pediatric PK studies for pediatric exclusivity

From: "Parasrampuria, Dolly [MCCUS]" DParasr@MCCUS.JNJ.com Subject: [NMusers] pediatric PK studies for pediatric exclusivity Date: Fri, December 10, 2004 9:13 am Hello: I wanted to get some input from the group regarding pediatric PK study designs that are required for pediatric exclusivity. If a study is required in a pediatric population (eg. adolescent) that either does not require a clinical study due to sound basis of disease similarity in adults and children or the opportunity to partner in clinical program has passed - what kind of a pediatric study design should one use? Assuming that this study needs to be conducted in healthy children and the drug has a relatively long half life of about 12 to 24 hours A traditional PK study with 8 or more blood draws, (approx sample size 24) OR A population PK approach with a slightly larger sample size but significantly less number of samples (approx sample size of 36 to 48) There are trade-offs for both approaches - cost (pop PK being more expensive than traditional PK; ethical sensitivities and considerations) and enrollment (more will be needed for pop PK) versus reduced number of blood draws and decreased discomfort/distress, potentially easier IRB approval (for pop PK) Appreciate feedback from the group. Best Regards Dolly __________________________________________________ Dolly A. Parasrampuria, PhD Clinical Pharmacology, M/C 263 McNeil Consumer & Specialty Pharmaceuticals 7050 Camp Hill Rd, Fort Washington PA 19034-2299 Tel: 215-273-7760 Fax: 215-273-4127

From: "Suliman Al-Fayoumi" alfayoumi@hotmail.com Subject: RE: [NMusers] pediatric PK studies for pediatric exclusivity Date: Fri, December 10, 2004 12:25 pm Hi Dolly; Both traditional and Pop PK studies have been utilized in the past to generate PK data in pediatrics. However, the use of Pop PK in pediatrics has generally been encouraged by the FDA due to ethical and practical considerations. Prior to embarking on the study, you might want to discuss any proposed study designs with the respective Clinical Division within FDA. In addition, the current regulations mandate that studies intended for pediatric exclusivity may only enroll pediatric patients. I hope you find those comments helpful. Take care. Suliman Al-Fayoumi Clinical Pharmacology Reviewer CDER/FDA _______________________________________________________