RÉSUMÉ
AIMS: To describe healthcare resource utilization (HCRU) and associated costs after initiation of injectable glucagon-like peptide-1 receptor agonist (GLP-1 RA) therapy by adult patients with type 2 diabetes (T2D) in the prospective, observational, 24-month TROPHIES study in France, Germany, and Italy. MATERIALS AND METHODS: HCRU data for cost calculations were collected by treating physicians during patient interviews at baseline and follow-up visits approximately 6, 12, 18, and 24 months after GLP-1 RA initiation with once-weekly dulaglutide or once-daily liraglutide. Costs were evaluated from the national healthcare system (third-party payer) perspective and updated to 2018 prices. RESULTS: In total, 2,005 patients were eligible for the HCRU analysis (1,014 dulaglutide; 991 liraglutide). Baseline patient characteristics were generally similar between treatment groups and countries. The largest proportions of patients using ≥2 oral glucose-lowering medications (GLMs) at baseline (42.9-43.4%) and month 24 (44.0-45.1%) and using another injectable GLM at month 24 (15.3-23.2%) were in France. Mean numbers of primary and secondary healthcare contacts during each assessment period were highest in France (range = 4.0-10.7) and Germany (range = 2.9-5.7), respectively. The greatest proportions (≥60%) of mean annualized costs per patient comprised medication costs. Mean annualized HCRU costs per patient varied by treatment cohort and country: the highest levels were in the liraglutide cohort in France (909) and the dulaglutide cohort in Germany (883). LIMITATIONS: Limitations included exclusion of patients using insulin at GLP-1 RA initiation and collection of HCRU data by physician, not via patient-completed diaries. CONCLUSIONS: Real-world HCRU and costs associated with the treatment of adults with T2D with two GLP-1 RAs in TROPHIES emphasize the need to avoid generalization with respect to HCRU and costs associated with a particular therapy when estimating the impact of a new treatment in a country-specific setting.
Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) have become frequent treatments of hyperglycemia in type-2 diabetes (T2D). Not all types of clinical study provide information about the cost of these treatments or the effects they might have on use of other medicines and equipment to control T2D or the need for visits to a doctor or nurse and different types of treatment in hospital. This study collected this information during the regular care of adults in France, Germany, or Italy who were prescribed either dulaglutide or liraglutide (both types of GLP-1 RAs) by their family doctor or a specialist in T2D. There were differences in costs and the need for other medicines and medical services between people using either dulaglutide or liraglutide and for people who were using the same GLP-1 RA in each of the three countries. The information from this study could be used to more accurately understand the overall costs and medical care needed when patients use dulaglutide or liraglutide in France, Germany, or Italy.
Sujet(s)
Diabète de type 2 , Peptides glucagon-like , Hypoglycémiants , Fragments Fc des immunoglobulines , Liraglutide , Protéines de fusion recombinantes , Humains , Diabète de type 2/traitement médicamenteux , Diabète de type 2/économie , Liraglutide/usage thérapeutique , Liraglutide/économie , Peptides glucagon-like/analogues et dérivés , Peptides glucagon-like/usage thérapeutique , Peptides glucagon-like/économie , Peptides glucagon-like/administration et posologie , Fragments Fc des immunoglobulines/usage thérapeutique , Fragments Fc des immunoglobulines/économie , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/usage thérapeutique , Protéines de fusion recombinantes/administration et posologie , Mâle , Hypoglycémiants/usage thérapeutique , Hypoglycémiants/économie , Femelle , Études prospectives , Adulte d'âge moyen , Sujet âgé , Ressources en santé/statistiques et données numériques , Ressources en santé/économie , Modèles économétriquesRÉSUMÉ
