Various Components Play Important Role in Companies’ Definition, Demonstration of Drugs’ Value

As prices for health care services, especially drugs, continue to be under the microscope of public scrutiny, life sciences companies are under pressure now more than ever to make sure that they can demonstrate the value of their products. But this may be easier said than done, as a patient’s perception of value may be quite different than, for example, that of a payer. But these conversations are starting to happen and need to continue to evolve and include all industry stakeholders to truly incorporate value into the equation, say life sciences experts.

According to Daniel C. Lane, Pharm.D., Ph.D., director of US Access Marketing-Customer & Portfolio Value Generation for Bristol Myers Squibb, “Value is something that’s very individualized.…If you think about value on a conceptual level, it’s a benefit being received over some type of value-assessment measure.…At the elemental level, that’s what we as a life science industry are really trying to understand — how do we communicate that?”

For a long time, the industry has focused on price, he told attendees at a recent webinar sponsored by Avalere Health, but showing a product’s value and what that means to various stakeholders is more important now than ever. For example, manufacturers may measure “value” in terms of additional life years gained, overall survival or progression free survival, he explained.

But when it comes to population health, that definition could be a bit different. “A lot of the things that we do as far as innovative treatments that provide value may shift the health of a population, and that has a significant value for some of our payers and other customers that we work with across the health care landscape.” For employers, value may be increased worker productivity and presenteeism.

What the industry is moving toward, he maintained, ultimately is showing value to one particular stakeholder: “trying to figure out how do we get all the stakeholders involved to understand how we can talk about value to the patient. A lot of those things are more patient-reported outcomes that we hear about — quality of life, thinking about it at the basic level, more time gained with their family, more quality experience, those types of things. So I think value is such a complicated area. [I think we have] the opportunity to really shape that discussion and work with our partners, and I think payers and some of these other groups recognize the importance of all these other stakeholders. I personally believe moving to a patient perspective is probably where we’re trying to get to from a value discussion.”

“Quality and value are intertwined, and everything is underscored by the need for great data,” asserted Sarah Alwardt, Ph.D., senior vice president and leader of Avalere’s healthcare transformation practice.

“There’s a growing body of evidence that suggests patient value isn’t a dimension by itself like part of the triple aim. It’s actually a connector to all of it, and understanding that patient value can improve outcomes and lower costs and benefit the whole ecosystem, and I think we can get behind that.”

For example, she pointed out, in situations where there is product competition within therapeutic classes, “patient value shared decision making might actually be a differentiating factor. And our challenge is really to work with patients to better understand, individually and collectively, what value means to them, which can be very nuanced — not just will I live longer but will I live better; will I be able to dance at my daughter’s wedding? This takes a much deeper understanding than we’ve traditionally taken.”

“What is most important to the patient [is] very, very nuanced, and this should really be considered across a product’s lifecycle,” she contended, including understanding perceived patient gaps in care during drug development. “I think we’re reaching that inflection point, understanding not only that patient centeredness in care, understanding the social determinants of health, diversity in clinical trials. We’re kind of pointing to that growing conversation around patient value.”

Moderator Elizabeth Carpenter, general manager and head of advisory services at Avalere, asked participants what the industry needs to do differently around the patient voice, and Lane maintained that this shift already has started to occur. “I think we’re seeing patients involved in clinical trial design,” and the FDA has emphasized it as well. “So I think that for us as the life science industry is really bringing them in earlier in that discussion and allowing them to help shape how we view discovery. But also more importantly, how do we think about how we bring drugs to market and their experience around receiving them and having those discussions, specifically around asking what’s their value? I think we’re starting to see some of that.”

Alwardt agreed, pointing out that “some of it’s coming from top down, so to speak.” Also critical to this shift is competition within clinical spaces, which is “such an amazing thing for patients.” Companies have shifted from the mindset that their drug is not facing competition, so patients have no choice but to take it, to the point of view of how they can distinguish their product from its competitors. “It’s the how do you differentiate, how do you really make sure that you’re developing something that not only treats the patient’s condition — I think we can agree that there’s a hierarchy of importance here — but it’s also meeting a patient’s need, filling a patient gap,” perhaps offering a new route of administration that a patient prefers. “There’s lots of things that kind of roll into that. And I think that that’s becoming more important, and it’s exciting that it is.”

Carpenter asked what the government’s role can be in driving forward the value-based conversation as it relates to drugs. In response, Milena Sullivan, managing director in Avalere’s policy practice, noted that policymakers long have been asking whether “the interventions we are paying for are priced relative to the benefit they bring to patients. But given the complexity of the health care system, I don’t think that they’ve ever been able to find the right formula of how to do that and how to think about that in a way that doesn’t hurt the very patients it’s supposed to benefit. What’s interesting now, though, is that we’re starting to see that evidence and value are considered by various government entities maybe more so than for a long time until very recently [when] we’ve had some precedent-setting actions,” such as the FDA granting accelerated approval to Biogen and Eisai’s Alzheimer’s disease treatment Aduhelm (aducanumab-avwa) and then CMS issuing a National Coverage Determination providing for Medicare coverage of the drug and other anti-amyloid monoclonal antibodies for Alzheimer’s only under certain circumstances.

“Through the coverage authority that Medicare is exercising, through the scrutiny of the accelerated approval pathway to where there’s a push to show better evidence to maybe incorporate real-world evidence into some of these decisions,” said Sullivan, “that in turn has implications for coverage and payment in ways that we haven’t seen before.”

