Development and validation of a claims-based algorithm to identify patients with Neuromyelitis Optica Spectrum disorder.

INTRODUCTION: No validated algorithm exists to identify patients with neuromyelitis optica spectrum disorder (NMOSD) in healthcare claims data. We developed and tested the performance of a healthcare claims-based algorithm to identify patients with NMOSD. METHODS: Using medical record data of 101 adults with NMOSD, multiple sclerosis (MS), or myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), we tested the sensitivity and specificity of claims-based algorithms developed through interviews with neurologists. We tested the best-performing algorithm's face validity using 2016-2019 data from IBM MarketScan Commercial and Medicare Supplemental databases. Demographics and clinical characteristics were reported. RESULTS: Algorithm inclusion criteria were age ≥ 18 years and (≥1 NMO diagnosis [or ≥ 1 transverse myelitis (TM) and ≥ 1 optic neuritis (ON) diagnosis] and ≥ 1 NMOSD drug) or (≥2 NMO diagnoses ≥90 days apart). Exclusion criteria were MS diagnosis or use of MS-specific drug after last NMO diagnosis or NMOSD drug; sarcoidosis diagnosis after last NMO diagnosis; or use of ≥1 immune checkpoint inhibitor. In medical record billing data of 50 patients with NMOSD, 30 with MS, and 21 with MOGAD, the algorithm had 82.0% sensitivity and 70.6% specificity. When applied to healthcare claims data, demographic and clinical features of the identified cohort were similar to known demographics of NMOSD. CONCLUSIONS: This clinically derived algorithm performed well in medical records. When tested in healthcare claims, demographics and clinical characteristics were consistent with previous clinical findings. This algorithm will enable a more accurate estimation of NMOSD disease burden using insurance claims datasets.

Expert Consensus Recommendations on the Management of Treatment-emergent Adverse Events Among Men with Prostate Cancer Taking Poly-ADP Ribose Polymerase Inhibitor + Novel Hormonal Therapy Combination Therapy.

BACKGROUND AND OBJECTIVE: Recent clinical trials have shown improvement in progression-free survival in men with metastatic prostate cancer (mPC) treated with combination poly-ADP ribose polymerase (PARP) inhibitors (PARPi) and novel hormonal therapy (NHT). Regulatory bodies in the USA, Canada, Europe, and Japan have recently approved this combination therapy for mPC. Common adverse events (AEs) include fatigue, nausea and vomiting, and anemia. Nuanced AE management guidance for these combinations is lacking. The panel objective was to develop expert consensus on AE management in patients with mPC treated with the combination PARPi + NHT. METHODS: The RAND/University of California Los Angeles modified Delphi Panel method was used. AEs were defined using the Common Terminology Criteria for Adverse Events. Twelve experts (seven medical oncologists, one advanced practice registered nurse, three urologists, and one patient advocate) reviewed the relevant literature; independently rated initial AE management options for the agent suspected of causing the AE for 419 patient scenarios on a 1-9 scale; discussed areas of agreement (AoAs) and disagreement (AoDs) at a March 2023 meeting; and repeated these ratings following the meeting. Second-round ratings formed the basis of guidelines. KEY FINDINGS AND LIMITATIONS: AoDs decreased from 41% to 21% between the first and second round ratings, with agreement on at least one management strategy for every AE. AoAs included the following: (1) continue therapy with symptomatic treatment for patients with mild AEs; (2) for moderate fatigue, recommend nonpharmacologic treatment, hold treatment temporarily, and restart at a reduced dose when symptoms resolve; (3) for severe nausea or any degree of vomiting where symptomatic treatment fails, hold treatment temporarily and restart at a reduced dose when symptoms resolve; and (4) for hemoglobin 7.1-8.0 g/dl and symptoms of anemia, hold treatment temporarily and restart at a reduced dose after red blood cell transfusion. CONCLUSIONS AND CLINICAL IMPLICATIONS: This expert guidance can support management of AEs in patients with mPC receiving combination PARPi + NHT therapy. PATIENT SUMMARY: A panel of experts developed guidelines for adverse event (AE) management in patients with metastatic prostate cancer treated with a combination of poly-ADP ribose polymerase inhibitors and novel hormonal therapy. For mild AEs, continuation of cancer therapy along with symptomatic treatment is recommended. For moderate or severe AEs, cancer therapy should be stopped temporarily and restarted at the same or a reduced dose when AE resolves.

