Date: October 15, 2015
Time: 9:00 a.m. to 5:00 p.m.
Location: FDA White Oak Campus
10903 New Hampshire Ave.
Building 31 Conference Center, The Great Room (Rm 1503 B+C)
Silver Spring, MD 20993
(Enter at Building 1 to clear security)
On October 15, 2015, FDA is conducting a public meeting on Patient-Focused Drug Development for nontuberculous mycobacterial (NTM) lung infections. Patient-Focused Drug Development is part of FDA’s performance commitments made as part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to allow FDA to obtain patient perspectives on the impact of NTM lung infections on daily life and patient views on treatment approaches. FDA is also interested in discussing issues related to scientific challenges in developing drugs to treat NTM lung infections. Common causes of NTM lung infections include Mycobacterium avium-intracellulare and M. abscessus. The questions for discussion on these topics are below.
In the afternoon, FDA will hold a workshop and provide information for and gain perspective from patients and patient advocacy organizations, health care providers, academic experts, and industry on various aspects of clinical development of drug products intended to treat NTM lung infections. The innput from this public meeting will help in developing topics for further discussion. Discussion topics for the afternoon will include the following: epidemiology and natural history of NTM lung infections, current treatment considerations, clinical trial designs, and clinical trial endpoints.
For each of these topics, a panel of patients and patient representatives/advocates will present comments to begin the dialogue and will be followed by a facilitated discussion inviting comments from all patients and patient representatives in the audience.
If you are interested in providing comments as part of the initial panel discussion, indicate so during the registration process. Potential panelists must send a brief summary of responses to the discussion questions below to PatientFocused@fda.hhs.gov. Panelists will be confirmed prior to the meeting.
There will also be an opportunity for patients, patient representatives and others to provide comments on issues other than topics 1 and 2 during an Open Public Comment session. Sign-up for Open Public Comment will take place the day of the meeting.
Webcast participants will also have an opportunity to provide input through webcast comments.
For more information, refer to the FDA meeting website at: http://www.fda.gov/Drugs/NewsEvents/ucm453877.htm
Topic 1: Disease symptoms and daily impacts that matter most to patients
- Of all the symptoms that you experience because of your condition, which 1-3 symptoms have the most significant impact on your life? (Examples may include cough, increased sputum production, shortness of breath, difficulty breathing, chest pain)
- Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition? (Examples of activities may include sleeping through the night, daily hygiene, driving, walking/running, exercising, etc.)
- How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days? (Examples may include limitations on the ability to undertake physically strenuous activities, restrictions on the ability to travel, inability to sleep, lack of appetite, fatigue, etc.)
- How has your condition and its symptoms changed over time?
- Do your symptoms come and go? If so, do you know of anything that makes your symptoms better? Worse?
- What worries you most about your condition?
Topic 2: Patients’ perspectives on current approaches to treating NTM lung infections
- What are you currently doing to help treat your condition or its symptoms? (Examples may include prescription medicines, over-the-counter products, nebulizers, and other therapies including non-drug therapies)
- What specific symptoms do your treatments address?
- How has your treatment regimen changed over time, and why?
- How well does your current treatment regimen treat the most significant symptoms of your disease?
- How well do these treatments stop or slow the progression of your disease?
- How well do these therapies improve your ability to do specific activities that are important to you in your daily life?
- How well have these treatments worked for you as your condition has changed over time?
- What are the most significant downsides to your current treatments, and how do they affect your daily life? (Examples of downsides may include bothersome side effects, need for multiple medications, need for injections, going to the hospital for treatment, etc.)
- Assuming there is no complete cure for your condition, what specific things would you look for in an ideal treatment for your condition?