This interview was originally published as a podcast. The audio is no longer available, but you can read the transcript below. For updates on our newly released content, visit our Insight Subscription page.

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 today by Brian Leinwand, Associate Principal, and Taylor Schwartz, Consultant II, who both work within Health Economics and Advanced Analytics.

This is the third and final episode discussing Avalere’s insights on infusing the patient perspective into value assessment.

In the first 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.

In the second episode, we discussed the need for a solution to infusing value drivers outside of traditional clinical, economic, and humanistic endpoints into formal value assessments. We introduced multi-criteria decision analysis (MCDA) as a means to systematically incorporate other important elements of healthcare value.

Today, we’re going to discuss the patient perspective value framework developed by Avalere Health and FasterCures, which is a form of MCDA. It provides a case study of how certain value elements that resonate with individual patients can be integrated into a value assessment framework.

In May 2017, Avalere and FasterCures released the patient perspective value framework, or PPFV, which was designed as a value assessment methodology that incorporates patient preferences and can be applied to a variety of settings and used by multiple audiences. Taylor, could you give us an overview of its development and design?

Taylor: Sure. The PPVF was developed under the guidance of a multistakeholder steering committee that included patient groups, payers, life sciences companies, and a range of other nonprofit organizations. The framework includes a set of patient-centered domains, criteria, and measures, as well as a broad and non-condition-specific methodology for assessing value driven by patient preferences.

Based on feedback that we received from over 270 participants, 84% of which came directly from patients, caregivers, and patient advocates, the PPVF’s 5 domains represent categories of considerations that are important to patients when making healthcare decisions.

Ali: Great, thanks, Taylor. Now I want to turn to Brian. So, Taylor mentioned the domains that the PPVF uses as its foundation. Brian, could you describe them in greater detail and how they depart from other US value frameworks in addressing value when viewing healthcare through the patient lens?

Brian: Absolutely. The PPVF is comprised of 5 domains: Patient Preferences, Patient-centered Outcomes, Patient and Family Costs, Usability and Transparency, and Quality and Applicability of the Evidence.

Patient-centered Outcomes assesses the clinical, functional, and quality-of-life benefits and drawbacks of different healthcare options.

Patient and Family Costs assesses the medical and non-medical costs and other financial considerations and burdens associated with different healthcare options.

Quality and Applicability of Evidence assesses the strength and consistency of the evidence as well as the degree to which the evidence may apply to an individual patient. This domain functions as the lens through which we view the Patient-centered Outcomes and Patient and Family Costs domains. It weights the outcome and cost data by the methodological rigor of the evidence that informs those domains.

Patient Preferences are really the levers that power the PPVF and assess the patient’s personal goals and value set. This domain is used to weight or effectively rank the Patient-centered Outcomes, Patient and Family Cost considerations, and Quality and Applicability of Evidence. So, think of this domain as the engine that powers the framework. It’s what captures an individual patient’s preferences and converts their value set into a weighting scheme.

During the first session, I discussed an example where patients may exhibit different priorities when it comes to the care they desire. For instance, one patient may want to optimize survival, say to experience the birth of a child or attend a child’s wedding, all at the expense of toxic side effects or crippling out-of-pocket costs. However, another patient may prioritize the quality of their remaining life and aim to limit treatment toxicity, even if a particular treatment isn’t best in class at lengthening survival. And a third patient may seek a treatment that limits their out-of-pocket costs to the extent possible with the intention of leaving their family without insurmountable debt when they pass.

These scenarios really capture the need to have a flexible, malleable value framework that can conform to whomever the end user may be, understanding that the individual patient can have a nuanced view of what they desire out of their healthcare. The value is truly in the eye of the beholder, and the PPVF, in this respect, incorporates this into the framework through its Patient Preferences domain.

Ali: Thanks for that overview of the PPVF domains. They are really the high-level foundation of the framework. It’s also going to be helpful to examine what’s under that foundation set by the domains. So Taylor, a moment ago, you mentioned the formal criteria and measures captured by the PPVF. Could you now share some additional context around those components?

Taylor: In short, the PPVF criteria are more granular elements of value that are used to characterize a particular domain, and I’ll provide a little bit more detail on that in a moment. The PPVF measures are specific factors used to assess the criteria within each of those domains. At this point, the picture we’ve painted is a bit abstract, so I want to bring it to life with a tangible example of a couple of the domains, criteria that comprise the domains, and measures that are used to assess the criteria.