Modified Delphi panel consensus recommendations for management of severe aplastic anemia.

ABSTRACT: Severe aplastic anemia (SAA) is a rare hematologic condition for which there is no clear management algorithm. A panel of 11 experts on adult and pediatric aplastic anemia was assembled and, using the RAND/University of California, Los Angeles modified Delphi panel method, evaluated >600 varying patient care scenarios to develop clinical recommendations for the initial and subsequent management of patients of all ages with SAA. Here, we present the panel's recommendations to rule out inherited bone marrow failure syndromes, on supportive care before and during first-line therapy, and on first-line (initial management) and second-line (subsequent management) therapy of acquired SAA, focusing on when transplant vs medical therapy is most appropriate. These recommendations represent the consensus of 11 experts informed by published literature and experience. They are intended only as general guidance for experienced clinicians who treat patients with SAA and are in no way intended to supersede individual physician and patient decision making. Current and future research should validate this consensus using clinical data. Once validated, we hope these expert panel recommendations will improve outcomes for patients with SAA.

Expert consensus on the management of infusion-related reactions (IRRs) in patients with sickle cell disease (SCD) receiving crizanlizumab: a RAND/UCLA modified Delphi panel.

Crizanlizumab, a monoclonal antibody against P-selectin, has been shown to reduce vaso-occlusive crises (VOCs) compared to placebo in patients ≥ 16 years with sickle cell disease (SCD). However, there have been rare reports of patients experiencing severe pain and subsequent complications within 24 hours of crizanlizumab infusions. These events are defined as infusion-related reactions (IRRs). Informed by current literature and clinical experience, a group of content experts developed clinical guidelines for the management of IRRs in patients with SCD. We used the RAND/University of California, Los Angeles (UCLA) modified Delphi panel method, a valid, reproducible technique for achieving consensus. We present our recommendations for managing IRRs, which depend on patient characteristics including: prior history of IRRs to other monoclonal antibodies or medications, changes to crizanlizumab infusion rate and patient monitoring, pain severity relative to patient's typical SCD crises, and severe allergic symptoms. These recommendations outline how to evaluate and manage IRRs in patients receiving crizanlizumab. Future research should validate this guidance using clinical data and identify patients at risk for these IRRs.

Managing hyperglycemia and rash associated with alpelisib: expert consensus recommendations using the Delphi technique.

Hyperglycemia and rash are expected but challenging adverse events of phosphatidylinositol-3-kinase inhibition (such as with alpelisib). Two modified Delphi panels were conducted to provide consensus recommendations for managing hyperglycemia and rash in patients taking alpelisib. Experts rated the appropriateness of interventions on a 1-to-9 scale; median scores and dispersion were used to classify the levels of agreement. Per the hyperglycemia panel, it is appropriate to start alpelisib in patients with HbA1c 6.5% (diabetes) to <8%, or at highest risk for developing hyperglycemia, if they have a pre-treatment endocrinology consult. Recommend prophylactic metformin in patients with baseline HbA1c 5.7% to 6.4%. Metformin is the preferred first-line anti-hyperglycemic agent. Per the rash panel, initiate prophylactic nonsedating H1 antihistamines in patients starting alpelisib. Nonsedating H1 antihistamines and topical steroids are the preferred initial management for rash. In addition to clinical trial evidence, these recommendations will help address gaps encountered in clinical practice.

