How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison
How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison
Aim: To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Patients & methods: Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies. Results: In Type 1 SMA, studies of risdiplam and nusinersen included similar populations. Indirect comparison results found improved survival and motor function with risdiplam versus nusinersen. Comparison with onasemnogene abeparvovec in Type 1 SMA and with nusinersen in Types 2/3 SMA was challenging due to substantial differences in study populations; no concrete conclusions could be drawn from the indirect comparison analyses. Conclusion: Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA.
MAIC, SMA, STC, indirect treatment comparison, nusinersen, onasemnogene abeparvovec, risdiplam, spinal muscular atrophy
347-370
Ribero, Valerie Aponte
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Daigl, Monica
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Martí, Yasmina
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Gorni, Ksenija
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Evans, Rachel
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Scott, David Alexander
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Mahajan, Anadi
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Abrams, Keith R
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Hawkins, Neil
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April 2022
Ribero, Valerie Aponte
3d9ae6e1-a858-4feb-97d3-dc7f3ed6a058
Daigl, Monica
17a040ab-59d1-4045-9ce7-bd629adbe7d2
Martí, Yasmina
45ae9f66-b25f-4417-8c49-48e1907973fd
Gorni, Ksenija
5a5c36eb-fd4d-48e9-b987-a1419ff79434
Evans, Rachel
a61a4bac-e744-45c2-aca2-59a1fbedb37b
Scott, David Alexander
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Mahajan, Anadi
cb32dc9e-026e-42b3-9f3a-9a1459914c9e
Abrams, Keith R
6db24e9a-24bb-4f34-8d32-a97352af4e09
Hawkins, Neil
1aa8112d-606d-4176-b306-9c22158c556d
Ribero, Valerie Aponte, Daigl, Monica, Martí, Yasmina, Gorni, Ksenija, Evans, Rachel, Scott, David Alexander, Mahajan, Anadi, Abrams, Keith R and Hawkins, Neil
(2022)
How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison.
Journal of comparative effectiveness research, 11 (5), .
(doi:10.2217/cer-2021-0216).
Abstract
Aim: To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Patients & methods: Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies. Results: In Type 1 SMA, studies of risdiplam and nusinersen included similar populations. Indirect comparison results found improved survival and motor function with risdiplam versus nusinersen. Comparison with onasemnogene abeparvovec in Type 1 SMA and with nusinersen in Types 2/3 SMA was challenging due to substantial differences in study populations; no concrete conclusions could be drawn from the indirect comparison analyses. Conclusion: Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA.
Text
cer-2021-0216
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More information
Accepted/In Press date: 14 December 2021
e-pub ahead of print date: 18 January 2022
Published date: April 2022
Additional Information:
Funding Information:
The systematic literature review and data extraction were conducted by Bridge Medical Consulting Ltd., London, UK and funded by F. Hoffmann-La Roche Ltd., Basel, Switzerland. Indirect treatment comparison analyses were designed by Visible Analytics, UK, funded by F. Hoffmann-La Roche Ltd. V Aponte Ribero, M Daigl, Y Martí and K Gorni are employees of and are stockholders in F. Hoffmann La Roche Ltd. R Evans, DA Scott, KR Abrams and N Hawkins are partners/employees of Visible Analytics Ltd., which designed the analysis and received consultancy fees and expenses from F. Hoffmann-La Roche Ltd. A Mahajan is an employee of Bridge Medical Consulting Ltd., which conducted this review and received consultancy fees and expenses from F. Hoffmann-La Roche Ltd. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Funding Information:
The systematic literature review and data extraction were conducted by Bridge Medical Consulting Ltd., London, UK and funded by F. Hoffmann-La Roche Ltd., Basel, Switzerland. Indirect treatment comparison analyses were designed by Visible Analytics, UK, funded by F. Hoffmann-La Roche Ltd. V Aponte Ribero, M Daigl, Y Mart? and K Gorni are employees of and are stockholders in F. Hoffmann La Roche Ltd. R Evans, DA Scott, KR Abrams and N Hawkins are partners/employees of Visible Analytics Ltd., which designed the analysis and received consultancy fees and expenses from F. Hoffmann-La Roche Ltd. A Mahajan is an employee of Bridge Medical Consulting Ltd., which conducted this review and received consultancy fees and expenses from F. Hoffmann-La Roche Ltd. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Publisher Copyright:
© 2022 F. Hoffmann-La Roche Ltd.
Keywords:
MAIC, SMA, STC, indirect treatment comparison, nusinersen, onasemnogene abeparvovec, risdiplam, spinal muscular atrophy
Identifiers
Local EPrints ID: 454926
URI: http://eprints.soton.ac.uk/id/eprint/454926
ISSN: 2042-6305
PURE UUID: bfb02d9e-b97a-443f-a0c3-7f2a79f6124a
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Date deposited: 01 Mar 2022 17:50
Last modified: 17 Mar 2024 04:02
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Contributors
Author:
Valerie Aponte Ribero
Author:
Monica Daigl
Author:
Yasmina Martí
Author:
Ksenija Gorni
Author:
Rachel Evans
Author:
David Alexander Scott
Author:
Anadi Mahajan
Author:
Keith R Abrams
Author:
Neil Hawkins
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