Cost-Effectiveness Analysis of a Prescription Digital Therapeutic in Type 2 Diabetes.

INTRODUCTION: BT-001 (AspyreRx™) prescription digital therapy, a form of personalized cognitive behavioral therapy, has demonstrated clinically meaningful and durable hemoglobin A1c reductions in patients with type 2 diabetes (T2D). The current study examined the cost-effectiveness of BT-001 plus standard of care (SoC) versus SoC alone in T2D over a lifetime horizon from a healthcare payer perspective. METHODS: We modeled the T2D pathway using an individual patient-level simulation; clinical data were sourced from the intention-to-treat subset of the BT-001 randomized clinical trial (RCT). SoC across both arms included the composition of oral and injectable treatments for T2D. Events were simulated using the United Kingdom Prospective Diabetes Study Outcomes Model 2 risk equation. A 3-month model cycle length was used in the first year, then annual model cycles were used in line with the original risk engine specifications. Patient characteristics informed event equations and Monte Carlo random sampling was used to assess the occurrence of events within each model cycle. Incidence of hypoglycemic events, drug discontinuation, costs, and health utilities and disutility values were sourced from the literature. RESULTS: From a payer perspective, BT-001 plus SoC versus SoC alone was dominant with a gain in quality-adjusted life years (QALYs) of 0.101 and cost savings of $7343 per patient over the lifetime horizon (i.e., more effective and less costly). BT-001 plus SoC was cost-effective at a willingness-to-pay of $100,000 per QALY (incremental net monetary benefit was $17,443). Savings with BT-001 were primarily driven by a reduction in drug acquisition costs. The reduction in hemoglobin A1c with BT-001 was associated with fewer T2D complications. CONCLUSIONS: BT-001 plus SoC was estimated to dominate SoC alone over the lifetime horizon from a payer perspective, suggesting that using BT-001 can empower patients to better manage their diabetes with the potential for lifelong advantages.

Costs in the Year Following Deceased Donor Kidney Transplantation: Relationships With Renal Function and Graft Failure.

Relationships between renal function and medical costs for deceased donor kidney transplant recipients are not fully quantified post-transplant. We describe these relationships with renal function measured by estimated glomerular filtration rate (eGFR) and graft failure. The United States Renal Data System identified adults receiving single-organ deceased donor kidneys 2012-2015. Inpatient, outpatient, other facility costs and eGFRs at discharge, 6 and 12 months were included. A time-history of costs was constructed for graft failures and monthly costs in the first year post-transplant were compared to those without failure. The cohort of 24,021 deceased donor recipients had a 2.4% graft failure rate in the first year. Total medical costs exhibit strong trends with eGFR. Recipients with 6-month eGFRs of 30-59 ml/min/1.73m2 have total costs 48% lower than those <30 ml/min/1.73m2. For recipients with graft failure monthly costs begin to rise 3-4 months prior to failure, with incremental costs of over $38,000 during the month of failure. Mean annual total incremental costs of graft failure are over $150,000. Total costs post-transplant are strongly correlated with eGFR. Graft failure in the first year is an expensive, months-long process. Further reductions in early graft failures could yield significant human and economic benefits.

Clinical Events and Renal Function in the First Year Predict Long-Term Kidney Transplant Survival.

