Study finds important health outcomes are not included in cost-effectiveness analyses for sickle cell, Alzheimer’s and MS treatments

Katie Koziara
Katie Koziara May 19, 2022

Older female patient talking with a doctor.

Share This

A new study from the Office of Health Economics (OHE) confirmed that the EQ-5D, a tool commonly used in health technology assessments (HTAs) often excludes outcomes important to patients and their caregivers who rely on treatments for multiple sclerosis, Alzheimer’s disease and sickle cell disease.

The OHE analysis shows that methods based on the quality adjusted life year, or QALY, used by many prominent HTA organizations, such as the Institute for Clinical and Economic Review, frequently overlook outcomes that patients value. When health plans rely on these assessments to set coverage or utilization management policies, they can prevent patients from accessing treatments.

One method used to quantify quality of life is the EQ-5D survey. While the survey does capture some aspects of health, like mobility, self-care and pain, it still has the same basic flaws as other QALY-based value assessments. By rolling up a range of quality-of-lie factors into a few simple measures, it devalues specific outcomes that are important to some patients and therefore may not recognize unique differences in how diseases impact a patient’s overall wellbeing. The OHE study explains the limitations of using EQ-5D across three disease areas:

Multiple Sclerosis: A chronic, neurological disease that affects around 900,000 people in the United States.

  • Assessments that use EQ-5D do not include impact on fatigue, vision degeneration, bladder dysfunction and cognition, and disease severity.
  • Another flaw is that respondents are asked to assess their health on the day of the assessment. Since people with multiple sclerosis experience fluctuations in their symptoms depending on the day, this is limiting and could undervalue treatments.

Alzheimer’s Disease: A progressive, neurological disease that causes damage to the brain and is estimated to affect 6.2 million people aged 65 and older in the United States.

  • EQ-5D method is less applicable in this patient population than other assessments that focus on outcomes that are important to people with Alzheimer’s disease, like memory loss, cognition, fatigue, incontinence and hearing problems.
  • The quality of life of a caretaker for patients with this disease is an important aspect to capture in value assessments too and the EQ-5D fails to capture that, which could lead to undervaluing treatments.

Sickle Cell Disease: An inherited, chronic disease that causes recurrent pain and hospitalizations that affects an estimated 100,000 people in the United States, including 1 out of every 365 Black Americans.

  • A main symptom of sickle cell disease is a vaso-occlusive crisis, which is painful and often requires hospitalization. These crises do not occur daily, but because the EQ-5D asks about a patients’ health ‘today’, it may fail to accurately reflect the pain of people with the disease. Treatments to lessen pain may therefore appear “less effective” as a result.
  • Gaps in assessments that use EQ-5D include the frequency of crises over the past 12 months and details about a patient’s most recent crisis.

In measuring health outcomes, it is important we use evidence and methods that reflect what matters to patients, are open and transparent, support doctor-patient decision-making and are based on rigorous science. Basing decision-making on HTAs that fail to capture what matters to patients ignores their needs and could lead to restrictions on access to treatments. If we’re going to get serious about value in the U.S. health care system, we need to get serious about valuing outcomes that matter to patients.

To learn more about value assessments, visit phrma.org/value-collaborative.