The US Food and Drug Administration (FDA) in March released a guidance on the content and placement of paediatric information within the labelling of drugs and biologics products. The guideline provides recommendations to ensure that information on the use of prescription drugs in paediatric populations (whether positive, negative, or inconclusive) is consistently placed in the proper sections and subsections within labelling so that the information is clear and accessible to health care providers.
In general, paediatric use information should be discussed in the Psediatric Use subsection and included in other sections of labelling as appropriate. When studies are waived under PREA (Paediatric Research Equity Act) because evidence strongly suggests that a drug would be ineffective or unsafe in a specific paediatric age group(s), the safety concern or lack of efficacy must be described in labelling (i.e., Paediatric Use subsection and summarized in other sections as appropriate).
The Agency breaks the information sponsors should include into four scenarios:
Scenario 1: The evidence supports the safety and effectiveness of a drug for an indication in paediatric patients (either all paediatric patients or in a specific paediatric age group(s));
Scenario 2: The evidence does not support the safety and effectiveness of a drug for an indication in paediatric patients […], because results of studies conducted in that population were negative (i.e., data from studies strongly suggest that the drug would be ineffective or unsafe) or inconclusive;
Scenario 3: There is no evidence available to support safety and effectiveness of an indication in paediatric patients […], because studies have not been conducted or are ongoing in that population and/or studies in paediatric patients […] have been waived under [the Paediatric Research Equity Act] PREA;
Scenario 4: Based on available evidence, the drug is contraindicated for use in all paediatric patients, a specific paediatric age group(s), or a specific subgroup of paediatric patients.”
For each of these scenarios, the guidance lays out which sections of labelling should include paediatric information, and what specific information should be included.
The final guidance also includes an expanded section on juvenile animal data, which previously only stated that such data should be included in the Paediatric Use subsection of the labelling. However, in the final guidance FDA has changed its recommendation to emphasize that juvenile animal data should be included only “when the data suggest an adverse signal(s) that has not been previously assessed in a paediatric clinical study.”
It is notable that a gap remains in EU labelling recommendations that does foresee a specific page for paediatric use of products even if the issue has been discussed several times.
The FDA Guideline is available at this link.