Number Of Therapy Days Research Articles

Haemophilia B therapy: impact of the reimbursability of a long-acting recombinant factor on pharmaceutical expenditure in Italy

to assess the variability in expenditure compared to 2022 assuming different rates of shifting of therapy days from current active ingredients used for the treatment of haemophilia B to nonacog beta pegolDesign: descriptive cross-sectional study. consumption in the year 2022 (data source: Medicines Utilisation Monitoring Centre, Italian Medicines Agency) of all medicinal products available in Italy containing coagulation factor IX. for each active ingredient, the total number of therapy days and the variability in expenditure compared to 2022 were estimated on the basis of a switch of therapy days, between 5% and 20%, to nonacog beta pegol. on the basis of considered scenarios, the analysis shows that the total annual expenditure for clotting factors used in the treatment of haemophilia B could remain at most unchanged or reduced. Particularly, the extent of the reduction in spending could vary from 0.11% to 2.26%. This trend would be in contrast to the stable increase seen in recent years, particularly in 2022. this predictive spending assessment may be useful in evaluating the economic impact from new treatment options, such as etranacogene dezaparvovec gene therapy already approved by the European Medicines Agency and the Food and Drug Administration, and to improve pharmaceutical governance.

Responsiveness of five measures of arm function in acute stroke rehabilitation

Objective: To determine the responsiveness of five arm function measures in people receiving acute inpatient stroke rehabilitation. Design: Inception cohort study. Setting: Comprehensive stroke unit providing early rehabilitation. Subjects: A total of 64 consecutively admitted stroke survivors with moderately severe disability (Modified Rankin Scale score median (interquartile range (IQR)): 4.0 (1.0)). Main measures: Responsiveness was analyzed by calculating effect size, standardized response mean and median-based effect size. Floor/ceiling effects were calculated as the percentage of participants scoring the lowest/highest possible scores. Results: Average length of stay and number of therapy days were 34 (SD = 27.9) and 12 (SD = 13.1), respectively. Box and Block Test and Functional Independence Measure–Self-Care showed the highest responsiveness with values in the moderate–large range (effect size = 1.09, standardized response mean = 1.07 and median-based effect size = 0.76; effect size = 0.94, standardized response mean = 1.04 and median-based effect size = 1.0). Responsiveness of Action Research Arm Test and Upper Limb–Motor Assessment Scale were moderate (effect size = 0.58, standardized response mean = 0.69 and median-based effect size = 0.59; effect size = 0.62, standardized response mean = 0.75 and median-based effect size = 0.67). For Manual Muscle Test, responsiveness was in the small–moderate range (effect size = 0.42, standardized response mean = 0.59 and median-based effect size = 0.5). Box and Block Test showed the largest floor effect on admission (28%), and Action Research Arm Test and Manual Muscle Test showed the largest ceiling effect on discharge (31%). Conclusion: These five measures varied in their ability to detect change with responsiveness ranging from the small to large range. Box and Block Test and Functional Independence Measure–Self-Care showed a greater ability to detect change; both demonstrated moderate–large responsiveness.

Analisi costo/efficacia della doxofillina vs. teofillina nella terapia dell’asma cronica reversibile dell’adulto

Doxofylline is a methyl-xanthine with bronchodilator activity which clinical efficacy in the treatment of asthma and COPD is equivalent to theophylline. Clinical trials prooved that while doxofylline is as effective as theophylline in the treatment of asthma, it is associated with lesser side effects. For our pharmacoeconomical evaluation, we employed the data of an international multi-centric study that compared the efficacy and tolerability of 12 weeks of treatment with either theophylline 250 mg tid, or doxofylline 400 mg tid, or placebo. The efficacy was evaluated with pulmonary function tests and by recording the number of asthma attacks and beta-2 agonist puffs: doxofylline 1200 mg/day and theophylline 750 mg/day resulted equally effective, but the former resulted better tolerated, as the adverse effects were lesser in number and severity. We performed a cost/effectiveness analysis in order to compare the two treatments, considering the perspective of the Italian health system (SSN) and of the Italian society. For the purposes of this analysis, the efficacy indices we considered were two: one simple, consisting in the number of avoided asthma attacks (AAA), and a more complex one, i.e. UATD (Utility Adjusted Therapy Days). For the construction of this index, we extracted the survival curve of the patients remaining in therapy; this curve showed that the cumulative number of therapy days is significantly greater in the doxofylline group, although the percentage of dropouts is similar at the end of the three months: this is due to the fact that the drop-outs in the theophylline group occur earlier after treatment start. The number of therapy days was then weighed for an utility index inversely proportional to the main daily number of avoided asthma attacks, thus obtaining the UATDs. The results of the cost/effectiveness analyses showed that doxofylline dominates theophylline treatment in asthma, as it is less expensive and more effective (in terms of AAAs and UATDs) for both considered decision-makers, i.e. Italian SSN and society. The consistency of these results was further confirmed by oneway sensitivity analyses.

