Assessing health care resource use, outcomes, and costs among Medicaid beneficiaries receiving factor IX prophylaxis for hemophilia B.

BACKGROUND: Hemophilia B is characterized by a deficiency of clotting factor IX (FIX), leading to excessive bleeding. Hemophilia B is commonly treated using replacement FIX therapy, which may be administered prophylactically or on-demand following a bleeding episode. Previous research has found high health care resource use (HCRU) and costs among Medicare and commercially insured people with hemophilia B (PwHB), with FIX therapy being a primary driver of health care costs. OBJECTIVE: To assess HCRU, outcomes, and costs among US Medicaid beneficiaries receiving FIX prophylaxis for hemophilia B. METHODS: This study employed a retrospective comparative cohort design to assess HCRU, outcomes, and costs among adult male Medicaid beneficiaries receiving FIX prophylaxis for hemophilia B, relative to a matched comparator population of beneficiaries without bleeding disorders. Nationwide Medicaid claims and enrollment data from 2015 to 2020 were used for this analysis. Adult male PwHB who received FIX prophylaxis, defined as not having identified gaps in FIX therapy exceeding 60-days during a 1-year measurement period, and were continuously enrolled in Medicaid for at least 2 years, were matched 1:4 to comparator beneficiaries without bleeding disorders based on baseline demographic and clinical characteristics. Key measures of HCRU and outcomes included inpatient hospital admissions, outpatient hematologist visits, and bleeding events. Measures of health care costs were assessed among a subset of beneficiaries enrolled in fee-for-service Medicaid. RESULTS: PwHB receiving FIX prophylaxis were significantly more likely to have multiple inpatient hospital admissions and had a longer cumulative length of stay per person relative to comparator beneficiaries (30.2 vs 14.8 days, respectively; P = 0.0473). PwHB receiving FIX prophylaxis also had significantly higher rates of bleeding events relative to comparator beneficiaries (0.54 vs 0.02 per person, respectively; P < 0.0001) and outpatient hematologist visits (1.58 vs 0.20 per person, respectively; P < 0.0001). Annual costs among PwHB receiving FIX prophylaxis were significantly higher than costs among comparator beneficiaries ($928,370 vs $34,553 per person, respectively; P < 0.0001) and were overwhelmingly driven by costs associated with FIX therapy. CONCLUSIONS: This analysis found higher rates of HCRU and costs among Medicaid beneficiaries receiving FIX prophylaxis for hemophilia B relative to a matched comparator population of beneficiaries without bleeding disorders. Future research should examine hemophilia B costs and outcomes within the context of new treatments with innovative mechanisms of action, such as gene therapies, RNA interference therapies, and antitissue factor pathway inhibitor therapies.

The PICOTS-ComTeC Framework for Defining Digital Health Interventions: An ISPOR Special Interest Group Report.

OBJECTIVES: Digital health definitions are abundant, but often lack clarity and precision. We aimed to develop a minimum information framework to define patient-facing digital health interventions (DHIs) for outcomes research. METHODS: Definitions of digital-health-related terms (DHTs) were systematically reviewed, followed by a content analysis using frameworks, including PICOTS (population, intervention, comparator, outcome, timing, and setting), Shannon-Weaver Model of Communication, Agency for Healthcare Research and Quality Measures, and the World Health Organization's Classification of Digital Health Interventions. Subsequently, we conducted an online Delphi study to establish a minimum information framework, which was pilot tested by 5 experts using hypothetical examples. RESULTS: After screening 2610 records and 545 full-text articles, we identified 101 unique definitions of 67 secondary DHTs in 76 articles, resulting in 95 different patterns of concepts among the definitions. World Health Organization system (84.5%), message (75.7%), intervention (58.3%), and technology (52.4%) were the most frequently covered concepts. For the Delphi survey, we invited 47 members of the ISPOR Digital Health Special Interest Group, 18 of whom became the Delphi panel. The first, second, and third survey rounds were completed by 18, 11, and 10 respondents, respectively. After consolidating results, the PICOTS-ComTeC acronym emerged, involving 9 domains (population, intervention, comparator, outcome, timing, setting, communication, technology, and context) and 32 optional subcategories. CONCLUSIONS: Patient-facing DHIs can be specified using PICOTS-ComTeC that facilitates identification of appropriate interventions and comparators for a given decision. PICOTS-ComTeC is a flexible and versatile tool, intended to assist authors in designing and reporting primary studies and evidence syntheses, yielding actionable results for clinicians and other decision makers.

Economic and humanistic burden in kidney transplant rejection: a literature review.

INTRODUCTION: Antibody-mediated rejection (ABMR) is a major cause of late kidney allograft failure, but its economic and humanistic impacts have not been well-characterized in the literature. AREAS COVERED: We reviewed available literature on economic burden (costs and healthcare resource use) and humanistic burden (health-related quality of life impacts [HRQOL] and utility estimates) in patients diagnosed with kidney transplant rejection; ABMR-specific studies were of particular interest. In total, 21 publications reporting economic and humanistic burden were included in the review; 9 of these reported ABMR-specific outcomes. The reviewed studies consistently showed a greater burden associated with ABMR-related transplant rejection than with non-ABMR transplant rejection. EXPERT OPINION: Evidence suggests greater economic burden and increased HRQOL impairment with ABMR-related kidney transplant rejection relative to non-ABMR, although small sample sizes and missing definitions for ABMR make meaningful comparisons between studies challenging. Because no International Classification of Diseases (ICD)-10 codes currently describe the etiologies of transplant rejection, it is difficult to characterize the burden of distinct types of transplant rejection. The paucity of high-quality data on the burden of ABMR in kidney transplant rejection demonstrates the need for more etiology-centric ICD-10 codes.

