{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,2]],"date-time":"2026-05-02T01:51:50Z","timestamp":1777686710574,"version":"3.51.4"},"reference-count":13,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2021,6,9]],"date-time":"2021-06-09T00:00:00Z","timestamp":1623196800000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JHD"],"published-print":{"date-parts":[[2021,6,9]]},"abstract":"<jats:p>Background: The most advanced disease-modifying therapies (DMTs) in development for Huntington\u2019s 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\u2019 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.<\/jats:p>","DOI":"10.3233\/jhd-200462","type":"journal-article","created":{"date-parts":[[2021,4,9]],"date-time":"2021-04-09T12:16:30Z","timestamp":1617970590000},"page":"303-311","source":"Crossref","is-referenced-by-count":4,"title":["The Impact of Upcoming Treatments in Huntington\u2019s Disease: Resource Capacity Limitations and Access to Care Implications"],"prefix":"10.1177","volume":"10","author":[{"given":"Mark","family":"Guttman","sequence":"first","affiliation":[{"name":"Centre for Movement Disorders, Toronto, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marco","family":"Pedrazzoli","sequence":"additional","affiliation":[{"name":"LSC Life Sciences Consultants, Milan, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marina","family":"Ponomareva","sequence":"additional","affiliation":[{"name":"LSC Life Sciences Consultants, Milan, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marsha","family":"Pelletier","sequence":"additional","affiliation":[{"name":"LSC Life Sciences Consultants, Milan, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Louisa","family":"Townson","sequence":"additional","affiliation":[{"name":"F. Hoffmann-La Roche Ltd, Basel, Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kopano","family":"Mukelabai","sequence":"additional","affiliation":[{"name":"F. Hoffmann-La Roche Ltd, Basel, Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Aaron","family":"Levine","sequence":"additional","affiliation":[{"name":"F. Hoffmann-La Roche Ltd, Basel, Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna-Lena","family":"Nordstr\u00f6m","sequence":"additional","affiliation":[{"name":"F. Hoffmann-La Roche Ltd, Basel, Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ralf","family":"Reilmann","sequence":"additional","affiliation":[{"name":"George Huntington Institute, Muenster, Germany"},{"name":"Department of Clinical Radiology, University of Muenster, Muenster, Germany"},{"name":"Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Tuebingen, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jean-Marc","family":"Burgunder","sequence":"additional","affiliation":[{"name":"Swiss Huntington\u2019s Disease Centre, Siloah, G\u00fcmligen, Switzerland"},{"name":"Department of Neurology, University of Bern, Bern, Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","reference":[{"issue":"2","key":"10.3233\/JHD-200462_ref1","doi-asserted-by":"crossref","first-page":"207","DOI":"10.3233\/JHD-160192","article-title":"Health care delivery practices in Huntington\u2019s disease specialty clinics: An international survey","volume":"5","author":"Frich","year":"2016","journal-title":"J Huntingtons Dis"},{"key":"10.3233\/JHD-200462_ref2","doi-asserted-by":"crossref","first-page":"710","DOI":"10.3389\/fneur.2019.00710","article-title":"International guidelines for the treatment of Huntington\u2019s disease","volume":"10","author":"Bachoud-Levi","year":"2019","journal-title":"Front Neurol"},{"issue":"4","key":"10.3233\/JHD-200462_ref4","doi-asserted-by":"crossref","first-page":"842","DOI":"10.1002\/ajmg.a.38494","article-title":"Therapy development in Huntington disease: From current strategies to emerging opportunities","volume":"176","author":"Dickey","year":"2018","journal-title":"Am J Med Genet A"},{"issue":"5","key":"10.3233\/JHD-200462_ref5","doi-asserted-by":"crossref","first-page":"801","DOI":"10.1016\/j.neuron.2019.01.039","article-title":"Huntingtin lowering strategies for disease modification in Huntington\u2019s disease","volume":"101","author":"Tabrizi","year":"2019","journal-title":"Neuron"},{"issue":"11","key":"10.3233\/JHD-200462_ref6","doi-asserted-by":"crossref","first-page":"1434","DOI":"10.1002\/mds.26007","article-title":"Targets for future clinical trials in Huntington\u2019s disease: What\u2019s in the pipeline?","volume":"29","author":"Wild","year":"2014","journal-title":"Mov Disord"},{"issue":"1","key":"10.3233\/JHD-200462_ref7","doi-asserted-by":"crossref","first-page":"1","DOI":"10.2217\/nmt.11.85","article-title":"A standard of care for Huntington\u2019s disease: Who, what and why","volume":"2","author":"Simpson","year":"2012","journal-title":"Neurodegener Dis Manag"},{"issue":"10","key":"10.3233\/JHD-200462_ref8","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1016\/S1474-4422(17)30280-6","article-title":"Therapies targeting DNA and RNA in Huntington\u2019s disease","volume":"16","author":"Wild","year":"2017","journal-title":"Lancet Neurol"},{"issue":"14","key":"10.3233\/JHD-200462_ref9","doi-asserted-by":"crossref","first-page":"6518","DOI":"10.1093\/nar\/gkw236","article-title":"The delivery of therapeutic oligonucleotides","volume":"44","author":"Juliano","year":"2016","journal-title":"Nucleic Acids Res"},{"issue":"2","key":"10.3233\/JHD-200462_ref10","doi-asserted-by":"crossref","first-page":"114","DOI":"10.1016\/j.pharmthera.2014.05.009","article-title":"Intrathecal delivery of protein therapeutics to the brain: A critical reassessment","volume":"144","author":"Calias","year":"2014","journal-title":"Pharmacol Ther"},{"key":"10.3233\/JHD-200462_ref11","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1007\/978-1-4939-7825-0_23","article-title":"Translating antisense technology into a treatment for Huntington\u2019s disease","volume":"1780","author":"Lane","year":"2018","journal-title":"Methods Mol Biol"},{"issue":"7","key":"10.3233\/JHD-200462_ref12","doi-asserted-by":"crossref","first-page":"899","DOI":"10.1177\/0883073815627882","article-title":"Intrathecal injections in children with spinal muscular atrophy: Nusinersen clinical trial experience","volume":"31","author":"Hache","year":"2016","journal-title":"J Child Neurol"},{"key":"10.3233\/JHD-200462_ref14","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1515\/tnsci-2017-0001","article-title":"ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy","volume":"8","author":"Ottesen","year":"2017","journal-title":"Transl Neurosci"},{"key":"10.3233\/JHD-200462_ref15","doi-asserted-by":"crossref","first-page":"227","DOI":"10.3233\/JND-190387","article-title":"Implementing a global Expanded Access Program (EAP) for infantile-onset spinal muscular atrophy (Type I): Understanding the imperative, impact and challenges","volume":"6","author":"Yong","year":"2019","journal-title":"J Neuromusc Dis"}],"container-title":["Journal of Huntington's Disease"],"original-title":[],"link":[{"URL":"https:\/\/content.iospress.com\/download?id=10.3233\/JHD-200462","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T08:55:59Z","timestamp":1777452959000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/full\/10.3233\/JHD-200462"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,6,9]]},"references-count":13,"journal-issue":{"issue":"2"},"URL":"https:\/\/doi.org\/10.3233\/jhd-200462","relation":{},"ISSN":["1879-6397","1879-6400"],"issn-type":[{"value":"1879-6397","type":"print"},{"value":"1879-6400","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,6,9]]}}}