FDA Workshop on the Development of Antibiotic Therapies for NTM

The FDA workshop on “The Development of Antibiotics for the Treatment of NTM Disease” on April 8, 2019 was very successful, with more than 100 people attending in person.

The agenda focused on issues surrounding clinical trials and the development of a validated Patient-Reported Outcome (PRO) tool. Panelists included experts in NTM and in PRO, from noted institutions across the country and from within the FDA.

NTMir represented the patient perspective, delivering remarks which included a report summarizing key findings of the recent survey on patient preferences in treatment outcomes and clinical trial design. We asked for your input as to what symptoms or changes we should be measuring in patients, and you responded! Your responses provided important information that can help develop and refine a PRO tool for use in clinical trials, helping the development of new treatments for NTM disease move forward.

We saw many familiar themes in your feedback, and overwhelmingly you reported fatigue, cough, and shortness of breath among the top symptoms and side effects you are dealing with.

The FDA put enormous thought and effort into convening this workshop, and we are grateful for the cooperative efforts we have seen from patients, clinicians, researchers, industry, and regulators, to make sure the new therapies developed for you help improve your lives and alleviate your illness.

The FDA webcast will be transcribed, and both will be archived on the FDA website. As soon as they are available, we will notify you. Meanwhile, the rest of the meeting materials, including our presentation, can be accessed at the following link: https://www.fda.gov/Drugs/NewsEvents/ucm629494.htm.

Thank you for participating in our surveys and research. Every time you do, it makes a difference, and we were proud to be your voice on April 8th, knowing the information we imparted was so impactful to the day’s work.

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