Academic Year 2006-2007
Fifth European Course
Evaluation of Medicinal Products in Children
Module on drug evaluation in children
ORGANIZING COMMITTEE
- Chairperson :- Gerard Pons (Paris V University, France)
- Coordination : - Agnes Saint-Raymond (EMEA, London, UK)
- Jean-Paul Langhendries (St Vincent Hospital, Liege, Belgium)
- Behrouz Kassaï (Eudipharm, Lyon, France)
- Jean-Marc Husson (Eudipharm, Paris, France)
- Organizing Committee members :
-John van den Anker (Sophia Children’s Hosp., Rotterdam, NL)
-Philippe Augere (Pierre Fabre, Castres, France)
-Elisabeth Autret-Leca (ToursUniversity, France)
-Maurizio Bonati (Mario Negri Institute, Milan, Italy)
-Daniel Brasseur (EMEA, London, UK)
-Imti Choonara (NottinghamUniversity, UK)
-Soizic Courcier-Duplantier (BMS, Paris, France)
-Tibor Ertl (Aok, Pecs, Hungary)
-Rafaël Gorodisher (Beer Sheva, ESDP, Israël)
-Kalle Hoppu (Helsinki University, Finland)
-Evelyne Jacqz-Aigrain (Paris VIUniversity, France)
-Betty Kalikstad (Rikshospitalet, Oslo, Norway)
-Ronald Kurz (CESP, Graz, Austria)
-Catherine Lassale (LEEM, Paris, France)
-Murray Lumpkin (FDA, USA)
-Bart van Overmeire (University Hospital, Antwerpen, Belgium)
-Laurent Perret (Servier, Paris, France)
-Yannick Pletan (Pfizer, Paris, France)
-Alain Puech (Sanofi-Aventis, Paris, France)
-Anders Rane (Karolinska Institute, Stockholm, Sweden )
-Hannsjörg Seyberth (Philipps Universität, Marburg, Germany)
-Josep Torrent-Farnell (Autonomous University Barcelona, EMEA, London, UK)
-
Year 2006-2007
OBJECTIVES
M 1 /Specific Aspects of Paediatric Pharmacology
/ (6h30)-Understand the differences between adults and children : growth, development and maturation of the child (1h30)
-Drug kinetics and effects (PK/PD relationship in children) (1h00)
-Drug evaluation in the main diseases unique to children (1h00)
-Pharmacogenomics during development (1h00)
-Explain the US legislation for paediatric drug evaluation (1h00)
-Understand why there are side effects occuring specifically in children (1h00).
M2 2 /
Specific Issues related to Clinical Trials and Drug Use in Children
/ (8h00)-Define and be aware of the extent of unlicenced and off-label use of medicinal products in children (1h00)
-Identify the needs for drug formulations adapted to children (1h00)
-Understand the impact of compliance and medication errors/misuse in children (1h00)
-Manage the specific aspects of pharmacovigilance in children (1h00)
-Understand the specific ethical issues in children including the use of placebo and obtention of consent form (1h00)
-Describe the directive on GCP and clinical trials. Determine the criteria Medicine Agencies are applying to authorize new drugs in children. Be aware of the EU existing legislative incentives (1h30)
-Explain and implement the methodological and technical specifications of clinical trials in children including placebo effect, choice and assessment of good endpoints (1h30).
-Understand the relevance of bridging PK-PD studies in children (1h00)
Manage the specific aspects of pharmacovigilance in children.(2h00)
M 3 /
Specific Aspects related to Drug Development
and Postmarketing Surveillance in Children
/ (8h00)-Understand the Common Technical Document (1h00)
-How to develop orphan drugs : academic & EMEA viewpoints (1h00)
-Understand the regulatory prerequisites specific to children (1h00)
-Understand the Paediatric Investigation Plan. Why initiating drug development in children compared to same process in adults at different phases of drug. The EMEA point of view (1h00).
