Brooks Blog

Monday • November 06, 2017

FDA Drug Approvals and Crowd-Sourcing Evidence

Review the FDA’s Novel Drug Approvals in 2017 and you’ll find an average rate of about one approval per week, many of which are ahead of schedule. While it’s fashionable to grumble at the speed of drug approvals, the FDA is already faster than the EU equivalent, the European Medicines Agency, and the speed to market looks to be headed on an even higher trajectory.

So what is driving this breakneck speed, and what will drive that trajectory in the future?

#1 The Past: Breakthrough Designations The recent flurry is partly due to congressional breakthrough therapy legislation passed in 2012 that fast-tracked treatments for conditions without “adequate” approved treatments, a policy that has met with optimism, skepticism and criticism.

#2 The Present: Research For One Purpose, Sell for Another?

Federal law prevents manufacturers from promoting to–but not communicating with–providers regarding off-label uses. Meanwhile off-label use is common and some say manufacturers would be the ideal conduit for information related to off-label uses. However, such a change would reduce the incentive to conduct clinical trials. Some argue this would reduce the scientific rigor behind drug approvals. Meanwhile Arizona passed legislation to allow these more communications, although manufacturers are still bound by federal laws against off-label promotion. While promoting off-label uses to providers can lead to some negative publicity in the present, the current administration could remove or reduce manufacturer liability in the future, which might move the needle on what already represents 20% of prescription rates.

#3 The Future: Crowdsourcing Data

As for the future trajectory for not just approvals but post-approval safety and efficacy, it is a stated goal of Scott Gottlieb, the Commissioner of Food and Drugs who some believe may replace Tom Price at HHS, to reduce the FDA’s absolute reliance on randomized clinical trials to ascertain both drug safety and drug efficacy. Gottlieb aims to integrate real world evidence (“RWE”) collected from the public and from electronic medical records, and his reasoning is straightforward:

“Should a product be marketed based on a data set that speaks to a limited and rigidly constructed circumstance, when the clinical use, and in turn the evidence we might have to evaluate the product, could have been far richer, far more diverse, and more informative?”

Incorporating RWE is not new to Gottieb, but the tone was more cautious as relast year. The concerns published by FDA officials in late 2016 were that RWE is too loosely defined, along with rigor and integrity of the data collected:

“The confluence of large data sets of uncertain quality and provenance, the facile analytic tools that can be used by nonexperts, and a shortage of researchers with adequate methodologic savvy could result in poorly conceived study and analytic designs that generate incorrect or unreliable conclusions.”

In Conclusion

Between redefining what constitutes drug safety evidence and potentially removing current regulations that prevent manufacturers from discussing or marketing off-label use of their products, the investment in clinical studies made today could see a major overhaul in the near term.

Speeding up approvals–or skipping them altogether–changes the equation in pharmaceutical market strategy in ways ranging from choosing which FDA approvals to seek to deciding what compounds have the most market potential down the road.

The Brooks Group will be watching to see how this trend continues.

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