The Impact of Upcoming Treatments in Huntington’s Disease: Resource Capacity Limitations and Access to Care Implications

Abstract

Background: The most advanced disease-modifying therapies (DMTs) in development for Huntington’s disease (HD) require intrathecal (IT) administration, which may create or exacerbate bottlenecks in resource capacity. Objective: To understand the readiness of healthcare systems for intrathecally administered HD DMTs in terms of resource capacity dynamics and implications for patients’ access to treatment. Methods: Forty HD centres across 12 countries were included. Qualitative and quantitative data on current capacity in HD centres and anticipated capacity needs following availability of a DMT were gathered via interviews with healthcare professionals (HCPs). Data modelling was used to estimate the current capacity gap in HD centres. Results: From interviews with 218 HCPs, 25% of HD centres are estimated to have the three components required for IT administration (proceduralists, nurses and facilities). On average, 114 patients per centre per year are anticipated to receive intrathecally administered DMTs in the future. At current capacity, six of the sampled centres are estimated to be able to deliver DMTs to all the anticipated patients based on current resources. The estimated waiting list for IT administration at current capacity will average 60 months (5 years) by the second year after DMT availability. Conclusion: Additional resources are needed in HD centres for future DMTs to be accessible to all anticipated patients. Timely collaboration by the HD community will be needed to address capacity gaps. Healthcare policymakers and payers will need to address costs and navigate challenges arising from country- or region-specific healthcare delivery schemes.

Canadian healthcare capacity gaps for disease-modifying treatment in Huntington’s disease: a survey of current practice and modelling of future needs

“…We confirmed that Canadian HD centres have limited current capacity to administer IT DMTs due to nursing, neurologist or room time. Overall, the incremental FTEs required in Canadian centres appear similar to those predicted internationally 16. Neurologist capacity and willingness to administer IT DMTs are also serious areas of concern, with shortages and waning interest in the specialty already a threat to the system 36 37.…”

“…Overall, the incremental FTEs required in Canadian centres appear similar to those predicted internationally. 16 Neurologist capacity and willingness to administer IT DMTs are also serious areas of concern, with shortages and waning interest in the specialty already a threat to the system. 36 37 Furthermore, when limitations in social worker capacity were accounted for, national capacity to treat decreased to only 6% of IT DMT-eligible patients with HD.…”

“…Using a case study, mixed methods framework,21 cross-sectional online surveys and follow-up phone interviews were modelled on a global study,16 modified by the Canadian study team, and validated by both Canadian neurologists with experience administering an IT DMT15 16 and a Canadian HD patient organisation representative. Clinical trial experience indicated that an HD IT DMT could be administered by a trained neurologist in a hospital or community setting, given adequate supports and capacity.…”

“…Considering the late stage exploration of intrathecal (IT) agents for movement disorders14 15 and anticipated healthcare capacity limitations,16–18 challenges were expected in the geographically vast, regionally diverse and publicly-funded Canadian healthcare system 19. In alignment with implementation science and practice improvement,20 we sought to understand current and future capacity for HD management in Canada and to qualify potential gaps arising from the theoretical introduction of an IT DMT.…”

Disease Burden of Huntington’s Disease (HD) on People Living with HD and Care Partners in Canada

“…The World Health Organization defines quality of life as the concept individuals have of their position in life, which includes perspective from their culture, as well as their evaluative systems, expectations, priorities, available infrastructure, and other factors [ 22, 23 ]; the jurisdiction from where HRQoL data originate is therefore important. Similarly, healthcare resources available to individuals with HD also vary between countries [ 24, 25 ], suggesting HRU associated with HD in Canada may be unique.…”