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FDA Leadership After Marty Makary: What Biotech Needs Next · Making Medicine

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  Speaker 1: At Making Medicine. We're reporting on the headlines, not making
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  Speaker 1: medical recommendations. For personal health questions, always ask your doctor.
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  Speaker 1: Nothing in this episode constitutes investment, financial, or legal advice.
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  Speaker 1: Consult your own advisors before making decisions. When there's uncertainty
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  Speaker 1: at the top of the agency, wherever it comes from,
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  Speaker 1: that uncertainty has a cost. Venture capital gets allocated elsewhere,
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  Speaker 1: trials get delayed, patients wait longer. Welcome back to the
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  Speaker 1: Making Medicine podcast, where science, policy and innovation meet. I'm
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  Speaker 1: your host, John Stanford. Today's episode is following the biggest
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  Speaker 1: headline in biotech, What's next at the FDA. We'll briefly
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  Speaker 1: catch up on the latest leadership news, which we've covered
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  Speaker 1: at length in previous episodes, before we turn to what
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  Speaker 1: the agency and whoever is leading it needs to be
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  Speaker 1: doing to shape this pivotal time in the development of
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  Speaker 1: new medicines. Let's get into the show. Let's start first
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  Speaker 1: by taking a look where things stand, because the news
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  Speaker 1: cycle has been moving fast over the past week. Multiple
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  Speaker 1: outlets have reported that the White House has signed off
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  Speaker 1: on a plan to replace FDA Commissioner McCarey. The President,
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  Speaker 1: when asked about it, directly said he quote knows nothing.
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  Speaker 1: One of the joys of recording a podcast weekly is
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  Speaker 1: some days we record Tuesday morning for episodes that air
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  Speaker 1: on Thursday. But as of this recording, the Commissioner is
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  Speaker 1: still in his job. He's still scheduled to appear before
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  Speaker 1: the Senate Appropriation Subcommittee on Wednesday, and still slated to
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  Speaker 1: be interviewed by Friend of the Show and points his
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  Speaker 1: Max Bear on Thursday. There's been a lot of reporting
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  Speaker 1: on why he might be let go, disputes over specific decisions,
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  Speaker 1: internal tensions at HHS, and pressure from outside groups. We're
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  Speaker 1: not going to re litigate any of that here, as
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  Speaker 1: it's been written about at nauseum. So what we're going
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  Speaker 1: to do is zoom out. It might surprise you to
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  Speaker 1: know that the f regulated products food, medical products, cosmetics,
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  Speaker 1: and even veterinary products represent about a fifth of the
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  Speaker 1: overall US economy. Some of the reported friction is stemmed
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  Speaker 1: from disputes over products outside of Incubate's lane. That's to say,
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  Speaker 1: not medicine, but the FDA is the gatekeeper between a
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  Speaker 1: promising molecule sitting in a lab and a therapy a
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  Speaker 1: patient can actually access. That's why We're focused on it
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  Speaker 1: today because when there's uncertainty at the top of the
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  Speaker 1: agency wherever it comes from, that uncertainty has a cost.
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  Speaker 1: Venture capital gets allocated elsewhere, Trials get delayed, patients wait longer.
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  Speaker 1: So at making medicine. We wanted to use the episode
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  Speaker 1: to talk about what makes a good FDA commissioner and
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  Speaker 1: how that leadership drives the agency. We broke it down
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  Speaker 1: into a few qualities that we'll talk about. None of
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  Speaker 1: these are radical, none of them are partisan. They're what
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  Speaker 1: every FDA commissioner of the modern era has been measured against,
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  Speaker 1: regardless of who appointed them, is being pro science. This
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  Speaker 1: is foundational. The FDA exists to determine whether treatments are
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  Speaker 1: safe and effective based on evidence. A commissioner who is
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  Speaker 1: pro science is one who respects the agency's career reviewers
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  Speaker 1: and treats their judgment as the starting point, not an obstacle.
