PUMA: paediatric-use marketing authorisation
A paediatric-use marketing authorisation (PUMA) covers a medicine developed only for children from an off-patent substance, with its own protection.
The PUMA was created by Article 30 of Regulation (EC) No 1901/2006, the Paediatric Regulation. It is a marketing authorisation for a product that is already authorised but no longer protected by a patent or supplementary protection certificate, developed exclusively for use in the paediatric population or subsets of it, with an age-appropriate formulation and strength. The application must include the data generated in compliance with an agreed paediatric investigation plan, and the product may keep the brand name of the existing adult product so that prescribers recognise the substance.
The incentive is Article 38 of the Regulation: a PUMA receives the data exclusivity and market protection of a new application, eight years of data protection and ten years of market protection, even though the substance has been known for decades. Article 31 allows the application to be submitted through the centralised procedure, which most sponsors use. The PUMA is assessed in the normal way, with the Paediatric Committee confirming compliance with the plan before validation. A PUMA also benefits from the fee reductions the Paediatric Regulation provides for paediatric procedures, and the product information presents the paediatric posology and formulation as the primary use rather than as an addition to adult labelling.
Few PUMAs have been granted since the Regulation entered into force, the first in 2011, because the economics are difficult: the adult product is cheap and widely available, and nothing stops prescribers using it off-label in children. Pricing and reimbursement bodies often refuse to pay a premium for the paediatric formulation, and national substitution rules can route pharmacists to the adult product. The Commission's own evaluation of the Paediatric Regulation recorded the low uptake.
What goes wrong: sponsors who count on the exclusivity without checking whether the adult product's off-label use leaves any commercial space; a paediatric investigation plan agreed with the Paediatric Committee that is then changed without a modification procedure, so compliance cannot be confirmed; and formulations that need excipients unsuitable for the youngest children, which the assessment will question. A PUMA also carries every obligation of a new authorisation, including an RMP, PSURs and a renewal, which small sponsors sometimes underestimate.
Timing
The paediatric investigation plan must be agreed, and its completion confirmed by the Paediatric Committee, before the PUMA application is validated. The centralised assessment then runs 210 active days with the usual clock-stops, followed by a Commission decision. Protection runs from the Commission decision: eight years of data exclusivity and ten years of market protection, independent of the adult product's history.
Used in
Before committing to a PUMA, estimate the application and annual fees for a paediatric product with the Regunaut fee calculator, including the paediatric reductions.
Related terms
- PIP: paediatric investigation planA paediatric investigation plan (PIP) is the agreed programme of studies in children a new medicine must complete, waive or defer before EU authorisation.
- Paediatric extension: six-month SPC extension for a completed PIPThe paediatric extension adds six months to a supplementary protection certificate as a reward for completing an agreed paediatric investigation plan.
- Data exclusivity: regulatory data protectionData exclusivity is the eight years after a reference product's first EU authorisation in which generics cannot rely on its preclinical and clinical data.
- Centralised procedure: single EU marketing authorisation via EMAThe centralised procedure grants one marketing authorisation valid across the EU and EEA after a single EMA assessment and a Commission decision.
- Pricing and reimbursement: national decisions on price and public fundingPricing and reimbursement are the national decisions, taken after authorisation, that set a medicine's price and whether a health system pays for it.
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