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An agency of the European Union Daniel Brasseur PDCO European Medicines Agency “5 th anniversary Paediatric Regulation” PDCO Achievements.

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Presentation on theme: "An agency of the European Union Daniel Brasseur PDCO European Medicines Agency “5 th anniversary Paediatric Regulation” PDCO Achievements."— Presentation transcript:

1 An agency of the European Union Daniel Brasseur PDCO European Medicines Agency “5 th anniversary Paediatric Regulation” PDCO Achievements

2 2 2 Paediatric Regulation

3 3 Take home message Good research in children is a need & time is needed to develop good research

4 4 Research Needs in Paediatric Medicines Total Opinions (+&-) 700

5 5 Research Needs in Paediatric Medicines PIP Opinions (= need) 500 Total Opinions 700

6 6 Research Needs in Paediatric Medicines PIP Opinions (= need) 500 Total Opinions % out of 700

7 77

8 8 PDCO Achievements PDCO Achievements Plan Implementation of procedures PIP Evaluation and Outcomes Survey of the Needs Priority list & FP7 Enprema Challenges

9 99 9 Overview of PDCO Procedure ReStart Clock Stop Clock 60 DAYS Adoption of Opinion & Final Report Start Clock Decision Evaluate Changes Find Agreement Finalize Plan Publish Decision Opportunity for the Company to introduce Modifications Understand Plan Detect Problems Identify Experts Propose Changes Adoption of Opinion, or Request for Modification

10 10 Overview PDCO of Procedure ReStart Clock Stop Clock 60 DAYS Adoption of Opinion & Final Report Start Clock Decision Evaluate Changes Find Agreement Finalize Plan Publish Decision Opportunity for the Company to introduce Modifications Understand Plan Detect Problems Identify Experts Propose Changes Adoption of Opinion, or Request for Modification

11 11 Overview PDCO of Procedure ReStart Clock Stop Clock 60 DAYS Adoption of Opinion & Final Report Start Clock Decision Evaluate Changes Find Agreement Finalize Plan Publish Decision Opportunity for the Company to introduce Modifications Understand Plan Detect Problems Identify Experts Propose Changes Adoption of Opinion, or Request for Modification

12 12

13 13

14 14 PDCO Achievements PDCO Achievements Plan Implementation of procedures PIP Evaluation and Outcomes Survey of the Needs Priority list & FP7 Enprema Challenges

15 15 5 CHMP members + 1 members per Member State not yet represented + 6 members from families & HCP associations Each member has an alternate PDCO

16 16 Rapporteurships

17 17 Update EMA/ PDCO Report Request for Modification PIP Procedure First step = 7 “expert” inputs Re-StartClock StopClock Peer Reviewer’s Comments Day 27 Rapporteur’s written Contribution Day 20 Members’ written Comments Day StartClock 1 st Rapp Oral Present. at PDCO Day 30 1 st PDCO Discussion +OE? Day 60 EMA Sm Report Day 1

18 18 Update EMA/ PDCO Report Request for Modification PIP Procedure First step = 7 “expert” inputs Re-StartClock StopClock Peer Reviewer’s Comments Day 27 Rapporteur’s written Contribution Day 20 Members’ written Comments Day StartClock 1 st Rapp Oral Present. at PDCO Day 30 1 st PDCO Discussion +OE? Day 60 EMA Sm Report Day 1

19 19 PIP Procedure Second step = 9 “expert” inputs 3 rd PDCO Discussion Final Report Day 120 OE= oral explanation Rapporteur’s Comments on Modifications Day 80 PReviewer’s Comments on Modifications Day 88 Update EMA/PDCO Sm Report Day 110 Members’ Final Comments to EMA Day 105 PDCOOpinion EMEADecision Re-StartClock Company’s PIP Re- Submission Day 61 EMA Sm Report Update Day 71 2 nd Rapp Oral Present. Day & 2 nd PDCO Discussion +OE? 90. OE? Final EMA Decision Day 130

20 20

21 21 Evaluation procedure of PIPs PDCO FWG Formulation Group – monthly meeting –PDCO members (Chair: Dr Siri Wang) + external experts (hospital, academia). –Discussion on formulation aspects and reporting to the PDCO. PDCO Paediatric Committee - monthly meeting

22 22 Applications assessed by FWG Around 1000 PIPs validated PIP/waiver applications (March 2011) 60 % 15/m

23 23 Critical Points for Paediatric Formulations Route of administration Appropriate dosage forms Excipients - 50% of the PIPs- choice excipient, safety, level, side effects…… Taste and palatability Delivery devices Rate of infusion Volume to be administered Wastage

24 24 Regulatory references 2 Regulatory references 2 (Draft) Guideline on pharmaceutical development of medicines for paediatric use Collaborative work between QWP, PDCO, and external experts. Public consultation aimed very soon.

