Interview with William Kennedy, MD, Senior Medical Director at Genentech

1. Addressing Core Operational Issues When Conducting Pediatric Clinical Trials with
Genentech
Interview with William Kennedy, MD, Senior Medical Director at Genentech
Increased pressure to conduct trials in children can be daunting
when faced with practical, legal and clinical considerations found
only in pediatric trials. Successfully implementing regulations and
ethical standards into study conduct requires a comprehensive
understanding of the unique concerns raised by ethics committees,
investigators and parents.
William Kennedy answered a series of questions written by marcus
evans before the forthcoming 9th Annual Pediatric Clinical Trials
Conference, April 24-26, 2013 in Philadelphia, PA. William shares
the challenges that came along with conducting pediatric clinical
trials. The responses below strictly reflect the views and beliefs of
William Kennedy and not necessarily those of Genentech.
What is the top challenge faced when designing clinical trials for pediatrics?
William Kennedy: I have been struck by the impact of medical associations in the field of
Rheumatology, the American College of Rheumatologists (ACR) and the European League Against
Rheumatism (EULAR) in particular, to enjoin industry efforts to develop new medicines. This has led
to the introduction and acceptance of new trial endpoints, ACR and DAS, in rheumatoid arthritis, the
development of new endpoints in lupus, SLE Responder Index, efforts to develop instruments to
measure changes in disease activity in lupus and changes in flare activity in lupus, in funding
consortia to collect academic and industry placebo trial data to understand variable placebo
responses across trials and to bring industry, academic and other interested parties together
through annual meetings to discuss how to make advances in clinical trials generally. As the
compliance rules for work between academic clinicians/scientisits and pharmaceutical industry
researchers become more regulated, the medical associations will become more important as
independent bodies that can guide and protect the scientific exchange of ideas and global
connections that are the lifeblood of scientific advances.
I believe a similar effort by the American Academy of Pediatrics would be invaluable for the work to
develop new medicines for children beyond vaccines. For example, there are no recognized clinical
trial endpoints for the evaluation of the benefits of pharmacotherapy in RSV trials; it is not much
better for hospitalized adults with influenza. Pharmaceutical industry researchers may have some
internal guidance and some external regulatory guidance for how they might develop a drug for
RSV, but case-by-case programs will be limited and standardization of methodologies will be lacking.
The AAP could be instrumental by advocating for appropriate drug development in children, through
coordination of annual meetings to include American and European regulatory colleagues with

2. interests in safety and pediatric medicines, and sponsorship of registries and initiatives that build
medical epidemiological frameworks for pediatric diseases and drug development.
Given that there is much pressure to streamlining drug development, what have you
found successful when incorporating alternative endpoints into the study?
WL: I anticipate the difficulty of success from the medical, regulatory and commercial sides early
enough that when the results come out, others will be able to focus not on what the results mean,
but on how they will effectively share that information for the new medicine as it moves forward in
development.
What have you found useful when standardizing how and when endpoints are measured
in multi-site trials?
WK: Intensive training of all investigators in clinical meaning of the endpoints, how the components
are measured, how much the quality of the information impacts the outcome of the study, use of
central, independent groups (laboratory, imaging readers, EEG interpretation, QT waveform
analysis, etc.) with validated tools with known intra- and inter-observer or measurement variability.
Periodic re-training, regional or selected site visits, sharing of interim safety data, weekly
teleconferences, willingness to amend protocols based on investigator feedback on study outcome
and detailed attention to the ongoing conduct of the trial are useful tools to consider.
How has the renewal of the Best Pharmaceuticals for Children Act (BCPA) and the
Pediatric Research Equity Act (PREA) affected your programs at Genentech?
Broadly, the renewal of these Acts is good for Pediatric Medicine and drug development. The
neonatal development issues brought forward in the Acts’ renewal is especially important.
Specifically, autoimmunity development programs at Genentech are currently not working in the
arena of neonatology as part of pediatric development. This could change in the future.
William Kennedy has been in Early Clinical Development at Genentech for 3 years conducting clinical
trials in rheumatoid arthritis and lupus. Prior to that, he worked in Clinical Pharmacology at Merck
for 4 years. Mr. Kennedy completed my Pediatric Infectious Disease Fellowship at Yale Medical
School in 2001 under Dr. George Miller and was attending physician in Pediatric ID from 2001-2003.
For more information please contact Michele Westergaard, Senior Marketing Manager, Media & PR,
312-540-3000 ext. 6625 or Michelew@marcusevansch.com.
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