How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison
To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneit...
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| Published in | Journal of comparative effectiveness research Vol. 11; no. 5; pp. 347 - 370 |
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| Main Authors | , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
England
Future Medicine Ltd
01.04.2022
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| Subjects | |
| Online Access | Get full text |
| ISSN | 2042-6305 2042-6313 2042-6313 |
| DOI | 10.2217/cer-2021-0216 |
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| Abstract | To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA).
Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies.
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.
Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA.
How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy? A systematic literature review and indirect treatment comparison. |
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| AbstractList | To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA).
Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies.
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.
Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA. 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.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. To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies. 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. Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA. 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. |
| Author | Evans, Rachel Scott, David Alexander Mahajan, Anadi Daigl, Monica Gorni, Ksenija Hawkins, Neil Martí, Yasmina Abrams, Keith R Ribero, Valerie Aponte |
| AuthorAffiliation | 3Visible Analytics, Oxford, OX2 0DP, UK 1Global Access, F. Hoffmann-La Roche Ltd, 4070, Basel, Switzerland 2PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, 4070, Basel, Switzerland 4Bridge Medical Consulting Ltd., Richmond, London, TW9 2SS, UK |
| AuthorAffiliation_xml | – name: 2PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, 4070, Basel, Switzerland – name: 4Bridge Medical Consulting Ltd., Richmond, London, TW9 2SS, UK – name: 1Global Access, F. Hoffmann-La Roche Ltd, 4070, Basel, Switzerland – name: 3Visible Analytics, Oxford, OX2 0DP, UK |
| Author_xml | – sequence: 1 givenname: Valerie Aponte orcidid: 0000-0002-0034-0182 surname: Ribero fullname: Ribero, Valerie Aponte organization: Global Access, F. Hoffmann-La Roche Ltd, 4070, Basel, Switzerland – sequence: 2 givenname: Monica orcidid: 0000-0002-9279-8288 surname: Daigl fullname: Daigl, Monica organization: Global Access, F. Hoffmann-La Roche Ltd, 4070, Basel, Switzerland – sequence: 3 givenname: Yasmina surname: Martí fullname: Martí, Yasmina organization: Global Access, F. Hoffmann-La Roche Ltd, 4070, Basel, Switzerland – sequence: 4 givenname: Ksenija surname: Gorni fullname: Gorni, Ksenija organization: PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, 4070, Basel, Switzerland – sequence: 5 givenname: Rachel orcidid: 0000-0002-4430-0262 surname: Evans fullname: Evans, Rachel organization: Visible Analytics, Oxford, OX2 0DP, UK – sequence: 6 givenname: David Alexander orcidid: 0000-0001-6475-8046 surname: Scott fullname: Scott, David Alexander organization: Visible Analytics, Oxford, OX2 0DP, UK – sequence: 7 givenname: Anadi surname: Mahajan fullname: Mahajan, Anadi organization: Bridge Medical Consulting Ltd., Richmond, London, TW9 2SS, UK – sequence: 8 givenname: Keith R orcidid: 0000-0002-7557-1567 surname: Abrams fullname: Abrams, Keith R organization: Visible Analytics, Oxford, OX2 0DP, UK – sequence: 9 givenname: Neil surname: Hawkins fullname: Hawkins, Neil organization: Visible Analytics, Oxford, OX2 0DP, UK |
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| Title | How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison |
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