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Protecting Innovation: Small Molecules and the Future of Medicine · Making Medicine

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  Speaker 1: You're listening to Making Medicine Stories from the Early stage
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  Speaker 1: life Sciences Ecosystem, a podcast that explores the people and
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  Speaker 1: deals that have led to the medicines, devices, and technologies
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  Speaker 1: that keep us healthy. Let's get into the show.
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  Speaker 2: Hello, and welcome back to the Making Medicine podcast. This
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  Speaker 2: is your host John Stanford. Today we have Kirsen Axelson
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  Speaker 2: from Incubate's Policy Lab joining us to talk about her
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  Speaker 2: latest white paper which is focused on small molecules. And
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  Speaker 2: this episode is dropping on nine thirteen, September thirteenth, and
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  Speaker 2: that is not a mistake because September thirteenth or nine
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  Speaker 2: thirteen is the day that Incubate and many other advocates
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  Speaker 2: from patient communities to biotech CEOs to investors are calling
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  Speaker 2: attention to the small molecule penalty and the fact that
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  Speaker 2: one type of medicine gets far less of a reward
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  Speaker 2: nine years versus the thirteen year received by biologics. That's
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  Speaker 2: why nine thirteen is a day to call attention to
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  Speaker 2: all the incredible innovation happening from small molecule companies. So
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  Speaker 2: it's great to have Kirsten on the show, who has
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  Speaker 2: just put out a white paper discussing all of these. So, Kirsten,
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  Speaker 2: it's great to have you back on the podcast. As always,
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  Speaker 2: this whole week we've been talking about small molecules. We've
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  Speaker 2: had members of Congress posting videos about how important small
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  Speaker 2: molecules are. We've had patients, we've had scientists, and here
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  Speaker 2: we are ready to talk about your white paper. But
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  Speaker 2: for our listeners, they're probably wondering what the heck are
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  Speaker 2: we talking about when we say small molecules. So can
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  Speaker 2: you lay it out in the most basic terms what
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  Speaker 2: we mean when we're talking about small molecules, large molecules, biologics,
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  Speaker 2: give us the one oh one on all things size
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  Speaker 2: of medicine.
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  Speaker 3: Thank you so much. Happy to be here with you
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  Speaker 3: today at participating with the Incubate Policy Lab. And as
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  Speaker 3: complex as molecular chemistry may be, I have to say,
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  Speaker 3: I think it may pale in comparison to some of
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  Speaker 3: these policy decisions that have been made that differentially impact
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  Speaker 3: small and large molecules. So to begin, just start at
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  Speaker 3: the very basics, right, the physical world is made up
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  Speaker 3: of atoms, and then atoms bind together to create larger
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  Speaker 3: structures that are called molecules, and those are all the
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  Speaker 3: building blocks of life. So you know our bodies are
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  Speaker 3: made up of water, carbon dioxide. Those are molecules. Molecules
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  Speaker 3: are also acetaminifin a mo oxacillin an insulin. Molecules come
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  Speaker 3: in different shapes and sizes. Small molecules can get inside
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  Speaker 3: of a cell. That can be useful if you're trying to,
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  Speaker 3: for example, kill the cell, such as in the case
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  Speaker 3: of an antibiotic. Small molecules can get inside of the brain.
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  Speaker 3: Large molecules attach to things in our bodies. So molecules
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  Speaker 3: come in different shapes and sizes, and that allows them
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  Speaker 3: to have access to different parts of our bodies and
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  Speaker 3: this helps relieve our pain, regulate our bood sugar, or
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  Speaker 3: kill bacteria. Both small and large molecules are incredibly important
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  Speaker 3: to therapeutics, to both drugs and vaccines. In addition to
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  Speaker 3: small and large molecules, there's also gene and cell therapies
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  Speaker 3: and stem cell therapies. These are derived from blood and
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  Speaker 3: can create an immune response in the body or repair
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  Speaker 3: damage tissue create new cells. So all of these types
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  Speaker 3: of medications or all of these types of delivery mechanisms
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  Speaker 3: are important to improving our health and none of them
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  Speaker 3: should be more punitively impacted by a certain policy than others.
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  Speaker 3: If we want science to follow, what is most curatives
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  Speaker 3: opposed to science to follow, what is the least likely
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  Speaker 3: to have a negative reimbursement effect as a result of
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  Speaker 3: a poorly informed policy decision.