Using a Modified Delphi Panel to Estimate Health Service Utilization for Patients with Advanced and Non-Advanced Systemic Light Chain Amyloidosis.

Purpose: Patients with diagnosed with systemic light chain (AL) amyloidosis at advanced Mayo stages have greater morbidity and mortality than those diagnosed at non-advanced stages. Estimating service use by severity is difficult because Mayo stage is not available in many secondary databases. We used an expert panel to estimate healthcare utilization among advanced and non-advanced AL amyloidosis patients. Patients and Methods: Using the RAND/UCLA modified Delphi method, expert panelists completed 180 healthcare utilization estimates, consisting of inpatient and outpatient visits, testing, chemotherapy, and procedures by disease severity and organ involvement during two treatment phases (the 1 year after starting first line [1L] therapy and 1 year following treatment [post-1L]). Estimates were also provided for post-1L by hematologic treatment response (complete or very good partial response [CR/VGPR], partial, no response or relapse [PR/NR/R]). Areas of disagreement were discussed during a meeting, after which ratings were completed a second time. Results: During 1L therapy, 55% of advanced patients had ≥1 hospitalization and 38% had ≥2 admissions. Rates of hematopoietic stem cell transplant (HSCT) in advanced patients were 5%, while pacemaker or implantable cardioverter defibrillator (ICD) placement were 15%. During post-1L therapy, rates of hospitalization in advanced patients remained high (≥1 hospitalization: 20-43%, ≥2 hospitalizations: 10-20%), and up to 10% of advanced patients had a HSCT. Ten percent of these patients underwent pacemaker/ICD placement. Conclusion: Experts estimated advanced patients, who would not be good candidates for HSCT, would have high rates of hospitalization (traditionally the most expensive type of healthcare utilization) and other health service use. The development of new treatment options that can facilitate organ recovery and improve function may lead to decreased utilization.

Apremilast Adherence and Persistence in Patients with Psoriasis and Psoriatic Arthritis in the Telehealth Setting Versus the In-person Setting During the COVID-19 Pandemic.

INTRODUCTION: Limited access to healthcare during the COVID-19 pandemic prompted patients to seek care using telehealth. In this study, we assessed whether treatment patterns differed for patients with psoriasis (PsO) or psoriatic arthritis (PsA) initiating apremilast by either a telehealth or an in-person visit. METHODS: We estimated adherence and persistence among US patients in the Merative© MarketScan© Commercial and Supplemental Medicare Databases who newly initiated apremilast between April and June 2020, categorized by the type of visit (telehealth or in-person) when apremilast was first prescribed. Adherence was defined as the proportion of days covered (PDC), with PDC ≥ 0.80 considered to indicate high adherence. Persistence was defined as having apremilast available to take without a 60-day gap during follow-up. Factors associated with high adherence and persistence were estimated with logistic and Cox regression. RESULTS: Among apremilast initiators (n = 505), the mean age was 47.6 years, 57.8% were female, and the majority had PsO (79.6%). Telehealth index visits were more likely among patients residing in Northeast USA (odds ratio [OR] 3.31, 95% confidence interval [CI] 1.63-6.71) and Western USA (OR 2.52, 95% CI 1.07-5.93]), those with a prescribing rheumatologist (OR 2.27, 95% CI 1.10-4.68), and those with any baseline telehealth visit (OR 1.91, 85% CI 1.20-3.04). Those initiating apremilast with a telehealth visit (n = 141) had similar mean PDC to those initiating apremilast with an in-person visit (n = 364) (0.695 vs. 0.728; p = 0.272). At the end of the 6-month follow-up, 54.3% of the overall population had high adherence (PDC ≥ 0.80) and 65.1% were persistent. After adjusting for potential confounders, patients initiating apremilast via telehealth had similar full adherence (OR 0.80, 95% CI 0.52-1.21) and persistence as those initiating apremilast in-person. CONCLUSION: Patients with PsO and patients with PsA initiating apremilast via telehealth or in-person during the COVID-19 pandemic had similar medication adherence and persistence during the 6-month follow-up period. These data suggest that patients initiating apremilast can be as effectively managed with telehealth visits as with in-person visits.

