Dr. Rosenberg: My parents were physicians. I became a physician. And I realized along my training ways that if I didn’t understand research and basic immunology and how that could apply to novel therapeutics, that I wasn’t going to do anything substantive.

Text Slide:

Amy Rosenberg, M.D.

Director

Division of Biotechnology Review and Research III

Office of Pharmaceutical Quality

Office of Biotechnology Products

Dr. Rosenberg: The mission of the Office of Biotechnology Products is to ensure the safety and effectiveness of the products that we regulate by evaluating product quality and consistency.

We regulate products that are generally derived from cells and they are protein therapeutics.

A lot of the problem with these products comes from the fact that because these look like foreign proteins to a patient, patients make immune responses to these proteins, which can nullify their effect or give them allergic type reactions.

This is the emphasis on improving patient outcome by engineering our therapeutic proteins for optimal effectiveness and safety. We try and foster research that helps our reviewers to really understand cutting-edge issues so that we can move forward. This research gives us the critical background to be able to identify therapeutics that might be outstandingly more effective than the ones that we currently have called breakthrough products, that we work with industry, that we work with sponsors, patient groups to improve our therapeutics to address central nervous system disease, muscle disease. The worst possible situation is where patients who have unmet medical needs can’t get a lifesaving therapeutic.

So modifying these proteins, these therapeutic proteins so that they don’t evoke an immune response. Because we know that that interferes with the therapeutic efficacy.

We’re modifying the patient’s immune response to the therapeutic via tolerance induction. So that’s what I mean by biobetters.

And biobetters, they’re twice blessed. Not only will they improve patient outcome, but economically they will help in deferring cost or in reducing the cost of these therapies by keeping patients healthier and having less of the terrible manifestations of their disease.

With us the focus is really on improving patient outcomes in whatever way we can do that. And so having that focus really resonates with me, having that as a sole focus and not any kind of monetary bottom line is for me what’s critical.