Open AccessModel‐based Meta‐Analysis on the Efficacy of Pharmacological Treatments for Idiopathic Pulmonary Fibrosis
Author(s) -
Chan Phyllis,
Bax Leon,
Chen Chunlin,
Zhang Nancy,
Huang ShuPang,
Soares Holly,
Rosen Glenn,
AbuTarif Malaz
Publication year - 2017
Publication title -
cpt: pharmacometrics and systems pharmacology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.53
H-Index - 37
ISSN - 2163-8306
DOI - 10.1002/psp4.12227
Subject(s) - pirfenidone , nintedanib , medicine , idiopathic pulmonary fibrosis , vital capacity , placebo , covariate , food and drug administration , clinical trial , leverage (statistics) , pooled analysis , meta analysis , pharmacology , statistics , pathology , alternative medicine , lung function , mathematics , lung , diffusing capacity
Recently, the US Food and Drug Administration (FDA) approved the first two drugs (pirfenidone and nintedanib) indicated for the treatment of idiopathic pulmonary fibrosis (IPF). The purpose of this analysis was to leverage publicly available data to quantify comparative efficacy of compounds that are approved or in development. An analysis‐ready database was developed, and the analysis dataset is composed of summary‐level data from 43 arms in 20 trials, with treatment durations ranging from 8–104 weeks. A hierarchical multivariable regression model with nonparametric placebo estimation was used to fit the longitudinal profile of change from baseline of percent predicted forced vital capacity (%predicted FVC) data. Pirfenidone and nintedanib were the only drugs identified to have significant estimated positive treatment effects. Model simulations were performed to further evaluate the covariate and time course of treatment effects on longitudinal change from baseline %predicted FVC to inform future trial designs and support decision making.