[Federal Register Volume 74, Number 176 (Monday, September 14, 2009)]
[Notices]
[Pages 47008-47010]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: E9-22012]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2009-N-0407]


Pediatric Clinical Trials Workshop: Unmet Needs, Trial Designs 
and Clinically Meaningful Safety and Effectiveness Outcomes

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop; request for comments.

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SUMMARY: The Food and Drug Administration (FDA) is announcing a public 
workshop entitled ``Pediatric Clinical Trials Workshop: Unmet Needs, 
Trial Designs and Clinically Meaningful Safety and Effectiveness 
Outcomes.'' The purpose of the public workshop is to solicit 
information from primary and secondary health care providers, academia, 
industry, and professional societies on various aspects of device 
clinical trials involving pediatric diseases and patients. Information 
from this public workshop will help stimulate interest in pediatric 
device clinical trial research methods, and develop topics for further 
discussion regarding the safety of pediatric device clinical trials. 
The information gathered in this and future workshops will help to 
develop future guidance for developing safe clinical trials for devices 
intended for pediatric patients. We encourage participation and 
comments from workshop attendees on the topics and questions discussed. 
Please see instructions for registration and for providing comments in 
the sections of this document entitled ``Registration'' and 
``Comments.''
    Dates and Times: The public workshop will be held on October 29, 
2009, from 8 a.m. to 5:30 p.m. and October 30, 2009, from 8 a.m. to 12 
noon.
    Location: The public workshop will be held at the Holiday Inn 
College Park located off I-95 at 10000 Baltimore Ave., College Park, MD 
20740. The hotel front desk number is 1-301-345-6700. For directions, 
please refer to the meeting Web page: http://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/ucm170938.htm
    Contact Person: Barbara Buch, Center For Devices and Radiological 
Health, Food and Drug Administration, Bldg. 66, rm. 1406, 10903 New 
Hampshire Ave., Silver Spring, MD 20993, 301-796-5650, FAX: 301-847-
8117, e-mail: [email protected]. If you need special 
accommodations due to a disability, (such as wheelchair access or a 
sign language interpreter), please notify Barbara Buch by September 30, 
2009.
    Registration: Registration and seating will be on a first-come, 
first-served basis and discussion preference will be afforded to 
clinical research investigators involved in pediatric clinical device 
trials, health care givers, and patient advocates. Please provide your 
name, title, organization affiliation, address, and e-mail contact 
information. There is no registration fee to attend the workshop. There 
will be no onsite registration. Please register electronically at 
http://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/default.htm by September 30, 2009. Due to limited space, and to 
maximize participation, attendees are asked to delegate one or two 
representatives from their organizations to participate in the general 
sessions. A report of The Workshop and The Information presented will 
be available following the meeting via a link on the meeting Web page. 
If you wish to make an oral comment during or to attend the public 
workshop, please note this in your registration information. The online 
registration form will instruct you as to the information you should 
provide prior to the meeting. In general, a summary of the presentation 
and an electronic copy of the presentation should be submitted by 
October 1, 2009. We will try to accommodate all persons who wish to 
make oral comments during the general sessions. However, we strongly 
recommend that you provide written comments as instructed in this 
document to ensure that your opinion, comments, and suggestions are 
captured. Please refer to the section, ``Comments'' for instructions on 
how to submit written comments.
    Comments: The deadline for submitting comments regarding this 
public workshop is November 30, 2009.
    Regardless of attendance at the public workshop, interested persons 
may submit written or electronic comments. Written comments should be 
submitted to the Division of Dockets Management (HFA-305), Food and 
Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. 
Electronic comments should be submitted to http://www.regulations.gov. 
Comments should be identified with the docket number found in brackets 
in the heading of this document. Received comments may be seen in the 
Division of Dockets Management between 9 a.m. and 4 p.m., Monday 
through Friday.

