88 results on '"Quinolones economics"'
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2. Pharmacokinetic Enhancement of Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis: A Cost Reduction Strategy to Address Global Disparities in Access.
- Author
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Hong E, Zampoli M, and Beringer PM
- Subjects
- Humans, Pyridines pharmacokinetics, Pyridines therapeutic use, Pyridines economics, Quinolines therapeutic use, Quinolines pharmacokinetics, Quinolines economics, Drug Costs, Health Services Accessibility economics, Healthcare Disparities economics, Chloride Channel Agonists therapeutic use, Chloride Channel Agonists pharmacokinetics, Chloride Channel Agonists economics, Pyrrolidines therapeutic use, Pyrrolidines pharmacokinetics, Cystic Fibrosis drug therapy, Cystic Fibrosis economics, Benzodioxoles therapeutic use, Benzodioxoles pharmacokinetics, Aminophenols therapeutic use, Aminophenols pharmacokinetics, Aminophenols economics, Drug Combinations, Quinolones therapeutic use, Quinolones pharmacokinetics, Quinolones economics, Indoles economics, Indoles therapeutic use, Indoles pharmacokinetics, Pyrazoles therapeutic use, Pyrazoles pharmacokinetics, Pyrazoles economics
- Published
- 2024
- Full Text
- View/download PDF
3. Clinical, economic, and societal burden of cystic fibrosis and the impact of the CFTR modulator, lumacaftor/ivacaftor: an assessment using linked registry data in Sweden.
- Author
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Lindblad A, Monestrol I, Gilljam M, Krantz C, McGarry LJ, Banefelt J, and Aldvén M
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- Humans, Male, Female, Sweden, Retrospective Studies, Child, Adolescent, Adult, Young Adult, Caregivers, Middle Aged, Absenteeism, Health Expenditures statistics & numerical data, Health Services statistics & numerical data, Health Services economics, Cystic Fibrosis drug therapy, Aminophenols therapeutic use, Aminophenols economics, Quinolones therapeutic use, Quinolones economics, Benzodioxoles therapeutic use, Benzodioxoles economics, Cost of Illness, Aminopyridines therapeutic use, Aminopyridines economics, Drug Combinations, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Registries
- Abstract
Aims: We aimed to describe the clinical, economic, and societal burdens of cystic fibrosis (CF) and impact of CF transmembrane conductance regulator modulator (CFTRm) treatment on people with CF, caregivers, and healthcare systems., Material and Methods: This retrospective study used linked real-world data from Swedish national population-based registries and the Swedish CF Quality Registry to assess clinical, economic, and societal burden and CFTR impact in CF. Records from people with CF and a ten-fold control population without CF matched by sex, birth year, and location were compared during 2019. Outcomes for a subset aged >6 years initiating lumacaftor/ivacaftor (LUM/IVA) in 2018 were compared 12 months pre- and post-treatment initiation., Results: People with CF ( n = 743) had >10 times more inpatient and outpatient specialist visits annually vs controls ( n = 7406). Those aged >18 had an additional 77·7 (95% CI: 70·3, 85·1) days of work absence, at a societal cost of €11,563 (95% CI: 10,463, 12,662), while caregivers of those aged <18 missed an additional 6.1 (5.0, 7.2) workdays. With LUM/IVA treatment, people with CF ( n = 100) had significantly increased lung function (mean change in ppFEV
1 [3·8 points; 95% CI: 1·1, 6·6]), on average 0·5 (95% CI: -0·8, -0·2) fewer pulmonary exacerbations and 45·2 (95% CI: 13·3, 77·2) fewer days of antibiotics. Days of work lost by caregivers of people with CF aged <18 decreased by 5·4 days (95% CI: 2·9, 7·9)., Conclusion: CF is associated with a high clinical economic and societal burden in Sweden. Improvements in clinical status observed in people with CF treated with LUM/IVA were reflected in reduced caregiver and societal burden.- Published
- 2024
- Full Text
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4. CFTR modulators: transformative therapies for cystic fibrosis.
- Author
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Dwight M and Marshall B
- Subjects
- Aminophenols economics, Aminophenols therapeutic use, Benzodioxoles economics, Benzodioxoles therapeutic use, Chloride Channel Agonists economics, Cystic Fibrosis economics, Cystic Fibrosis genetics, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Drug Approval economics, Drug Combinations, Humans, Indoles economics, Indoles therapeutic use, Medical Assistance, Mutation, Pyrazoles economics, Pyrazoles therapeutic use, Pyridines economics, Pyridines therapeutic use, Quinolines economics, Quinolines therapeutic use, Quinolones economics, Quinolones therapeutic use, Treatment Outcome, United States, United States Food and Drug Administration, Chloride Channel Agonists therapeutic use, Cystic Fibrosis drug therapy, Cystic Fibrosis Transmembrane Conductance Regulator agonists, Drug Costs
- Abstract
DISCLOSURES: No funding contributed to the writing of this commentary. Both authors are employed by the Cystic Fibrosis Foundation. The Cystic Fibrosis Foundation has entered into therapeutic development award agreements and licensing agreements to assist with the development of CFTR modulators that may result in intellectual property rights, royalties, and other forms of consideration provided to CFF. Some of these agreements are subject to confidentiality restrictions and, thus, CFF cannot comment on them.
- Published
- 2021
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- View/download PDF
5. The effectiveness and value of novel treatments for cystic fibrosis.
- Author
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Tice JA, Kuntz KM, Wherry K, Seidner M, Rind DM, and Pearson SD
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- Adolescent, Aminophenols economics, Aminophenols therapeutic use, Aminopyridines economics, Aminopyridines therapeutic use, Benzodioxoles economics, Benzodioxoles therapeutic use, Child, Chloride Channel Agonists economics, Cystic Fibrosis economics, Cystic Fibrosis genetics, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Drug Approval economics, Drug Combinations, Drug Costs, Health Policy economics, Humans, Indoles economics, Indoles therapeutic use, Mutation, Pyrazoles economics, Pyrazoles therapeutic use, Pyridines economics, Pyridines therapeutic use, Quinolines economics, Quinolines therapeutic use, Quinolones economics, Quinolones therapeutic use, Treatment Outcome, United States, United States Food and Drug Administration, Chloride Channel Agonists therapeutic use, Cost-Benefit Analysis, Cystic Fibrosis drug therapy, Cystic Fibrosis Transmembrane Conductance Regulator agonists, Models, Economic
- Abstract
DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, California Health Care Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, America's Health Insurance Plans, Anthem, Allergan, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare. Seidner, Rind, and Pearson are employed by ICER. Tice reports contracts to his institution, University of California, San Francisco, from ICER during the conduct of this study. Wherry has nothing to disclose.
- Published
- 2021
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6. Ozenoxacin: A novel topical antibiotic.
- Author
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Santhosh P and Thomas MH
- Subjects
- Administration, Topical, Aminopyridines chemistry, Aminopyridines economics, Anti-Bacterial Agents chemistry, Anti-Bacterial Agents economics, Contraindications, Drug, Drug Interactions, Drug Resistance, Bacterial, Humans, Molecular Structure, Ointments, Quinolones chemistry, Quinolones economics, Aminopyridines pharmacology, Anti-Bacterial Agents pharmacology, Quinolones pharmacology
- Published
- 2021
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7. Cost-Effectiveness of Ivacaftor Therapy for Treatment of Cystic Fibrosis Patients With the G551D Gating Mutation.
- Author
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Wherry K, Williamson I, Chapman RH, and Kuntz KM
- Subjects
- Aminophenols economics, Chloride Channel Agonists economics, Cost-Benefit Analysis, Cystic Fibrosis drug therapy, Cystic Fibrosis genetics, Drug Costs, Female, Humans, Male, Mutation genetics, Quality-Adjusted Life Years, Quinolones economics, Time Factors, Aminophenols therapeutic use, Chloride Channel Agonists therapeutic use, Cystic Fibrosis economics, Health Care Costs statistics & numerical data, Quinolones therapeutic use
- Abstract
Objectives: Cystic fibrosis (CF) is a rare genetic disease with no cure. Until recently, treatment has targeted symptoms of the disease and not the disease-causing genetic defect. Ivacaftor is included in a new class of breakthrough drugs targeting the genetic defects of CF. We sought to estimate the long-term cost-effectiveness of ivacaftor from a US payer perspective., Methods: We developed an individual-level microsimulation model that followed a cohort of heterogeneous US CF patients over a lifetime. The primary outcome of interest was quality-adjusted life years (QALYs). We also compared unadjusted life years, count of acute pulmonary exacerbations, and count of lung transplants over a lifetime between patients treated with ivacaftor plus best supportive care and patients treated with best supportive care alone. We conducted one-way and probabilistic sensitivity analyses to test the impact of various model inputs and uncertainties., Results: We found a substantial increase in QALYs, life years, and treatment costs over a lifetime for patients treated with ivacaftor plus best supportive care versus best supportive care alone. Discounted results for ivacaftor were 22.92 QALYs and $8 797 840 in total lifetime costs compared to 16.12 QALYs and $2 336 366 lifetime costs for best supportive care alone. The incremental cost-effectiveness ratios (ICERs) were $950 217 per QALY. Results from the probabilistic sensitivity analysis indicated a 0% chance that ivacaftor was cost-effective at a willingness-to-pay (WTP) threshold of $500 000 per QALY., Conclusions: Treatment with ivacaftor plus best supportive care versus best supportive care alone is not cost-effective at or near commonly accepted WTP thresholds., (Copyright © 2020 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)
- Published
- 2020
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8. Ozenoxacin (Xepi) for the Treatment of Impetigo.
- Author
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Eudaley S
- Subjects
- Administration, Topical, Aminopyridines economics, Anti-Bacterial Agents economics, Child, Child, Preschool, Humans, Infant, Quinolones economics, Aminopyridines administration & dosage, Anti-Bacterial Agents administration & dosage, Impetigo drug therapy, Quinolones administration & dosage
- Published
- 2020
9. Adherence to lumacaftor-ivacaftor therapy in patients with cystic fibrosis in France.
- Author
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Olivereau L, Nave V, Garcia S, Perceval M, Rabilloud M, Durieu I, and Reynaud Q
- Subjects
- Adult, Age Factors, Cost-Benefit Analysis, Drug Combinations, Female, Forced Expiratory Volume, France epidemiology, Homozygote, Humans, Male, Respiratory Function Tests methods, Respiratory Function Tests statistics & numerical data, Retrospective Studies, Treatment Outcome, Aminophenols economics, Aminophenols therapeutic use, Aminopyridines economics, Aminopyridines therapeutic use, Benzodioxoles economics, Benzodioxoles therapeutic use, Chloride Channel Agonists economics, Chloride Channel Agonists therapeutic use, Cystic Fibrosis drug therapy, Cystic Fibrosis economics, Cystic Fibrosis epidemiology, Cystic Fibrosis genetics, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Medication Adherence statistics & numerical data, Quinolones economics, Quinolones therapeutic use
- Abstract
Background: Lumacaftor-ivacaftor combination is a promising treatment for cystic fibrosis (CF) patients homozygous for the F508del-CFTR mutation. Optimal adherence is essential to achieve full health outcomes benefits., Methods: This retrospective study used pharmacy refills data to calculate proportion of days covered (PDC). Adherence was defined as a PDC ≥80%. A logistic regression analysis was conducted to examine factors associated with medication adherence., Results: Ninety-six patients were included in the final cohort for analysis. The mean PDC was 96% ± 14 at 6 months, and 91% ± 17 at 12 months. The proportion of adherent patients was 89% and 83% at 6 and 12 months respectively. Age and ppFEV1 were found to affect medication adherence., Conclusions: Considering the medico-economic impact of CFTR modulator therapy, high adherence rates to lumacaftor-ivacaftor found in this study are encouraging., Competing Interests: Declaration of Competing Interest The authors declare that they have no conflicts of interests., (Copyright © 2019 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.)
- Published
- 2020
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10. Health Care Cost in Patients With Schizophrenia Treated With Brexpiprazole Versus Other Oral Atypical Antipsychotic Therapy.
