@article{b934fe5874eb4cc186de138bde7fd668,
title = "Development of Drugs for Nontuberculous Mycobacterial Disease: Clinicians{\textquoteright} Interpretation of a US Food and Drug Administration Workshop",
abstract = "The US Food and Drug Administration convened a workshop to discuss clinical trial design challenges and considerations related to the treatment of nontuberculous mycobacterial pulmonary disease, to include topics such as clinical trial end points, duration, and populations. The clinicians participating in the meeting provide here their interpretation of the discussion, which included US Food and Drug Administration and industry representatives. The treatment of nontuberculous mycobacterial pulmonary disease typically includes multiple antibiotics for a prolonged period and can be difficult to tolerate; there is a great need for new treatment options. Most individuals have a microbiologic response to therapy, but data correlating decreasing bacillary load with patient-reported outcomes or measured functional improvement are lacking. Accordingly, trial designs for new therapeutic agents should incorporate both microbiologic and clinical outcome measures and select appropriate study candidates with capacity for measurable change of such outcome measures. The need for shorter study designs, early primary end points, and placebo control arms was highlighted during the workshop.",
keywords = "clinical trials, drug development, mycobacteria",
author = "Flume, {Patrick A.} and Griffith, {David E.} and Chalmers, {James D.} and Daley, {Charles L.} and Kenneth Olivier and Anne O'Donnell and Timothy Aksamit and Shannon Kasperbauer and Amy Leitman and Winthrop, {Kevin L.}",
note = "Funding Information: FUNDING/SUPPORT: This publication was supported in part by the Intramural Research Program of the National Heart, Lung, and Blood Institute / National Institutes of Health and in part by the National Center for Advancing Translational Sciences of the National Institutes of Health [Grant UL1 TR001450 ]. Funding Information: Financial/nonfinancial disclosures: The authors have reported to CHEST the following: P. A. F. reports grants and personal fees from Insmed, Savara Pharmaceuticals, and the Cystic Fibrosis Foundation; grants from Novoteris; and personal fees from Janssen Research & Development and from Merck , outside of the submitted work. D. E. G. reports grants, personal fees, and nonfinancial support from Insmed Inc.; and personal fees from Spero, Merck, and Johnson & Johnson, outside of the submitted work. J. D. C. reports grants and personal fees from GlaxoSmithKline , Boehringer Ingelheim, Bayer HealthCare , Grifols, and Insmed; personal fees from Napp and Aradigm Corporation; and grants from AstraZeneca and Gilead Sciences, outside of the submitted work. C. L. D. reports grants from Insmed; personal fees from Insmed, Paratek, Johnson & Johnson, Meiji, Matinas BioPharma, Cipla, and Beyond Air; and grants and personal fees from Spero, outside of the submitted work. K. O. reports grants and nonfinancial support from Beyond Air, as well as grants from Matinas BioPharma, outside of the submitted work; and has also served in an (unpaid) advisory capacity for Merck & Co, Qrumpharma, Spero Therapeutics, AN2 Therapeutics, and Oricula Therapeutics. A. O. reports grants from Insmed and the COPD Foundation; and personal fees from Insmed, Merck, Electromed, and Xellia, outside of the submitted work. S. K. reports personal fees from Insmed, outside of the submitted work. K. L. W. reports grants and personal fees from Insmed; and personal fees from Johnson & Johnson, Paratek, RedHill Biopharma, Horizon, and Spero, outside of the submitted work. None declared (T. A., A. L.). ",
year = "2021",
month = feb,
doi = "10.1016/j.chest.2020.08.2055",
language = "English (US)",
volume = "159",
pages = "537--543",
journal = "Chest",
issn = "0012-3692",
publisher = "American College of Chest Physicians",
number = "2",
}