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Infusing the Patient Perspective into Value Assessment, Part II

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Summary

Tune into another episode of Avalere Health Essential Voice in our Start Your Day with Avalere series. In this segment, we continue our series on infusing the patient perspective into value assessment with a discussion on the need for a solution to incorporating value drivers outside of traditional endpoints, and introduce a proposed approach to quantify those elements of healthcare value.
“Although still in the relatively early stages of development and utilization as far as analytic methods are concerned, MCDA has an inherent flexibility that allows for the assessment of healthcare from a number of different stakeholder perspectives, including that of the patient.” Brian Leinwand

Panelists

Moderator
Ali Silverstein , Consultant II, Health Economics & Advanced Analytics

Ali Silverstein collaborates with life sciences clients to develop research and analysis demonstrating client product and service value.

Speaker
Brian Leinwand , Associate Principal, Health Economics & Advanced Analytics

Brian Leinwand provides strategic health economics support throughout a product’s life-cycle and helps clients build evidence packages that demonstrate and support the value of their products.

Speaker
Taylor Schwartz , Consultant II, Health Economics & Advanced Analytics

Taylor Schwartz works collaboratively with clients to develop, design, and execute epidemiological and health economics and outcomes research studies in a variety of therapeutic areas, in addition to healthy policy analyses and medical innovation value assessment studies.

Transcription

Ali: Hello, and welcome to another episode of Avalere Health Essential Voice in our Start Your Day with Avalere series. My name is Ali Silverstein and I’m a Consultant II in the Health Economics and Advanced Analytics practice here at Avalere. I’m joined again today by Brian Leinwand, Associate Principal, and Taylor Schwartz, Consultant II, who both work within Health Economics and Advanced Analytics.

This is the second episode of 3 discussing Avalere’s insights on infusing the patient perspective into value assessment. In the prior session, we gave an overview of value assessments and discussed some of the limitations of current value assessment frameworks as they pertain to capturing a holistic view of healthcare value through the lens of the patient.

Brian defined value in 3 ways: humanistic, economic, and clinical, and described how different stakeholders in the healthcare system place varying levels of importance on each of these definitions.

Taylor described health technology assessments, or HTAS, and how those bodies use cost effectiveness to evaluate medical innovations for coverage decisions in developed countries around the world, while highlighting how in the US, the FDA determines the safety and efficacy of treatments, and coverage decisions are negotiated between the manufacturer and the payer.

Independent organizations such as ASCO, IVI, and ICER have created different value frameworks to assess new technologies, but they incorporate the patient perspective on value to varying degrees.

Taylor also discussed many of the novel elements of value as defined by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) using the benefits of a treatment for COVID-19 to illustrate these broader definitions of value that are salient to the patient.

Today, we’re going to dive a bit deeper and discuss the need for a solution to systematically incorporating value drivers outside of traditional economic, clinical, and humanistic endpoints into value assessments, and introduce an approach that has been proposed to quantify those important elements of healthcare value.

So, despite the emergence of formal value assessment frameworks over the past 5-6 years, it is difficult to achieve a consensus as to what constitutes value and what it should be primarily composed of.

Taylor, could you provide your take on some of the limitations of many prominent value frameworks, especially when viewed through the patient lens?

Taylor: Absolutely. So, when addressing the needs or perspective of patients, 2 fundamental limitations, or complicating factors, are the assessments’ intended audiences, as well as the perspectives from which they model clinical benefits, costs, and other attributes of treatments.

Frameworks’ intended audiences shape which elements of value are incorporated into the value assessment analysis. For instance, ICER’s analyses are oriented toward healthcare payers and as such, many elements of value outlined in the prior session, such as fear of contagion, insurance value, and option value, are not fundamentally incorporated into the qualitative analysis. These elements and others may be of utmost importance to patients. However, a patient perspective must fundamentally infuse elements of value that resonate with them.

Value is also in the eye of the beholder and what may be of particular importance to one patient may not be critical to another. For instance, as Brian outlined in the prior session, a patient with stage 4 terminal lung cancer may aim to live as long as possible and desire a treatment that can extend life to the greatest degree regardless of the outpatient costs or toxicity burden. We see this frequently with patients that may want to meet a landmark in their life, such as a child’s wedding, the birth of a grandchild, or something of that sort.

