Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Potency Assignment of Factor IX Medicinal Products for the Treatment of Hemophilia B: Regulatory meets Practice ***

Dr. Oliver Walisko (Abschlußjahr: 2019)

Summary
Language: English
The determination of a biological product´s potency is crucial for its safe and effective application in clinical practice.
While the legislative as well as scientific foundations for potency assays have been long established, they are challenged by the emergence of new generation (long-acting) FIX products for the treatment of hemophilia B due to the occurrence of assay discrepancies when the FIX International Standard (IS) Concentrate is used as reference.
In the light of the 2013 NIBSC collaborative study that has systematically compared new generation FIX products in potency assays, this thesis is presenting and discussing the consequentially implemented regulatory measures aiming to maintain the WHO FIX IS Concentrate as reference for potency labelling of new FIX products.
As these regulatory measures have been implemented prior to granting marketing authorization of long-acting FIX products, the MAH´s strategy chosen for potency labelling is presented and discussed based on publically available information contained in the product´s SmPCs.
In addition, potential issues concerning the One-Stage Clotting Assay are raised with regard to assay validity when used for potency assignment of recombinant and long-acting FIX products against the WHO FIX IS Concentrate.
Pages: 40