ISSUE:

Qualitative methods can be used by health technology assessment (HTA) bodies to incorporate the views of patients and their carers in their decisions. We will present a case study and discuss how patient involvement adds value for decision makers. Andrew Lloyd will introduce the session and outline the challenges with understanding patient burden in rare diseases and how these data can support the work of decision makers. Eliza Kruger will present a case study from people with glycogen storage disorder Ia, where novel methods were used to capture the patient perspective. Val Buxton will discuss the challenges that they face as a patient organisation in communicating the needs of patients and the burden of disease to decision makers. Val will reflect on the value of the data in the case study. Heidi Livingstone (NICE) will introduce new processes regarding how patient organisations can add value and provide constructive input to decision making. The discussion will delve into the benefits and importance of capturing the patient perspective for decision making and which sorts of approaches may or may not be acceptable.

OVERVIEW:

Rare conditions and their impact on patients can be poorly understood. Qualitative methods can capture these data in a more standardised approach and have advantages over other approaches. For many rare diseases there is no validated patient reported outcomes measure available for measuring burden or impact. Qualitative data can be well suited for supporting the discourse at committee hearings regarding both the burden of disease and the benefits of treatment. But a clearer understanding of the methodological challenges is needed. These will include questions around what role such data should have in a decision, what methods are best suited for the rare disease context, the potential solutions, the barriers to implementation, and the need for different methodologies and policy implications.