Abstract

The importance of considering the patient perspective in health technology assessments (HTA) is widely acknowledged. The aim of this research was to understand how HTA bodies incorporate patient input to inform their decision-making and whether certain approaches are more impactful. This study focused on 12 countries (9 European, Australia, Brazil and Canada). Information was retrieved from peer reviewed publications, grey literature, and publicly available information from national and regional HTA bodies. Information was reviewed to identify and map formal pathways for patient input within the HTA appraisal and decision-making process. Eligibility criteria, submission format and level of interaction were compared to determine potential for patient input to impact HTA outcomes. Significant variation was identified across the processes for incorporating patient input of the scope countries. Cross-country comparison identified 5 approach types with varying levels of opportunity to impact decision-making. Ranging from routine inclusion of patient experts in HTA committee meetings (Australia (PBAC), England (NICE), Germany (G-BA) and Scotland (SMC)) to the acceptance of online patient submissions (Brazil (CONTEC), Germany (IQWIG), Netherlands (ZIN), Norway (NoMA), Spain (CatSalut)). This research found considerable difference in the level of transparency of HTA agencies publicly reporting the processes for patient involvement. Payers face an increasingly challenging environment with a burgeoning pipeline of orphan drugs, cell and gene therapies. These treatments are often for diseases with a high unmet need and further raise the importance for considering the patient perspective in decision-making. There is a trend for countries with a longer history of HTA to have HTA bodies with more evolved and transparent processes for patient involvement. However, it is crucial that all HTA bodies seek to implement best practices by developing transparent, formal processes that ensure patient input is provided by appropriate patient experts to inform reimbursement decision-making.

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