The Organisational Impact of Upcoming Treatments in Huntington’s Disease: Resource Capacity Gaps and Access to Care Implications
Marco Pedrazzoli1, Marina Ponomareva1, Mattia Moro1, Marsha Pelletier1, Louisa Townson2, Kopano Mukelabai2, Aaron Levine2, Anna-Lena Nordström2, Mark Guttman3, Jean-Marc Burgunder4,5, Ralf Reilmann6
1LSC Life Sciences Consultants, 2F. Hoffmann-La Roche Ltd, 3Centre for Movement Disorders, 4Swiss Huntington's Disease Centre, 5Department of Neurology, University of Bern, 6George Huntington Institute
To assess and understand (1) the impact of upcoming disease-modifying-therapies (DMTs) for Huntington’s disease (HD) on healthcare systems and (2) possible resource capacity gaps and its implications for access to care.

HD is a genetic, progressive neurodegenerative disease. While no DMTs are available, several approaches are in clinical development. The investigational drugs most advanced in clinical development are administered intrathecally as chronic treatment. The intrathecal procedure will require additional resources in HD clinics. Also, individuals not yet seen at HD clinics or not previously diagnosed are expected to seek access to care.


Thirty-five HD specialist centres from 9 countries were involved in a prospective study to assess their capacity to perform intrathecally administered treatments. Data on current resource availability, utilisation, skills and equipment were collected by conducting interviews with >170 healthcare professionals. Capacity gaps were assessed by comparing resources currently available in each HD centre to an approximation of the amount of resources that will be required in the future for the estimated eligible patient population.


Only 20% of participating HD teams currently have the required resources to perform intrathecal injections: a skilled “proceduralist” (e.g. trained neurologist, anesthesiologist, interventional radiologist), one or more nurses to assist in the procedure, and the appropriate space. When considering all resources available in the hospital (e.g. neurology department, infusion suites), only 17% of HD specialist clinics are estimated to have sufficient capacity to serve the eligible population for intrathecally administered DMTs. When simulating the additional referral-in of patients from non-HD-specialized clinics, only 6% of HD clinics have sufficient capacity.


To ensure adequate care, capacity constrained healthcare systems will need to plan adequately and ensure providers have sufficient training and resources to be able to deliver new DMTs, while coping with an increased demand for diagnosis, treatment and follow-up.