2025 Drugs to Watch: Market Access, Innovation, and the Future of Reimbursement

In this interview, guest experts John Stahl and Roy Moore explore the evolving payer landscape in 2025, discussing the growing role of value-based agreements, real-world evidence, and innovative reimbursement models in supporting the adoption of precision medicine, GLP-1 therapies, radiopharmaceuticals, and emerging digital health technologies.

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John Stahl: My name is John Stahl. I'm a partner with our commercial strategy consulting team here at Clarivate. I have over 20 years of experience in the health care industry and life sciences space. Here at Clarivate, I lead our US pricing and market access team.

Roy Moore: Thanks for having me. Hi everyone, my name is Roy Moore. I have nearly two decades of experience in the US health care and global market access space. I currently work on a team of analysts who put together syndicated reports looking at the market access landscape for certain diseases and emerging therapies. It's nice to be with you all.

Given the increasing focus on precision medicine, how should payers approach the reimbursement of therapies targeting smaller, genetically defined patient populations, where real-world evidence might be limited at launch?

Stahl: I'll go first and then would love to hear your perspective. I think this is a real opportunity that we should think about as we continue to have these amazing innovations coming to market. Drug research and development is moving to this world of more targeted therapies and genetically defined patient populations.

With that often comes some questions about the type of evidence we need to support these therapies. How soon or how quickly can that evidence be developed? How do we ultimately pay for these new innovations? Maybe looking at a couple of those angles here, from a payer perspective and as we think about that reimbursement angle, we're seeing more and more adoption of value-based contracts or value-based payment models, value-based agreements (VBAs).

Here in the US, historically, they have been nicer to have as opposed to need to have. But this is something that we are seeing many payers and manufacturers engaging in more and more, where you're looking at these types of payment models, whether they're outcomes-based models or more innovative ones that are looking at pay-over-time or subscription-type models, to align that reimbursement with the drug’s or the solution’s real-world performance. So, I think getting more creative—and both manufacturers and payers getting more involved with value-based agreements—is certainly going to be crucial for further adoption of precision medicine.

Secondly, it comes down to the evidence. There's a lot of evidence that is mandated or necessary for drug approval, but payers often want and need more. How do the drugs continue to perform in the real world? Again, partnerships between manufacturers and payers to generate that real-world evidence will be key to continued adoption of these precision therapies. That can be through patient registries or other post-marketing studies, but that will help to support those payer decisions around reimbursement.

One last theme is collaborative engagement between payers and manufacturers throughout the drug development life cycle early on around the evidence that is needed or wanted. Then, post-launch, continuing to engage manufacturers and payers, as well as engaging with patient advocacy groups, to encourage continued evidence generation and access to these new innovations.

Moore: I would just echo what John said. He said it perfectly. One key thing I would hit on is, again, the evidence generation. Clinical trials are expensive. You can't cover all populations you want for a drug, even if you have the data down to the particular level.

You need to consider evidence generation throughout the entire life cycle of the drug, even after launch. The generation continues after the drug is approved because you may want to expand populations or better understand how effective the drug is. We've actually seen this with some drugs where they will make agreements with payers and continue to generate evidence. We saw this in the cardiology space—what happened is we saw that the reimbursement status of those drugs actually improved when they had the evidence. I would say that manufacturers should always look at evidence generation as a process to be developed throughout the entire life cycle of the drug, not just in the phase 3 trials.

With next-generation GLP-1 therapies transforming the obesity market by demonstrating substantial efficacy in weight reduction and related metabolic benefits, how do you foresee payers addressing the high demand and associated costs of these treatments? What frameworks might emerge to assess long-term value in obesity management?

Moore: I'll go ahead and take the lead on this, and John can chime in. First of all, the GLP-1s are a remarkable technology. They're probably one of the most exciting areas for development right now in the health care space. We already see that they have shown significant change in addressing weight management and weight reduction, as well as cardiometabolic benefits such as reduced risk of stroke, lower blood pressure, and other ancillary benefits.

The challenge of these drugs is that although they are very effective, they come with a price tag, and the sheer number of patients that would qualify or can benefit from the drug is so substantial that it creates a major budget impact for payers. That's why, historically, we have seen somewhat limited uptake of these drugs in the commercial space. A lot of commercial insurers are not paying for them—not because they're not effective, but because they're worried about the long-term budget impact. We have seen that in Medicare, of course, as well. We are seeing that evolve because, obviously, these drugs do have a positive benefit. Particularly on the part of employers, they're looking to cover some of these drugs for their workers because they know that there will be downstream benefits.

When it comes to measuring the value framework and long-term benefit of these drugs, I think we'll stick with what we've always had. This is a heavily rebated space. These drugs were used for diabetes, and we know those drugs are heavily contracted with significant rebates. That will continue. However, looking at the long-term benefit or long-term value—particularly for employers whose employees will stay there for years—you could see the rebates tied to long-term efficacy benefits, such as reduced hospitalizations and reduced risk of strokes. You could see frameworks tied to the downstream impact on medical benefits and medical costs, as well as overall cost of care.

