# Presentation: 375942

## The European Paediatric Initiative

***Agnès Saint Raymond, MD***
***Scientific Advice and Orphan Drugs***
**The European Medicines Evaluation Agency**

## The EU System

**Two European procedures for marketing authorisation  from January 1995 (Regulation 2309/93):    **
****** ****centralised (biotech and innovative medicines)**
****** ****mutual recognition (all except biotech)**
******  ****Creation of the European Agency for the Evaluation of Medicinal Products (EMEA) **
******   ****Rapid and EU-wide authorisation for innovative medicines (210 days)**
******   ****One evaluation, One authorisation, 11-13 languages!**

## The EMEA

**  ****EMEA is not an FDA for Europe!**
**Member States have pooled their sovereignty for authorisation of medicines**
**EMEA coordinates the existing scientific resources of Member States**
**An interface with all partners**
****** ****All parties linked by an IT network (EudraNet)**

## EMEA and the EU system

**Committee for Proprietary**
**Medicinal Products**
**(CPMP)**

**Committee for Veterinary**
**Medicinal Products**
**(CVMP)**

**Committee for Orphan**
**Medicinal Products**
**(COMP)**

**EMEA**
**Management Board**

**National competent authorities**
**2,500 European experts**

**EU institutions:****Commission and Parliament**

## CPMP: 

**CPMP: **
**Licensing scientific committee**

**Chairman: Dr. Daniel BRASSEUR****2 Members /15 Member States + Repres. from Norway and Iceland + European Commission**

## Working Parties

- + ***Ad-hoc***** EXPERT GROUPS:**
** ****e.g. : Paediatric Expert Group, Bioterrorism, Oncology, Anti HIV drugs**

**Biotech WP**
**Prof. J.H. Trouvin**

**Safety WP**
**Prof.B. Silva Lima**

**Quality WP**
**Dr. J.L. Robert**

**Blood and Plasma WP**
**Dr. M. Haase**

**Efficacy WP**
**Dr. B. van Zwieten Boot**

**Pharmacovigilance WP**
**Dr. A. Castot**

## The EU situation

- 20% of the EU population, ie 75 million, is aged less than 16
- 46% medicines prescribed to children either unlicensed or off-label (hosp) 1
- 67% of children receive unlicensed/off-label medicines (hosp) 1
- Up to 90% of ICU paediatric medicines are not licensed 2
*1 Conroy S et al. Br Med J. 2000;320:79-82*
*2 Chalumeau M et al. Arch Dis Child. 2000;83: 502-5*

## European Initiative

- Round table on Medicines for Children 1997
- French Memorandum in July 2000
- Council Resolution in December 2000
- Consultation Paper Feb 2002 **** April 2002
- Draft Legislation 3Q 2002
- Council and European Parliament readings
- Implementation in 2003-2004?

## PEG objectives

- Co-ordination of paediatric actions, advice on paediatric development (Guidelines) and market access
- Needs / Priorities for studies of products
- Information (web) between MS, to Health professionals and the public
- Relations with patients, industry, learned societies and other Agencies

## Paediatric Expert Group

- Experts proposed by Member States
- Composition based on expertise at difference of other groups.

## PEG composition

- Paediatric formulations: 1 pharmacist
- Preclinical requirements: 1 preclinical expert
- Methodology: 1 ‘Bayesian’ expert
- PK and pharmacology: 2
- Clinical efficacy: 8
- Neonatology, Adolescent medicine, Gal Paediatrics, Immunology, Nephrology
- Pharmacovigilance: 1 from National Agency (vaccines)
- Links with existing COMP and Working Parties: 5
- + Observers

## Ad-hoc group

***Ad-hoc***** group**
**on Paediatrics**

**MRFG**

**CPMP**

**PEG Networking**

**COMP**

**CPMP, Working Parties **
**+ *****Ad-hoc***** groups**

## Paediatric formulations

- Paediatric formulations of choice
- List of most used adult medicines
- Extemporaneous preparations (hospital pharmacists) -  Good Practice guide

## Preclinical issues

- Concept Paper on Toxicity studies in Juvenile animals
- Review of existing data
*Are we adding useful requirements or are we setting disincentives?*

## Methodology

- A problem common to Orphan drugs and Paediatrics
- Clinical trials for efficacy and small sample size
- Inventory of existing methods
- Introduction into methodology guidelines
*Meeting on 22 October 2002 at the EMEA*

## Inventory of the needs

- Existing compendia (UK, France, Sweden)
- Published information
- US studies
- French Data
- Harmonising the needs
- Setting priorities
*In view of (public) funding of studies*
*Developing network of paediatric investigators*

## Paediatric pharmacovigilance

- Long-term surveillance
- New requirements – cohort, register,
- Post authorisation studies
- Funding?

## Database of Products

- From all European sources
- According to age groups
- Extrapolation of data, or specific?
- In the future, paediatric EPARS?

**Database of Products**

## Conclusions

- In the mean time, practical actions to be taken with the PEG
- Needs and Priorities
- Information - Awareness
- Networking