Considering the Needs of Older Patients in Value Assessments

When we consider health care value, how can we assess the challenges faced by older patients with multiple diseases and their caregivers when making unique choices about their treatment options? It’s a question that is top of mind for Sue Peschin, MHS, President and CEO at the Alliance for Aging Research, a nonprofit organization dedicated to accelerating the pace of scientific discoveries and their application to vastly improve the universal human experience of aging and health. With the growing interest in value assessment frameworks as a way to evaluate health care treatments and costs, the Alliance has raised concerns about basing certain economic calculations of a therapy’s “value” on the health status, disability and age of the patient instead of on the benefits therapies provide. In commentaries and a new website outlining these concerns, the Alliance said these calculations could result in discrimination against older patients or people living with disabilities. National Pharmaceutical Council President and Chief Executive Officer Dan Leonard spoke with Sue to get her take on the health needs of America’s aging population, the role of value assessments, and their potential impact on access and research moving forward.

Dan: Welcome, Sue. First, please tell us about the Alliance for Aging Research’s work and its impact on patient care, and how the needs of an aging population may affect health care today and in the future. How does that relate to value assessments?

Sue: The Alliance for Aging Research has been around since 1986. Our mission is to advance research as a way to promote healthy aging. The older population has been on a growth trajectory that will continue through 2030. Older adults are the largest consumers of health care in the United States and will continue to be for decades to come. So any conversations around value in health care have to include the preferences of and considerations for older adults.

Dan: When I’ve heard you speak about value assessment frameworks, you’ve mentioned concerns that the existing frameworks don’t adequately capture the unique choices facing patients with multiple comorbidities, particularly older patients. What are some of the Alliance’s specific concerns? Thoughts on how to address them?

Sue: The European Medicines Agency has set a good example with its Geriatric Medicines Strategy. The Strategy seeks to capture the unique geriatric considerations in clinical development and clinical care for older adults. Innovative measures, including ones that allow for multiple chronic conditions in clinical trial participation, allow for a more real-world design and application of research once a new medical product goes to market. We should be thinking about these considerations before we even get to value assessments.

Where parts of Europe get it wrong, however, is after a medical product is approved, cost effectiveness models that use quality-adjusted life years, or QALYs, are utilized to assess value. So the value assessment side cancels out the positive work on the clinical development side. We need a balance that incorporates older patients’ preferences in both clinical development and value assessment.

Countries that use QALYs as the basis for cost effectiveness to make coverage decisions, particularly in Europe, tend to have worse health outcomes in major disease areas compared to the United States. These decisions often impact patient access issues, so people have to wait longer for new treatments.

Dan: Many groups have questioned the reliance on QALYs to judge value, and the Alliance is no exception. What are the particular concerns about QALYs in evaluating treatments for older populations? What are better alternatives?

Sue: QALYs don’t adjust for quality of life issues based on age, disability or severity of illness. There is no allowance for heterogeneity in the patient population.  It puzzles me how that’s helpful from a payer perspective.  No insured population is homogenous, so these assessments are limited and not reflective of the populations served. For example, a 2013 study from the European Consortium in Health Care Outcomes and Cost Benefit Research surveyed 1,300 people from four countries. They found that the QALY model was highly divergent and inconsistent in recommendations on costs, patient preferences and access.

The QALY is a measure invented to estimate the cost-effectiveness of a medical treatment using mortality, morbidity and cost data. When applied to health care decisions, the Institute for Clinical and Economic Review’s methodology can mean older adults, people with disabilities and veterans are deemed “too expensive” to receive care. ICER's description of its “equal value of life years gained” metric, which is simpler than QALYs, states they do not adjust for quality of life differences arising from “age, severity of illness or level of disability.”

ICER’s use of QALYs means that if a 30-year-old and a 60-year-old have a fatal disease and receive identical treatments, which result in a 100 percent cure and expectation of living to 80 years of age, it is 2.5x more cost effective to treat the younger patient than the older one. This is health rationing and takes treatment options off the table for doctors and their patients.

We need to have committed discussions about doing the research to develop new models.

I see a role across the National Institutes of Health, in coordination with the Patient-Centered Outcomes Research Institute (PCORI), to develop some alternative measures that better capture the needs and preferences of patient subgroups.

Dan: There’s also a need for more evidence about how treatments work in the “real-world,” especially for elderly patients. How can we encourage researchers to develop this evidence and health care decision-makers to consider it as part of value assessments?

Sue: There are opportunities with patient registry data and pragmatic trials to gain more insight into the real-world experience of older adult patients. I think patient registries are increasingly being adopted in coverage decision-making, but we have a long way to go in terms of using pragmatic clinical trials. The National Institute on Aging has been a lead group in promoting the use of pragmatic clinical trials as well as age-inclusion across the life span, but it is much less common across industry which is where the rubber hits the road. Clinical trials for the Food and Drug Administration approval tend to be more restrictive.

Dan: As frameworks are developed and undergo revisions, such as ICER, what can developers do to ensure patients’ individual needs and priorities are taken into consideration? What about the unique perspectives of older populations?

Sue: The primary issue is whether it is enough to be open to modifying your existing model or whether we need to be exploring other models. If you have a discriminatory model to start with, it doesn’t matter if you are checking a box and talking to patient advocacy groups. We need to have committed discussions about doing the research to develop new models. I would argue that comparative clinical effectiveness research, like the research funded by PCORI, has a role to play and can serve as a model for integration of patient preferences and is particularly valuable in clinical areas where there are multiple medical options available that could be evaluated against each other.

Dan: How can organizations like ICER improve their engagement processes with patients and patient-focused policy organizations like the Alliance?

Sue: ICER solicits comments from disease-specific organizations from the clinical area that they are focusing on, but they do it after the fact and in a “check the box” manner. Survey work from the Partnership to Improve Patient Care and from anecdotal experiences of patient advocacy groups reveal a lot of frustration and over-extension above and beyond their day jobs to engage with ICER on a review.  They are spending time and resources on something they don’t have the bandwidth for that takes away from their existing missions and service to patients.

We all have been led to believe that ICER is the only game in town, therefore you have to “go along to get along.”  The Alliance has taken the position that what ICER is selling, we’re not buying.  We need alternatives and not to limit ourselves to old ways of thinking that originated to initiate health rationing. We have to be able to do better than that.