Statistics are like a bikini. What they show you is interesting, but what they conceal is essential.
Nevertheless, someone at the FDA owes the Pink Sheet a thank-you note.
The Pink Sheet reports that:
The agency has been trumpeting the 30 new molecular entity and new biologic entity approvals from CDER in 2011 – the most since 2004 and a welcome sign of recovery after the approvals doldrums in between – but it also deserves recognition for achieving an unprecedented, near-perfect rate of PDUFA review goal compliance (96.7%) while maintaining healthy rates of first-cycle approvals (63.3%), and time to approval (an average of 15 months)
|
CDER Review Performance In The PDUFA IV Era |
|||||
|
Year |
Approvals (average time to approval) |
User fee compliance |
First-cycle review share |
First-cycle share, Standard |
First-cycle share, Priority |
|
2011 |
30 (15 mo.) |
96.7% |
63.3% |
43.8% |
85.7% |
|
2010 |
21 (15.1 mo.) |
86% |
76.2% |
75% |
77.8% |
|
2009 |
25 (17.3 mo) |
72% |
58% |
47% |
66.7% |
|
2008 |
24 (17.9 mo.) |
70% |
67% |
53.3% |
88.9% |
Consistency is becoming a hallmark of FDA’s review activity, with nearly identical average times to approval in the two years following the adjustment period for implementation of the FDA Amendments Act. The striking difference between 2010 and 2011 is the number of approvals, not the parameters of the reviews – showing FDA can deliver the same performance with a higher volume.
Innovative drugs drove the positive review trends, with 12 first-in-class agents among the CDER NMEs and NBEs, representing 40% of calendar year 2011 approvals.
First-in-class status correlates closely but imperfectly with priority review status, another marker of innovation that incorporates not only pioneer mechanisms but also unmet medical needs. By the metrics, the 2011 class did not vary significantly from the level of innovation in recent years, though the significance of some of the innovative products cleared in 2011 certainly makes the class notable.
The difference in time to approval between the first-cycle and second-cycle approvals is stark. On average, the penalty for going to the second review cycle was 19.6 months, the difference between the 7.8 month average time to approval for all NMEs and novel BLAs (CDER and CBER) approved in one review cycle and the 27.5 month average for products requiring two review cycles.
FDA could not have posted a good year for new drug approvals without plentiful applications based on well-constructed clinical programs – an aspect in the hands of industry. But the agency can also make its own luck. The case studies of drug approvals published each month by Pharmaceutical Approvals Monthly suggest that the agency will show great creativity and flexibility in analyzing and acquiring data for new products that address unmet medical needs.
The Pink Sheet ends it’s analysis with the comment, “As the agency heads into PDUFA reauthorization, FDA’s review success will be a powerful argument that the agency knows what it is doing. Just look at the numbers.”
Just so, but as Vince Ventimiglia, Former Assistant Secretary for Legislation at the US Department of Health and Human Services, commented at a recent Center for Medicine in the Public Interest conference on PDUFA, numbers are very much determined by who provides them. He called one myth about PDUFA, “Lie to Me." When it comes to numbers, he asks, “Are they always true? And the answer is no. It behooves you to look very carefully at FDA numbers, Department of HHS numbers, and OMB numbers as a package. They’re not always in sync with each other."
As Mark Twain said, “Facts are stubborn things, but statistics are more pliable.”
