Commentary: Determining Economic Factors That Matter to People With Intellectual and Developmental Disabilities and Their Caregivers: A Process Framework.

BACKGROUND: The 2020-2029 strategic plan for the Patient-Centered Outcomes Research Trust Fund calls for addressing data infrastructure gaps that are critical for studying issues around intellectual and developmental disabilities (I/DD). Specifically, the plan calls for data collection on economic factors that affect person-centered approaches to health care decision-making. Among people with I/DD and their caregivers, such economic factors may include financial costs of care, decreased opportunities for leisure and recreation, income losses associated with caregiving, and foregone opportunities for skill acquisition or other human capital investments. OBJECTIVE: This commentary supports responsiveness to the Patient-Centered OutcomesResearch Trust Fund (PCORTF) calls by conceptualizing and operationalizing a framework for identifying preferences on economic factors that are relevant to people with I/DD and their caregivers. MAIN ARGUMENTS: The framework outlined in this commentary addresses barriers to data collection that hinder measure development in the study of I/DD. This work is significant and timely given the continued movement to integrate and maintain people with I/DD within communities and recent methodological advances for eliciting preferences among people with I/DD. RELEVANCE TO THE SPECIAL ISSUE: Readers will be introduced to a framework for building data capacity in the study of economic outcomes among a population that is a high research priority for federal funding agencies. This commentary aims to be useful to researchers in planning, developing, and initiating projects in this area.

Cost effectiveness of non-drug interventions that reduce nursing home admissions for people living with dementia.

INTRODUCTION: Six million Americans live with Alzheimer's disease and Alzheimer's disease and related dementias (AD/ADRD), a major health-care cost driver. We evaluated the cost effectiveness of non-pharmacologic interventions that reduce nursing home admissions for people living with AD/ADRD. METHODS: We used a person-level microsimulation to model the hazard ratios (HR) on nursing home admission for four evidence-based interventions compared to usual care: Maximizing Independence at Home (MIND), NYU Caregiver (NYU); Alzheimer's and Dementia Care (ADC); and Adult Day Service Plus (ADS Plus). We evaluated societal costs, quality-adjusted life years and incremental cost-effectiveness ratios. RESULTS: All four interventions cost less and are more effective (i.e., cost savings) than usual care from a societal perspective. Results did not materially change in 1-way, 2-way, structural, and probabilistic sensitivity analyses. CONCLUSION: Dementia-care interventions that reduce nursing home admissions save societal costs compared to usual care. Policies should incentivize providers and health systems to implement non-pharmacologic interventions.

Economic implications of FDA platelet bacterial guidance compliance options: Comparison of single-step strategies.

BACKGROUND: Bloodborne pathogens pose a major safety risk in transfusion medicine. To mitigate the risk of bacterial contamination in platelet units, FDA issues updated guidance materials on various bacterial risk control strategies (BRCS). This analysis presents results of a budget impact model updated to include 5- and 7-day pathogen reduced (PR) and large volumed delayed sampling (LVDS) BRCS. STUDY DESIGN AND METHODS: Model base-case parameter inputs were based on scientific literature, a survey distributed to 27 US hospitals, and transfusion experts' opinion. The outputs include hospital budget and shelf-life impacts for 5- and 7-day LVDS, and 5- and 7-day PR units under three different scenarios: (1) 100% LVDS, (2) 100% PR, and (3) mix of 50% LVDS - and 50% PR. RESULTS: Total annual costs from the hospital perspective were highest for 100% LVDS platelets (US$2.325M) and lowest for 100% PR-7 units (US$2.170M). Net budget impact after offsetting annual costs by outpatient reimbursements was 5.5% lower for 5-day PR platelets as compared to 5-day LVDS (US$1.663 vs. US$1.760M). A mix of 7-day LVDS and 5-day PR platelets had net annual costs that were 1.3% lower than for 100% 7-day LVDS, but 1.3% higher than for 100% 5-day PR. 7-day PR platelets had the longest shelf life (4.63 days), while 5-day LVDS had the shortest (2.00 days). DISCUSSION: The model identifies opportunities to minimize transfusion center costs for 5- and 7-day platelets. Budget impact models such as this are important for understanding the financial implications of evolving FDA guidance and new platelet technologies.

A longitudinal evaluation of family caregivers' willingness to pay for an in-home nonpharmacologic intervention for people living with dementia: results from a randomized trial.