Expert Consensus on the Use of On-Demand Treatments for OFF Episodes in Parkinson's Disease: A Modified Delphi Panel.

Background: On-demand treatments can treat OFF episodes in Parkinson's disease, however, there is limited information regarding when to prescribe them. Objective: Develop expert consensus to determine appropriate clinical factors for considering on-demand treatments. Methods: Using a RAND/UCLA modified Delphi panel method, a panel developed consensus on the use of on-demand treatments for OFF episodes. Results: The panel agreed on-demand treatments were appropriate when OFF episodes were associated with greater functional impact and interfered with basic daily activities. The panel also agreed on-demand treatment may be appropriate for patients with morning akinesia and/or delayed ON of first levodopa dose and >1 type of OFF episode (eg, early morning OFF or wearing OFF regardless of frequency). Conclusions: Experts agreed on-demand treatment is appropriate for many patients with OFF episodes. The greater the functional impact of OFF episodes, the more likely experts agreed that on-demand treatment is appropriate to prescribe.

In-hospital mortality in amyloid light chain amyloidosis: analysis of the Premier Healthcare Database.

Aim: Describe the clinical and economic burden of hospitalizations for amyloid light chain (AL) amyloidosis. Materials & methods: This retrospective analysis used nationally representative hospital discharge data (2017-2020) to report discharge status, resource use and costs for hospitalizations among patients with AL amyloidosis. Results: Of 1341 patients identified, 92% were discharged alive and 8% experienced in-hospital death. Compared with the average US hospital stay during 2017-2019 (4.7 days, mean costs of $13,046 and mean charges of $54,496), hospital stays for AL amyloidosis were longer and costlier (9.7 days, $27,098.61, $111,233.91), especially in patients with in-hospital death (12.2 days, $44,966, $182,338.18). Conclusion: AL amyloidosis is associated with significant clinical and economic burden.

Impact of early detection on cancer curability: A modified Delphi panel study.

Expert consensus on the potential benefits of early cancer detection does not exist for most cancer types. We convened 10 practicing oncologists using a RAND/UCLA modified Delphi panel to evaluate which of 20 solid tumors, representing >40 American Joint Committee on Cancer (AJCC)-identified cancer types and 80% of total cancer incidence, would receive potential clinical benefits from early detection. Pre-meeting, experts estimated how long cancers take to progress and rated the current curability and benefit (improvement in curability) of an annual hypothetical multi-cancer screening blood test. Post-meeting, experts rerated all questions. Cancers had varying estimates of the potential benefit of early cancer detection depending on estimates of their curability and progression by stage. Cancers rated as progressing quickly and being curable in earlier stages (stomach, esophagus, lung, urothelial tract, melanoma, ovary, sarcoma, bladder, cervix, breast, colon/rectum, kidney, uterus, anus, head and neck) were estimated to be most likely to benefit from a hypothetical screening blood test. Cancer types rated as progressing quickly but having comparatively lower cure rates in earlier stages (liver/intrahepatic bile duct, gallbladder, pancreas) were estimated to have medium likelihood of benefit from a hypothetical screening blood test. Cancer types rated as progressing more slowly and having higher curability regardless of stage (prostate, thyroid) were estimated to have limited likelihood of benefit from a hypothetical screening blood test. The panel concluded most solid tumors have a likelihood of benefit from early detection. Even among difficult-to-treat cancers (e.g., pancreas, liver/intrahepatic bile duct, gallbladder), early-stage detection was believed to be beneficial. Based on the panel consensus, broad coverage of cancers by screening blood tests would deliver the greatest potential benefits to patients.

