Document ID: FDA-2019-N-4041-0001
Agency: fda
Document Type: Notice
Title: Advancing the Development of Pediatric Therapeutics: Pediatric Clinical Trial Endpoints for Rare Diseases With a Focus on Pediatric Patient Perspectives; Public Workshop
Posted Date: 2019-10-10T04:00Z

[Federal Register Volume 84, Number 197 (Thursday, October 10, 2019)]
[Notices]
[Pages 54611-54612]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-22187]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-N-4041]

Advancing the Development of Pediatric Therapeutics: Pediatric 
Clinical Trial Endpoints for Rare Diseases With a Focus on Pediatric 
Patient Perspectives; Public Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Office of Pediatric Therapeutics, Food and Drug 
Administration (FDA), is announcing a public workshop entitled 
``Advancing the Development of Pediatric Therapeutics (ADEPT 6): 
Pediatric Clinical Trial Endpoints for Rare Diseases with a Focus on 
Pediatric Patient Perspectives.'' The purpose of this workshop is to 
discuss pediatric patient-specific engagement in the development of 
clinical trial endpoints for rare diseases.

DATES: The public workshop will be held on November 12, 2019, from 8 
a.m. to 4:30 p.m. See the SUPPLEMENTARY INFORMATION section for 
registration date and information.

ADDRESSES: The public workshop will be held at the FDA White Oak 
Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great 
Room (Rm. 1503-A), Silver Spring, MD 20993-0002. Entrance for the 
public workshop participants (non-FDA employees) is through Building 1, 
where routine security check procedures will be performed. For parking 
and security information, please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.

FOR FURTHER INFORMATION CONTACT: Terrie L. Crescenzi, Office of 
Pediatric Therapeutics, Food and Drug Administration, 10903 New 
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-8646, email: 
terrie.crescenzi@fda.hhs.gov; or Elizabeth Sanford, Office of Pediatric 
Therapeutics, Food and Drug Administration, 10903 New Hampshire Ave., 
Silver Spring, MD 20993-0002, 301-796-8659, email: 
elizabeth.sanford@fda.hhs.gov.

SUPPLEMENTARY INFORMATION: 

I. Background

    Patient engagement is critical in the development of patient-
focused study endpoints that measure clinical benefit in clinical 
trials. Asking patients what aspects of their disease they consider 
important to measure is especially important for rare diseases, given 
the lack of established endpoints for many rare diseases, the small 
number of patients available for enrollment in trials, and the 
heterogeneity of disease manifestations (e.g., between patients and 
over time). While there is increased emphasis on incorporating the 
patient voice in rare disease drug development activities, there is an 
increased need for pediatric patient-specific engagement efforts. 
Pediatric rare disease drug development would benefit from direct and 
early involvement of pediatric patients and their caregivers in 
determining the most relevant and clinically meaningful endpoints and 
outcome assessment tools for use in clinical trials.

II. Topics for Discussion at the Public Workshop

    In this workshop, FDA will obtain the pediatric patient perspective 
on their disease/condition and what is most important to consider when 
designing rare disease trials. There will also be discussion regarding 
patients' thoughts on clinical endpoints that are currently being used 
in clinical trials, potential areas of innovation, and how to create 
processes that might include pediatric patients and their caregivers as 
collaborators in endpoint development in early stages of medical 
product development (e.g., protocol design). The morning session will 
focus on identifying endpoints that capture important aspects of how 
pediatric patients feel and function. The afternoon session will focus 
on steps for development of clinical outcome assessment tools for use 
in pediatric patient populations and the potential role of child and 
youth friendly technology in endpoint assessments.

III. Participation in the Public Workshop

    Registration: Persons interested in attending this public workshop 
must register online at: https://www.eventbrite.com/e/adept-6-workshop-pediatric-clinical-trial-endpoints-for-rare-diseases-registration-67523118465 by November 5, 2019. For those without internet access, 
please contact Terrie Crescenzi or Elizabeth Sanford (see FOR FURTHER 
INFORMATION CONTACT) to register.
    Registration is free and based on space availability, with priority 
given to early registrants. Onsite registration on the day of the 
meeting will be based on space availability. Registration information, 
the agenda, and additional background materials can be found at http://
wcms-internet.fda.gov/news-

[[Page 54612]]

events/fda-meetings-conferences-and-workshops/advancing-development-
pediatric-therapeutics-adept-6-pediatric-clinical-trial-endpoints-rare.
    If you need special accommodations due to a disability, please 
contact Elizabeth Sanford (see FOR FURTHER INFORMATION CONTACT) at 
least 7 days in advance. Persons attending the meeting are advised that 
FDA is not responsible for providing access to electrical outlets.
    Streaming Webcast of the Public Workshop: This public workshop will 
also be webcast. Login URL: https://collaboration.fda.gov/adept6/.
    If you have never attended a Connect Pro event before, test your 
connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. To get a quick overview of the Connect Pro program, 
visit https://www.adobe.com/go/connectpro_overview. FDA has verified 
the website addresses in this document, as of the date this document 
publishes in the Federal Register, but websites are subject to change 
over time.
    Transcripts: Please be advised that as soon as a transcript of the 
public workshop is available, FDA will post it at http://wcms-internet.fda.gov/news-events/fda-meetings-conferences-and-workshops/advancing-development-pediatric-therapeutics-adept-6-pediatric-clinical-trial-endpoints-rare.

    Dated: October 4, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-22187 Filed 10-9-19; 8:45 am]
 BILLING CODE 4164-01-P