OBJECTIVE: To determine the willingness-to-pay (WTP) of family caregivers to learn care strategies for persons living with dementia (PLwD). DESIGN: Randomized clinical trial. SETTING: Community-dwelling PLwD and their caregivers (dyads) in Maryland and Washington, DC. PARTICIPANTS: 250 dyads. INTERVENTION: Tailored Activity Program (TAP) compared to attention control. TAP provides activities tailored to the PLwD and instructs caregivers in their use. MEASUREMENT: At baseline, 3 and 6 months, caregivers were asked their WTP per session for an 8-session 3-month in-home nonpharmacologic intervention to address behavioral symptoms and functional dependence. RESULTS: At baseline, 3 and 6 months, caregivers assigned to TAP were willing to pay $26.10/session (95%CI:$20.42, $33.00), $28.70 (95%CI:$19.73, $39.30), and $22.79 (95%CI: $16.64, $30.09), respectively; attention control caregivers were willing to pay $37.90/session (95%CI: $27.10, $52.02), $30.92 (95%CI: $23.44, $40.94), $27.44 (95%CI: $20.82, $35.34), respectively. The difference in baseline to 3 and 6 months change in WTP between TAP and the attention control was $9.58 (95%CI: -$5.00, $25.47) and $7.15 (95%CI: -$5.72, $21.81). The difference between TAP and attention control in change in the proportion of caregivers willing to pay something from baseline to 3 and 6 months was -12% (95%CI: -28%, -5%) and -7% (95%CI:-25%, -11%), respectively. The difference in change in WTP, among caregivers willing to pay something, between TAP and attention control from baseline to 3 and 6 months was $17.93 (95%CI: $0.22, $38.30) and $11.81 (95%CI: -$2.57, $28.17). CONCLUSIONS: Family caregivers are willing to pay more for an intervention immediately following participation in a program similar to which they were asked to value.

Exploring beliefs about pneumococcal vaccination in a predominantly older African American population: the Pharmacists' Pneumonia Prevention Program (PPPP).

Objectives: To assess the association of the Pharmacists' Pneumonia Prevention Program (PPPP) with changes in beliefs related to pneumonia vaccination (PV) in a predominately older African American population.Methods: PPPP was an educational intervention delivered using a senior center model of care consisting of a formal pharmacist presentation, live skit, small group action planning, and optional PV. A 15-item instrument assessed participants' beliefs at baseline, post-test, and three months across four domains: pharmacists and pharmacies, vaccination, pneumococcal disease, and physicians. Friedman tests and pairwise Wilcoxon signed rank tests were used to determine the statistical significance of the mean change in belief responses across timepoints.Results: 190 older adults participated; the sample was majority female (76.3%) and African American (80.5%), and had a mean age of 74.3 years. Statistically significant improvements in beliefs at post-test were observed in the following domains: pharmacists and pharmacies, vaccination, and the pneumococcal disease; however, some of these gains were incompletely sustained at three months.Conclusion: PPPP positively impacted beliefs post-program regarding the pneumococcal disease, pharmacists and pharmacies, and vaccination; however, sustained efforts may be needed to reinforce these gains.Policy implications: Support for pharmacist educational services in senior centers should be considered.

Competencies for Professionals in Health Economics and Outcomes Research: The ISPOR Health Economics and Outcomes Research Competencies Framework.

The need for Health Economics and Outcomes Research (HEOR) has expanded globally, fueling demand for professionals trained in the discipline. By leveraging the expertise and perspectives of its members, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) established a set of competencies for HEOR professionals. The resulting 41 competencies were organized into 13 topic domains that collectively comprise the ISPOR Health Economics and Outcomes Research Competencies Framework. In this article, we explain the collaborative process used by the ISPOR Institutional Council and Faculty Advisor Council to identify and validate the framework. This process entailed expertise from the council members combined with natural language processing to examine competencies included in ISPOR Career Center HEOR job postings, qualitative input from a focused Institutional Council-Faculty Advisor Council workgroup, and quantitative input from 3 surveys of mutually exclusive member groups: a general member survey to assess importance and relevance of each competency, a faculty member survey to assess the extent to which HEOR graduate degree programs cover each of the competencies, and a student member survey to assess exposure to each of the competencies. Organization of the competencies into topic domains was achieved by engaging the Education Council, which applied a taxonomy consistent with ISPOR's educational programming. The resulting ISPOR Health Economics and Outcomes Research Competencies Framework has the important potential of serving as a tool to guide academic curricula, fellowships, and continuing education programs, and assessment of job candidates. As the HEOR field advances, so do the job types and the breadth of topics in which professionals must demonstrate competence. Future work will entail revisiting the competencies to ensure their currency and comprehensiveness, and tailoring the framework according to major specialty areas.

