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Remarks from FDA Commissioner Scott Gottlieb, M.D., as prepared for oral testimony before the U.S. Senate Committee on Health, Education, Labor & Pensions Hearing, “Implementation of the 21st Century Cures Act: Progress and the Path Forward for Medical Innovation

For Immediate Release:

Chairman Alexander, Ranking Member Murray, and members of the committee, thank you for the invitation to testify at this hearing to discuss the implementation of the 21st Century Cures Act.

“Cures” set FDA on a transformative path. It set out to optimize our investments in science by modernizing how FDA oversees breakthrough technologies. You asked us to advance innovations more efficiently, while maintaining our gold standard for protecting patients.

This focus on innovation couldn’t come at a better time. Across multiple fields of science, we stand at an inflection point in medicine – where new technology is creating foundational opportunities to treat and cure disease in ways that weren’t possible just a short time ago.

Take, for example, our recent experience with gene therapy.

We’ve seen two recent approvals of CAR-T therapies for cancer, where a patient’s own immune cells are re-engineered – using the tools of gene therapy – to target a patient’s individual cancer. This form of gene therapy represents a whole new paradigm in treating cancer. And the early results are changing the way we treat serious tumors.

This experience shows how a single, fundamental breakthrough in science can open up a whole new way of combatting disease.

In gene therapy, that breakthrough has been the development of vehicles that can deliver genes more efficiently to their target inside the body. These often are referred to as vectors. And they’ve taken the form of viruses that are specially engineered for this purpose.

In particular, the advent of a specific kind of largely inert adeno-associated virus – or “AAV” vector – was an inflection point in this field.

I liken the advent of AAV vectors to the development of processes for developing antibody drugs and making these medicines nearly identical to the fully human cells that they were mimicking.

Monoclonal antibodies represented a promising field of potentially breakthrough medicines in the 1990s. But for a long time, these therapeutic drugs fell short of their promise.

That was because these drugs were made with antibodies from mice, and the antibody drugs themselves were soon rejected by patients’ immune systems. Then came the science for humanizing these antibodies, so they’d more fully mimic their normal human counterparts. And pretty soon, we saw many breakthrough drugs result. A whole new field of medicine grew up very fast.

I believe we’re at a similar turning point when it comes to gene therapy. Over the next several years, we’ll see this approach become a mainstay of treating, and probably curing, a lot of our most devastating and intractable illness. At FDA, we’re focused right now on establishing the right policy framework to capitalize on this scientific opening.

Researchers at MIT recently estimated that about 40 gene therapies might win FDA approval by the end of 2022 from a current pipeline of 932 development candidates. They estimate that 45 percent of the total gene therapy drugs are expected to target cancer.

I can’t affirm their assessment. But I can confirm that we’re at the early stages of a transformation in medical treatment as a consequence of this new technology. And the benefits are likely to accelerate quickly.

The advance of this field is not risk-free. Yet it’s a good example of how FDA’s embrace of the Cures Act – and our efforts to build on what Congress set out to do in balancing safety with scientific promise – is expanding our ability to capitalize on breakthrough innovation.

In this case, Cures provided a pathway for certain regenerative medicine products to receive expedited review by FDA through the RMAT designation. We extended that opportunity.

FDA has considered CAR-T products to be a form of gene therapy, since the key therapeutic manipulation that’s made to the cells is through a gene product delivered by a vector.

In FDA’s new draft guidance on “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, FDA clarified that regenerative medicine therapies would include gene therapies that lead to a durable modification of cells or tissues – including genetically modified cells. This would include CAR-T products, when these gene therapy products lead to a durable modification of cells or tissues and therefore deliver a sustainable effect in the body.

For example, if a gene therapy alters tissue to allow the body to express a certain therapeutic protein, or if CAR-T cells have resiliency and maintain a presence that delivers a sustainable therapeutic effect, we would consider them to be regenerative medicine therapies.

By FDA taking these science-based decisions, it means that gene therapies – including CAR-T – may be eligible for the RMAT designation.

Next year, we’ll be building on these opportunities. We’ll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products.

We intend to lay out modern and more efficient parameters – including new clinical measures – for the evaluation and approval review of gene therapy for different high-priority diseases where the platform is being targeted. We plan to focus the first guidance document on the use of gene therapy in hemophilia.

Other documents will address clinical areas where there’s a lot of interest in using these techniques, such as certain more common single gene disorders. We’ll provide innovators with advice on development pathways, including potential accelerated approval endpoints.

Gene therapy is just one opportunity transforming medicine. This year FDA may be on track to approve the highest or second highest number of novel medicines across our combined biologics and drug centers in FDA’s entire history. We’ll also approve the highest number of generic medicines ever. And we’re on pace to approve the highest number of novel medical devices in our modern history.

All this year. All at one moment in time.

This progress is a reflection of scientific opportunity.

It’s also a reflection of sound policy – advanced by Congress – that gave FDA key resources and authorities at the very moment that those new policies lined up with a turning point in science.

The result is measured in these product approvals, and in the impact of some highly novel medicines like the new gene therapies.

Ultimately, this progress will be measured in its human impact. We witnessed an inflection point in the survivability of cancers after the advent of a lot of new therapies in the late 1990s and early 2000s. The most significant were the antibody drugs. I predict we’ll see a similar turning point in how we cope with illness as we consolidate the latest wave of advances into new ways of combatting disease.

I look forward to answering your questions and, on behalf of my colleagues at FDA, I want to thank you for your support of our mission.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.



Deborah Kotz

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