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# Gottlieb: Science Contributing to ‘Inflection Point in Medicine
- URL: https://www.fdaweb.com/gottlieb-science-contributing-to-inflection-point-in-medicine/
- Published: 2017-12-07T12:00:00.000Z
- Updated: 2026-09-14T23:06:28.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5140297

FDA commissioner **Scott Gottlieb** says that across multiple science fields, “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.” He made these remarks during a 12/7 Senate Health, Education, Labor and Pensions hearing reflecting on the implementation of the 21st Century Cures Act at its one-year anniversary.

To illustrate the path science is taking, Gottlieb pointed to advances 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,” he told the hearing. “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.”

Gottlieb likens advances in gene therapy to what was seen with monoclonal antibodies in the 1990s, and once these were humanized many breakthrough drugs resulted. “A whole new field of medicine grew up very fast,” he said. “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.”

Gottlieb noted that MIT researchers recently estimated that about 40 gene therapies might win FDA approval by the end of 2022 from a current pipeline of 932 candidates. Of these, 45% are expected to target cancer. “I can’t affirm their assessment,” he said. “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.”

Gottlieb told the hearing that next year the agency will be building on new opportunities offered by the Cures Act’s focus on innovation and breakthrough therapies. “We’ll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products,” he said. “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.”

The first guidance document will likely focus on the use of gene therapy in hemophilia, according to Gottleib. And other guidances will address clinical areas of interest, such as single gene disorders. “We’ll provide innovators with advice on development pathways, including potential accelerated approval endpoints,” he said.

Additionally, Gottlieb noted that 2017 is on track to see the highest or second-highest number of novel medicines across the biologics and drug Centers approved in FDA’s history. Generic drug approvals are expected to be at an all-time high, and so are novel medical devices, Gottlieb testified.