[Federal Register Volume 86, Number 185 (Tuesday, September 28, 2021)]
[Notices]
[Pages 53663-53664]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-21000]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2021-N-0689]


Public Workshop: Analgesic Clinical Trial Designs, Extrapolation, 
and Endpoints in Patients From Birth to Less Than Two Years of Age

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Food and Drug Administration (FDA) is announcing a public 
workshop entitled ``Analgesic Clinical Trial Designs, Extrapolation, 
and Endpoints in Patients from Birth to Less Than Two Years of Age.'' 
The purpose of the public workshop is to discuss the state of science, 
data gaps, and challenges in drug development for drugs intended to 
treat acute pain in patients less than 2 years of age.

DATES: The public workshop will be held virtually on October 13 and 14, 
2021, from 10 a.m. to 2 p.m. Eastern Standard Time. See the 
SUPPLEMENTARY INFORMATION section for registration date and 
information.

ADDRESSES: The public workshop will be held in virtual format only and 
will not be held at a specific location. Please note that due to the 
impact of the COVID-19 pandemic, all meeting participants will be 
joining this public meeting via an online teleconferencing platform. 
The public workshop will be held at https://go.umd.edu/analgesic-clinical-trial.

FOR FURTHER INFORMATION CONTACT: Heather Buck at 
[email protected], 301-796-1413 or Kerri-Ann Jennings at [email protected], 301-796-2919, Food and Drug Administration, 
Center for Drug Evaluation and Research, 10903 New Hampshire Ave., 
Bldg. 22, Rm. 6467, Silver Spring, MD 20903-0002.

SUPPLEMENTARY INFORMATION:

I. Background

    In 2009, FDA convened a scientific workshop with experts in 
pediatric pain, pediatric clinical trial design, pediatric ethics, and 
pediatric drug development \1\. Based on the available data at the 
time, the expert panel recommended extrapolation of efficacy in 
patients 2 years and older, relying on matching effective drug 
exposures in adults. The current approach to study drugs with well-
established mechanisms of action, such as opioids, non-steroidal anti-
inflammatory drugs (NSAIDS), acetaminophen, and local anesthetics, 
relies on matching safe and effective drug exposures in adults to 
support the efficacy of drugs used to treat acute pain in pediatric 
patients at least 2 years of age. Controlled efficacy trials are only 
required in patients from birth to less than 2 years of age. When 
controlled efficacy trials are needed, FDA has recommended an ``add-
on'' design using opioid-sparing calculation rather than the change in 
pain intensity used in efficacy trials of analgesics in adults.
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    \1\ Berde, CB, et.al., Pediatrics 2012 Feb;129(2):354-64. 
https://www.fda.gov/advisory-committees/advisory-committee-calendar/april-12-2016-pediatric-advisory-committee-meeting-announcement-04122016-04122016.
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    Despite these advances in clinical trial design, there continues to 
be unmet needs in the availability of products to treat acute pain, 
especially in patients less than 2 years of age. There is currently 
only one analgesic labeled for use in patients less than 2 years of 
age: Ibuprofen is approved for the treatment

[[Page 53664]]

of pain in children 6 months of age and older. Furthermore, controlled 
trials in patients less than 2 years of age have been difficult to 
complete and the data obtained from completed trials have often been 
difficult to interpret.
    The purpose of the public workshop is to discuss the current state 
of therapies to treat acute pain in children, identify data gaps, and 
consider methods to improve the current drug development paradigm for 
acute pain in patients less than 2 years of age (e.g., use of pediatric 
extrapolation, and novel clinical trial designs). The workshop is 
intended to focus on drugs with well-established mechanisms of action 
(NSAIDs, acetaminophen, local anesthetics, opioids), rather than drugs 
with novel mechanisms of action.

II. Topics for Discussion at the Public Workshop

    The main objective of the ``Analgesic Clinical Trial Designs, 
Extrapolation, and Endpoints in Patients from Birth to Less Than Two 
Years of Age'' workshop is to discuss the current state of therapies to 
treat acute pain in children, identify data gaps, and discuss feasible 
trial designs and methods (e.g., use of pediatric extrapolation) to 
improve the current drug development paradigm for acute pain in 
patients less than 2 years of age. The workshop will include 
regulators, industry, academia, and patient organizations to optimize 
the discussion of the selected topics.

III. Participating in the Public Workshop

    Registration: Please visit the following website to register for 
this public workshop: https://go.umd.edu/analgesic-clinical-trial. 
Please provide complete contact information for each attendee, 
including name, title, affiliation, address, email, and telephone.
    Streaming Webcast of the Public Workshop: This public workshop will 
also be webcast at the following site: https://collaboration.fda.gov/rz3mubd491lo/.
    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. For an 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.

    Dated: September 22, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-21000 Filed 9-27-21; 8:45 am]
BILLING CODE 4164-01-P