FDA caught up on domestic preapproval inspections, foreign inspections still a challenge

Regulatory NewsRegulatory News | 20 June 2022 |  By 

The US Food and Drug Administration (FDA) eliminated its backlog of overdue domestic preapproval inspections, yet such progress has been stunted at the international level as getting to sites in China has remained a challenge amid the country’s COVID-19 pandemic restrictions.

So asserted Patrizia Cavazzoni, the director of FDA’s Center for Drug Evaluation and Research (CDER), who provided an update on the center’s activities and priorities for 2022 at the Food and Drug Law Institute’s (FDLI) annual meeting last week. Other priorities include accelerating the development of drugs to treat rare diseases, as well as spurring the use of real-world evidence (RWE) in clinical trials.
Despite the pandemic, the center has maintained historical trends in approving new drug and generic drugs, said Cavazzoni. Since last year’s FDLI conference, CDER approved 41 novel treatments in 2021, including 27 new drug applications (NDAs) and 14 biologics license applications (BLAs), with 23 of the 41 treatments indicated for rare diseases, Cavazzoni said. There were also 563 generic drugs approved during that time, and of these, 87 were first-time generic approvals and 56 were competitive generics.
Cavazzoni said that progress has also been made on the domestic front in inspecting sites awaiting product approval. “We are now in a position where all domestic preapproval inspections that had been delayed have been completed. Obviously, we used alternative tools” for some of these inspections, Cavazzoni said, adding that “there are always situations that we have no choice but to physically inspect the facility in order to make a decision on the applications.”
Yet this backlog has not yet been resolved when it comes to international facilities. Cavazzoni said that “all delayed CDER BLA inspections have been completed, with the exception of those in India and China. China remains a problem because of the ‘Zero COVID’ policy that exists in China which makes physical inspections exceedingly difficult, so these alternative tools will be particularly important at this juncture.”
According to FDA’s resiliency roadmap issued last year, as of September 2021, 60 approval applications had been delayed because of the inability of inspectors to get to sites. (RELATED:  FDA says it far exceeded its projections for inspections in FY2021, Regulatory Focus 22 November 2021)
Doubled down on rare diseases
In other areas, the center has “doubled down” on programs to accelerate the development of rare diseases.
From 2015 to 2021, CDER approved 163 drugs for rare disease, which represents about half of all novel drug approvals during that time.
FDA has approved over 600 treatments for rare diseases since the passage of the orphan drug act in 1983. Cavazzoni said that while 600 orphan approvals is a notable achievement, “there are over 7,000 rare diseases so that there is lot of work to do. We cannot rest on our laurels.”
To expedite the development of these treatments, FDA has launched a new pilot program to identify novel endpoints for rare diseases. The program, called the rare disease endpoint advance (RDEA) pilot, is included in the PDUFA VII program. (RELATED: PDUFA VII commitment letter: RWE, rare diseases see renewed attention, Regulatory Focus, 23 August 2021)
This program would also complement CDER’s new program called Accelerating Rare disease Cures (ARC) that aims to “turbocharge” the development of treatments that meet unmet medical needs for rare diseases. (RELATED: This Week at FDA: House holders user fee markup, CDER’s ARC for rare diseases, Regulatory Focus, 13 May 2022)
Another priority is to accelerate the adoption of real-world evidence to support clinical studies for new drugs in the US. Cavazzoni said that while uptake of RWE has grown during the pandemic, sponsors are still relying too much on data from other countries, such as Hong Kong and Denmark.
“Somehow we have been lagging in the US largely because our healthcare system is more complex and more fractionalized, so there are more challenges with respect linking outcomes data,” said Cavazzoni.
FDLI annual meeting


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