- Author
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Yan T, Greene M, Chang E, Houle CR, Waters HC, Tarbox MH, and Broder MS
- Subjects
- Administration, Oral, Adult, Antipsychotic Agents therapeutic use, Aripiprazole economics, Aripiprazole therapeutic use, Female, Health Care Costs, Humans, Lurasidone Hydrochloride economics, Lurasidone Hydrochloride therapeutic use, Male, Medicaid economics, Medicare economics, Middle Aged, Olanzapine economics, Olanzapine therapeutic use, Paliperidone Palmitate economics, Paliperidone Palmitate therapeutic use, Piperazines economics, Piperazines therapeutic use, Quetiapine Fumarate economics, Quetiapine Fumarate therapeutic use, Quinolones therapeutic use, Risperidone economics, Risperidone therapeutic use, Schizophrenia drug therapy, Thiazoles economics, Thiazoles therapeutic use, Thiophenes therapeutic use, United States, Antipsychotic Agents economics, Hospitalization economics, Quinolones economics, Schizophrenia economics, Thiophenes economics
- Abstract
Purpose: Brexpiprazole is an oral atypical antipsychotic (OAA) for the treatment of schizophrenia (SCZ). This study compared all-cause and psychiatric inpatient hospitalization and medical costs in adult patients with SCZ newly treated with brexpiprazole versus other US Food and Drug Administration-approved OAAs in a real-world setting., Methods: This retrospective cohort study analyzed data from: (1) the IBM MarketScan Commercial and Medicare Supplemental databases, and the MarketScan Multi-State Medicaid database; and (2) the de-identified Optum Clinformatics Datamart. Adult patients were identified if they had SCZ and initiated either brexpiprazole or another OAA during the study identification period (July 1, 2015, to September 30, 2016, for MarketScan Commercial and Medicare Supplemental and for Optum; July 1, 2015, to June 30, 2016, for MarketScan Multi-State Medicaid) and had ≥12 months of continuous enrollment before (baseline) and after (follow-up) the first treatment date. Linear regression analyses were performed to test associations between treatment groups (brexpiprazole vs another OAA) and costs (total and medical); negative binomial regression models were used to estimate number of hospitalizations per year, adjusting for baseline characteristics and medication adherence to index treatment during the 12-month follow-up., Findings: The final study sample consisted of 6254 patients with SCZ: 176 initiated brexpiprazole; 391, ziprasidone; 453, paliperidone; 523, lurasidone; 786, aripiprazole; 1234, quetiapine; 1264, olanzapine; and 1427, risperidone. Controlling for baseline characteristics and medication adherence, the adjusted number of hospitalizations (both all-cause and psychiatric), all-cause total costs, and all-cause medical costs did not differ across groups. Brexpiprazole users had the lowest mean psychiatric costs among all OAA users ($12,013; 95% bootstrap CI, 7488-16,538). Compared with brexpiprazole users, paliperidone (incidence rate ratio [95% CI], 1.52 [1.05-2.19]; P = 0.027) and quetiapine (incidence rate ratio [95% CI], 1.47 [1.04-2.07]; P = 0.029) users had more psychiatric hospitalizations per year. Paliperidone had higher psychiatric costs than brexpiprazole (total, $32,066 [95% bootstrap CI, 28,779-35,353] vs $23,851 [18,907-28,795]; medical, $19,343 [16,294-22,392] vs $12,013 [7488-16,538]). Psychiatric medical costs were also $6744 higher in olanzapine users (95% bootstrap CI, 1694-11,795; P = 0.009) than in brexpiprazole users., Implications: Patients with SCZ treated with brexpiprazole had fewer psychiatric hospitalizations and lower psychiatric costs than those treated with paliperidone. Differences in the number of all-cause hospitalizations and medical costs among treatments were not statistically significant. Although treatment decisions are driven by a number of factors (eg, clinical circumstances and drug costs), choice of OAA may affect health care costs., (Copyright © 2019 The Author(s). Published by Elsevier Inc. All rights reserved.)
- Published
- 2020
- Full Text
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11. Orphan Drug Pricing and Costs: A Case Study of Kalydeco and Orkambi.
- Author
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Hollis A
- Subjects
- Cost-Benefit Analysis, Drug Combinations, Drug Costs statistics & numerical data, Humans, Aminophenols economics, Aminopyridines economics, Benzodioxoles economics, Chloride Channel Agonists economics, Cystic Fibrosis drug therapy, Cystic Fibrosis economics, Orphan Drug Production economics, Quinolones economics
- Abstract
Background: A common narrative is that high prices are necessary for "orphan drugs" because of the fewer patients. In the context of state health insurance systems, the high prices create significant challenges because of limited budgets., Results: This study carefully examines both costs and revenues of two drugs for cystic fibrosis (ivacaftor and lumacaftor), showing that, for this important example, prices are not high because of fewer patients. The study then explores the justifications usually given for high orphan drug prices, including the need to support research and development for new drugs. Each of these standard justifications is shown to be inadequate; instead, it appears that the exercise of market power in the presence of insurance is the dominant driver of high prices., Interpretation: Insurers need to re-examine how they address high-priced drugs., (Copyright © 2019 Longwoods Publishing.)
- Published
- 2019
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12. Medication Adherence, Health Care Utilization, and Costs in Patients With Major Depressive Disorder Initiating Adjunctive Atypical Antipsychotic Treatment.
- Author
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Broder MS, Greene M, Yan T, Chang E, Hartry A, and Yermilov I
- Subjects
- Adolescent, Adult, Aged, Antipsychotic Agents economics, Depressive Disorder, Major economics, Emergency Service, Hospital economics, Emergency Service, Hospital statistics & numerical data, Female, Health Care Costs, Hospitalization economics, Hospitalization statistics & numerical data, Humans, Lurasidone Hydrochloride economics, Male, Medicaid economics, Medicare economics, Medication Adherence statistics & numerical data, Middle Aged, Patient Acceptance of Health Care, Quetiapine Fumarate economics, Quinolones economics, Thiophenes economics, United States, Young Adult, Antipsychotic Agents therapeutic use, Depressive Disorder, Major drug therapy, Lurasidone Hydrochloride therapeutic use, Quetiapine Fumarate therapeutic use, Quinolones therapeutic use, Thiophenes therapeutic use
- Abstract
Purpose: The purpose of this study was to compare medication adherence, health care utilization, and cost among patients receiving adjunctive treatment for major depressive disorder (MDD) with brexpiprazole, quetiapine, or lurasidone., Methods: Using Truven Health MarketScan® Commercial, Medicaid, and Medicare Supplemental Databases, we identified adults with MDD initiating adjunctive treatment with brexpiprazole, quetiapine, or lurasidone (index atypical antipsychotic [AAP]). We compared medication adherence and persistence measured by proportion of days covered (PDC) and treatment duration of index AAP, all-cause and psychiatric hospital care (hospitalization or emergency department visit), and medical costs during 6-month follow-up. Models performed included logistic regression for hospital care, linear regression for PDC and cost, and Cox proportional hazards regression for time to discontinuation, adjusting for demographic, clinical, and utilization differences during the 6 months before index AAP., Findings: The total sample included 778 brexpiprazole, 626 lurasidone, and 3458 quetiapine therapy initiators. Adjusting for baseline differences, the risk of discontinuation of index AAP was statistically significantly higher for quetiapine than for brexpiprazole (hazard ratio [HR] = 1.13; 95% CI, 1.02-1.25; P = 0.023) and did not differ between lurasidone and brexipiprazole (HR = 1.14; 95% CI, 1.00-1.29; P = 0.054). The adjusted rate of all-cause hospitalization or emergency department visit in the postindex period was lowest for brexpiprazole at 27.4% (95% CI, 24.0%-31.0%), compared with 31.1% (95% CI, 27.3%-35.2%) for lurasidone and 35.3% (95% CI, 33.5%-37.1%) for quetiapine (P< 0.001 for all comparisons). Quetiapine users had increased all-cause costs compared with brexpiprazole users (estimate = $2309; 95% CI, $31-$4587; P = 0.047); all-cause medical costs did not differ between lurasidone and brexpiprazole (estimate = $913; 95% CI, $-2033 -$3859; P = 0.543). Adjusted psychiatric hospital care, psychiatric costs, and PDC did not differ significantly among the groups., Implications: In patients with MDD and a variety of insurance types, brexpiprazole use was associated with statistically significantly lower risks of discontinuation, risk of hospital care (hospitalization and ED visits), and all-cause medical costs compared with adjunctive quetiapine. Differences between brexpiprazole and lurasidone were not statistically significant. These findings suggest that drug choice is associated with subsequent health care utilization and costs., (Copyright © 2019 The Authors. Published by Elsevier Inc. All rights reserved.)
- Published
- 2019
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13. Cost-effectiveness of twice-daily indacaterol/glycopyrrolate inhalation powder for the treatment of moderate to severe COPD in the US.
- Author
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Rajagopalan K, Bloudek L, Marvel J, Dembek C, and Kavati A
- Subjects
- Administration, Inhalation, Adrenergic beta-2 Receptor Agonists adverse effects, Bronchodilator Agents adverse effects, Cost-Benefit Analysis, Drug Administration Schedule, Drug Combinations, Forced Expiratory Volume, Glycopyrrolate adverse effects, Humans, Indans adverse effects, Lung physiopathology, Markov Chains, Models, Economic, Muscarinic Antagonists adverse effects, Nebulizers and Vaporizers economics, Powders, Pulmonary Disease, Chronic Obstructive diagnosis, Pulmonary Disease, Chronic Obstructive physiopathology, Quinolones adverse effects, Randomized Controlled Trials as Topic, Severity of Illness Index, Time Factors, Treatment Outcome, United States, Adrenergic beta-2 Receptor Agonists administration & dosage, Adrenergic beta-2 Receptor Agonists economics, Bronchodilator Agents administration & dosage, Bronchodilator Agents economics, Drug Costs, Glycopyrrolate administration & dosage, Glycopyrrolate economics, Indans administration & dosage, Indans economics, Lung drug effects, Muscarinic Antagonists administration & dosage, Muscarinic Antagonists economics, Pulmonary Disease, Chronic Obstructive drug therapy, Pulmonary Disease, Chronic Obstructive economics, Quinolones administration & dosage, Quinolones economics
- Abstract
Background: Indacaterol 27.5 µg/glycopyrrolate 15.6 µg (IND/GLY 27.5/15.6 µg) inhalation powder, a twice-daily, fixed-dose combination of a long-acting beta2-agonist (LABA) and a long-acting antimuscarinic antagonist (LAMA), is indicated in the US for long-term maintenance treatment of airflow obstruction in patients with COPD. The safety and efficacy of IND/GLY 27.5/15.6 µg have been established, but cost-effectiveness is not yet known. This study compared the cost-effectiveness of IND/GLY 27.5/15.6 µg with other long-acting COPD maintenance therapies., Methods: A Markov model was constructed from the US payer perspective. Health states were defined as mild (post-bronchodilator FEV
1 ≥80% of predicted), moderate (50% ≤FEV1 <80% of predicted), severe (30% ≤FEV1 <50% of predicted), and very severe (FEV1 <30% of predicted) COPD. Patients entering the model transitioned through health states based on placebo-adjusted change from baseline in trough FEV1 for each comparator at week 12. Comparators included other US Food and Drug Administration-approved LABA/LAMA fixed-dose combinations as well as commonly prescribed LAMA and LABA/inhaled corticosteroid agents. One-way and probabilistic sensitivity analyses were conducted to test the model assumptions and the overall robustness of the results., Results: Using the model, IND/GLY 27.5/15.6 µg treatment for 12 weeks resulted in total costs of US $23,375 vs US $9,365 for placebo. Compared with placebo, IND/GLY 27.5/15.6 treatment resulted in the highest improvement in FEV1 across all comparators and the lowest cost per decline in 100 mL FEV1 . IND/GLY 27.5/15.6 µg was also among the most cost-effective treatment option as measured by St George's Respiratory Questionnaire response rate, at US $3,518 per additional responder at 12 weeks compared with placebo. In addition, IND/GLY 27.5/15.6 µg had the lowest cost per severe exacerbation avoided vs placebo across all comparators (US $87,686)., Conclusion: This model, developed from the US payer perspective with a 5-year time horizon, found IND/GLY 27.5/15.6 µg to be a cost-effective treatment option for patients with moderate to severe COPD., Competing Interests: Disclosure KR was an employee of Sunovion Pharmaceuticals Inc at the time the study was conducted. CD is an employee of Sunovion Pharmaceuticals Inc. JM and AK are employees of Novartis Pharmaceuticals Corporation. LB was an employee of Xcenda LLC when the study was conducted. The authors report no other conflicts of interest in this work.- Published
- 2018
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14. Cost-Effectiveness and Budget Impact of Lumacaftor/Ivacaftor in the Treatment of Cystic Fibrosis.
