Agenda
November 15, 1999
Ethical Issues
8:00 a.m. Call to Order/Introductions
P. Joan Chesney, M.D., Pediatric Advisory Subcommittee
Chair
Conflict of Interest Statement
Jayne E. Peterson, R.Ph., J.D., Executive Secretary
8:10 a.m. Welcome and Review of Meeting Agenda
Background Information and Overview
Dianne Murphy, M.D., Associate Director of Pediatrics,
Center for Drug Evaluation and Research (CDER), FDA
8:25 a.m. Compliance Issues
Paul Goebel, Associate Director for Human Subject
Protection, CDER, FDA
8:40 a.m. Institutional Review Board Issues
Susan Kornetsky, M.P.H., Children's Hospital, Boston, MA
8:55 a.m. Pharmaceutical Research and Manufacturer's
Association
Stephen Spielberg, M.D., Ph.D., Janssen Research
Laboratories, Titusville, NJ
9:10 a.m. Investigator Comments
Ralph Kauffman, M.D., The Children's Mercy Hospital,
Kansas City, MO
9:25 a.m. Questions and Comments from the Advisory
Subcommittee
10:00 a.m. Break
10:15 a.m. Presentation of Case Studies/Questions
Dianne Murphy, M.D.
10:30 a.m. Topic Presentations
· Research on Healthy Children: History
Jeffrey Botkin, M.D., M.P.H., University of Utah
· Benefit in Pediatric Research
Norman Fost, M.D., M.P.H., University of Wisconsin
· Risk in Pediatric Research
Benjamin Wilfond, M.D., National Institutes of Health
· Assent/Consent/Permission
Ellen Clayton, M.D., J.D., Vanderbilt University
· Compensation
Jonathan Rackoff, National Institutes of Health
· Non-Beneficial Research with Relatively Sick
Children
Eric Kodish, M.D., Case Western Reserve University
12:30 p.m. Lunch
1:30 p.m. Open Public Hearing
(**30 minutes allocated unless public participation
does not last that long.)
2:00 p.m. Committee Discussion - Case Studies/Questions
Case Study No. 1
Case Study No. 2
3:30 p.m. Break
3:45 p.m. Case Study No. 3
Case Study No. 4
Case Study No. 5
5:25 p.m. Closing Remarks
Dianne Murphy, M.D.
5:30 p.m. Adjourn
Agenda
November 16, 1999
Issues Regarding a Pediatric Drug
Development Program for the Treatment of Insomnia
8:00 a.m. Call to Order/Introductions
P. Joan Chesney, M.D., Subcommittee Chair
Conflict of Interest Statement
Jayne E. Peterson, R.Ph., J.D., Executive Secretary
8:05 a.m. FDA Introduction/Overview of Issues
Thomas Laughren, M.D., Acting Deputy Director,
Neuropharmacological Drug Products, CDER, FDA
8:10 a.m. General Pediatrician Perspective
Leslie Clapp, M.D., Main Pediatrics, Buffalo, NY
8:25 a.m. Child Psychiatrist Perspective
Richard Malone, M.D., Eastern Pennsyl. Psychiatric
Institute, Philadelphia, PA
8:40 a.m. Expert Perspective
Richard Ferber, M.D., Children's Hospital, Boston, MA
8:55 a.m. American Academy of Child and Adolescent
Psychiatry (AACAP)
Mark Riddle, M.D., Johns Hopkins Medical Institutions,
Baltimore, MD
9:10 a.m. Pharmaceutical Industry Perspective
Stephen Spielberg, M.D., Ph.D., Janssen Research
Laboratories, Titusville, NJ
9:25 a.m. Epidemiologic Issues: Office of
Post-Marketing Drug Risk Assessment (OPDRA)
Carolyn McCloskey, M.D., M.P.H., CDER, FDA (presenting)
Amarilys Vega, M.D., M.P.H., CDER, FDA
9:45 a.m. Break
10:00 a.m. Open Public Hearing
(**30 minutes allocated unless public participation
does not last that long.)
