We have many inquiries regarding user fee funding. We’ll update this week with details on remaining balances. We’re also closely evaluating when we will cross a threshold, beyond which, we have to declare that we will not be able to meet medical product user fee goals for 2019.
Remember the number 62. That is the new benchmark for US drug approvals set in 2018 by a biopharma sector propelled by billions of dollars in investment and a regulatory agency in overdrive.
The US government’s shutdown must cast some doubt over whether such a feat can be repeated in 2019. While user fee-supported drug reviews are so far continuing, the FDA is running out of that revenue stream, and no new applications can be received. Assuming that the shutdown ends in the coming days, drug reviews will resume at a similar pace, but the pause in new applications could mean that approvals slated for 2019 could spill into 2020. A longer delay could spell even worse news.
The FDA commissioner, Scott Gottlieb, told the JP Morgan healthcare conference last week that if the shutdown was not resolved the agency would run out of PDUFA revenue within a month, curtailing its activities. And he acknowledged that the agency could reach a point at which drug review activity would cease entirely.
A government shutdown of that length would be unprecedented – the previous record is 21 days. There are no signs that President Donald Trump and Congress are moving closer to a deal that would reopen much of the government.
Yesterday, Aimmune Therapeutics said the review of its peanut allergy project A101, submitted the day before the shutdown began on December 22, would not start until after funding was restored. And at least one biopharma analyst is cautioning that even short delays will increase costs to sponsors, and that the knock-on effects of the shutdown will continue even once it is resolved.
In the context of the bumper year of 2018, any signs that 2019 will underperform will disappoint investors. The number of annual drug approvals is only one measure of productivity, and an imperfect one at that, but it is closely watched.
So to see 62 approvals – a number that includes decisions from both the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research – should please biopharma bulls, not to mention executives and patient advocates. Exceeding 2017’s 57 approvals is no mean feat, and a sign of a sector in rude health.
|NME approval numbers and time over the past decade|
|Priority review||Standard||Breakthrough therapy|
|Year||Number||Avg time (mth)||Number||Avg time (mth)||Number||Avg time (mth)|
Looking at individual approvals, 2018 saw the launches of an entire class of new migraine drugs in Aimovig, Emgality and Ajovy; 17 cancer drugs, including six for haematological cancers; the first RNAi therapeutic in Alnylam’s Onpattro; and the first-ever antiviral to treat smallpox, Siga Technologies’ Tpoxx.
Behind the raw numbers, 2018 saw a slight reduction in average approval times, to 10.6 months. This came because of a faster average standard review cycle, while priority reviews slowed slightly. There was one potentially concerning trend: drugs awarded breakthrough therapy designations did not move through especially swiftly, with an average approval time greater than for those with priority review.
The apparent slowdown for the highest-priority drugs could be down to volume: 31 of the 62 approved drugs had priority review and 13 had breakthrough status. It might come as no surprise that the agency struggled to keep approval turnarounds on an even pace: the greatest number of priority review projects before 2018 was 17, in both 2015 and 2005. Such trends could make regulators reconsider which submissions ought to qualify for either priority review or breakthrough therapy designations.
Matching or exceeding a record year like 2018 would have been a big task under any circumstances, and the government shutdown will not help matters in 2019. Investors will probably tolerate the minor risk that delayed submissions entail, but if pending drugs reviews are suspended this will be taken poorly.
In light of Mr Gottlieb’s warning, biopharma investors and companies alike will be hoping that the shutdown is resolved and that the FDA is back to full capacity soon. But it is hard to tell when this might happen. If the situation is the same in a month’s time investors will really start to worry.
A Product Manager with expertise in pharma marketing and sales operations