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Understanding patient preferences and willingness to pay for hemophilia therapies
BACKGROUND: Despite clearly improved clinical outcomes for prophylaxis compared to on-demand therapy, on average only 56% of patients diagnosed with severe hemophilia receive prophylactic factor replacement therapy in the US. Prophylaxis rates generally drop as patients transition from childhood to...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Dove Medical Press
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4646600/ https://www.ncbi.nlm.nih.gov/pubmed/26635471 http://dx.doi.org/10.2147/PPA.S92985 |
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author | Chaugule, Shraddha S Hay, Joel W Young, Guy |
author_facet | Chaugule, Shraddha S Hay, Joel W Young, Guy |
author_sort | Chaugule, Shraddha S |
collection | PubMed |
description | BACKGROUND: Despite clearly improved clinical outcomes for prophylaxis compared to on-demand therapy, on average only 56% of patients diagnosed with severe hemophilia receive prophylactic factor replacement therapy in the US. Prophylaxis rates generally drop as patients transition from childhood to adulthood, partly due to patients becoming less adherent when they reach adulthood. Assessment of patient preferences is important because these are likely to translate into increased treatment satisfaction and adherence. In this study, we assessed preferences and willingness to pay (WTP) for on-demand, prophylaxis, and longer acting prophylaxis therapies in a sample of US hemophilia patients. METHODS: Adult US hemophilia patients and caregivers (N=79) completed a discrete-choice survey that presented a series of trade-off questions, each including a pair of hypothetical treatment profiles. Using a mixed logit model for analysis, we compared the relative importance of five treatment characteristics: 1) out-of-pocket treatment costs (paid by patients), 2) factor dose adjustment, 3) treatment side effects, 4) availability of premixed factor, and 5) treatment effectiveness and dosing frequency. Based on these attribute estimates, we calculated patients’ WTP. RESULTS: Out-of-pocket treatment costs (P<0.001), side effects (P<0.001), and treatment effectiveness and dosing frequency (P<0.001) were found to be statistically significant in the model. Patients were willing to pay US $410 (95% confidence interval: $164–$656) out of pocket per month for thrice-weekly prophylaxis therapy compared to on-demand therapy and $360 (95% confidence interval: $145–$575) for a switch from thrice-weekly to once-weekly prophylaxis therapy. CONCLUSION: Improvements in treatment effectiveness and dosing frequency, treatment side effects, and out-of-pocket costs per month were the greatest determinants of hemophilia treatment choice and WTP. The positive preferences and WTP for longer acting prophylactic therapies suggest that the uptake is likely to increase adherence, improving treatment outcomes. These preferences should also inform the Food and Drug Administration’s assessment of new longer acting hemophilia therapies. |
format | Online Article Text |
id | pubmed-4646600 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2015 |
publisher | Dove Medical Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-46466002015-12-03 Understanding patient preferences and willingness to pay for hemophilia therapies Chaugule, Shraddha S Hay, Joel W Young, Guy Patient Prefer Adherence Original Research BACKGROUND: Despite clearly improved clinical outcomes for prophylaxis compared to on-demand therapy, on average only 56% of patients diagnosed with severe hemophilia receive prophylactic factor replacement therapy in the US. Prophylaxis rates generally drop as patients transition from childhood to adulthood, partly due to patients becoming less adherent when they reach adulthood. Assessment of patient preferences is important because these are likely to translate into increased treatment satisfaction and adherence. In this study, we assessed preferences and willingness to pay (WTP) for on-demand, prophylaxis, and longer acting prophylaxis therapies in a sample of US hemophilia patients. METHODS: Adult US hemophilia patients and caregivers (N=79) completed a discrete-choice survey that presented a series of trade-off questions, each including a pair of hypothetical treatment profiles. Using a mixed logit model for analysis, we compared the relative importance of five treatment characteristics: 1) out-of-pocket treatment costs (paid by patients), 2) factor dose adjustment, 3) treatment side effects, 4) availability of premixed factor, and 5) treatment effectiveness and dosing frequency. Based on these attribute estimates, we calculated patients’ WTP. RESULTS: Out-of-pocket treatment costs (P<0.001), side effects (P<0.001), and treatment effectiveness and dosing frequency (P<0.001) were found to be statistically significant in the model. Patients were willing to pay US $410 (95% confidence interval: $164–$656) out of pocket per month for thrice-weekly prophylaxis therapy compared to on-demand therapy and $360 (95% confidence interval: $145–$575) for a switch from thrice-weekly to once-weekly prophylaxis therapy. CONCLUSION: Improvements in treatment effectiveness and dosing frequency, treatment side effects, and out-of-pocket costs per month were the greatest determinants of hemophilia treatment choice and WTP. The positive preferences and WTP for longer acting prophylactic therapies suggest that the uptake is likely to increase adherence, improving treatment outcomes. These preferences should also inform the Food and Drug Administration’s assessment of new longer acting hemophilia therapies. Dove Medical Press 2015-11-11 /pmc/articles/PMC4646600/ /pubmed/26635471 http://dx.doi.org/10.2147/PPA.S92985 Text en © 2015 Chaugule et al. This work is published by Dove Medical Press Limited, and licensed under Creative Commons Attribution – Non Commercial (unported, v3.0) License The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. |
spellingShingle | Original Research Chaugule, Shraddha S Hay, Joel W Young, Guy Understanding patient preferences and willingness to pay for hemophilia therapies |
title | Understanding patient preferences and willingness to pay for hemophilia therapies |
title_full | Understanding patient preferences and willingness to pay for hemophilia therapies |
title_fullStr | Understanding patient preferences and willingness to pay for hemophilia therapies |
title_full_unstemmed | Understanding patient preferences and willingness to pay for hemophilia therapies |
title_short | Understanding patient preferences and willingness to pay for hemophilia therapies |
title_sort | understanding patient preferences and willingness to pay for hemophilia therapies |
topic | Original Research |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4646600/ https://www.ncbi.nlm.nih.gov/pubmed/26635471 http://dx.doi.org/10.2147/PPA.S92985 |
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