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Scott Gottlieb gave a savvy review of what we can expect at the FDA; When times got rough, a biotech CEO demonstrated real integrity

Smart, reasonable and extremely well prepared: What did we learn from Scott Gottlieb’s confirmation hearing?

On Wednesday, when Scott Gottlieb showed up for his confirmation hearing at the Senate, it became quickly apparent that the prospective FDA commissioner was the best prepared person talking that day. What the Dems had billed as a sharp attack on Gottlieb’s industry ties quickly disintegrated into a series of random, disjointed comments directed against largely unrelated issues.

Commissioner-designate Scott Gottlieb at his hearing Zach Gibson/Getty Images


The Republicans were there to pat him on the back, which they managed to do just fine. A few used the occasion to preen to their voters. It’s what lawmakers do. No matter.

It was a non-event if you were looking for fireworks and confrontation. If you listened carefully, though, there was some real meat to this exchange as well, and some vitally important insights into how he sees his role in managing reform from the inside rather than establishing himself as the agency’s biggest critic.

It was a hugely important moment for the biopharma industry, which has had to fear the worst over the past three months.

Gottlieb looked like he had been prepping for this moment for the past 20 years, or more. He has a background in the FDA and deep ties to the industry. Over the past decade he has amassed a lineup of connections to investment groups and biopharma companies. He will not set himself in opposition to the industry, nor should he. More importantly, he’s prepared to champion the R&D gold standard on safety and efficacy.

Three cheers. But in addition to all those jobs he pursued under the Obama administration, there was one more position he volunteered for: FDA commissioner-in-waiting.

Back before his nomination, I took a close look at Gottlieb. Based on the public record and people close to him, it was clear he had a specific agenda in mind. And he ticked off most of those boxes to the senators.

He was going to go after complex generics, clearing the way for new competition to some very extensive therapies. He would celebrate Richard Pazdur’s clear achievements in accelerating cancer drug reviews — a role that has overhauled and speeded a massive amount of product development in the field — and promised to get some laggards at the agency up to speed.

He likes adaptive trial designs, looking for new and better ways to intelligently assess safety/efficacy.

Drugs for rare diseases also look like they’ll be a priority. There’s a way for skilled FDA staff to oversee small, focused studies for new drugs that can avoid the mess that Sarepta created and still suffers from.

I would have liked to have heard more specifics, such as publishing CRLs (or transparency in general), how he will overhaul the orphan drug process and doing away with the need for some outcomes studies ahead of approvals, when they can be done afterwards.

(Though can you also hear payers sharpening their knives for those drugs? When regulatory risk declines, payer risk rises disproportionately these days. And after Gilead’s Sovaldi primed the pump, it’s a brand new day in the payer universe.)

Maybe we can get some of that extra detail in the final Q&A round to come. Publishing CRLs won’t overly disturb the responsible players in the industry and Gottlieb could add some much-needed transparency as a way to distinguish his stint as an independent commissioner. It’s not all going to be cozy and fun.

What I was particularly pleased to hear is that Gottlieb views recruiting talent as his biggest challenge, while looking for a “bottom up” approach from the centers to drive reform. Gottlieb’s stint as FDA commissioner would be long and fruitless if he took a confrontational position with staff.

So what do we get?

We get a commissioner who’s pledged to speed things up some more, responsibly. He’s going to bring in new, badly needed talent. And we hope he clearly chooses transparency over the long and unacceptable black box that the agency has long been required to work in — though that has yet to be seen.

I’ll take it.


Integrity matters more than ever in biopharma

I’ve been writing about biopharma on a daily basis for close to 14 years now. And I’m more than familiar with the restructuring drill.

Company X runs into some trouble. The biotech boat becomes a life raft, or a log, and staffers have to go. There’s a press release, a canned quote from the CEO saying how grateful they were to the newly jobless, and on to the next thing.

So I was more than a little shocked when CEO Ron Cohen reached out to talk to me about the layoffs and downsizing at Acorda this week following a setback on his patent front for Ampyra. That never happens. But he spelled out what the cuts meant, where the ax would fall and how they could monetize assets.

Cohen has a Plan B. He hasn’t been standing on the tracks, waiting for the train to come. It’s extraordinarily high risk, and Plan B hasn’t always gone according to plan over the years — which is par for this unique course. And now failure is not an option.

That’s not a great place to be. No one envies his position. But instead of ducking and taking the easy way out, Cohen demonstrated real integrity in standing up and taking it all on, directly.

Sure, you could throw a cheap shot or two in his direction. But in this industry integrity matters more than ever. And I respect it more than ever. We all should.


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John Carroll, Editor and Co-Founder

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