Patient perspectives play a crucial role in shaping healthcare decisions, and AMCP 2024 session speaker Sarah Emond discusses the importance of patient engagement in drug value assessments within the managed care framework, providing examples of successful collaboration between patient groups and managed care pharmacy.

Hi, I'm Sarah Emond. I'm the president and CEO of a group called the Institute for Clinical and Economic Review, a non-profit organization conducting what's known as health technology assessments so that we can try to get our health care system to move towards paying for value. 

My professional history has sort of been a health policy nerd my whole career without knowing it. I started out working in clinical research, then I did corporate communications for biopharmaceutical companies, which I really enjoyed, and then transitioned to the role at ICER back in 2009 when I finished graduate school, my graduate degree in public policy with a concentration in health policy. And I started as a chief operating officer with ICER in 2009 and then just took over as president and CEO this January. 

Can you explain the importance of collaboration between managed care pharmacy and patient groups in ensuring optimal patient outcomes? 

Yeah, this is such an interesting area that's been evolving over time. I think that generally the health care system is learning a lot by listening to patients and patient groups about the outcomes that matter to them, what they want from therapy, how they want to be able to access their medicines. And so I think we've seen an evolution in how managed care pharmacy has been thinking about this issue. And so we know that at ICER we've learned a lot from the work that we've done engaging with patients and our comparative effectiveness reviews and when we started doing our drug reviews 14, 15 years ago I will admit that we weren't great at doing patient engagement and we've learned a lot about how to do it better and now have an entire chapter of our report dedicated to the patient experience. We partner with the patient organizations during our review to understand outcomes that matter most to them. And we have a final report that reflects policy recommendations that we hope are patient-centered, but also balancing the needs of the ultimate decision-makers in our health care system by thinking about population health as well. So that's a known tension when we think about patient-centered and population health, but we think ICERs work can help bridge that gap. 

And I think it's important for managed care pharmacies to start thinking about their patient engagement strategy. Do they want to include a step in their P&T process where they talk to the major patient organizations for a particular disease? Do they want to include patient representatives in their P&T committees? Do they want to rely on independent research like ICER to help inform the patient's perspective? I think all of these options exist, but I think it's important that we are all thinking about how the decisions we're making from a formulary decision perspective, from a coverage policy perspective, are trying to do their best to reflect the outcomes that matter most to patients. 

What specific efforts are needed to successfully engage patients in drug value assessments within the managed care framework? Can you provide examples of successful patient engagement? 

I think that the first place is to start from a place of humility. When we take on a value assessment at ICER, we are not experts in the disease. And so we have to respect that the patients, the patient advocacy organizations, their families have the lived experience to be experts in the disease. So we actually start our reviews by talking to the patients. Before we even announce the review publicly, we want to hear directly from them about what they care about when it comes to potential treatment outcomes, what the experience has been like so far with the existing treatment paradigms, and so we start by talking with them. The patients are then able to participate throughout the entire review, whether they're commenting on the model that we developed, that's the economic model that'll help us know the comparative cost effectiveness of these therapies, all the way to the public meeting where they sit front and center. and are served as experts for our independent panel that's deliberating on the evidence that we produce, all the way to providing comments on the draft report as well.

And so I think that the framework can be different for different organizations. This has served us well at ICER because our process is meant to be very public and have lots of points for public input and so make it that patients and the patient groups know how to participate in those different parts of the process has been really important to us. When you think about how it gets applied to managed care pharmacy decisions, it can take a different format, it can, those organizations again can rely on work already produced that's summarizing the patient experience and hearing from patients about the outcomes they care about, but it can also include talking to patients directly. 

So a few examples of what we've seen in the ecosystem. So we had a review a few years ago that we did on sickle cell disease. Not our most recent review on the gene therapies, this was a review of some of the chronic therapies. And one thing that was really interesting that we learned by talking to patients had nothing to do with the therapies that we were looking at. It had to do with the challenges they faced when they had a vaso-occlusive crisis and ended up in the emergency room. And so many patients we talked to told us about waiting and changing into professional-looking clothes before they went to the emergency room in the hopes that they would be taken more seriously by the doctors and not to be assumed to be drug-seeking and that they would hopefully get access to the medicine that they needed. We know that one regional payer, upon learning this information through the ICER report, actually changed their prior authorization guidance for people with sickle cell disease to make sure there were no barriers from their end to accessing pain medicine.

And while that had nothing to do with the review that we were doing of two new therapies for sickle cell disease, it's an example of how listening to patients and hearing their experience can end up actually changing and improving the payer coverage decision on the other end. 

