Journal of Biometrics & Biostatistics

Ascendis Pharma Inc., Palo Alto, CA, USA

Publications

• Research Article   
  Developing and then Confirming a Hypothesis Based on a Chronology of Several Clinical Trials: A Bayesian Application to Pirfenidone Mortality Results
  Author(s): Zhengning Lin* and Donald A Berry
  
  Background: Designing a study for independent confirmation of a treatment effect is sometimes not practical due to required large sample size. Post hoc pooling of studies including those for learning purposes is subject to selection bias and therefore not scientifically solid. We propose a Bayesian approach which calibrates the role of prior information from historical studies for learning and confirming purposes. The method is illustrated in the analysis of mortality data for the pirfenidone NDA. Methods: The pirfenidone NDA includes three placebo-controlled studies to demonstrate efficacy for idiopathic pulmonary fibrosis (IPF), a rare and ultimately fatal lung disease with no approved treatment in the US at the time of NDA. The results of two earlier conducted studies PIPF-004 and PIPF-006 suggested that pirfenidone might reduce mortality.. Read More»
  DOI: 10.37421/2155-6180.2020.11.442

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