Diabetic macular edema (DME) is a degenerative disease of the macular area in diabetes mellitus and can lead to vision loss, disability, and significantly reduced quality of life. Faricimab is the only bispecific antibody for DME therapy that targets two pathogenic pathways (Ang-2 and VEGF-A). PURPOSE: This study comparatively evaluates the clinical and economic feasibility of faricimab and other angiogenesis inhibitors in patients with DME. MATERIAL AND METHODS: This article analyzed literature on the efficacy and safety of intravitreal injections (IVI) of ranibizumab 0.5 mg, aflibercept 2 mg, and faricimab 6 mg. A model of medical care was developed for patients with DME receiving anti-angiogenic therapy. Pharmacoeconomic analysis was performed using cost minimization and budget impact analysis (BIA) methods. Modeling time horizon was 2 years. The research was performed from the perspective of the healthcare system of the Russian Federation. RESULTS: The efficacy and safety of faricimab in a personalized regimen (up to one IVI in 16 weeks) are comparable to those of aflibercept and ranibizumab, administered in various regimens. The use of faricimab is associated with the lowest number of IVIs. Over 2 years, the maximum costs of drug therapy were associated with the use of ranibizumab (about 914 thousand rubles), while the minimum costs were associated with the use of faricimab (614 thousand rubles). The reduction in inpatient care costs with faricimab therapy was 36% compared to aflibercept (216 and 201 thousand rubles in inpatient and day hospitals, respectively) and 82% compared to ranibizumab (486 and 451 thousand rubles in inpatient and day hospitals, respectively). BIA demonstrated that the use of faricimab will reduce the economic burden on the healthcare system by 11.3 billion rubles (9.8%) over 2 years. CONCLUSION: The use of faricimab is a cost-effective approach to treatment of adult patients with DME in Russia.
Sujet(s)
Inhibiteurs de l'angiogenèse , Rétinopathie diabétique , Pharmacoéconomie , Oedème maculaire , Récepteurs aux facteurs de croissance endothéliale vasculaire , Protéines de fusion recombinantes , Humains , Oedème maculaire/traitement médicamenteux , Oedème maculaire/étiologie , Oedème maculaire/économie , Inhibiteurs de l'angiogenèse/économie , Inhibiteurs de l'angiogenèse/administration et posologie , Rétinopathie diabétique/traitement médicamenteux , Rétinopathie diabétique/économie , Russie , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/administration et posologie , Récepteurs aux facteurs de croissance endothéliale vasculaire/administration et posologie , Injections intravitréennes , Ranibizumab/administration et posologie , Ranibizumab/économie , Analyse coût-bénéfice , Anticorps bispécifiques/économie , Anticorps bispécifiques/administration et posologie , Résultat thérapeutiqueRÉSUMÉ
INTRODUCTION: Recombinant factor IX (rFIX) and recombinant FIX Fc fusion protein (rFIXFc) are standard half-life and extended half-life FIX replacement therapies, respectively, and represent established treatment options indicated for adults and children with haemophilia B. These FIX replacement therapies can be administered as prophylaxis (to prevent bleeding) or 'on-demand' (to stop bleeding). This analysis aimed to estimate the cost-effectiveness of once-weekly prophylaxis with rFIXFc versus on-demand treatment with rFIX in patients with haemophilia B without inhibitors in the Italian healthcare setting. METHODS: A Markov model was developed to assess a hypothetical cohort of adolescent or adult male patients (≥ 12 years) with haemophilia B (FIX level of ≤ 2 IU/dL) without inhibitors. Model inputs were derived from the pivotal phase 3 clinical studies for rFIXFc and rFIX, published literature and assumptions when published data were unavailable. The model employed a lifelong time horizon with 6-monthly transitions between health states, and it estimated total costs, total quality-adjusted life years (QALYs), number of bleeds, number of surgeries and incremental cost-effectiveness ratio. RESULTS: rFIXFc prophylaxis was associated with lower total costs per patient (5,308,625 versus 6,564,510) and greater total QALYs per patient (15.936 versus 11.943) compared with rFIX on-demand; rFIXFc prophylaxis was therefore the dominant treatment strategy. The model also demonstrated that rFIXFc prophylaxis was associated with fewer incremental bleeds (- 682.29) and surgeries (- 0.39) compared with rFIX on-demand. CONCLUSIONS: rFIXFc prophylaxis provides improved health outcomes and lower costs, and represents a cost-effective treatment option compared with rFIX on-demand for adolescent and adult male patients with haemophilia B. This comparative assessment of cost-effectiveness should help to inform both clinicians and healthcare policy makers when making treatment decisions for patients with haemophilia B.
