Advancing the Development of Pediatric Therapeutics: Pediatric Clinical Trial Endpoints for Rare Diseases With a Focus on Pediatric Patient Perspectives; Public Workshop, 54611-54612 [2019-22187]

Download as PDF 54611 Federal Register / Vol. 84, No. 197 / Thursday, October 10, 2019 / Notices of ‘‘Coronary, Peripheral, and Neurovascular Guidewires— Performance Tests and Recommended Labeling’’ may send an email request to CDRH-Guidance@fda.hhs.gov to receive an electronic copy of the document. Please use the document number 16007 to identify the guidance you are requesting. IV. Paperwork Reduction Act of 1995 This guidance refers to previously approved collections of information. These collections of information are subject to review by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501–3521). The collections of information in the following FDA regulations and guidance have been approved by OMB as listed in the following table: 21 CFR part; guidance; or FDA form Topic 807, subpart E ......................................................................... 812 ........................................................................................... ‘‘Requests for Feedback on Medical Device Submissions: The Q-Submission Program and Meetings with Food and Drug Administration Staff’’. 800, 801, and 809 .................................................................... 820 ........................................................................................... Premarket notification ............................................................. Investigational Device Exemption ........................................... Q-submissions ......................................................................... 0910–0120 0910–0078 0910–0756 Medical Device Labeling Regulations ..................................... Current Good Manufacturing Practice (CGMP); Quality System (QS) Regulation. 0910–0485 0910–0073 Dated: October 4, 2019. Lowell J. Schiller, Principal Associate Commissioner for Policy. [FR Doc. 2019–22194 Filed 10–9–19; 8:45 am] BILLING CODE 4164–01–P 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. 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 SUMMARY: VerDate Sep<11>2014 19:50 Oct 09, 2019 Jkt 250001 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. PO 00000 Frm 00027 Fmt 4703 Sfmt 4703 OMB control No. 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-6workshop-pediatric-clinical-trialendpoints-for-rare-diseases-registration67523118465 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 https://wcms-internet.fda.gov/news- E:\FR\FM\10OCN1.SGM 10OCN1 54612 Federal Register / Vol. 84, No. 197 / Thursday, October 10, 2019 / Notices events/fda-meetings-conferences-andworkshops/advancing-developmentpediatric-therapeutics-adept-6pediatric-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 https://wcms-internet.fda.gov/newsevents/fda-meetings-conferences-andworkshops/advancing-developmentpediatric-therapeutics-adept-6pediatric-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] DEPARTMENT OF HEALTH AND HUMAN SERVICES [Docket No. FDA–2013–N–1428] Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Electronic Drug Product Reporting for Human Drug Compounding Outsourcing Facilities Under Section 503B of the Federal Food, Drug, and Cosmetic Act Food and Drug Administration, VerDate Sep<11>2014 20:53 Oct 09, 2019 Jkt 250001 The Food and Drug Administration (FDA) is announcing that a proposed collection of information has been submitted to the Office of Management and Budget (OMB) for review and clearance under the Paperwork Reduction Act of 1995. DATES: Fax written comments on the collection of information by November 12, 2019. ADDRESSES: To ensure that comments on the information collection are received, OMB recommends that written comments be faxed to the Office of Information and Regulatory Affairs, OMB, Attn: FDA Desk Officer, Fax: 202– 395–7285, or emailed to oira_ submission@omb.eop.gov. All comments should be identified with the OMB control number 0910–0827. Also include the FDA docket number found in brackets in the heading of this document. SUMMARY: FOR FURTHER INFORMATION CONTACT: Domini Bean, Office of Operations, Food and Drug Administration, Three White Flint North, 10A–12M, 11601 Landsdown St., North Bethesda, MD 20852, 301–796–5733, PRAStaff@ fda.hhs.gov. In compliance with 44 U.S.C. 3507, FDA has submitted the following proposed collection of information to OMB for review and clearance. SUPPLEMENTARY INFORMATION: OMB Control Number 0910–0827— Extension Food and Drug Administration HHS. Notice. Electronic Drug Product Reporting for Human Drug Compounding Outsourcing Facilities Under Section 503B of the Federal Food, Drug, and Cosmetic Act BILLING CODE 4164–01–P AGENCY: ACTION: The Drug Quality and Security Act added section 503B to the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 353b) creating a category of entities called ‘‘outsourcing facilities.’’ Outsourcing facilities, as defined in section 503B(d)(4) of the FD&C Act, are facilities that must meet all the requirements described in section 503B, including registering with FDA as an outsourcing facility and submitting regular reports identifying the drugs PO 00000 Frm 00028 Fmt 4703 Sfmt 4703 compounded by the outsourcing facility during the previous 6-month period. The first of these reports must be submitted upon initial registration as an outsourcing facility. Thereafter, semiannual product reports must be submitted, once during the month of June and once during the month of December, for as long as an establishment remains registered as an outsourcing facility. In addition, drug products compounded in an outsourcing facility can qualify for exemptions from the FDA approval requirements in section 505 of the FD&C Act (21 U.S.C. 355) and the requirement to label products with adequate directions for use under section 502(f)(1) of the FD&C Act (21 U.S.C. 352(f)(1)) if the requirements in section 503B are met. To help respondents understand the statutory requirements, how we interpret them, and the associated information collection, we developed the guidance document entitled ‘‘Electronic Drug Product Reporting for Human Drug Compounding Outsourcing Facilities Under Section 503B of the Federal Food, Drug, and Cosmetic Act.’’ The guidance is available from our website at: https://www.fda.gov/media/ 90173/download. The guidance explains that, once an entity has elected to register as an outsourcing facility, it must submit reports identifying the drugs compounded by the outsourcing facility. The guidance also communicates who must report, the format of the report, the content to include in each report, when to report, how reports are submitted to FDA, and the consequences of outsourcing facilities’ failure to submit reports. In the Federal Register of July 17, 2019 (84 FR 34184), we published a 60day notice requesting public comment on the proposed collection of information. No comments were received. We therefore estimate the burden of the information collection as follows: E:\FR\FM\10OCN1.SGM 10OCN1

Agencies

[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.

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

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: 
[email protected]; or Elizabeth Sanford, Office of Pediatric 
Therapeutics, Food and Drug Administration, 10903 New Hampshire Ave., 
Silver Spring, MD 20993-0002, 301-796-8659, email: 
[email protected].

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 https://
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 https://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

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