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  Speaker 2: Well, I appreciate that, and it is good to go
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  Speaker 2: back to chemistry one on one, and I think I
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  Speaker 2: agree that at times, certainly, our healthcare policy is far
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  Speaker 2: more complex than basic kinesiology and pharmacology. I want to
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  Speaker 2: stay with this idea for a little bit of these
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  Speaker 2: different types of medicines. Is it fair to say that
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  Speaker 2: fifty years ago, if one of us got sick, we
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  Speaker 2: would buy and large probably be being prescribed a small
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  Speaker 2: molecule medicine. Are these other modalities that you've talked about
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  Speaker 2: by logics selling gene therapies?
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  Speaker 4: Are those newer?
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  Speaker 2: What's the timeline like on the development of these things?
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  Speaker 3: Large molecules cell gene therapy are newer, and both fifty
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  Speaker 3: years ago and today, if one of us gets sick,
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  Speaker 3: we're probably going to be prescribed a small molecule medicine.
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  Speaker 3: In fact, we're probably going to be prescribed a small
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  Speaker 3: molecule generic medicine because ninety percent of all the medications
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  Speaker 3: in the drug delivery system, more than ninety percent are generics,
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  Speaker 3: and the vast majority of those are small molecule medicines.
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  Speaker 3: Small molecule medicines are easy to manufacture, they are generally
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  Speaker 3: stable on the shelf. There are complex small molecule medicines
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  Speaker 3: such as long acting injectables, but yes, the majority of
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  Speaker 3: the medications we take are small molecule medicines, which have
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  Speaker 3: worked to dramatically reduce depth from heart disease. For example,
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  Speaker 3: most heart disease. Vast majority of heart disease medicines are
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  Speaker 3: small molecules, as are antibiotics, and many of the medicines
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  Speaker 3: for cancer are also small molecules, as well as medicines
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  Speaker 3: that help people not reject their transplants after a major surgery.
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  Speaker 3: So small molecules medicines are important on their own, but
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  Speaker 3: they're also important as an adjunct to other major interventions
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  Speaker 3: such as surgery.
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  Speaker 2: And it sounds like not only were they around a
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  Speaker 2: long time ago, but we're also getting new small molecule
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  Speaker 2: drugs all the time. Is it unfair to frame small
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  Speaker 2: molecule as any less innovative? Are there still breakthrough medicines
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  Speaker 2: coming from the small molecule community.
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  Speaker 3: There are still breakthrough medicines. In fact, one of the
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  Speaker 3: novel approaches to developing small molecule medicines is synthetic chemistry
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  Speaker 3: and artificial intelligence, which can be used to strigreen thousands
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  Speaker 3: of potential drug candidate molecules, and one application is using
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  Speaker 3: as developing targeted therapeutics that can cross the bacterial membranes
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  Speaker 3: to kill bacteria. This is particularly important in as we
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  Speaker 3: consider antibiotic resistance and the need to be able to
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  Speaker 3: develop new antibiotics that can kill different bacteria as they
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  Speaker 3: mutate and respond to existing antibiotics. So yes, there are
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  Speaker 3: still novel mechanisms in small molecule development. Small molecules are
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  Speaker 3: also being used for targeted cancer therapeutics. There are still
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  Speaker 3: many new development programs and a lot of innovation happening
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  Speaker 3: in small molecule medicines.
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  Speaker 2: You laid out there, here's in a lot of different
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  Speaker 2: therapeutic areas, and I think this is something that's really
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  Speaker 2: gotten a lot of patients focused on some of the
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  Speaker 2: issues around policy and the size of drugs.
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  Speaker 4: As you've laid.
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  Speaker 2: Out, but maybe zooming out where are the therapeutic areas, you
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  Speaker 2: see small molecule drugs and medicines having the most impact.
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  Speaker 2: And are there any therapeutic areas where frankly, small molecules
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  Speaker 2: are our only option for having therapeutic impact.
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  Speaker 3: Well, I think important thing to remember is one hundred
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  Speaker 3: percent of large molecule medicines cannot cross the blood brain barrier,
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  Speaker 3: and two percent of small molecules can. So when we
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  Speaker 3: think about therapeutics that need to get inside of a cell,
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  Speaker 3: get inside of the brain and act on the brain,
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  Speaker 3: and that can include cancer, that can include neurological disease,
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  Speaker 3: that is an essential, deliberate mechanism. But again I go
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  Speaker 3: back to saying both small and large molecule medicines are important.