Background: Estimated glomerular filtration rate (eGFR) at 1 year post transplantation has been shown to be a strong predictor of long-term graft survival. However, intercurrent events (ICEs) may affect the relationship between eGFR and failure risk. Methods: The OPTN and USRDS databases on single-organ kidney transplant recipients from 2012 to 2016 were linked. Competing risk regressions estimated adjusted subhazard ratios (SHRs) of 12-month eGFR on long-term graft failure, considering all-cause mortality as the competing risk, for deceased donor (DD) and living donor (LD) recipients. Additional predictors included recipient, donor, and transplant characteristics. ICEs examined were acute rejection, cardiovascular events, and infections. Results: Cohorts comprised 25,131 DD recipients and 7471 LD recipients. SHRs for graft failure increased rapidly as 12-month eGFR values decreased from the reference 60 ml/min per 1.73 m2. At an eGFR of 20 ml/min per 1.73 m2, SHRs were 13-15 for DD recipients and 12-13 for LD recipients; at an eGFR of 30 ml/min per 1.73 m2, SHRs were 5.0-5.7 and 5.0-5.5, respectively. Among first-year ICEs, acute rejection was a significant predictor of long-term graft failure in both DD (SHR=1.63, P<0.001) and LD (SHR=1.51, P=0.006) recipients; cardiovascular events were significant in DD (SHR=1.24, P<0.001), whereas non-CMV infections were significant in the LD cohort (SHR=1.32, P=0.03). Adjustment for ICEs did not significantly reduce the association of eGFR with graft failure. Conclusions: Twelve-month eGFR is a strong predictor of long-term graft failure after accounting for clinical events occurring from discharge to 1 year. These findings may improve patient management and clinical evaluation of novel interventions.

Patient-reported outcomes in hemodialysis vascular access: A call to action.

While access-related dysfunction is a clear driver of clinical outcomes and costs, the full impact of vascular access dysfunction on patient experience and quality of life is not fully characterized in the literature. One way to more comprehensively characterize the patient experience from the patient perspective is through patient reported outcomes (PROs). However, the limited implementation of PROs in clinical trials, patient registries, quality measurement, and other research settings has significantly constrained the patient voice in evaluation of vascular access outcomes and vascular access decision-making. To address these issues, the Kidney Health Initiative, a public-private partnership between the American Society of Nephrology and the U.S. Food and Drug Administration, assembled an interdisciplinary workgroup to enhance uptake of access-related PROs with the aims of: (1) reviewing the domains of HRQOL that are affected by vascular access, collect information on existing instruments that measure access-specific HRQOL in hemodialysis, and identify gaps in existing measures; (2) identifying and critically assessing barriers to widespread use of access-specific PRO measures; and (3) defining initiatives to overcome barriers and make recommendations for strategies to improve the use and utility of access-specific PRO measures. A consensus group process identified potential barriers to use of PRO measures in six categories: (1) PRO misperceptions, (2) patient factors, (3) regulators and payers, (4) instrument factors, (5) study design, and (6) physicians. The workgroup provided recommendations for actions to promote the widespread utilization of vascular access-related PRO measures in five categories: (1) development of vascular access-specific PRO measures, (2) ensuring comprehensive assessment when using vascular access PRO measures, (3) ensuring accessibility and applicability of vascular access PRO measures to all end stage kidney disease populations, (4) establishing universal guidelines and accepted vascular access PRO measures, and (5) engaging stakeholders across all facets.

Defining a minimal clinically meaningful difference in 12-month estimated glomerular filtration rate for clinical trials in deceased donor kidney transplantation.

BACKGROUND: A Minimal Clinically Meaningful Difference (MCMD) has not been defined for Estimated glomerular filtration rate (eGFR). Our goal was to define the MCMD for eGFR anchored to kidney graft failure. METHODS: A systematic review of studies with 12-month eGFR and subsequent renal graft failure was conducted. For observational studies, we calculated hazard ratio (HR) differences between adjacent eGFR intervals weighted by population distribution. Interventional trials yielded therapeutically induced changes in eGFR and failure risk. OPTN data analysis divided 12-month eGFR into bands for Cox regressions comparing adjacent eGFR bands with a death-censored graft survival outcome. RESULTS: Observational studies indicated that lower eGFR was associated with increased death-censored graft failure risk; each 5 ml/min/1.73 m2 12-month eGFR band associated with a weighted incremental HR = 1.12 to 1.23. Clinical trial data found a 5 ml/min/1.73 m2 difference was associated with incremental HR = 1.16 to 1.35. OPTN analyses showed weighted mean HRs across 10, 7, and 5 ml/min/1.73 m2 bands of 1.47, 1.30, and 1.19. CONCLUSIONS: A 5 ml/min/1.73 m2 difference in 12-month eGFR was consistently associated with ~20% increase in death-censored graft failure risk. The magnitude of effect has been interpreted as clinically meaningful in other disease states and should be considered the MCMD in renal transplantation clinical trials.

