Date: April 6, 2016
Time: 1:00 p.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 April 6, 2017 FDA is conducting a public meeting on Patient-Focused Drug Development for Sarcopenia. 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). FDA is interested in the perspectives of patients with sarcopenia on i) symptoms and the daily impacts of their condition ii) current approaches to treatment, and iii) decision factors taken into account when selecting a treatment.
The questions that will be asked of patients and patient stakeholders at the meeting are listed below, organized by topic. For each topic, a panel of patients and patient representatives 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 attending the meeting in person and 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 by March 20, 2017. 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/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm527587.htm.
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 difficulty walking, feeling unsteady and falling frequently, having a decreased level of activity etc.)
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 of activities may include participation in social activities, household chores, daily hygiene, etc.)
3) How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days?
4) How have your condition and its symptoms changed over time?
a) Would you define your condition today as being well managed?
5) What worries you most about your condition?
Topic 2: Patients’ perspectives on current approaches to treatment
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 modification.)
a) What specific symptoms do your treatments address?
b) How has your treatment regimen changed over time, and why?
2) How well does your current treatment regimen control your condition?
a) How well do your treatments address specific activities that are important to you in your daily life?
b) How well have these treatments worked for you as your condition has changed over time?
3) What are the most significant downsides to your current treatments, and how do they affect your daily life? (Examples of downsides may include going to the hospital or clinic for treatment, time devoted to treatment, etc.)
4) What specific things would you look for in an ideal treatment for your condition?
a) What would you consider to be a meaningful improvement (for example symptom improvements or functional improvements) in your condition that a treatment could provide?