-How to investigate drug in children : Ethical Committee point of view (1h00)
-Explain the prerequisites for investigators/centres (1h00)
-Identify the process used to develop a paediatric drug in the Pharmaceutical Industry (1h00)
-- Learn from the experience of applications for Marketing Authorization for medicinal products in children at EMEA (1h00)Understand the specific requirements for investigator/center to qualify for drug evaluation in children. (1h00)
M 4 / Pre/Perinatal Drug Evaluation and Use / (7h45)
-Explain drug evaluation by Regulatory Authorities and specific issues linked to their use during pregnancy : viewpoint of the toxicologist (1h00)
-Writing product information/labeling, questioning clinical trials on medicinal products possible during pregnancy (1h00)
-Understand the principles and identify the methodological issues on the evaluation of placental drug transfer (1h00)
-Explain the potential impact of drugs in the treatment of pregnant women : assess the risk of drug exposure at different stages of pregnancy. Evaluate the consequences for drug use in pregnant women (1h00)
-Analyze specific cases of drug use during pregnancy (1h30)
-Understand the principles and identify the methodological issues on the evaluation of drug transfer into breast milk. Evaluate the consequences in lactating mothers (2h001h00)
-Review the different clinical situations related to fetal drug therapy. Elaborate on the methodological issues on drug evaluation in these situations (1h15)
M 5 / Drug Evaluation in Various Specific Therapeutic Area in Children. State of the Art. Protocol Design (workshop) to be continued … / (7h30)
-Review the state of the art on drug evaluation in various specific therapeutic areas in children through evidence-based medicine (1h30)
-How to design a specific protocol & CRF in a specific therapeutic area in children (3h30)
-How to write a protocol and the CRF (2h30).
M 6 / Drug Evaluation in Various Specific Therapeutic Area in Children. State of the Art. Protocol Design (workshop)… continued / (7h30)
-How to write and present a protocol and the CRF (3h30)
-Describe specific aspects of the evaluation of drugs in epilepsia in children/adults (1h30)
-Describe specific aspects the evaluation of drugs for pain in children/adults (1h30)
-Learn from the experience of a Paediatric Network in private practice (1h00)
M 7 / Drug evaluation in a specific therapeutic area in Children. State of the Art / (4h30)
-Describe specific aspects of the evaluation of drugs in cancer diseases in children (1h30)
-Describe specific aspects of clinical practice related to liver diseases when using drugs in children (1h30)
-Learn from the experience of a hospital paediatric network regarding drug evaluation (1h30)
Year 2006-2007
Modules on evaluation of medicinal products in children
TIME-SCHEDULE (2 periods)
-M1 Thursday 22 February 2007
-M2 Friday23 February 2007
-M3 Saturday24 February 2007
-M4 Wednesday14 March 2007
-M5 Thursday15 March 2007
-M6 Friday16 March 2007
-M7 Saturday17 March 2007
VALIDATION
-5 modulesrequired : M1, M2, M3, M4, M5 or M6
VENUE
University Rene Descartes Paris V
Academic Year 2006-2007
Fifth European Course
Evaluation of Medicinal Products in Children
Preliminary PROGRAMME
Co-ordination : Gerard Pons, Agnes Saint-Raymond, jean-Marc HUSSON, Behrouz Kassaï, Jean-Paul LanghendrieS
DAY 1 M 0 Thursday 22 February 2007
8h00 - 8h30 : Registration / Welcome to participants
8h30 - 9h00 :Introduction to the course
-Paris V University (Rene Descartes Medical School)
-European Diploma in Pharmaceutical Medicine
-ENDIC paediatricians and pharmacologists (European Society for Developmental, Perinatal and Paediatric Pharmacology)
DAY 1 M 1 (6h30) Thursday 22 February 2007
Specific Aspects of Paediatric Pharmacology
1) 9h00 - 10h30 : Jean-Paul Langhendries, St Vincent Hospital, Liege and UCL, Belgium (1h30).
-Differences between adults and children : growth, development and maturation of the child.
-Impact of demographic data, prevalence of diseases and public health in children.
2) 10h30 - 11h30 :Anders Rane, Huddinge University, Sweden (1h00)
-Pharmacokinetics and pharmacodynamics (PK/PD) changes in children during maturation and diseases
11h30 - 12h00 Break
3) 12h00 - 13h00 : Hannsjörg Seyberth, Marburg University, Germany (1h00)
-Main diseases unique to children requiring a specific drug evaluation.
