Dr Aimee Tharaldson Outlines the Pipeline, Role of Emerging Specialty Medications

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In the current pipeline of specialty medications seeking approval, a majority of development lies within orphan drugs and cancer medications, said Aimee Tharaldson, PharmD, of Express Scripts.


In the current pipeline of specialty medications seeking approval, a majority of development lies within orphan drugs and cancer medications, with other areas to monitor including therapies for Duchenne muscular dystrophy, hemophilia, and Alzheimer disease, said Aimee Tharaldson, PharmD, senior clinical pharmacist of Emerging Therapeutics at Express Scripts.


At this year's AMCP Nexus 2020 virtual meeting, you will feature in a discussion on specialty pharmaceuticals currently in development. Can you first outline what specialty medications have been recently approved and any notable impact that these approvals may have on managed care pharmacy?

Over the past 5 years, over two-thirds of drug approvals have been for specialty medications. Just this year alone, we've already had 28 novel specialty drugs approved. Of those, 16 are for cancer, and of those cancer drugs, 10 are oral, so they're going to be impacting the pharmacy benefit. We're actually even seeing some competition among some of these oral therapies for cancer.

We had Retevmo [selpercatinib] approved. This is an oral RET inhibitor for patients with RET-positive non–small-cell lung cancer (NSCLC), and also some patients with RET-positive thyroid cancer—that was approved in May. Then, Gavreto [pralsetinib], another RET inhibitor, was approved for NSCLC in September. It's going to get an expanded thyroid cancer indication in February, but again, these are very rare cancer types where there's similar mechanisms, other options available. Although, unfortunately, it's not bringing down the price of these drugs—they cost about $240,000 per year, those 2 medications.

We also see focus on really, again, niche cancer types. So, another example is Pemazyre [pemigatinib]. So, this is a new drug to treat patients with cholangiocarcinoma, or bile duct cancer. That only affects about 8000 patients per year in the US, and this is specifically for patients that have FGFR2 mutations, which is only a subset of those patients, about 9-14% had those mutations. So, this is a new drug available for those patients. It costs about $300,000 per year. There is a pharmacogenetic test available to identify the appropriate patients for therapy.

Also, there's a big focus on orphan medication development. So, of the 28 specialty drugs approved so far this year, 20 are orphan drugs. Just one I wanted to highlight is Evrysdi [risdiplam]. This is a new medication for patients with spinal muscular atrophy, and it's an oral medication. So, this could compete with Spinraza [nusinersen], which is an intrathecal medication. So, we could see some of the shift from the medical to the pharmacy benefit for these patients. There's also Zolgensma [onasemnogene abeparvovec-xioi] available that's a gene therapy, it's a one-time infusion that costs $2.1 million. So, that's another player in this space as well.

Can you speak on the current pipeline for specialty medication approvals?

So, the majority of spend or actually development for specialty pipeline is, again, orphan drugs and cancer medications. When you look at orphan drugs, some areas to watch for are Duchenne muscular dystrophy. We actually could see a gene therapy approved at the end of next year for Duchenne muscular dystrophy, and it could be used in all patients potentially. So, right now we just have those available for patients that have certain mutations. So, that's one to watch for.

We also have a lot of hemophilia. So, watch for that as well—probably 2022 will be the first time we see that approved, but those are going to be between $2-3 million for a one-time infusion—very expensive, but they also have the potential to minimize the need of those infused factors, which can be very expensive, as well.

Cancer is another area again of a lot of development that we've had. Potentially, we're going to have 21 cancer drugs approved just this year alone, and we have 2 more CAR [chimeric antigen receptor] T therapies that are pending approval at FDA. So, we have lisocabtagene, this is going to be approved next month for patients with large B-cell lymphoma, and then we're going to have idecabtagene for patients with multiple myeloma. So, this will be the first CAR T for patients with multiple myeloma.

Another area where there's a lot of activity is atopic dermatitis, and there are specialty drugs that are going to be approved for patients with moderate to severe atopic dermatitis or eczema. So, right now we have Dupixent [dupilumab] on the market. This is a biologic, it's a subcutaneous injection every 2 weeks and it really dominates that market, but now we're going to have several oral [Janus kinase] inhibitors approved in 2021 for moderate to severe atopic dermatitis.

Now, I think because of the safety concerns with this class, they may be used after Dupixent, but we could see abrositinib approved in April, Rinvoq [upadacitinib] could get an expanded indication in June, and then Olumiant [baricitinib] could also be approved next year. So, watch that space. There’s actually a novel drug in development for moderate to severe atopic dermatitis as well. It's called tralokinumab and this is an interleukin-13 inhibitor. So, it's a biologic drug, subcutaneous injection every 2 weeks, similar to Dupixent, to watch for as well.

Then another one we're watching is aducanumab. So, this could be approved for patients with Alzheimer disease (AD). And this would be the first disease-modifying drug for AD. So, we're really watching this closely. It's an [intravenous] infusion that's given every 4 weeks, and there was 2 pivotal trials, evaluating this medication. Those were previously stopped because it didn't look like they're going to reach the primary endpoints, but then they did another analysis of one of these trials, and they found that patients who were on it long-term at the high dose may have some benefit.

So, an advisory panel was actually going to be discussing this product November 6, so it'd be really interesting to hear what they have to say. Right now, analysts are all over the place whether they think it's going to be approved. I think estimates are around 30-50% chance of approval, but it'll be very interesting to hear what the ad board has to say if it's positive. This could be approved before its March 7 action date.

As specialty medications continue to be approved, what impact will this have on the health care industry? Managed care pharmacy?

According to Express Scripts’ 2019 Drug Trend Report data, 48% of spend under the pharmacy benefit is for specialty medications. We're expecting that by 2020—so this year, once we do the data at the end of the year—we're expecting that specialty drug spend is going to overtake traditional drugs spend under the pharmacy benefit, but it's important to realize that only 3% of patients take specialty medications; but of course, they're very expensive, costing over $3000 per month, on average.

So, the health care industry has to deal with this issue of paying for these very expensive medications. And so that's why it's important for the managed care industry to ensure that patients with these devastating conditions have access to medications that can help prolong or improve their lives significantly. We also have to make sure that we manage the spend on these medications as well so as not to overwhelm the health care system.

There are a lot of tools available. Obviously, the first most important responsibilities to the patients. So, you want to make sure you have highly trained pharmacists and nurses working through specialty pharmacies to make sure patients understand their medications. They can help monitor for side effects, they can minimize waste, and then there's also just managed spend tools available, like formulary management step therapies, prior authorizations. Then, there's new tools available for patients like now we have the embark program, and that helps to provide access to these expensive gene therapies as well. So, I just think continued innovation is going to be really important to maintaining this balance of access and affordability.