Rare Diseases, RWD, Supply Chain Headline CDER’s Priority List

The FDA’s Center for Drug Evaluation and Research (CDER) is looking past the pandemic in its current goals for 2022, says Director Patrizia Cavazzoni, focusing on initiatives on rare diseases, real-world data (RWD), supply chain surveillance and increased patient engagement in drug development.

In the past year, Cavazzoni told attendees at the Food and Drug Law Institute’s annual conference yesterday, the FDA reviewed more than 460 trials related to COVID-19 and granted emergency use authorization to 14 COVID-19 treatments and full approval to two treatments.

Now, the agency is turning its attention to what comes after the public health emergency, she said, “to ensure long-term access to safe and effective therapies against COVID-19 across the entire spectrum of disease severity.”

In addition to new guidance related to COVID-19, CDER is also examining the use of real-world data (RWD) in advancing clinical research, Cavazzoni said, and noted that legislation to reauthorize the Prescription Drug User Fee Act (PDUFA) currently before Congress includes a potential new RWD pilot program to evaluate its worth.

“We have seen an expansion of the utilization of real-world data during the pandemic, which is great, but at the same time, more data doesn’t mean better evidence,” she said.

Cavazzoni also pointed to another program outlined in the PDUFA legislation, CDER’s Rare Disease Endpoint Advancement (RDEA) pilot that will allow sponsors to collaborate with the FDA on novel efficacy endpoints for rare diseases. She also highlighted the Accelerating Rare disease Cures (ARC) Program, CDER’s new initiative to speed and increase the development of treatments for rare diseases, as a bright spot in CDER’s plans (DID, May 12).

CDER has also been focused on drug supply chain surveillance, Cavazzoni said. To develop more insight into the supply chain and mitigate shortages, she said CDER is deploying advanced analytics to examine availability, supply and demand, and encouraging investment in advanced manufacturing technology and in “strengthening quality management maturity.”

Patient-focused drug development is another area of focus, said Cavazzoni, adding that CDER is working on a series of guidances to further the advancement and use of systematic approaches to collect and use patient and caregiver input to inform medical product development and regulatory decisionmaking.

CDER is also planning to modernize the regulatory framework and processes for the FDA’s postmarket surveillance program over the next three years, she said. 

To try to upgrade the program any sooner than that “is intellectually appealing, but not terribly realistic,” said Cavazzoni.

Turning to inspections of drug manufacturers, she said CDER is excited to more permanently implement alternative tools that were used to continue oversight during the pandemic. During COVID, CDER completed more than 1,000 remote regulatory assessments of drug manufacturing sites. Now, the number of facilities needing preapproval and prelicense inspections has been reduced by about 52 percent. — Suz Redfearn