- Author
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Vadagam P, Kamal KM, Covvey JR, Giannetti V, and Mukherjee K
- Subjects
- Clinical Decision-Making, Cost-Benefit Analysis, Cystic Fibrosis diagnosis, Cystic Fibrosis physiopathology, Decision Support Techniques, Drug Combinations, Forced Expiratory Volume, Humans, Lung drug effects, Lung physiopathology, Models, Economic, Quality-Adjusted Life Years, Time Factors, Treatment Outcome, Aminophenols economics, Aminophenols therapeutic use, Aminopyridines economics, Aminopyridines therapeutic use, Benzodioxoles economics, Benzodioxoles therapeutic use, Budgets, Cystic Fibrosis drug therapy, Cystic Fibrosis economics, Drug Costs, Quinolones economics, Quinolones therapeutic use, Respiratory System Agents economics, Respiratory System Agents therapeutic use
- Abstract
Background: Cystic fibrosis (CF) is a chronic, progressive, genetic disease affecting more than 30,000 people in the United States and 70,000 people globally. The goals of treatment are to slow disease progression, reduce pulmonary exacerbations, relieve chronic symptoms, and improve the patient's quality of life. Lumacaftor/ivacaftor is a new therapy for CF that has demonstrated good clinical outcomes, including improved absolute percentage predicted forced expiratory volume in 1 second (FEV
1 %). However, given the high cost of therapy, there is a need to evaluate the overall value of lumacaftor/ivacaftor in CF management., Objectives: To (a) conduct a cost-effectiveness analysis (CEA) of lumacaftor/ivacaftor to understand the overall effectiveness of the drug compared with its costs and (b) conduct a budget impact analysis (BIA) to understand the potential financial effect of introducing a new drug in a health plan., Methods: Two static decision models were developed using Microsoft Excel to evaluate the cost-effectiveness and budget impact of lumacaftor/ivacaftor over a 1-year time frame from a payer perspective. Model inputs included drug costs (wholesale acquisition costs), drug monitoring schedules (package inserts), drug monitoring costs (Centers for Medicare & Medicaid physician fee schedule and published literature), FEV1 % predicted and pulmonary exacerbation values (clinical trials), and cost to treat pulmonary exacerbations (published literature). The outcomes in the CEA included total cost of therapy; average cost-effectiveness ratio (ACER), defined as cost per FEV1 % predicted; and incremental cost-effectiveness ratio (ICER), defined as the difference in the ratio of cost per FEV1 % predicted of lumacaftor/ivacaftor and placebo. Outcomes in the BIA included total budget impact; cost per member per month (PMPM), defined as total budget impact per hypothetical plan population; and cost per treated member per month (PTMPM), defined as total budget impact per target CF population. All costs were adjusted to 2016 dollars, and one-way sensitivity analyses were conducted to test the model robustness given uncertainty in model inputs and study assumptions., Results: The annual cost of therapy per patient for lumacaftor/ivacaftor was $379,780. The ACER for lumacaftor/ivacaftor was $151,912, while the ICER for lumacaftor/ivacaftor compared with placebo was $95,016 per FEV1 % predicted. The annual total budget impact due to the inclusion of lumacaftor/ivacaftor on the health plan formulary was $266,046. The PMPM cost was $0.02 and the PTMPM cost was $6.21., Conclusions: In patients with CF, lumacaftor/ivacaftor has demonstrated better clinical effectiveness compared with placebo alongside an increased drug acquisition cost. However, the therapy may be a viable alternative to existing standard therapy over a short time horizon. Health care payers, both private and public, need to evaluate the cost-effectiveness and the financial effect when considering expansion of new drug coverage in CF management., Disclosures: No outside funding supported this study. Covvey and Kamal have received research funding from Novartis Pharmaceuticals. Covvey, Giannetti, and Kamal have received research funding from the College of Psychiatric and Neurologic Pharmacists. Kamal serves as a consultant to the Lynx Group (Cranbury, NJ) and Manticore Consulting Group (Scottsdale, AZ). Mukherjee has nothing to disclose. A related poster abstract was presented at the AMCP Managed Care & Specialty Pharmacy Annual Meeting; March 27-30, 2017; Denver, CO.- Published
- 2018
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15. Enumerating the economic cost of antimicrobial resistance per antibiotic consumed to inform the evaluation of interventions affecting their use.
- Author
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Shrestha P, Cooper BS, Coast J, Oppong R, Do Thi Thuy N, Phodha T, Celhay O, Guerin PJ, Wertheim H, and Lubell Y
- Subjects
- Anti-Bacterial Agents therapeutic use, Bacteria classification, Bacteria drug effects, Bacteria genetics, Bacteria isolation & purification, Bacterial Infections microbiology, Carbapenems economics, Carbapenems therapeutic use, Drug Utilization economics, Humans, Macrolides economics, Macrolides therapeutic use, Quinolones economics, Quinolones therapeutic use, Thailand, United States, Anti-Bacterial Agents economics, Bacterial Infections drug therapy, Bacterial Infections economics, Drug Resistance, Bacterial
- Abstract
Background: Antimicrobial resistance (AMR) poses a colossal threat to global health and incurs high economic costs to society. Economic evaluations of antimicrobials and interventions such as diagnostics and vaccines that affect their consumption rarely include the costs of AMR, resulting in sub-optimal policy recommendations. We estimate the economic cost of AMR per antibiotic consumed, stratified by drug class and national income level., Methods: The model is comprised of three components: correlation coefficients between human antibiotic consumption and subsequent resistance; the economic costs of AMR for five key pathogens; and consumption data for antibiotic classes driving resistance in these organisms. These were used to calculate the economic cost of AMR per antibiotic consumed for different drug classes, using data from Thailand and the United States (US) to represent low/middle and high-income countries., Results: The correlation coefficients between consumption of antibiotics that drive resistance in S. aureus , E. coli, K. pneumoniae , A. baumanii, and P. aeruginosa and resistance rates were 0.37, 0.27, 0.35, 0.45, and 0.52, respectively. The total economic cost of AMR due to resistance in these five pathogens was $0.5 billion and $2.9 billion in Thailand and the US, respectively. The cost of AMR associated with the consumption of one standard unit (SU) of antibiotics ranged from $0.1 for macrolides to $0.7 for quinolones, cephalosporins and broad-spectrum penicillins in the Thai context. In the US context, the cost of AMR per SU of antibiotic consumed ranged from $0.1 for carbapenems to $0.6 for quinolones, cephalosporins and broad spectrum penicillins., Conclusion: The economic costs of AMR per antibiotic consumed were considerable, often exceeding their purchase cost. Differences between Thailand and the US were apparent, corresponding with variation in the overall burden of AMR and relative prevalence of different pathogens. Notwithstanding their limitations, use of these estimates in economic evaluations can make better-informed policy recommendations regarding interventions that affect antimicrobial consumption and those aimed specifically at reducing the burden of AMR., Competing Interests: Not applicable. Not applicable. The authors declare that they have no competing interests. Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
- Published
- 2018
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16. Cost-effectiveness of indacaterol/glycopyrronium in comparison with salmeterol/fluticasone combination for patients with moderate-to-severe chronic obstructive pulmonary disease: a LANTERN population analysis from Singapore.
- Author
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Tee A, Chow WL, Burke C, and Guruprasad B
- Subjects
- Aged, Bronchodilator Agents, Cohort Studies, Computer Simulation, Cost-Benefit Analysis, Drug Combinations, Drug Costs, Female, Fluticasone-Salmeterol Drug Combination administration & dosage, Forced Expiratory Volume, Glycopyrrolate administration & dosage, Hospitals, Humans, Indans administration & dosage, Male, Middle Aged, Probability, Quality-Adjusted Life Years, Quinolones administration & dosage, Singapore epidemiology, Fluticasone-Salmeterol Drug Combination economics, Glycopyrrolate economics, Indans economics, Pulmonary Disease, Chronic Obstructive drug therapy, Pulmonary Disease, Chronic Obstructive economics, Quinolones economics
- Abstract
Introduction: In light of the growing evidence base for better clinical results with the use of the dual bronchodilator indacaterol/glycopyrronium (IND/GLY) over inhaled corticosteroid-containing salmeterol/fluticasone combination (SFC), this study aimed to evaluate the cost-effectiveness of IND/GLY over SFC in patients with moderate-to-severe chronic obstructive pulmonary disease (COPD) who are at low risk of exacerbations, in the Singapore healthcare setting., Methods: A previously published patient-level simulation model was adapted for use in Singapore by applying local unit costs. The model was populated with clinical data from the LANTERN and ECLIPSE studies. Both costs and health outcomes were predicted for the lifetime horizon from a payer's perspective and were discounted at 3% per annum. Costs were expressed in 2015 USD exchange rates. Uncertainty was assessed through probabilistic sensitivity analysis., Results: Compared to SFC, use of IND/GLY increased mean life expectancy by 0.316 years and mean quality-adjusted life years (QALYs) by 0.246 years, and decreased mean total treatment costs (drug costs and management of associated events) by USD 1,474 over the entire lifetime horizon. IND/GLY was considered to be 100% cost-effective at a threshold of 1 × gross domestic product per capita. The cost-effectiveness acceptability curve showed that IND/GLY was 100% cost-effective at a willingness-to-pay threshold of USD 0 (additional cost) when compared to SFC., Conclusion: IND/GLY was estimated to be highly cost-effective compared to SFC in patients with moderate-to-severe COPD who are not at high risk of exacerbations in the Singapore healthcare setting., (Copyright: © Singapore Medical Association.)
- Published
- 2018
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17. Cost-effectiveness analysis of a fixed-dose combination of indacaterol and glycopyrronium as maintenance treatment for COPD.
