Pharmaceutical Outsourcing Q2 2024 - 27

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Nazim Kanji, Executive Director, Pediatric Services, Quotient
Sciences: Based on our experience working with sponsor
companies, we see increased interest in developing a wide and
full range of liquid and solid dose formats. This indicates the many
drivers and preferences for the development of bespoke, ageappropriate
dosage forms. There appears to be a preference for a
single formulation which can be flexibly dosed to a wide age range
of children and in dosage forms such as solutions, suspensions,
powder for reconstitution, minitablets, and sprinkles.
One key factor in determining the product development strategy
is to understand the taste attributes and palatability of the active
pharmaceutical ingredient (API). We are seeing an increasing trend
of taste characterization studies on APIs, with no prior taste data,
in our Phase I clinics in the USA and UK before the commencement
of formulation development activities. The data from these taste
studies helps to inform the formulation and taste-masking strategies
employed in the development of pediatric dosage forms.
Gus LaBella, Director of Formulation Development, Mikart: A
patient-centric approach is preferred when developing dosage
forms to meet the specific needs of pediatric patients. This trend
emphasizes the importance of flexibility in dosing options, such
as adjustable dosages based on weight or age, to ensure optimal
efficacy and safety. Many drug products were developed only for
the adult market leaving pediatric patients to suffer the poor taste
of a ground up tablet for many indications. We see many 505(b)(2)
products now being converted to solutions and suspensions which
are more amenable to the pediatric population. Another trend
includes diversification of dosage forms beyond traditional dosage
forms like tablets or suspensions and syrups. Dosage forms such
as orally disintegrating tablets, chewable tablets, multiparticulate
sprinkles and minitablets provide additional options that are more
suited for pediatric patients.
Can you provide insights into the
key players dominating the pediatric
dosage form market and their respective
market shares?
LaBella:
The key players dominating the market are large
pharmaceutical companies that focus on pediatric dosage
formulations. Some of these include Pfizer, Johnson and Johnson,
GSK, Sanofi, and Novartis. These companies invest significantly in
research and development to maintain market leadership in the
pediatric form segment. In addition to these key players, it's worth
noting that there are smaller mid-sized Contract Development and
Manufacturing Organizations (CDMOs) that offer specialized services
for pediatric dosage forms. These CDMOs can provide biotech and
pharmaceutical companies with tailored contract development and
manufacturing solutions, including formulation development, scaleup,
and commercial manufacturing of pediatric medications.
What are the major challenges faced
by pharmaceutical companies in
developing pediatric dosage forms,
particularly concerning safety,
efficacy, and palatability?
Shanmugam: Pharmaceutical companies are expected to address
pediatric challenges such as dose flexibility, dose accuracy, and
dose convenience while ensuring safety, efficacy, and regulatory
compliance. This requires a significant investment of resources and
time. Achieving a balance between need-based dosing solutions
for pediatrics and economic feasibility is a predominant challenge
faced by pharmaceutical companies.
Ensuring safety and navigating ethical considerations are important
when conducting complex clinical trials, particularly when
managing a limited patient population. Addressing potential longterm
effects on developing systems is crucial. Achieving efficacy
requires formulating precise and flexible dosages suitable for
diverse age groups, from newborns to adolescents, considering
their developmental phases and needs.
Palatability also poses a persistent challenge, requiring the
development of acceptable formulations that mask bitterness or
unpleasant taste of the drug. Regulatory compliance, economic
viability, and the limited patient population for pediatric studies
further compound these challenges.
Addressing these multifaceted issues demands collaboration
among pharmaceutical companies, regulatory bodies, healthcare
professionals, and caregivers to deliver safe, effective, and
acceptable pediatric dosage forms.
Kanji: There has been a consistent demand for more pediatric
medications over the past two decades, especially ones that are
appropriately optimized for young patients. The sponsor and its
outsourcing partner(s) must consider numerous factors to develop
an acceptable pediatric product that achieves clinical, regulatory,
and commercial success.
The initial stage involves defining the target product profile for
intended pediatric patient population(s) and understanding the
associated challenges and risks. The properties of the API (including
taste and palatability), formulation factors including excipient
selection for the target age group, and the administration method
all must be factored in, along with any clinical and regulatory
considerations, dose extrapolation from adult clinical data, and the
target pharmacokinetic (PK) profile.
After formulation development, a clinical assessment of the
proposed pediatric formulations in adult volunteer panels typically
comes next to evaluate and optimize the taste and/or PK attributes
prior to dosing in pediatric patient studies. Once the pediatric
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Pharmaceutical Outsourcing Q2 2024

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