Town Hall: FDA Braces for PDUFA V, Possible Loss of Biosimilars Authority

Janet Woodcock looks forward to having more cash and resources with new user fee legislation. Meanwhile, she raises concerns about the Agency’s authority over biosimilars should the Supreme Court strike down the Affordable Care Act.

By Paul Thomas, Senior Editor

At the BIO 2012 exhibition in Boston yesterday, FDA CDER director Janet Woodcock presided over a town hall meeting in which discussion leaned heavily towards PDUFA V—the impending reauthorization of the Prescription Drug User Fee Act—and what its repercussions will be for the drug industry. (Here is FDA info on PDUFA V, which one commentator calls “the only health care bill that is guaranteed to pass Congress this summer.”)

Woodcock was joined on the podium by CBER director Karen Midthun—who summarized her Center’s current priorities, from pandemic preparedness to biosimilars—but it was the gregarious CDER director who did most of the talking, during her presentation and the Q&A with the audience that followed. Woodcock showed a typical feistiness in the face of questions about "regulatory uncertainty" and blanket statements about whether the Agency is as effective and efficient as it could be.

Woodcock discussed CDER priorities, suggesting that PDUFA V, as well as GDUFA (for generics user fees, also expected to be enacted soon), will infuse the Agency with funds to undertake much-needed initiatives. The $299 million anticipated annual intake from GDUFA, she noted, will enable FDA to fulfill its commitments to, for example:
•    Review ANDAs within 10 months
•    Eliminate its review backlog
•    Expand and bring parity to inspections of foreign facilities

What follows is an abridged version of the Q&A:

Audience Question: Where is FDA in terms of mitigating potential drug shortages?

Woodcock: We have a very good drug shortage program once a shortage happens, but the real thing we need to do is prevent these things from happening in the first place. That will take some analysis and introspection on our part.

Audience Question: PDUFA V calls for increased patient input [on drug safety and efficacy, etc.]. What tools or forums will be best to get that input?

Woodcock: We’ve already talked to patient organizations about this. There is no monolithic patient population . . . the challenge is how to get not only authentic input but also representative input …we would hope to not do this all ourselves, but to leverage the work of patient groups and other groups and have them work on this as well.

Audience Question: How does FDA determine which therapies constitute “significant breakthroughs” that deserve accelerated review?

Woodcock: People get confused between “review” and “development.” The main issue is development . . . the thing about breakthrough therapies is, we’re not saying we know one when we see one. We know a potential one . . . in some cases, you have new therapies that are so much more effective than what’s out there that we should think differently about their development. It has very little to do with review—I think the focus on FDA review is extremely misplaced, especially in the PDUFA era. The point is [for the drug sponsor or manufacturer] to get it right from the get-go.

Audience Question: What will be the greatest challenges in implementing PDUFA V?

Midthun: Making sure we have a platform that allows us to implement it. The expectation is that come October 1 we’re doing this. So there are systems that we need to put in place . . . a lot of our work at this point is anticipatory—having our ducks in a row.

Woodcock: On the review side, we have to think through what that would look like. Because patient-focused drug development is very novel, that’s gong to be very hard. None of us know how to do it, and we’re going to be experimenting for a while. But getting the authentic patient voice into the process will help us at the end of the day.

Audience Question: PDUFA V seems to pay attention to addressing “regulatory uncertainty.” How are REMS and other issues that could extend review time being addressed?

Woodcock: What a lot of people mean by “regulatory uncertainty” is really “scientific uncertainty”…but as far as more predictability, yes. One of the goals is to have more first-cycle approvals. …and to have more communication and transparency …for us, more cycles is time-consuming, so we’d like to reduce that as much as possible as well..But unfortunately, we can’t reduce scientific uncertainty too much.

Audience Question: From a management perspective, how do you encourage FDA staff at all levels to embrace the open communications with drug companies that the Agency is striving for?

Midthun: It’s building on many things that we’re already doing . . . people really have learned that you really do have a much smoother process if you have that communication . . . for me, it’s inserting that [communications] piece into drug development that we’re already doing in [drug] review.

Woodcock: When I was in CBER I think we had a more interactive environment. But over time the process got extremely formalized. . . . We recognize in CDER we have to strike the right balance between formality and being official and having back and forth and regulatory exchange. Some people [in different departments within FDA] already feel very confident in having back and forth, while others say, “No way do we do that now.”


Audience Question: What’s the possibility of FDA coming out with its own SBIR (small business investigational research grants, from NIH)?

Woodcock: We aren’t really funded as a research organization. We contribute some to consortia, but usually that’s very highly leveraged . . . the public is actually our customer, but Congress doesn’t think of us that way (as it does NIH). So we aren’t seen as a granting agency—we would require additional authority and funding.

Audience Question: There are additional responsibilities for FDA that are going to be enacted as part of PDUFA. Will those pieces have the same dramatic negative impact on your time and resources that the FDA Amendment Act (FDAAA) had?

Woodcock: I don’t think so. I think the coin is still spinning in terms of what’s going to be in there, but a lot of it is reporting, which does not sap time and resources  . . . there’s nothing [in PDUFA] of the magnitude that has all these legal and regulatory consequences [that FDAAA had].

Midthun: Will there be certain challenges? Sure. But we don’t see anything of the magnitude of the FDA Amendments Act.

Audience Question: If President Obama’s Affordable Care Act were struck down by the U.S. Supreme Court, the new biosimilars statute would be struck down, too. You could make a legal argument that FDA would still have the authority to approve products as biosimilars, but I’ve heard the opposite as well. What do you think? Would biosimilars move forward?

Woodcock: I’m not a lawyer. [She laughs. An audience member jokes: “Thank goodness for that!”] However, we would have done this before if we could have done it. Everybody says FDA is behind Europe [in regards to biosimilars], but it’s because we didn’t have statutory authority before . . . I don’t think the Food and Drug legal minds think we have the authority to [continue to regulate biosimilars]. The general consensus is that we don’t. I always listen to the lawyers even if I don’t like what they tell me.

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