- Author
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Chan MC, Tan EC, and Yang MC
- Subjects
- Adrenergic beta-2 Receptor Agonists adverse effects, Adult, Bronchodilator Agents adverse effects, Computer Simulation, Cost-Benefit Analysis, Disease Progression, Drug Combinations, Female, Forced Expiratory Volume, Glycopyrrolate adverse effects, Humans, Indans adverse effects, Lung physiopathology, Male, Models, Economic, Muscarinic Antagonists adverse effects, Pulmonary Disease, Chronic Obstructive diagnosis, Pulmonary Disease, Chronic Obstructive physiopathology, Quality of Life, Quality-Adjusted Life Years, Quinolones adverse effects, Severity of Illness Index, Taiwan, Time Factors, Treatment Outcome, Adrenergic beta-2 Receptor Agonists administration & dosage, Adrenergic beta-2 Receptor Agonists economics, Bronchodilator Agents administration & dosage, Bronchodilator Agents economics, Drug Costs, Glycopyrrolate administration & dosage, Glycopyrrolate economics, Indans administration & dosage, Indans economics, Lung drug effects, Muscarinic Antagonists administration & dosage, Muscarinic Antagonists economics, Pulmonary Disease, Chronic Obstructive drug therapy, Pulmonary Disease, Chronic Obstructive economics, Quinolones administration & dosage, Quinolones economics
- Abstract
Objective: The aim of this study was to evaluate the cost-effectiveness of the long-acting beta-2 agonist (LABA)/long-acting muscarinic antagonist (LAMA) dual bronchodilator indacaterol/glycopyrronium (IND/GLY) as a maintenance treatment for COPD patients from the perspective of health care payer in Taiwan., Patients and Methods: We adopted a patient-level simulation model, which included a cohort of COPD patients aged ≥40 years. The intervention used in the study was the treatment using IND/GLY, and comparators were tiotropium or salmeterol/fluticasone combination (SFC). Data related to the efficacy of drugs, incidence of exacerbation, and utility were obtained from clinical studies. Direct costs were estimated from claims data based on the severity of COPD. The cycle length was 6 months (to match forced expiratory volume in 1 second [FEV
1 ] data), and the time horizons included 1, 3, 5, 10 years, and lifetime. Deterministic and probabilistic sensitivity analyses were conducted to test the robustness of the model results. Costs were expressed in US dollars with a discount rate of 3.0%., Results: Compared to tiotropium and SFC, the incremental cost-effectiveness ratios (ICERs) per quality-adjusted life year (QALY) gained of patients treated with IND/GLY were US$5,987 and US$14,990, respectively. One-way sensitivity analysis revealed that the improvement in FEV1 provided by IND/GLY, the distribution of patients with regard to the severity of COPD, and acute exacerbation rate ratio were the key drivers behind cost-effectiveness. Adopting a willingness to pay of US$60,000 per QALY gained as the threshold, there was a 98.7% probability that IND/GLY was cost-effective compared to tiotropium. Similarly, there was a 99.9% probability that IND/GLY was cost-effective compared to SFC., Conclusion: As a maintenance treatment for COPD, we consider the dual bronchodilator IND/GLY as a cost-effective strategy when compared to either tiotropium or SFC., Competing Interests: Disclosure The authors report no conflicts of interest in this work.- Published
- 2018
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18. Indacaterol/glycopyrronium is cost-effective compared to salmeterol/fluticasone in COPD: FLAME-based modelling in a Swedish population.
- Author
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Bjermer L, van Boven JFM, Costa-Scharplatz M, Keininger DL, Gutzwiller FS, Lisspers K, Mahon R, Olsson P, and Roche N
- Subjects
- Aged, Double-Blind Method, Drug Combinations, Female, Fluticasone-Salmeterol Drug Combination administration & dosage, Glycopyrrolate administration & dosage, Humans, Indans administration & dosage, Male, Middle Aged, Pulmonary Disease, Chronic Obstructive drug therapy, Pulmonary Disease, Chronic Obstructive epidemiology, Quinolones administration & dosage, Sweden epidemiology, Cost-Benefit Analysis methods, Fluticasone-Salmeterol Drug Combination economics, Glycopyrrolate economics, Indans economics, Models, Economic, Population Surveillance methods, Pulmonary Disease, Chronic Obstructive economics, Quinolones economics
- Abstract
Background: This study assessed the cost-effectiveness of indacaterol/glycopyrronium (IND/GLY) versus salmeterol/fluticasone (SFC) in chronic obstructive pulmonary disease (COPD) patients with moderate to very severe airflow limitation and ≥1 exacerbation in the preceding year., Methods: A previously published and validated patient-level simulation model was adapted using clinical data from the FLAME trial and real-world cost data from the ARCTIC study. Costs (total monetary costs comprising drug, maintenance, exacerbation, and pneumonia costs) and health outcomes (life-years (LYs), quality-adjusted life-years (QALYs)) were projected over various time horizons (1, 5, 10 years, and lifetime) from the Swedish payer's perspective and were discounted at 3% annually. Uncertainty in model input values was studied through one-way and probabilistic sensitivity analyses. Subgroup analyses were also performed., Results: IND/GLY was associated with lower costs and better outcomes compared with SFC over all the analysed time horizons. Use of IND/GLY resulted in additional 0.192 LYs and 0.134 QALYs with cost savings of €1211 compared with SFC over lifetime. The net monetary benefit (NMB) was estimated to be €8560 based on a willingness-to-pay threshold of €55,000/QALY. The NMB was higher in the following subgroups: severe (GOLD 3), high risk and more symptoms (GOLD D), females, and current smokers., Conclusion: IND/GLY is a cost-effective treatment compared with SFC in COPD patients with mMRC dyspnea grade ≥ 2, moderate to very severe airflow limitation, and ≥1 exacerbation in the preceding year.
- Published
- 2017
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19. Forecasting the Long-Term Clinical and Economic Outcomes of Lumacaftor/Ivacaftor in Cystic Fibrosis Patients with Homozygous phe508del Mutation.
- Author
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Dilokthornsakul P, Patidar M, and Campbell JD
- Subjects
- Adult, Aminophenols economics, Aminopyridines economics, Benzodioxoles economics, Cystic Fibrosis genetics, Cystic Fibrosis physiopathology, Drug Combinations, Forced Expiratory Volume, Homozygote, Humans, Markov Chains, Mutation, Quinolones economics, Severity of Illness Index, Treatment Outcome, United States, Aminophenols administration & dosage, Aminopyridines administration & dosage, Benzodioxoles administration & dosage, Cystic Fibrosis drug therapy, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Quality-Adjusted Life Years, Quinolones administration & dosage
- Abstract
Objectives: To forecast lifetime outcomes and cost of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis (CF) with homozygous phe508del mutation from the US payer perspective., Methods: A lifetime Markov model was developed from a US payer perspective. The model included five health states: 1) mild lung disease (percent predicted forced expiratory volume in 1 second [FEV
1 ] >70%), 2) moderate lung disease (40% ≤ FEV1 ≤ 70%), 3) severe lung disease (FEV1 < 40%), 4) lung transplantation, and 5) death. All inputs were derived from published literature. We estimated lumacaftor/ivacaftor's improvement in outcomes compared with a non-CF referent population as well as CF-specific mortality estimates., Results: Lumacaftor/ivacaftor was associated with additional 2.91 life-years (95% credible interval 2.55-3.56) and additional 2.42 quality-adjusted life-years (QALYs) (95% credible interval 2.10-2.98). Lumacaftor/ivacaftor was associated with improvements in survival and QALYs equivalent to 27.6% and 20.7%, respectively, for the survival and QALY gaps between CF usual care and their non-CF peers. The incremental lifetime cost was $2,632,249., Conclusions: Lumacaftor/ivacaftor increased life-years and QALYs in CF patients with the homozygous phe508del mutation and moved morbidity and mortality closer to that of their non-CF peers but it came with higher cost., (Copyright © 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)- Published
- 2017
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20. The pharmacokinetic interaction between ivacaftor and ritonavir in healthy volunteers.
- Author
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Liddy AM, McLaughlin G, Schmitz S, D'Arcy DM, and Barry MG
- Subjects
- Adult, Aminophenols economics, Aminophenols therapeutic use, Area Under Curve, Chloride Channel Agonists economics, Chloride Channel Agonists therapeutic use, Chromatography, Liquid methods, Cross-Over Studies, Cystic Fibrosis drug therapy, Drug Administration Schedule, Drug Interactions, Female, Half-Life, Healthy Volunteers, Humans, Male, Quinolones economics, Quinolones therapeutic use, Tandem Mass Spectrometry methods, Young Adult, Aminophenols pharmacokinetics, Chloride Channel Agonists pharmacokinetics, Cytochrome P-450 CYP3A metabolism, Cytochrome P-450 CYP3A Inhibitors pharmacokinetics, Quinolones pharmacokinetics, Ritonavir pharmacokinetics
- Abstract
Aims: The aim of this study was to determine the pharmacokinetic interaction between ivacaftor and ritonavir., Methods: A liquid chromatography mass spectrometry (LC-MS) method was developed for the measurement of ivacaftor in plasma. An open-label, sequential, cross-over study was conducted with 12 healthy volunteers. Three pharmacokinetic profiles were assessed for each volunteer: ivacaftor 150 mg alone (study A), ivacaftor 150 mg plus ritonavir 50 mg daily (study B), and ivacaftor 150 mg plus ritonavir 50 mg daily after two weeks of ritonavir 50 mg daily (study C)., Results: Addition of ritonavir 50 mg daily to ivacaftor 150 mg resulted in significant inhibition of the metabolism of ivacaftor. Area under the plasma concentration-time curve from time 0 to infinity (AUC
0-inf obv ) increased significantly in both studies B and C compared to study A (GMR [95% CI] 19.71 [13.18-31.33] and 19.77 [14.0-27.93] respectively). Elimination half-life (t1/2 ) was significantly longer in both studies B and C compared to study A (GMR [95% CI] 11.14 [8.72-13.62] and 9.72 [6.68-12.85] respectively). There was no significant difference in any of the pharmacokinetic parameters between study B and study C., Conclusion: Ritonavir resulted in significant inhibition of the metabolism of ivacaftor. These data suggest that ritonavir may be used to inhibit the metabolism of ivacaftor in patients with cystic fibrosis (CF). Such an approach may increase the effectiveness of ivacaftor in 'poor responders' by maintaining higher plasma concentrations. It also has the potential to significantly reduce the cost of ivacaftor therapy., (© 2017 The British Pharmacological Society.)- Published
- 2017
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21. Ivacaftor for cystic fibrosis: An evaluation of real world utilisation and expenditure in the Irish Healthcare Setting.
- Author
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Corcoran A, Hickey N, Barry M, Usher C, and McCullagh LM
- Subjects
- Adolescent, Adult, Aminophenols economics, Child, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Female, Forced Expiratory Volume, Health Expenditures, Humans, Ireland, Male, Mutation, Quinolones economics, Young Adult, Aminophenols therapeutic use, Cystic Fibrosis drug therapy, Quinolones therapeutic use
- Abstract
In Ireland, Ivacaftor is reimbursed, on the High-Tech Drug Scheme, for the treatment of cystic fibrosis in patients age 6 years and older who have the G551D mutation. The aim of this study was to analyse the utilisation and expenditure of Ivacaftor on this scheme in the 12 month period post-reimbursement. All patients who had received Ivacaftor (regardless of General Medical Services Scheme eligibility/ineligibility) were included. A total of 140 individuals (male=74; 53%) received Ivacaftor over the defined 12 month study period (from January 2015 to December 2015 inclusive). The cohort ranged in age from 6 years to 61 years. The mean age was 22 years; a positive skew in age distribution indicated that a greater number of the cohort were in the younger age groups. No statistically significant difference was detected in the mean ages of the male and female subgroups. Drug acquisition expenditure by the Health Services Executive on Ivacaftor over the 12 month study period was €29.81 million.
- Published
- 2017
22. Cost-effectiveness of brexpiprazole adjunctive treatment for major depressive disorder.
- Author
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Sussman M, Yu J, Kamat SA, Hartry A, Legacy S, Duffy R, and Aigbogun MS
- Subjects
- Adolescent, Adult, Aged, Akathisia, Drug-Induced economics, Akathisia, Drug-Induced etiology, Antipsychotic Agents therapeutic use, Benzodiazepines therapeutic use, Cost-Benefit Analysis, Depressive Disorder, Major economics, Disorders of Excessive Somnolence chemically induced, Disorders of Excessive Somnolence economics, Drug Costs, Drug Therapy, Combination, Fatigue chemically induced, Fatigue economics, Female, Health Care Costs, Humans, Male, Middle Aged, Models, Economic, Olanzapine, Patient Selection, Quetiapine Fumarate therapeutic use, Quinolones economics, Serotonin Agents economics, Thiophenes economics, Weight Gain, Young Adult, Antidepressive Agents therapeutic use, Depressive Disorder, Major drug therapy, Quinolones therapeutic use, Serotonin Agents therapeutic use, Thiophenes therapeutic use
- Abstract
Background: Major depressive disorder (MDD) is a debilitating psychiatric illness with a high cost burden. This analysis evaluates the cost-effectiveness of adjunctive brexpiprazole versus comparator branded adjunctive treatment for MDD and background antidepressant therapy (ADT) alone from a US payer perspective., Methods: An economic model was developed to assess the cost-effectiveness of brexpiprazole versus comparator adjunctive treatment and ADT alone on total direct medical costs using a 6-week cycle time frame for a total of 48 weeks, with treatment response and remission as primary outcomes. The model consisted of 3 parts, 1 to represent the acute treatment phase and 2 to represent the maintenance stage., Results: In the base-case analysis, brexpiprazole as reference treatment resulted in cost per additional responder ranging from $19,442-$48,745 and cost per additional remitter ranging from $27,196-$71,839 versus comparator treatments over 48 weeks. Sensitivity analyses showed treatment with brexpiprazole was more costly, but more clinically effective in all probabilistic simulations., Limitations: This representation of disease natural history over 48 weeks may not account for all possible health states. Resource utilization on treatment was estimated using the resource use data from previous trials, and may overestimate medical costs compared to the real-world setting. Treatment comparators were limited to branded therapies, and head-to-head studies were not available to obtain data inputs., Conclusion: Compared to other branded adjunctive therapies, brexpiprazole increases response and remission at 6 weeks; medical care cost savings were observed with the use of brexpiprazole. These findings may assist clinicians and formulary decision makers when selecting treatment for MDD., (Copyright © 2016 The Authors. Published by Elsevier B.V. All rights reserved.)