SUPPLEMENTARY INFORMATION:

I. Why Are We Holding This Public Workshop?

    The purpose of the public workshop is to solicit expert input on 
topics related to pediatric device clinical trials. The agency seeks 
discussion between FDA and other interested parties regarding the 
conduct of clinical trials to investigate device use in pediatric 
populations. Other purposes of the public workshop are, to identify any 
gaps in such research, and to provide information about evaluating the 
short- and long-term safety and effectiveness of pediatric medical 
devices using valid and sound scientific methods. Since the 2007 Food 
and Drug Administration Amendments Act was signed into law, there has 
been increased interest in conducting scientifically sound clinical 
research related to pediatric populations. It is hoped that this 
meeting will provide a forum for open discussion and information 
exchange among interested parties, FDA, and other stakeholders to lay a 
framework for further research into the use of devices to treat 
disorders and diseases that affect pediatric patients.

II. What Will Be the Format for the Meeting?

    The format for the meeting will include a general session in the 
morning on the first day. Invited expert speakers will present 
information regarding current needs and concerns about clinical trials 
that involve pediatric patients. These presentations will provide the 
topics for the small breakout groups, which will begin in the afternoon 
session of day one and continue through the morning of day two of the 
public workshop. Each of the smaller breakout group discussion sessions 
will be led and moderated by a panel of experts in each of the 
specialty focus areas listed in section III of this document. Each 
small group session will begin with an invited presentation to describe 
the issues of concern in the specific specialty. This will be followed 
by a moderated question and comment session including both prespecified 
questions posed to the assembled group and any that arise during the 
workshop's discussions. Those in attendance will have the opportunity 
in these small group discussions to participate in the discussion, ask 
questions, and provide comments for consideration. Small group 
discussions will be concluded in the morning of day two. Small group 
participation will be limited by space and will be available on a 
first-come, first-served basis. When registering for

[[Page 47009]]

the meeting, you should also designate which small group discussion you 
would like to attend because each participant may register for only one 
of the small group sessions.
    At the conclusion of day two's small group discussions, the general 
session will reconvene. After the general session reconvenes, each 
small group will report to the general session the results of the 
discussions related to the general questions posed to each group in 
outline form.

III. What Are the General Topic Areas We Intend to Address at the 
Public Workshop?

    We plan to discuss a number of general disease/anatomical topical 
issues at the conference, including the following issues:
     Musculoskeletal disease,
     Cardiovascular disease,
     Abdominal disorders and gastrointestinal (GI) diseases,
     Neurologic disorders and conditions,
     Renal diseases, and
     Audiologic disorders.
    The challenges posed by developing diagnostic tests for pediatric 
patients will be addressed as a part of discussion under each topical 
area breakout session. For each of the general disease/anatomical topic 
areas, we will pose questions to elicit and solicit scientific and 
clinical discussion in the breakout sessions. These include, but are 
not limited to the following questions:
    What are the most urgent unmet needs?
    What are the best practices for conduct of clinical research, 
including clinical trial design?
    What are specific patient/caregiver issues to consider?
    What are the surrogate endpoints for lifelong patient safety and 
effectiveness?
    What are appropriate clinical assessments?
    What are appropriate endpoints that determine clinical success?
    The questions, listed in section IV of this document, will be the 
focus of the expert-moderated breakout discussions in the afternoon of 
day one and the morning of day two.

