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# FDA Revamping its Industry Advice for New Technologies: Gottlieb
- URL: https://www.fdaweb.com/fda-revamping-its-industry-advice-for-new-technologies-gottlieb/
- Published: 2017-09-07T12:00:00.000Z
- Updated: 2026-09-14T22:47:25.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139577

FDA is planning to revamp its early pre-clinical involvement with medical product developers as part of a new “Strategic Policy Roadmap” that will be unveiled soon, commissioner **Scott Gottlieb** told an online Research America 2017 National Health Research Forum in Washington, DC 9/7\. New policies are being formulated to “adapt our regulatory principles to the new challenges we face in properly evaluating a very different set of scientific opportunities,” he said, particularly for gene therapy, regenerative medicine and other new platforms. He noted that FDA currently has more than 550 active IND applications related to gene therapy products, and 76 active INDs related to CAR-T cell products.

Gottlieb told the forum that the agency’s policies and regulatory framework are out-of-date to properly evaluate the safety and effectiveness of new technology platforms. “Our ability to fully capitalize on new science, and maintain FDA’s gold standard for product review, means FDA also needs to modernize itself alongside the new platforms that we’re evaluating,” he said. “We need to make certain our principles for regulation allow and facilitate beneficial new innovation while making sure that FDA continues to meet its gold standard for safety and effectiveness.”

Enhancing its regulatory framework will have FDA asking new questions, according to Gottlieb. “For one thing, how do we adjust our approach when the primary complexity and uncertainty inherent in a new product isn’t just the clinical questions related to whether it works, but the features of its delivery platform, or how it’s being manufactured?” he asked. “What do we do, for example, when the most complicated risks aren’t acute toxicities that can be observed up front? Instead, they’re long term and more theoretical risks and uncertainties, such as the potential for off target effects of gene therapy interventions?”

Because gene therapy offers situations where “products are delivered transiently, and are sometimes just a single administration,” Gottlieb said, “determining safety and effectiveness doesn’t boil down to a judgment made at a single point in time. It’s not a binary measure – a single, discrete threshold. The proper evaluation of safety is an ongoing process. It crosses over the threshold of initial approval. This means more emphasis needs to be placed on how new technologies perform in clinical use; during routine care. Long-term risks and benefits need to be carefully monitored. This is similar to the way that we evaluate many medical devices. Our questions related to these new platforms would increasingly relate to their long-term performance. More of our emphasis will naturally shift to our post market tools.”

Revamped pre-clinical efforts will focus on the advice and engagement FDA offers before new technologies are moved into the three phases of clinical development, Gottlieb told the forum. “Our evidence shows that earlier and more frequent engagement can help make the initial stages of development more efficient, and increase the odds for success,” he said. “We know that academic and individual medical sponsors, as well as startups and some small-sized biotechnology companies, sometimes don’t have a full understanding of what it will take to get an IND or to get a BLA filed with FDA for their product. It’s often the smaller companies or individual researchers who are working with the most novel technology platforms. At FDA, we’re therefore encouraging very early meetings to provide early feedback to drug developers.”

He noted that that some sponsors overestimate the amount of information that is needed to file an IND, “and that too many of the costs of development therefore get front-loaded, increasing the cost of initiating new science. Ideally, it would be easier to get products into development, with more of the costs pushed further out, after some of the initial pre-clinical work is already done, and there’s a better understanding of whether a new product has clinical promise. Toward these goals, the FDA’s review staff is sometimes able to help significantly streamline the early development process by eliminating unnecessary pre-clinical tests or by suggesting optimal pre-clinical or clinical designs such as more adaptive trials for early stage research, or Bayesian approaches to statistical evaluation of results where randomization is not possible in early stage clinical trials.”

Additionally, Gottlieb said that new approaches to pre-clinical scientific engagement will be outlined in “educational materials” that will be released soon. “We’re going to be taking other new steps to make sure that our policies governing early, pre-clinical science are more closely matched to the complexion of modern technologies. One is evidenced in how clinical trials are initially designed. In certain cases, there are a lot of common features across the same platform, even as it’s used to target different genes or proteins. There may be plausible reason to recognize how a product or platform can work across multiple disease states – and leverage the learning from one setting in other opportunities.”

For example, Gottlieb said the agency may be able to take an “adaptive approach” in its pre-clinical evaluation of different therapies that “share a lot of common characteristics in the overall platform used to deliver a gene product. Consider two gene therapy vectors that contain CRISPR constructs differing by only one base pair. These two products might not need nearly the same amount of pre-clinical data for the second variant as was required for the first one, if we can borrow what we learn across different clinical applications of the same basic construct. By comparison – if the CRISPR inserts were different at five or more base pairs – that might require more data. The bottom line is this: We need to carefully but efficiently evolve our pre-clinical regulatory models to adjust to what we need as the science evolves and indicates what is critically necessary for safe application of the new technology.”

Gottlieb also told the forum that in the coming weeks he will “discuss how these same policy goals will be made evident in how we approach the clinical portion of the development process, and our life-cycle approach to medical product stewardship.”