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The role of EMEA The role of EMEA in in
Orphan Drug Orphan Drug DevelopmentDevelopment
Thomas Lönngren, EMEA Thomas Lönngren, EMEA Stockholm, 15 February 2005Stockholm, 15 February 2005
15 Feb 2005 – Thomas LönngrenSlide: 2
European Medicines Agency www.emea.eu.int
The European Medicines AgencyThe European Medicines Agency
• EMEA is the European Union body responsible for the evaluation and supervision of medicines in Europe.
• Its main responsibility is the protection and promotion of public and animal health.
• The EMEA works as a network, bringing together the scientific resources of the Member States.
• The Agency cooperates closely with international partners on a wide range of regulatory issues (e.g. ICH, WHO, FDA, etc)
• The Agency was created in 1995 and is headquartered in London.
15 Feb 2005 – Thomas LönngrenSlide: 3
European Medicines Agency www.emea.eu.int
Drug Therapy in Rare DiseasesDrug Therapy in Rare Diseases
Persons suffering from rare diseases have the same rights as their fellow citizens to safe and effective therapies
15 Feb 2005 – Thomas LönngrenSlide: 4
European Medicines Agency www.emea.eu.int
Orphan RegulationsOrphan Regulations
• Regulation (EC) No 141/2000 of the European Parliament and of the Council on Orphan Medicinal Products of 16 December 1999
• Commission Regulation (EC) No 847/2000 of 27 April 2000
15 Feb 2005 – Thomas LönngrenSlide: 5
European Medicines Agency www.emea.eu.int
What is an Orphan Medicinal ProductWhat is an Orphan Medicinal Product
Orphan Medicinal Products• for rare diseases (affecting less than 5 in
10,000 persons)• development costs > expected return on
investment• life-threatening or very serious
Lack of sponsors developing orphan medicinal products
15 Feb 2005 – Thomas LönngrenSlide: 6
European Medicines Agency www.emea.eu.int
What are the EU incentives ?What are the EU incentives ?
Protocol Assistancefree scientific advice
to optimise development
EU-Funded Researchgrants from Community
& Member State programmes
Fee Reductionsreduction of centralised
regulatory fees via a special fundfrom EU budgetary authority
Market Exclusivityfor 10 years after grant of
EU marketing authorisation Centralised Procedure
direct access to EMEAcentralised procedure for marketing authorisation
15 Feb 2005 – Thomas LönngrenSlide: 7
European Medicines Agency www.emea.eu.int
Application for Orphan DesignationApplication for Orphan Designation
Application should demonstrate orphan criteria have been met:
• life-threatening or debilitating nature of condition• medical plausibility• prevalence < 5 in 10,000 or unlikely to generate
sufficient return on investment• no satisfactory methods exist or medicinal
product will be of significant benefit
All claims should be substantiated by references
15 Feb 2005 – Thomas LönngrenSlide: 8
European Medicines Agency www.emea.eu.int
Committee for Orphan Medicinal Committee for Orphan Medicinal Products (COMP)Products (COMP)
EMEA Committee: 31 members + Chairman• 1 Member per Member State• 3 representatives from patients groups• 3 members proposed by the EMEA
COMP responsible for:• opinions on designation• advising on general EU policies• international co-operation
15 Feb 2005 – Thomas LönngrenSlide: 9
European Medicines Agency www.emea.eu.int
Procedure for Orphan DesignationProcedure for Orphan Designation
Evaluation Opinion Decision-Making
DesignationValidation
Day 1 Day 90 + 30 days
Pre-submission
15 Feb 2005 – Thomas LönngrenSlide: 10
European Medicines Agency www.emea.eu.int
Status of Orphan ApplicationsStatus of Orphan Applications
0
20
40
60
80
100
120
2000 2001 2002 2003 2004
submitted
positive opinions
negative opinions
withdrawals
Commissiondecisions
Up to January 2005Up to January 2005
15 Feb 2005 – Thomas LönngrenSlide: 11
European Medicines Agency www.emea.eu.int
Distribution of opinionsDistribution of opinionsimmunology
11%
oncology36%
cardiovascular and respiratory
9%
antiinfectious5%
metabolism11%
musculoskeletal and nervous system
7%
other21%
immunology oncologycardiovascular and respiratory antiinfectiousmetabolism musculoskeletal and nervous systemother
