To ensure that value assessment accounts for patient needs, we need a better understanding of the outcomes that are most important to patients, write authors from COVIA Health Solutions and the University of Washington School of Pharmacy.
In recent years—and perhaps in reaction to rising health care costs and increased attention towards achieving greater efficiency out of current spending—various methodological frameworks have emerged which seek to assess the value of health care treatments and interventions and inform pricing, coverage, and reimbursement decisions.
As these critical endeavors move forward, one factor is emerging with growing consensus: the need for value assessment research to be more inclusive of the patient perspective. And while great strides have been made to determine what outcomes truly matter to patients and therefore should be foundational to assessing and tracking value in health care, the field is lacking a means to align value with patient-centered outcomes (PCOs) that can be captured, measured, and operationalized.
Traditional value assessment methods, which rely on the conventional Quality Adjusted Life Year (QALY), have their limitations. In particular, basing a therapy’s value solely on the QALY may overlook some elements of value that matter most to patients, such as the treatment’s impact on their ability to work or stay active, the potential for reduced side effects, or the impact on family and caregivers.
These patient-derived value elements, such as the high caregiver burden experienced by family members and friends of patients diagnosed with complex diseases such as cystic fibrosis or multiple myeloma, the value of hope for treatment, and fear of contagion are often quantitatively excluded from traditional value assessment models.1 However, these costs can be substantial. A new study estimating the economic effects of coronavirus disease 2019 (COVID-19) found that China lost $275 billion during the Chinese Lunar New Year week due to the fear of contagion.2 Ensuring such elements become a crucial part of the value conversation should be the end goal of any attempt to redefine measurements of treatment value.
But in order to ensure that value assessment accounts for what matters most to patients, we need a better understanding of the outcomes that are most important to patients. To date, some progress has been made on developing PCO measures, most of which have the goal of advancing research or clinical care.
Capturing the patient voice in value assessment has been an ongoing objective of the PhRMA Foundation’s Value Assessment Initiative. Researchers at the Patient-Driven Values in Healthcare Evaluation (PAVE) Center at the University of Maryland are working to develop a framework for identifying patient-driven value elements for chronic conditions, starting with chronic obstructive pulmonary disease (COPD), childhood allergies, and hepatitis C. A team at the University of Colorado’s Center for Pharmaceutical Value (pValue) is testing methods to allow greater flexibility in accounting for attributes most important to stakeholders in assessing value in health care. Others, including the Center for Enhanced Value Assessment (CEVA) at Tufts Medical Center also aim to incorporate broader elements of value into economic evaluations.
So, where do we go from here?
First, we need to identify all pertinent PCOs by disease, and perhaps even by severity of within a specific disease state. We can learn from a number of ongoing efforts that aim to identify specific core outcome sets by disease. The Core Outcome Measures in Effectiveness Trials (COMET) Initiative, which seeks to establish comprehensive reviews and a searchable database of core outcomes sets, offers a useful model. Other efforts such as the ICHOM standard sets, and the Green Park Collaborative’s Core Outcomes Set Initiative are instructive as well.
These projects aim to establish a minimum set of core outcomes to be measured and reported in clinical studies for treatment of specific disease conditions and offer a useful model for how to incorporate patient perspectives into clinical research. However, it is not yet clear whether these efforts are sufficient to capture all pertinent PCOs, nor do these efforts appear to specifically link these core outcomes to treatment value.
We need to determine the degree to which existing activities are capturing pertinent PCOs and fill in the gaps where they may occur. Second, we must explore the effort to link all pertinent PCOs in a national repository that could serve as a go-to resource for anyone interested in understanding the value of health care interventions. This repository, which would organize key PCOs on a disease-specific basis, could serve as a “one stop shop” for researchers looking to build patient-centered comparative effectiveness research or include patient-centered value elements into economic models. Such a repository could also inform outcomes-based contracting, quality measurement, and regulatory decision-making.
Establishing a PCO repository will require the active support of all stakeholders, including the patient community, payers, health outcomes researchers, and providers with disease-specific expertise to make it part of their mission. Redefining current value assessment methods in a manner that truly captures the patient perspective is a challenge for the entire health care community to tackle.
We have several steps to take before we can achieve such an endeavor, but working together to establish a common repository of PCOs will bring us closer to a health care system that offers the value all stakeholders are seeking.
Sachin Kamal-Bahl, PhD, is president of COVIA Health Solutions. Bryan Luce, PhD, MBA, is an affiliate professor at the University of Washington School of Pharmacy.
1. Lakdawalla DN, Doshi JA, Garrison LP, Phelps CE, Basu A, Danzon PM. Defining elements of value in health care – a health economics approach: An ISPOR special task force report. Value in Health. 2018;21(2):131-139. doi:10.1016/j.jval.2017.12.007
2. Ma S, Kim DD, Cohen JT, Neumann PJ. Measuring “fearonomics fffects” in valuing therapies: An application to Covid-19 in China. Value in Health. 2020;23(11):1405-1408. doi:10.1016/j.jval.2020.06.002