Healthcare resource utilization among commercially insured patients with cold agglutinin disease in the United States.

Aims: Cold agglutinin disease (CAD) is a rare subtype of autoimmune hemolytic anemia associated with increased thromboembolism risk and early mortality. Healthcare resource utilization (HRU) in CAD has not been reported. We aimed to compare HRU of patients with CAD with a matched non-CAD cohort in the United States.Materials and methods: Patients with CAD were identified from 2006 to 2016 in the Optum-Humedica database using CAD terms in clinical notes and hematologist review. Patients were required to have Integrated Delivery Network records and ≥6 months' follow-up before and after the first CAD mention date (index date). Patients with CAD were matched to a non-CAD cohort based on demographics. Multivariate analyses assessed inpatient hospitalizations, outpatient visits, emergency room visits, and transfusion use between cohorts 6 months before and 12 months after the index date.Results: Of 814 patients with CAD, 410 met inclusion criteria and were matched to 3,390 patients without CAD. Mean age of patients with CAD was 68.0 years; approximately 62% were female. In the 12 months after the index date, mean inpatient hospitalizations (0.83 vs. 0.25), outpatient visits (17.26 vs. 6.77), emergency room visits (0.55 vs. 0.32), and transfusion days (1.05 vs. 0.05) were higher for patients with CAD than the matched non-CAD cohort (all p < .0001). Similarly, in the 6 months before the index date, patients with CAD had higher HRU than matched patients without CAD for all measures evaluated.Limitations: Results of this study are based on patient information from the Optum-Humedica database, which is limited to commercially insured patients and may not represent the overall CAD population.Conclusions: CAD places a substantial burden on patients and healthcare systems. In addition, the high HRU for patients with CAD observed in the 6 months before diagnosis indicates that disease awareness and better diagnostic practices may be needed.

Toward Patient-Centered Innovation: A Conceptual Framework for Patient-Reported Outcome Measures for Transformative Kidney Replacement Devices.

Individuals with dialysis-dependent kidney failure experience considerable disease- and treatment-related decline in functional status and overall well-being. Despite these experiences, there have been few substantive technological advances in KRT in decades. As such, new federal initiatives seek to accelerate innovation. Historically, integration of patient perspectives into KRT product development has been limited. However, the US Food and Drug Administration recognizes the importance of incorporating patient perspectives into the total product life cycle (i.e., from product conception to postmarket surveillance) and encourages the consideration of patient-reported outcomes in regulatory-focused clinical trials when appropriate. Recognizing the significance of identifying patient-reported outcome measures (PROMs) that capture contemporary patient priorities, the Kidney Health Initiative, a public-private partnership between the American Society of Nephrology and US Food and Drug Administration, convened a workgroup to (1) develop a conceptual framework for a health-related quality of life PROM; (2) identify and map existing PROMs to the conceptual framework, prioritizing them on the basis of their supporting evidence for use in the regulatory environment; and (3) describe next steps for identifying PROMs for use in regulatory clinical trials of transformative KRT devices. This paper summarizes the proposed health-related quality-of-life PROM conceptual framework, maps and prioritizes PROMs, and identifies gaps and future needs to advance the development of rigorous, meaningful PROMS for use in clinical trials of transformative KRT devices.

Angiotensin II for the treatment of distributive shock in the intensive care unit: A US cost-effectiveness analysis.

