European Congenital Heart Disease Organisation (ECHDO) Berlin 23 – 24 March 2007 H. W. Seyberth Chairman of the Commission on Drug Safety of German Society of Pediatrics (DGKJ) Member of the Paediatric Working Party (PEG) at the EMEA Save Drugs for Children with CHD: Save Drugs for Children with CHD: New European Regulation New European Regulation
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European Congenital Heart Disease Organisation (ECHDO)
Berlin 23 – 24 March 2007
H. W. Seyberth
Chairman of the Commission on Drug Safety
of German Society of Pediatrics (DGKJ)
Member of the Paediatric Working Party (PEG) at the EMEA
Save Drugs for Children with CHD:Save Drugs for Children with CHD:
New European RegulationNew European Regulation
The labelled percentage of 41 “standard drugs” used in a neonatal and pediatric intensive care unit
(Heidelberg, 1981).
– newborns infants children100%
50%
0%
5 13 23
32%
57%
11%
H.W. Seyberth, Kinderarzt 1984
Consequences of unlicenced and off label drug Consequences of unlicenced and off label drug perscriptions:perscriptions:
No adequate pediatric formulations
No dosing regimen
No warning of possible adverse drug reactions
No information about possible drug interactions
No product liability for the drug by the sponsor
No long term surveillance
When we choose a drug for a child ...When we choose a drug for a child ...
We rely on:
- Personal experience (trial/error)?
- Advice from colleagues/mentors?
- Anecdotal reports in the literatur?
- Extrapolation from adult data?
- Small „trending“/inclusive trials?
-
-
10
5
0
3.9
6.0
1.4
3.4
ADR% In label and licensed
Of label and unlicensed
On the wards (UK) In the outpatient clinics (FR)
(Turner et al, Acta Paed. 1999) (Horen et al, Brit J Clin Pharm 2002)
AdverseAdverse Drug Reactions (ADR) of Drug Reactions (ADR) of
unlicensed and of label drug prescription in paediatricsunlicensed and of label drug prescription in paediatrics
What has happened in the past ?
Some examples !
fluid restriction
furosemide
digoxin
NSAID´s ACE- inhibitor
Adverse effects of geriatric heart failure therapy Adverse effects of geriatric heart failure therapy applied to the preterm infant with sPDA:applied to the preterm infant with sPDA:
arrhythmias with cerebral bleeding
marked volume depletion renal hypoperfusion
PG-stimulationnephrocalcinosis
intestinal perforationrenal failure
Congenital salt losing tubulopathies (SLTs)Congenital salt losing tubulopathies (SLTs)Different age at manifestation and ontogeny of targets Different age at manifestation and ontogeny of targets (Jeck et al., AJ P 2005)(Jeck et al., AJ P 2005)
dosis:0.05 mg/kg/min infusionrate until painlessness
Legislative Incentives in Europe?
Objectives of the EU-Regulation on Medicinal Products Objectives of the EU-Regulation on Medicinal Products for Paediatric Use for Paediatric Use
• To improve the health of the children of Europe, by:
- increasing high quality research into medicines for them - promoting the development and authorization of such medicines - improving the information on medicines designed for children
• While avoiding unnecessary studies in children and not delaying the authorization of medicines for adults
Key measures for patent medicinesKey measures for patent medicines
Requirement at the time of applications for new medicines for:
• Data in children as agreed by PC
or
• A waiver from requirement
or
• A deferral of the timing of the studies
Key measures for Key measures for patentedpatented medicines medicines
Rewards for studies conduced in children:
- 6-months extension of the supplementory protection certificate
(in-effect, a patent extension)
- For orphan medicines, 2-years additional market exclusivity (10+2 years)
Key measures for Key measures for offoff-patent medicines-patent medicines
The Pediatric Use Marketing Authorisation (P.U.M.A.) :
- enabeling 10-years data protection
- use of existing brand name (brand recognition)
- amended data requirements
Institutional or horizontalInstitutional or horizontal key measures of the EU-Regulation: key measures of the EU-Regulation:
• A new expert paediatric committe (PC) at the EMEA, which negotiates with the MAH the padiatric investigation plan (PIP)
• Free scientific advice from EMEA
• European network of experts
• Information tools – inventory of therapeutic needs, new product labelling requirements, database of studies
• Public funding for studies into off-patent medicines, e.g. accepted
application in the FP7
• Enhanced safety monitoring for marked products
Specific paediatric needs and priorities in Europe (particular for the neonates):
• Analgesics• Sedatives• Immunomodulators, e.g. rheuma and TPL
Child Health in FPVII (2007-2013) 2nd call due June 07 and 3rd call due September 07
Overarching topics on Child Health:
•Adopting off-patent medicines to specificneeds of paediatric populations
Studies include the assessment of PK-data, efficacy,safety and/or the development of age appropriateformulation
Funding scheme: collaborative project with a maximum of EC contribution of 3 000 000 €for each (3 year) project ( 30 Mio € are available !)
Members of the European Network Drug Investigation in Children
(ENDIC1997 in the ESDP)
Elisabeth Autret-Leca, Tours Jean-Paul Langhendries, Liege
Maurizio Bonati, Milano Gérard Pons, Paris
Imti Choonara, Derby Anders Rane, Stockholm
Rafaël Gorodischer, Beer-Sheva Hannsjörg W. Seyberth, Marburg
Kalle Hoppu, Helsiniki John N. van den Anker, Rotterdam
Evelyne Jacqz-Aigrain, Paris Bart van Overmeire, Antwerp
Thank you for your attention !
EMEA=European Medicines Agency
Milestones of the EU Paediatric regulation
• EC Round Table, EMEA - 18 December 1997
• EU Council resolution - 14 December 2000
• Public consultations - 2002 and 2004
• EU Commission proposal - 29 September 2004
• European Parliament 1. vote - 7. Sept. 2005
• Adoption by EU Council - 9 December 2005
• 2nd vote and adoption by EP - 1. June 2006
• Entry into force - 26. January 2007
Assessment of Paediatric Needs by the Paediatric Expert Group at the EMEA in the last three years( http://www.emea.eu.int/htms/human/peg/pegassessment.htm )