دورية أكاديمية
Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls.
| العنوان: | Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls. |
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| المؤلفون: | Muntoni, Francesco, Signorovitch, James, Sajeev, Gautam, Goemans, Nathalie, Wong, Brenda, Tian, Cuixia, Mercuri, Eugenio, Done, Nicolae, Wong, Hallee, Moss, Jackson, Yao, Zhiwen, Ward, Susan J, Manzur, Adnan, Servais, Laurent, Niks, Erik H, Straub, Volker, de Groot, Imelda Jm, McDonald, Craig, North Star Clinical Network |
| المصدر: | Neuromuscular Disorders, 32 (4), 271-283 (2022-02-25) |
| بيانات النشر: | Elsevier Ltd, 2022. |
| سنة النشر: | 2022 |
| مصطلحات موضوعية: | Clinical trials, Drug evaluation, Duchenne muscular dystrophy, External controls, Natural history data, Real-world data, Pediatrics, Perinatology and Child Health, Neurology, Neurology (clinical), Genetics (clinical), Human health sciences, Pediatrics, Sciences de la santé humaine, Neurologie, Pédiatrie |
| الوصف: | Using external controls based on real-world or natural history data (RWD/NHD) for drug evaluations in Duchenne muscular dystrophy (DMD) is appealing given the challenges of enrolling placebo-controlled trials, especially for multi-year trials. Comparisons to external controls, however, face risks of bias due to differences in outcomes between trial and RWD/NHD settings. To assess this bias empirically, we conducted a multi-institution study comparing mean 48-week changes in North Star Ambulatory Assessment (NSAA) total score between trial placebo arms and RWD/NHD sources, with and without adjustment for baseline prognostic factors. Analyses used data from three placebo arms (235 48-week intervals, N = 235 patients) and three RWD/NHD sources (348 intervals, N = 202 patients). Differences in mean ΔNSAA between placebo arms and RWD/NHD sources were small before adjustment (-1.2 units, 95% CI: [-2.0 -0.5]) and were attenuated and no longer statistically significant after adjustment (0.1 units (95% CI: [-0.6, 0.8]). Results were similar whether adjusting using multivariable regression or propensity score matching. This consistency in ΔNSAA between trial placebo arms and RWD/NHD sources accords with prior findings for the six-minute walk distance, provides a well-validated framework for baseline adjustment of prognostic factors, and supports the suitability of RWD/NHD external controls for drug evaluations in ambulatory DMD. |
| نوع الوثيقة: | journal article http://purl.org/coar/resource_type/c_6501Test article |
| اللغة: | English |
| العلاقة: | https://api.elsevier.com/content/article/PII:S096089662200061X?httpAccept=text/xmlTest; urn:issn:0960-8966; urn:issn:1873-2364 |
| DOI: | 10.1016/j.nmd.2022.02.009 |
| الوصول الحر: | https://orbi.uliege.be/handle/2268/289970Test |
| حقوق: | restricted access http://purl.org/coar/access_right/c_16ecTest info:eu-repo/semantics/restrictedAccess |
| رقم الانضمام: | edsorb.289970 |
| قاعدة البيانات: | ORBi |
| DOI: | 10.1016/j.nmd.2022.02.009 |
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