Date: May 13, 2014
Time: 1:00 p.m. to 5:00 p.m.
Location: FDA White Oak Campus
10903 New Hampshire Ave.
Building 31, Room 1503A (Great Room)
Silver Spring, MD 20993
On May 13, 2014, FDA is conducting a public meeting on Pulmonary Arterial Hypertension (PAH) Patient-Focused Drug Development. FDA is interested in obtaining patient input on the impact of pulmonary arterial hypertension on daily life (topic 1) and currently available treatments to treat the condition (topic 2). The questions for discussion on these topics are below.
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 other patients and patient representatives.
If you are interested in providing comments as part of the initial panel discussion, indicate so during the registration process. 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.
For more information, refer to the FDA meeting website at:http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm379694.htm.
Questions for Discussion
Topic 1: Disease symptoms and daily impacts that matter most to patients
1) 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 symptoms such as chest pain, shortness of breath, difficulty concetrating, and others.)
2) 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 may include activities such as household chores, walking up the stairs.)
a) How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days?
3) How have your condition and its symptoms changed over time?
Topic 2: Patients’ perspectives on current approaches to treating PAH
1) What are you currently doing to help treat your condition or its symptoms? (Examples may include prescription medicines, over-the-counter products, and other therapies including non-drug therapies such as diet modifications.)
a) How has your treatment regimen changed over time?
b) How well does your current treatment regimen treat the most significant symptoms of your disease?
c) Have the medications for pulmonary arterial hypertension made a difference to you? If so, in what ways?
2) What are the most significant downsides to your current treatments, and how do they affect your daily life? (Examples may include downsides such as bothersome side effects, going to the hospital for treatment, and others.)
3) Assuming there is no complete cure for your condition, what specific things would you look for in an ideal therapy for your condition?