WHO guideline aims to facilitate the development of “more durable” pediatric medicines
The World Health Organization (WHO) has released a draft guideline to encourage the development of better-formulated and higher-quality pediatric medicines. The guideline targets member countries and manufacturers in assessing and developing these medicines.The document focuses on specific areas of attention and is not meant to address "general regulatory principles," according to WHO. Additionally, it does not cover extemporaneous preparations and compounding, which are covered in a separate guidance.
Due to a lack of suitable formulations for pediatric patients, pharmacists, healthcare professionals, parents, and caregivers often need to modify adult medications in ways not specified in the Summary of Product Characteristics (SmPC) or product labeling, according to the document. These modifications can include breaking, crushing, or cutting tablets. WHO said the risks associated with manipulating dosage forms for children have been documented over “many decades.”
The guideline builds on previous WHO efforts to promote the development of innovative pediatric medicines and enhance their quality, safety, and efficacy. These efforts include the Global Accelerator for Pediatric Formulations (GAP-f), which was launched in 2020 to establish a sustainable mechanism to “ensure the safe and effective use of pediatric medicines” and promote “more durable” pediatric formulations within an accelerated timeline.
The document outlines a set of general principles, including administration issues, the needs of end users, and acceptability criteria. It also discusses formulation design, which encompasses quality and the Biopharmaceutical Classification System, as well as excipients and considerations for packaging and labeling. Additionally, it covers routes of administration, such as oral or enteral, rectal, parenteral, and dermal or transdermal administration.
In the formulation design section, the guideline states that medicines should ideally come in ready-to-administer formulations to minimize the need for health professionals, parents, or caregivers to adjust the dose before administering the medication. Yet, there may be situations where some adjustments cannot be avoided.
The guideline further advises avoiding developing drugs that require frequent dosing, such as more than twice daily, which may conflict with the schedules of older children.
The guideline also advises manufacturers to devise questionnaires, preference studies, or modeling methods to gauge drug acceptability. It states that “early focus on the appropriateness of the formulation for the target age group is essential since a poorly acceptable formulation might also affect the outcome” of a pediatric trial.
It also advises manufacturers to consult with organizations dealing with pediatric and neonatal patients to get accurate information on dosing. For example, groups in Canada and the United Kingdom have conducted research on teaching swallowing techniques to children in taking oral solid dosage forms to reduce the need for liquid formulations.
The guideline also urges manufacturers to avoid using coloring agents to avoid provoking allergic reactions, particularly in medicines for infants and young children.
Their use may be justified to prevent accidental dosing errors for medications produced in various strengths or to mask an “unpleasant” color associated with the active pharmaceutical ingredient.
The document was released for comment in January. Plans are to review the comments and release a working document for discussion in March 2025. WHO’s Expert Committee on Specifications for Pharmaceutical Preparations (ECSPP) is expected to adopt the guideline in September 2025.
WHO is accepting feedback on the guideline through an online platform. The deadline for submitting comments is 7 March 2025.
WHO guideline