- Published
- 2017
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23. Cost Effectiveness of the Long-Acting β2-Adrenergic Agonist (LABA)/Long-Acting Muscarinic Antagonist Dual Bronchodilator Indacaterol/Glycopyrronium Versus the LABA/Inhaled Corticosteroid Combination Salmeterol/Fluticasone in Patients with Chronic Obstructive Pulmonary Disease: Analyses Conducted for Canada, France, Italy, and Portugal.
- Author
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Reza Maleki-Yazdi M, Molimard M, Keininger DL, Gruenberger JB, Carrasco J, Pitotti C, Sauvage E, Chehab S, and Price D
- Subjects
- Administration, Inhalation, Aged, Bronchodilator Agents economics, Canada, Cost-Benefit Analysis, Drug Costs, Drug Therapy, Combination, Fluticasone administration & dosage, Fluticasone economics, France, Glycopyrrolate administration & dosage, Glycopyrrolate economics, Health Care Costs, Humans, Indans administration & dosage, Indans economics, Italy, Male, Portugal, Pulmonary Disease, Chronic Obstructive economics, Quinolones administration & dosage, Quinolones economics, Salmeterol Xinafoate administration & dosage, Salmeterol Xinafoate economics, Bronchodilator Agents therapeutic use, Fluticasone therapeutic use, Glycopyrrolate therapeutic use, Indans therapeutic use, Pulmonary Disease, Chronic Obstructive drug therapy, Quinolones therapeutic use, Salmeterol Xinafoate therapeutic use
- Abstract
Objective: The objective of this study was to assess the cost effectiveness of the dual bronchodilator indacaterol/glycopyrronium (IND/GLY) compared with salmeterol/fluticasone combination (SFC) in patients with moderate-to-severe chronic obstructive pulmonary disease (COPD) who had a history of one or no exacerbations in the previous year, in Canada, France, Italy, and Portugal., Methods: A patient-level simulation was developed to compare the costs and outcomes of IND/GLY versus SFC based on data from the LANTERN trial (NCT01709903). Monte-Carlo simulation methods were employed to follow individual patients over various time horizons. Population and efficacy inputs were derived from the LANTERN trial. Considering the payers' perspective, only direct costs were included. Costs and health outcomes were discounted annually at 3.0 % for all countries. Unit costs were taken from publically available sources with all costs converted to euros (€). The cost base year was 2015. Deterministic and probabilistic sensitivity analyses were undertaken to test the robustness of the model results., Results: IND/GLY was found to be the dominant (more effective and less costly) treatment option compared with SFC in all four countries. The use of IND/GLY was associated with mean total cost savings per patient over a lifetime of €6202, €1974, €1611, and €220 in Canada, France, Italy, and Portugal, respectively. Sensitivity analysis showed that exacerbation rates had the largest impact on incremental costs and quality-adjusted life-years (QALYs). The probability of IND/GLY being cost effective was estimated to be >95 % for thresholds above €5000/QALY., Conclusion: In patients with moderate to severe COPD, IND/GLY is likely to be a cost-effective treatment alternative compared with SFC.
- Published
- 2016
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24. Controversies with Kalydeco: Newspaper coverage in Canada and the United States of the cystic fibrosis "wonder drug".
- Author
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Rachul C, Toews M, and Caulfield T
- Subjects
- Canada, Cross-Cultural Comparison, Humans, Reimbursement Mechanisms statistics & numerical data, United States, Aminophenols economics, Aminophenols therapeutic use, Cystic Fibrosis drug therapy, Cystic Fibrosis economics, Drug Costs, Health Services Accessibility statistics & numerical data, Newspapers as Topic statistics & numerical data, Orphan Drug Production economics, Quinolones economics, Quinolones therapeutic use
- Abstract
Background: The cystic fibrosis drug, Kalydeco, has attracted attention both for its effectiveness in particular CF patients and its substantial price tag. An analysis of newspaper portrayals of Kalydeco provides an opportunity to examine how policy issues associated with rare diseases and orphan drugs are being represented in the popular press., Methods: We conducted a content analysis of 203 newspaper articles in Canada and the U.S. that mention Kalydeco. Articles were analyzed for their main frame, discussion of Kalydeco, including issues of drug development, patient access, and reimbursement, and overall tone., Results: In Canadian newspaper coverage, 77.4% of articles were framed as human interest stories featuring individual patients seeking public funding for Kalydeco, yet only 7.5% mentioned any budgetary limitations in doing so. In contrast, U.S. newspaper coverage was framed as a financial/economic story in 43.1% of articles and a medical/scientific story in 27.8%., Conclusions: Newspaper coverage varied significantly between Canada, where Kalydeco is predominantly a story about increasing patient access through full government funding, and the U.S., where Kalydeco is largely a financial story about the economic impact of Kalydeco. The difference in coverage may be due to differences in public funding between the healthcare systems of these two countries., (Copyright © 2016 The Authors. Published by Elsevier B.V. All rights reserved.)
- Published
- 2016
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25. Healthcare resource utilization associated with ivacaftor use in patients with cystic fibrosis.
- Author
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Suthoff ED, Bonafede M, Limone B, O'Callaghan L, Sawicki GS, and Wagener JS
- Subjects
- Adolescent, Adult, Age Factors, Aminophenols therapeutic use, Child, Chloride Channel Agonists therapeutic use, Costs and Cost Analysis, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Female, Health Expenditures statistics & numerical data, Humans, Insurance, Health statistics & numerical data, Male, Middle Aged, Mutation, Quinolones therapeutic use, Retrospective Studies, Sex Factors, Young Adult, Aminophenols economics, Chloride Channel Agonists economics, Cystic Fibrosis drug therapy, Cystic Fibrosis economics, Health Services economics, Health Services statistics & numerical data, Quinolones economics
- Abstract
Objective: Ivacaftor was approved in 2012 to treat patients with cystic fibrosis (CF) with specific CFTR gene mutations. The objective of this analysis was to analyze the impact of ivacaftor on health resource utilization through analysis of claims data., Methods: Patients diagnosed with CF aged ≥6 years prescribed ivacaftor between January 1, 2012 and July 31, 2014 with ≥12 months of continuous insurance coverage prior to and following the prescription were identified. All-cause and CF-specific healthcare resource utilization during the pre- and post-prescription periods and ivacaftor adherence levels were studied., Results: The 79 identified patients had a mean age of 20.8 years, and 54% were female. The proportion of patients with inpatient admissions (all-cause and CF-related) was significantly higher in the pre index compared to post index period (p ≤ 0.05). Mean ivacaftor medication possession ratio was 0.8 (SD = 0.3), and 73% of patients had a medication possession ratio >0.80., Limitations: Only a small number of patients met the inclusion criteria. Additionally, claims data may contain errors or inconsistencies and cannot be used to determine if medications were taken as prescribed., Conclusions: Ivacaftor therapy was associated with significant reductions in hospitalizations along with high rates of adherence to treatment over 12 months.
- Published
- 2016
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26. Cystic fibrosis drug is not cost effective, says NICE.
- Author
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Gulland A
- Subjects
- Aminophenols economics, Aminopyridines economics, Benzodioxoles economics, Cost-Benefit Analysis, Cystic Fibrosis economics, Humans, Quinolones economics, Respiratory System Agents economics, Aminophenols therapeutic use, Aminopyridines therapeutic use, Benzodioxoles therapeutic use, Cystic Fibrosis drug therapy, Quinolones therapeutic use, Respiratory System Agents therapeutic use
- Published
- 2016
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27. Promising gene therapies pose million-dollar conundrum.
- Author
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Check Hayden E
- Subjects
- Aminophenols economics, Anemia, Sickle Cell economics, Anemia, Sickle Cell genetics, Anemia, Sickle Cell therapy, Antibodies, Monoclonal economics, Antibodies, Monoclonal, Humanized, Biotechnology economics, Biotechnology trends, Cystic Fibrosis drug therapy, Cystic Fibrosis economics, Drug Industry economics, Genetic Therapy statistics & numerical data, Genetic Therapy trends, Humans, Insurance, Health economics, Neoplasms economics, Neoplasms genetics, Neoplasms immunology, Neoplasms therapy, Quinolones economics, Sofosbuvir economics, Drug Costs, Genetic Therapy economics
- Published
- 2016
- Full Text
- View/download PDF
28. Forecasting US ivacaftor outcomes and cost in cystic fibrosis patients with the G551D mutation.
- Author
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Dilokthornsakul P, Hansen RN, and Campbell JD
- Subjects
- Aminophenols economics, Cohort Studies, Computer Simulation, Cost-Benefit Analysis, Cystic Fibrosis economics, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Drug Therapy trends, Forecasting, Health Status, Humans, Lung Transplantation, Markov Chains, Monte Carlo Method, Quality-Adjusted Life Years, Quinolones economics, Respiratory Function Tests, Treatment Outcome, United States, Aminophenols therapeutic use, Cystic Fibrosis drug therapy, Cystic Fibrosis genetics, Forced Expiratory Volume drug effects, Mutation, Quinolones therapeutic use
- Abstract
Ivacaftor, a breakthrough treatment for cystic fibrosis (CF) patients with the G551D genetic mutation, lacks long-term clinical and cost projections. This study forecasted outcomes and cost by comparing ivacaftor plus usual care versus usual care alone.A lifetime Markov model was conducted from a US payer perspective. The model consisted of five health states: 1) forced expiratory volume in 1 s (FEV1) % pred ≥70%, 2) 40%≤ FEV1 % pred <70%, 3) FEV1 % pred <40%, 4) lung transplantation and 5) death. All inputs were extracted from published literature. Budget impact was also estimated. We estimated ivacaftor's improvement in outcomes compared with a non-CF referent population.Ivacaftor was associated with 18.25 (95% credible interval (CrI) 13.71-22.20) additional life-years and 15.03 (95% CrI 11.13-18.73) additional quality-adjusted life-years (QALYs). Ivacaftor was associated with improvements in survival and QALYs equivalent to 68% and 56%, respectively, for the survival and QALY gaps between CF usual care and their non-CF peers. The incremental lifetime cost was $3 374 584. The budget impact was $0.087 per member per month.Ivacaftor increased life-years and QALYs in CF patients with the G551D mutation, and moved morbidity and mortality closer to that of their non-CF peers. Ivacaftor costs much more than usual care, but comes at a relatively limited budget impact., (Copyright ©ERS 2016.)
- Published
- 2016
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29. Lumacaftor/ivacaftor (Orkambi) for cystic fibrosis.
