Differentiation of Health-Related Quality of Life Outcomes Between Five Disease Areas: Results From an International Survey of Patients

Abstract

Objectives:Health-related quality of life (HRQoL) data generated by generic, preference-based instruments (i.e., EQ-5D) are highly demanded in health policy decision making, because they allow for direct comparisons of HRQoL outcomes between disease areas. We aimed to quantify HRQoL outcomes in breast cancer (BC), rheumatoid arthritis (RA), multiple sclerosis (MS), rare cancers (RC), and rare disease (RD) patients and understand the patterns that differentiate HRQoL outcomes between these disease areas, and more specifically between rare and more common disease population groups.Methods:An international, Web survey of patients measured HRQoL (EQ-5D-5L), self-perceived health (EQ-5D-5L Visual Analogue Scale), and additional QoL dimensions, such as patient disability level.Results:We received 675 completed responses. Average utility loss was 53.5 percent, 32.5 percent, and 33.3 percent for RD, RA, and MS patients, respectively, in contrast to 18.6 percent for BC and RC patients. Statistically significant differences (p < .05) were observed between disease groups in all EQ-5D-5L domain outcomes, apart from that of “Anxiety/Depression.” Severe and/or extreme problems were reported in performing usual activities for RD and RC (34 percent and 13 percent of overall problems reported respectively), mobility for MS (18 percent), pain/discomfort for RA (13 percent), and anxiety/depression for BC (7 percent) patients.Conclusions:We demonstrated significant differences in the dimensions that drive HRQoL outcomes between rare and more common diseases and showcased that the same EQ-5D utility may reflect very different severities depending on the patient population under investigation. Future research should examine whether outcomes in other, critical HRQoL domains not included in generic measures also highlight significant differences across disease areas.

Evaluating the Health-Related Quality of Life of the Rare Disease Population in Hong Kong Using EQ-5D 3-Level

“…Only 3 other studies have attempted to determine HRQOL of patients across several RD categories using the EQ-5D measure. 5,22,23 Findings from the current study were corroborated with evidence from the 3 published cohorts, combined as part of a meta-analysis. The pooled utility score was 0.57 (SD 0.09) across 4 studies.…”

“…Similar to this study, patients with rare neurologic diseases consistently report the lowest utility scores among all RD categories in other studies. 5,21,22,[38][39][40] Neurologic diseases are characterized by dysfunction in the brain or nervous system, resulting in poor physical or mental functioning. 41 Progressive muscle degeneration, fatigue, and difficulty in respiration and ambulation drastically decrease the HRQOL in these patients.…”

“…21 To date, only 3 other studies have investigated the HRQOL of the RD population as a whole using EQ-5D. 5,22,23 All 3 studies highlighted significantly lower HRQOL than the general population and other chronic illnesses. Nevertheless, there is an apparent gap in existing literature on HRQOL of patients with RD in Asia and cohorts with a variety of RDs.…”

The promise of Immuno-oncology: implications for defining the value of cancer treatment

“…There is increasing interest in ‘going beyond QALYs’, to measure and systematically incorporate patient reported outcomes (PRO) in oncology [57–59], as there are signals (from markets outside the U.S.) that surrogate endpoints like PFS may not be closely associated with improvements in health-related quality of life in oncology clinical trials [60], or that current health-related quality of life instruments lack uniformity when applied across therapeutic areas [61]. While various work has suggested how to set standards for PRO use for cancer clinical trials with international standards [62], or in clinical trial protocols [63], there is more to be done before this work is ready for inclusion in value assessments.…”

Construct Validity of a Korean Version of the WHOQOL-BREF and EQ-5D Questionnaire Applied on Various Cancer Survivors Undergoing Palliative Rehabilitation Programs

“…2020;27(3):212-219 concepts and are intended to provide scores that are sensitive to all ranges of the concept being measured. By contrast, some measures are designed to assess the aspects of health status re sulting from a particular pathology and to view the attribution of functional limitations to the specific condition [4,5].…”