Stahl: Those are great thoughts, Roy. In terms of maybe other innovative approaches that are out there, Roy mentioned the use again of these value-based arrangements. But potentially, we may start to see specific patient segmentations that are used, managing the riskiest patient populations, such as patients with obesity plus other comorbid conditions. That's certainly something that payers are exploring in order to provide access appropriately for patients.

As we look at how GLP-1s are used in the obesity space, perhaps there are evolutions that come along where GLP-1 medications may be used for initial weight loss, and then other therapies may be used for longer-term maintenance therapy once we see the initial weight loss from the GLP-1.

There's a lot to consider here. Roy highlights the tremendous innovation of GLP-1s. At the end of the day, how do payers ensure that they can manage that long-term budget through appropriate access for patients?

Your report highlights the transformative potential of radiopharmaceutical theranostics. How can payers evaluate the cost-benefit ratio of these therapies, particularly in balancing upfront treatment costs with potential reductions in disease progression and hospitalizations?

Stahl: Continuing on this theme of tremendous innovation over the last couple of years, radiopharmaceutical theranostics are a great advance, especially in the world of oncology. They offer the potential for being very targeted in their approach to cancer treatment, minimizing systemic side effects and damage to surrounding healthy tissues. They're able to be used in cases where other drugs have not worked and where you might have resistance that has crept in, such as in metastatic castration-resistant prostate cancer.

But as you highlight, they do come with challenges. They come with higher costs upfront, some complex logistics that are involved for their delivery, and payers need to balance both the pros—that targeted, very effective use in resistant populations—with some of the cons in terms of the higher upfront costs.

I think it comes down to developing these frameworks that allow you to follow patients over time. At the end of the day, if we are focused on moving away from fee-for-service to fee-for-value and population-based health care, we need to take a longer view to the benefits of these medications. So, being able to track the decrease in future medical costs that might be a result of using the radiopharmaceutical theranostics, being able to manage, track, and quantify the reduction in side effects, and build that into to the value frameworks. Certainly, from a patient-reported outcomes perspective, being able to look at patient quality of life on these new innovations and being able to build them into to the value frameworks, that will help with further adoption.

It's easy to look at the pills that were developed in metastatic castration-resistant prostate cancer as an example and just look at the pharmacy costs. But if we are going to be moving to, again, more of this fee-for-value and population health focus here in the US, taking that longer-term view, looking at those cost offsets, looking at reduced need for additional treatments, and building in that patient angle from a quality-of-life perspective is certainly going to be crucial to further adoption here.

Moore: To piggyback on what John said, what he said was perfect. When it comes to balancing the upfront treatment costs, we have to keep in mind that these drugs are significantly more expensive. We saw in the report at Pluvicto, it's more than double the price of its comparators. When it comes to balancing that, I could see payers managing the prior authorization restrictions, maybe having them tied upfront so they can target the patients who will be most likely to benefit from the therapy. And then when they have more evidence, usually third generation, they can expand that out to actually loosen restrictions so you can get a wider population. I think that's how they would probably look to balance the cost with the benefit.

Drugs like Fitusiran and SEL-212 showcase the use of cutting-edge technologies, such as RNA-based therapies and immune tolerance platforms, in chronic conditions. How can payers assess the real-world applicability and sustainability of such highly specialized treatments?

Stahl: There are a lot of advances that we're seeing from SEL-212. Payers will continue to demand that real-world evidence. As we think about what that looks like, [it looks like] being able to track patients over time, have better data collection processes or data collection protocols, managing patients—perhaps through patient registries—and identifying the metrics that will allow us to appropriately identify the efficacy and safety benefits that these new innovations are bringing.

If we're talking about Fitusiran in hemophilia, [we want to be] able to track reduced bleeding events, reduced hospitalizations, and reduced need for additional factor administration in acute settings. That requires payers to take, again, that holistic view—not just that upfront pharmacy spend, but being able to follow their patients over time, evaluate the cost offsets and cost savings that will be arriving as a result of reduced medical expenditures, and track those long-term patient outcomes through electronic health record (EHR) integration and things of that sort. That's ultimately how we move to this fee-for-value world that we are needing to move the US to.

Moore: Well said, as always, John. I guess the only thing I would add is that with  Fitusiran, hemophilia is a heavy Medicaid population—around 30%. So, you could see some innovation there. Obviously, we're seeing the cell and gene access model, and we could see something similar for diseases that heavily targets the Medicaid population, with some innovative models there.

There is a growing emphasis on patient-reported outcomes in clinical trials. How can payers effectively integrate this patient-centric data into their coverage determinations to ensure therapies meet patient needs and provide value?

