Daniel J. Isaacman, m.D., FAAP

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Presentation transcript:

Waivers and Deferrals for Pediatric Study Plans: Principles of Justification Daniel J. Isaacman, m.D., FAAP Professor of Pediatrics, Drexel University School of medicine President, Clinical Pharma Solutions, LLC

Important Laws Affecting Pediatric Drug Developing Pediatric Research and Equity Act (PREA) Best Pharmaceutical for Children Act (BCPA) FDA Safety and Innovation Act (FDASIA)

The Goal for All of These Pieces of Legislation Assure that children receive the same attention from drug developers that has been previously reserved for adults Incentivize drug developers to comply with this mandate Develop labels that contain pre-marketing drug development information for children as well as adults

PREA is triggered by the following: New Indication New Dosage Form New Dosing Regimen New Route of Administration New Active Ingredient

When Is a Waiver Appropriate Under PREA?

PREA: Waiver Criteria Necessary studies are impossible or highly impracticable OR Evidence strongly suggests the drug/biologic would be ineffective or unsafe 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 OR Reasonable attempts to produce a pediatric formulation necessary for that age group have failed (partial waiver only)

PREA: Waiver and Deferrals FDA review divisions and sponsors should discuss PREA requirements early in the drug development process PSP 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

When is it reasonable to defer pediatric studies? The drug/biologic is ready for approval for use in adults before pediatric studies are complete OR Pediatric studies should be delayed until additional safety or effectiveness data have been collected OR There is another appropriate reason for deferral (e.g., scientific issues exists regarding study design or endpoints)

PREA - Deferral Requirements The sponsor must submit Certification of the grounds for deferring the assessments AND A Pediatric Study Plan AND Evidence that the studies are being conducted or will be conducted with due diligence and at the earliest possible time AND A timeline for the completion of such studies

How can we help? We can review the product profile to determine if it qualifies for a waiver If appropriate, we can then draft a written justification for the waiver If needed, we can meet with the regulatory authority to defend said justification