Evaluation of safety, efficacy and immunogenicity of a recombinant FVIII molecule

We propose a meta-analytic project which aims at summarizing Post-Authorization Surveillance Safety data on Advate, a recombinant FVIII concentrates used to treat patients with Hemophilia A. The study will involve the largest cohort of hemophilia patients on FVIII concentrates analyzed in the literature. The project represents an attempt to sustain a methodologically strong approach in the field of Hemophilia, as paradigm of rare disease. We will accompany a classic and more common metaanalytic modeling with a Bayesian modeling, able to provide measures of efficacy and safety after repetitive simulations with a probabilistic construct resembling the physician’s decisional process. The partner organization will benefit from a state-of-the-art assessment of the safety profile of their prodict in a post-authorization context, which will facilitate plans for the development of future molecules and will be used for lobbying with the regulators in improving the registration process.

Faculty Supervisor:

Drs. Lehana Thabane & Alfonso Iorio

Student:

Maura Marcucci & Ji Cheng

Partner:

Baxter Corporation Canada

Discipline:

Biochemistry / Molecular biology

Sector:

Life sciences

University:

McMaster University