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  Speaker 3: There are large molecule cancer medicines. There are the majority
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  Speaker 3: of cancer medicines, though are small molecules. The majority of
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  Speaker 3: heart disease medicines are small molecules. But there's again innovation
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  Speaker 3: going on in both. So the vast majority of therapeutic
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  Speaker 3: areas now are still small molecules, because the vast majority
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  Speaker 3: of medicines are still small molecules, and we do not
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  Speaker 3: want to deter the development of small molecules more than
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  Speaker 3: large molecules for a number of reasons. One is a
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  Speaker 3: therapeutic mechanism that small molecules can achieve. But also when
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  Speaker 3: a small molecule medicine becomes a generic it is the
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  Speaker 3: cost savings are dramatic because again those medicines are inexpensive
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  Speaker 3: to produce. You typically have multiple competitors in a large
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  Speaker 3: therapeutic area, and we've seen billions of dollars of savings
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  Speaker 3: from small molecule medicines. So we want to encourage both
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  Speaker 3: the development of new medicines, but also remember that with
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  Speaker 3: a robust marketplace of small molecule medicines, we also get
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  Speaker 3: a lot of cost savings at the end of the
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  Speaker 3: patent life.
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  Speaker 4: Yeah, I think that's important.
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  Speaker 2: We've talked before on the show that trade off with society,
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  Speaker 2: the importance of intellectual property. And the great news is
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  Speaker 2: that when that intellectual property and those protections expire for
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  Speaker 2: small molecules, especially, that competition is right there in the
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  Speaker 2: form of generic and the prices dropped down to very
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  Speaker 2: low amounts, almost at times too low, but we'll save
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  Speaker 2: that for another episode. You mentioned about savings, and this
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  Speaker 2: is something that incubators working with the Congressional Budget Office
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  Speaker 2: to change how they model some of the projected savings
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  Speaker 2: from the Inflation Reduction Act. And that's along the lines
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  Speaker 2: of if the small molecule penalty plays out ten fifteen
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  Speaker 2: years down the road, and investors have moved away in
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  Speaker 2: a meaningful way towards other modalities in seeking better rewards
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  Speaker 2: and reward terms, and we have fewer small molecules from
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  Speaker 2: a cost perspective, not necessarily a curative or therapeutic perspective.
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  Speaker 2: Is there a difference between a patient accessing a small
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  Speaker 2: molecule versus accessing a biologic and what kind of consequence
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  Speaker 2: might that have for the US Treasury, which saving them
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  Speaker 2: dollars was one of the big drivers of the Inflation
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  Speaker 2: Reduction Act in the first place.
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  Speaker 3: Absolutely, small molecule medicines tend to be even branded, tend
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  Speaker 3: to be lower cost on average relative to large molecule medicines.
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  Speaker 3: You know, the vast majority of large molecule medicines are
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  Speaker 3: what we would call specialty drugs with price tags and
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  Speaker 3: the multiple thousands of dollars per month. Often in medicare,
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  Speaker 3: those specialty drugs are accessed through their insurance plan on
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  Speaker 3: what's called a co insurance tier, weaning the patient pace
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  Speaker 3: twenty five percent or more of the cost of that drug.
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  Speaker 3: Small molecule medicines are more likely to be accessed through
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  Speaker 3: a copay type structure, where they're paying forty five dollars
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  Speaker 3: typically on average for third tier placement and a copay,
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  Speaker 3: so they tend to be both lower cost to the
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  Speaker 3: payer and lower cost to the patient when it's a
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  Speaker 3: small molecule drug. But then again, the tremendous savings comes
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  Speaker 3: when there is generic or in the case of a
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  Speaker 3: large molecule biosimilar competition. Biosimilar competition tends to come in
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  Speaker 3: at around fifteen to fifty percent discount to the innovator
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  Speaker 3: a large molecule and stays around there, some achieving somewhat
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  Speaker 3: more than fifty percent. But with small molecules, within six months,
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  Speaker 3: savings tend to be ninety to ninety five percent of
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  Speaker 3: relative to the innovator medicine before it went off patent,
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  Speaker 3: So you just get much more savings from the small
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  Speaker 3: molecule medicines. They are cheaper to manufacturer, so it can
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  Speaker 3: be at more straightforward manufacture, so there's a different approval
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  Speaker 3: pathway to the FDA. Because a small molecule medicine can
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  Speaker 3: be copied almost identically and can be expected to work
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  Speaker 3: in a very similar way in a person's body.
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  Speaker 2: That makes a lot of sense that the sort of
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  Speaker 2: market forces that drive down the costs are greater on
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  Speaker 2: that side of the house.
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  Speaker 4: And so I think you know, as you've said before, we.
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  Speaker 2: Really want to be in a world where science is
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  Speaker 2: leading the way on what we decide, and we want
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  Speaker 2: to live in a world with small molecules alongside biologics
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  Speaker 2: and selling gene therapies and stem cells. But they're the
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  Speaker 2: workhorse of our healthcare system. And you've been so gracious
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  Speaker 2: with your time, and so I want to kind of
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  Speaker 2: close with this question. We've discussed that there are certain
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  Speaker 2: therapeutic areas where the value of small molecules really seems
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  Speaker 2: to differentiate themselves. So there are places where they're just
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  Speaker 2: simply better for the patients, and you laid out a
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  Speaker 2: few including challenges like AMR or post surgery, and a
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  Speaker 2: lot of this is detailed in the white paper, which
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  Speaker 2: you can find at Incubatecoalition dot org. We've also talked
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  Speaker 2: about that there are some cost savings to the system.