BACKGROUND: Patients with distributive shock who are unresponsive to traditional vasopressors are commonly considered to have severe distributive shock and are at high mortality risk. Here, we assess the cost-effectiveness of adding angiotensin II to the standard of care (SOC) for severe distributive shock in the US critical care setting from a US payer perspective. METHODS: Short-term mortality outcomes were based on 28-day survival rates from the ATHOS-3 study. Long-term outcomes were extrapolated to lifetime survival using individually estimated life expectancies for survivors. Resource use and adverse event costs were drawn from the published literature. Health outcomes evaluated were lives saved, life-years gained, and quality-adjusted life-years (QALYs) gained using utility estimates for the US adult population weighted for sepsis mortality. Deterministic and probabilistic sensitivity analyses assessed uncertainty around results. We analyzed patients with severe distributive shock from the ATHOS-3 clinical trial. RESULTS: The addition of angiotensin II to the SOC saved .08 lives at Day 28 compared to SOC alone. The cost per life saved was estimated to be $108,884. The addition of angiotensin II to the SOC was projected to result in a gain of .96 life-years and .66 QALYs. This resulted in an incremental cost-effectiveness ratio of $12,843 per QALY. The probability of angiotensin II being cost-effective at a threshold of $50,000 per QALY was 86 percent. CONCLUSIONS: For treatment of severe distributive shock, angiotensin II is cost-effective at acceptable thresholds.

Vascular access-specific health-related quality of life impacts among hemodialysis patients: qualitative development of the hemodialysis access-related quality of life (HARQ) instrument.

BACKGROUND: End stage kidney disease and hemodialysis dependence are associated with impairments in health-related quality of life (HRQOL), which may be related to vascular access (VA). Few HRQOL measures are VA-specific and none differentiate HRQOL impact by VA type. We developed a VA-targeted HRQOL measure to distinguish the impact of fistulas, grafts and catheters. METHODS: We created an initial item pool based on literature review and then conducted focus groups at 4 US sites with 37 adults and interviews with nine dialysis clinicians about VA's impact on HRQOL. We then drafted the Hemodialysis Access-Related Quality of Life (HARQ) measure and cognitively tested it with 17 hemodialysis patients. Focus group and cognitive interview participants were diverse in age, gender, years on dialysis, and VA. RESULTS: We identified six domains for the HARQ: symptoms, physical functioning, emotional impacts, social and role functioning, sleep, and care-related burdens. Cognitive interviews indicated that items were easily understood and supported content validity. Attributing HRQOL impact to VA as opposed to other hemodialysis burden was challenging for some items. Some items were dropped that were considered redundant by patients, limitations while dressing was added, and reference to VA-specific impact was included for each item. The average Flesch-Kincaid reading grade level for the revised 47-item HARQ was 5.3. CONCLUSIONS: The HARQ features VA-specific content not addressed in other HRQOL measures, making it ideal for comparisons of different VA types and new VA technologies. The psychometric properties of the HARQ will be evaluated in future research.

Patency of ePTFE Arteriovenous Graft Placements in Hemodialysis Patients: Systematic Literature Review and Meta-Analysis.

Arteriovenous grafts (AVGs) are an appropriate option for vascular access in certain hemodialysis patients. Expanded polytetrafluoroethylene (ePTFE) has become the dominant material for such grafts, due in part to innovations in graft design and surgical interventions to reduce complications and improve patency rates. Comprehensive evidence syntheses have not been conducted to update AVG performance in an era in which both access choice and ePTFE graft functioning may have changed. We conducted a systematic review and meta-analysis summarizing outcomes from recent studies of ePTFE AVGs in hemodialysis, following PRISMA standards. Literature searches were conducted in multiple databases to identify observational and interventional studies of AVG patency and infection risk. Primary, primary-assisted, and secondary patency rates were analyzed at 6, 12, 18, and 24 months postplacement. Kaplan-Meier graft survival plots were digitized to recreate individual patient-level data. Patency rates were pooled using a random effects model. We identified 32 studies meeting our selection criteria that were published from 2004 through 2019. A total of 38 study arms of ePTFE grafts were included, representing 3381 AVG accesses placed. The mean primary, primary-assisted, and secondary patency rates at 1 year were 41% (95% CI, 35% to 47%), 46% (95% CI, 41% to 51%), and 70% (95% CI, 64% to 75%), respectively. Mean 24-month patency rates were 28% (95% CI, 22% to 33%), 34% (95% CI, 27% to 41%), and 54% (95% CI, 47% to 61%), respectively. A high degree of heterogeneity across studies was observed. Overall risk of infection was not consistently reported, but among available studies the pooled estimate was 9% per patient-year (95% CI, 6% to 12%). This meta-analysis provides an up-to-date estimate of the performance of ePTFE AVGs, within the context of improved graft designs and improved interventional techniques.

