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Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey
OBJECTIVES: The aim of the Patient preferences to Assess Value IN Gene therapies (PAVING) study was to investigate trade‐offs that adult Belgian people with haemophilia (PWH) A and B are willing to make when choosing between prophylactic factor replacement therapy (PFRT) and gene therapy. METHODS: T...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9293173/ https://www.ncbi.nlm.nih.gov/pubmed/34472162 http://dx.doi.org/10.1111/hae.14401 |
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author | van Overbeeke, Eline Hauber, Brett Michelsen, Sissel Peerlinck, Kathelijne Lambert, Catherine Hermans, Cedric Lê, Phu Quoc Goldman, Michel Simoens, Steven Huys, Isabelle |
author_facet | van Overbeeke, Eline Hauber, Brett Michelsen, Sissel Peerlinck, Kathelijne Lambert, Catherine Hermans, Cedric Lê, Phu Quoc Goldman, Michel Simoens, Steven Huys, Isabelle |
author_sort | van Overbeeke, Eline |
collection | PubMed |
description | OBJECTIVES: The aim of the Patient preferences to Assess Value IN Gene therapies (PAVING) study was to investigate trade‐offs that adult Belgian people with haemophilia (PWH) A and B are willing to make when choosing between prophylactic factor replacement therapy (PFRT) and gene therapy. METHODS: The threshold technique was used to quantify the minimum acceptable benefit (MAB) of a switch from PFRT to gene therapy in terms of ‘Annual bleeding rate’ (ABR), ‘Chance to stop prophylaxis’ (STOP), and ‘Quality of life’ (QOL). The design was supported by stakeholder involvement and included an educational tool on gene therapy. Threshold intervals were analysed using interval regression models in Stata 16. RESULTS: A total of 117 PWH completed the survey. Mean thresholds were identified for all benefits, but substantial preference heterogeneity was observed; especially for the STOP thresholds, where the distribution of preferences was bimodal. Time spent on the educational tool and residence were found to impact MAB thresholds. The most accepted (88% of PWH) gene therapy profile investigated in this study comprised of zero bleeds per year (vs. six for PFRT), 90% chance to stop prophylaxis, no impact on QoL, and 10 years of follow‐up on side effects (vs. 30 for PFRT). CONCLUSIONS: Results from this study proved the value of educating patients on novel treatments. Moreover, preference heterogeneity for novel treatments was confirmed in this study. In gene therapy decision‐making, preference heterogeneity and the impact of patient education on acceptance should be considered. |
format | Online Article Text |
id | pubmed-9293173 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-92931732022-07-20 Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey van Overbeeke, Eline Hauber, Brett Michelsen, Sissel Peerlinck, Kathelijne Lambert, Catherine Hermans, Cedric Lê, Phu Quoc Goldman, Michel Simoens, Steven Huys, Isabelle Haemophilia Original Articles OBJECTIVES: The aim of the Patient preferences to Assess Value IN Gene therapies (PAVING) study was to investigate trade‐offs that adult Belgian people with haemophilia (PWH) A and B are willing to make when choosing between prophylactic factor replacement therapy (PFRT) and gene therapy. METHODS: The threshold technique was used to quantify the minimum acceptable benefit (MAB) of a switch from PFRT to gene therapy in terms of ‘Annual bleeding rate’ (ABR), ‘Chance to stop prophylaxis’ (STOP), and ‘Quality of life’ (QOL). The design was supported by stakeholder involvement and included an educational tool on gene therapy. Threshold intervals were analysed using interval regression models in Stata 16. RESULTS: A total of 117 PWH completed the survey. Mean thresholds were identified for all benefits, but substantial preference heterogeneity was observed; especially for the STOP thresholds, where the distribution of preferences was bimodal. Time spent on the educational tool and residence were found to impact MAB thresholds. The most accepted (88% of PWH) gene therapy profile investigated in this study comprised of zero bleeds per year (vs. six for PFRT), 90% chance to stop prophylaxis, no impact on QoL, and 10 years of follow‐up on side effects (vs. 30 for PFRT). CONCLUSIONS: Results from this study proved the value of educating patients on novel treatments. Moreover, preference heterogeneity for novel treatments was confirmed in this study. In gene therapy decision‐making, preference heterogeneity and the impact of patient education on acceptance should be considered. John Wiley and Sons Inc. 2021-09-01 2021-11 /pmc/articles/PMC9293173/ /pubmed/34472162 http://dx.doi.org/10.1111/hae.14401 Text en © 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
spellingShingle | Original Articles van Overbeeke, Eline Hauber, Brett Michelsen, Sissel Peerlinck, Kathelijne Lambert, Catherine Hermans, Cedric Lê, Phu Quoc Goldman, Michel Simoens, Steven Huys, Isabelle Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey |
title | Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey |
title_full | Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey |
title_fullStr | Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey |
title_full_unstemmed | Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey |
title_short | Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey |
title_sort | patient preferences for gene therapy in haemophilia: results from the paving threshold technique survey |
topic | Original Articles |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9293173/ https://www.ncbi.nlm.nih.gov/pubmed/34472162 http://dx.doi.org/10.1111/hae.14401 |
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