Published on November 10th, 2021 📆 | 5244 Views ⚑0
Inclusion In Health Technology Assessments: The First Step Toward Equity
Many researchers working on health technology assessments (HTAs) are keen to address disparities in health care. But the COVID-19 pandemic and recent prominence of the Black Lives Matter movement have cast a bright spotlight on structural racism in the US and elsewhere, showing that addressing health disparities after they are established is both too late and not enough. To eliminate health disparities, researchers have been called on to examine underlying inequities, specifically the ways that systematic exclusions are baked into the principles, data, approaches, and even objective methods that we use in our work. To be accountable for our own role in perpetuating health disparities, researchers must examine how our assumptions and approaches perpetuate racism.
Health technology assessment should be at the top of the accountability list, for at least two reasons:
First, this work is consequential. While, US health insurers use varying approaches for determining when or at what price to cover medical interventions, formal HTA methods are gaining momentum as one potential solution to ever-escalating health care expenditures. The US is now poised to expand on its localized, insurer-driven patchwork of value assessment and expenditure management. In response to this need, the Institute for Clinical and Economic Review (ICER) has built a catalog of completed cost-effectiveness analyses, with more ongoing. New attention to value assessment could bring about dramatic changes in the way that insurers cover and pay for treatments and services in the US.
Second, exclusionary practices may be built into the very principles guiding HTAs, especially when that assessment is based on the quality-adjusted life-year (QALY). The QALY is typically measured through population surveys and instruments such as the EQ-5D. Cost per life-year estimates are also typically adjusted by “utilities” for different states of health, and these too are usually estimated from surveys of the population. Survey participants are asked to assign decrements of value for living one year in a state of illness, pain, disability, or older age compared to living one year in a state of perfect health at a younger age. In the classical QALY approach, these preferences should by definition be expressed by community members and not by patients.
QALYs, which are at the foundation of most HTAs, have been the subject of significant criticism. A recent systematic review of these criticisms helpfully catalogues specific published criticisms of the QALY, distinguishing those based in methodological concerns that may be addressed by changes to the QALY itself from those based in ethical concerns that need to be addressed through political or policy discussions.
Well-documented concerns about the QALY point to its systematic discrimination toward older adults, the chronically ill, and the disabled. A guiding principle of most current HTA practices is that people with lived experience of the condition of interest should not be recruited for surveys that support the QALY-based HTA. Those with lived experience of the condition of interest are therefore systematically excluded from two core aspects of QALY measurement: determining which outcomes are used to measure value and determining how to adjust utilities for different health states. They are excluded on the assumption that those with lived experience are in a conflict of interest when placing value on interventions they might receive and that societal-level estimates are the only way to generate apples-to-apples comparisons of value across conditions and interventions. For those in the disability rights community, societal estimates of utility systematically undervalue disability care.
We posit that in this effort to generate comparable estimates, the approach bakes in societal-level biases, systematic exclusions, and structural racism. This problem is not merely a matter of the public or policy deliberation that follows production of a QALY estimate. It is also a function of the processes used to estimate QALYs.
Too Little, Too Late
For instance, although ICER and other HTA organizations have engaged patients in public deliberations, these efforts have not fundamentally changed the exclusion of patient voices from these two key QALY inputs. The still-reigning method excludes the value of experience and perspective of patients in generating value estimates.
Patient involvement in public deliberations after the data are collected and estimates are generated is again too little and too late. Keeping the lived experience of people with health needs at the center of research is possible, including in HTAs. Patient-centeredness as a concept has “caught on” in clinical care, beginning with the patient-centered medical home. Measurement of care quality followed next with federal payers establishing programs to bring patients into the quality measurement lifecycle.
Now, more than a decade beyond the founding of the Patient-Centered Outcomes Research Institute (PCORI), the US has joined other countries in addressing the role of the patient in research. One of the last holdouts in establishing patients at the center of health care and research are the formally developed and endorsed methods for valuing health care interventions. To date, there have been several, repeated missed opportunities for incorporating a representative patient perspective in the data that are used to inform health care valuation. Some trials submitted for regulatory approval have fallen short of adequate representation of patients from communities of color, for example, in cancer and ophthalmology and Alzheimer’s disease.
The same kinds of exclusions have left the US poorly informed in other areas of health. For instance, public health experts were ill prepared to address high rates of vaccine hesitancy due to a lack of adequate data on patient values and preferences.
Collecting and using the perspective of patients—getting to know the outcomes for health interventions that patients value—is a way forward. We have an opportunity right now—through a window opened up by our shared experience of COVID-19—to address HTAs’ dominant White medical culture with a much more diverse and inclusionary involvement of patients representing their lived experience in the US health care system. Here’s how:
Multiple-Criteria Decision Analysis
Understanding the experiences of different types of patients is key. The elements exist here in the US to improve health technology assessment. The comparative effectiveness research approach central to the research funded by PCORI, coupled with the focus on patient-centered outcomes, is part of the equation. Identifying outcomes that matter most to all types of patients requires focused investment. So too does the work of generating a robust body of evidence on inclusive patient-centered comparative effectiveness for diverse populations. Without these pieces, we cannot get value assessment right for people who have been excluded from health research and are disproportionately affected by poor health outcomes. Currently, methods used in other fields are being adapted for use in valuing medical interventions. One such method, multiple-criteria decision analysis (MCDA), has the advantage of allowing for inclusion of multiple, diverse stakeholder views in the criteria that are part of the decision analysis and in the weighting of those criteria.
The current cultural moment in medicine makes wider acceptance and use of more inclusive methods in HTA possible. We believe seizing this opportunity will fortify the ethical foundations of our work. We are calling on researchers to stop business as usual and to apply an inclusion and an equity lens to all we do, while avoiding appropriation of resources better used by those with equity research expertise and lived experience. Methodologists’ attention to outreach and inclusion of patients, clinicians, and caregivers is critical. This outreach must include non-White communities in a way far more robust than what has been accomplished so far. Just as attention to stakeholder engagement requires awareness and expertise, so does attention to health equity. Deliberative methods, technology-enabled surveys, and online crowdsourcing offer substantial advantages to get past current-day exclusions of those with relevant lived experience.
Due to heightened awareness among the majority White research and clinical communities of the need to meaningfully change the race and ethnicity inequities built into the system of health care, we have the opportunity now to improve methods for health technology assessments. Ensuring inclusiveness of views incorporated into HTAs and pushing HTA measures beyond the usual can support methods that address racial and ethnic equity by design. As the US is poised to scale up HTAs, the methods used need to be the right ones.
This post is based on work funded by the PhRMA Foundation and PhRMA. Frank and Concannon received the PhRMA Foundation Value Assessment Challenge Award in 2019 (1st place) in recognition of their work on the Patient Engaged Healthcare Valuation Strategy. They also receive funding from the Innovation and Value Initiative (IVI) and PhRMA for related work.