From Then Until Now: A Top-Down View Of The Affordable Care Act.

How does the Patient Protection and Affordable Care Act (ACA) of 2010 differ from the act as it stands today? Many changes have occurred, in Medicaid expansion, private insurance coverage, the American Health Benefit Exchanges, and the individual mandate.

Knowledge, Activation, and Costs of the Pharmacists' Pneumonia Prevention Program (PPPP): A Novel Senior Center Model to Promote Vaccination.

BACKGROUND: Vaccination is the best way to prevent pneumococcal disease (PD), but 40% of older adults remain unvaccinated nationwide, with even greater nonvaccination rates among African Americans (AAs). Prior studies suggest that insufficient knowledge contributes to low vaccination rates. The Pharmacists' Pneumonia Prevention Program (PPPP) was designed to improve older adults' knowledge about PD and pneumococcal vaccination (PV). OBJECTIVE: To measure PPPP's effect on knowledge and activation in a predominantly AA population and determine program costs. METHODS: PPPP uses a senior center model with a pharmacist presentation, actors' skit, and small-group action planning. Knowledge about PD risk, transmission, symptoms, and PV side effects was assessed at baseline (BL), postintervention (PT), and 3 months (M3) and analyzed using an intention-to-treat (ITT) approach. Actions taken (got vaccinated, spoke to doctor or pharmacist, discussed with family/friends) were assessed at M3. PPPP costs ($US 2013) included staff time, PV, actor, and site fees. RESULTS: Of 276 attending PPPP, 190 consented and were included in the ITT sample, which was largely black (80.5%) and female (76.3%) and had a mean age of 74.4 years. Knowledge improved by 46.8% (BL vs PT), with significant gains in all domains. At M3, knowledge improved by 54.2% vs BL, indicating sustained gains; 37.2% of previously unvaccinated participants reported receiving PV by M3. Program cost was $119 per attendee. CONCLUSION: PPPP significantly improved PD and PV knowledge. It could be delivered more efficiently by holding larger events on fewer dates, staffing with volunteers where appropriate, and utilizing a local pharmacy to manage the vaccine supply.

IMPACT OF PHYSICIAN COMMUNICATION ON DIABETIC EYE EXAMINATION ADHERENCE: Results From a Retrospective Cohort Analysis.

PURPOSE: To evaluate the effect of written communication between an ophthalmologist and a primary care physician (PCP) on patient adherence to diabetic eye examination recommendations. METHODS: In a retrospective cohort study of a multiethnic population at an urban ophthalmology center, records of all patients with diabetes and clinic visits between 2007 and 2010 were reviewed. Data collected included patient demographics, insurance status, hemoglobin A1C, severity of diabetic retinopathy, follow-up examinations, and written communication between a patient's ophthalmologist and PCP. Statistical analyses were performed to examine the relationship between physician communication and adherence to diabetic eye examination based on the American Academy of Ophthalmology-published recommendations. RESULTS: A total of 1,968 people with diabetes were included. Written communication from an ophthalmologist to a PCP was associated with increased adherence to follow-up eye examination recommendations (Odds Ratio: 1.49; 95% Confidence Interval: 1.16-1.92; P = 0.0018). Communication from a PCP to an ophthalmologist was also associated with increased adherence (Odds Ratio: 1.94; 95% Confidence Interval: 1.37-2.77; P = 0.0002). Multivariable analysis controlling for other factors associated with examination adherence confirmed that communication both to and from an ophthalmologist was independently and significantly associated with increased follow-up adherence. CONCLUSION: Patients with communication between ophthalmologists and PCPs are more likely to adhere to diabetic eye examinations.

Cost-effectiveness of allopurinol and febuxostat for the management of gout.

