[Federal Register Volume 82, Number 42 (Monday, March 6, 2017)]
[Notices]
[Pages 12611-12613]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-04229]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-N-0136]


Public Meeting on Patient-Focused Drug Development for Autism; 
Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public meeting; request for comments.

-----------------------------------------------------------------------

SUMMARY: 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.

DATES: 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 SUPPLEMENTARY INFORMATION for instructions). Submit either 
electronic or written comments on the public meeting by July 5, 2017. 
Late, untimely filed comments will not be considered. Electronic 
comments must be submitted on or before July 5, 2017. The https://www.regulations.gov electronic filing system will accept comments until 
midnight Eastern Time at the end of July 5, 2016. Comments received by 
mail/hand delivery/courier (for written paper submissions) will be 
considered timely if they are postmarked or the delivery service 
acceptance receipt is on or before that date. See the SUPPLEMENTARY 
INFORMATION section for registration date and information.

ADDRESSES: 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 http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    You may submit comments as follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
will be posted to the docket unchanged. Because your comment will be 
made public, you are solely responsible for ensuring that your comment 
does not include any confidential information that you or a third party 
may not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on https://www.regulations.gov.
     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'').

Written/Paper Submissions

    Submit written/paper submissions as follows:
     Mail/Hand delivery/Courier (for written/paper 
submissions): Division of Dockets Management (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     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.''
    Instructions: All submissions received must include the Docket No. 
FDA-2017-N-0136 for ``Public Meeting on Patient-Focused Drug 
Development for Autism.'' Received comments, those filed in a timely 
manner (see DATES), will be placed in the docket and, except for those 
submitted as ``Confidential Submissions,'' publicly viewable at https://www.regulations.gov or at the Division of Dockets Management between 9 
a.m. and 4 p.m., Monday through Friday.
     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 https://www.regulations.gov. 
Submit both copies to the Division of Dockets Management. If you do not 
wish your name and contact information to be made publicly available, 
you can provide this information on the cover sheet and not in the body 
of your comments and you must identify this information as 
``confidential.'' Any information marked as ``confidential'' will not 
be disclosed except in accordance with 21 CFR 10.20 and other 
applicable disclosure law. For more information about FDA's posting of 
comments to public dockets, see 80 FR 56469, September 18, 2015, or 
access the information at: https://www.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the

[[Page 12612]]

electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in 
the heading of this document, into the ``Search'' box and follow the 
prompts and/or go to the Division of Dockets Management, 5630 Fishers 
Lane, Rm. 1061, Rockville, MD 20852.
    FDA will post the agenda approximately 5 days before the meeting 
at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm529043.htm.

FOR FURTHER INFORMATION CONTACT: 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, [email protected].

SUPPLEMENTARY INFORMATION:

I. Background on Patient-Focused Drug Development

    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 http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf.
    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 Federal Register 
(78 FR 08441) announcing the disease areas for meetings in fiscal years 
(FYs) 2013-2015, the first 3 years of the 5-year PDUFA V time frame. 
The Agency used several criteria outlined in that notice to develop the 
list of disease areas. FDA obtained public comment on the Agency's 
proposed criteria and potential disease areas through a public docket 
and a public meeting that was convened on October 25, 2012. In 
selecting the set of disease areas, FDA carefully considered the public 
comments received and the perspectives of review divisions at FDA. FDA 
initiated a second public process for determining the disease areas for 
FY 2016-2017, and published a notice in the Federal Register on July 2, 
2015, announcing the selection of eight disease areas. More 
information, including the list of disease areas and a general schedule 
of meetings, is posted at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm347317.htm.

II. Purpose and Scope of the Meeting

    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 ADDRESSES).

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 
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?

Topic 2: Patients' Perspectives on Current Approaches to Treating

    (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?

[[Page 12613]]

    (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)

III. Meeting Attendance and Participation

    If you wish to attend this meeting, visit https://autismpfdd.eventbrite.com. Persons interested in attending this public 
meeting must register by April 24, 2017. If you are unable to attend 
the meeting in person, you can register to view a live Webcast of the 
meeting. You will be asked to indicate in your registration if you plan 
to attend in person or via the Webcast. Registration is free and based 
on space availability, with priority given to early registrants. Early 
registration is recommended because seating is limited; therefore, FDA 
may limit the number of participants from each organization. 
Registrants will receive confirmation once they have been accepted. 
Onsite registration on the day of the meeting will be based on space 
availability. If you need special accommodations because of a 
disability, please contact Shanon Woodward (see FOR FURTHER INFORMATION 
CONTACT) at least 7 days before the meeting.
    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 
[email protected] a brief summary of responses to the topic 
questions by April 17, 2017. Panelists will be notified of their 
selection approximately 7 days before the public meeting. We will try 
to accommodate all patients and patient representatives who wish to 
speak, either through the panel discussion or audience participation; 
however, the duration of comments may be limited by time constraints.
    Transcripts: Please be advised that as soon as a transcript is 
available, it will be accessible at https://www.regulations.gov. It may 
be viewed at the Division of Dockets Management (see ADDRESSES). A link 
to the transcript will also be available on the Internet at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm529043.htm.

    Dated February 28, 2017.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2017-04229 Filed 3-3-17; 8:45 am]
BILLING CODE 4164-01-P