Estimating the Impact of Novel Digital Therapeutics in Type 2 Diabetes and Hypertension: Health Economic Analysis.

BACKGROUND: Behavioral interventions can meaningfully improve cardiometabolic conditions. Digital therapeutics (DTxs) delivering these interventions may provide benefits comparable to pharmacologic therapies, displacing medications for some patients. OBJECTIVE: Our objective was to estimate the economic impact of a digital behavioral intervention in type 2 diabetes mellitus (T2DM) and hypertension (HTN) and estimate the impact of clinical inertia on deprescribing medications. METHODS: Decision analytic models estimated health resource savings and cost effectiveness from a US commercial payer perspective. A 3-year time horizon was most relevant to the intervention and payer. Effectiveness of the DTx in improving clinical outcomes was based on cohort studies and published literature. Health resource utilization (HRU), health state utilities, and costs were drawn from the literature with costs adjusted to 2018 dollars. Future costs and quality-adjusted life years (QALYs) were discounted at 3%. Sensitivity analyses assessed uncertainty. RESULTS: Average HRU savings ranged from $97 to $145 per patient per month, with higher potential benefits in T2DM. Cost-effectiveness acceptability analyses using a willingness-to-pay of $50,000/QALY indicated that the intervention would be cost effective at total 3-year program costs of $6468 and $6620 for T2DM and HTN, respectively. Sensitivity analyses showed that reduced medication costs are a primary driver of potential HRU savings, and the results were robust within values tested. A resistance to deprescribe medications when a patient's clinical outcomes improve can substantially reduce the estimated economic benefits. Our models rely on estimates of clinical effectiveness drawn from limited cohort studies with DTxs and cannot account for other disease management programs that may be implemented. Performance of DTxs in real-world settings is required to further validate their economic benefits. CONCLUSIONS: The DTxs studied may provide substantial cost savings, in part by reducing the use of conventional medications. Clinical inertia may limit the full cost savings of DTxs.

Costs Attributable to Arteriovenous Fistula and Arteriovenous Graft Placements in Hemodialysis Patients with Medicare coverage.

INTRODUCTION: Hemodialysis (HD) in end-stage renal disease (ESRD) patients requires vascular access (VA) through an arteriovenous fistula (AVF), a prosthetic arteriovenous graft (AVG), or a central venous catheter. While AVF or AVG is commonly used for HD, the economic implications of AVF versus AVG use have not been fully established. We describe the healthcare resource utilization and costs of AVF and AVG use for incident ESRD patients in the United States. METHODS: This observational cohort study of AVF and AVG placements used data from the United States Renal Data System to identify and follow access placements. AVF and AVG placements after ESRD onset for incident patients from 2012 to 2014 with continuous Medicare primary coverage were included. All-cause and access-related Medicare costs were averaged over the placement lifetime and expressed as per dialysis-month costs. RESULTS: The analysis included 38,035 AVF placements and 12,789 AVG placements. Total all-cause monthly costs for AVF averaged USD 8,508; mean monthly costs were USD 3,027 for inpatient (IP), USD 3,139 for outpatient (OP), USD 1,572 for physician services, and USD 770 for other care settings. Access-related monthly costs averaged USD 1,699 and represented 20% of all-cause charges for AVFs. Mean all-cause monthly costs for AVG were USD 9,605; by setting monthly costs were USD 3,811 for IP, USD 3,034 for OP, USD 1,881 for physician services and USD 879 for other care settings. Access-related monthly costs averaged USD 2,656 and represented 28% of all-cause charges for AVGs. DISCUSSION/CONCLUSIONS: This study indicates that costs due to VA are a significant burden on Medicare budgets and on patients. The factors driving access-related utilization and costs merit attention in future research. Both optimizing process of care and discovery innovation may significantly accelerate better stewardship of available healthcare resources.

