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000144995 0247_ $$2doi$$a10.1002/pst.1992
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000144995 041__ $$aEnglish
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000144995 1001_ $$0P:(DE-HGF)0$$aBenda, Norbert$$b0$$eCorresponding author
000144995 245__ $$aEnrichment designs using placebo nonresponders.
000144995 260__ $$aNew York, NY$$bWiley$$c2020
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000144995 520__ $$aEnrichment designs that select placebo nonresponders have gained much attention during the last years in areas with high placebo response rates, eg, in depression. Proposals were made that re-randomize patients who did not respond to placebo during a first study phase as the sequential parallel design (SPD). This design uses in a second phase an enriched patient population where the treatment effect is expected to be more pronounced. This may be problematic if an effect in the overall population is claimed. Proposals were made to combine the treatment effects in the overall population from study phase 1 and the enriched population from study phase 2, alleviating but not solving the issue of a potential selection bias. This paper shows how this bias corresponding to the effect difference between the overall population and the enriched population depends on the variability of a potential subject-by-treatment interaction. Sample sizes are given, which lead to a significant result in the combining test with a given probability if actually the average effect in the overall population is zero. If, on the other hand, no subject-by-treatment interaction is given, the enrichment is shown to be inefficient. We conclude that enrichment designs using placebo nonresponders are not able to claim a positive average effect in the overall population if a subject-by-treatment interaction cannot be excluded. It cannot be used to demonstrate positive efficacy in the overall population in a pivotal phase III trial but may be used in early phases to demonstrate varying treatment effects between patients.
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000144995 650_2 $$2MeSH$$aData Interpretation, Statistical
000144995 650_2 $$2MeSH$$aDouble-Blind Method
000144995 650_2 $$2MeSH$$aHumans
000144995 650_2 $$2MeSH$$aModels, Statistical
000144995 650_2 $$2MeSH$$aPlacebo Effect
000144995 650_2 $$2MeSH$$aRandomized Controlled Trials as Topic: statistics & numerical data
000144995 650_2 $$2MeSH$$aResearch Design: statistics & numerical data
000144995 650_2 $$2MeSH$$aTreatment Outcome
000144995 7001_ $$0P:(DE-2719)2810511$$aHänisch, Britta$$b1$$eLast author$$udzne
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000144995 773__ $$0PERI:(DE-600)2083706-9$$a10.1002/pst.1992$$gVol. 19, no. 3, p. 303 - 314$$n3$$p303-314$$tPharmaceutical statistics$$v19$$x1539-1604$$y2020
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000144995 9201_ $$0I:(DE-2719)1013010$$kAG Hänisch ; AG Hänisch$$lPharmacoepidemiology in Neurodegenerative Disorders$$x0
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