With greater innovation on the horizon in health care delivery, the regulatory environment must adapt to support the advancement of novel technologies.
 
At the Asembia Specialty Pharmacy Summit 2019, Scott Gottlieb, MD, 23rd commissioner of the FDA discussed the agency's role in rewriting the modern rules of new regulatory paradigms for a number of recent advancements in medicine. Developments in novel areas such as gene therapy, targeted medicine, and digital health have required the agency to rethink how to regulate these novel products, according to Gottlieb.
 
“The old rules didn’t apply,” he said to the audience. “We had to rewrite the rules of how we were going to approach these new technologies and really lay the foundation for what the regulatory architecture would look like going forward.”
 
Gottlieb touched on areas of innovation that emerged during his time in office, including gene therapy, cell-based regenerative medicine, targeted medicine, analytical tools, and electronic health records (EHRs). Many of these bring individual challenges that required the agency to develop new ways of approaching regulating these products.
 
For example, many of the regulatory challenges for gene therapy aren’t derived from the clinical portion of the review. Instead, Gottlieb explained that the complexity for the agency is primarily caused by the product portion, which lends itself to a whole new paradigm. In addition, targeted medicine represented a challenge because the current regulatory rules did not provide readily available tools to test these products, Gottlieb said. Approaches such as basket trials and tissue agnostic trials, which allowed drugs to receive approvals for multiple tissue types, allowed for more efficient development of these therapies.
 
Digital health tools such as apps also posed challenges, especially for pre-market testing. Instead of regulating the product itself, the agency took an approach that included regulating the firm’s standard operating procedures for testing their own software, according to Gottlieb.
 
But he noted that perhaps the most profound trend was the agency’s transition to EHRs and practical data. For the first time, the agency has a major buildout of the ability to use data from EHRs to conduct post-marketing surveillance and, eventually, practical clinical trials in a real-world setting.
 
“The idea is to see if the agency can develop a paradigm for replicating clinical studies using real-world evidence,” he said.
 
Gottlieb added that the FDA is adopting a more structured model to standardize the submission of applications to foster a more consistent approach to drug review.
 
Gottlieb also discussed current challenges with reimbursement in today’s health care landscape, which will be determinative in patients being able to access groundbreaking therapies.
 
“I think we are really at a crossroads when it comes to the reimbursement environment,” he said.
 
Gottlieb indicated that these newer therapies will require a change in how we reimburse products, particularly in the context of Medicare Part B. He touched on the average sales price (ASP) plus 6% payment model for Medicare reimbursement for Part B drugs.
 
“When we created that model, that model was designed to recognize that CMS was going to be a price taker,” he explained. “So we wanted a system that was designed to mitigate against price increases. And ASP plus 6% was designed to do that.”
 
Moreover, determining payment models for products such as chimeric antigen receptor (CAR)-T cell therapy remains a structural challenge for Medicare, Gottlieb said. He expressed concern that this may lead to diminished investment in these areas.
 
Despite reimbursement challenges, the agency’s ability to keep pace with these emerging technologies represents a promising outlook for the future of medicine. With the framework set in motion, Gottlieb said that writing modern rules for a more efficient review and development of these products is going to help make them more available to patients.
 
“I think the future is really bright,” Gottlieb concluded. “I saw when I was at the FDA unlimited opportunity in what we are capable of doing.”