Presentation (CTI)

OBJECTIVES: Evidence across various categories, from disease burden to management of rare diseases, is generally quite low, and result in highly uncertain environments for HTA bodies. Patient-based evidence is becoming increasingly important in HTA, but given evidence scarcity, there’s higher reliance on qualitative data to convey value. We sought to understand which qualitative methods are commonly used and how key HTA bodies like NICE, HAS, G-BA, and CDA (CADTH) consider qualitative data presented in submissions.
METHODS: We identified products that were evaluated by NICE under the highly specialised technology (HST) pathway since 2020. HTA evaluations for these products by NICE, HAS, G-BA, and CDA/CADTH were considered, including any publicly available committee papers and submission / guidance documents. A systematic review was conducted to assess the use of qualitative data, data sources, and methodology; evidence categories in which these were used, i.e. for elucidating specific aspects of disease burden to treatment implementation, or patient preference.
RESULTS: Qualitative evidence was used in almost all the submissions considered (N=22); explicitly in >90% of submissions. These took the form of patient, clinician or caregiver expert reports and narratives, and to a lesser extent patient/caregiver interviews. Evidence presented pertained to illuminating aspects of disease burden, particularly around health-related quality of life (HRQoL). Qualitative evidence in all submissions was considered by all three HTA bodies.
CONCLUSIONS: Patient expert reports or narratives, caregiver impacts and clinician narratives have become standardised practice in committee discussions across all three bodies. Evidence for impact on daily living, families and caregivers was predominantly qualitative in these submissions, and featured heavily in discussions around HRQoL impact. As a key source of patient voice in decision-making, it’s imperative to have guidance asserting transparent and rigorous data collection and analysis of qualitative evidence to ensure credibility and usefulness.