Putting Patients First® in the Value Equation: An Interview With the National Health Council’s Dr. Eleanor Perfetto

Eleanor M. Perfetto

National Pharmaceutical Council President Dan Leonard caught up this week with Eleanor Perfetto, PhD, MS, Senior Vice President of Strategic Initiatives at the National Health Council (NHC), the only organization that brings together all segments of the health community to provide a united voice for the more than 133 million people with chronic diseases and disabilities and their family caregivers. Dr. Perfetto has extensive experience in public policy and research. The NHC and its member patient advocacy organizations recently developed two NHC tools: the Patient-Centered Value Model Rubric, created to help patients and other stakeholders assess the patient centeredness of a value framework and supporting models, and the NHC’s Value Framework Get-Ready Checklist, which helps patient advocacy groups prepare and increase their capacity to engage with value framework developers.

Dan Leonard (DL):  As you know, there are several organizations that have developed value assessment frameworks and are using them to review a wide range of treatments. What’s your take on this latest health care trend?

Eleanor Perfetto (EP): Payers and biopharmaceutical companies have been constructing and debating economic models and methods for assessing the value of products for quite some time. Companies want to demonstrate the value of their product to gain access to formularies. Payers counter with their own cost-effectiveness analyses using data from their own populations to determine if the value argument holds up.

What really is new is that frameworks are being constructed by third parties and often do include some type of economic model as a component. The fact that these frameworks come from third parties gives the impression the approaches are more impartial than if they come from a pharma company or insurer. The important questions, from the National Health Council’s view, are two-fold: Do these models really depict value from the perspective of patients and their families, and were patients and their families engaged every step of the way?

DL: Tell me a little more about the NHC Rubric and the Get-Ready Checklist. What prompted NHC to develop them, and how are patients using them?

EP: Toward the end of last year, we were hearing concerns from patient groups that they did not know enough about the value frameworks. Frankly, the groups that had frameworks released in their disease area of interest felt blindsided. We held an education session on the topic at our annual Washington Representatives Retreat in December 2015 and got an overwhelming response from our members. They asked for help in myriad ways. Hearing that plea and out of brainstorming with them, our value initiative took shape and resulted in the creation of the rubric and the checklist, among other things.

DL: It appeared that early attempts at structuring value frameworks deprioritized the role and preferences of patients. Did you find that as well, and if so, why do you think that was the case?

EP: Yes, version 1.0 of the frameworks released in the last few years had two important limitations in this regard. They either only minimally engaged patients, if at all, and/or when they did engage patients, did not transparently describe the level and means of engagement so that it was known and could be assessed. Many of the advocacy organizations that felt blindsided by these efforts expressed a great deal of frustration in their attempts to engage with framework developers in order to share the perspective of the patients they represent.

In our view, if you do not include patients in the discussion from inception, if you don’t include the outcomes patients believe are important and care about, then you are probably not getting the question right. If the question is not framed correctly, then it doesn’t really matter how rigorous the rest of the methods are. 

DL: What do you see as next steps, not just for the Rubric but for the value assessment field as well?

EP: I see the framework developers refining their work in many ways and have had numerous discussions with them on patient engagement. Most are trying to do a better job of being patient centered. Several have admitted quite publically that they missed this the first time around.

I have seen and been a part of their recent efforts to get it right as they move forward with the next, updated versions of their frameworks. This will inevitably give us better tools in the future, if that commitment to patient centeredness and engagement is really there.

DL: You’ve been working with patient organizations that have been asked to participate in a value assessment. What have you learned about the process?

EP: That it is evolving! Many patient organization experiences with frameworks a year or so ago were quite bad. They felt ignored, insulted, patronized, etc. I am now hearing that the process is changing, and the organizations are beginning to have better, respectful, meaningful dialogue. Maybe not in all cases, but the tide seems to be changing. Even the ones now having good experiences are still a bit skeptical.  They are saying, “I feel like I am being listened to. But we have to wait and see if we’ve really been heard based upon what the final products look like. Will they really reflect what we’ve said?” We have started seeing instances where input that was received from patients has been used to frame the work of value model developers. Now we must wait to see whether this more appropriate framing will result in more patient-centered assessments.

DL: How would you recommend a patient or organization prepare for a value assessment in their disease/treatment area?

EP: The Get-Ready Checklist provides guidance to the patient organizations on how to prepare. We suggest that groups do their homework, understand who the developers are and what opportunities they have to engage, to look internally to identify the data they have to offer, to reach out and make introductions, and to keep their organizations and constituents informed.

DL: Thanks for sharing your thoughts on this topic. We’re looking forward to learning more from you during our September 29 conference, Assessing Value: Promise and Pitfalls.


Mark your calendars: NPC will bring together health care stakeholders on September 29 in Washington, DC, for a conference, “Assessing Value: Promise and Pitfalls,” to examine value assessment frameworks. To register to attend in-person or watch live online, visit NPC’s website.