diagnosis of residual glioma Yondelis for soft tissue sarcoma Tasigna for chronic myeloid leukaemia Torisel for renal cell carcinoma Thalidomide Celgene for multiple myeloma Volibris for pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension Firazyr for angioedema 2008 Ceplene for acute myeloid leukaemia Kuvan for hyperphenylalaninaemia Mepact for osteosarcoma Vidaza for acute myeloid leukaemia and myelodysplastic syndromes
OD Marketing Authorisations Nymusa for primary apnoea in premature newborns Afinitor for renal cell carcinoma Mozobil for mobilization of progenitor cells prior to stem cell transplantation Cayston for gram negative bacterial lung infection in cystic fibrosis 2009 Arcalyst for Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) Ilaris for Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) Nplate for idiopathic thrombocytopenic purpura (ITP) Firdapse for Lambert-Eaton Myasthenic Syndrome 2010 Revolade for idiopathic thrombocytopenic purpura Tepadina for conditioning prior haematopoietic progenitor cell transplantation Arzerra for chronic lymphocytic leukemia
Distribution of Marketing authorizations per therapeutic area � 62 orphan drugs 4% 4% received marketing 13% 13 approval so far 10% 10 46% 46 � More than one third antineoplastic and 2% 2% 25% 25% immunomodulating, antineoplastic and immunomodulating agents metabolism blood musculoskeletal and nervous system followed by agents for cardiovascular others metabolic diseases
The story so far… � A Simple Framework that has produced measurable output: � 724 designated orphan products across the entire spectrum of human disease � 62 Orphan Medicinal Products have received Marketing Authorisation
Thank you
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