Use of rhDNase therapy and costs of respiratory-related care in patients with cystic fibrosis.

To assess the relationship between level of use of recombinant deoxyribonuclease I (rhDNase) therapy and costs of respiratory-related care in patients with cystic fibrosis. Retrospective, cohort study using healthcare claims data from a large New England health insurer. All cystic fibrosis patients five years of age and older who began therapy with rhDNase in 1994 (the year it was first marketed in the US). Healthcare claims were compiled for six months prior to first receipt of rhDNase (pretreatment) and for 30 months subsequently (follow-up). Patients were stratified according to their level of rhDNase use during follow-up, based on whether it was above or below the median number of therapy days for the sample. Costs of rhDNase, all antibiotics, and all respiratory-related outpatient (physician, home health, hospital outpatient) and inpatient care were included. All costs were expressed on an annualized basis. Twenty-four patients with cystic fibrosis who began treatment with rhDNase in 1994 met all entry criteria; the median number of therapy days over a 30-month period was 355. Among patients with low (i.e., below the median) rhDNase use (n = 12), mean +/- SD annualized costs of respiratory-related care increased by almost $17,000 between pretreatment and follow-up, from $29,251 +/- $37,919 to $46,109 +/- $40,944. Among high-use patients (n = 12), costs decreased by approximately $2500, from $37,178 +/- $48,476 to $34,592 +/- $22,591. The change in both groups was accounted for primarily by a change in the number of respiratory-related hospitalizations. Prolonged use of rhDNase may reduce costs of respiratory-related care in patients with cystic fibrosis; further study is required, however, to confirm these findings.

Use of rhDNase Therapy and Costs of Respiratory-Related Care in Patients with Cystic Fibrosis

OBJECTIVE:To assess the relationship between level of use of recombinant deoxyribonuclease I (rhDNase) therapy and costs of respiratory-related care in patients with cystic fibrosis.DESIGN:Retrospective, cohort study using healthcare claims data from a large New England health insurer.PATIENTS:All cystic fibrosis patients five years of age and older who began therapy with rhDNase in 1994 (the year it was first marketed in the US). Healthcare claims were compiled for six months prior to first receipt of rhDNase (pretreatment) and for 30 months subsequently (follow-up). Patients were stratified according to their level of rhDNase use during follow-up, based on whether it was above or below the median number of therapy days for the sample.MAIN OUTCOME MEASURES:Costs of rhDNase, all antibiotics, and all respiratory-related outpatient (physician, home health, hospital outpatient) and inpatient care were included. All costs were expressed on an annualized basis.RESULTS:Twenty-four patients with cystic fibrosis who began treatment with rhDNase in 1994 met all entry criteria; the median number of therapy days over a 30-month period was 355. Among patients with low (i.e., below the median) rhDNase use (n = 12), mean ± SD annualized costs of respiratory-related care increased by almost $17000 between pretreatment and follow-up, from $29251 ± $37919 to $46109 ± $40944. Among high-use patients (n = 12), costs decreased by approximately $2500, from $37178 ± $48476 to $34592 ± $22591. The change in both groups was accounted for primarily by a change in the number of respiratory-related hospitalizations.CONCLUSIONS:Prolonged use of rhDNase may reduce costs of respiratory-related care in patients with cystic fibrosis; further study is required, however, to confirm these findings.

Clinical Experience with Atovaquone on a Treatment Investigational New Drug Protocol for Pneumocystis carinii Pneumonia

The clinical experience of human immunodeficiency virus (HIV) + patients treated with oral atovaquone for acute Pneumocytstis carinii pneumonia (PCP) under a Treatment Investigational New Drug (IND) protocol (mild or moderate PCP) and an Open-Label Study protocol (severe PCP) was evaluated. A total of 940 patients intolerant of or unresponsive to trimethoprimsulfamethoxazole were enrolled from private practices, clinics, and institutional HIV treatment centers in the United States. Demographics data and the history and severity of PCP were collected at enrollment. The number of therapy days, adverse experiences, clinical response to therapy, and mortality were collected at day 21. Reporting of serious, unexpected adverse experiences attributable to therapy was required. Of the 760 (96%) patients with mild to moderate disease for whom follow-up observation was complete, 591 (78%) responded clinically to treatment, 177 patients (23%) discontinued treatment prematurely, and 50 patients (7%) died. Of the 140 patients (95%) with severe PCP with follow-up data, 79 (56%) responded to treatment, 45 (32%) discontinued treatment early, and 53 patients (38%) died. Adverse events that resulted in temporary or permanent discontinuation of therapy included diarrhea, vomiting, elevated liver enzyme levels, nausea, and fever. No serious unexpected adverse events attributable to the drug were reported. The treatment IND mechanism enabled a large number of patients with acute PCP to be treated with this experimental therapy while the drug was under regulatory view.