2004
DOI: 10.1002/sim.1808
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Adaptive urn designs for estimating several percentiles of a dose–response curve

Abstract: Dose--response experiments are crucial in biomedical studies. There are usually multiple objectives in such experiments and among the goals is the estimation of several percentiles on the dose--response curve. Here we present the first non-parametric adaptive design approach to estimate several percentiles simultaneously via generalized Pólya urns. Theoretical properties of these designs are investigated and their performance is gaged by the locally compound optimal designs. As an example, we re-investigated a… Show more

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Cited by 12 publications
(5 citation statements)
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“…There are several important methodologic obstacles to drug development in this area that might be best amenable to expert consensus on the: (1) definition of A/A for inclusion; (2) choice of primary outcome; (3) role of adaptive designs to minimize exposure to a drug or placebo that is “not working” (Mugno et al , 2004; Kairalla et al , 2012); (4) standardization of non-pharmacological person-centered interventions (Gitlin et al , 2012) in both arms; and (5) placebo run-in-period prior to randomization. Once eligibility is confirmed at baseline, only patients with persisting A/A or at higher levels of A/A severity would participate in the treatment period.…”
Section: Discussionmentioning
confidence: 99%
“…There are several important methodologic obstacles to drug development in this area that might be best amenable to expert consensus on the: (1) definition of A/A for inclusion; (2) choice of primary outcome; (3) role of adaptive designs to minimize exposure to a drug or placebo that is “not working” (Mugno et al , 2004; Kairalla et al , 2012); (4) standardization of non-pharmacological person-centered interventions (Gitlin et al , 2012) in both arms; and (5) placebo run-in-period prior to randomization. Once eligibility is confirmed at baseline, only patients with persisting A/A or at higher levels of A/A severity would participate in the treatment period.…”
Section: Discussionmentioning
confidence: 99%
“…For adaptive dose finding design, the method of continual re-assessment method (CRM) in conjunction with Bayesian approach is usually considered [ 34 - 36 ]. Mugno et al (2004) introduced a nonparametric adaptive urn design approach for estimating a dose-response curve [ 37 ]. More details regarding PhRMA's current position and proposed statistical methods, the reader should consult with a special issue recently published at the Journal of Biopharmaceutical Statistics , Vol.…”
Section: What Is Adaptive Design?mentioning
confidence: 99%
“…ROBBINS-MONRO YÖNTEMİ [25][26][27][28] Yöntem, yanıt eğrisine ait herhangi bir yüzdelik değerin tahmin edilmesinde tahmin edilmek istenilen yüzdelik değere yakınsayan bir noktalar dizisi oluşturmaktadır. Yönteme göre amaç, y(x) yanıt fonksiyonu ve F(θ) = p, 0 < p < 1 koşulunu sağlayan F(x) bilinmeyen dağılım fonksiyonu olmak üzere p olasılık değerine sahip, θ'nın değerini tahmin etmektir.…”
Section: % P ) öLümunclassified