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Assessment of face ...
Assessment of face validity of a disease model of nonsense mutation Duchenne muscular dystrophy: a multi-national Delphi panel study
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Landfeldt, E. (author)
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Zhang, R. R. (author)
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Childs, A. M. (author)
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Johannsen, J. (author)
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O'Rourke, D. (author)
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- Sejersen, T. (author)
- Karolinska Institutet
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Strautmanis, J. (author)
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Schara-Schmidt, U. (author)
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- Tulinius, Mar, 1953 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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Walter, M. C. (author)
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Willis, T. (author)
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Buesch, K. (author)
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(creator_code:org_t)
- 2022-06-14
- 2022
- English.
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In: Journal of Medical Economics. - : Informa UK Limited. - 1369-6998 .- 1941-837X. ; 25:1, s. 808-816
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https://gup.ub.gu.se...
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Abstract
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- Objective The objective of this study was to assess the face validity of a disease model evaluating the cost-effectiveness of ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). Methods This was a Delphi panel study comprising of physicians with first-hand experience of ataluren for the treatment of nmDMD. Consensus was sought for previously unvalidated model data, including patient health status and quality of life measured using the Health Utility Index (HUI), mortality, informal caregiving, and the expected benefit of early ataluren treatment across four states: (1) ambulatory, (2) non-ambulatory, not yet requiring ventilation support, (3) non-ambulatory, night-time ventilation support, and (4) non-ambulatory, full-time ventilation support. Results Nine experts from five countries participated in the Delphi panel. Consensus was obtained for all questions after three panel rounds (except for two HUI-questions concerning hand function [dexterity]). Consensus HUI-derived utilities for state (1) were 1.0000 for ataluren on top of best supportive care (BSC) and 0.7337 for BSC alone. Corresponding estimates for state (2) were 0.3179 and 0.2672, for state (3) 0.1643 and 0.0913, and for state (4) -0.0732 and -0.1163. Consensus mortality rates for states (1), (2), and (3) were 4%, 13%, and 33%, and life expectancy in state (4) was agreed to be 3 years. Panelists further agreed that two informal caregivers typically provide day-to-day care/support to patients with nmDMD, and that starting treatment with ataluren at 2 versus 5 years of age would be expected to delay loss of ambulation by an additional 2 years, and initiation of night-time and full-time ventilation support by an additional 3 years, respectively. Limitations The main limitation concerns the size of the Delphi panel, govern primarily by the rarity of the disease. Conclusion This study confirms the face validity of key clinical parameters and assumptions underlying the ataluren cost-effectiveness model.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Medicinsk genetik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Medical Genetics (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Allmänmedicin (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- General Practice (hsv//eng)
Keyword
- Duchenne muscular dystrophy
- quality of life
- mortality
- treatment
- health policy
- model
- quality-of-life
- ataluren
- dmd
- Health Care Sciences & Services
- General & Internal Medicine
Publication and Content Type
- ref (subject category)
- art (subject category)
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- By the author/editor
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Landfeldt, E.
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Zhang, R. R.
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Childs, A. M.
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Johannsen, J.
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O'Rourke, D.
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Sejersen, T.
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show more...
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Strautmanis, J.
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Schara-Schmidt, ...
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Tulinius, Mar, 1 ...
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Walter, M. C.
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Willis, T.
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Buesch, K.
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Basic Medicine
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and Medical Genetics
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and General Practice
- Articles in the publication
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Journal of Medic ...
- By the university
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University of Gothenburg
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Karolinska Institutet