BACKGROUND: Gout is the most common inflammatory arthritis in the United States. OBJECTIVE: To evaluate the cost-effectiveness of urate-lowering treatment strategies for the management of gout. DESIGN: Markov model. DATA SOURCES: Published literature and expert opinion. TARGET POPULATION: Patients for whom allopurinol or febuxostat is a suitable initial urate-lowering treatment. TIME HORIZON: Lifetime. PERSPECTIVE: Health care payer. INTERVENTION: 5 urate-lowering treatment strategies were evaluated: no treatment; allopurinol- or febuxostat-only therapy; allopurinol-febuxostat sequential therapy; and febuxostat-allopurinol sequential therapy. Two dosing scenarios were investigated: fixed dose (80 mg of febuxostat daily, 0.80 success rate; 300 mg of allopurinol daily, 0.39 success rate) and dose escalation (≤120 mg of febuxostat daily, 0.82 success rate; ≤800 mg of allopurinol daily, 0.78 success rate). OUTCOME MEASURES: Discounted costs, discounted quality-adjusted life-years, and incremental cost-effectiveness ratios. RESULTS OF BASE-CASE ANALYSIS: In both dosing scenarios, allopurinol-only therapy was cost-saving. Dose-escalation allopurinol-febuxostat sequential therapy was more costly but more effective than dose-escalation allopurinol therapy, with an incremental cost-effectiveness ratio of $39 400 per quality-adjusted life-year. RESULTS OF SENSITIVITY ANALYSIS: The relative rankings of treatments did not change. Our results were relatively sensitive to several potential variations of model assumptions; however, the cost-effectiveness ratios of dose escalation with allopurinol-febuxostat sequential therapy remained lower than the willingness-to-pay threshold of $109 000 per quality-adjusted life-year. LIMITATION: Long-term outcome data for patients with gout, including medication adherence, are limited. CONCLUSION: Allopurinol single therapy is cost-saving compared with no treatment. Dose-escalation allopurinol-febuxostat sequential therapy is cost-effective compared with accepted willingness-to-pay thresholds. PRIMARY FUNDING SOURCE: Agency for Healthcare Research and Quality.

A home-based intervention to reduce depressive symptoms and improve quality of life in older African Americans: a randomized trial.

BACKGROUND: Effective care models for treating older African Americans with depressive symptoms are needed. OBJECTIVE: To determine whether a home-based intervention alleviates depressive symptoms and improves quality of life in older African Americans. DESIGN: Parallel, randomized trial stratified by recruitment site. Interviewers assessing outcomes were blinded to treatment assignment. (ClinicalTrials.gov: NCT00511680). SETTING: A senior center and participants' homes from 2008 to 2010. PATIENTS: African Americans aged 55 years or older with depressive symptoms. INTERVENTION: A multicomponent, home-based intervention delivered by social workers or a wait-list control group that received the intervention at 4 months. MEASUREMENTS: Self-reported depression severity at 4 months (primary outcome) and depression knowledge, quality of life, behavioral activation, anxiety, function, and remission at 4 and 8 months. RESULTS: Of 208 participants (106 and 102 in the intervention and wait-list groups, respectively), 182 (89 and 93, respectively) completed 4 months and 160 (79 and 81, respectively) completed 8 months. At 4 months, participants in the intervention group showed reduced depression severity (difference in mean change in Patient Health Questionnaire-9 score from baseline, -2.9 [95% CI, -4.6 to -1.2]; difference in mean change in Center for Epidemiologic Studies Depression Scale score from baseline, -3.7 [CI, -5.4 to -2.1]); improved depression knowledge, quality of life, behavioral activation, and anxiety (P < 0.001); and improved function (P = 0.014) compared with wait-list participants. More intervention than wait-list participants entered remission at 4 months (43.8% vs. 26.9%). After treatment, control participants showed benefits similar in magnitude to those of participants in the initial intervention group. Those in the initial intervention group maintained benefits at 8 months. LIMITATION: The study had a small sample, short duration, and differential withdrawal rate. CONCLUSION: A home-based intervention delivered by social workers could reduce depressive symptoms and enhance quality of life in most older African Americans. PRIMARY FUNDING SOURCE: National Institute of Mental Health.

Economic Analysis of the Tailored Activity Program: A Nonpharmacological Approach to Improve Quality of Life in People Living With Dementia and their Caregivers.

We investigated costs of delivering the Tailored Activity Program (TAP) and cost savings from two perspectives (health sector and societal) for people living with dementia (PLWD) and their caregivers (dyads) compared to attention control (AC) using data from a randomized controlled trial. The evaluation assessed intervention delivery costs and caregiver reported health care utilization. The total intervention cost of TAP was $1707/dyad versus $864/dyad for AC, and total costs over 6 months for TAP dyads as compared to AC were $1299 (CI: -$10,496, $7898) less from the healthcare perspective, and $761 (CI: -$10,133, $8611) less from the societal perspective. TAP cost savings are driven by lower use of healthcare services among participating dyads, but further analyses with larger samples is warranted to confirm its financial impact.

