Pediatric Study PlansA High Level View

DANIEL J. ISAACMAN, M.D., FAAPPROFESSOR OF PEDIATRICS, DREXEL UNIVERSITY SCHOOL OF MEDICINEPRESIDENT, CLINICAL PHARMA SOLUTSIONS, LLC

History of FDA Initiatives to Foster Pediatric Drug Development

1979 - Labeling Requirement

1994 - Final Rule: Pediatric labeling-extrapolation of efficacy

1997 - FDAMA/Pediatric Exclusivity Provision

1998 - Final Rule: Pediatric Studies Required

2001 - Subpart D: Additional Safeguards for Children in Clinical Investigations of FDA-regulated products

History of FDA Initiatives to Foster Pediatric Drug Development (cont)

2002 - Best Pharmaceuticals for Children Act

2003 - Pediatric Research Equity Act

2007 - Both Reauthorized under FDAAA

2012 - Both Made Permanent under FDASIA

Purpose of PREA (Pediatric Research and Equity Act)

Encourage sponsors to identify pediatric studies as early as possible in product development

Facilitate making pediatric data available to the public as soon as possible

Timeline for Submission of Pediatric Study Plans

How Much Detail is Required in the Initial Pediatric Study Plan?

Should provide an outline of all intended studies

General plans should be described, but full protocols not required

Protocol and statistical analysis plan must be submitted prior to initiation of each particular study in the plan

Ideally, studies should be completed before the submission of the NDA, BLA or Supplement

What is Expected in the Pediatric Study Plan (PSP)

What is Expected in the PSP (cont)

What is Expected in the Pediatric Study Plan (cont)

Assistance We Can Provide

Drafting of the PSP

Advice whether or not your given product qualifies for a Waiver from the PSP

Drafting of Waiver Request

Planning of Individual Studies

Drafting of Individual Study Protocols

Drafting of Informed Consent Forms

Recruitment of Study Investigators