In First Speech Since Stepping Down as FDA Commissioner, Gottlieb Reflects on Agency's Push for Innovation

In his first speech since stepping down as FDA commissioner, Scott Gottlieb, MD, reflected on his time in office during a session at World Health Care Congress 2019.

During his speech, Gottlieb set a focus on the FDA's approaches to embracing innovation while under his leadership. “We have a lot of new things behind us and a lot of new opportunities in front of us in terms of new fields of technology that we really need to create modern rules for how they’ll be regulated,” he said, adding that dealing with how to pay for these technologies is something he’ll be focusing on as he returns to the American Enterprise Institute.

Looking at innovations that occurred during his time in office, each required the agency to think differently about regulation because old rules did not apply to these new areas, he explained.

Gene therapies: When it comes to gene therapy, there aren’t unanswered questions regarding whether or not the therapy works, as efficacy is often demonstrated early on in the development process, said Gottlieb. Instead, the majority of the complexity and uncertainty surrounds the product features, such as the vector and how the vector will behave, which lends itself to a much different approach to regulation. It also leads to more opportunities, with questions including: can you allow 2 gene therapies targeting the same disease that effectively have the same gene facet but much different vectors to both be eligible for market exclusivity?

Cell-based regenerative medicine: While Gottlieb was at the FDA, the agency put out a series of guidance documents to lay the groundwork for how regenerative medicine should be regulated. Recognizing that many of these products are developed at academic institutions by individuals investigators who don’t have the ability to file for FDA approval, the FDA made the decision to permit individual investigators to pool their data, so long as they follow common manufacturing data, and file for 1 common license, explained Gottlieb.

Targeted medicine: Gottlieb also touted the agency’s promotion of basket trials and tissue agnostic approvals, where a drug can be approved for a specific mutation regardless of what organ or tissue is impacted. In oncology, for example, the FDA approved larotrectinib for all solid tumors that have a neurotrophic receptor tyrosine kinase gene infusion. “For the first time, you can get approval based on genotype rather than phenotype,” said Gottlieb.

Analytical tools: With uptake of new analytical tools like artificial intelligence (AI) and next-generation sequencing, the FDA is not able to regulate these tools in a typical fashion, explained Gottlieb. For example, the basis of AI is that it is a self-learning system, which means the tool will make mistakes and needs to make these mistakes to learn. In response, the FDA does not require pre-marketing testing in the traditional sense. Rather, the tool’s validity and utility is compared against publicly available databases and if they meet a certain metric or standard with that data, it’s deemed sufficient for demonstrating effectiveness needed for receiving approval.

Electronic health records (EHRs): One of the biggest transitions is the one to EHRs and the use of real-world data, which Gottlieb said will change the culture of the FDA and open more opportunities for manufacturers, including giving the agency the ability to conduct post-marketing surveillance and eventually clinical trials in real-world settings.

While there are challenges with reimbursement and regulation for innovations like these, “there couldn’t be a better time for life sciences,” said Gottlieb, who recalled getting to brief President Donald Trump on the approval of the first gene therapy. “We are at a moment where we can talk about curing diseases."

He gave the example of sickle cell disease. Whereas 10 years ago, talking about a cure would have been perceived as “highly irresponsible,” it’s now understood by most people that a cure is possible, with some estimating there will be a cure within the next 5 to 10 years, said Gottlieb.