Register Now! June 10th FDA Patient-Focused Disease-Specific Meeting

May 21, 2014

FDA Public Meeting information

  •   Title: “Neurological Manifestations of Inborn Errors of Metabolism”
  •   Goal: To obtain patient input on the impact of the neurological manifestations of inborn errors of metabolism on daily life and patient views on treatment options.
  •   Date: June 10, 2014
  •   Time: 9am-to-1pm
  •   Location:US Food and Drug Administration (White Oak campus) 10903 New Hampshire Avenue
    Building 31, Room 1503A (Great Room)
    Silver Spring, MD 20993

 You can participate in 1 of 3 ways:

  1. As a speaker (panelist). 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.
  2. On day of meeting to speak during the open public session. There will also be an opportunity for patients, patient representatives and others to provide comments on issues other than the topics during an Open Public Comment session. Sign up for Open Public Comment will take place the day of the meeting.
  3. By webinar. Webcast participants will also have an opportunity to provide input through webcast comments.
  •   Registration is required to attend in person, or by webinar. Visit Registration closes May 27, 2014. These meetings reach full capacity quickly.
  •   Topics and accompanying Questions:
    The FDA has posted the following topics to get patients to focus on the most prevalent symptoms, and symptoms that are not adequately addressed by current or “ideal” treatments.


For more information, refer to the FDA meeting website at:

Topic 1: Neurologic/ neuropsychological 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?

page1image24216 Topic 2:  Current available approaches to treat the neurologic manifestations of inborn errors of metabolism

1. What are you/child currently 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?


Background & General Information on FDA Public Meetings 

  • Patient-Focused Drug Development is an initiative launched by FDA to improve drug development and review by taking a more systematic approach to looking at benefit-risk assessment including the disease severity and unmet medical needs.
  • The goal of this initiative to obtain a better understanding of patients’ perspectives on their disease/condition and the available therapies to treat their condition.
  •  As part of this commitment, the FDA will hold 20 specific disease-related meetings between 2013 and 2017. The June 10th meeting on neurological manifestations of inborn errors of metabolism is one of these 20 meetings.

Topics for each meeting include:

  • The impact of the disease on patients
  • The spectrum of severity for those who have the disease
  • The measures of benefit that matter most to patients, and o The adequacy of the existing treatment options for patients
  • These meetings are public and published in the Federal Register.
  • Participants include FDA review divisions, the relevant patient advocacy community and other interested stakeholders.
  • FDA will issue a report will summarize the input provided by patients and patient representatives at each meeting.

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