This is a brief summary of PREA Waivers. It is a general overview but hlepful nonetheless.

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4/9/2015
Waivers and Deferrals of
Pediatric Studies under PREA

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Recent Laws Affecting Pediatric
Drug Development
 Pediatric Research and Equity Act (PREA) of 2002
 Best Pharmaceuticals for Children Act (BCPA) of 2002
 Both PREA and BCPA reauthorized under Title IV of
FDAAA (FDA Amendments Act)
 Both PREA and BCPA made permanent under FDA
Safety and Innovation Act (FDASIA)
4/9/2015

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The Goal of All of These Legislative Acts
 Assure that children receive the same attention from
Sponsors previously reserved for adults
 Incentivize drug developers to comply with
these mandates
 Develop product labeling that contains pre-marketing
information for children as well as adults
4/9/2015

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The requirement for Pediatric Studies under
PREA is triggered by the following:
 New Indication
 New Dosage Form
 New Dosing Regimen
 New Route of Administration
 New Active Ingredient
4/9/2015

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When is it reasonable to defer
pediatric studies until after approval?
 The drug/biologic is ready for approval for use in adults
before pediatric studies are complete OR
 Pediatric studies must be delayed to collect additional
safety or effectiveness data OR
 There is another appropriate scientific reason for
deferral (e.g., lack of agreement in the medical
community regarding study design or endpoints)
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PREA - Deferral Requirements
 Certification of the grounds for deferring the
assessments
 A Pediatric Study Plan
 Evidence that the studies are being conducted or will
be conducted with due diligence and at the earliest
possible time
 A timeline for the completion of such studies
The Sponsor must submit all of the following:
4/9/2015

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When is a waiver of pediatric
studies appropriate under PREA?
4/9/2015

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PREA: Waiver Criteria
 Necessary studies are impossible or highly
impracticable (Section 505B(a)(4)(A)(i)) OR
 Evidence strongly suggests the drug/biologic would be
ineffective or unsafe (Section 505B(a)(4)(A)(ii)) OR
 Drug/biologic does not represent a meaningful therapeutic
benefit over existing therapies for pediatric patients AND is
not likely to be used by a substantial number of pediatric
patients (Section 505B(a)(4)(A)(ii)) OR
 Reasonable attempts to produce a pediatric formulation
necessary for that age group have failed (partial
waiver only)
4/9/2015

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PREA: Waiver and Deferrals
 Office of New Drugs Review Divisions and Sponsors
should discuss PREA requirements early in the drug
development process
 Pediatric Study Plan needs to include plans to request
deferrals, waivers or partial waivers with supporting data
 Final deferral and waiver decisions are made at the time
of NDA/BLA approval
4/9/2015

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How Can Clementi & Associates Help?
 We can review the product / profile to determine if a
Waiver or Deferral are justifiable
 If appropriate, we can prepare a written justification for
the Waiver or Deferral
 If needed, we can support interactions with the
regulatory authority to defend the Waiver or Deferral
request
4/9/2015