IV. What Are the Issues That Will Be Discussed and Considered?

Questions for Discussion Regarding Pediatric Device Clinical Trials

1. What Are the Five Most Important Unmet Research Needs in Each 
Specific Disease/Anatomic Category? (musculoskeletal disease, 
cardiovascular disease, abdominal disorders and GI diseases, neurologic 
disorders and conditions, renal diseases, and audiologic disorders)
    Although there are obvious barriers to clinical trials such as 
concerns about the effects on child development, there are significant 
needs in both rare and common diseases or disorders that have not been 
met with modifications of adult devices. We will start by asking 
questions such as:
    a. What are the most important unmet device needs in each category?
    b. What are the scientific or clinical barriers or other potential 
barriers to developing devices to meet those needs?
2. What Are Some Clinical Trial Designs That Encourage Enrollment of 
Pediatric Patients While Providing Quality Data to Support Safety and 
Effectiveness of Devices?
    We need to understand:
    a. What are appropriate controls to use in pediatric trials to 
satisfy the legal regulatory definitions of valid scientific evidence 
as described in 21 CFR 860.7?
    b. How can followup be maximized?
    c. What timeframes are needed given the age of patients and the 
expected lifetime of the device/disease being treated?
    d. How do we understand the long term effect on development and 
growth in a short clinical trial?
3. Preclinical and Animal Studies
    Although there are examples of immature and fetal animal studies 
that are well established for pharmaceuticals, how do we translate 
those concepts for devices?
    a. What types of endpoints and timeframes translate into outcomes 
in the human population? How do we know that?
    b. How do we set a standard to judge subsequent trial outcomes as 
acceptable and safe?
    c. What animal models exist or are appropriate for studying each of 
the diseases or disorders we identify as significant unmet needs?
4. How Do We Measure Safety and Effectiveness in a Pediatric 
Population?
    The pediatric population cannot always describe symptoms or 
functional problems in the same way that adults can. It therefore 
follows that the same assessment tools and surrogate endpoints will not 
apply to a pediatric population.
    Therefore we are striving to understand:
    a. What validated assessments are needed or exist for the pediatric 
population being treated?
    b. What surrogate markers or endpoints are needed for each disease?
    c. What surrogates are needed or are available to determine long-
term outcomes?
    d. How do we validate surrogate endpoints?
5. How Do We Know That the Study and the Treatment Are Successful?
    Assessment and judgment of patient outcomes varies considerably for 
a pediatric population. The needs of the patient and his caregiver or 
parent must be considered. The longevity of, and durability of, devices 
captures a new meaning when the lifespan is 50 to 60 years; remaining 
lifespan in adults is very different. We are soliciting feedback on:
    a. What constitutes successful or unsuccessful treatment outcomes?
    b. What criteria should be used to determine successful or 
unsuccessful treatment outcomes?
    c. What human factors in each case need to be considered?
    d. What patient factors unique to the pediatric population have to 
be considered?
    e. What criteria are required to acknowledge that successful 
treatment for a patient has been achieved?
    f. What constitutes a successful clinical trial?
    g. How long should a device or treatment last to be considered 
effective?
    Please note funding options for research have already been 
discussed at prior public meetings and will not be discussed at this 
workshop. Information regarding funding sources is available on 
government Web sites as well as other public Web sites dedicated to 
pediatric health.

V. Where Can I Find Out More About This Public Workshop?

    Background information on the public workshop, registration 
information, the agenda, information about lodging, and other relevant 
information will be posted, as it becomes available, on the Internet at 
http://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/default.htm.

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Organization and Basic Instructions for Comments

    To facilitate information gathering, we invite written comments on 
the questions presented in section IV of this document. We intend to 
discuss and expand on these same questions during the small group 
discussions. If you wish to comment in writing on a particular 
question, please identify the question that you are addressing before 
providing your response to the question. For example, your comment 
could take the following format:
    ``Question 1--[Quote the question].''
    ``Response--[Insert your response].''
    You do not have to address each question. Additionally, for those 
questions pertaining to the prevalence of a particular need, problem or 
scientific question, please provide data and/or references so that we 
may understand the basis for your comment, figures, and any assumptions 
that you used. Additionally, the goal of this public workshop is to 
gain a greater understanding of treatment needs and needs for 
innovative solutions to those needs. Accordingly, we look forward to 
participation and comments from manufacturers, innovators, and 
organizations that either market or have in development technologies 
that could be used to treat pediatric patients.

    Dated: September 3, 2009.
Catherine M. Cook,
Associate Director for Regulations and Policy, Center for Devices and 
Radiological Health.
[FR Doc. E9-22012 Filed 9-11-09; 8:45 am]
BILLING CODE 4160-01-S