Medicines for children: Guidance for applicants / Paediatric investigation plans (PIPs), waivers and modifications

Medicines for children
Guidance for applicants
Paediatric investigation plans (PIPs), waivers and modifications
The Paediatric Regulation requires, where necessary, the early submission of a development plan for medicines - the paediatric investigation plan (PIP). The normal development of a medicine requires that various studies be performed to ensure its quality, safety and efficacy. The development plan can be modified at a later stage, with increasing knowledge and if the applicant encounters such difficulties with its implementation as to render the plan unworkable or no longer appropriate.

A paediatric investigation plan is a development plan aimed at ensuring that the necessary data are obtained through studies in children, when it is safe to do so, to support the authorisation of the medicine for children. The plan should be submitted by pharmaceutical companies to the Paediatric Committee, which is responsible for agreement or refusal of the plan.

The paediatric investigation plan includes a description of the studies and of the measures to adapt the way the medicine is presented (formulation) to make its use more acceptable in children. For example, children cannot swallow big tablets, so a liquid formulation may be more appropriate.

The plan should cover the needs of all age groups of children, from birth to adolescence.

The plan also defines the timing of studies in children compared to adults. In some cases, studies will be deferred until after the studies in adults have been conducted, to ensure that research with children is done only when it is safe and ethical to do so. Even when studies are deferred, the PIP should include details on the studies and the timelines.

As some diseases do not affect children (for example Parkinson's disease), the development of medicines for these diseases should not be performed in children, and so a paediatric investigation plan will not be required by the Paediatric Committee; the requirement for a PIP will therefore be waived in these cases. A list of these "class" waivers can be found here.

On 24 September 2008, the European Commission published the guideline entitled 'Guideline on the format and content of applications for agreement or modification of a paediatric investigation plan and requests for waivers or deferrals and concerning the operation of the compliance check and on criteria for assessing significant studies' (Commission Communication 2008/C 243/01).

Relevant documents:
Guidance:

-European Commission guideline on format and content of applications for paediatric investigation plans
-Applicants are reminded that the format of the scientific document (parts B to E) for PIP/waiver applications has changed, in accordance with the new EU guideline. The document should follow the outline published in the last page of the "Electronic template for PIP applications".
-Frequently asked questions on regulatory aspects of Regulation (EC) No 1901/2006 (Paediatric Regulation) amended by Regulation (EC) No 1902/2006 (EMEA/520085/2006). Updated 2 September 2008
-Procedural advice Updated 4 January 2010
Contact details for PDCO members and alternates for sending PIP application

Templates and forms:

-Template for letter of intent to submit an application Updated 2 February 2010 Updated on Martes, 02 de Febrero de 2010
-Template for PIP applications or request for waiver (part A) Updated 29 January 2010 Updated on Viernes, 29 de Enero de 2010
-To use the template you will need version 8 or above of the free Adobe Reader on your PC, which you can download here.
-Form for non-clinical and clinical studies proposed in a PIP application Correction published 15 January 2010
-Template to change applicant name or details Published 4 January 2010
Template for the PDCO Summary Report (including internal guidance - published for information ]
-Request for modification of an agreed paediatric investigation plan Published 28 May 2009
-Template for annual report on a deferral

Compliance check request form
.Request of confirmation of the applicability of the Agency decision on class waivers Published 21 July 2008
.Deadlines* and PDCO meeting dates:

.PDCO meeting dates 2010
.PDCO meeting dates 2011
.Submission deadlines 2010-2011 for PIPs and Answers to RfM
.Submission deadlines 2010-2011 for modification of an agreed PIP
.Submission deadlines 2010-2011 for compliance check
* Deadlines for submission of applications / responses to the EMA: please note that the published date refers to the last acceptable day of receipt of electronic documents (via Eudralink or CD/DVD)

abrir aquí:
http://www.emea.europa.eu/htms/human/paediatrics/pips.htm