Clinical Outcomes of Arteriovenous Access in Incident Hemodialysis Patients with Medicare Coverage, 2012-2014.

BACKGROUND: Chronic hemodialysis requires a mode of vascular access through an arteriovenous fistula (AVF), a prosthetic arteriovenous graft (AVG), or a central venous catheter (CVC). AVF is recommended over AVG or CVC due to increased patency and decreased intervention rates for those that mature. AVG are preferred over CVC due to decreased infection and mortality risk. The aims of this study were to evaluate the lifespan of AVF and AVG in maturation, sustained access use, and abandonment. METHODS: The United States Renal Data System (USRDS), Medicare claims, and CROWNWeb were used to identify access placements. Patients with a first end-stage renal disease (ESRD) service from January 1, 2012 to June 30, 2014 with continuous coverage with Medicare as primary payer and ≥1 AVF or AVG placed after ESRD onset were included. Maturation was defined as the first use of the access for hemodialysis recorded in CROWNWeb. Sustained access use was defined as 3 consecutive months of use without catheter placement or replacement. Accesses that were never used at any time post-placement were considered abandoned. RESULTS: The cohort included 38,035 AVF placements and 12,789 AVG placements. Sixty-nine percent of AVF and 72% of AVG matured. Fifty-two percent of AVF and 51% of AVG achieved sustained access use. One quarter of AVF and 14% of AVG were abandoned without use as recorded in CROWNWeb. CONCLUSION: Although considered the gold standard for vascular access, only half of AVF and AVG placements achieved sustained access use. The USRDS database has inherent limitations but provides useful clinical insight into maturation, sustained use, and abandonment.

Patient-level costs of major cardiovascular conditions: a review of the international literature.

OBJECTIVE: Robust cost estimates of cardiovascular (CV) events are required for assessing health care interventions aimed at reducing the economic burden of major adverse CV events. This review synthesizes international cost estimates of CV events. METHODS: MEDLINE database was searched electronically for English language studies published during 2007-2012, with cost estimates for CV events of interest - unstable angina, myocardial infarction, heart failure, stroke, and CV revascularization. Included studies provided at least one estimate of patient-level direct costs in adults for any identified country. Information on study characteristics and cost estimates were collected. All costs were adjusted for inflation to 2013 values. RESULTS: Across the 114 studies included, the average cost was US $6,466 for unstable angina, $11,664 for acute myocardial infarction, $11,686 for acute heart failure, $11,635 for acute ischemic stroke, $37,611 for coronary artery bypass graft, and $13,501 for percutaneous coronary intervention. The ranges for cost estimates varied widely across countries with US cost estimate being at least twice as high as European Union costs for some conditions. Few studies were found on populations outside the US and European Union. CONCLUSION: This review showed wide variation in the cost of CV events within and across countries, while showcasing the continuing economic burden of CV disease. The variability in costs was primarily attributable to differences in study population, costing methodologies, and reporting differences. Reliable cost estimates for assessing economic value of interventions in CV disease are needed.

Systematic Review of Low-Density Lipoprotein Cholesterol Apheresis for the Treatment of Familial Hypercholesterolemia.

