1. Alfonso Iorio, on behalf of the working group
Working group on Pharmacokinetics and Population Pharmacokinetics of factor concentrates
Alfonso Iorio, on behalf of the working group

2. DISCLOSURES Financial conflicts of interests
McMaster University has received funds for research and service agreements from Bayer, Baxalta, Biogen, NovoNordisk, Octapharma, and Pfizer.
Non-financial (intellectual) conflicts
PI for the WAPPS project (
No mention of unlicensed products

3. WORKING GROUP Core members Additional members PK and PopPK consultants
A Iorio, V Blanchette, P Collins, K Fischer, J Blatny, E Neufeld
Additional members
D Lillicrap, D Hart, M Makris
PK and PopPK consultants
A Edginton, S Ito

4. Previous SSC Communications
The design and analysis of half-life and recovery
studies for factor VIII and factor IX.
Factor VIII/Factor IX Scientific and Standardization Committee of the International Society for Thrombosis and Haemostasis.
Morfini M, Lee M, Messori A.
Thromb Haemost Sep 2;66(3):384-6.

5. Previous SSC Communications
Scientific and Standardization Committee Communication
The Design and Analysis of Pharmacokinetic Studies of
Coagulation Factors
On behalf of the Subcommittee on Factor VIII and Factor IX of the Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis
M. Lee, M. Morfini, S. Schulman, J. Ingerslev
and the Factor VIII/Factor IX Scientific and Standardization Committee of the ISTH
Posted on the ISTH SSC on March 21, 2001

6. Previous SSC Communications
… the approaches recommended here are primarily intended for clinical trials of factor concentrates that are being compared to other, similar preparations. … studies on individual patients for diagnostic or therapeutic purposes may not necessarily be relevant to what is discussed here.

7. Previous SSC Communications

8. AIMS: Primary PK and PopPK estimates of factor concentrates: Reporting
Interpretation
Assessment in clinical practice
Study design for “real world/PM” PopPK assessment

9. PK and PopPK estimation:
AIMS: Secondary
PK and PopPK estimation:
During registration phase
To compare different molecules
Choice of time points ….

10. How (and whether) to best involve:
PROCESS
How (and whether) to best involve:
Pharmaceutical companies PK/PopPK experts
FDA / EMA PK/PopPK experts
Authors of PK/PopPK published papers
Patient representatives

11. PROGRESS
2 conference calls
1 panel survey
Bi-monthly conference calls
Review of the existing literature
Review of regulators guidance
Draft recommendations on primary goals

12. Thank you for attention
Questions time
Thank you for attention
Slides posted on: