Date: June 10, 2014
Time: 9:00 a.m. to 1:00 p.m.
Location: FDA White Oak Campus
10903 New Hampshire Ave.
Building 31 Conference Center, The Great Room (Rm 1503)
Silver Spring, MD 20993
On June 10, 2014, FDA is conducting a public meeting on Neurologic Manifestations of Inborn Errors of Metabolism (IEM) Patient-Focused Drug Development. FDA is interested in obtaining patient input on the neurologic/neuropsychological signs, symptoms, and daily impacts that matter most to patients (topic 1), and current available approaches to treat the neurologic manifestations of inborn errors of metabolism (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 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. 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/ucm387057.htm
Questions for Discussion
Topic 1: Disease signs, 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 neurologic/neuropsychological signs and/or symptoms have the most significant impact on you/your child's life? (Examples may include seizures, decreased muscle tone, sensory issues, etc.)
2) Are there specific activities that are important to you/your child but that you/your child cannot do because of these neurologic/neuropsychological signs or symptoms? (Examples of activities may include sleeping through the night, daily hygiene, going up the stairs, etc.)
3) How have you/your child's neurologic/neuropsychological signs or symptoms changed over time?
Topic 2: Patient perspectives on current approaches to treating neurologic manifestations of inborn errors of metabolism
1) What are you/child currenty doing to help treat the condition or its signs/symptoms? (Examples may include prescription medicines, herbal therapies, acupuncture, over-the-counter products, and other therapies including non-drug therapies such as diet modification.)
a) How well does this current treatment regimen treat the neurological symptoms of you/your child's disease? For example, how well do the treatments improve you/your child's ability to do specific activities?
2) Assuming there is no complete cure for you/your child's condition, what specific attributes would you look for in an ideal treatment for the condition?
3) The process of informed consent is an important way for researchers to communicate the purpose of a clinical trial, and the potential benefits and risks of the trial so that people can make an informed decision about whether to participate. Informed consent also ensures that parents are fully informed and are given opportunities to ask questions about the clinical trial. In addition to informed consent from parents, assent from children may also be needed. Assent is the term used to describe when a child agrees to be in a clinical trial. Among other considerations, children should be old enough to understand basic facts about the clinical trial in order to provide assent to participate.
a) In the informed consent process, what are important considerations to take into account in cases when the potential participant is a child? For example, how should the informed consent clearly communicate to the patient the potential benefits and risks of a study?