Medication Adherence and Healthcare Utilization in Relapsing-Remitting Multiple Sclerosis Patients Using Teriflunomide.

Purpose: Multiple sclerosis (MS) is a costly, immune-mediated disease of the central nervous system. Most patients have relapsing-remitting MS (RRMS) for which disease-modifying therapies (DMTs) provide an effective treatment option by reducing relapse rates. However, adherence to DMTs is suboptimal. This study examines the association between adherence to teriflunomide and clinical and healthcare utilization outcomes. Patients and Methods: Patients with RRMS who started treatment with teriflunomide between 1/1/2018 and 12/31/2019 were analyzed using IQVIA PharMetrics® Plus data. RRMS patients were identified via diagnosis codes and treatment types; the first prescription date for teriflunomide was the index date. Highly and poorly adherent patients were identified based on the proportion of days covered (PDC) post-index (PDC ≥0.8 and PDC ≤0.5, respectively). Patient demographics, clinical characteristics, healthcare utilization during the year pre- and post-index, and relapse rate post-index were reported descriptively. Outcomes were compared between highly and poorly adherent patients through logistic regression. Models were adjusted for demographics, comorbidities, and utilization measures during the baseline period. Results: Among the 922 RRMS patients identified, 534 (57.9%) were highly adherent to teriflunomide, while 249 (27.0%) had PDC ≤0.5. The two groups were not statistically different in terms of demographic characteristics and comorbidities; however, poorly adherent patients were more likely to have emergency department (ED) or inpatient visits during baseline (36.9% versus 26.8%, P=0.004; 17.3% versus 10.9%, P=0.013, respectively). Unadjusted results suggested lower likelihood of both relapses and utilization during follow-up among highly adherent patients compared to poorly adherent patients. Adjusted results confirmed that high adherence was associated with decreased likelihood of post-index relapses, ED utilization, and inpatient utilization (OR [95% CI]: 0.55 [0.39-0.76], 0.49 [0.34-0.71], and 0.51 [0.27-0.97], respectively) even after controlling for baseline utilization. Conclusion: High adherence to teriflunomide was found to be associated with fewer relapses and lower healthcare utilization among patients with RRMS.

Diagnostic amyloid light chain amyloidosis hospitalizations associated with high acuity and cost: analysis of the Premier Healthcare Database.

Aim: Estimate the frequency and costs of diagnostic admissions among hospitalized patients with amyloid light chain (AL) amyloidosis. Materials & methods: This retrospective analysis used nationally representative hospital discharge data from 2017 to 2020 to report resource use and cost for hospitalizations during which AL amyloidosis was diagnosed. Results: Of 1341 admissions, 17.6% were diagnostic. Bone marrow (79.5%) and kidney (44.9%) biopsies were the most common qualifying biopsies. Diagnostic hospitalizations had longer length of stay (14.5 vs 8.4 days; p < 0.001) and higher cost ($40,052 [USD] vs $24,360; p < 0.001) than nondiagnostic ones. Conclusion: Diagnostic admissions are more likely to be urgent/emergent, require longer stays and have higher costs compared with hospitalizations in known AL amyloidosis patients. Improved diagnostic pathways toward early diagnosis are needed.

National burden of achondroplasia: an analysis of the National Inpatient and Nationwide Ambulatory Surgery Samples.

Background: To estimate the cost of US hospital admissions and outpatient surgeries associated with achondroplasia. Materials & methods: Using 2017 data from nationally representative databases, this study identifies hospital admissions and outpatient encounters with an achondroplasia diagnosis. Descriptive measures are reported. Results: There were 1985 achondroplasia admissions nationwide. The most frequent admissions were neonatal care (33.7%) in children and musculoskeletal (22.7%) in adults. Average hospital length of stay was 6.8 days, 2.2 days longer than the US mean. Total mean inpatient costs were US$19,959, $7789 greater than the US mean. In the outpatient setting, children 5-14 years accounted for 56.9% of procedures. Conclusion: Achondroplasia is a serious condition with a wide range of lifelong complications frequently requiring hospitalization and surgical intervention.

