Aim Recent decades have seen the development of regulatory frameworks aimed at increasing the availability of medicines authorised for children. In Europe, the Paediatric Use Marketing Authorisation (PUMA) was introduced to incentivise the development of off-patent drugs as many of the products used in children have not been adequately tested and are often not available in a dosage form suitable for children.¹ As of 2022 only six products have been granted a PUMA. While these products were exclusively developed and centrally authorised for children, anecdotal evidence suggests that patient access to these products varies across and within countries. This study aimed to identify factors affecting real-world accessibility to PUMA products in the United Kingdom (UK).

Method The archives of the Neonatal and Paediatric Pharmacy Group (NPPG) online forum (approx. 500 members) was searched for the period between 2011 and February 2022. The forum is a UK-based online platform for paediatric pharmacy professionals to discuss topics related to the pharmaceutical care of children in both primary and secondary settings. Relevant posts were identified using the brand name of the six products and their generic names as search terms. Inductive thematic analysis was performed by two independent researchers using NVivo software. A web-based survey was also distributed via the NPPG in September 2022 over a 4-week period. The survey consisted of mixed multiple-choice and open questions on the availability of the six products in the respondent’s organisation. Data were analysed using descriptive statistics.

Results Thematic analysis generated three themes: affordability, market availability, and scope of the approved conditions of use. Affordability was discussed mostly in the context of higher cost of PUMA products, where there is evidence that access is restricted by cost pressure. Alkindi^® hydrocortisone granules are available in four strengths, designed to enable accurate and flexible dosing; yet only four hospitals surveyed (8%, 4/50) stocked all available strengths, corroborating with forum comments indicating continued off-label use of other hydrocortisone products in practice that involves manipulation of dosage form. Forum members commented on the unavailability of Hemangiol^® solution in the UK market, which is consistent with survey results showing that none of the hospitals stocked this product. Buccolam^® oromucosal solution was most widely stocked by hospitals (94%, 47/50), of which 95.7% (45/47) had access to all strengths available. Forum members and survey respondents also commented on the restricted scope of the approved conditions of use of these products including selective patient groups, both in terms of age and therapeutic indications, as well as route of administration.

Conclusion Despite targeted legislative efforts to increase the availability of medicines authorised for children, our results show that authorisation does not equate to market availability, which in turn does not guarantee patient access. Our study provides evidence substantiating the most voiced concern that a PUMA does not offer meaningful market exclusivity in practice.¹ The data also suggest that cost pressure drives local procurement decisions and seems to significantly eclipse the long-standing problems associated with off-label use and manipulation of medicines in children.

Reference

1. European Commission. State of Paediatric Medicines in the EU 10 years of the EU Paediatric Regulation. 2017. https://ec.europa.eu/health/system/files/2017-11/2017_childrensmedicines_report_en_0.pdf (Accessed 07 July 2023).