10:30 a.m. Advisory Subcommittee Discussion of
Questions
12:30 p.m. Adjourn
Case Studies/Questions to the
Subcommittee
November 15, 1999
The following scenarios are based on proposals received at
the FDA and represent potential ethical questions.
1. A manufacturer wishes to taste test a new elixir
formulation of an antibiotic that has been approved for use in adults. The intended study
population is asymptomatic, healthy children. The study design is to provide each child
with a single dose of the antibiotic, observe for one hour and record reactions. For
children who are capable, a short questionnaire about taste tolerance and palatability
will be given.
A. Does this study exceed the threshold of a "minor
increase over minimal risk"?
B. Would any precautions or exclusions minimize risk?
C. Could this study be done in children who cannot give
assent (under a certain age)?
D. Would it make a difference if the children had a
disease potentially responsive to this therapy?
E. Would it make a difference if this were an
investigational drug (not approved in adults or children)?
2. A sponsor has developed a new formulation of an
anticonvulsant approved for use in adults. The intended study population is asymptomatic,
healthy children. The study design is to provide each child with a single dose of the
anticonvulsant, observe, and obtain one or two blood samples for participation in a
population pK study.
A. Does this study exceed the threshold of a "minor
increase over minimal risk"?
B. Would any precautions or exclusions minimize risk?
C. Could this study be done in children who cannot give
assent (under a certain age)?
D. Would it make a difference if the children had the
disease for which the drug is indicated in adults?
E. Would it make a difference if this were an
investigational drug (not approved in adults or children)?
F. If the pharmacokinetic design was to obtain samples at
1, 2, 4, 6, 8, 10, 12, 18, and 24 hours, would you allow the study to proceed or place any
restrictions on the study?
G. Would your answers to A through F be different if the
formulation under study were an antihistamine instead of an anticonvulsant?
Case Studies/Questions to the
Subcommittee (cont.)
November 15, 1999
3. A sponsor has developed a new formulation of an
ophthalmic agent approved for use in adults. The intended study population is
asymptomatic, healthy children ages 3-8. The study design is to provide each child with a
single dose of the ophthalmic agent in the eye, observe for 2 hours for adverse events
and, if no adverse events are noted, then progress to a multidose 6 week study. It is not
known if such agents would have any unique impact on visual acuity in this age group where
visual acuity is still developing.
A. Does this study exceed the threshold of a "minor
increase over minimal risk"?
B. Would any precautions or exclusions minimize risk?
C. Could this study be done in children who cannot give
assent (under a certain age)?
D. Would it make a difference if this were an
investigational drug (not approved in adults or children)?
4. A sponsor is developing a new MRI contrast agent and
wishes to test safety and tolerance in children. The study design is to administer one
dose of the intravenous contrast agent to hospitalized children with indwelling catheters,
or previously established intravenous access, and observe the children for two hours
A. Does this study exceed the threshold of a "minor
increase over minimal risk"?
B. Would any precautions or exclusions minimize risk?
C. Could this study be done in children who can not give
assent (under a certain age)?
D. Would it make a difference if this were an
investigational drug (not approved in adults or children)?
E. Would your answers to A through D be different if
children were being admitted for placement of drainage tubes in the ear and, instead of a
new MRI contrast agent, an investigational antibiotic were to be given prior to the
surgery with subsequent sampling of middle ear fluid and serum?
5. What is the impact of compensation on parent/child
permission/assent:
A. Would compensation unduly influence a child's assent?
Should a child be aware/told of compensation prior to giving assent?
B. Does compensation compromise a parent's permission to
allow participation of their child in a clinical trial? How would the nature, amount, and
recipient of the compensation affect this decision?
Questions to the Subcommittee
November 16, 1999
1. Is there a public health need for FDA to encourage
(through written requests) or require (as described in the final rule) that hypnotic
medications be considered a therapeutic option to treat insomnia in the pediatric
population? If yes, what are the age ranges that it would be appropriate to study?
2. Is there any other sleep disorder in the pediatric age
group that presents a public health need requiring drug development of hypnotics in this
population? If so, are there other issues that should be considered in the design/conduct
of the pediatric studies?
FDA/Center for Drug Evaluation and Research
Last Updated: March 08, 2001
Originator: OTCOM/DML
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