BACKGROUND: Apheresis is an important treatment for reducing low-density lipoprotein cholesterol (LDL-C) in patients with familial hypercholesterolemia (FH). We systematically reviewed the current literature surrounding LDL-C apheresis for FH. METHODS AND RESULTS: Electronic databases were searched for publications of LDL-C apheresis in patients with FH. Inclusion criteria include articles in English published in 2000-2013 that provide descriptions of practice patterns, efficacy/effectiveness, and costs related to LDL-C apheresis in patients with FH. Data were stratified by country and FH genotype where possible. Thirty-eight studies met the inclusion criteria: 8 open-label clinical trials, 11 observational studies, 17 reviews/guidelines, and 2 health technology assessments. The prevalence of FH was not well characterized by country, and underdiagnosis was a barrier to FH treatment. Treatment guidelines varied by country, with some guidelines recommending LDL-C apheresis as first-line treatment in patients with homozygous FH and after drug therapy failure in patients with heterozygous FH. Additionally, guidelines typically recommended weekly or biweekly LDL-C apheresis treatments conducted at apheresis centers that may last 2 to >3 hours per session. Studies reported a range for mean LDL-C reduction after apheresis: 57-75% for patients with homozygous FH and 58-63% for patients with heterozygous FH. Calculated annual costs (in US$2015) may reach US$66 374 to US$228 956 per patient for weekly treatment. CONCLUSIONS: LDL-C apheresis treatment may be necessary for patients with FH when drug therapy is inadequate in reducing LDL-C to target levels. While apheresis reduces LDL-C, high per-session costs and the frequency of guideline-recommended treatment result in substantial annual costs, which are barriers to the optimal treatment of FH.

US Payer Perspectives on Evidence for Formulary Decision Making.

PURPOSE: The perspective of commercial payers on comparative effectiveness research (CER) has not been well researched. This study aims to describe how US commercial payers use and value CER for formulary decision making in different disease states. METHODS: We recruited 20 medical and pharmaceutical directors from national and regional plans who are involved in pharmaceutical and therapeutics committees to participate in the study. We conducted in-depth qualitative interviews with the payers and asked them to rate the usefulness of CER study types across various disease states and market conditions. The results were analyzed for thematic content. RESULTS: Our findings indicate that payers are interested in a broad range of CER study types, are unsatisfied with the current state of CER, and would like to partner with research groups to develop research and treatment guidelines to better leverage CER. Payers value CER less so in oncology than in other disease states because of limitations in their ability to manage oncology therapies. CONCLUSION: To improve formulary design processes and support payers in providing more effective health care, policy makers should consider involving commercial payers in the development of CER as well as in the creation of research and treatment guidelines.

US payer perspectives on evidence for formulary decision making.

PURPOSE: The perspective of commercial payers on comparative effectiveness research (CER) has not been well researched. This study aims to describe how US commercial payers use and value CER for formulary decision making in different disease states. METHODS: We recruited 20 medical and pharmaceutical directors from national and regional plans who are involved in pharmaceutical and therapeutics committees to participate in the study. We conducted in-depth qualitative interviews with the payers and asked them to rate the usefulness of CER study types across various disease states and market conditions. The results were analyzed for thematic content. RESULTS: Our findings indicate that payers are interested in a broad range of CER study types, are unsatisfied with the current state of CER, and would like to partner with research groups to develop research and treatment guidelines to better leverage CER. Payers value CER less in oncology than in other disease states because of limitations in their ability to manage oncology therapies. CONCLUSION: To improve formulary design processes and support payers in providing more effective healthcare, policy makers should consider involving commercial payers in the development of CER as well as in the creation of research and treatment guidelines.

Use of electronic medical records (EMR) for oncology outcomes research: assessing the comparability of EMR information to patient registry and health claims data.

UNLABELLED: Electronic medical records (EMRs) are used increasingly for research in clinical oncology, epidemiology, and comparative effectiveness research (CER). OBJECTIVE: To assess the utility of using EMR data in population-based cancer research by comparing a database of EMRs from community oncology clinics against Surveillance Epidemiology and End Results (SEER) cancer registry data and two claims databases (Medicare and commercial claims). STUDY DESIGN AND SETTING: DEMOGRAPHIC, CLINICAL, AND TREATMENT PATTERNS IN THE EMR, SEER, MEDICARE, AND COMMERCIAL CLAIMS DATA WERE COMPARED USING SIX TUMOR SITES: breast, lung/bronchus, head/neck, colorectal, prostate, and non-Hodgkin's lymphoma (NHL). We identified various challenges in data standardization and selection of appropriate statistical procedures. We describe the patient and clinic inclusion criteria, treatment definitions, and consideration of the administrative and clinical purposes of the EMR, registry, and claims data to address these challenges. RESULTS: Sex and 10-year age distributions of patient populations for each tumor site were generally similar across the data sets. We observed several differences in racial composition and treatment patterns, and modest differences in distribution of tumor site. CONCLUSION: Our experience with an oncology EMR database identified several factors that must be considered when using EMRs for research purposes or generalizing results to the US cancer population. These factors were related primarily to evaluation of treatment patterns, including evaluation of stage, geographic location, race, and specialization of the medical facilities. While many specialty EMRs may not provide the breadth of data on medical care, as found in comprehensive claims databases and EMR systems, they can provide detailed clinical data not found in claims that are extremely important in conducting epidemiologic and outcomes research.