Sujet(s)
Facteur IX , Hémophilie B , Fragments Fc des immunoglobulines , Protéines de fusion recombinantes , Adolescent , Adulte , Enfant , Humains , Mâle , Adulte d'âge moyen , Jeune adulte , Analyse coût-bénéfice , Facteur IX/usage thérapeutique , Facteur IX/économie , Hémophilie B/traitement médicamenteux , Hémophilie B/économie , Hémorragie/prévention et contrôle , Fragments Fc des immunoglobulines/usage thérapeutique , Fragments Fc des immunoglobulines/économie , Italie , Chaines de Markov , Années de vie ajustées sur la qualité , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/usage thérapeutique , Protéines recombinantes/usage thérapeutique , Protéines recombinantes/économieRÉSUMÉ
BACKGROUND/OBJECTIVES: Diabetic macular oedema (DMO) is a leading cause of blindness in developed countries, with significant disease burden associated with socio-economic deprivation. Distributional cost-effectiveness analysis (DCEA) allows evaluation of health equity impacts of interventions, estimation of how health outcomes and costs are distributed in the population, and assessments of potential trade-offs between health maximisation and equity. We conducted an aggregate DCEA to determine the equity impact of faricimab. METHODS: Data on health outcomes and costs were derived from a cost-effectiveness model of faricimab compared with ranibizumab, aflibercept and off-label bevacizumab using a societal perspective in the base case and a healthcare payer perspective in scenario analysis. Health gains and health opportunity costs were distributed across socio-economic subgroups. Health and equity impacts, measured using the Atkinson inequality index, were assessed visually on an equity-efficiency impact plane and combined into a measure of societal welfare. RESULTS: At an opportunity cost threshold of £20,000/quality-adjusted life year (QALY), faricimab displayed an increase in net health benefits against all comparators and was found to improve equity. The equity impact increased the greater the concerns for reducing health inequalities over maximising population health. Using a healthcare payer perspective, faricimab was equity improving in most scenarios. CONCLUSIONS: Long-acting therapies with fewer injections, such as faricimab, may reduce costs, improve health outcomes and increase health equity. Extended economic evaluation frameworks capturing additional value elements, such as DCEA, enable a more comprehensive valuation of interventions, which is of relevance to decision-makers, healthcare professionals and patients.
Sujet(s)
Inhibiteurs de l'angiogenèse , Analyse coût-bénéfice , Rétinopathie diabétique , Équité en santé , Oedème maculaire , Années de vie ajustées sur la qualité , Ranibizumab , Protéines de fusion recombinantes , Humains , Rétinopathie diabétique/traitement médicamenteux , Rétinopathie diabétique/économie , Oedème maculaire/traitement médicamenteux , Oedème maculaire/économie , Inhibiteurs de l'angiogenèse/économie , Inhibiteurs de l'angiogenèse/usage thérapeutique , Royaume-Uni , Équité en santé/économie , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/usage thérapeutique , Ranibizumab/économie , Ranibizumab/usage thérapeutique , Ranibizumab/administration et posologie , Mâle , Récepteurs aux facteurs de croissance endothéliale vasculaire/usage thérapeutique , Injections intravitréennes , Femelle , Facteur de croissance endothéliale vasculaire de type A/antagonistes et inhibiteurs , Bévacizumab/économie , Bévacizumab/usage thérapeutique , Coûts des médicaments , Adulte d'âge moyen , Évaluation du Coût-EfficacitéRÉSUMÉ
