Tuesday, June 20, 2017

Scott Gottlieb's Magical Risk-Based Powers and Orphan Drug Applications

Interesting Numbers

In a Senate Hearing today, FDA Director said that there are over 200 orphan designation applications pending with the agency. He then promised a turnaround on ALL (yup, you read that right) pending applications within 90 days. For anyone interested, that is approximately 2.2 applications per day. Now assume a day is 8 hours and FDA employees are jailed to their desks even on weekends...

A few days ago, I was positive after reading his blog post and I even reported on it here. Now, I am just perplexed to say the least (and pretend that I do not want to say something else altogether!). The FDA is not a clearing agency. Orphan designation requests are not like Fishing Permits or Back Country Hiking Permits. How could we expect any relevant level of review under such stringent timelines?

I understand it is important to clear back logs. I understand the importance of orphan drugs/devices/diagnostics and how anxious patients must be. But there is efficiency and then, there is general madness.

Trust Issues

The FDA's success in keeping US patients safe is seminal to ongoing trust people place on it. Slowly, reluctantly, agencies world over are following the FDA. To go on a mad rampage, potentially endangering patient safety to just move rapidly on applications makes for bad precedent. And, without providing any actual strategy, Gottlieb simply repeats the words "risk based approach".

The magical incantation?

You hear risk based tools once, and you go, okay, that sounds nice. When someone keeps offering the same thing as a "fix it" for everything, you start getting suspicious. Earlier this week, he touted risk-based tools as means to approve digital health applications. Okay. And I do see how they can be applied on all applications. But there is more to a review of applications.

In his defense, he did also say "modern" tools as well, and wants to improve the orphan designation application form. However, none of this sounds too comforting when taken in context.. Yes, risk based approaches to regulation will help improve things. However, they are not a panacea. You obviously need good quality reasoning. Is the condition indeed rare? Are there indeed no other drugs/devices that work? Is the applicant trying to abuse the orphan designation to take advantage of anxious patients? These are all important considerations, and I fail to see, how in about 3.5 hours or so, a decision on all these pertinent questions can be made. This is essentially what Gottlieb promised Congress.

A warning on orphan designations

Some in industry might see dollar signs and be thrilled with this laissez-faire attitude. However, I will remind you, that all is well, only till something goes wrong. Already, the pricing of orphan drugs has created quite a storm, with many questioning whether the Orphan Drug Law is being abused. If applications get a very light review, and the result is either more expensive, poorly effective drugs and devices, or if it results in serious patient injury, it will be a disaster. Patients and advocates engaged in less covered disease and therapy areas are already very vocal and will not excuse failure. Therefore, I sincerely hope this is not a thinly veiled attempt to reduce the quality of the FDA review process.

Since I have always been interested in oprhan drugs, I will keep a close eye to see how he directs the agency to rapidly respond to applications, with the level of scientific review, focused on safety and effectiveness and report back.


1. The RAPS Article: http://www.raps.org/Regulatory-Focus/News/2017/06/20/27941/FDA-Commissioner-Vows-to-Eliminate-Backlog-of-Orphan-Drug-Designation-Requests/?utm_source=Email&utm_medium=Informz&utm_campaign=Informz-Emails

2. Image Courtesy, Pexels: https://www.pexels.com/photo/blur-bokeh-dark-evening-75360/

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