Effect of reimbursement changes on erythropoiesis-stimulating agent utilization and transfusions.

Cancer patients frequently develop chemotherapy-induced anemia, which can be treated with erythropoiesis-stimulating agents. These agents have shifted the standard of chemotherapy-induced anemia treatment away from the previous mainstay of red blood cell transfusions. In July 2007, the Centers for Medicare and Medicaid Services issued a National Coverage Decision restricting reimbursement for erythropoiesis-stimulating agents to those chemotherapy patients who have hemoglobin levels <10 g/dL at initiation of therapy. This decision was hypothesized to place a greater reliance on transfusions for chemotherapy-induced anemia treatment. This observational study examined transfusions and erythropoiesis-stimulating agent utilization rates within defined episodes of chemotherapy care using electronic medical records from seven practices consisting of 39 sites of care across seven states. We compared the frequency of myelosuppressive chemotherapy treatment, erythropoiesis-stimulating agent administrations, and red blood cell transfusions before and after the National Coverage Decision in oncology patients with chemotherapy-induced anemia. Although exposure to myelosuppressive chemotherapy was not different, erythropoiesis-stimulating agent administrations significantly decreased and blood transfusions significantly increased after implementation of the National Coverage Decision. The 31% increase in transfusions for patients aged 65 years and older was significant (P = 0.007) and higher than the 8% increase for patients younger than 65 years (P = 0.358). Changes in practice patterns for chemotherapy-induced anemia treatment that followed the Centers for Medicare and Medicaid Services reimbursement decision for erythropoiesis-stimulating agents seem to be impacting practice patterns. Further research is necessary to determine whether these changes represent a widespread and durable shift in patient treatment.

Value of biologic therapy: a forecasting model in three disease areas.

OBJECTIVE: Forecast the return on investment (ROI) for advances in biologic therapies in years 2015 and 2030, based upon impact on disease prevalence, morbidity, and mortality for asthma, diabetes, and colorectal cancer. METHODS: A deterministic, spreadsheet-based, forecasting model was developed based on trends in demographics and disease epidemiology. 'Return' was defined as reductions in disease burden (prevalence, morbidity, mortality) translated into monetary terms; 'investment' was defined as the incremental costs of biologic therapy advances. Data on disease prevalence, morbidity, mortality, and associated costs were obtained from government survey statistics or published literature. Expected impact of advances in biologic therapies was based on expert opinion. Gains in quality-adjusted life years (QALYs) were valued at $100,000 per QALY. RESULTS: The base case analysis, in which reductions in disease prevalence and mortality predicted by the expert panel are not considered, shows the resulting ROIs remain positive for asthma and diabetes but fall below $1 for colorectal cancer. Analysis involving expert panel predictions indicated positive ROI results for all three diseases at both time points, ranging from $207 for each incremental dollar spent on biologic therapies to treat asthma in 2030, to $4 for each incremental dollar spent on biologic therapies to treat colorectal cancer in 2015. If QALYs are not considered, the resulting ROIs remain positive for all three diseases at both time points. CONCLUSIONS: Society may expect substantial returns from investments in innovative biologic therapies. These benefits are most likely to be realized in an environment of appropriate use of new molecules. LIMITATIONS: The potential variance between forecasted (from expert opinion) and actual future health outcomes could be significant. Similarly, the forecasted growth in use of biologic therapies relied upon unvalidated market forecasts.