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Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study.
Holzhauer, Björn; Hampson, Lisa V; Gosling, John Paul; Bornkamp, Björn; Kahn, Joseph; Lange, Markus R; Luo, Wen-Lin; Brindicci, Caterina; Lawrence, David; Ballerstedt, Steffen; O'Hagan, Anthony.
Afiliação
  • Holzhauer B; Global Drug Development, Novartis Pharma AG, Basel, Switzerland.
  • Hampson LV; Global Drug Development, Novartis Pharma AG, Basel, Switzerland.
  • Gosling JP; Department of Mathematical Sciences, Durham University, Durham, UK.
  • Bornkamp B; Global Drug Development, Novartis Pharma AG, Basel, Switzerland.
  • Kahn J; Global Drug Development, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
  • Lange MR; Global Drug Development, Novartis Pharma AG, Basel, Switzerland.
  • Luo WL; Global Drug Development, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
  • Brindicci C; Global Drug Development, Novartis Pharma AG, Basel, Switzerland.
  • Lawrence D; Global Drug Development, Novartis Pharma AG, Basel, Switzerland.
  • Ballerstedt S; Global Drug Development, Novartis Pharma AG, Basel, Switzerland.
  • O'Hagan A; School of Mathematics and Statistics, The University of Sheffield, Sheffield, UK.
Pharm Stat ; 21(5): 1005-1021, 2022 09.
Article em En | MEDLINE | ID: mdl-35373454
Pharmaceutical companies regularly need to make decisions about drug development programs based on the limited knowledge from early stage clinical trials. In this situation, eliciting the judgements of experts is an attractive approach for synthesising evidence on the unknown quantities of interest. When calculating the probability of success for a drug development program, multiple quantities of interest-such as the effect of a drug on different endpoints-should not be treated as unrelated. We discuss two approaches for establishing a multivariate distribution for several related quantities within the SHeffield ELicitation Framework (SHELF). The first approach elicits experts' judgements about a quantity of interest conditional on knowledge about another one. For the second approach, we first elicit marginal distributions for each quantity of interest. Then, for each pair of quantities, we elicit the concordance probability that both lie on the same side of their respective elicited medians. This allows us to specify a copula to obtain the joint distribution of the quantities of interest. We show how these approaches were used in an elicitation workshop that was performed to assess the probability of success of the registrational program of an asthma drug. The judgements of the experts, which were obtained prior to completion of the pivotal studies, were well aligned with the final trial results.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Asma / Desenvolvimento de Medicamentos Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Revista: Pharm Stat Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Asma / Desenvolvimento de Medicamentos Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Revista: Pharm Stat Ano de publicação: 2022 Tipo de documento: Article