13h00 - 14h00 Lunch
4) 14h00 - 15h00 :Evelyne Jacqz-Aigrain, Robert Debre Hospital, Paris, France (1h00)
-Prospects of pharmacogenomics in paediatric pharmacology.
5) 15h00 - 16h00 : Dianne Murphy, FDA, Washington, USA (1h00)
-Legislation on paediatric drug evaluationin USA
16h00 - 16h30 Break
6) 16h30 - 17h30 : Gerard Pons, St Vincent de Paul Hospital, Paris V University, France (1h00)
-Extrapolability to children of side effects in adults.
-Potential long term side effects of drugs related to exposure during growth and maturation.
DAY 2 M 2 (8h00)Friday 23 February 2007
Specific Issues related to Clinical Trials and Drug Use in Children
1) 8h30 - 9h30 : Imti Choonara, Nottingham University, Derby, UK (1h00)
-Extent of unlicenced and off-label use of medicinal products in children.
2) 9h30 - 10h30 : Tony Nunn, Royal Liverpool Children’s NHS Trust, Liverpool, UK (1h00)
-The needs for pharmaceutical forms of medicinal products adapted to children.
10h30 - 11h00 Break
3) 11h00 - 12h00 : Maurizio Bonati, Mario Negri Institute, Milan, Italy (1h00)
Impact of compliance and medication errors/misuse in children, the role of proper information to children and parents.
4) 12h00 - 13h00 : Stephen Evans, London School of Hygiene & Tropical Medicine, UK (1h00)
-Manage the specific aspects of pharmacovigilance in children.
13h00 - 14h00 Lunch
5) 14h00 - 15h00 : Peter Sauer, Groningen University Medical Centre, The Netherlands (1h00)
-Specific ethical issues concerning clinical trials in children including the use of placebo and obtention of consent form.
-Main cultural differences within Europe.
6) 15h00 - 16h30 : Agnes Saint-Raymond, EMEA, London, UK (1h30)
-Directive on GCP and clinical trials (basic and appended texts) : specific issues concerning clinical research in children.
-ICH E11 note for guidance.
-EU legislative incentives.
16h30 - 17h00 Break
7) 17h00 - 18h00 : Gerard Pons, St Vincent de Paul Hospital, Paris V University, France(1h00)
-Explain and implement the methodological and technical specifications of clinical trials in children, including placebo effect, choice and assessment of good endpoints.
8) 18h00 - 18h30 : Behrouz Kassaï, Laënnec University, Lyon, France (30mn)
-Clinical cases: meta-analysis in paediatrics.
DAY 3 M 3 (8h00)Saturday 24 February 2007
Specific Aspects related to Drug Development and Postmarketing Surveillance in Children
1)8h30 - 9h30 : Jean-Marc Husson, Eudipharm, Paris, France (1h00)
-The Common Technical Document on the basis of a practical example.
2) 9h30 - 10h30 :Kerstin Westermark, EMEA, London, UK(1h00)
-Rare diseases, orphan drug and how to develop an orphan drug : academic and EMEA/COMP viewpoints.
10h30 - 11h00 Break
3) 11h00 - 12h00 :Beatriz Silva-Lima, Lisbon University, Portugal & EMEA, CPMP/Safety WP, UK (1h00)
Required preclinical studies for the marketing authorization of a new medicinal product to be used in children (including juvenile animal models) : the authorities viewpoint.
Preclinical studies for a new drug application in children : industry strategy
4) 12h00 - 13h00 :Nathalie Seigneuret, EMEA, London, UK (1h00)
Paediatric Investigation Plan
Reasons to initiate drug development in children at different phases of drug development in adults : the regulatory (EMEA/CPMP) viewpoint (scientific advice...)
13h00 - 14h00 Lunch
5) 14h00 - 15h00 : Jean-Louis Bernard, Salvador Hospital, Marseille, France (1h00)
Problems related to drug investigation in children faced by an Ethical Committee
6) 15h00 - 16h00 : John van den Anker,Rotterdam University, NL/George Washington University, USA (1h00)
Specific requirements for investigators/centres to qualify for drug evaluation in children.
