This document summarizes the experiences of Camargo Pharmaceutical Services in negotiating initial Pediatric Study Plans (iPSPs) with the FDA for drug products using the 505(b)(2) regulatory pathway. It presents three case studies demonstrating different strategies used to minimize or waive pediatric study requirements based on available data. For the first case, a literature-based NDA for an IV product, the agreed iPSP included a post-marketing PK and safety study in adolescents and a deferred juvenile toxicity study. The second case involved an oral formulation, where partial waivers for certain age groups were granted based on safety concerns and literature demonstrating a lack of meaningful therapeutic benefit. The third case involved an IV product with changed excipients

1. We reviewed our works and present our
experience in negotiating initial PSPs with
the Food and Drug Administration (FDA).
Preparation of Pediatric Study Plans for Drug Development Programs
Using the 505(b)(2) Pathway
Vien Lai, Loan Pham, Eric Kendig, Marc Wiles, Ruth Stevens – Camargo Pharmaceutical Services, LLC
APPROACH
RESULTS
CONCLUSION
RESULTS
The proposed iPSP included:
 Age 0 – 12 years: A partial waiver based on literature data
showing low prevalence [referencing section 505B(a)(4)(B)(i)
of the FD&C Act]
Case Study 2: A new oral formulation
The strategy included:
 Ages 0 – 6 years: A partial waiver based on the
Agency’s finding that the LD was unsafe in this age group
[referencing section 505B(a)(4)(B)(ii) of the FD&C Act]
PURPOSE
Drug B is a modified released oral formulation.
The listed drug (LD) was an immediate-release formulation.
Poster
Number
04T0100
Identify strategies
to minimize pediatric
requirements under
PREA for products
that use the 505(b)(2)
regulatory pathway.
Pediatric Research Equity Act (PREA)
Best Pharmaceuticals for Children Act (BPCA)
How to Comply with PREA
(FDA Draft Guidance, 2005)
Qualifying for Pediatric Exclusivity
History
Who Needs a Pediatric Study Plan?
• A new active ingredient
• A new indication
• A new dosage form
• A new dosing regimen
• A new route of administration
A PSP is required unless
the drug was granted
orphan designation for
the proposed indication.
Drug A is an intravenous (IV) product with a planned literature-based 505(b)(2)
NDA. Drug A has been marketed for years as an unapproved product.
 Ages 12 to ≤ 17 years:
 Extrapolation of efficacy data from adults to pediatric patients based on
literature data showing similarity of disease course and drug effect
[referencing section 505B(a)(2)(B)(i) of the FD&C Act]
 Safety data was referenced from the published literature.
Agreed Pediatric Study Plan:
 One post-marketing pharmacokinetic (PK) and safety study in patients ages
12 – 17 years (28 subjects)
 One juvenile toxicity study in rats (deferred)
Agreed Pediatric Study Plan:
 No clinical or nonclinical studies were needed
 Ages 6 to ≤ 17 years: A partial waiver because the proposed product did not
represent a meaningful therapeutic benefit over existing and is not likely to be
used in a substantial number of pediatric patients [referencing section
505B(a)(4)(A)(iii) of the FD&C Act]
Many regulatory strategies
exist that may allow Sponsors
employing the 505(b)(2) to
avoid unnecessary pediatric
studies. When evaluating the
strategy for a PSP, it is critical
to identify all potential
strategies based on available
information to guide the unique
strategy for each drug product.
Case Study 3: Change in
excipients for an IV product
Drug C is an IV product
identical to the LD in the
active ingredient, indication,
dosing regimen, and route of
administration. Drug C does
not qualify as a generic due
to the change in excipients.
Agreed Pediatric Study Plan:
 Not needed; PREA was not
applicable per agreement in the PIND
Meeting
Strategy:
 In vitro binding study showed similar
binding constants and free drug release
profile compared to the LD.
 Data from the published literature to
justify that substitution of the solubility
enhancer would not alter PK, efficacy,
or safety of the drug.
 BA/BE was deemed by the Agency to
be self-evident and in vivo BA/BE
studies were waived.
Case Study 1: The active ingredient has never been approved