The Patient-centered Outcomes domain is comprised of 4 criteria. We have quality of life, complexity of regimen, efficacy and effectiveness, and safety. Most of these are captured in many value frameworks, namely quality of life, efficacy, and safety. So, in short, there’s really nothing groundbreaking here.

However, the criteria that stands out is complexity of regimen. This Patient-centric Outcome has 4 associated measures. We have the dosing schedule, treatment length, site of care, and the route of administration. Again, think through how the Patient Preferences domain can be employed through this criterion. Some patients may prioritize being treated at home rather than needing to travel to a facility setting. Others may have a fear of needles and prioritize an oral therapy rather than an injectable therapy. And other patients may prioritize the less complex medication schedule, say once a month rather than daily. These nuances are important to capture in a patient’s assessment of value, but it is not possible to capture them in many of the currently available quantitative value frameworks.

Next, I want to talk about the Patient and Family Costs domain, which is comprised of 3 criteria. We have medical out-of-pocket costs, non-medical costs to the patient and family, as well as future costs of care. There are 2 things that I’d like to point out that are not prevalent in other US value frameworks.

First, notice that these costs are from the patient perspective. Out-of-pocket costs are truly the responsibility of the patient and/or their family, not the financial impact to the payer or the cost borne to society.

Second are the non-medical costs, which are novel when it comes to a lot of the value frameworks that we see today. The measures housed under the non-medical cost criterion are the cost of travel, the cost of child or elder care, lost wages, and lifestyle or behavioral changes, such as healthy eating, along with a few others. Patients are at the center of healthcare decision making and many argue that characterizing value without considering a definition of value that captures the needs, goals, expectations, and financial tradeoffs of patients is suboptimal. For instance, a patient might value a therapy that enables them to avoid additional childcare or eldercare for someone in their family when going to get their treatment.

Ali: Thanks for that helpful context. It is rather clear that while patient outcomes and costs are traditional value domains in US value frameworks, the PPVF takes a unique approach to characterize these value domains. That said, I want to cover another domain, the Quality and Applicability of Evidence. Brian, could you tell us a bit about this domain?

Brian: Sure, happy to. This one is a bit complex, so bear with me for a moment. This domain helps the patient understand the level of confidence that a healthcare option will have the intended effects. So how is that determined? This PPVF domain is comprised of quality of evidence, consistency of evidence, and differences in treatment effects.

Quality of evidence refers to how methodologically robust a particular study is and follows the evidence hierarchy or pyramid and considers additional nuanced threats to the validity of study results.

Consistency of evidence captures variability of results across studies. So, think of a dartboard full of darts representing the results of similar studies. When making treatment decisions, any decision maker would prefer the darts centered around one point on the board rather than all over the board. This criterion aims to capture the degree to which there is significant variability in study results, which would erode the confidence of a decision maker.

Ali: I appreciate that detail on the Quality and Applicability of Evidence domain. Now I want to pivot to the content we discussed in our second session on MCDA. Before we conclude today’s session, Brian, could you briefly detail how the PPVF is linked to MCDA?

Brian: Absolutely. Personally, I think the tie is clear. During our second session, we described some common characteristics of different types of MCDA, and I’ll recap 2 of them here.

First, they formally and quantitatively incorporate different domains of value. And second, whomever utilizes the framework to make decisions values these domains to represent what resonates most with them, and effectively weights each domain to illustrate their preferences.

As we discussed today, the PPFV certainly checks these 2 boxes and serves as a model for employing a form of MCDA to capture treatment value from the viewpoint of individual patients.

Ali: Fantastic. Thanks, Brian. The PPVF, as the 2 of you have outlined, provides a more comprehensive view of value through the eyes of patients, developed with substantial input from patients, patient advocates, and other relevant stakeholders. Nontraditional elements of value are quantitatively considered in the PPVF, such as complexity of regimen and non-medical costs to patients. Patient Preferences, or the mechanism through which the PPVF is able to accommodate patients’ variation in value sets and treatment goals, is clearly a valuable addition to the scope of value frameworks.

Once again, it has been such a pleasure to speak with both of you as we outlined several US value frameworks and the degree to which they apply a patient-centric view of value. We positioned MCDA as an approach to formally infusing the patient perspective into a quantitative value framework, and today, this series culminated in describing how the PPVF employed a form of MCDA to develop an approach to capture value according to patients.

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.