Addressing racial inequality and its effects on vaccination rate: A trial comparing a pharmacist and peer educational program (MOTIVATE) in diverse older adults.

BACKGROUND: The mortality, morbidity, health care utilization, and cost attributable to vaccine-preventable diseases are substantial for those aged 50 years and older. Although vaccination is the most cost-effective strategy to prevent common infectious diseases in older adults, vaccination rates remain below US Centers for Disease Control and Prevention benchmarks, especially among racial minorities. Historical mistrust, structural racism within the US medical system, and misinformation contributed to lower immunization rates among minorities, especially Black Americans. To address the critical need to increase knowledge and trust in vaccination, 2 community-based educational interventions were tested: a pharmacist-led didactic session (PHARM) and a peer-led educational workshop (PEER). OBJECTIVE: To determine and compare the effectiveness and costs of PEER and PHARM community-based education models in improving knowledge and trust in vaccinations. METHODS: The Motivating Older adults to Trust Information about Vaccines And Their Effects (MOTIVATE) study was a cluster-randomized trial conducted in the greater Delaware Valley Region sites from 2017 to 2020. The included sites (7 senior centers, 3 housing units, 1 church, and 1 neighborhood family center) predominantly served Black communities. Participants were randomized to either PHARM or PEER sessions covering influenza, pneumococcal disease, herpes zoster, and beliefs related to vaccines. Peer leaders facilitated smaller workshops (5-10 participants), whereas pharmacists conducted larger didactic lectures with 15-43 participants. Outcomes were captured through a self-administered survey at baseline, postprogram, and 1 month after the program. Intervention costs were measured in 2017 US dollars. RESULTS: 287 participants were included. Their mean age was 74.5 years (SD = 8.94), 80.5% were women, 64.2% were Black, and 48.1% completed some college. Knowledge scores within groups for all 3 diseases significantly increased postprogram for both PEER and PHARM and were sustained at 1 month. Between-group knowledge differences were significant only for influenza (PEER participants had significantly larger improvement vs PHARM). Vaccination trust significantly increased in both groups. Total program costs were $11,411 for PEER and $5,104 for PHARM. CONCLUSIONS: Both interventions significantly improved knowledge and trust toward vaccination and retained their effect 1 month after the program. The 2 effective community-based education models should be expanded to ensure timely and trusted information is available to educate older adults about vaccine-preventable diseases. Further research is encouraged to assess the long-term cost-effectiveness of these models' utilization on a larger scale. DISCLOSURES: Dr Schafer is an employee of Merck; however, at the time of the project, he was a professor at Thomas Jefferson University. The other authors have no conflicts of interest to disclose. This study was supported in part by a research grant from the Investigator-Initiated Studies Program of Merck Sharp & Dohme Corp. The opinions expressed in this article are those of the authors and do not necessarily represent those of Merck Sharp & Dohme Corp. The sponsor played no role in the design and conduct of the study; in the collection, analysis, and interpretation of the data; or in the preparation, review, or approval of the article. Study Registration Number: NCT03239665.

Manhattan Vision Screening and Follow-up Study (NYC-SIGHT): Baseline Results and Costs of a Cluster-Randomized Trial.

PURPOSE: To describe the 15-month baseline results and costs of the Manhattan Vision Screening and Follow-up Study, which aims to investigate whether innovative community-based eye health screening can improve early detection and management of glaucoma and other eye diseases among high-risk populations. DESIGN: Five-year prospective, cluster-randomized controlled trial. METHODS: Individuals aged 40+ years were recruited from public housing buildings in New York City for an eye health screening (visual acuity (VA) with correction, intraocular pressure measurements (IOP), and fundus photography). Participants with VA 20/40 or worse, IOP 23-29 mm Hg, or an unreadable fundus image failed the screening and were scheduled for an optometric examination at the same location; those with an abnormal image were referred to ophthalmology. A cost analysis was conducted alongside the study. RESULTS: A total of 708 participants were screened; mean age 68.6±11.9 years, female (65.1%), African American (51.8%) and Hispanic (42%). 78.4% (n = 555) failed the eye health screening; 35% (n= 250) had an abnormal image and were also referred to ophthalmology. 308 participants attended the optometric exam; 218 were referred to ophthalmology. Overall, 66.1% were referred to ophthalmology. The cost per participant to deliver the eye health screening and optometric examination was $180.88. The cost per case of eye disease detected was $273.64. CONCLUSIONS: This innovative study in public housing developments targeted high-risk populations, provided access to eye-care, and improved early detection of ocular diseases in New York City. The study has identified strategies to overcoming barriers to eye care to reduce eye health disparities.