We have another example that we haven't quite yet seen the payer experience change, but maybe hoping for this a little bit, when we reviewed a new therapy for pulmonary arterial hypertension. We actually heard from the patient groups how difficult it was for a lot of their community to access the right type of bottled oxygen. And that was something we had no idea was going to be on our radar as we did this review of a new therapy for the condition. And we developed policy recommendations calling on the payer community to streamline access for bottled oxygen for people with this condition. And so those are the types of things that can happen when you engage with patient groups and hear about their experience. We can identify ways as a community that we can do better in terms of access. 

Well I think what's so informative is how much our system has been focused on doing things the way we've always done them and there's a real reckoning that we all have to have now. Whether it's thinking about social determinants of health and health equity and institutionalized racism or all of these things that really just sort of been built into the system. When you take a step back and you actually ask patients and their families what they care about, you might be surprised by the answer. And so it's been such an education for us as an organization and an evolution. We're continuing to learn the best practices and how to do this even better than we do now. But I think there's opportunities for the managed care community to really evolve how it thinks about patient input in some of these decisions. 

How do patient perspectives play a role in shaping health care decisions and how can they be effectively incorporated into the decision-making process? 

That's a big question and I think that it depends on what we're talking about. If we're talking about the decision-making between a patient and their clinician, there's a lot of excellent work happening about shared decision-making and the work of the Patient-Centered Outcomes Research Institute that's trying to develop information that will help support those types of decisions.

If we're thinking about more population health level decisions, if we're thinking about a coverage policy, if we're thinking about a guideline decision, I think that the ways to incorporate patient preferences are a little bit different than when you're thinking about shared decision making. 

So a few ideas here. One is that you do need a base of evidence to try to inform what you're doing with your coverage policy or your clinical guideline. And so often, we are relying on the clinical trial program for this because we might be making decisions about a new drug that just got FDA approval. So we're not going to have all the evidence that we wish we had. We don't have real-world data yet, we don't have maybe registry data or other sources of information that could inform some of our decisions. So we're going to rely on the clinical trial program.

So the clinical trial program might feel a little less than ideal for answering some of the questions about patient preferences and patient-centered outcomes. And so what I think that commands of us is to think about design and coverage policies that have the best chance of incorporating what patients want out of the treatment in terms of access. And so this means things like flexibility or a robust appeals process. So you might write a coverage policy based on the best evidence in the FDA label, but it might turn out that there are a subset of patients, or some patients, who have a particular comorbidity or a different manifestation of the disease who might fall outside the label. And it's kind of not their fault that they weren't included in the clinical trial so I think it's incumbent upon us to make sure that there is a robust appeals process an exceptions process that's easy to navigate in case that's the right therapy for that patient. 

I think that the other ways we can think about incorporating patient preferences is to think about our coverage policy decisions as a snapshot in time. That's how we think about our comparative effectiveness reviews we're looking at the evidence in front of us right at that minute. That evidence is going to change and evolve and so making sure that there's the ability and the structure for updating policies when new information is available and even hearing from patients and patient groups about ways the coverage policy might need to evolve to reflect the latest evidence. 

How can health technology assessments be leveraged to enhance patient engagement and improve overall health care decision-making processes? 

This is such an interesting question because health technology assessments by definition take a societal or health system perspective, which means we're talking about averages and we're talking about what's happening for a population. So they're not always that helpful for a shared decision-making conversation between a patient and a doctor, and that's okay. What they can do, though, is highlight areas where we either have gotten it right with the evidence. So if we hear from a patient community that the outcomes that matter most to them are reflected in the clinical trial data or the data set that we're reviewing in our health technology assessment, I think that's something we can celebrate and we can be excited and say this is great and maybe we need some more evidence, but at least we know we're headed in the right direction.
More often than not, a health technology assessment can help us identify areas where we can do better. So if the clinical trial data are missing the outcomes that matter most patients, or we know that a registry or some real-world evidence would really enhance our understanding of the right patient population to be using a therapy, that's a commitment we can all make to developing that evidence even after a health technology assessment.

I think that one of the things that I have learned by doing patient engagement at ICER is that a sense of humility when approaching the question of how do we make our system more patient-centered and still balance things like access and affordability, is a really helpful starting point. Because we thought we knew what we were doing and that we had it all figured out and we knew how to do patient engagement and boy, did we have a lot to learn. 

So that place of humility, I think, has led us to where we are now. We're really proud of our patient engagement program, and of course it can continue to evolve. But now we're seen as an example that some managed care organizations look to, that other health technology assessment groups look to internationally, and so I would recommend for anyone in a managed care organization who's thinking about trying to tackle this very complicated issue, it's okay to not have it right when you get started and a place of humility is probably a good place to start.