{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,25]],"date-time":"2026-04-25T17:42:18Z","timestamp":1777138938267,"version":"3.51.4"},"reference-count":31,"publisher":"Springer Science and Business Media LLC","issue":"3","license":[{"start":{"date-parts":[[2026,3,9]],"date-time":"2026-03-09T00:00:00Z","timestamp":1773014400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2026,3,9]],"date-time":"2026-03-09T00:00:00Z","timestamp":1773014400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"name":"Takeda Development Center Americas Inc."}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ther Innov Regul Sci"],"published-print":{"date-parts":[[2026,5]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Quantitative benefit-risk assessment (qBRA) can reveal how patients balance benefits and risks of cancer treatments. To align with qBRA good practice guidelines, researchers must address challenges including attribute value dependence, double counting, attribute dominance, and uncertainty associated with immature clinical trial outcomes. We present a case study illustrating these challenges in a qBRA of treatment preferences among patients with Philadelphia chromosome-positive acute lymphoblastic leukemia. Preferences were elicited using a discrete choice experiment (DCE). First, we explain how we mitigated potential dominance of survival outcomes by narrowing the range of overall survival (OS) durations that each participant considered. Second, we describe how we acknowledged the conceptual interaction between OS and duration of remission (DOR) attributes and tested for a statistical interaction. Third, we detail how we conducted qBRA with uncertain efficacy data using bivariate sensitivity analysis. Bivariate sensitivity analysis based on DCE-elicited preferences and head-to-head clinical performance data showed that if the considered treatments \u2013 ponatinib\u2009+\u2009chemotherapy and imatinib\u2009+\u2009chemotherapy \u2013 had equivalent efficacy, 52.9% (95% CI: 52.5%\u201353.4%) of DCE participants would be expected to choose ponatinib over imatinib. If ponatinib offered 10-month longer DOR and 20-month longer OS vs. imatinib, 71.6% (95% CI: 67.2%\u201376.0%) would choose ponatinib. Probabilistic sensitivity analyses showed that the probability of\u2009 \u2265\u200970% of patients preferring ponatinib is 77.5% if ponatinib offers 15-month longer OS and DOR and 93.0% if it offers 45-month longer OS and DOR. Preference heterogeneity analyses identified that the overall choice probability results hold for all subgroups in nearly all scenarios.<\/jats:p>","DOI":"10.1007\/s43441-026-00935-x","type":"journal-article","created":{"date-parts":[[2026,3,9]],"date-time":"2026-03-09T13:23:31Z","timestamp":1773062611000},"page":"617-628","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Challenges in Conducting Quantitative Patient-Centered Benefit-Risk Assessments: A Case Study in Ph\u2009+\u2009ALL with Immature Efficacy Data"],"prefix":"10.1007","volume":"60","author":[{"given":"Ajibade","family":"Ashaye","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Caitlin","family":"Thomas","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vamsi","family":"Kota","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nicolas","family":"Krucien","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kevin","family":"Marsh","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2026,3,9]]},"reference":[{"issue":"2","key":"935_CR1","doi-asserted-by":"publisher","first-page":"415","DOI":"10.1007\/s43441-020-00230-3","volume":"55","author":"MY Smith","year":"2021","unstructured":"Smith MY, van Til J, DiSantostefano RL, et al. Quantitative benefit\u2013risk assessment: state of the practice within industry. Ther Innov Regul Sci. 2021;55(2):415\u201325. https:\/\/doi.org\/10.1007\/s43441-020-00230-3.","journal-title":"Ther Innov Regul Sci"},{"issue":"4","key":"935_CR2","doi-asserted-by":"publisher","first-page":"449","DOI":"10.1016\/j.jval.2022.12.006","volume":"26","author":"T Tervonen","year":"2023","unstructured":"Tervonen T, Veldwijk J, Payne K, et al. Quantitative benefit-risk assessment in medical product decision making: a good practices report of an ISPOR task force. Value Health. 2023;26(4):449\u201360. https:\/\/doi.org\/10.1016\/j.jval.2022.12.006.","journal-title":"Value Health"},{"key":"935_CR3","unstructured":"International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). ICH harmonised guideline: General considerations for patient preference studies. E22. (Draft version). 2025. Updated 19 November 2025. Accessed 19 January 2026. 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CT is an employee of PPD Evidera Patient-Centered Research, part of the PPD clinical research business of Thermo Fisher Scientific, which received funding from Takeda to conduct the work reported in this manuscript. VK is employed by the Section of Hematology and Oncology, Georgia Cancer Center at Augusta University, Augusta, GA, USA, and was paid by Takeda Development Center Americas Inc. for work related to this study. NK is an employee of PPD Evidera Patient-Centered Research, part of the PPD clinical research business of Thermo Fisher Scientific, which received funding from Takeda to conduct the work reported in this manuscript. NK is a minority shareholder of Thermo Fisher Scientific as part of his employment with PPD Evidera Patient-Centered Research. KM is an employee of PPD Evidera Patient-Centered Research, part of the PPD clinical research business of Thermo Fisher Scientific, which received funding from Takeda to conduct the work reported in this manuscript. KM is a minority shareholder of Thermo Fisher Scientific as part of his employment with PPD Evidera Patient-Centered Research.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interests"}},{"value":"The preference study described in this article was approved by Salus IRB ([formerly Ethical and Independent Review Services]; MO, USA; 19162\u201301) and performed in accordance with the ethical standards as laid down in the 1964 Declaration of Helsinki and its later amendments or comparable ethical standards.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}