Paediatric development update and initiatives

At the first of a number of regular meetings between regulators and industry representatives to discuss areas of product development support, early discussions and interactions to shape paediatric development featured highly.

EMA to redesign process for very early dialogue for paediatric development plans

A pilot launched in June 2015 to encourage very early discussion on paediatric drug development between the European Medicines Agency (EMA) has been less than successful; only 9 of 36 (25%) appropriate requests for this type of meeting were made.

The intended scope of an early interaction meeting was to establish the appropriate paediatric condition and target population for the product and encourage early development of a paediatric strategy that would be, wherever feasible, integrated into a product’s overall development plan.

Most of the meeting requests received by EMA during the pilot related to products whose development was already at an advanced stage, for which a discussion during a paediatric investigation plan (PIP) or scientific advice procedure was more appropriate.

Industry requested more clarity about the optimal timing of this early interaction and how it fits in with other interactions such as scientific advice and PRIME.  The EMA is to redesign the process ensuring that it will paediatric development to be incorporated into overall development programmes.

Interacting with the FDA and EMA over paediatric development programmes

Industry representatives made a strong case for global paediatric development saying it would deliver optimal and streamlined product development for the benefit of children.  It was proposed that inclusion of sponsors in the existing dialogue between the US Food and Drugs Administration (FDA) and EMA would be beneficial, particularly for programmes with identified differences on opposite sides of the Atlantic.

Four ways to aid global paediatric development programmes were presented:

  • Parallel EMA/FDA scientific advice including paediatric experts
  • Joint pre-submission meeting with scientific discussion
  • Enhanced common commentary during PIP/PSP process
  • Mutual reliance on paediatric plan assessment.

The EMA has recently introduced a number of measures to support global paediatric product development, such as:

  • Cross-attendance at Paediatric Committee (PDCO)/ Paediatric Medicines Office (PME) and Pediatric Review Committee (PeRC) meetings
  • Joint EMA/FDA early paediatric interactions within paediatric cluster, issuing a common commentary

In the future we will see the first joint EMA, FDA and Health Canada paediatric development workshop (on paediatric pulmonary arterial hypertension).

Ways to optimise the discussion on paediatric medicines development

A number of initiatives were discussed, the first of which was a proposal from industry to identify paediatric needs with a multi-stakeholder approach as one potential basis for agreeing paediatric investigation plans (PIPs).  Such an initiative needs to be part of a holistic approach; indeed, there have been first discussions at the PDCO to explore further methodologies for defining paediatric needs.  With this point in mind, it was recognised to include the experience with the current inventory of paediatric needs as well as with ongoing multi-stakeholder activities such as ‘Accelerate’ in paediatric oncology and the ‘International Neonatal Consortium’ in neonatology.

The EMA presented further initiatives to increase scientific dialogue.  The PDCO is increasing collaboration with other committees to support the medicine’s lifecycle, from early development to marketing authorisation and beyond.

PDCO members are routinely involved in scientific advice procedures relevant to paediatric development. In 2016 there were 142 paediatric scientific advice procedures and PDCO members were involved in all but four of them.

Recently, the agency has begun exploring ways for the PDCO to increase collaboration, dialogue and discussion with the CHMP on topics of common interest based on ongoing procedures at either the PDCO or the CHMP.

To read about the other areas of discussion, please click here.