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BioWorld – Tsunami of change to challenge PDUFA VII

BioWorld – Tsunami of change to challenge PDUFA VII

Although PDUFA VI still has two years of life left to it, PDUFA VII is already in the birthing process, with the use of real-world data (RWD), AI, and a coming surge of novel cell and gene therapies looking to be prominent features of the next five-year user fee agreement.

Politics likely will play a role as well.

“I think there’s a tsunami of new things coming to the FDA,” Russ Altman, a Stanford University professor of bioengineering, genetics, medicine and biomedical data science, said Thursday at the FDA’s opening discussion of PDUFA VII.

Over the lifespan of PDUFA VII, which would run from fiscal 2023 through 2027, Altman said novel therapies will be invented and discovered in unusual ways, using artificial intelligence and other new technologies. Data will be more complex and will require computational analysis. And the pace for postmarket monitoring will be more dynamic.

Several speakers at the meeting recognized that the FDA’s Center for Biologics Evaluation and Research (CBER) will face the biggest challenges, as it could be overwhelmed by the expected wave of cell and gene therapies. Currently, CBER is dealing with 900 active investigational new drugs (INDs), and 200 more INDs are expected to be submitted each year, said Rachel Sher, vice president for regulatory and government affairs at the National Organization for Rare Disorders. Consequently, between 40 to 60 new cell or gene therapies are expected to launch over the next decade.

That wave would outstrip CBER’s resources unless proactive steps are taken to ensure adequate resources and optimal review management practices, said Cartier Esham, executive vice president for emerging companies at the Biotechnology Innovation Organization.

That means if the FDA is to be prepared for the coming tsunami, PDUFA VII will have to do more than build on the current agreement. Other industry priorities for what Esham called a patient-centric and effective PDUFA VII include:

  • incorporating lessons learned from COVID-19;
  • investing in FDA hiring and resource management accountability;
  • modernizing the generation of regulatory evidence;
  • facilitating innovative review approaches;
  • advancing the use of complex innovative trial designs;
  • conducting patient-centric drug reviews;
  • advancing digital health technologies;
  • modernizing the agency’s data and technology infrastructure;
  • improving FDA/sponsor interactions;
  • advancing biologic manufacturing.

Next steps

Thursday’s meeting was the first in a series of public meetings in which the FDA will hear from consumer advocacy groups, patients, health care providers, the scientific/academic community, as well as industry, on what they want to see in PDUFA VII.

Following the public meetings, the FDA and industry will negotiate the fees and the agency’s performance goals, with a Jan. 22, 2022, deadline for delivering PDUFA VII to Congress. One thing that’s off limits in the actual negotiations is policy, said Andrew Kish, director of the Office of Program and Strategic Analysis at the FDA’s Center for Drug Evaluation and Research.

That’s not to say that policy, also known as politics, won’t find its way into the final legislation. Congress typically adds its own provisions to the negotiated agreement. But if some consumer advocacy groups have their way, policy could be in play much earlier in the process – and the negotiating table would become more crowded.

“We’re not at the table for PDUFA, and that’s very unfortunate,” Diana Zuckerman, president of the National Center for Health Research, said at Thursday’s meeting. Being at public meetings is a lot different than being at the negotiating table, she added.

Consumer advocacy groups, some of which are politicized, position themselves as looking out for patients, but their stance is often at odds with the patient voice that all stakeholders agree is vital to drug development and PDUFA VII. That was evident Thursday in the priorities consumer groups and patients laid out for PDUFA VII.

“We support more resources for the FDA … but those resources will not be helping patients if they are focused really on speed,” Zuckerman said, referencing performance goals in the current and past user fee agreement that focused on reducing the time it takes the FDA to review a new drug or biologic application.

Sally Greenberg, executive director of the National Consumers League, agreed, saying that the FDA’s performance should be measured by more than the speed with which it reviews and approves a new product. There’s too little emphasis on performance goals focusing on drug safety and effectiveness and too much on the speed of the review, she said.

Speaking from the patient perspective, Jeff Allen, president and CEO of the Friends of Cancer Research, pushed back, saying, “Time certainly matters,” especially for patients with life-threatening diseases that have no approved treatments.…