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FDA will maintain the pace, vows Woodcock, but warns of skills gap

Top official unconcerned by dip in approval rate

FDA

The departure of Scott Gottlieb in April robbed the FDA of one of its most dynamic ever Commissioners, and industry observers have been wondering how this might change the long-term direction of the US regulator.

His successor, Ned Sharpless has been drafted in from his former role as head of the National Cancer Institute (NCI), but remains for now an interim Commissioner, and one certainly less voluble on social media.

Also significant is a notable drop off in the number of new drug marketing authorisations at the FDA this year compared to last, when it achieved a record 59 novel drug and biologic approvals.

This time last year, the US regulator has approved 20 new drugs, whereas the tally stands at just 14 so far this year.

Janet Woodcock

Janet Woodcock

Responding to these trends on Tuesday was one of the agency’s longest serving and most senior officials, Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research.

She was speaking at the SVB Leerink Therapeutics Day in Boston, and reassured the biopharma sector that the agency was still focused on accelerating the drug approval process.

A detailed note from SVB seen by FiercePharma reported that Woodcock was confident that approval levels would remain high.

“In fact, Dr. Woodcock believes that the drug industry is just beginning to see the fruits of the human genome sequencing and expects more and more approvals in future years,” the SVB report said.

Woodcock said the agency had already shifted its own expectations about safety and efficacy data based on the clear move towards therapies for more difficult-to-treat conditions, and had listened to patient views that uncertain efficacy and potentially higher toxicities were acceptable.

These cases have, of course, not been without controversy – one of the most notable being the approval of Sarepta’s Duchenne treatment Exondys 51 in 2016, despite questionable data. This shift has also led to a huge increase in the number of conditional marketing approvals - and critics say the FDA hasn't pursued companies who haven't met post-marketing obligations, or have failed to demonstrate overall survival benefits.

Despite a few doubtful cases, however, confidence in the FDA remains high, and Woodcock was willing to concede that the “gold standard" of randomised, placebo-controlled, double-blind studies may no longer be tenable.

That means the FDA is open to broader use of non-inferiority and other trial protocols, such as dose comparisons and delayed start trials, said the SVB note.

However Woodcock warned that this new approach could bring with it a number of challenges, not least its own Center for Biologics Evaluation and Research (CBER) struggling to meet demand from a potential flood of new cell and gene therapy products.

Woodcock also noted a “wide skill gap” in the industry between big and small companies when it comes to compiling regulatory dossiers, noting that many smaller companies had faced rejection after selecting endpoints in clinical trials that were not clinically meaningful.

Also eyecatching were Woodcock’s comments on two high profile FDA approvals of spinal muscular atrophy (SMA) treatments, Biogen’s Spinraza and Novartis’ gene therapy Zolgensma. She noted that both drugs carried labels that allowed for broad use - but warned that insurance companies might interpret use much more narrowly in such cases.

Before Gottlieb departed earlier this year, he unveiled a new division aimed at building up the FDA’s capacity to deal with the rapid pace of change in  drug R&D and regulation – the Office of Drug Evaluation Science (ODES).

Woodcock said that plans for the ODES were still in progress, but needed approval from Congress before it could be set up.

She was able to reveal that a division focused on neuroscience will operate within the ODES to review novel therapies for psychiatric disorders, neurodegenerative disease, pain and addiction, as this field is expected to see a renaissance in the coming years.

Article by
Andrew McConaghie

11th July 2019

From: Regulatory

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