OBJECTIVES: Considering patient insights in healthcare decision making is particularly relevant in rare diseases where the patient population is small and published clinical evidence may be limited. Different health technology assessment (HTA) bodies place differing emphasis and have different preferences when it comes to assessing the inclusion of patient insights in submissions. This research aims to understand how best to utilise patient insights in HTA submissions, drawing on four representative country HTA’s Australia, France, Italy, and the Netherlands focusing on late-onset Pompe disease (LOPD).

METHODS: HTA body websites (Pharmaceutical Benefits Advisory Committee, Haute Autorité de Santé, Agenzia Italiana del Farmaco and Zorginstituut Nederland) and publications relating to the use of patient insights were analysed for guidance on including the patient voice in submissions. Learnings from this analysis were then implemented to collect insights from 40 people with LOPD. The SF-36 tool was used to derive patients’ Health-Related Quality of Life, supplemented with additional insights. 10 patients were then selected from this cohort for follow-up interviews to gain deeper understanding of the disease helping contextualise the patient perspective in HTA decision making.

RESULTS: In general, HTA bodies prefer the inclusion of validated widely used patient-reported outcome measures. Except for Australia, HTA bodies require patient-reported outcomes to be collected in the country of submission. There are many validated tools that are appropriate to use to collect patient insights for HTA submissions. SF-36 was chosen as it is generic and has been used in LOPD previously.

CONCLUSIONS: Patient insights allow HTA bodies to understand the impact of disease on patients’ lives and are becoming of increasingly important in reimbursement decisions, particularly in rare diseases. Using a validated tool alongside disease specific questions may allow organisations to submit patient insights that can support HTA submissions for reimbursement.