16h00 - 16h30 Break
7) 16h30 - 17h30 :Khazal Paradis, Genzyme Europe, Naarden, The Netherlands (1h00)
Rationale, barriers and opportunities for developing a paediatric medicine : a research based pharmaceutical industry approach.
8) 17h30 - 18h30 : Daniel Brasseur, EMEA/CPMP, London, U.K.(1h00)
Experience of applications for Marketing Authorization for medicinal products in children at EMEA
DAY 4 M 4 (7h45)Wednesday 14 March 2007
Pre/Perinatal Drug Evaluation and Use
1) 8h30 - 9h30 : Per Spindler, Copenhagen University, Denmark (1h00)
Drug evaluation by Regulatory Authorities and specific issues linked to their use during pregnancy : viewpoint of the toxicologist.
2) 9h30 - 10h30 : Sandra Kweder, FDA, Rockville, USA(1h00)
-Drug evaluation by Regulatory Authorities and specific issues linked to their use during pregnancy : product information/labeling.
-Questioning clinical trials on medicinal products possible during pregnancy
10h30 - 11h00 Break
3) 11h00 - 12h00 : Donald Mattison, National Institute of Child Health and Human Development, Rockville, MD, USA (1h00)
-Principles and methodological issues on the evaluation of placental drug transfer.
4) 12h00 - 13h00 : Elisabeth Elefant, Trousseau Hospital, Paris, France(1h00)
-Drug therapy in pregnant women.
-Risk of drug exposure at different stages of pregnancy : consequences for drug use in pregnant women.
13h00 - 14h00 Lunch
5) 14h00 - 15h30 : Moderator Donald Mattison, NICHD, Rockville, MD, USA (1h30)
Panel discussion with : Elisabeth Elefant, Donald Mattison,Sandra Kweder, Per Spindler, Gerard Pons,
-Case studies : 3 cases / 6 groups
15h30 - 16h00 Break
6) 16h00 - 17h00 :Gerard Pons, St Vincent de Paul Hospital, Paris V University, France
-Principles and methodological issues on the evaluation of drug transfer into breast milk. Consequences for breast fed children.(30 mn)
-Case studies(30 min)
7) 17h00 - 18h15 : Michael D. Reed, Rainbow Babies & Children’s Hospital, Cleveland, U.S.A.
-Different clinical situations related to fetal drug therapy. Methodological issues on drug evaluation in these situations and available evidence-based data. (45 min)
-Case study(30 min)
DAY 5 M 5 (7h30)Thursday 15 March 2007
Drug Evaluation in Various Specific Therapeutic Areas in Children.
State of the art. Protocol Design (workshop)
1) 8h30 - 10h00 : Bart van Overmeire, Antwerpen University Hospital, Belgium (1h30)
-Specific aspects of drug evaluation in ductus arteriosus in neonates
10h00 - 10h30 Break
2) 10h30 - 12h30 :Gary Fowler, Pfizer Ltd., Sandwich, U.K. (2h00)
Practical training on the design of one specific protocol with its CRF in a therapeutic area : growth hormone in GH-deficient children. (lecture)
-Position of the specific problem : state of the art through evidence based medicine on drug evaluation in GH-deficient children.
-Identification of all ethical, methodological, regulatory issues relevant to the design of the study protocol/CRF.
12h30 - 13h30 Lunch
3) 13h30 - 15h00 : Gary Fowler, Pfizer Ltd., Sandwich, U.K. (1h30)
-Practical training on the design of one specific protocol with its CRF, on growth hormone in GH-deficient children. (instructions)
4) 15h00 - 16h30 : Co-ordination by Gary Fowler,Gerard Pons and Jean-Marc Husson (1h30)
-Drafting the protocol and the CRF by students in 3 working groups
16h30 - 17h00 Break
5) 17h00 - 18h00 : Co-ordination by Gary Fowler,Gerard Pons and Jean-Marc Husson (1h00)
-Drafting the protocol and the CRF by students in 3 working groups (continued...)
