www.biopharminternational.com March 2021 eBook BioPharm International 35
These case studies also exem-
plify how pre-competitive efforts—
leveraging data and k nowledge
exchange from collaborating phar-
maceutical companies, regulatory
agencies, and contract research
organizations—contribute to the
implementation and reg ulator y
accept a nce of i n novat ive a nd
well-justified scientific approaches.
The PWG within IQ-CPLG has
leveraged its forum to facilitate
multi-stakeholder discussions to
advance the thinking on its applica-
bility in modern drug development.
Due to the diverse expertise and
perspectives of PWG members, this
forum has helped foster the evolu-
tion on the implementation of quan-
titative and Bayesian approaches,
and other methodologies for use in
pediatric extrapolation. The influ-
ence can be seen not only within
each member's respective companies
and the impact on pediatric develop-
ment plans, but also on policy-mak-
ing in global pharmaceutical trade
organizations (European Federation
of Pharmaceutical Industries and
A s s o c i at io n s , P h a r m a c e ut ic a l
R e s e a r c h a n d M a nu f a c t u r e r s
of A mer ica, or Biotec h nolog y
Innovation Organization) and deci-
sion-making at regulatory agencies
(e.g., Entresto [sacubitril/valsartan] 2019
FDA supplemental new drug application
approval for the treatment of symp-
tomatic heart failure with systemic
left ventricular systolic dysfunction
in pediatric patients aged one year
and older) (16).
Importantly, the International
Council for Harmonization (ICH)
is work ing towar d a global consen-
sus guideline intended to provide
detailed guidance (17) on the use
of pediatric extrapolation to sup-
por t successf u l accelerat ion of
marketing authorizations. Some
members of the PWG have overlap-
ping roles in the pediatric extrapo-
lation ICH E11A expert work group
and have been able to leverage and
incorporate consistency to what is
expected to be an influential inter-
national consensus guidance.
It is the authors' perspective that
the IQ CPLG PWG has played a cru-
cial role in facilitating information
sharing and influence on the appli-
cability and appropriateness of the
pediatric extrapolation framework,
which has the potential to hasten
access of medicines to children.
REFERENCES
1. H. Sun, et al., Ther Innov & Regul Sci. 1–7
(Aug. 18, 2017).
2. EMA, EMA Public Workshop on
Extrapolation of Efficacy and Safety in
Medicine Development (London, 2016).
3. ICH, E11(R1) Guideline on Clinical
Investigation of Medicinal Products in the
Pediatric Population, Step 5 version (2017).
4. AAP, "Update: Anti-Epileptic Drug Efficacy
In Adults Can Be Extrapolated to Pediatric
Patients," (April 6, 2016).
5. IQ Consortium, "Challenges and
Opportunities in Extrapolation," Pre-
conference at ASCPT meeting (March 21,
2018).
6. IQ Consortium, "Strategies to Address
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7. IQ Consortium and M-CERSI, "Pediatric
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8. J. S. Barrett, et al., Clin Pharmacol Ther
103 (3) 419–433 (2018).
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11. E. G. Yan and K. M. Munir, Ethics and
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15. C. Bucci-Rechtweg, Clinical Ther 39 (10)
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16. Novartis, "Novartis Entresto Receives
FDA Aapproval for Pediatric Heart Failure,
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01, 2019.
17. ICH, "Final Concept Paper Pediatric
Extrapolation," Endorsed by Management
Committee (Oct. 17, 2017).
BP
ABOUT THE AUTHORS
Sebastian Haertter*, sebastian.
haertter @boehringer-ingelheim.
com, is clinical pharmacology expert
(director) translational medicine and
clinical pharmacology, Boehringer
Ingelheim; S.Y. Amy Cheung is
senior director, Certara; Angela
James is director, Astellas Pharma
Global Development; Ashley Strougo
is head of M&S Germany, pharma-
cokinetics, dynamics and metabo-
lism, translational medicine, Sanofi;
Christina Bucci-Rechtweg is global
head, pediatric and maternal health
policy, Novartis Pharmaceuticals
Corporation; Jing Liu is senior
director, clinical pharmacology,
Pfizer; Meina Tao Tang is princi-
pal scientist, clinical pharmacology,
Genentech, Inc.; Paulien Ravenstijn
is senior consultant, qPharmetra
LLC; Raafat Bishai is global clini-
cal program lead, AstraZeneca;
Satyendra Suryawanshi is director,
clinical pharmacology and pharma-
cometrics, Bristol Myers Squibb; and
Solange Corriol-Rohou is senior reg-
ulatory affairs and policy director for
Europe, CMO office and chair of AZ
Paediatric Work Group Regulatory
Excellence, AstraZeneca; all authors
are members of the IQ Consortium.
* To whom a l l cor resp ondence
should be addressed.
Regulatory Sourcebook Quality Collaboration
Extrapolation
supported the
successful approval
of a pediatric
indication for dasatinib
and informed dosing
and posology.
