ISPOR News

Kelly Lenahan, MPH, ISPOR, Lawrenceville, NJ, USA and Brian O’Rourke, PharmD, ISPOR President-Elect, and Independent Healthcare Advisor, Ottawa, ON, Canada

There is a growing emphasis for payers and health technology assessment (HTA) bodies to incorporate “novel” elements of value when undertaking an assessment of pharmaceuticals and other health technologies. Recognizing the need for education and collaboration, ISPOR introduced a strategy to increase engagement with payers in 2019. Since then, ISPOR has leveraged its unique position and world-renowned reputation to promote the use of health economics and outcomes research that informs healthcare policies and decisions. For example, an ISPOR Speaks column¹ published in 2020 in Value & Outcomes Spotlight highlighted ISPOR’s initial efforts to engage payers. In 2020, ISPOR launched its first annual Payer Summit to allow for a more intimate dialogue between payers and industry stakeholders^2,3 and that tradition continues today.

In fact, examining the novel elements of value in assessing health technologies was the focus of the 2022 ISPOR Payer Summit, which was held virtually on September 15, 2022. Attendees of this summit represented multistakeholder perspectives from European and North American payer organizations, HTA bodies, patient groups, and industry stakeholders from the ISPOR Institutional Council.

"ISPOR has leveraged its unique position and world-renowned reputation to promote the use of health economics and outcomes research that informs healthcare policies and decisions."

 

The 2022 summit presented 3 different perspectives on expanding the concept of value for high-cost innovations: (1) alternative concepts of value (presented by Peter Neumann, ScD, Tufts Medical Center, Boston, MA, USA); (2) what does value mean to a patient (presented by Alan Balch, PhD, Patient Advocate Foundation and National Patient Advocate Foundation, Washington, DC, USA); and (3) what does value mean to a payer (presented by Iga Lipska, MD, PhD, MPH, Medical University of Gdańsk, Gdańsk, Poland).

After the panel discussion, participants were divided into 3 groups to discuss different topics: (1) avoided versus avoidable costs; (2) incorporating patient-centered research and endpoints into value assessment; and (3) how to incorporate broader societal value into clinical and economic assessments. Participants were equally divided based on their stakeholder relationship, geography, and gender.

The group that focused on avoided versus avoidable costs discussed how different countries such as Germany, Belgium, and the United States assess a product and whether they include costs when determining the value of a product. A part of the discussion also focused on how to appropriately measure health system costs. Ultimately, all parties involved in the breakout group agreed that they are trying to minimize uncertainty and maximize value. The main action point that arose from the discussion is the need to better differentiate avoidable costs from costs that are avoided, perhaps by learning how other sectors, such as manufacturing, have defined and measured these costs.

The second group focused on how to better incorporate patient-centered research and patient-important outcomes into payer-based value assessments. A case study involving patients with multiple sclerosis was presented by a representative from a European HTA body. They determined that patients want disease-modifying therapies throughout their life and do not want to stop therapy as a milestone for disability status; however, when patient preference information was input into their cost-effectiveness analysis, the model showed that patients should be taking a lower-priced disease-modifying therapy based on their disability status. They also found that when community preference data were input into the cost-effectiveness model for multiple sclerosis, the model prefers the community preferences instead of individual patient preferences. A payer from the United States discussed how they conducted a Delphi panel of patients with multiple sclerosis and found that the patients were much more concerned about slowing disability and keeping activities of daily living instead of the predicted outcomes of wanting to avoid adverse effects of medications. These examples demonstrate the importance of incorporating patient preferences into payer decision making. Some payers in the United States that are very interested in using Patient-Reported Outcomes Measurement Information System (PROMIS) data, especially with their outcomes-based contracts; international payers were not aware of PROMIS data. Low- and middle-income countries in Europe have found that it is difficult to explain the value of a product to patients, especially when describing the budget impact. They have found that patients care much more about ease of treatments instead of costs. Ultimately, all stakeholders in group 2 agreed that they should be engaging patients earlier in the decision-making process.

"HTA is much more than an economic assessment of the value of a technology—clinical assessments are just as important to the HTA process and while the ISPOR Value Flower is a good starting point, many feel that it focuses too much on the economic assessments of value and not clinical outcomes."

The final breakout group focused on how to incorporate broader societal values into clinical and economic assessments. An emphasis was made on remembering that HTA is much more than an economic assessment of the value of a technology—clinical assessments are just as important to the HTA process and while the ISPOR Value Flower is a good starting point, many feel that it focuses too much on the economic assessments of value and not clinical outcomes. All payers and HTA bodies in this group were unsure if the novel elements of value defined by the Value Flower could even fully be included into the assessment process.

Some important influences on payers in the United States when making a decision include input and policies from employer groups, patient and caregiver engagement, and reports from the Institute for Clinical and Economic Review (ICER), especially their cost-utility analyses. It was questioned if patients and caregivers should be considered as 2 distinct stakeholder groups, since the caregiver perspective might be overlooked in areas where the caregiver burden is very important, such as for Alzheimer’s disease.

The next ISPOR Payer Summit will take place on May 7, 2023 at ISPOR 2023 in Boston, MA, USA. For more information on the ISPOR Payer Summit or if you would like to inquire about future participation in 2024, please contact HTACouncil@ispor.org.

References

1. Naaman N. Dialogue with decision makers: collaborating with payers to advance health economics and outcomes research. Value & Outcomes Spotlight. 2020. 6(2):7.

2. O’Rourke B, Cabra HA, Orsini LS, Guerino J. Quality, access, and the use of real-world data by payers: where are we now? where do we want to be in the future? Value & Outcomes Spotlight. 2021. 7(2):11-13.

3. O’Rourke B, Trusheim M, Cabra HA. Benefits and challenges of performance-based managed entry agreements: report from an ISPOR Payer Summit. Value & Outcomes Spotlight. 2022. 8(2):13-15.