New FDA Commissioner Scott Gottlieb intends to release a far-reaching plan aimed at modernizing regulatory processes and speeding approvals for new drugs and devices, which could allow greater patient access to new medicines through unprecedented scientific and technological advancements.

Key elements of the plan, called the Innovation Initiative, include using computer models and virtual patients to develop and evaluate new drugs or devices, particularly those targeting personalized medicine and rare disease populations, and creating new regulatory standards to apply expedited pathways for regenerative medicine products. The FDA will also move forward with guidance in areas including novel clinical trial design and use of real-world evidence in drug approvals, which has been made possible through recently developed digital technologies.

Gottlieb, who announced the new initiative in a blog post, said the plan builds on the 21st Century Cures Act, which made substantial changes to FDA regulations and gave the agency new powers to speed approval of drugs. The commissioner said goals of the Innovation Initiative include ensuring regulatory principles are “modern and efficient” and incorporate the “most up to date” scientific knowledge.

“We are at a point in science where new medical technologies hold out the promise of better treatments for a widening number of vexing conditions,” Gottlieb said. “We don’t want to present regulatory barriers to the beneficial new medical innovations that add to the time, cost and uncertainty of bringing these technologies forward if they don’t add to our understanding of the product’s safety and benefits.”

The Innovation Initiative was revealed as the FDA faces a central challenge of balancing regulations that promote speed and innovation of new drugs with the need for comprehensive benefit/risk data to ensure patient safety. Many industry groups express optimism about the agency’s recent steps to accelerate drug development and approval processes, yet the industry doesn’t support a less rigorous FDA review process that could put patients at risk and make it harder to convince physicians and insurance companies that new treatments delivered value. 

“We are fully confident that the FDA’s gold-standard commitment to safe and efficacious products will be maintained, even as the agency deploys new technologies, such as modeling and simulation, to advance products in development through the stages of clinical trials more efficiently,” said Doug Peddicord, Ph.D., executive director of Association of Clinical Research Organizations (ACRO). “We look forward to new and updated guidance about the use of such ‘in silico’ tools across the spectrum of product development.”

Industry groups and experts also praised the new commissioner’s commitment to support and broaden the Cures Act, which was enacted in December 2016 during the Obama administration. The Cures Act includes requirements for the FDA to develop a new framework for incorporating patient perspectives into the drug development process and the use of real-world evidence in the approval of new uses for existing drugs. In his blog post, Gottlieb announced that the final work plan for steps to implement different aspects of the Cures Act has been posted.

“Since the 21st Century Cures Act was enacted in the final days of the Obama Administration, one might worry that the Trump Administration would not embrace it. But that is simply not the case. The FDA seems to be implementing it with zeal. And indeed, that’s the benefit of having produced the 21st Century Cures Act in a bipartisan manner,” said Bradley Merrill Thompson, a lawyer with the firm Epstein Becker & Green, who specializes in FDA and clinical trial issues. “The FDA is enthusiastically embracing the idea embedded in the legislation that the FDA has a responsibility to ensure that innovation flourishes. I really have to applaud the FDA for this initiative.”

A key way that the Innovation Initiative proposes to modernize processes is through new FDA guidance regarding the use of computer modeling and simulation in clinical trials to improve the efficiency of both drug development and regulatory review. Computer models can help narrow possible drug designs, predict product safety, evaluate potential adverse event mechanisms, inform alternate study designs and help advance new therapies more quickly through the different clinical trial phases.

The FDA has begun to use computer modeling to build natural history databases, Gottlieb said, that could allow the FDA to better evaluate new therapies for Parkinson’s disease, Huntington’s disease, Alzheimer’s disease and muscular dystrophy. The agency has also begun to develop a “family of virtual patients” for testing new devices and is investing in high-performance computing capabilities to allow review staff to manage the large data sets that have become a common element of drug applications.

“I am very glad to see that the FDA is investing in upgrades to its IT infrastructure that will allow it to make maximum use of some of the software analytics that will help the FDA spot potential safety risks, but also do so quickly and with the least burden on industry applicants,” said Thompson. “We’ve made great strides in computer modeling and simulation the folks in industry have been now using for a while. Putting the FDA in the position to likewise use those techniques is a real step forward that will both enhance safety and lessen the regulatory burden.”

As part of the Innovation Initiative, in September the FDA will announce a comprehensive framework for development and oversight of regenerative medicine. The agency recently implemented the Regenerative Medicine Advanced Therapy (RMAT) designation, which provides an additional pathway to access expedited programs for certain cell and therapeutic tissue engineering therapies. The new effort will include a series of guidance documents that outline regulatory aspects governing regenerative medicine, including policies for demonstrating safety and effectiveness of these novel products.

The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the gene and cellular therapies and broader regenerative medicine sector, commended the FDA’s new initiative. ARM had previously expressed concerns about proposals that sought to change approval pathways for regenerative medicine products by reducing or, in some cases, eliminating the FDA’s regulatory authority, which could have allowed untested and unproven treatments to reach the market.

“We’re very enthusiastic about the regenerative medicine-specific provisions in this announcement, especially the RMAT designation, as it’s specifically designed, for the first time in the U.S., to apply expedited pathways to regenerative medicine products. Its implementation will ensure that safe and effective products reach patients in need as quickly as possible,” said Michael Werner, ARM’s executive director and head of legislative activities.

“We look forward to reviewing the details of Commissioner Gottlieb’s Innovation Initiative once they emerge,” said Sara Radcliffe, president and CEO of California Life Sciences
Association. “The FDA’s work heavily impacts the pace at which innovation is driven in California’s life sciences industry, as well as the delivery of life-changing medicines and technologies far beyond the state of California.”

This article was reprinted from Volume 21, Issue 28, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »