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Development of novel agents for idiopathic pulmonary fibrosis: progress in target selection and clinical trial design.
Chest 2015; 148(4):1083-1092Chest

Abstract

Idiopathic pulmonary fibrosis (IPF) is a progressive, fatal disease. Until recently, the standard therapy for this disease has been essentially supportive, with the exception of a minority of patients who were eligible for lung transplantation. The development pathway for novel medications for IPF has been complicated. There have been several challenges, including an incomplete understanding of the pathogenesis, unpredictable clinical course, lack of validated biomarkers, the low clinical predictive value of animal models of lung injury, and the need to commit to large clinical trials of long duration to obtain initial evidence of clinical efficacy. Despite these challenges, the combination of recent advances in translational medicine and the unprecedented increase in clinical data accumulated from recent large clinical trials has stimulated an increase in the number of clinical development programs for IPF. Clinical programs are increasingly characterized by rational target selection, preclinical optimization of therapeutic molecules, and an emphasis on efficient clinical trial design. A lower rate of functional decline in patients treated with pirfenidone and nintedanib was demonstrated in large clinical trials. In October 2014, these two drugs became the first agents to be approved by the US Food and Drug Administration for the treatment of IPF. (Pirfenidone had already been approved in several countries outside the United States.) In November 2014, the European Medicines Agency approved the use of nintedanib for IPF. The landscape for management of IPF has markedly changed with the advent of approved therapeutic options for IPF. In this article, we review the strategies that are being used to increase the likelihood of success in clinical development programs of novel disease-modifying agents in IPF.

Authors+Show Affiliations

Gilead Sciences Inc, Seattle, WA.Gilead Sciences Inc, Foster City, CA.Center for Interstitial Lung Disease, University of Washington, Seattle, WA. Electronic address: graghu@uw.edu.

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

26020856

Citation

O'Riordan, Thomas G., et al. "Development of Novel Agents for Idiopathic Pulmonary Fibrosis: Progress in Target Selection and Clinical Trial Design." Chest, vol. 148, no. 4, 2015, pp. 1083-1092.
O'Riordan TG, Smith V, Raghu G. Development of novel agents for idiopathic pulmonary fibrosis: progress in target selection and clinical trial design. Chest. 2015;148(4):1083-1092.
O'Riordan, T. G., Smith, V., & Raghu, G. (2015). Development of novel agents for idiopathic pulmonary fibrosis: progress in target selection and clinical trial design. Chest, 148(4), pp. 1083-1092. doi:10.1378/chest.14-3218.
O'Riordan TG, Smith V, Raghu G. Development of Novel Agents for Idiopathic Pulmonary Fibrosis: Progress in Target Selection and Clinical Trial Design. Chest. 2015;148(4):1083-1092. PubMed PMID: 26020856.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Development of novel agents for idiopathic pulmonary fibrosis: progress in target selection and clinical trial design. AU - O'Riordan,Thomas G, AU - Smith,Victoria, AU - Raghu,Ganesh, PY - 2015/5/29/entrez PY - 2015/5/29/pubmed PY - 2016/1/8/medline SP - 1083 EP - 1092 JF - Chest JO - Chest VL - 148 IS - 4 N2 - Idiopathic pulmonary fibrosis (IPF) is a progressive, fatal disease. Until recently, the standard therapy for this disease has been essentially supportive, with the exception of a minority of patients who were eligible for lung transplantation. The development pathway for novel medications for IPF has been complicated. There have been several challenges, including an incomplete understanding of the pathogenesis, unpredictable clinical course, lack of validated biomarkers, the low clinical predictive value of animal models of lung injury, and the need to commit to large clinical trials of long duration to obtain initial evidence of clinical efficacy. Despite these challenges, the combination of recent advances in translational medicine and the unprecedented increase in clinical data accumulated from recent large clinical trials has stimulated an increase in the number of clinical development programs for IPF. Clinical programs are increasingly characterized by rational target selection, preclinical optimization of therapeutic molecules, and an emphasis on efficient clinical trial design. A lower rate of functional decline in patients treated with pirfenidone and nintedanib was demonstrated in large clinical trials. In October 2014, these two drugs became the first agents to be approved by the US Food and Drug Administration for the treatment of IPF. (Pirfenidone had already been approved in several countries outside the United States.) In November 2014, the European Medicines Agency approved the use of nintedanib for IPF. The landscape for management of IPF has markedly changed with the advent of approved therapeutic options for IPF. In this article, we review the strategies that are being used to increase the likelihood of success in clinical development programs of novel disease-modifying agents in IPF. SN - 1931-3543 UR - https://www.unboundmedicine.com/medline/citation/26020856/Development_of_novel_agents_for_idiopathic_pulmonary_fibrosis:_progress_in_target_selection_and_clinical_trial_design_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0012-3692(15)50298-5 DB - PRIME DP - Unbound Medicine ER -