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  Speaker 2: So I want to finish with something that's very popular
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  Speaker 2: and rightfully so discussed in the world of healthcare access today.
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  Speaker 2: That's a concept of equity. So with that same theoretical
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  Speaker 2: future where we've moved a little bit away from small molecules,
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  Speaker 2: what consequences can this mean in terms of certain populations
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  Speaker 2: being able to access the medicines they need.
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  Speaker 3: Well, when you think about going back to some of
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  Speaker 3: my earlier points on the transportation of small molecule medicines,
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  Speaker 3: how they need to be stored, small molecule medicines don't
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  Speaker 3: require special handling, for the most part, can be accessed
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  Speaker 3: through most pharmacies typically are taken by the person, not
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  Speaker 3: taken in the doctor's office. So anyone who has restricted
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  Speaker 3: access to care, or is living far away from specialty
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  Speaker 3: pharmacy or an infusion center will be more able to
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  Speaker 3: use a small molecule treatment, again, if that is appropriate
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  Speaker 3: for their health condition. A lot of the conditions where
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  Speaker 3: small molecules are the vast majority of the treatment, such
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  Speaker 3: as heart disease, transplant, even cancer, have much higher incidents
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  Speaker 3: in certain racial and ethnic groups, in particular black men
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  Speaker 3: and black women in certain cancers. Multiple myloma is an
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  Speaker 3: example we discuss in the paper where black men have
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  Speaker 3: a much higher rate of that disease and it is
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  Speaker 3: treated with both small and large molecule medicines in complement.
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  Speaker 3: So we really need to have again the access to
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  Speaker 3: the full spectrum of treatments to be able to both
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  Speaker 3: reach people where they are, including in the home setting,
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  Speaker 3: as well as to be able to treat some of
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  Speaker 3: the conditions that are most affecting all people, including people
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  Speaker 3: who are typically underserved by the healthcare system, such as
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  Speaker 3: people with low income and certain racial and ethnic minorities.
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  Speaker 3: In the US, we think about the example of antibiotic resistance.
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  Speaker 3: Being able to deliver small molecule antibiotics to the world
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  Speaker 3: as we see mutations of bacteria and infections is going
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  Speaker 3: to be essential to global health as well as health
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  Speaker 3: of the US, because obviously those infections eventually make it
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  Speaker 3: to us, even if they originate in another country. And
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  Speaker 3: there are already challenges to the economics of developing antibiotics.
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  Speaker 3: We don't want to put up additional hurdles with policy
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  Speaker 3: that is more punitive to small molecule medicines, such as
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  Speaker 3: the policy and the Inflation Reduction Act. When evaluating the
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  Speaker 3: potential for small molecules, whether considering the economics, the potential
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  Speaker 3: to reach patients around the world even if they are
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  Speaker 3: far from a healthcare setting, and their potential to improve
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  Speaker 3: human health. They are critical to health care delivery and
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  Speaker 3: should not be the target of punitive health care policy.
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  Speaker 2: Is Kirsen Axelson talking about the latest white paper coming
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  Speaker 2: out of Incubates Policy Lab, a deeper dive looking at
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  Speaker 2: the role small molecules play in our society. This is
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  Speaker 2: part of Incubates Week of Small Molecules ending today on
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  Speaker 2: September thirteenth, nine to thirteen, where we are calling attention
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  Speaker 2: to the important role that small molecules play in our society,
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  Speaker 2: and to Kirsten's point right there at the end, they
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  Speaker 2: have a role to play in equitable access to medicine,
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  Speaker 2: not just here in the United States, but around the world.
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  Speaker 4: We need to live in a world where.
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  Speaker 2: Small molecule innovation is happening, and that's exactly what some
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  Speaker 2: policymakers in Congress are trying to do right as we speak. So, Kirsen,
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  Speaker 2: thank you so much for the time. As always, it's
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  Speaker 2: great to have you on. We look forward to your
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  Speaker 2: next white paper and ongoing discussions here. Thanks as always
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  Speaker 2: to our listeners and audience for sending in ideas for shows.
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  Speaker 2: Thanks to our producer Grace Moster for help with this
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  Speaker 2: episode and other episodes. You can find more about Incubate
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  Speaker 2: at www dot Incubatecoalition dot org, including the white paper
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  Speaker 2: we've been discussing today, and as always, keep innovating.
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