Oxford BioMedica PLC (OXBDF)

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In the coming months, we also intend to further clarify our approach to the regulation of gene therapy. We’re going to provide a comprehensive framework that will include new guidance on manufacturing standards and approaches; as well as clinical guidance that lays out development pathways and potential accelerated approval endpoints for products that target specific disease areas like haemophilia.
We’ve reached an inflection point when it comes to gene therapy. The key enabling technology was the advent of more reliable vectors to deliver the target genes.
We’re now at a turning point where these treatments have the potential to become a backbone of modern medicine.

It’s similar to the inflection point when we first perfected the humanization of antibodies. It was a turning point in the use of antibodies as drugs. In a similar way, the advent of the lentiviral vector is an analogous product innovation that has opened up a huge clinical opportunity. Gene therapies using lentiviral and certain other vectors now hold out the promise of curative treatments for many vexing disorders.

Our obligation is to design a framework for the evaluation of these products that enables the efficient advance of these opportunities, while taking steps to ensure their safety.

When it comes to gene therapy, often the hard questions aren’t around efficacy. Many of these treatments look like they’ll be highly effective. Efficacy is often established early in the course of development, and in small series of patients.
The critical questions don’t necessarily turn on the clinical portion of the review, but on the product related issues.

These include the theoretical risks from off target effects, and on the durability of the response. They turn on complex issues associated with manufacturing and gene delivery.

A lot of the IP is also embedded in the manufacturing processes. And in some cases, we may be seeing product developers re-engineer these manufacturing processes in ways that may be geared less toward improving the products, and more toward circumventing some of the stacked royalties that exist in this field. Our job is to make sure these manufacturing changes don’t also introduce new risks. So we need to make sure we’re applying proper attention to all of these product-related regulatory issues.

In short, some of the hardest regulatory questions may turn less on the clinical evaluation of the technologies and more on the product specific and manufacturing issues that relate to the long run performance of these constructs.
This requires us to re-think our regulatory model when it comes to these products, to make sure we’re taking an efficient and science-based approach. Where the critical questions turn on long-term safety and durability, we need adequate policies to evaluate these products post approval.

For gene therapies that target serious conditions and offer potentially curative effects, the accelerated approval process may be ideally suited to allowing us to achieve a twin purpose – to expedite market entry while, at the same time, ensuring that we have strong post-market tools to continue to evaluate the safety of these product effects post approval.

Keynote Addresss by Commissioner Gottlieb to the 2018 FDLI Annual Conference
Speech by Scott Gottlieb, M.D.
Commissioner of Food and Drugs
Keynote Address at the 2018 Food and Drug Law Institute Annual Conference
Washington, DC
May 3, 2018
HTTPS://www.fda.gov/NewsEvents/Speeches/ucm606541.htm