Moore: Patient-reported outcomes are quite important, generally speaking, because they speak to how well the patient feels, how they are responding to treatment, and how their disease is progressing. However, it is a challenge for payers because they are going to focus overwhelmingly on the efficacy, safety, and tolerability.

Patient reported outcomes may give a broader view of the patient. The payers themselves will focus on the patient-reported outcomes. That's not to say that you can't include patient-reported outcomes as part of your value messaging, because some actually will be more receptive toward the message that the patient does feel better.

So, that can go to your value message, but, again, they're going to focus mostly on the efficacy, safety, and tolerability data. But if you can have some patient-reported outcome measures that are focused on that—such as functional state, decline in activity, or something tied to that—they would be receptive to that.

Stahl: Just to add, I'm a big proponent of this fee-for-value and the Triple Aim, and part of that Triple Aim is patient outcomes. How can we improve patient quality of life? We see this increasingly being adopted outside of the US. It's part of many health technology assessments, and we're starting to see US payers, led by Centers for Medicare & Medicaid Services (CMS), as well as some of the big national and regional payers, starting to ask for these types of data around patient-reported outcomes, voice of the patient being used during clinical trial development, and then certainly post-launch being used as part of these value assessment frameworks.

We are heading in the right direction. As we think about it, it's this theme of manufacturers working alongside payers and patient advocacy groups to ensure that patient voice is represented across that clinical trial continuum. And then identifying those metrics that, as Roy pointed out, are really able to show the benefits from use of that medication, whatever one is being studied.

The integration of artificial intelligence (AI) and digital health technologies is transforming drug development and patient monitoring. How can early engagement between payers and developers of these technologies optimize coverage policies and support value-based care models?

Moore: Obviously, there is always going to be innovation in drug development. Payers don't like to be surprised when these sorts of things come out. That's why they do horizon scanning activities, the same as payers all over the world. When there is a technology that is tied to a drug, whether it's AI or digital health, that also needs to be communicated early to the payers so they're up to speed and understand the science behind it.

If you just hand them data and say, "This is what this does," at the time the drug's approved, they're not going to understand it, and it's going to delay actual adoption and reimbursement of the drug. You need to have those conversations early, and you need to get feedback from them because it might actually feed into how you design your trials—thankfully phase 3.

What are the key takeaways you hope the audience gains from this interview?

Stahl: I'll tackle a few. I think there are three crucial things that, as we're seeing tremendous amounts of innovation here in life sciences and health care, we need to remember. One is early engagement. As you've heard Roy and I say on many of the questions today, early engagement between manufacturers and payers [is essential]. Bringing multiple stakeholders into the discussion—the patient advocacy groups, providers, other individuals, and bodies that are important to drug access and drug reimbursement—making sure that those discussions happen early in the development life cycle. Making sure that payers are aware of these new innovations, the value and the benefits that they bring, how they compare to current therapies, and allowing that discussion and that collaboration to influence the clinical trial developments and the evidence generation strategies.

Secondly, there really needs to be a focus on appropriate reimbursement frameworks. Again, going back to this theme of value-based care and looking to more deeply embed these value-based agreements into the drug reimbursement ecosystem. We're seeing more and more adoption of it. We're seeing more and more acceptance of it. Getting to models that are two-sided risk, where both parties, whether it's a payer and a manufacturer or a manufacturer and an employer, have incentives and are both looking for those win-win solutions. We need to see more experimentation there. We need to see more adoption there, but that's how we are going to be able to provide that appropriate reimbursement for these innovative solutions.

Lastly, continuous solution evidence generation. Roy has mentioned it. I've mentioned it. It doesn't stop once you launch. How do we continue to develop that real-world evidence to support the continued access and reimbursement for the approved indications while making sure that we understand how these drugs might be working within specific populations, expanding populations appropriately based upon that evidence? Continuing to generate that real-world evidence is going to be key.

Moore: Yes, again, I would echo what John said. One thing that I would also take away is that we're in a golden era as far as technologies for development. We have GLP-1s, cell and gene therapies, radio theranostics, and mRNA technology as well. But with the US payer system, it's so fragmented between Medicare, Medicaid, commercial, and then within those you have the different payers themselves.

It is going to be a challenge, but it is also a time for innovation. One thing that I did want to mention, of course, is that in some cases, the government's actually leading some of the innovation as far as reimbursement. We saw the creation of the cell and gene therapy access model. We didn't really go into much detail on it in this call, but they announced the first two drugs for it just last December. Those are the ones for sickle cell disease. In this model, essentially the states defer negotiations to CMS, which negotiates long-term reimbursement, supplementary rebates that could take years, maybe a decade in some instances.

I would just say, look for innovation from these agreements between the manufacturers and the payers themselves, because if they want to measure a benefit, you need to have a longer time horizon. That's going to take creativity, probably greater creativity than we've traditionally seen from the US insurance market.

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