A comparison of approaches to incorporate patient-selected and patient-ranked outcomes in clinical trials
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arXiv
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| Auteurs principaux: | , |
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| Format: | Preprint |
| Publié: |
2025
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| _version_ | 1866908589726105600 |
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| author | Robertson, David S. Jaki, Thomas |
| author_facet | Robertson, David S. Jaki, Thomas |
| contents | A key aspect of patient-focused drug development is identifying and measuring outcomes that are important to patients in clinical trials. Many medical conditions affect multiple symptom domains, and a consensus approach to determine the relative importance of the associated multiple outcomes ignores the heterogeneity in individual patient preferences. Patient-selected outcomes offer one way to incorporate individual patient preferences, as proposed in recent regulatory guidance for the treatment for migraine, where each patient selects their most bothersome migraine-associated symptom in addition to pain. Patient-ranked outcomes have also recently been proposed, which go further and consider the full ranking of the relative importance of all the outcomes. This can be assessed using a composite DOOR (Desirability of Outcome Ranking) endpoint. In this paper, we compare the advantages and disadvantages of using patient-selected versus patient-ranked outcomes in the context of a two-arm randomised controlled trial for multiple sclerosis. We compare the power and type I error rate by simulation, and discuss several other important considerations when using the two approaches. |
| format | Preprint |
| id |
arxiv_https___arxiv_org_abs_2510_11578 |
| institution | arXiv |
| publishDate | 2025 |
| record_format | arxiv |
| spellingShingle | A comparison of approaches to incorporate patient-selected and patient-ranked outcomes in clinical trials Robertson, David S. Jaki, Thomas Methodology 62P10 A key aspect of patient-focused drug development is identifying and measuring outcomes that are important to patients in clinical trials. Many medical conditions affect multiple symptom domains, and a consensus approach to determine the relative importance of the associated multiple outcomes ignores the heterogeneity in individual patient preferences. Patient-selected outcomes offer one way to incorporate individual patient preferences, as proposed in recent regulatory guidance for the treatment for migraine, where each patient selects their most bothersome migraine-associated symptom in addition to pain. Patient-ranked outcomes have also recently been proposed, which go further and consider the full ranking of the relative importance of all the outcomes. This can be assessed using a composite DOOR (Desirability of Outcome Ranking) endpoint. In this paper, we compare the advantages and disadvantages of using patient-selected versus patient-ranked outcomes in the context of a two-arm randomised controlled trial for multiple sclerosis. We compare the power and type I error rate by simulation, and discuss several other important considerations when using the two approaches. |
| title | A comparison of approaches to incorporate patient-selected and patient-ranked outcomes in clinical trials |
| topic | Methodology 62P10 |
| url | https://arxiv.org/abs/2510.11578 |