Another patient, however, may prefer to live their life free from intolerable toxicities, with survival and cost taking a backseat. Yet another patient may seek to prevent financial toxicity to their surviving family, indicating their preference set is aligned with limiting out-of-pocket costs.

Due to this varying preference set among patients, an ideal framework from the patient perspective needs to be malleable enough to weight aspects of treatment that resonate with a particular patient. Based on what we discussed in the prior session, a more expansive approach to value determination is needed that can account for both the traditional and non-traditional elements of value. This has been a challenge for framework developers to tackle as formal methods to quantitatively capture some of these elements are in their relative infancy. What is very clear is that value assessment frameworks are oriented toward different audiences and this drives the choice of value elements used. In this multiplicity of value elements, some resonate with particular stakeholders and some resonate with others.

Ali: Thanks, Taylor. It is important to understand how key the audiences of these are to the structure and the content of the framework. Now I want to turn to Brian. There have been some solutions proposed to tackle this value assessment challenge, but the one that appears to be most mature in the literature is multi-criteria decision analysis (MCDA). Could you describe this approach to decision making?

Brian: Sure, Ali, but let me take a step back for a moment. Taylor mentioned the multiplicity of value elements, and due to the complexity of making decisions around multi-dimensional problems, we sometimes utilize intuitive approaches to simplify the decision-making process. But because of that, we may underappreciate or even exclude critical value drivers which can lead to choices that do not align with a decision-making body’s or other stakeholder’s priorities. As a result, the decision-making process tends to be informed almost exclusively by evidence from health economic analyses with many patient- centric value drivers evaluated on a qualitative and ad hoc basis.

Therefore, when faced with multiple tradeoffs across a range of patient-centric values, decision makers oftentimes are not armed to make well-informed or holistic decisions and making decisions under this type of uncertainty may really lessen the credibility of those decisions.

So, as you mentioned, Ali, the use of MCDA has been proposed by a range of methodologists as an approach to solve the problem of a multiplicity of value elements. Although still in the relatively early stages of development and utilization as far as analytic methods are concerned, MCDA has an inherent flexibility that allows for the assessment of healthcare from a number of different stakeholder perspectives, including that of the patient. It can be used to drive coverage and reimbursement decision making, as well as physician patient shared treatment decision making. It can be used for quantifying benefits, risks, and uncertainties in order to aid the decision-making process by considering an explicit set of criteria and their relative importance to a particular stakeholder in a very transparent manner, while incorporating a wide range of stakeholder views to express some more societal or social perspective.

While there are different models or approaches to operationalizing MCDA, there are several common features. First, they formally and quantitatively incorporate multiple elements of value. Second, end users determine which value domains resonate most with them and assign weights to each domain to quantify their order of importance. Third, they combine each intervention’s performance across each weighted value domain into a comprehensive score that is used to rank interventions.

Ali: Brian, you mentioned that this method is in its relative infancy with respect to its use in value assessment. What are some of the roadblocks to its broad-based uptake in the near future?

Brian: Decision makers, whether they are patients, providers, policymakers, payers, or other stakeholders, must choose which attributes or domains of value to include in the decision-making calculus and specify weights to signify the relative importance of each attribute. In some cases, they must also score interventions on subjective attributes, such as improves equity or possible scientific breakthrough for which no specific measures exist.

Learning how to best accomplish these tasks in different settings, from individual patient choices, to payer coverage and reimbursement decision making, to systemwide investment decisions, stands as one of the most important barriers to widespread use of MCDA to guide healthcare decision making. At the moment, we don’t really have enough experience with these models in either individual patients or group organizational decisions to know how to maximize their usability and their subsequent use.

That said, the body of literature around MCDA and its associated models is growing as methodologists understand the importance in making decisions based on a full holistic version of what may constitute value.

Ali: Well, MCDA certainly appeals to our search for a solution to incorporate many elements of value tailored to a variety of audiences, and especially to elevate the patient perspective on value and capture individual preferences. To your previous point, we do need more experience with these methods before they become more common approaches to assessing the value of medical innovations and perhaps informing healthcare decision making.

In our final episode of this series, we’ll be covering a case study around a similar approach to MCDA that Avalere took to develop the patient perspective value framework.

It’s always a pleasure to speak with you, Brian and Taylor. Thank you for joining me today. And thank you all for tuning in to Avalere Health Essential Voice. Please stay tuned for more episodes in our Start Your Day with Avalere series.

If you would like to learn more, please visit us at our website www.avalere.com.

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