The Paediatric Use Marketing Authorisation (PUMA) was introduced in the European Union to incentivise the development of off-patent medicines in children. However, there is limited data on the accessibility of PUMA products at the healthcare provider level. This study aimed to identify factors affecting real-world accessibility to PUMA products in the United Kingdom (UK). Inductive thematic analyses of the archives of the Neonatal and Paediatric Pharmacy Group (NPPG) online forum were conducted. A web-based survey was also distributed to NPPG members in September 2022 regarding the availability of PUMA products in their organisations. Thematic analysis generated five themes: authorisation, availability, affordability, appropriateness and acceptability. Restricted scope of the product’s marketing authorisation, market access variation, higher cost of PUMA products, product appropriateness and patient acceptability were reasons for continued off-label use and use of unlicensed products in clinical practice. Conclusion: Despite targeted legislative efforts to bring off-label uses in children into authorised use, this study provides evidence that authorisation alone does not equate to market availability, which in turn does not guarantee patient access. The study findings also suggest that cost pressure drives local procurement decisions and overshadows the long-standing problems associated with off-label use and manipulation of medicines in children.<Table Float="No" ID="Taba"> <tgroup cols="1"> <colspec align="left" colname="c1" colnum="1" /> <tbody> <row> <entry align="left" colname="c1"> What is already known about this subject • The Paediatric Use Marketing Authorisation (PUMA) was introduced in the European Union (applied to the UK at the time) to incentivise the development of off-patient medicines exclusively for use in the paediatric population. • It is widely acknowledged that the PUMA concept has not achieved its intended goal, as evidenced by the few products authorised through this route. </entry> </row> <row> <entry align="left" colname="c1"> What this study adds • This study shows inequalities in children's access to PUMA products in the UK. • Determinants impeding patient access to child-appropriate paediatric medicines can be categorised into five dimensions: authorisation, availability, affordability, appropriateness, and acceptability. </entry> </row> </tbody> </tgroup> </Table>

Access to child-appropriate medicines: an exploratory survey of the use of paediatric use marketing authorisation products in the UK

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2025年1月25日

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Mandy Wan,
Amin Houshian,
Stephen Tomlin,
Asia N. Rashed

摘要

The Paediatric Use Marketing Authorisation (PUMA) was introduced in the European Union to incentivise the development of off-patent medicines in children. However, there is limited data on the accessibility of PUMA products at the healthcare provider level. This study aimed to identify factors affecting real-world accessibility to PUMA products in the United Kingdom (UK). Inductive thematic analyses of the archives of the Neonatal and Paediatric Pharmacy Group (NPPG) online forum were conducted. A web-based survey was also distributed to NPPG members in September 2022 regarding the availability of PUMA products in their organisations. Thematic analysis generated five themes: authorisation, availability, affordability, appropriateness and acceptability. Restricted scope of the product’s marketing authorisation, market access variation, higher cost of PUMA products, product appropriateness and patient acceptability were reasons for continued off-label use and use of unlicensed products in clinical practice. Conclusion: Despite targeted legislative efforts to bring off-label uses in children into authorised use, this study provides evidence that authorisation alone does not equate to market availability, which in turn does not guarantee patient access. The study findings also suggest that cost pressure drives local procurement decisions and overshadows the long-standing problems associated with off-label use and manipulation of medicines in children.

What is already known about this subject

• The Paediatric Use Marketing Authorisation (PUMA) was introduced in the European Union (applied to the UK at the time) to incentivise the development of off-patient medicines exclusively for use in the paediatric population.

• It is widely acknowledged that the PUMA concept has not achieved its intended goal, as evidenced by the few products authorised through this route.

What this study adds

• This study shows inequalities in children's access to PUMA products in the UK.

• Determinants impeding patient access to child-appropriate paediatric medicines can be categorised into five dimensions: authorisation, availability, affordability, appropriateness, and acceptability.