Executive Summary

CDER Director says US FDA needs to develop statistical and other guidances faster to keep up with the pace of science and drug development.

The US FDA wants to speed up its policy-making process to better keep up with the speed of scientific advances affecting drug reviews.

Center for Drug Evaluation and Research Director Janet Woodcock said guidances and other policies need to be written faster and more efficiently to ensure drug sponsors receive the most up-to-date advice.

“We have to figure out a way to have rapid policy development,” she said during the recent Drug Information Association-FDA Statistics Forum. “How do we get rapid statistical policy, rapid guidance out, because the science is changing very fast and we need to figure out a way to keep up with that.”

Woodcock added that a portion of that goal includes streamlining FDA processes for making policy prescriptions.

“We, internally, really need to work on efficiency with all these assignments,” she said. “We at FDA really need to keep focused on the drug development pathways, how do we think about the armamentarium, how do we think about the disease, how do we think about the advice that we give.”

The idea fits with the message of new FDA Commissioner Scott Gottlieb, who wants agency staff to share best practices in order to increase efficiency. (Also see “Gottlieb Promotes ‘Bottom-Up’ Review To Increase FDA Efficiency, Consistency” – Pink Sheet, 6 Apr, 2017.)

Woodcock did not hint at what may encompass rapid policy development. But it seems possible any changes could coincide with her reorganization of the Office of New Drugs.

Following the departure of long-time director John Jenkins, Woodcock took over OND temporarily and has been talking with staff about changes to make reviews more uniform. (Also see “CDER Director Woodcock Plans Changes To Drug Reviews During OND Transition” – Pink Sheet, 6 Mar, 2017.)

If FDA can provide faster policy development, it may come in the form of formal meeting advice to sponsors, rather than guidance. The agency and industry embraced an increase in formal meetings both before application submission and during the review for complex generics, as well as biosimilars and new drugs, to help speed development.

Lawmakers also are interested in expanding the concept of formal meetings during review to generics looking to enter markets with little or no competition. (Also see “Breakthrough-Style Program For ANDAs Added To House User Fee Bill” – Pink Sheet, 18 May, 2017.)

Clearance Process May Remain A Barrier

Making guidance available to the public faster still may prove difficult, in part because a portion of the process is out of FDA’s control.

Once a draft guidance is completed, it can be weeks or months before it is published as it winds through the clearance process, where HHS and White House staff review it.

Indeed, an anticipated biosimilars guidance is among the documents now navigating the process. Lisa LaVange, director of the CDER Office of Biostatistics, said during the conference that draft guidance on statistical approaches for evaluating analytical similarity data to show biosimilarity was in the clearance process.

Draft guidance on meta-analysis of randomized control trials to evaluate the safety of human drugs and biologics also is awaiting clearance, LaVange said.

Both documents were included on CDER’s 2017 guidance agenda.

FDA released guidance on biosimilar interchangeability in January. (Also see “Interchangeability: FDA Sets ‘Stringent’ Standard On Design Differences” – Pink Sheet, 24 Jan, 2017.) It was among the dozens of guidances and other documents the agency released in the waning days of the Obama Administration. (Also see “FDA’s Document Dump: Guidance Release Skyrockets Ahead Of Trump’s Arrival” – Pink Sheet, 22 Jan, 2017.)

FDA also is working on several guidances intended to help sponsors better incorporate patient-reported outcomes into clinical trials that were mandated by the 2016 21st Century Cures Act (Also see “Woodcock, Califf Give Thumbs Up To Certain 21st Century Cures Provisions” – Pink Sheet, 14 Dec, 2016.).

The prescription drug user fee reauthorization also requires additional work on the patient-focused drug development effort launched as part of the 2012 program renewal. (Also see “You Just Have To Wait: FDA Can’t Hurry PDUFA VI Guidances” – Pink Sheet, 15 Aug, 2016.)

Progress Depends on Adoption, Mullin Says

Theresa Mullin, director of CDER’s Office of Strategic Programs, said during the conference that a major portion of the patient data and statistics policy development will include putting the new standards into practice.

Mullin said more training and increased communication will be necessary to build confidence in the new approaches.

“I think the pace of our progress here, real progress, not just talking in conferences, is going to depend on how well we can get to clear definitions of these various terms and widely understood definitions that we’re using, whether it be patient area or real-world evidence or any of them, that we have clarity around definitions, that we have data standards that support and work with those definitions,” she said.

Also important to future use of the standards will be the ease that data can be incorporated into drug labeling as new uses.

The Friends of Cancer Research has called for more discussion on how to add quality clinical experience data to labels when a supplemental NDA submission is not feasible. (Also see “Label Expansion: Could Clinical Experience Data Find Supplement Work-Around?” – Pink Sheet, 20 Apr, 2017.)

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