- Subjects
- Administration, Oral, Aminophenols administration & dosage, Aminophenols adverse effects, Aminophenols economics, Aminophenols pharmacokinetics, Aminopyridines administration & dosage, Aminopyridines adverse effects, Aminopyridines economics, Aminopyridines pharmacokinetics, Benzodioxoles administration & dosage, Benzodioxoles adverse effects, Benzodioxoles economics, Benzodioxoles pharmacokinetics, Cystic Fibrosis diagnosis, Cystic Fibrosis economics, Cystic Fibrosis genetics, Cystic Fibrosis Transmembrane Conductance Regulator drug effects, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Drug Combinations, Drug Costs, Drug Interactions, Humans, Membrane Transport Modulators administration & dosage, Membrane Transport Modulators adverse effects, Membrane Transport Modulators economics, Membrane Transport Modulators pharmacokinetics, Mutation, Quinolones administration & dosage, Quinolones adverse effects, Quinolones economics, Quinolones pharmacokinetics, Risk Factors, Treatment Outcome, Aminophenols therapeutic use, Aminopyridines therapeutic use, Benzodioxoles therapeutic use, Cystic Fibrosis drug therapy, Membrane Transport Modulators therapeutic use, Quinolones therapeutic use
- Published
- 2016
30. Precision Medicine: At What Price?
- Author
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Ferkol T and Quinton P
- Subjects
- Aminophenols therapeutic use, Aminopyridines therapeutic use, Benzodioxoles therapeutic use, Cost-Benefit Analysis, Cystic Fibrosis economics, Cystic Fibrosis genetics, Drug Combinations, Humans, Quinolones therapeutic use, Respiratory System Agents therapeutic use, United States, Aminophenols economics, Aminopyridines economics, Benzodioxoles economics, Cystic Fibrosis drug therapy, Precision Medicine economics, Quinolones economics, Respiratory System Agents economics
- Published
- 2015
- Full Text
- View/download PDF
31. Orkambi's Slick Unveiling Puts Insurers in a Bind.
- Author
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Silverman E
- Subjects
- Cystic Fibrosis drug therapy, Drug Combinations, Humans, United States, Aminophenols economics, Aminopyridines economics, Benzodioxoles economics, Drug Costs, Drug Industry economics, Insurance Coverage economics, Insurance, Pharmaceutical Services, Quinolones economics
- Published
- 2015
32. Settlement reached over medicaid coverage of cystic fibrosis drug: Arkansas federal court case highlights medical necessity, high cost of targeted therapies.
- Subjects
- Aminophenols therapeutic use, Arkansas, Cystic Fibrosis economics, Health Services Accessibility legislation & jurisprudence, Humans, Poverty, Quinolones therapeutic use, United States, Aminophenols economics, Cystic Fibrosis drug therapy, Medicaid, Patient Rights legislation & jurisprudence, Quinolones economics
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- 2015
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33. Foundation receives $3.3-billion windfall for Kalydeco.
- Author
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Senior M
- Subjects
- Aminophenols therapeutic use, Cystic Fibrosis drug therapy, Cystic Fibrosis Transmembrane Conductance Regulator therapeutic use, Humans, Quinolones therapeutic use, Aminophenols economics, Cystic Fibrosis Transmembrane Conductance Regulator economics, Financing, Organized, Foundations, Quinolones economics
- Published
- 2015
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34. Cost-effectiveness of the LABA/LAMA dual bronchodilator indacaterol/glycopyrronium in a Swedish healthcare setting.
- Author
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Price D, Keininger D, Costa-Scharplatz M, Mezzi K, Dimova M, Asukai Y, and Ställberg B
- Subjects
- Aged, Albuterol analogs & derivatives, Albuterol economics, Albuterol therapeutic use, Androstadienes economics, Androstadienes therapeutic use, Bronchodilator Agents administration & dosage, Bronchodilator Agents therapeutic use, Cost-Benefit Analysis, Double-Blind Method, Drug Combinations, Drug Costs statistics & numerical data, Drug Therapy, Combination, Female, Fluticasone, Forced Expiratory Volume drug effects, Glycopyrrolate administration & dosage, Glycopyrrolate therapeutic use, Humans, Indans administration & dosage, Indans therapeutic use, Male, Middle Aged, Monte Carlo Method, Pulmonary Disease, Chronic Obstructive economics, Pulmonary Disease, Chronic Obstructive physiopathology, Quinolones administration & dosage, Quinolones therapeutic use, Salmeterol Xinafoate, Sweden, Bronchodilator Agents economics, Glycopyrrolate economics, Indans economics, Pulmonary Disease, Chronic Obstructive drug therapy, Quinolones economics
- Abstract
Background: Indacaterol/glycopyrronium (IND/GLY) is a once-daily inhaled fixed-dose combination of indacaterol (IND), a long-acting β2-adrenergic agonist (LABA), and glycopyrronium (GLY), a long-acting muscarinic antagonist (LAMA) for use as maintenance treatment to relieve symptoms of chronic obstructive pulmonary disease (COPD) in adults., Objective: To determine the economic benefits of IND/GLY compared with the free combination of indacaterol and glycopyrronium (IND + GLY), and with the fixed-dose combination of salmeterol/fluticasone (SFC), in a moderate-to-severe COPD population with low-exacerbation risk. The model-based analysis extrapolated results up to lifetime time horizon and calculated costs per quality-adjusted life year., Methods: Assuming equal efficacy, a cost-minimisation analysis compared IND/GLY vs IND + GLY using model inputs from the double-blind, randomised SHINE trial. The double-blind, randomised ILLUMINATE and TORCH trials were used to analyse cost-effectiveness versus SFC. To consider ICS-related pneumonia events, published odds ratio comparing an ICS-exposed group to a control group of COPD patients was used. Direct and indirect drug costs as well as drug acquisition costs (in Swedish Krona [SEK]) were derived from published Swedish sources. Cost and effects were discounted with 3%. Uncertainty was assessed by one-way and probabilistic sensitivity analyses (PSA)., Results: IND/GLY was cost-saving vs IND + GLY with incremental savings of SEK (EUR) 768 (85), and 3309 (368) per patient over one and five years. IND/GLY was found to be less costly and more effective compared to SFC with cost savings of SEK (EUR) 2744 (303), 8854 (976), 13,938 (1536), 27,495 (3031) and 43,033 (4744) over one, three, five, ten years and lifetime. The PSA indicated that all iterations produced dominant results for IND/GLY., Conclusion: IND/GLY is cost-minimising vs IND + GLY and dominates SFC in the maintenance treatment of COPD patients in Sweden. Encouraging dual bronchodilator therapy over an ICS-containing combination results in lower total costs and better outcomes compared to combination therapy including fluticasone in moderate-to-severe COPD patients with low exacerbation risk., (Copyright © 2014 Elsevier Ltd. All rights reserved.)
- Published
- 2014
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35. Cost-effectiveness of aripiprazole once-monthly compared with paliperidone palmitate once-monthly injectable for the treatment of schizophrenia in the United States.
- Author
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Citrome L, Kamat SA, Sapin C, Baker RA, Eramo A, Ortendahl J, Gutierrez B, Hansen K, and Bentley TG
- Subjects
- Antipsychotic Agents adverse effects, Aripiprazole, Decision Support Techniques, Drug Administration Schedule, Humans, Injections, Intramuscular, Isoxazoles administration & dosage, Paliperidone Palmitate, Palmitates administration & dosage, Piperazines administration & dosage, Quinolones administration & dosage, Schizophrenia economics, United States, Antipsychotic Agents administration & dosage, Antipsychotic Agents economics, Cost-Benefit Analysis, Isoxazoles economics, Palmitates economics, Piperazines economics, Quinolones economics, Schizophrenia drug therapy
- Abstract
Objective: To develop a decision-analytic model to estimate the cost-effectiveness of initiating maintenance treatment with aripiprazole once-monthly (AOM) vs paliperidone long-acting injectable (PLAI) once-monthly among patients with schizophrenia in the US., Methods: A decision-analytic model was developed to evaluate a hypothetical cohort of patients initiating maintenance treatment with AOM or PLAI. Rates of relapse, adverse events (AEs), and direct medical costs were estimated for 1 year. Patients either remained on initial treatment or discontinued treatment due to lack of efficacy, AEs, or other reasons, including non-adherence. Data from placebo-controlled pivotal trials and product prescribing information (PI) were used to estimate treatment efficacy and AEs. Analyses were performed assuming dosing of clinical trials, real-world practice, PIs, and highest therapeutic dose available, because of variation in practice settings. The main outcome of interest was incremental cost per schizophrenia hospitalization averted with AOM vs PLAI., Results: Based on placebo-controlled pivotal trials' dosing, AOM improved clinical outcomes by reducing schizophrenia relapses vs PLAI (0.181 vs 0.277 per person per year [pppy]) at an additional cost of US$1276 pppy, resulting in an incremental cost-effectiveness ratio (ICER) of US$13,280/relapse averted. When PI dosing was assumed, this ICER increased to US$19,968/relapse averted. When real-world dosing and highest available dosing were assumed, AOM was associated with fewer relapses and lower overall treatment costs vs PLAI., Conclusions: AOM consistently provided favorable clinical benefits. Under various dosing scenarios, AOM results indicated fewer relapses at lower overall costs or a reasonable cost-effectiveness threshold (i.e., less than the cost of a hospitalization relapse) vs PLAI. Given the heterogeneous nature of schizophrenia and variability in treatment response, health plans may consider open access for treatments like AOM. Since model inputs were based on data from separate placebo-controlled trials, generalization of results to the real-world setting is limited.
- Published
- 2014
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36. Sweat chloride is not a useful marker of clinical response to Ivacaftor.
- Author
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Barry PJ, Jones AM, Webb AK, and Horsley AR
- Subjects
- Aminophenols economics, Biomarkers analysis, Body Height, Body Weight, Cohort Studies, Cystic Fibrosis genetics, Cystic Fibrosis physiopathology, Forced Expiratory Flow Rates, Humans, Lung physiopathology, Prospective Studies, Quinolones economics, Spirometry, Aminophenols therapeutic use, Chlorides analysis, Cystic Fibrosis drug therapy, Quinolones therapeutic use, Sweat chemistry
- Abstract
Clinical trials have revealed that Ivacaftor significantly reduces sweat chloride in patients with cystic fibrosis who carry the G551D mutation. This finding has been incorporated into the commissioning guidelines in the UK with a sweat chloride reduction of 30% or below 60 mmol/L, specified as the main criteria for continued funding of Ivacaftor for individual patients. In a cohort of 24 adults who were prescribed Ivacaftor, there was no correlation between absolute or relative reductions in sweat chloride and improvements in lung function. This questions the validity of sweat chloride as a surrogate marker of clinical efficacy., (Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.)
- Published
- 2014
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37. Cystic Fibrosis Trust's clarification of Cohen and Raftery's article on cystic fibrosis drug development.
- Author
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Owen E
- Subjects
- Humans, Aminophenols economics, Cystic Fibrosis drug therapy, Drug Discovery economics, Drug Industry economics, Foundations economics, Quinolones economics
- Published
- 2014
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38. A new model for drug development using a multi-stakeholder consortium.
- Author
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Hall AK
- Subjects
- Humans, Aminophenols economics, Cystic Fibrosis drug therapy, Drug Discovery economics, Drug Industry economics, Foundations economics, Quinolones economics
- Published
- 2014
- Full Text
- View/download PDF
39. Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis.
