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Accelerated Approval or Risk Reduction? How Response Biomarkers Advance Therapeutics through Clinical Trials in Cystic Fibrosis.
Mayer-Hamblett, N; VanDevanter, D R.
Afiliação
  • Mayer-Hamblett N; University of Washington, Seattle, WA, USA; Seattle Children's Hospital, Seattle, WA, USA. Electronic address: nicole.hamblett@seattlechildrens.org.
  • VanDevanter DR; Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Trends Mol Med ; 26(12): 1068-1077, 2020 12.
Article em En | MEDLINE | ID: mdl-32868171
ABSTRACT
Progress in the development of new therapies for cystic fibrosis (CF) has benefited from therapeutically responsive biomarkers to streamline drug development. Paradoxically, these response biomarkers have been proven to be essential even in the presence of data demonstrating a lack of correlation with clinical outcomes across individuals with CF. This finding is unsurprising, particularly in the setting of a rare disease given complex disease processes and an often-limited pool of clinically effective therapies by which to link biomarkers and clinical responsiveness. While many response biomarkers will be unable to progress from their status as markers of biological efficacy to either established correlates of clinical efficacy or surrogate endpoints, they remain critical to the overall success of therapeutic development.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Aprovação de Drogas / Fibrose Cística / Comportamento de Redução do Risco Tipo de estudo: Etiology_studies / Prognostic_studies / Risk_factors_studies Limite: Humans Idioma: En Revista: Trends Mol Med Assunto da revista: BIOLOGIA MOLECULAR Ano de publicação: 2020 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Aprovação de Drogas / Fibrose Cística / Comportamento de Redução do Risco Tipo de estudo: Etiology_studies / Prognostic_studies / Risk_factors_studies Limite: Humans Idioma: En Revista: Trends Mol Med Assunto da revista: BIOLOGIA MOLECULAR Ano de publicação: 2020 Tipo de documento: Article