Modification of the All Wales Medicines Strategy Group appraisal process provides faster access to children's medicines in Wales

Health Technology Assessment International (HTAi) Annual Conference, June 2024, Seville, Spain.

Introduction: Medicines routinely funded for use in Wales undergo health technology appraisal by the All Wales Medicines Strategy Group (AWMSG) or the National Institute for Health and Care Excellence (NICE). This includes paediatric licence extensions (PLE) notwithstanding any existing advice in adults. Review of the PLE process was conducted with the aim of providing faster access to children’s medicines in Wales. 

Methods:  Data were collected for PLE appraisals of medicines previously approved for adults by AWMSG or NICE that subsequently went through the original PLE process between January 2010 and December 2020, or a simplified PLE process between January 2021 and March 2023. Data were analysed by descriptive statistics, mean ( standard deviation) and a two tailed t-test (unequal variance) to test the null hypothesis that the difference between the two means was zero. An alpha of < 0.05 was considered significant. Feedback was obtained from relevant stakeholders (Association of the British Pharmaceutical Industry (Wales) (ABPI) and Royal College of Paediatrics).   

Results: AWMSG issued positive recommendations for all PLE appraisals included in the data collected, and these were endorsed by Welsh Government. Appraisals that went through the original PLE process (n = 56) took a mean 229.8 days (± 55.6) while those that went through the simplified PLE process (n = 15) took a mean 102.6 days (± 48.1), p < 0.0001. The rapid access to children’s medicines was welcomed by the Association of the British Pharmaceutical Industry (Wales) and Royal College of Paediatrics.    

Conclusions: The 2020 and 2023 reviews of the PLE process facilitated faster access to clinically and cost effective medicines for children in Wales. In March 2023, AWMSG and Welsh Government reviewed these results and agreed that because all PLE medicines were approved for use within NHS Wales irrespective of the process used, AWMSG would no longer be required to routinely appraise PLEs.