25 25

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27 27 WGroups ReStart Clock Stop Clock DAY 0 or 30 DAY 60 or 90 Start Clock Opinion Opportunity to discuss need for juvenile animal studies Opportunity to discuss need for juvenile animal studies Non-Clinical

28 28

29 29 WGroups ReStart Clock Stop Clock DAY 0 or 30 DAY 60 or 90 Start Clock Opinion Opportunity to discuss extrapolations statistics ?modelling Methodology Opportunity to discuss extrapolations statistics ?modelling

30 30 Reasonable to assume equivalence of disease Children = Adults - similar progression? - similar response to intervention? Reasonable to assume similar concentration response (C-R) in Children = Adults? PK Conduct PK studies SE Conduct S & E trials Conduct PK studies to achieve levels similar to Adults S Conduct S trials PKPD Conduct PK/PD studies to get CR for PD measurement PK Conduct PK to achieve target concentration based on CR SE Conduct S & E trials E Is there a PD measurement that can be used to predict E? No No No Yes Yes Yes Yes

31 31 “Classical Power” Overall population Affected population Sick population (patients) Available Population (volunteers) Resources Project Diagnosis Prevalence Participating Population (Cases) Protocol CT Methodological constraints in small populations

32 32 Modelling and Simulation!

33 33 Definition of Models Published Paradigm PBPK (Physiologically Based PK) PBPK-PD POP PK (Population Based PK) POP PK/PD Disease progression models and response models Kinetic (K)–PD models Toxicity/AE models

34 34

35 35 PDCO Achievements PDCO Achievements Plan Implementation of procedures PIP Evaluation and Outcomes Survey of the Needs Priority list & FP7 Enprema Challenges

36 36

37 37 60 %

38 38

39 39 How many trials have effectively started? How many children to be included? How many indications to be developed? What is the expected product attrition rate? What are finalisation delays overall? 6%6%

40 40 PDCO Achievements PDCO Achievements Plan Implementation of procedures PIP Evaluation and Outcomes Survey of the Needs Priority list & FP7 Enprema Challenges

41 An agency of the European Union Presented by: Dr. Radu Botgros Scientific Administrator, Safety and Efficacy of Medicines REPORT ON THE SURVEY OF ALL PAEDIATRIC USES OF MEDICINAL PRODUCTS IN EUROPE established according to article 42 of Regulation (EC) No 1901/2006 of the European Parliament and of the Council on medicinal products for paediatric use

42 42 Results (I) Most frequent medicines used off-label/ unauthorised : –antiarrhythmics, –antihypertensives (rennin-angiotensin inhibitors and beta- blockers), –proton pump inhibitors and H2-receptor antagonists, –antiasthmatics, and –antidepressants (mainly selective serotonin reuptake inhibitors, serotonin-norepinephrine reuptake inhibitors and tricyclic antidepressants). High rate of off-label use of oral contraceptives in ados –mainly in Scandinavia.

43 43 Results (II) Extensive off-label use of ABs in very young children. –macrolides –betalactamines plus betalactamase inhibitors –carbapenems Corticosteroids used off-label in the systemic treatment of very young children. –Some for systemic use (e.g. dexamethasone) are not even authorised in some countries (Norway)

44 44 Results (III) Analysis of the pharmaceutical forms both oral and parenteral formulations are being used unauthorised or off-label, common reason: lack of appropriate dosages and strengths for the treated age groups. off-label use of –multivitamins –many antiasthmatics.

45 45 PDCO Achievements PDCO Achievements Plan Implementation of procedures PIP Evaluation and Outcomes Survey of the Needs Priority list & FP7 Enprema Challenges

46 46

47 47 PDCO evaluation List DG Research evaluation PIP DG Research EMA Funding Applicant’s project

48 48 PDCO evaluation List DG Research evaluation PIP DG Research EMA Funding Applicant’s project

49 49 PDCO PIP List DG ResearchEMA Applicant’s draft PIP

50 50 PDCO PIP List DG ResearchEMEA Applicant’s project + agreed PIP Funding

51 51 PDCO Achievements PDCO Achievements Plan Implementation of procedures PIP Evaluation and Outcomes Survey of the Needs Priority list & FP7 Enprema Challenges

52 52 Researcher HCP Industry Regulators Academia

53 53 PDCO Achievements PDCO Achievements Plan Implementation of procedures PIP Evaluation and Outcomes Survey of the Needs Priority list & FP7 Enprema Challenges

54 54

55 55

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57 57 Scientific Advice (paediatric only) 57 SA +PA Paed %

58 58 PDCO Achievements PDCO Achievements Conclusion Change of mindset with Industry 475 PIPs agreed Start of Network of Networks Limited view of immediate impact… Better overview of Paediatric needs Trials of off-label drugs (FP7) Reflection ongoing to improve/ facilitate work

59 59 Take home message Good research in children is a need & time is needed to develop good research

60 60


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