Transparency in Action: FDA Comments on Pirfenidone

It's no secret: FDA review managers would like to make "complete response" letters public.

We've heard that directly from them, and now the idea is moving forward as one of a number of proposals from the agency's Transparency Task Force.

This idea, to put it mildly, provokes mixed feelings in industry. "Mixed" in the sense that sponsors love the idea of seeing their competitors' complete response letters, but hate the idea of having their own made public.

But a recent presentation by FDA officials during the American Thoracic Society's annual conference has us wondering what the fuss is all about.

As reported by The Pink Sheet (relying on a transcript by one of the Wall Street analyst firm, ThinkEquity, who attended the briefing), medical officer Banu Karimi-Shah discussed the recent complete response letter for Intermune's IPF therapy pirfenidone, and outlined the agency's position that the existing dataset does not meet the statutory definition of "substantial evidence," and therefore another clinical trial is required. Karimi-Shah also discussed issues around the adequacy of forced vital capacity as a surrogate endpoint, and the relevance (or lack thereof) of Japanese clinical data with a different formulation of pirfenidone.

FDA rejected the application May 4, over-ruling an FDA advisory committee that voted in favor of approval--though the committee had significant misgivings about the sufficiency of the data as a basis for approval.

This is not be the first time an FDA official has commented on a non-approval decision during a public forum, but the extensive discussion of FDA's issues with the application can hardly be described as routine. So we asked the agency whether the remarks were cleared by top FDA officials or posed any concerns regarding the appropriateness of discussing the elements of a Complete Response.

Here is what the agency told us, by email:

"The information presented by Dr. Karimi-Shah was presented and discussed at the Pulmonary-Allergy Drugs Advisory Committee meeting on March 9, 2010, so her presentation was already in the public domain. Intermune disclosed in a press statement on May 4, 2010, that the FDA issued a CR letter and the FDA requested an additional clinical trial to support the efficacy of pirfenidone, so this information was also in the public domain."

See? There is really no reason to argue about making CR letters public after all. Right?

Okay, our tongue is firmly in our cheek on that. We'll have much more on the reaction to the transparency proposals in an upcoming issue of The RPM Report.

Drug Safety...or Food Safety...or Tobacco Safety...or Pet Food Safety...or Dietary Supplement Safety?

So which is most important to you? Well, if you're in charge of FDA, they all are. That came through loud and clear during a health care reporters breakfast with commissioner Andrew von Eschenbach organized by the folks at Health Affairs.

But fortunately for us here at the IN VIVO Blog, we only have to worry about drugs and biologics. With Congress taking off for their August recess, it looks like the Prescription Drug User Fee Act reauthorization as part of the much grander FDARA drug bill will have to wait until September to get passed.

So wait, doesn't that mean droves of FDA employees will be out of a job come September 1st because there will be no money to pay them (FDA gets half of its drug review budget from user fees)? Not so much. Von Eschenbach said the agency has already dipped into "carry-over" funds to ensure the agency will run smoothly while they wait for lawmakers to act. He expects/hopes the bill will pass in the first few days when the House and Senate come back from recess. Von Eschenbach emphasized he was working very closely with specific players in Congress to make sure that happens. The "carry-over" money, which seems to have magically appeared, is enough to last a couple of months before FDA would have to send out lay-off notices.

The RPM Report will be hosting a webinar on August 14 in partnership with the law firm Ropes & Gray to explain the commercial impact of the new drug safety rules for pharma and biotech companies. This will be the first in a series of audioconferences on the pending drug safety reforms.

The RPM Report has fancy new e-digs

Our colleagues from The RPM Report (and fellow bloggers here at The IN VIVO Blog) have a beautiful new web site over at http://therpmreport.com/, which we wholeheartedly encourage you to visit for the best analysis of biopharma regulation/policy/market access on these here internets.