Biologic Initiation Rate in Systemic-Naïve Psoriatic Arthritis Patients Starting Treatment with Apremilast vs Methotrexate: 1-Year Retrospective Analysis of a US Claims Database.

Purpose: To compare the rate of biologic initiation after commencing treatment with apremilast (APR) vs methotrexate (MTX), in systemic-naïve patients with psoriatic arthritis (PsA). Patients and Methods: Systemic-naïve patients with PsA who started treatment with either APR or MTX between 01/01/2015 and 12/31/2018 were analyzed using claims data from the IBM® MarketScan® Commercial and Medicare Supplemental databases (2014-2019). PsA patients were identified via diagnosis codes; the first prescription date for APR or MTX was the index date. Patient demographics, clinical characteristics, healthcare utilization during the year pre-index (baseline) and the year post-index (follow-up), and median time to biologic initiation were reported descriptively. The rates and risk of biologic initiation during follow-up were compared between APR and MTX users by logistic and Cox regressions, respectively. Models were adjusted for demographics, clinical and utilization measures during the baseline period. Results: A total of 2116 patients with PsA newly treated with APR (n = 534) or MTX (n = 1582) were identified. Mean age was similar (50.5 vs 50.4; P = 0.938), and proportion of females was higher for APR vs MTX users (59.4% vs 54.0%; P = 0.031). Mean time to biologic initiation among patients who initiated during follow-up was 194.1 vs 138.7 days between APR vs MTX users (P < 0.001). After adjusting for confounders, the likelihood of biologic initiation was 58% lower (OR, 0.42 [95% CI, 0.32-0.54]; P < 0.001) with APR, with a significantly lower predicted rate of biologic initiation among APR users when compared to MTX users during follow-up (20.0% [95% CI, 16.6-23.9%] vs 37.5% [95% CI, 35.0-40.1%]). Additionally, APR users had a lower risk of biologic initiation than MTX users (HR, 0.46 [95% CI, 0.37-0.57]; P < 0.001) during the 1-year follow-up. Conclusion: Systemic-naïve patients with PsA have a lower rate of, and longer time to, biologic initiation over one-year following APR initiation, compared to those initiating MTX.

Biologic initiation rates in systemic-naive psoriasis patients after first-line apremilast versus methotrexate use.

Aim: To compare rates of biologic initiation after commencing treatment with apremilast (APR) versus methotrexate (MTX) in systemic-naive patients with psoriasis (PsO). Methods: This was a retrospective cohort study of systemic-naive patients with PsO who initiated treatment with APR or MTX between 1 January 2015 and 31 March 2018. Outcomes: Adjusted rates of biologic initiation during follow-up were compared by logistic and Cox regressions. Results: APR initiators had 58% lower likelihood of biologic initiation (odds ratio: 0.42; 95% CI: 0.37-0.48; p < 0.001), lower adjusted biologic initiation rate (14.4% [95% CI: 13.2-15.7%] vs 28.6% [95% CI: 26.8-30.5%]), lower risk of biologic initiation (hazard ratio: 0.45; 95% CI: 0.40-0.51; p < 0.001) compared with MTX initiators. Conclusion: Systemic-naive patients with PsO have a lower rate of biologic initiation over 1 year following APR initiation.

An Adaptation of the RAND/UCLA Modified Delphi Panel Method in the Time of COVID-19.