Using real-world data, explained Alwardt, allows companies to “uncover a lot of things that we didn’t understand in a clinical trial. Our challenge — and I think that this is something that shouldn’t be taken very lightly — is we’re just getting to a point where we can feel somewhat good about using regular real-world data as part of a regulatory submission.” She clarified that “regular real-world data” is “like administrative data or data collected through an EMR [i.e., electronic medical record]. Those types of real-world data that are customarily used, we have some comfort with them, we’ve been using them for a couple of decades, even if we haven’t been necessarily using them with a regulatory intent.

“So now we add on a layer of complexity,” she continued. “Now we have patient-reported outcomes, where if we thought that regular real-world data were messy, patient-reported outcome data [are] super messy. So I think we’re going to need to spend some time understanding what data quality looks like, what sort of data measurement tools we need and how we validate all of this. But it’s doable, and I think that it’s going to take really cross-ecosystem partnerships — none of us are going to be able to push this rock up the hill ourselves — but as we work together, I think that we’ll be able to get to a better understanding of the utility of the data that we can collect.”

Lane stated that he was “excited to see a lot of these partnerships starting to occur. There are coalitions that are forming for us to work together with different real-world data providers so they can tackle some of these issues because…there’s no one single source of data; we’re getting it from multiple different places. The standardization of real-world data is not where the trial space is at this point, so we have to continue to evolve that, so I’m excited to see the progress in that.”

He acknowledged that many areas have seen a lot of work, but there are “some that are still lagging behind, but I think the key is those coalitions working together. For me, if I’m thinking about how we sort of leverage this, that’s the key. The reality is drugs are taking a while to get onto the market, and the reality is we want to know when they come to market how they’re going to behave, so it’s a topic that continually comes up” in discussions with his company’s various customers.

“Are there faster ways for us to leverage different real-world data sources or put any new systems in place? Maybe that will be clinical trial continuations; maybe we learn to link some of the clinical trial data to some of the real-world data sources, registries and those type of things,” said Lane. “That’s what we’re going to have to leverage if we’re going to meet some of the needs.…We’re going to have to find creative ways. Admittedly, traditional administrative claims are not going to meet that level of evidence.”

In a participant poll at the beginning of the panel, the Institute for Clinical and Economic Review (ICER) was cited by many attendees as one of the top entities that they look to on value.

“It’s interesting how much people are paying attention to ICER and ICER decisions,” asserted Sullivan. “ICER is certainly a very opportunistic organization and one that is continuing to try to assert its role or have a more central role in the U.S. health care system. But do I think that we’re closer to having a de facto HTA [i.e., health technology assessment] body in the U.S. today than we maybe were five years ago? Maybe only slightly.”

She pointed toward CMS’s decision on Aduhelm and the anti-amyloid monoclonal antibodies. “Some of the real impact of evidence and value could be exactly through where we saw…in the metrics that CMS was looking for in order to allow broader access to the” drugs “because the implications are significant on the budget, on the premiums, on a very large population. We haven’t seen as many coverage determinations for drugs over the years, but this was a perfect mix of budget impact and some gaps in the evidence, and thrown together, it showed that Medicare can exercise its muscles without needing an external body to determine value. And so then that means that the evidence-generation strategy has to evolve for companies going back to how they conduct their clinical trials and then the approval pathway that they choose because then this could have potentially more implications down the line for reimbursement and coverage.”

The life sciences industry, asserted Lane, needs to acknowledge price as a component of value, and “as we all know,…price is different to multiple different people. Having some rules about transparency when we’re putting some prices out there [is important], but the reality is that those prices, depending on the payer and the organization, are going to be very different.” It’s important to connect “that strong value story” about the components of a price and the benefits of that price to each stakeholder. “Price is a reality, and we all have to put that out there, but let’s understand what is that price not just for people but also what is that value and how do we show what you’re getting for that? And I think having that conscious conversation and us being more proactive about how we can have those discussions in a compliant way is probably the key for us in advancing the discussion.”

Sullivan noted that “one of the things we’re also trying to solve for as we work with clients is the question of equity because when you’re telling the value story, all the elements of value or the drivers of value, economic models account for them in different ways, and they don’t necessarily do it in a way that’s sensitive to the diversity of the population, particularly in a country like the United States.” That equity, she said, “is something that we think about in terms of quality of care, we think about it in terms of impact on patients, payers are thinking about it more, but certainly the government is now at a place where it’s thinking about it in a serious way, so I think value assessment and equity considerations have the potential of clashing in a very serious way, especially if all the other things are not in place with the other data. So we have to do a better job of thinking about the clinical value across different patient populations. And I think that’s something that hopefully the government will be aware of as well.”

“I think the most important thing is for us to continue to have the conversation” around value and all of its components, said Alwardt. “This isn’t a one-and-done conversation; we’re not going to solve everything. And cost isn’t going to go away, and it shouldn’t. I don’t think we’re saying that we need to…squeeze the balloon and just have it go ‘bloop’ out the other end. It’s how do we take all of these factors into consideration so we’re making balanced decisions with all of the data that we have available to us, and then we’re going to find the win-win-wins. And by ignoring part [of the components], it’s going to make a flat wheel; the wheel is flat on one side, and I think it’s going to take all of it to really bring it home.”

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