- Author
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Whiting P, Al M, Burgers L, Westwood M, Ryder S, Hoogendoorn M, Armstrong N, Allen A, Severens H, and Kleijnen J
- Subjects
- Adolescent, Adult, Age Factors, Child, Cost-Benefit Analysis, England, Female, Humans, Lung Transplantation economics, Male, Models, Economic, Mutation, Quality-Adjusted Life Years, Randomized Controlled Trials as Topic, Respiratory Function Tests, Sex Factors, State Medicine, Aminophenols economics, Aminophenols therapeutic use, Cystic Fibrosis drug therapy, Cystic Fibrosis genetics, Quinolones economics, Quinolones therapeutic use
- Abstract
Background: Ivacaftor (Kalydeco(®), Vertex Pharmaceuticals) is the first of a new class of drugs that target the underlying protein defect in cystic fibrosis (CF). It is aimed at patients with the G551D (glycine to aspartate change in nucleotide 1784 in exon 11) mutation; 5.7% of patients with CF in the UK have this mutation., Objectives: To review the clinical effectiveness and cost-effectiveness of ivacaftor for the treatment of CF in patients aged ≥ 6 years who have the G551D mutation., Methods: Ten databases, including MEDLINE and EMBASE, were searched from inception to July 2012. Studies that evaluated ivacaftor for the treatment of adults and children (≥ 6 years) with at least one G551D mutation were eligible. There were insufficient data to conduct a formal meta-analysis. The manufacturer of ivacaftor, Vertex Pharmaceuticals, submitted a deterministic patient-level simulation model for the assessment of the lifetime cost-effectiveness of ivacaftor. We modified the model where values were not UK-specific or not recent, or where better estimates could be found. The only change to the model structure was the addition of lung transplantations. We changed utility values, annual decline in percentage predicted forced expiratory volume in 1 second (FEV1), and the baseline exacerbation rate, and used data from the CF Registry to estimate the relation between costs, age and percentage predicted FEV1. Estimates of treatment effect of ivacaftor came from the clinical effectiveness review. We modelled three scenarios for the longer-term effects of ivacaftor. We also modelled an 'optimistic' scenario for patients aged < 12 years with little lung damage. We conducted a budget impact analysis to estimate the total cost to the NHS of introducing ivacaftor in England., Results: Three studies were included: a randomised controlled trial (RCT) in adults (n = 167) (≥ 12 years), a RCT in children (n = 26) (6-11 years), and an open-label extension study of the two RCTs. Both RCTs reported significantly greater changes from baseline in all measures of lung function in patients receiving ivacaftor than in those receiving placebo. The mean difference in change in percentage predicted FEV1 was 10.5 [95% confidence interval (CI) 8.5 to 12.5] percentage points in the adults' study and 10.0 (95% CI 4.5 to 15.5) percentage points in the children's study at 48 weeks. Improvements in lung function were seen across all subgroups investigated (age, sex, study region and lung function). There were significantly greater improvements in the ivacaftor group than in the placebo group for all outcomes assessed (exacerbations, quality of life, sweat chloride and weight) with the exception of quality of life in children. Improvements were maintained in the open-label trial. Adverse events were mainly minor and comparable across treatment groups. Both RCTs reported more withdrawals in the placebo group than in the ivacaftor group. The incremental cost-effectiveness ratio varied between £335,000 and £1,274,000 per quality-adjusted life-year gained. The total additional lifetime costs for all eligible CF patients in England ranged from £438M to £479M; the lifetime cost for standard care only was £72M., Conclusions: The available evidence suggests that ivacaftor is a clinically effective treatment for patients with CF and the G551D mutation; the high cost of ivacaftor may prove an obstacle in the uptake of this treatment. The main priority for further research is the long-term effectiveness of ivacaftor., Study Registration: This study is registered as PROSPERO CRD42012002516., Source of Funding: The National Institute for Health Research Health Technology Assessment programme.
- Published
- 2014
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- View/download PDF
40. Paying twice: questions over high cost of cystic fibrosis drug developed with charitable funding.
- Author
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Cohen D and Raftery J
- Subjects
- Aminophenols therapeutic use, Drug Discovery ethics, Drug Discovery organization & administration, Drug Industry ethics, Humans, Quinolones therapeutic use, Research Support as Topic, United Kingdom, United States, Aminophenols economics, Cystic Fibrosis drug therapy, Drug Discovery economics, Drug Industry economics, Foundations economics, Quinolones economics
- Published
- 2014
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- View/download PDF
41. Single-tablet regimens in HIV: does it really make a difference?
- Author
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Aldir I, Horta A, and Serrado M
- Subjects
- Adenine administration & dosage, Adenine analogs & derivatives, Adenine economics, Adenine therapeutic use, Anti-HIV Agents economics, Carbamates administration & dosage, Carbamates economics, Carbamates therapeutic use, Cobicistat, Deoxycytidine administration & dosage, Deoxycytidine analogs & derivatives, Deoxycytidine economics, Deoxycytidine therapeutic use, Drug Administration Schedule, Drug Combinations, Emtricitabine, Humans, Nitriles administration & dosage, Nitriles economics, Nitriles therapeutic use, Organophosphonates administration & dosage, Organophosphonates economics, Organophosphonates therapeutic use, Pyrimidines administration & dosage, Pyrimidines economics, Pyrimidines therapeutic use, Quinolones administration & dosage, Quinolones economics, Quinolones therapeutic use, Reverse Transcriptase Inhibitors administration & dosage, Reverse Transcriptase Inhibitors economics, Reverse Transcriptase Inhibitors therapeutic use, Rilpivirine, Tablets therapeutic use, Tenofovir, Thiazoles administration & dosage, Thiazoles economics, Thiazoles therapeutic use, Anti-HIV Agents administration & dosage, Anti-HIV Agents therapeutic use, HIV Infections drug therapy, HIV-1 drug effects
- Abstract
Objectives: Review of the available data on the currently available single-tablet regimens (STRs), from the analysis of efficacy and safety to the key points of value in terms of adherence, quality of life and pharmacoeconomic evaluation., Methods: For this narrative review, literature searches have been performed in PubMed, IndexRevMed and Cochrane, using the search terms HIV, single-tablet, one-pill, single dose, fixed-dose, and STR. These have been reviewed and complemented with the most recent publications of interest., Results: Fixed-dose combinations are a significant advance in antiretroviral treatment simplification, contributing to an increase in compliance with complex chronic therapies, thus improving patients' quality of life. Reducing the number of pills and daily doses is associated with higher adherence and better quality of life. As a fixed-dose combination tablet given once daily, EFV/FTC/TDF was the first available STR combining efficacy, tolerability and convenience, with the simplest dosing schedule and smallest numbers of pills of any ART combination therapy. The RPV/FTC/TDF is a next-generation NNRTI-based STR, a once daily complete ART regimen for the treatment of HIV-1 infection. Recently the combination of EVG/COBI/FTC/TDF was also approved by the European Commission, and is the first integrase inhibitor-based STR. Receiving antiretroviral therapy as once daily STR is associated with both clinical and economic benefits, which confirms previous research., Conclusions: The associated benefits of STRs provide a valid strategy for the treatment of HIV-infected patients.
- Published
- 2014
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42. Aripiprazole for the treatment and prevention of acute manic and mixed episodes in bipolar I disorder in children and adolescents: a NICE single technology appraisal.
- Author
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Uttley L, Kearns B, Ren S, and Stevenson M
- Subjects
- Adolescent, Antipsychotic Agents adverse effects, Antipsychotic Agents economics, Aripiprazole, Bipolar Disorder economics, Bipolar Disorder physiopathology, Child, Clinical Trials as Topic, Cost-Benefit Analysis, Humans, Piperazines adverse effects, Piperazines economics, Precision Medicine, Quinolones adverse effects, Quinolones economics, Antipsychotic Agents therapeutic use, Bipolar Disorder drug therapy, Piperazines therapeutic use, Quinolones therapeutic use
- Abstract
As part of its single technology process, the National Institute for Health and Care Excellence (NICE) invited the manufacturers of aripiprazole (Otsuka Pharmaceutical Co. and Bristol Myers Squibb) to submit evidence of the clinical and cost effectiveness of aripiprazole for the treatment and prevention of acute manic and mixed episodes in bipolar I disorder in children and adolescents. The School of Health and Related Research Technology Appraisal Group at the University of Sheffield was commissioned to act as the independent Evidence Review Group (ERG). The ERG produced a critical review of the evidence for the clinical and cost effectiveness of the technology, based upon the manufacturers' submission to NICE. The evidence, which was derived mainly from a double-blind, phase III, placebo-controlled trial of aripiprazole in patients aged 10-17 years, showed that aripiprazole performed significantly better than placebo in reducing mania according to the primary outcome measurement (the Young Mania Rating Scale at 4 weeks). Safety outcomes indicated that aripiprazole was significantly more likely to cause extrapyramidal symptoms and somnolence than placebo. The manufacturers also presented a network meta-analysis of aripiprazole versus other atypical antipsychotics commonly used to treat manic episodes (olanzapine, quetiapine and risperidone) to show that aripiprazole performed similarly to the comparator drugs in terms of efficacy and safety. Aripiprazole was demonstrated to perform better in safety outcomes of (1) less weight gain than olanzapine and quetiapine; and (2) less prolactin increase than olanzapine, quetiapine and risperidone. Results from the manufacturers' economic evaluation showed that use of aripiprazole second-line dominated all of the other treatment strategies that were considered. However, there was considerable uncertainty in this result, and clinical advisors indicated that the actual treatment strategy employed in practice is likely to be dependent upon the patient's characteristics. The ERG demonstrated that if this personalised medicine resulted in improved cost effectiveness for any of the other treatment strategies, then they had the potential to dominate use of aripiprazole second-line. In conclusion, whilst a strategy including aripiprazole appeared to be cost effective relative to a strategy without it, there was not robust enough evidence to recommend a specific place for aripiprazole within the treatment pathway.
- Published
- 2013
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- View/download PDF
43. Pricing for orphan drugs: will the market bear what society cannot?
- Author
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O'Sullivan BP, Orenstein DM, and Milla CE
- Subjects
- Aminophenols economics, Aminophenols therapeutic use, Cost Sharing, Cystic Fibrosis drug therapy, Drug Discovery, Drug Industry ethics, Health Expenditures, Humans, Quinolones economics, Quinolones therapeutic use, United States, Drug Costs, Drug Industry economics, Orphan Drug Production economics, Social Justice
- Published
- 2013
- Full Text
- View/download PDF
44. Cost-effectiveness of lurasidone vs aripiprazole among patients with schizophrenia who have previously failed on an atypical antipsychotic: an indirect comparison of outcomes from clinical trial data.
- Author
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Rajagopalan K, Hassan M, O'Day K, Meyer K, and Grossman F
- Subjects
- Adult, Antipsychotic Agents adverse effects, Antipsychotic Agents economics, Antipsychotic Agents therapeutic use, Aripiprazole, Cardiovascular Diseases chemically induced, Cardiovascular Diseases economics, Clinical Trials as Topic statistics & numerical data, Comorbidity, Cost-Benefit Analysis, Diabetes Mellitus chemically induced, Diabetes Mellitus economics, Hospitalization economics, Hospitalization statistics & numerical data, Humans, Lurasidone Hydrochloride, Markov Chains, Models, Economic, Outcome Assessment, Health Care statistics & numerical data, Recurrence, Schizophrenia complications, United States, Clozapine adverse effects, Clozapine economics, Clozapine therapeutic use, Isoindoles adverse effects, Isoindoles economics, Isoindoles therapeutic use, Piperazines adverse effects, Piperazines economics, Piperazines therapeutic use, Quinolones adverse effects, Quinolones economics, Quinolones therapeutic use, Schizophrenia drug therapy, Schizophrenia economics, Thiazoles adverse effects, Thiazoles economics, Thiazoles therapeutic use
- Abstract
Objective: Compare long-term costs and outcomes of lurasidone to aripiprazole among adults with schizophrenia in the US who previously failed ≥1 atypical antipsychotic (olanzapine, risperidone, quetiapine, or ziprasidone) based on an indirect comparison of outcomes data from clinical trials., Methods: A 5-year Markov cohort model was developed to compare long-term effectiveness of lurasidone to aripiprazole, including total discontinuations, relapse rates, and hospitalization rates. Cost inputs included pharmacy, mental health, and medical costs associated with cardiometabolic risks (diabetes and cardiovascular [CV] events). Effectiveness inputs were derived from an indirect comparison of aripiprazole and lurasidone using common comparators from CATIE. Cardiometabolic risks were derived from claims data analysis for diabetes, weight change and CV events, and Framingham body mass index (BMI) risk equation. Cost inputs were derived from published sources and Red Book. Costs and outcomes were discounted at 3% and tested with sensitivity analyses., Results: Over 5 years, total discounted costs for lurasidone and aripiprazole patients were $86,480 and $90,500, respectively. During this period, the number of relapses per patient, hospitalizations per patient, diabetes rates, and CV events per 1000 patients, respectively, were estimated to be lower for lurasidone (0.442, 0.245, 7.29%, and 37.3) than aripiprazole (0.478, 0.369, 7.36%, and 37.8). Results were sensitive to lurasidone and aripiprazole hospitalization rates. At a willingness-to-pay threshold of $50,000 per hospitalization avoided, lurasidone had a 100% probability of being more cost-effective than aripiprazole., Limitations: The model was based on results from various comparative clinical trials. Differences in patient population and study methods may change estimates from the model. The model does not account for patient heterogeneity., Conclusions: Based on this model, when switching from another atypical antipsychotic, lurasidone had fewer relapses and hospitalizations with a lower incidence of diabetes and CV events than aripiprazole. Additionally, lurasidone may be less costly than aripiprazole among adults with schizophrenia.
