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

• Published in: Journal of Huntington's disease Vol. 10; no. 2; pp. 303 - 311
• Main Authors: Guttman, Mark, Pedrazzoli, Marco, Ponomareva, Marina, Pelletier, Marsha, Townson, Louisa, Mukelabai, Kopano, Levine, Aaron, Nordström, Anna-Lena, Reilmann, Ralf, Burgunder, Jean-Marc
• Format: Journal Article
• Language: English
• Published: London, England SAGE Publications 01.01.2021; IOS Press BV; IOS Press
• Subjects: Health care access; Health Facilities; Health Resources; Health Services Accessibility; Health Workforce; Humans; Huntington Disease - therapy; Huntington's disease; Huntingtons disease; Injections, Spinal; Research Report; Spinal Puncture; and services; health care facilities; health resources; intrathecal injection; spinal puncture; Huntington’s disease; health services accessibility; manpower; capacity building
• ISSN: 1879-6397; 1879-6397; 1879-6400
• DOI: 10.3233/JHD-200462

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 time 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.