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  Speaker 1: That doesn't mean a commissioner is a rubber stamp. Benefit
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  Speaker 1: risk calls are genuinely hard. Sometimes leadership has to weigh in,
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  Speaker 1: but the default has to be the science leads. Decisions
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  Speaker 1: are grounded in data. Advisory committees do their work in
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  Speaker 1: public Frankly, advisory committees meet at all, Descents from career
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  Speaker 1: staff get aired, not buried. The agency's credibility has been
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  Speaker 1: its most valuable asset. So when commissioners override career scientists
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  Speaker 1: without a good reason why or a public reason why,
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  Speaker 1: that credibility erodes, and once it's gone, it'll take a
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  Speaker 1: while to rebuild. The second quality we think is important
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  Speaker 1: is regulatory experience. Is one of the most complex regulatory
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  Speaker 1: institutions on the planet. It runs on user fee agreements
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  Speaker 1: negotiated with industry. It has statutorily defined review pathways from
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  Speaker 1: accelerated approval, breakthrough designation, priority review, rare, pediatric disease voucher programs,
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  Speaker 1: all of which we've covered on the show in detail,
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  Speaker 1: and every one of them has their own nuances. Those
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  Speaker 1: nuances are covered by decades of guidance documents that shape
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  Speaker 1: how every clinical trial in this country gets designed. A
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  Speaker 1: commissioner doesn't need to have run a seater review to
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  Speaker 1: be effective, but the FDA is at a particularly pivotal moment.
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  Speaker 1: We could talk about any number of opportunities for modernization,
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  Speaker 1: but to put some color on this, we want to
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  Speaker 1: dig deeply into one of them. There's been so much
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  Speaker 1: hype about what AI can do for drug development, and
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  Speaker 1: while a lot of it remains hype, it's important that
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  Speaker 1: the FDA focus right now on developing the guidance and
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  Speaker 1: regulatory structure to embrace AI when it came and make
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  Speaker 1: a meaningful difference. One example in particular that we've been
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  Speaker 1: thinking about is how AI could be used to identify
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  Speaker 1: patients that might be interested in clinical trials. As making
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  Speaker 1: medicine listeners will know, clinical trials are one of the
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  Speaker 1: longest phases in developing a new medicine, and if we
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  Speaker 1: can truncate that, if we can shrink it, not only
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  Speaker 1: will that mean that we're moving drugs faster to patients,
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  Speaker 1: it's also going to improve our global competitiveness. Check out
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  Speaker 1: any one of our episodes on China or Australia or
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  Speaker 1: other countries that have found ways to bring drugs to
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  Speaker 1: market faster, and you're going to be looking at what
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  Speaker 1: they do differently with clinical trials. So there's a lot
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  Speaker 1: of promise that AI could help identify patients faster and
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  Speaker 1: get them into those but AI runs on data and
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  Speaker 1: in the US, unlike in the United Kingdom, with the
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  Speaker 1: National Health Service or other governments that have centralized data
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  Speaker 1: repositories are data is fragmented. It's with insurers, it's with hospitals,
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  Speaker 1: it's with individual patients. So for us to have an
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  Speaker 1: AI system that actually makes clinical trials more relevant to
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  Speaker 1: American patients, we're going to have to be thinking about
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  Speaker 1: how we integrate that data, how we make it available,
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  Speaker 1: and how industry on the drug development side talks with
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  Speaker 1: industry on the claims and hospital side. That's a conversation
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  Speaker 1: that the FDA could be leading right now, bringing people
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  Speaker 1: together to think about how we're going to be ready
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  Speaker 1: for when really meaningful things can be done with artificial intelligence.
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  Speaker 1: It's for that reason that expertise in healthcare and regulatory
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  Speaker 1: has to be one of the critical qualities of an
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  Speaker 1: FDA commissioner, and regulatory experience also means knowing what the
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  Speaker 1: agency can't do. The FDA isn't a pricing authority, it
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  Speaker 1: isn't a coverage authority, and confusing the agency's mission with
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  Speaker 1: adjacent policy goals, even well intentioned ones, tends to produce
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  Speaker 1: bad outcomes for everyone. The third quality we want to
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  Speaker 1: see is someone who wants to oversee a stable, predictable
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  Speaker 1: This is the one that the investment community feels most acutely.
