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Event Title
Remarks on FDA’s Role in Advancing a Modern Framework for Gene Therapy
December 19, 2017

Remarks by Dr. Scott Gottlieb
Commissioner of Food and Drugs
FDA’s Role in Advancing a Modern Framework for Gene Therapy
Silver Spring, MD
December 19, 2017

As we approach the end of a landmark year in medicine, I’d like to reflect on the intersection of policy and science that’s opened new paths for the discovery and development of innovative technologies to treat and cure disease in ways that weren’t possible just a short time ago. We’re at an inflection point in medicine and health. We’re witnessing the advent of brand new technology platforms that have the potential to improve health, and cure disease, in fundamentally novel ways. Applications like gene therapy and regenerative medicine may cure inherited disorders and rejuvenate damaged cells and organs. Tools like digital applications and more sophisticated diagnostics have the capability to more fully empower consumers with information to tailor their medical care and inform them about their treatments.

With each of these and with similar products, the novelty of the technology that the FDA is being asked to evaluate challenge the agency to adapt its usual approaches to product regulation in some novel ways. We need to make sure that we’re taking policy steps to enable these innovations to efficiently advance to benefit patients, while we maintain our gold standard for ensuring the safety and efficacy of new products.

By working closely with our partners in the research and development community to create tailored standards for evaluating the safety and effectiveness of novel technologies, we’re helping to make the entire development process more efficient, while ensuring that the FDA is a bridge, not a barrier, to patients accessing potentially transformative treatments.

These standards build on the agency’s commitment to science-based regulation, enabling us to better understand the fundamental clinical risks and potential benefits presented by the different types of technology platforms under review. Gene therapy is one area where the FDA is seeing this approach come to fruition. Today we mark an important step in this progress.

The approval of the first directly administered gene therapy is especially notable. Not only for what the new treatment does and how it works – by engineering a virus as a vehicle to deliver the gene directly to its target inside the body – but also for how we’ve expanded the use of gene therapy beyond the treatment of cancer to the treatment of vision loss for children and adults.

Today’s action reinforces the potential of this innovative technology in treating a wide-range of challenging diseases.
We’re taking steps to extend the opportunities offered by gene therapy innovation. We recently announced our comprehensive policy framework for regenerative medicine, including a draft guidance that describes the expedited programs that may be available to sponsors of these therapies. In the draft guidance we also describe a novel method for how we intend to tailor our traditional regulatory approach in order to accommodate the origin of much of this innovation.

Individual academic investigators are often the ones pioneering regenerative medicine products. They might lack the knowledge and resources to address all the aspects of product approval and regulation. So we delineate a route to allow these innovators to more easily navigate the regulatory process while meeting the requirements for demonstrating safety and effectiveness.

To expand similar policy efforts when it comes to facilitating the advance of gene therapy, next year we’ll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products. These guidance documents will be part of a modern, comprehensive framework for how we’ll help advance the field of gene therapy while making sure new products meet the FDA’s standards for safety and benefit.

These policies will articulate, among other things, new clinical measures for the evaluation of gene therapy for different high-priority diseases where the platform is being targeted. These new documents will address clinical areas where there’s a lot of interest in using these gene therapy techniques, such as certain more common single gene disorders. We intend to provide innovators with clear advice on safe and effective development pathways, including potential accelerated approval endpoints.
The spirit of these efforts was embodied in the review of this new gene therapy product for the treatment of an inherited form of vision loss. We recognize that with novel therapies for some of the most vexing diseases and conditions, often comes a need for new clinical measures to allow these technologies to go forward.

In this case, it meant the validation of a new standard that will help guide the development of future therapies that address severe vision disorders. Our Office of Tissues and Advanced Therapies, which had familiarity with the issues involved in the study of products for vision loss, recognized that showing differences in visual loss or visual acuity might be difficult. In this review, the FDA suggested and worked with the product sponsor to develop and validate a new, more practical endpoint that could reflect true clinical benefit in both pediatric and adult patients instead of relying on only visual acuity or visual field testing.

Gene therapy products are now being studied for the treatment of many diseases and conditions, including genetic disorders, autoimmune diseases, heart disease, cancer, diabetes, and HIV/AIDS. We will work closely with the research community to help advance potential new therapies in these serious disease areas—which affect millions of Americans and their families.

Other similar medical opportunities lie just behind this milestone. To give you just one example related to the agency’s current approval action; there are more than 600 active investigational new drug applications related to gene therapy products. Researchers at the Massachusetts Institute of Technology estimate that about 40 gene therapies might win approval by 2022, from a current pipeline of 932 development candidates. They estimate that 45 percent relate to treatments for cancer.

I can’t confirm their estimate. But I can affirm that we’re at the early stages of a transformation in medical care as a consequence of these and other transformative platforms. The benefits of this science – and the products that become available – are likely to accelerate. How we define the modern framework for safely advancing these opportunities will determine whether we’re able to fully realize the benefits that these new technologies can offer.

I want to thank you for joining us today and as we move forward together to unlock the full potential of gene therapy for patients. Now I’d like to introduce Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research – the FDA center that’s tasked with regulating regenerative medicine, including gene therapies, to talk more specifically about today’s approval.

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