Food and Drug Administration, HHS.
Notice of public meeting; request for comments.
The Food and Drug Administration (FDA, the Agency, or we) is announcing a public meeting and an opportunity for public comment on Patient-Focused Drug Development for autism. 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 as well as patient views on treatment approaches for autism.
The public meeting will be held on May 4, 2017, from 1 p.m. to 5 p.m. Registration to attend the meeting must be received by April 24, 2017 (see
The public meeting will be held at the FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (Rm. 1503), Silver Spring, MD 20993–0002. Entrance for the public meeting participants (non-FDA employees) is through Building 1 where routine security check procedures will be performed. For more information on parking and security procedures, please refer to
You may submit comments as follows:
Submit electronic comments in the following way:
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• If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see “Written/Paper Submissions” and “Instructions”).
Submit written/paper submissions as follows:
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• For written/paper comments submitted to the Division of Dockets Management, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in “Instructions.”
• Confidential Submissions—To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states “THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.” The Agency will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on
FDA will post the agenda approximately 5 days before the meeting at:
Shanon Woodward, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 1141, Silver Spring, MD 20993–0002, 240–402–6167, FAX: 301–847–8443,
FDA has selected autism as the focus of a public meeting under Patient-Focused Drug Development, an initiative that involves obtaining a better understanding of patient perspectives on the severity of a disease and the available therapies for that condition. Patient-Focused Drug Development is being conducted to fulfill FDA performance commitments that are part of the reauthorization of the PDUFA under Title I of the Food and Drug Administration Safety and Innovation Act (Pub. L. 112–144). The full set of performance commitments is available at
FDA committed to obtain the patient perspective on at least 20 disease areas during the course of PDUFA V. For each disease area, the Agency is conducting a public meeting to discuss the disease and its impact on patients' daily lives, the types of treatment benefit that matter most to patients, and patients' perspectives on the adequacy of the available therapies. These meetings will include participation of FDA review divisions, the relevant patient communities, and other interested stakeholders.
On April 11, 2013, FDA published a notice in the
As part of Patient-Focused Drug Development, FDA will obtain input of patients and patient representatives on the symptoms of autism that matter most to patients and on current approaches to treating autism. Autism is a neurodevelopmental disorder characterized in varying degrees by difficulties with social interaction, verbal and non-verbal communication challenges, and repetitive behavior patterns. FDA has approved products for irritability related to autism including risperidone and aripiprazole. In addition to pharmacological treatments, behavioral and educational interventions are also common treatment options. FDA is interested in the perspectives of patients with autism and caregivers on (1) symptoms and the daily impacts of their condition, (2) current approaches to treatment, and (3) decision factors taken into account when selecting a treatment.
The questions that will be asked of patients and patient representatives at the meeting are listed in this section, organized by topic. For each topic, a brief initial patient/caregiver panel discussion will begin the dialogue. This will be followed by a facilitated discussion inviting comments from other patient and patient representative participants. In addition to input generated through this public meeting, FDA is interested in receiving patient input addressing these questions through written comments, which can be submitted to the public docket (see
(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 concentrating, 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 through the night, daily hygiene, 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?
(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 as 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 devoted to treatment, etc.)
(4) What specific things would you look for in an ideal treatment for your/your child's 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? (Common 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 risks associated with the treatments? (Examples of less common but serious risks are infections, organ damage or failure, suicidal thoughts)
If you wish to attend this meeting, visit
Patients and patient representatives who are interested in presenting comments as part of the initial panel discussions will be asked to indicate in their registration which topic(s) they wish to address. These patients and patient representatives also must send to