2010-19044. Design of Clinical Trials of Aerosolized Antimicrobials for the Treatment of Cystic Fibrosis; Public Workshop

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice of public workshop.

SUMMARY:

The Food and Drug Administration (FDA) is announcing a public workshop regarding scientific issues in clinical development of aerosolized antimicrobials for the management and/or treatment of patients with cystic fibrosis. Aerosolized antimicrobials are used to treat chronic bacterial infection in the lungs and thus improve the respiratory symptoms in patients with cystic fibrosis. This public workshop is intended to provide information for and gain perspective from health care providers, patients and patient advocacy organizations, academia, and industry on various aspects of the design of clinical trials of aerosolized antimicrobials in patients with cystic fibrosis. The input from this public workshop will help in developing topics for further discussion.

Dates and Times: The public workshop will be held on September 23, 2010, from 8:30 a.m. to 5:30 p.m. and on September 24, 2010, from 8 a.m. to 4 p.m.

Location: The public workshop will be held at the Crowne Plaza Hotel,8777 Georgia Ave., Silver Spring, MD 20910. Seating is limited and available only on a first-come, first-served basis.

Contact Persons: Chris Moser or Lori Benner, Center for Drug Evaluation and Research,Food and Drug Administration, Office of Antimicrobial Products, 10903 New Hampshire Ave., Bldg. 22, rm. 6209, Silver Spring, MD 20993-0002, 301-796-1300.

Registration: Registration is free for the public workshop. Interested parties are encouraged to register early because space is limited. Seating will be available on a first-come, first-served basis. To register electronically, e-mail registration information (including name, title, firm name, address, telephone, and fax number) to [e-mail] Persons without access to the Internet can call 301-796-1300 to register. Persons needing a sign language interpreter or other special accommodations should notify Christine Moser or Lori Benner (see Contact Persons) at least 7 days in advance.

SUPPLEMENTARY INFORMATION:

FDA is announcing a public workshop regarding scientific considerations in the design of clinical trials of aerosolized antimicrobials to treat chronic bacterial infection in the lungs and thus improve the respiratory symptoms in patients with cystic fibrosis. The development of clinical trial endpoints to establish efficacy is a major challenge in the design of informative clinical trials of aerosolized antimicrobials for the management and/or treatment of patients with cystic fibrosis. The workshop will include discussion of clinical trial endpoints to establish efficacy, such as timing and definitions of pulmonary exacerbations, changes in the results of pulmonary function testing, and changes on patient reported outcome measures. An important consideration will be the evaluation of new aerosolized antimicrobials in the context of approved aerosolized antimicrobials on the basis of these or other efficacy endpoints. Other issues in the design of clinical trials of aerosolized antimicrobials include: The development of drug resistance and other safety concerns, microbiologic testing such as sputum bacterial density, the utility of in-vitro susceptibility testing, and the need for pediatric use information.

The agency encourages individuals, patient advocates, industry, consumer groups, health care professionals, researchers, and other interested persons to attend this public workshop.

Transcripts: Please be advised that as soon as a transcript is available, it will be accessible at http://www.regulations.gov. It may be viewed at the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD. A transcript will also be available in either hardcopy or on CD-ROM, after submission of a Freedom of Information request. Written requests are to be sent to Division of Freedom of Information (HFI-35), Office of Management Programs, Food and Drug Administration, 5600 Fishers Lane, rm. 6-30, Rockville, MD 20857. Transcripts will also be available on the Internet http://www.fda.gov/​Drugs/​NewsEvents/​ucm205809.htm approximately 45 days after the workshop.

Dated: July 29, 2010.

Leslie Kux,

Acting Assistant Commissioner for Policy.