- Published
- 2013
- Full Text
- View/download PDF
45. A UK-based cost-utility analysis of indacaterol, a once-daily maintenance bronchodilator for patients with COPD, using real world evidence on resource use.
- Author
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Price D, Asukai Y, Ananthapavan J, Malcolm B, Radwan A, and Keyzor I
- Subjects
- Aged, Albuterol administration & dosage, Albuterol analogs & derivatives, Albuterol economics, Cost-Benefit Analysis, Drug Administration Schedule, Female, Humans, Male, Markov Chains, Middle Aged, Models, Economic, Salmeterol Xinafoate, Scopolamine Derivatives administration & dosage, Scopolamine Derivatives economics, Tiotropium Bromide, Treatment Outcome, United Kingdom, Bronchodilator Agents administration & dosage, Bronchodilator Agents economics, Drug Costs statistics & numerical data, Indans administration & dosage, Indans economics, Pulmonary Disease, Chronic Obstructive drug therapy, Pulmonary Disease, Chronic Obstructive economics, Quinolones administration & dosage, Quinolones economics
- Abstract
Introduction: Chronic Obstructive Pulmonary Disease (COPD) is a chronic, progressive disease that is not curable. However, there are effective treatments available. In the UK, long-acting bronchodilators are first-line treatments for COPD patients requiring maintenance therapy, and there are several options available. The aim of this study is to establish, from the UK National Health Service (NHS) perspective, the cost-effectiveness profile of indacaterol, the first once-daily long-acting beta2-agonist (LABA), compared with tiotropium and salmeterol, in patients with moderate to severe COPD. In assessing the cost-effectiveness of COPD therapies, this study has the advantage of using real world evidence on the resource use associated with COPD management across the spectrum of the disease., Methods: A Markov model was developed with four health states following the GOLD classification for severity of airflow limitation. The model time horizon was 3 years, and the cycle length was 3 months. From each state, patients could experience a severe or non-severe exacerbation, move to a different COPD state, remain in the current state or die. Transition probabilities were based on data from the indacaterol clinical trials. The majority of the resource use data was taken from the Optimum Patient Care Research Database (OPCRD), which contains data from over 20,000 COPD patients in England and Scotland. Cost data were taken from UK-based sources and published literature and presented for the cost year 2011. Health-related quality of life was the main outcome of interest and utility data for the COPD states were based on data from the indacaterol clinical trials and disutility due to exacerbations were taken from the literature. Both one way and probabilistic sensitivity analyses were performed to test the robustness of the results., Results: Indacaterol dominated in the comparison with salmeterol producing an incremental QALY gain of 0.008 and cost savings of £110 per patient over a 3-year time horizon. In the comparison with tiotropium over the same time horizon, indacaterol remained the dominant strategy, producing an incremental QALY gain of 0.008 and cost savings of £248 per patient. The one-way sensitivity analysis indicates that the proportion of patients in each of the COPD stages and the mortality rate associated with Very Severe COPD are the variables with the largest impact on the results. The probabilistic sensitivity analyses showed that over 72 % and 89 % of the iterations when compared with salmeterol and tiotropium, respectively, produced dominant results for indacaterol., Conclusion: The analyses demonstrate that indacaterol dominates both tiotropium and salmeterol in the base case and is likely to remain cost-effective under a range of assumptions.
- Published
- 2013
- Full Text
- View/download PDF
46. Macrolides vs. quinolones for community-acquired pneumonia: meta-analysis of randomized controlled trials.
- Author
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Skalsky K, Yahav D, Lador A, Eliakim-Raz N, Leibovici L, and Paul M
- Subjects
- Adult, Anti-Bacterial Agents adverse effects, Anti-Bacterial Agents economics, Community-Acquired Infections mortality, Drug-Related Side Effects and Adverse Reactions epidemiology, Drug-Related Side Effects and Adverse Reactions pathology, Humans, Macrolides adverse effects, Macrolides economics, Pneumonia, Bacterial mortality, Quinolones adverse effects, Quinolones economics, Randomized Controlled Trials as Topic, Survival Analysis, Treatment Failure, Anti-Bacterial Agents therapeutic use, Community-Acquired Infections drug therapy, Macrolides therapeutic use, Pneumonia, Bacterial drug therapy, Quinolones therapeutic use
- Abstract
The relative efficacy, safety and ecological implications of macrolides vs. quinolones in the treatment of community-acquired pneumonia (CAP) are debatable. We performed a systematic review and meta-analysis of randomized controlled trials comparing any macrolide vs. any quinolone for the treatment of CAP among adult inpatients or outpatients, as monotherapy or both in combination with a beta-lactam. We did not limit inclusion by pneumonia severity, publication status, language or date of publication. The primary outcomes assessed were 30-day all-cause mortality and treatment failure. Two authors independently extracted the data. Fixed effect meta-analysis of risk ratios (RRs) with 95% confidence intervals was performed. Sixteen trials (4989 patients) fulfilling inclusion criteria were identified, mostly assessing outpatients with mild to moderate CAP. All-cause mortality was not significantly different for macrolides vs. quinolones, RR 1.03 (0.63-1.68, seven trials), with a low event rate (2%). Treatment failure was significantly lower with quinolones, RR 0.78 (0.67-0.91, 16 trials). The definition of failure used in the primary studies was not clearly representative of patients' benefit. Microbiological failure was lower with quinolones, RR 0.63 (0.49-0.81, 13 trials). All adverse events, adverse events requiring discontinuation and any premature antibiotic discontinuation were significantly more frequent with macrolides, mainly on account of gastrointestinal adverse events. Resistance development was not assessed in the trials. Randomized controlled trials show an advantage of quinolones in the treatment of CAP with regard to clinical cure without need for antibiotic modification at end of treatment and gastrointestinal adverse events. The clinical significance of this advantage is unclear., (© 2012 The Authors. Clinical Microbiology and Infection © 2012 European Society of Clinical Microbiology and Infectious Diseases.)
- Published
- 2013
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- View/download PDF
47. New cystic fibrosis drug paves the way for orphan diseases.
- Author
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Cooney D
- Subjects
- Cystic Fibrosis Transmembrane Conductance Regulator genetics, Drug Approval, Drug Costs, Humans, Mutation, Respiratory System Agents economics, Respiratory System Agents pharmacology, United Kingdom, Aminophenols economics, Aminophenols pharmacology, Cystic Fibrosis drug therapy, Cystic Fibrosis economics, Cystic Fibrosis genetics, Precision Medicine economics, Precision Medicine methods, Quinolones economics, Quinolones pharmacology, Rare Diseases economics
- Published
- 2013
- Full Text
- View/download PDF
48. Improved adherence expected with new HIV combo treatment.
- Author
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Morrow T
- Subjects
- Adenine economics, Adenine therapeutic use, Aged, Aged, 80 and over, Anti-HIV Agents economics, Carbamates economics, Deoxycytidine economics, Drug Combinations, Drug Labeling, Elvitegravir, Cobicistat, Emtricitabine, Tenofovir Disoproxil Fumarate Drug Combination, Humans, Middle Aged, Organophosphonates economics, Quinolones economics, Randomized Controlled Trials as Topic, Thiazoles economics, United States, Adenine analogs & derivatives, Anti-HIV Agents therapeutic use, Carbamates therapeutic use, Deoxycytidine therapeutic use, HIV Infections drug therapy, Medication Adherence, Organophosphonates therapeutic use, Quinolones therapeutic use, Thiazoles therapeutic use
- Published
- 2012
49. Cystic fibrosis in an era of genomically guided therapy.
- Author
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Barrett PM, Alagely A, and Topol EJ
- Subjects
- Aminophenols economics, Cystic Fibrosis economics, Cystic Fibrosis genetics, Genetic Predisposition to Disease, Humans, Mutation, Quinolones economics, Aminophenols therapeutic use, Cystic Fibrosis drug therapy, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Molecular Targeted Therapy economics, Orphan Drug Production economics, Quinolones therapeutic use
- Abstract
Although affecting only 4-5% of those with cystic fibrosis (CF), the G551D-CFTR mutation is the target of the recently approved 'orphan drug', ivacaftor. The promise of such genomically guided therapies heralds a new era in the management of CF. A phase 3 trial demonstrated significant improvements in forced expiratory volume in 1 s (FEV(1)) from baseline, average weight gain, concentration in sweat chloride and reductions in pulmonary exacerbations [Ramsey, B.W., et al. A CFTR potentiator in patients with CF and the G551D mutation. N. Engl. J. Med., 2011. 365: 1663-1672.)]. Ivacaftor is among a group of recently approved, novel, mutation guided 'orphan drug' therapies that have established clinical benefits within their respective disease categories. They do not, however, offer a cure. Pharmaceutical and biotech companies have leveraged the incentivized benefits of the Orphan Drug Act to develop more of these drugs for orphan disorders affecting populations of <200 000 patients. With marked clinical efficacy via DNA sequence guidance, these drugs have also set a precedent in terms of the substantial annual costs and if this trend continues, such expenditures may become unsustainable. This paper explores the genomic pathophysiology of CF and how therapies such as ivacaftor provide benefit to those with the disease but at a considerably elevated price point.
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- 2012
- Full Text
- View/download PDF
50. Evaluating the impact of a novel restricted reimbursement policy for quinolone antibiotics: a time series analysis.
- Author
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Manns B, Laupland K, Tonelli M, Gao S, and Hemmelgarn B
- Subjects
- Aged, Aged, 80 and over, Alberta, Anti-Bacterial Agents therapeutic use, Drug Utilization, Female, Formularies as Topic, Humans, Linear Models, Male, Outpatients, Regression Analysis, Anti-Bacterial Agents economics, Drug Prescriptions economics, Practice Patterns, Physicians' statistics & numerical data, Quinolones economics, Quinolones therapeutic use, Reimbursement Mechanisms, Universal Health Insurance economics
- Abstract
Background: Publicly-funded drug plans often use prior authorization policies to limit drug prescribing. To guide physician prescribing of a class of antibiotics with broad antimicrobial activity (quinolone antibiotics) in accordance with new prescribing guidelines, Alberta's provincial health ministry implemented a new mechanism for formulary restriction entitled the optional special authorization (OSA) program. We conducted an observational study to determine the impact of this new formulary restriction policy on antimicrobial prescription rates as well as any clinical consequences., Methods: Quinolone antibiotic use, and adherence with quinolone prescribing guidelines, was assessed before and after implementation of the OSA program in patients with common outpatient infections using an administrative data cohort and a chart review cohort, respectively. At the same time this policy was implemented to limit quinolone prescribing, two new quinolone antibiotics were added to the formulary. Using administrative data, we analysed a total of 397,534 unique index visits with regard to overall antibiotic utilization, and through chart review, we analysed 1681 charts of patients with infections of interest to determine the indications for quinolone usage., Results: Using segmented regression models adjusting for age, sex and physician enrollment in the OSA program, there was no statistically significant change in the monthly rate of all quinolone use (-3.5 (95% CI -5.5, 1.4) prescriptions per 1000 index visits) following implementation of the OSA program (p = 0.74). There was a significant level change in the rate of quinolone antibiotic use for urinary tract infection (-33.6 (95% CI: -23.8, -43.4) prescriptions and upper respiratory tract infection (-16.1 (95%CI: -11.6, -20.6) prescriptions per 1000 index visits. Among quinolone prescriptions identified on chart review, 42.5% and 58.5% were consistent with formulary guidelines before and after the implementation of the OSA program, respectively (p = 0.002). There was no change in hospitalization, mortality or use of physician services after implementation of the OSA program., Conclusions: Despite the addition of two new quinolone antibiotics to the formulary, we found that there was no change in the use of quinolones after implementation of a new formulary restriction policy for outpatients with common outpatient infections.
- Published
- 2012
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