Paediatric-use marketing authorisation

Paediatric-use marketing authorisation (PUMA) is a regulatory pathway established by the European Medicines Agency (EMA) aimed at improving the health of children in Europe by facilitating the development and availability of medicines for children aged 0 to 17 years. PUMA is specifically designed for medicines that are not protected by a patent or a supplementary protection certificate (SPC) and are intended exclusively for use in the paediatric population.

Overview

The concept of PUMA was introduced with the adoption of the Paediatric Regulation by the European Union in 2006. The regulation was established in response to the lack of specifically authorized paediatric medicines, which often forced healthcare professionals to use adult medicines in an off-label manner, leading to potential risks of inefficacy or adverse reactions. PUMA aims to encourage pharmaceutical companies to invest in the research and development of paediatric medicines by offering incentives such as data protection and market exclusivity.

Procedure

To obtain a PUMA, a pharmaceutical company must submit an application to the EMA that includes a comprehensive paediatric investigation plan (PIP). The PIP outlines the proposed studies to be conducted in the paediatric population, ensuring that the medicine's safety, efficacy, and pharmacokinetic properties are appropriately assessed. Upon successful completion of the PIP and demonstration of the medicine's benefit to children, the EMA can grant a PUMA, which provides 10 years of market exclusivity for the product.

Benefits

The PUMA initiative offers several benefits:

• It encourages the development of medicines tailored specifically to the paediatric population, addressing the unique therapeutic needs of children.
• It ensures that paediatric medicines undergo rigorous evaluation for safety and efficacy before their authorization.
• It provides incentives to pharmaceutical companies, promoting innovation in paediatric drug development.

Challenges

Despite its advantages, the PUMA pathway faces challenges, including:

• The financial and logistical hurdles associated with conducting clinical trials in children.
• The limited market size for paediatric-only medicines, which may deter investment from pharmaceutical companies.
• The need for ongoing surveillance and research to monitor the long-term effects of paediatric medicines.

Conclusion

The Paediatric-use marketing authorisation represents a significant step forward in improving paediatric healthcare by ensuring that children have access to safe and effective medicines. While challenges remain, the continued support and refinement of the PUMA pathway are essential for advancing paediatric drug development and addressing the unmet medical needs of children.