DAY 6 M 6 (7h30)Friday 16 March 2007
Drug Evaluation in Various Specific Therapeutic Areas. State of the art.
Protocol Design (workshop continued...)
1) 8h30 - 10h30 : Co-ordination by Gary Fowler,Gerard Pons and Jean-Marc Husson (2h)
-Drafting the protocol and the CRF by students in 3 working groups (continued...end)
10h30 - 11h00 Break
2) 11h00 - 12h30 : Reports by the 3 different student working groups (1h30).
12h30 - 13h30 Lunch
DAY 6 M 7 Friday 16 March 2007
Drug Evaluation in a Specific Therapeutic Area in Children. State of the art.
3) 13h30 - 15h00 :Catherine Chiron, Necker Hospital, Paris, France (1h30 mn)
-Specific aspects of the evaluation of epilepsia in children, differences with adults.
4) 15h00 - 16h30 : Chantal Wood, Robert Debre Hospital, Paris, France(1h30)
-Specific aspects of the evaluation of pain in children, differences with adults.
5) 16h30 - 17h30 : Robert Cohen, ACTIV, St Maur des Fossés, France (1h00)
-Lessons from the experience of a French paediatric network in private practice on the basis of an observational study.
DAY 7 M 7 (4h45)Saturday 17 March 2007
Drug Evaluation in a Specific Therapeutic Area in Children. State of the art.
1) 8h30 - 10h00 : Gilles Vassal, Gustave Roussy Institute, Villejuif, France (1h30)
- Specific aspects of the evaluation of drugs in cancer diseases in children.
2) 10h00 - 11h30 : Etienne Sokal, U.C.L., Brussels, Belgium (1h30)
-Drugs and liver : examples significant to clinical practice.
11h30 - 12h00 Break
3) 12h00 - 13h30 :Fred Zepp, Johannes Gutenberg University, Mainz, Germany (1h30)
-Lessons from the experience of the German Paediatric Network (PaedNet), on the basis of one specific clinical study.
4) 13h30 - 13h45 : Conclusions on the meeting : Gerard Pons, Jean-Marc Husson (15mn).
Evaluation of Medicinal Products in Children
Paris, 22-23-24 February 2007 and 14-15-16-17 March 2007
At Paris V University Rene Descartes
Registration form
Please complete and return this form as soon as possible to the Organizing Secretariat:
Nadine PONS – Pharmacologie Clinique – Hôpital St Vincent de Paul
82, av. Denfert Rochereau – 75674 Paris Cx 14 France
Tel: +33.1.40.48.82.22 – Fax: +33.1.40.48.83.28 – E-mail:
Please use block letters or type
Please register me to attend the Seminar on “Evaluation of Medicinal Products in Children”
Mr Mrs Dr Pr
Family name :...... …………..First Name:……………...... ……....
Organization/Company : ...... …………………………...... …………………......
Position:...... …………...... …………...... …....
Address (Street) : ...... …………..………………………..………………….………....
Postal code: ...... …….City:...... …….…...... Country:.… ………...... …….
Telephone : ...... ………...... ….Fax : .…...... …………...... …...
E-mail: ……...... …………...... …………...... ……….
PAYMENT :
Course registration fee 3000 €
Registration fee for academics and non-profit organizations 750 €
Registration fee for students, residents, assistants 300 €
➣REGISTRATION WILL BE VALID ON PAYMENT RECEIPT
by enclosed cheque to : NATURALIA & BIOLOGIA
by bank transfer to NATURALIA & BIOLOGIA Bank C.C.F. Odeon Paris (France)
Account number : 00705443530 EUR,
IBAN FR76 305 6000 7000 7054 4353 086 Code BIC CCFRFRPP
- Please mention delegate’s name
ACCOMODATION :
We are available to provide you with information to find a hotel if needed.
February : Modules 1,2,3 :
Arrival date :………..….Departure date : ……………..
March : Modules 4,5,6.7 :
Arrival date :……..…....Departure date : …...... ……..
Signature ………….………..…………………………………Date ………………………………
Print 02/11/1808:11