Well-structured value assessments should allow stakeholders to compare different treatment options and arm them with information that enables them to choose the treatment that meets patient preferences and treatment goals. The challenge with current assessment methodologies is that they may be unable to account for vast patient differences and treatment preferences – even though value assessment is closely tied to how patients and payers interact and view a medicine.

At UCB, patients are at the center of all we do, and we have been proactive about identifying how to center the patient perspective in value assessment. This has culminated in UCB developing its own value assessment framework and releasing UCB’s Principles for Value Assessment in the U.S.

UCB has also partnered with other stakeholders, including the Innovation and Value Initiative (IVI), a non-profit organization whose work is vital to ensuring frameworks include elements that matter to patients. UCB was proud to partner with IVI during the development of its open-source value model in rheumatoid arthritis by providing comments and insights related to patient experience on its framework.

Below, UCB’s U.S. Head of Health Economics and Outcomes Research, Edward Lee, and IVI’s Executive Director, Jennifer Bright, talk about patient-centricity as a guiding force in value assessment and what we lose when we do not focus on the patient.

Note: The conversation has been edited and condensed for clarity. This conversation is part one of two. You can find part two here.

What can a well-structured value assessment process contribute to the health care system, patients and society?

Jennifer Bright: Value assessment has to start with the patient. It considers traditional elements of efficacy, cost, risks and benefits, but right now we are missing key components that are vital in understanding the patient experience. And in our view, value assessment needs to focus on who and what it is for, not what inputs we have.

Ultimately, value assessment should help us achieve better outcomes and use our resources better. This is more than just price - we need to account for families, caregivers, etc. and incorporate them into our calculation of value to ensure we’re getting the best value we possibly can.

Edward Lee: I couldn’t agree more. One of the big advantages to a well-structured process is consistency in how we mine and measure value elements, but also in how we capture different perspectives, from patients, to payers, etc.

When we have consistency, it allows findings to be more actionable because we have a common understanding of how value is defined, measured and derived, and informed by assumptions and limitations. That enables the decisionmaker to understand how to interpret the findings and identify what is best for the patient.

Why is it so critical that the patient voice is central to any value assessment methodology?

Bright: Patients are the individuals with the most experience about their condition, the impact on their health and functioning, their quality of life as well as the impact on their family, caregivers, friends. We also need to understand how health care interventions are helping or harming and how patients and their families decide on these trade-offs.

Unfortunately, we do a poor job of measuring these aspects because patients are very heterogeneous, and these issues are complex and individualized. Many value assessment methodologies skip over this because it’s not well-defined, but at IVI we’re in this up to our elbows. We work with clinicians, employers and payers with our models. We deliberately start by understanding the patient experience, and what patients mean when they say, “this has value to me.” This is painstaking work, but it’s critical.

Lee: At UCB, we understand that each patient’s experience can vary significantly. We look at factors from how their disease manifests, to how easy a treatment is to take or how efficacious it is, their prior experience with disease management, and how the disease impacts quality of life. We know we can’t include every factor that is important to every patient, but we have to do our due diligence to make sure we’ve captured the attributes that are the most important in how a patient considers whether an intervention was successful or not. Then we formally include that in our value assessment process.

Bright: I have an example from our model on rheumatoid arthritis (RA), which is a highly variable disease in terms of how different individual patients experience disease through their lifetime. What we learned by talking to patients is that the things that matter to patients are not what we measure, for example fatigue. Fatigue affects whether a person can work, care for their family, function. And we do a poor job of measuring it and incorporating that element into our conceptualization of value.

Lee: UCB provided feedback and comments to IVI on their RA open-source model, and the model allows patients to choose and make trade-offs based on the elements of value that matter to them. We need to allow for that in the value assessment process, even if it’s difficult. This is what’s going to be most meaningful for them.

What can we get wrong if we don’t include the patient voice?

Bright: Ultimately, we want to know the actual treatment patterns people are following and whether patients are getting the best outcomes. But we also need to know if we are measuring the outcomes that matter to the individual with a specific condition. We may be able to achieve specific treatment metrics, but what is the impact? Are they going to work, paying their bills, maintaining their relationships with family and social connections? This has implications for society, for employers, for the fabric of family life.

We are in a time of an emphasis on, “are we delivering the best?” That’s great, but we need to invest in asking better questions, developing better data, and using our best methods and smartest people to really think about value in a holistic way. Otherwise, we are going to hard code inequity, disparity in access, disparity in the types of treatment that are available to people, which will not lead to good health outcomes.

Lee: Each framework has its own unique set of strengths and weaknesses, but what’s clear is that we need to continue to give attention to, support and promote value frameworks that continue to put patients at the center and attribute value to organizations that bring those innovations to market.

Frameworks need to be transparent about how they capture and include patient-centric elements. Ultimately, stakeholders should utilize all the frameworks that are available, particularly if they’re for a certain population or disease state. This enables payers and providers to make more informed decisions about what value is to patients and what therapies they make available in terms of access in a transparent and systematic manner.

How can we build trust with communities with the goal of furthering equity and ensuring all patient populations feel like they have a seat at the table and that their voices are being heard?

Bright: You just heard Eddie talk about transparency, and at its core, it’s about being honest about what we can and cannot answer. We need to lead with “tell me what I need to understand,” not “here are 10 clinical assumptions that we see in the data.” It’s also about creating an honest dialogue about where data can support decision-making, where we still need to improve, and what we need to understand about patients’ journeys in terms of subgroups.

But there’s also a bottom-up approach: engaging with communities who have high degrees of prevalence of a disease but who are also underserved and distrustful of health systems at large. The fundamental ingredient is transparent conversation and one in which we are willing to acknowledge what we don’t have figured out yet. We need to convey an understanding that a PhD in health economics is on a level playing field with someone who has a PhD in living with rheumatoid arthritis.

These conversations aren’t technical, they are humanistic.

Lee: I think there are many times where we see the reports out of a community from a value assessment where they brought in a single patient to provide their perspective on how they manage the disease or how they're coping with it. I think it's important to bring in more than one patient. So, bring in patients that were recently diagnosed, patients that have been living with the disease, even patients that are in remission so that we can get a much broader perspective.

It’s also very important that we weigh the patient feedback as being just as important as the provider feedback. We need to know we’re choosing the right outcomes when we define efficacy, optimal goal response, or what’s going to lead to treatment discontinuation or not. Patients are committing to providing their life stories, so we must put those factors at the forefront of how we define value.

The last thing a patient wants to see is that they’ve provided this feedback and then it isn’t formally included because there wasn’t enough information. It wears down the patient.

Bright: Agreed. We can’t leave key questions on the cutting room floor because we don’t have data to answer them. We need turn back around and say, “how can we answer these questions?” That’s authentic. That not only builds the evidence base, but it also builds trust.