5. Special cases in marketing authorisations: Paediatric medicines

Waivers and deferrals apply to any new or in patent drug for which a MA or a MA variation is applied for (articles 7 and 8 of the Paediatric Regulation),

Waivers for specific medicines or classes of medicines:

• grant exemption to produce results from measures/studies in one or more paediatric subsets, for a given condition.
• do not prohibit performance of (paediatric) studies;
• allow rewards only if some measures have been completed, in compliance with a PIP

Can be given:

• for medicines that are likely to be ineffective or unsafe in part or all of the paediatric population;
• for medicines that do not represent a significant therapeutic benefit over existing treatments for paediatric patients
• when development of a paediatric medicine is not needed or is not appropriate, such as for diseases that only affect adults;

Deferrals:

• help avoid delaying marketing authorisation in adults, i.e. a MAA for adults is possible before completion of one or more studies/measures in the PIP;
• does NOT mean that a PIP application can be submitted late (e.g. after completion of adult development) but that the agreed measures in the PIP can be initiated and/or completed later in relation to the adult development.
• may be applicable to some or all measures in a PIP;
• are granted when adult study results are deemed necessary prior to initiating studies in children or when paediatric studies will take longer to conduct than studies in adults;
• even when studies are deferred, the PIP will include details of the paediatric studies and their timelines