Timothy Hugh Barker, Lemma N Bulto, Grace Holland, Camille Schubert, Tracy Merlin, Samer G Karam, Wojtek Wiercioch, Danielle Pollock, Cindy Stern, Jay Beasley-Hall, Nipun Shrestha, Steve Vucic, Lynne Giles, Zachary Munn
This review provides a comprehensive synthesis of the values and preferences of the broad MND community. This review has been conducted following rigorous best-practice methodology, to directly inform the selection and prioritisation of outcomes for the development of the Australian MND Guideline, ensuring the guideline adheres to a patient-centred approach. Standardisation of preference elicitation methods from the MND community, and the development of a core outcome set for future MND research are key future priorities.
AIM: To synthesise quantitative evidence on the values and preferences of people living with Motor Neurone Disease (MND), caregivers, and genetic carriers regarding health-related outcomes to inform the Australian MND Guideline.
METHODS: A systematic review was conducted following Cochrane and GRADE guidance, informed by an a priori protocol. Major electronic databases (including MEDLINE, Embase, CENTRAL) and trial registries were searched to identify studies that met the eligibility criteria. Risk of bias of the studies that met the eligibility criteria was assessed using the Risk of Bias in Studies of Values and Utilities (ROBVALU) tool. Data on health state utility values were synthesised using meta-analysis where appropriate, while other quantitative data deemed inappropriate for meta-analysis were synthesised narratively. The certainty of the evidence for each outcome was assessed using the GRADE approach.
RESULTS: Twenty-four studies (n = 10,397) were included. Overall health-related quality of life (hrQoL) utility values for adults with MND varied significantly based on the regional preference set utilised (mean EQ-5D utility ranging from 0.57 in UK cohort (high certainty in the evidence) to 0.72 in Chinese cohorts (low certainty in the evidence). Utility values declined consistently with increasing disease severity across multiple staging systems, such as King's and MiToS. Narrative synthesis identified clear preferences across physical, psychosocial, and healthcare domains regarding both current and hypothetical treatment strategies.
CONCLUSION: This review provides a comprehensive synthesis of the values and preferences of the broad MND community. This review has been conducted following rigorous best-practice methodology, to directly inform the selection and prioritisation of outcomes for the development of the Australian MND Guideline, ensuring the guideline adheres to a patient-centred approach. Standardisation of preference elicitation methods from the MND community, and the development of a core outcome set for future MND research are key future priorities.