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  Speaker 1: Investors put capital at risk on decade long timelines. A
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  Speaker 1: biotech raising a series A today is making decisions based
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  Speaker 1: on the regulatory pathway of the twenty thirties. Predictability doesn't
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  Speaker 1: mean rigidity. Science evolves, standards get updateds. Novel techniques like
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  Speaker 1: gene therapies require the agency to adapt. That's healthy and
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  Speaker 1: on that front, this FDA has been doing real work
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  Speaker 1: that incubate and our members have supported the push on
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  Speaker 1: new approach methodologies, alternatives to traditional animal testing, organ on
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  Speaker 1: chip systems, human derived models, computational simulation. That's meaningful modernization
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  Speaker 1: and a lot of credit should go to this FDA
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  Speaker 1: for willing to push the boundaries in a way that
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  Speaker 1: can be effective. Only a couple weeks ago, we had
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  Speaker 1: our partners at menat Health on that shared these exact
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  Speaker 1: views from sixteen investors, and I encourage you to check
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  Speaker 1: out that episode to dig deeper about what vcs want
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  Speaker 1: out of an FDA. Well close today by talking about
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  Speaker 1: why this matters, bringing the conversation back to patients, because
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  Speaker 1: it's easy for a conversation about FDA leadership and capital
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  Speaker 1: allocation to lose sight of what this is actually about.
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  Speaker 1: Take for example, that ninety five percent of diseases still
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  Speaker 1: have no FDA approved treatment. Thirty million Americans are living
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  Speaker 1: with a rare disease, as we talked about our episode
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  Speaker 1: in Rare Disease Week. So for every patient out there
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  Speaker 1: that's hoping a better drug is coming along, the difference
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  Speaker 1: between a commissioner who runs a predictable, science driven process
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  Speaker 1: and one who doesn't isn't abstract whether that treatment reaches
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  Speaker 1: them in time or doesn't. We've been watching this play
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  Speaker 1: out in real time. A few weeks ago, we covered
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  Speaker 1: the Casear Life Sciences story, a cautionary tale if there
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  Speaker 1: ever was one, about what an effective FDA means, what
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  Speaker 1: an ineffective FDA can mean to a small biotech. Earlier
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  Speaker 1: this month, Passage Bio announced its restructuring, cutting it it's
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  Speaker 1: workforce by seventy five percent after the FDA pushed back
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  Speaker 1: on its lead gene therapy for dementia. The CEO put
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  Speaker 1: it plainly, FDA wouldn't support a single arm trial for
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  Speaker 1: the indication and said a randomized control trial would be required.
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  Speaker 1: That's a company trying to treat a devastating disease that
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  Speaker 1: has natural history to back it up running into a wall.
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  Speaker 1: So this is a story about patients and the FDA
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  Speaker 1: has to get it right, but it's also a competitive story.
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  Speaker 1: China has now overtaken the United States and the number
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  Speaker 1: of new drug clinical trials initiated each year. It's not
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  Speaker 1: a headline we ever expected to read. And this happens
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  Speaker 1: right in the very week where President Trump is headed
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  Speaker 1: over to China to talk about the two countries' futures.
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  Speaker 1: We'll note that there are no biotech CEOs traveling with
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  Speaker 1: the President, so we're not sure that we're on the agenda.
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  Speaker 1: But the US has led in biopharmaceutical innovation for decades,
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  Speaker 1: strong IP, deep capital markets, world class research universities, and
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  Speaker 1: an FDA that set a global standard for regulatory rigor.
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  Speaker 1: So that final point, that last leg of what has
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  Speaker 1: built our dominance over the decades is the big question
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  Speaker 1: mark for our industry and that's why it's been getting
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  Speaker 1: the headlines. So to whoever is sitting in the Commissioner's
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  Speaker 1: chair when this episode drops Thursday morning, and whoever is
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  Speaker 1: sitting it in a year from now, Our message at
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  Speaker 1: making medicine won't change. We're looking for someone pro science,
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  Speaker 1: We're looking for someone with real regulatory experience, and most importantly,
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  Speaker 1: we're looking for someone who wants to oversee a stable
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  Speaker 1: and predictable process. By doing that, that FDA Commissioner will
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  Speaker 1: ensure that we're doing what's right for patients and remaining
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  Speaker 1: globally competitive. These aren't controversial asks. They're what built the
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  Speaker 1: FDA into the institution it is today. Thanks for listening,
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  Speaker 1: and tune in next week to see how some of
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  Speaker 1: this conversation unfolds. Until then, be sure to subscribe on
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  Speaker 1: YouTube and follow us on our other channels so you
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  Speaker 1: don't miss those upcoming conversations. And if you have thoughts
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  Speaker 1: on anything we discussed today it might affect your portfolio
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  Speaker 1: or your company, let us know we might feature your
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  Speaker 1: comment in next week's episode. Thanks for listening, and as always,
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  Speaker 1: keep innovating