Interactions between CHMP and PDCO; reflections from a CHMP perspective

Tomas Salmonson

vice-chair, CHMP

5 years of the Paediatric regulation whose objectives are:

– to increase the availability of medicines intended for children

– to make information on those medicines widely available

– to stimulate high quality paediatric research.

Early concerns from CHMP

• Delay in drug development

• Timing of studies in children

• PIP vs SA

• Too "heavy" procedure (for industry and regulators)

Early concerns from CHMP

• Delay in drug development

• Timing of studies in children

• PIP vs SA

• Too "heavy" procedure (for industry and regulators)

CHMP – PDCO interactions

• PDCO-members (bridge to CHMP)

CHMP – PDCO interactions

• PDCO-members (bridge to CHMP)– has not worked - I still believe my proposal

would have been a better one

CHMP – PDCO interactions

• PDCO-members (bridge to CHMP)

• SAWP

CHMP – PDCO interactions

• PDCO-members (bridge to CHMP)

• SAWP– 250 PIPs received EMA scientific advice or protocol

assistance before or after PIP opinion– PDCO members / EMA paed staff have been

involved in 205 SAWP procedures – PDCO delegates are automatically involved in paediatric SA/PA

– an empty "PDCO-chair " at SAWP– SAWP not consulted in PIP-discussions

CHMP – PDCO interactions

• PDCO-members (bridge to CHMP)

• SAWP

• Guidelines

CHMP – PDCO interactions

• PDCO-members (bridge to CHMP)

• SAWP

• Guidelines– PDCO involvement in guidelines drafting

(e.g.: adopted this year Paediatric addendum for PAH, Clinical investigation of recombinant and human plasma-derived factor VIII products and factor IX)

CHMP – PDCO interactions

• PDCO-members (bridge to CHMP)

• SAWP

• Some examples (MAAs)

Examples

• Enbrel; RoActemra (extrapolation of efficacy)– Enbrel: to extend the lower age range in

polyarticular juvenile idiopathic arthritis (JIA) from four to two years.

Examples

• Enbrel/RoActemra (extrapolation of efficacy)

• Buccolam (PUMA)

European Medicines Agency gives first positive opinion forpaediatric-use marketing authorisation

Buccolam recommended for treatment of prolonged, acute, convulsive seizures in paediatric patients from 3 months to 18 years

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given its first positive opinion for a paediatric-use marketing authorisation (PUMA) for Buccolam(midazolam), from ViroPharma SPRL, intended for the treatment of prolonged, acute, convulsive seizures in paediatric patients from the age of 3 months to 18 years.

June 2011 (PIP approved August 2009)

Dr Agnès Saint-Raymond said:

“We welcome this first opinion for a PUMA. Since the coming into force of the Paediatric Regulation in 2007 companies have given too little attention to the incentives for paediatric-use marketing authorisations.”

Examples

• Enbrel/RoActemra (extrapolation of efficacy)

• Buccolam (PUMA)

• Schizofrenia (joint SAG, Invega)

In summary…

• Lots of PIPs

In summary…

• Lots of PIPs

• Many SA related to paediatrics

In summary…

• Lots of PIPs

• Many SA related to paediatrics

• Significant interactions between SAWP and PDCO (still room for improvement)

In summary…

• Lots of PIPs

• Many SA related to paediatrics

• Significant interactions between SAWP and PDCO (still room for improvement)

• Few direct interactions between PDCO and CHMP (big room for improvement)

In summary…

• Lots of PIPs

• Many SA related to paediatrics

• Significant interactions between SAWP and PDCO (still room for improvement)

• Few direct interactions between PDCO and CHMP (big room for improvement)

• Few applications – too early to judge??– eg: 26 applications for PIPs for PUMAs have been received

and 7 opinions have been given by the PDCO. (June 2011)

The future…… (?)

• PRAC - PASS

• CAT – conditional approvals

• CHMP – PAES, HTA

• PDCO – PIP

The future…… (?)

• PRAC - PASS

• CAT - conditional approvals

• CHMP – PAES, HTA

• PDCO – PIP

We (CHMP) need a system to initially provide an outline of a follow-up measure and later a more detailed advice (through SAWP). This may be a model that can be explored for other committees