Executive Summary

Deputy Office of New Drugs director says IND evaluation and post-market surveillance could be part of restructuring effort along with changes to application review.

The US FDA’s restructuring of the Office of New Drugs could extend well beyond new drug application reviews. Changes to many of its pre- and post-market business practices also are being considered.

Peter Stein, deputy OND director, said “we put everything on the table” as the office in charge of review and approval of new drug products considers how to adapt to new drug development paradigms.

“We are thinking about how we look at the IND level reviews, how we do application reviews for NDAs and BLAs and supplements, how should post-marketing surveillance be considered, what are the things that we can do to make these processes more efficient,” he said Nov. 14 during the Biopharma Congress, sponsored by Prevision Policy and the Friends of Cancer Research.

The wider review underway is in addition to the already well-publicized look at OND’s staffing structure.

After the departure of long-time OND Director John Jenkins, Center for Drug Evaluation and Research Director Janet Woodcock became acting OND director and initiated the reorganization process. Woodcock has said that several options are under consideration, such as whether review teams can be designed to move between divisions if workload demands. It is in part intended to generate more consistency within the office. (Also see “CDER Director Woodcock Plans Changes To Drug Reviews During OND Transition” – Pink Sheet, 6 Mar, 2017.)

Stein said OND now is dealing with more rare disease applications and narrowly targeted specific therapies and those workload changes will be considered as the restructuring process moves forward.

Pilot projects for potential changes are under consideration, but Stein said the process will be careful, thoughtful and rigorous, and that results will emerge over the “next couple of months and years.”

“These are complex processes,” Stein said. “We want to make changes that are going to advance things and not just sort of confuse the situation.”

Among the changes potentially appearing soon are to OND’s guidance documents. Woodcock said during the conference that some will be much shorter and contain a series of bullet points that the agency wants to communicate, rather than long and sometimes confusing prose. (See sidebar.)

Another idea would create multi-disciplinary teams to handle a product’s entire life-cycle. Commissioner Scott Gottlieb also has said OND is considering creating a single shared review memo to ensure interaction across disciplines. (Also see “Gottlieb Promotes ‘Team’ Work For Product Reviews” – Pink Sheet, 3 Nov, 2017.)

Is A Rare Disease Team Coming?

Stein also suggested OND leadership is considering a carve out within the new office structure focused on rare diseases.

When asked whether FDA was thinking about replicating the Oncology Center of Excellence idea for rare diseases, Stein said no proposal has been made. But he also said the agency has been thinking about it, and “it’s possible” that the structure for evaluating rare diseases “within OND and actually with other offices may change.”

“I think we recognize this is a rapidly moving area,” Stein said. “I think that does suggest the potential for trying to make this a more cohesive approach.”

The Oncology Center of Excellence was created in 2016 and intended to bring oncology experts from the various product centers together to help complete product reviews faster. (Also see “FDA’s Pazdur Jumps Over To New ‘Moonshot’ Role” – Pink Sheet, 29 Jun, 2016.)

Stein said reviewers who work in the rare disease space have a unique set of skills and “those that have done rare disease reviews and are experienced with it have a different mindset.”

A separate rare disease-focused review team has long been an ask of some rare disease stakeholders and advocates. Because the drugs involve small patient populations, they often employ small and atypical clinical trials and require FDA to use its flexibility in judging efficacy. Reviewer consistency also has been a problem.

Gottlieb has argued for creation of an orphan drug review division and planned to create expert review teams that could help reviewers with complex issues such as gene therapy or molecularly-targeted drugs. (Also see “Gottlieb’s FDA: Specialized Groups Could Create Review Consistency, Speed” – Pink Sheet, 23 Mar, 2017.)

The prescription drug user fee program renewal also required FDA’s rare disease staff to be integrated into the existing review divisions to help with orphan applications when needed. (Also see “Rare Disease Integration Into FDA Reviews Will Grow Under PDUFA VI” – Pink Sheet, 18 Jul, 2016.)

Needed Expertise May Help Determine OND Structure

Woodcock also said the scientists and clinicians that will be needed to review the applications will help determine how the agency will be organized going forward.

“In the future we’ll have to really figure out how this trans-disciplinary effort needs to go on, and then the organizational structure should flow from that,” she said. “The question is what do we need to do the assessment of these products, and then once we figure that out, then how do you organize yourself in an efficient way.”

Indeed, several former FDA officials have warned that reorganizing FDA’s drug center entirely by disease area may not be wise or efficient. (Also see “Don’t Rush To Reorganize FDA, McClellan Says” – Pink Sheet, 29 Feb, 2016.)

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