The RAND/UCLA modified Delphi panel method is a formal group consensus process that systematically and quantitatively combines expert opinion and evidence by asking panelists to rate, discuss, then re-rate items. The method has been used to develop medical society guidelines, other clinical practice guidelines, disease classification systems, research agendas, and quality improvement interventions. Traditionally, a group of experts meet in person to discuss results of a first-round survey. After the meeting, experts complete a second-round survey used to develop areas of consensus. During the COVID-19 pandemic, this aspect of the method was not possible. As such, we have adapted the method to conduct virtual RAND/UCLA modified Delphi panels. In this study, we present a targeted literature review to describe and summarize the existing evidence on the RAND/UCLA modified Delphi panel method and outline our adaptation for conducting these panels virtually. Transitioning from in-person to virtual meetings was not without challenges, but there have also been unexpected advantages. The method we describe here can be a cost-effective and efficient alternative for researchers and clinicians.

Cost of cancer management by stage at diagnosis among Medicare beneficiaries.

OBJECTIVE: Estimate the annual cost of care in the 5 years following a cancer diagnosis for 17 invasive cancer types, by stage at diagnosis. METHODS: We used 2012-2016 data from the Surveillance, Epidemiology, and End Results (SEER) registry-Medicare claims database to examine cost of care among Medicare beneficiaries with a confirmed cancer diagnosis based on International Classification of Diseases for Oncology, Third Edition histology codes reported in SEER. Beneficiaries contributed to the annual cost calculations (Years 1-5) using their observed time after diagnosis. Beneficiaries were continuously enrolled in fee-for-service Medicare Parts A/B and Part D during follow-up. Total, inpatient, outpatient, and pharmacy cancer-related service costs were calculated. RESULTS: From 2012 to 2016, we identified 597,778 Medicare beneficiaries with incident cancer diagnosis within 5 years (Stage I, II, III, and IV: 32.6%, 33.4%, 15.9%, and 18.0%, respectively). In Year 1, mean (standard deviation) total costs for Stage I diagnoses varied from $7640 ($17,378) (prostate) to $94,636 ($117,636) (pancreas). Total costs increased by stage and reached $58,783 ($92,344) (prostate) to $156,982 ($175,009) (stomach) for Stage IV diagnoses in Year 1. Costs in Year 1 were significantly higher for Stage IV diagnoses than for earlier stages across all cancer types. In Years 2-5, total costs were lower than in Year 1 but continued to increase by stage. CONCLUSIONS: Beneficiaries diagnosed at later stages of cancer have higher costs of care (up to 7 times as much) than those diagnosed at earlier stages. Earlier cancer diagnosis may lead to more efficient treatment and decreased management cost.

Real-world impact of disease on functioning and activity: what is missed when using general instruments to estimate quality-adjusted life years.

OBJECTIVE: Economic evaluations conducted to inform healthcare resource allocation often rely on quality-adjusted life years (QALYs) to measure therapeutic benefit. However, QALYs, with underlying health utilities estimated using the EQ-5D or SF-36, may fail to capture the impact of disease for all patients. How well-being and heath utility differ across several common conditions was explored. METHODS: This study examined eight diseases: arthritis, asthma, cancer, depression, diabetes, heart disease, lung disease and stroke. Health utilities for each disease were obtained from published literature. Other measures of disease burden, including physical functioning, cognitive functioning and physical activity, were estimated from the National Health and Nutrition Examination Survey (NHANES). Group rankings by these measures were compared to rankings by health utility. RESULTS: Health utilities were lowest for patients with depression (0.44), and highest for those with cancer (0.81). Physical functioning was most limited (higher score) among those with stroke (28.2) and had the least impact for cancer (24.4). Physical activity was most impacted by heart disease (27.3) and least impacted by depression (40.7). Cognitive functioning was lowest in stroke (41.6) and highest in asthma (52.0). CONCLUSION: Differences in rankings of disease severity by metric indicate that the results of cost-utility analyses might be biased against treatments for certain diseases. As patient preferences for clinical outcomes vary, the full burden of disease should be considered in evaluations. Restricting access to treatments based on an incomplete estimate of burden could lead to misallocation of resources and a withholding of therapies that patients find valuable.