Up to December Up to December 20042004
15 Feb 2005 – Thomas LönngrenSlide: 12
European Medicines Agency www.emea.eu.int
Opinions designated based on Opinions designated based on significant benefitsignificant benefit
• Up to January 2005: 182 out of 262 opinions (69%) based on assumption of significant benefit over authorised treatments in the orphan condition
• Significant benefit to be reviewed at the time of Marketing Authorisation to maintain orphan status
15 Feb 2005 – Thomas LönngrenSlide: 13
European Medicines Agency www.emea.eu.int
Protocol AssistanceProtocol Assistance
Article 6 of Regulation (EC) No 141/2000
• Protocol Assistance = Scientific Advice for companies developing Orphan Medicinal Products
• Revised procedure adopted by the CHMP 2003• Implementation of changes from new
Pharmaceutical Regulation by end 2005
15 Feb 2005 – Thomas LönngrenSlide: 14
European Medicines Agency www.emea.eu.int
Protocol Assistance – Key FeaturesProtocol Assistance – Key Features
• Systematic pre-submission meeting with the EMEA
• Oral explanations in the majority of cases
• Additional and specific expertise to participate in SAWP
• Involvement of 2 representatives of the Committee for Orphan Medicinal Products in SAWP (Significant Benefit issues)
• Fee reduction (currently 100% = free)
15 Feb 2005 – Thomas LönngrenSlide: 15
European Medicines Agency www.emea.eu.int
Scientific Advice / Protocol AssistanceScientific Advice / Protocol AssistanceProceduresProcedures
65
13
75
22
77
32
0
50
100
2002 2003 2004
15 Feb 2005 – Thomas LönngrenSlide: 16
European Medicines Agency www.emea.eu.int
Orphan Medicinal ProductsOrphan Medicinal Products Application for Marketing Authorisation (MAA) Application for Marketing Authorisation (MAA)
At the stage of MAA:• Filing can currently be through Mutual
Recognition Procedure or Centralised Procedure• To obtain Market Exclusivity MA must be
granted by all Member States in Mutual Recognition
• In November 2005, Centralised filing obligatory• Fee reductions are granted by some MS’s and
by EMEA for centralised applications
15 Feb 2005 – Thomas LönngrenSlide: 17
European Medicines Agency www.emea.eu.int
Status of Orphan Marketing Status of Orphan Marketing Authorisation Applications Authorisation Applications
18 authorisations granted to date
• Fabrazyme for Fabry disease• Replagal for Fabry disease• Glivec for chronic myeloid leukaemia• Tracleer for pulmonary arterial hypertension• Trisenox for acute promyelocytic leukaemia• Somavert for acromegaly• Zavesca for Gaucher disease• Carbaglu for hyperammonaemia
15 Feb 2005 – Thomas LönngrenSlide: 18
European Medicines Agency www.emea.eu.int
Status of Orphan Marketing Status of Orphan Marketing Authorisation Applications cont’dAuthorisation Applications cont’d
• Aldurazyme for Mucopolysaccharidosis• 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• Photobarr for Barret’s oesophagus• Wilzin for Wilson's disease • Xagrid for Thrombocythaemia
15 Feb 2005 – Thomas LönngrenSlide: 19
European Medicines Agency www.emea.eu.int
Status of Orphan Marketing Status of Orphan Marketing Authorisation Applications cont’dAuthorisation Applications cont’d
Two CHMP Opinions in decision-making• Orfadin for Hereditary tyrosinemia type 1 • Prialt for chronic painThree extensions of indication• Glivec for Gastrointestinal Stromal Tumours• Glivec for first line use in Chronic Myeloid Leukaemia• Glivec for paediatric use in Chronic Myeloid LeukaemiaNine centralised applications in review process
Four applications filed through Mutual Recognition
15 Feb 2005 – Thomas LönngrenSlide: 20
European Medicines Agency www.emea.eu.int
Overview of “evidence” Overview of “evidence” in authorised productsin authorised products
• Data provided– in 28% of products phase III studies (double
blind, randomised, placebo controlled)– in 44% of products phase II studies
• 61% of Marketing Authorisations granted under “exceptional circumstances”
15 Feb 2005 – Thomas LönngrenSlide: 21
European Medicines Agency www.emea.eu.int
Orphan Marketing AuthorisationsOrphan Marketing Authorisations
240 designated
45 marketingapplic’ns
19authorised
830,000 patients