May 4, 2017
1:00 p.m. – 5:00 p.m.
FDA White Oak Campus
10903 New Hampshire Ave.
Building 31, Conference Center; The Great Room (Rm 1503 B and C)
Silver Spring, MD 20993
Enter at Building 1 to clear security
(Information about arrival to FDA’s White Oak campus)
On May 4, 2017, FDA is conducting a public meeting on Patient-Focused Drug Development for Autism. FDA is interested in obtaining patient perspectives on the impact of autism on daily life and patient views on treatment approaches. 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 autism on daily life, patient views on treatment approaches for autism.
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 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 April 17th. 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 the Topic 1 and Topic 2 Discussion Questions (see below) 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/ucm529043.htm
Topic 1: Disease symptoms and daily impacts that matter most to patients
1) Of all the symptoms that you/your child experiences because of the condition, which 1-3 symptoms have the most significant impact on your/your child's life? (Examples may include behavioral symptoms, difficulty with motor coordination, difficulty sleeping, difficulty concentration, seizures, etc.)
2) Are there specific activities that are important to you/your child but that you/your child cannot do at all or as fully as you would like because of these symptoms? (Examples of activities may include sleeping throught the night, daily hygeine, eating, dressing, participation in sports or social activities, etc.)
a) How do these symptoms and their negative impacts affect daily life on the best days? On the worst days?
3) How has your/your child's condition and its symptoms changed over time?
4) What worries you/your child most about your/your child's condition?
Topic 2: Patients' perspective on current treatment approaches
1) What are you/your child currently doing to help treat the condition or its symptoms? (Examples may include prescription medicines, over-the-counter products, and other therapies including non-drug therapies such as behavioral interventions)
a) How has your/your child's treatment regimen changed over time, and why?
2) How well does your/your child's current treatment regimen treat the most significant symptoms of the condition?
a) How well do your/your child's treatments address specific activities that are important to you/your child's daily life?
b) How well have these treatments worked for you/your child as the condition has changed over time? Which symptoms are not addressed well?
3) What are the most significant downsides to your/your child's current treatments, and how do they affect your daily life? (Examples of downsides may include bothersome side effects, interacts with other medications, time devotd to treatment, etc.)
4) Assuming there are no complete cures for your/your child's condition, what specific things would you/your child 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/your child's condition that a treatment could provide?
5) What factors do you/your child take into account when making decisions about selecting a course of treatment?
a) What information on potential benefits of these treatments factors most into your/your child's decision?
b) How do you/your child weigh the potential benefits of these treatments versus the common side effects of the treatments? (Commom side effects could include headache, nausea, fatigue, weight gain)
c) How do you/your child weigh potential benefits of these treatments versus the less common but serious risk associated with the treatments? (Examples of less common but serious risk are infections, organ damage or failure, suicidal thoughts)