Development of Drugs for Nontuberculous Mycobacterial Disease: Clinicians’ Interpretation of a US Food and Drug Administration Workshop

Patrick A. Flume, David E. Griffith, James D. Chalmers, Charles L. Daley, Kenneth Olivier, Anne O'Donnell, Timothy Aksamit, Shannon Kasperbauer, Amy Leitman, Kevin L. Winthrop

Research output: Contribution to journalReview articlepeer-review

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.

Original languageEnglish (US)
Pages (from-to)537-543
Number of pages7
JournalCHEST
Volume159
Issue number2
DOIs
StatePublished - Feb 2021

Keywords

  • clinical trials
  • drug development
  • mycobacteria

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine
  • Critical Care and Intensive Care Medicine
  • Cardiology and Cardiovascular Medicine

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