Clinical and Economic Burden Associated With Acute Graft-Versus-Host Disease After Allogeneic Hematopoietic Cell Transplantation in Germany.

BACKGROUND: Acute graft-vs-host disease (aGVHD) is a serious complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT), yet there are limited data on the clinical and economic burden of aGVHD in Germany. This real-world study aimed to evaluate clinical and economic outcomes among patients in Germany with or without aGVHD after allo-HSCT. METHODS: This retrospective cohort study used administrative claims extracted from the German statutory health insurance database. Eligible adult patients underwent allo-HSCT between 1 January 2009 and 31 December 2017 for any hematological malignancy. Clinical (severe infections and mortality) and economic (health care resource use [HCRU] and costs) outcomes were compared in "aGVHD" patients and "no GVHD" patients. Propensity score matching (1:1) was used to balance covariates between the aGVHD and no GVHD groups. RESULTS: After propensity score matching, 95 aGVHD and 95 no GVHD patients were included in the analysis. The aGVHD group had significantly higher odds of mortality than the no GVHD group (odds ratio [OR] 2.2; 95% CI 1.2-4.0). Odds of severe infection were similar between the 2 groups (OR 1.7; 95% CI 0.9-3.3). Patients in the aGVHD group had significantly more overnight hospitalizations per patient-year (mean [SD]: 3.7 [3.0] and 2.7 [2.5], P = .029), and total direct costs were 1.6-fold higher than those in the no GVHD group. CONCLUSION: Among patients who underwent allo-HSCT, aGVHD was associated with significantly higher mortality, HCRU, and costs, highlighting the need for effective prophylaxis and treatment options to prevent or reduce the incidence of aGVHD.

Clinical and economic burden associated with graft-versus-host disease following allogeneic hematopoietic cell transplantation in France.

The real-world clinical and economic burden of graft-versus-host disease (GVHD) following allogeneic hematopoietic stem cell transplantation has not been comprehensively studied in France. Clinical outcomes, healthcare resource utilization and costs associated with acute GVHD (aGVHD), chronic GVHD (cGVHD), acute plus chronic GVHD (a+cGVHD) versus no GVHD were compared using French administrative claims data. After propensity score matching, 1934, 408, and 1268 matched pairs were retained for the aGVHD, cGVHD, and a+cGVHD cohorts, respectively. Compared with patients with no GVHD, odds of developing severe infection were greater in patients with aGVHD (odds ratio: 1.7 [95% confidence interval: 1.4, 2.1]). Compared with patients with no GVHD, mortality rates were higher in patients with aGVHD (rate ratio (RR): 1.6 [1.4, 1.7]) and with a+cGVHD (RR: 1.1 [1.0, 1.2]) but similar in patients with cGVHD (RR: 0.9 [0.7, 1.1]). Mean overnight hospital admission rates per patient-year were significantly higher in patients with aGVHD and a+cGVHD compared with no GVHD. Total direct costs (range €174,482-332,557) were 1.2, 1.5, and 1.9 times higher for patients with aGVHD, cGVHD, and a+cGVHD, respectively, versus patients with no GVHD. These results highlight the significant unmet need for effective treatments of patients who experience GVHD.

How Useful Are Digital Health Terms for Outcomes Research? An ISPOR Special Interest Group Report.

OBJECTIVES: This study aimed to review definitions of digital health and understand their relevance for health outcomes research. Four umbrella terms (digital health, electronic health, mobile health, and telehealth/telemedicine) were summarized in this article. METHODS: PubMed/MEDLINE, Embase, Cochrane Library, and EconLit were searched from January 2015 to May 2020 for systematic reviews containing key Medical Subject Headings terms for digital health (n = 38) and synonyms of "definition." Independent pairs of reviewers performed each stage of the review, with reconciliation by a third reviewer if required. A single reviewer consolidated each definition for consistency. We performed text analysis via word clouds and computed document frequency-and inverse corpus frequency scores. RESULTS: The search retrieved 2610 records with 545 articles (20.9%) taken forward for full-text review. Of these, 39.3% (214 of 545) were eligible for data extraction, of which 134 full-text articles were retained for this analysis containing 142 unique definitions of umbrella terms (digital health [n = 4], electronic health [n = 36], mobile health [n = 50], and telehealth/telemedicine [n = 52]). Seminal definitions exist but have increasingly been adapted over time and new definitions were created. Nevertheless, the most characteristic words extracted from the definitions via the text analyses still showed considerable overlap between the 4 umbrella terms. CONCLUSIONS: To focus evidence summaries for outcomes research purposes, umbrella terms should be accompanied by Medical Subject Headings terms reflecting population, intervention, comparator, outcome, timing, and setting. Ultimately a functional classification system is needed to create standardized terminology for digital health interventions denoting the domains of patient-level effects and outcomes.

Medication compliance and persistence: terminology and definitions.

OBJECTIVE: The aim of the study is to provide guidance regarding the meaning and use of the terms "compliance" and "persistence" as they relate to the study of medication use. METHODS: A literature review and debate on appropriate terminology and definitions were carried out. RESULTS: Medication compliance and medication persistence are two different constructs. Medication compliance (synonym: adherence) refers to the degree or extent of conformity to the recommendations about day-to-day treatment by the provider with respect to the timing, dosage, and frequency. It may be defined as "the extent to which a patient acts in accordance with the prescribed interval, and dose of a dosing regimen." Medication persistence refers to the act of continuing the treatment for the prescribed duration. It may be defined as "the duration of time from initiation to discontinuation of therapy." No overarching term combines these two distinct constructs. CONCLUSIONS: Providing specific definitions for compliance and persistence is important for sound quantitative expressions of patients' drug dosing histories and their explanatory power for clinical and economic events. Adoption of these definitions by health outcomes researchers will provide a consistent framework and lexicon for research.