Comparative effectiveness research for prostate cancer radiation therapy: current status and future directions.

Comparative effectiveness research aims to help clinicians, patients and policymakers make informed treatment decisions under real-world conditions. Prostate cancer patients have multiple treatment options, including active surveillance, androgen deprivation therapy, surgery and multiple modalities of radiation therapy. Technological innovations in radiation therapy for prostate cancer have been rapidly adopted into clinical practice despite relatively limited evidence for effectiveness showing the benefit for one modality over another. Comparative effectiveness research has become an essential component of prostate cancer research to help define the benefits, risks and effectiveness of the different radiation therapy modalities currently in use for prostate cancer treatment.

A community-integrated home based depression intervention for older African Americans: [corrected] description of the Beat the Blues randomized trial and intervention costs.

BACKGROUND: Primary care is the principle setting for depression treatment; yet many older African Americans in the United States fail to report depressive symptoms or receive the recommended standard of care. Older African Americans are at high risk for depression due to elevated rates of chronic illness, disability and socioeconomic distress. There is an urgent need to develop and test new depression treatments that resonate with minority populations that are hard-to-reach and underserved and to evaluate their cost and cost-effectiveness. METHODS/DESIGN: Beat the Blues (BTB) is a single-blind parallel randomized trial to assess efficacy of a non-pharmacological intervention to reduce depressive symptoms and improve quality of life in 208 African Americans 55+ years old. It involves a collaboration with a senior center whose care management staff screen for depressive symptoms (telephone or in-person) using the Patient Health Questionnaire (PHQ-9). Individuals screened positive (PHQ-9 ≥ 5) on two separate occasions over 2 weeks are referred to local mental health resources and BTB. Interested and eligible participants who consent receive a baseline home interview and then are randomly assigned to receive BTB immediately or 4 months later (wait-list control). All participants are interviewed at 4 (main study endpoint) and 8 months at home by assessors masked to study assignment. Licensed senior center social workers trained in BTB meet with participants at home for up to 10 sessions over 4 months to assess care needs, make referrals/linkages, provide depression education, instruct in stress reduction techniques, and use behavioral activation to identify goals and steps to achieve them. Key outcomes include reduced depressive symptoms (primary), reduced anxiety and functional disability, improved quality of life, and enhanced depression knowledge and behavioral activation (secondary). Fidelity is enhanced through procedure manuals and staff training and monitored by face-to-face supervision and review of taped sessions. Cost and cost effectiveness is being evaluated. DISCUSSION: BTB is designed to bridge gaps in mental health service access and treatments for older African Americans. Treatment components are tailored to specific care needs, depression knowledge, preference for stress reduction techniques, and personal activity goals. Total costs are $584.64/4 months; or $146.16 per participant/per month. TRIAL REGISTRATION: ClinicalTrials.gov #NCT00511680.

Payer decision-making with limited comparative and cost effectiveness data: the case of new pharmacological treatments for gout.

CONTEXT: The need for comparative effectiveness (CE) data continues to grow, fuelled by market demand as well as health reform. There may be an assumption that new drugs result in improved efficacy compared with the standard of care, therefore warranting premium prices. Gout treatment has recently become controversial, as expensive new drugs enter the market with limited CE data. METHODS: The authors reviewed published clinical trials and conducted a cost effectiveness analysis on a new drug (febuxostat) versus the standard (allopurinol) to illustrate the limitations in using these data to inform evidence-based decision-making. FINDINGS: Although febuxostat trials included allopurinol as a comparator, methodological limitations make comparative effectiveness evaluations difficult. However, when available trial data were input to a decision analytic model, the authors found that a significant reduction in febuxostat cost would be required in order for it to dominate allopurinol in cost effectiveness analysis. This case exemplifies the challenges of using clinical trial data in comparative and cost effectiveness analyses.