Benefits associated with a broad selection of dosage strengths for recombinant factor VIII products.

Factor VIII (FVIII) concentrates for haemophilia A patients are dosed according to body weight. This results in a continuous range of prescribed doses, which challenges pharmacies to find dosage strengths closest to the prescribed dose while utilizing the least number of vials. This study was conducted to determine whether a broader selection of FVIII dosage strengths results in improved dispensing accuracy and an increased number of single-vial users. This research retrospectively analyzed a US pharmacy database of prescriptions filled in 2008. Recombinant FVIII (rFVIII) therapies were classified by the range of dosage strengths offered in 2008: Group 1 had three dosage strengths; Group 2 had four dosage strengths; and Group 3 had six dosage strengths. A total of 76,584 dispensed doses of rFVIII for 1,244 patients were included in this analysis. Dispensing accuracy (calculated as both the absolute and relative difference between dispensed and prescribed dose) was significantly better for Group 3 (23.2 IU, 1.2%) than Groups 1 (33.5 IU, 1.6%) and 2 (50.2 IU, 2.4%) (both P < 0.01). In addition, the average number of unique actual rFVIII potencies dispensed per month was highly correlated (-0.977) with dispensing accuracy for each dosage strength group. Among Groups 1, 2 and 3, 23.0%, 44.9% and 73.4% of patients, respectively, had at least one single vial option dispensed (P < 0.0001). A broader selection of rFVIII dosage strengths and more actual rFVIII potencies were associated with improved dispensing accuracy and more single-vial users. This may translate into less waste, cost savings, increased convenience and improved adherence to physician-prescribed regimens.

Retrospective analysis of differences in annual factor VIII utilization among haemophilia A patients.

Finding differences in drug utilization patterns within rare patient populations is challenging without access to a large sample. Our objective was to identify patient and treatment-related factors associated with differences in annual recombinant factor VIII (rFVIII) utilization in a large cohort of haemophilia A patients. This retrospective analysis utilized a large, US specialty pharmacy dispensing database from January 2006 to September 2009. Differences in median annual FVIII utilization (IU kg(-1) year(-1)) by age, severity, treatment regimen, rFVIII product type and health insurance plan were tested using non-parametric statistics and regression analysis. A total of 1011 haemophilia A patients were included in the overall analysis. Severe haemophilia patients had higher median annual FVIII utilization than mild/moderate patients (P < 0.0001). Median annual FVIII utilization was also significantly different between treatment regimens (episodic = 1429 IU kg(-1) year(-1) vs. prophylaxis = 3993 IU kg(-1) year(-1) for severe patients, P < 0.0001). Children (0-12 years old), adolescents (13-18 years old) and adults (19+ years old) with severe haemophilia A receiving prophylaxis utilized 4588, 4082 and 3223 IU kg(-1) year(-1) (P < 0.0001). After controlling for age, severity, treatment regimen and insurance type, regression analysis revealed B domain-deleted recombinant FVIII (BDD-rFVIII) was associated with 33% higher FVIII consumption compared with full-length recombinant FVIII (FL-rFVIII) (P = 0.0172). Similar results were also seen when matching BDD-rFVIII and FL-rFVIII patients. Health insurance type was not associated with annual FVIII utilization. As expected, age, severity and treatment regimen were significantly associated with FVIII utilization. After controlling for confounders, patients receiving FL-rFVIII prophylactically were associated with lower annual FVIII utilization compared with patients receiving BDD-rFVIII prophylactically.

Patient and parent preferences for haemophilia A treatments.

Little is known about the relative importance of factor VIII (FVIII) treatment attributes to haemophilia A patients and their willingness to accept trade-offs among these attributes. To quantify patient and parent preferences for FVIII treatments and compare the relative importance of treatment attributes. Adult patients and parents of children with severe haemophilia A in the US completed a web-enabled, choice-format conjoint survey that presented a series of 12 trade-off questions, each including a pair of hypothetical treatment profiles. Each profile was defined by percent of bleeds stopped with one or two infusions, chance of developing an inhibitor, risk of viral infection, preparation volume, dosage strengths available, and history of supply shortage. Trade-off questions were based on a D-optimal experimental design. Preference weights for attribute levels were estimated using random-parameters logit. One hundred and forty seven subjects completed the survey. Over the ranges of attribute levels included in the study, risk of viral infection was the most important attribute. Remaining attributes were ranked in decreasing order of importance as follows: chance of developing an inhibitor, dosage strengths available, percent of bleeds stopped with one or two infusions, history of supply shortage, and preparation volume. Risk of viral infection was 6.0 times as important as percent of bleeds stopped with one or two infusions and 2.7 times as important as the chance of developing an inhibitor. While risk of viral infection was the most important attribute, this research demonstrates that many FVIII treatment attributes are important in the decision-making process.

Selection of clinical, patient-reported, and economic end points in acute exacerbation of chronic bronchitis.

BACKGROUND: Acute exacerbation of chronic bronchitis (AECB) places tremendous burden on patients, providers, employers, and health care systems. OBJECTIVE: The purpose of this paper is to (1) review the clinical, patient-reported, and economic measures used to evaluate disease burden and treatment effectiveness in AECB in clinical trials and (2) propose a guide for selecting study end points in AECB that will help capture all the relevant disease outcomes. METHODS: Two literature searches of the PubMed database were conducted to identify studies of clinical trials in bronchitis and evaluate the clinical, patient-reported, and economic end points used in these studies. RESULTS: Previous studies have focused primarily on clinician-assessed outcomes, which do not capture the full impact of AECB on patients' lives. Reporting mechanisms for most end points have been inconsistent, limiting the ability to compare information or interpret differences. Previous studies have given limited attention to patient-reported outcomes and the economic implications of AECB. Patient-reported outcomes such as speed of symptom relief and work productivity are important parameters for assessing treatment effectiveness and provide practical information for treatment evaluation. CONCLUSIONS: Additional research is needed to develop, examine, and validate patient-reported outcomes and the indirect costs of AECB. Measuring the relevant clinical, economic, and patient-reported outcomes in AECB patients using standardized methods may lead to a clearer understanding of the disease burden and the role, effectiveness, and cost-effectiveness of antibiotic treatment.

Workplace costs associated with acute exacerbation of chronic bronchitis: a comparison of moxifloxacin and levofloxacin.

Past research on acute exacerbation of chronic bronchitis (AECB) has not quantified related work loss. This exploratory study used data from a comparative trial of moxifloxacin versus levofloxacin to examine and compare workplace-related indirect costs. Both groups reported considerable absenteeism and lowered work productivity. However, patients taking moxifloxacin reported significantly higher work productivity than those taking levofloxacin (70% vs. 50%; P = .03). This translated into substantial indirect cost savings with moxifloxacin of $726/patient/year. Antibiotic choice in AECB can affect worker productivity and workplace-related indirect costs. Both insurers and employers should consider workplace-related indirect costs in addition to direct medical expenses when making formulary selections.