MHRA, EMA and FDA requirements for pediatric study
Pediatric Studies: Impact of Brexit on your Pediatric Investigational Plans (PIP)
The uncertainty of Brexit has significant implications for all aspects of product development and Pediatric Investigational Plans (PIPs) are no exception. The recently posted MHRA Guidance: Procedures for UK-PIPs in the event the UK leaves the EU without a deal provides some clarification and states that “UK aims to simplify the PIP application process for applicants by offering an expedited assessment where possible”. However, the overall impact is that there is an additional step of submission of a PIP to MHRA in addition to the submission to EMA, thus adding an additional application to an already complex and lengthy process of PIP preparation and submission.
To successfully navigate this road, pharma companies often seek assistance from a partner with a specialized mix of scientific, therapeutic, regulatory and operational expertise with the aim of ensuring their pediatric studies are successfully designed, planned and executed – with patient safety as the centerpiece of the program.
To address this expanding need, IQVIA has put together a team of experts with backgrounds in pediatric drug development, regulatory, modeling and simulation and pharmacokinetics (PK), to assist clients in the evaluation of their products or programs and recommend practical and informed strategies for pediatric drug development. Our team can evaluate client product profile(s) using a formalized methodology based on the Guidances of major regulatory agencies such as EMA and FDA, but also taking in consideration other Health Authority requirements. Our pediatric evaluation team will assess each unique scenario to determine whether the client will: a) need to develop a full Pediatric Investigational Plan; or b) potentially be eligible for a waiver or deferral. We are familiar not only with the unique challenges and principles inherent in pediatric trials but can leverage the specific therapeutic area background needed to provide customers with product specific advice targeted to their compounds. We can assist with coordination of a PIP and the Pediatric Study Plan if FDA submission is also being planned. Finally, we can also assist with PIP Modifications that may be needed as well as the final PIP Compliance Check that is required prior to the submission of the Marketing Authorisation Application.
A consultation from IQVIA can save clients significant time and expense by providing an intensive and independent pre-review based on regulatory requirements. If this review suggests a waiver is likely to be granted, IQVIA can provide a written justification in submission-ready format. If IQVIA’s review suggests a waiver is not likely to be granted, we can assist as needed in developing a Pediatric Investigational Plan/ Study Plan.
As this requirement applies to any new medicinal product that is seeking marketing authorization approval in the UK, it has significant implications for drug developers. It is important to facilitate discussion about pediatric development plans early on in the product’s development lifecycle, ideally following human PK studies.
Please contact us at: RareDisease_PediatricCOE@iqvia.com
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