Presentation (CTI)

OBJECTIVES: In Ireland, the National Centre for Pharmacoeconomics (NCPE) calls for submissions from patient organisation (PO) groups upon receipt of a company health technology assessment (HTA). Many PO’s also function as patient advocacy groups (PAG) for rights and access to treatment. Inclusion of the patient voice ensures that their perspective is fairly considered in the HTA process. This research investigated the long-term trends in PO submissions submitted to the NCPE, and future patient-centric perspectives.
METHODS: HTA assessments completed between 2018 and 2024 were compiled into a database and technical summary reports reviewed. Descriptive statistics were used to quantify the number of PO submissions received overall, within oncology-specific, non-oncology, and rare-disease indications.
RESULTS: HTA submissions (N=139) from 2018-2024 were mostly oncology-specific (58%) versus non-oncology (42%), or non-rare (63%) (rare-disease, 37%). Of all PO submissions identified (51/139; 37%): most were non-oncology (61%) versus oncology-specific (39%), with rare-diseases accounting for 55% (non-rare, 45%). Long-term PO trends showed a marked increase from 3 (2.16%/139) in 2018 to 15 (10.8%/139) in 2023. Although some 2024 HTAs are still in progress, 4 of 9 available technical reports (44.4%) had PO submissions suggesting a continuing long-term trend.
CONCLUSIONS: The results show a numerically increasing long-term trend in PO submissions to the NCPE since 2018. This highlights the growing involvement of patient advocacy in the HTA process. The high levels of rare disease advocacy reflect the unique challenges faced by patients with these conditions and importance of their representation in the HTA process. Linear regression analysis will focus on examining the trend prior and after 2018 following the NCPE public consultation to implement strategies to refine the PO submission process. Another future perspective is earlier utilisation of PAGs at the NCPE rapid review stage to increase the strength of the patient voice throughout the entire HTA process.