BACKGROUND: Promacta (eltrombopag; EPAG) and Nplate (romiplostim; ROMI) have not been compared in head-to-head trials for treatment of chronic immune thrombocytopenia (cITP); however, indirect treatment comparisons have indicated similar efficacy and safety outcomes, and the drugs are generally accepted as therapeutic alternatives. OBJECTIVE: To determine which of the 2 therapies would result in the lowest overall cost from a US health plan perspective, under the assumption of equivalent clinical efficacy and safety. METHODS: A cost-minimization model was developed in Microsoft Excel. The model incorporated only costs that differ between the treatments, including drug acquisition, administration, and monitoring costs, over a 52-week horizon. Average dosing for EPAG and ROMI was taken from the long-term EXTEND trial and from a published metaanalysis of 14 clinical trials, respectively. ROMI is injectable and EPAG is oral, so only ROMI had administration costs. The model assumed patients used 25 mg EPAG tablets and the 250 µg vial size of ROMI. ROMI wastage was included in drug acquisition costs by rounding up average dose to the nearest whole vial. Monitoring requirements were determined from US prescribing information, with platelet monitoring assumed equal, and hepatic panel testing every 4 weeks for EPAG. The model was adjustable to commercial, Medicare, and Medicaid plan perspectives, with optional inclusion of drug wastage, monitoring, or administration costs. RESULTS: The base case used a commercial plan perspective, with average dosing of 51.5 mg/day for EPAG and 4.20 µg/kg/week for ROMI. The analysis found a cost difference per treated patient of $64,770 in favor of EPAG on an annual basis. Breakdown by unique costs for EPAG included drug-acquisition cost of $123,135 and monitoring cost of $705. Breakdown by unique costs for ROMI included drug-acquisition cost of $183,234, with wastage of $63,179 and administration cost of $5,377. Based on a hypothetical commercial plan with 1 million members and an estimated 11 patients with cITP receiving ROMI, potential annual savings for switching all patients from ROMI to EPAG is $712,473 or $0.06 per member per month. EPAG remained the less costly option for all plan types and assumptions. A sensitivity analysis found that the result was most sensitive to drug pricing and wastage inputs. CONCLUSIONS: Because of lower drug-acquisition costs (including drug wastage) and administration costs, treatment of cITP with EPAG is associated with a lower net cost per patient than ROMI. DISCLOSURES: This study was funded by Novartis Pharmaceuticals Corporation. Proudman, Lucas, and Nellesen are employees of Analysis Group, Inc., which received funding from Novartis Pharmaceuticals Corporation to conduct this study. Patwardhan was employed by Novartis Pharmaceuticals Corporation at the time of this study; Allen is an employee of Novartis. This research was presented as an e-poster at the AMCP 2020 Virtual, April 2020.
Sujet(s)
Benzoates/économie , Maladie chronique/économie , Hydrazines/économie , Pyrazoles/économie , Protéines de fusion recombinantes/économie , Thrombopénie/traitement médicamenteux , Thrombopoïétine/économie , Adolescent , Adulte , Maîtrise des coûts , Coûts des médicaments , Femelle , Humains , Mâle , Adulte d'âge moyen , Récepteur Fc , États-Unis , Jeune adulteRÉSUMÉ
OBJECTIVES: Onasemnogene Abeparvovec-xioi (AVXS-101) is a gene therapy intended for curative treatment of spinal muscular atrophy (SMA) with an expected price of around 2 000 000. The goal of this study is to perform a cost-effectiveness analysis of treatment of SMA I patients with AVXS-101 in The Netherlands including relapse scenarios. METHODS: An individual-based state-transition model was used to model treatment effect and survival of SMA I patients treated with AVXS-101, nusinersen and best supportive care (BSC). The model included five health states: three health states according to SMA types, one for permanent ventilation and one for death. Deterministic and probabilistic sensitivity analyses were performed. Effects of relapsing to lower health states in the years following treatment was explored. RESULTS: The base-case incremental cost-effectiveness ratio (ICER) for AVXS-101 versus BSC is 138 875/QALY, and 53 447/QALY for AVXS-101 versus nusinersen. If patients relapse within 10 years after treatment with AVXS-101, the ICER can increase up to 6-fold, with effects diminishing thereafter. Only relapses occurring later than 50 years after treatment have a negligible effect on the ICER. To comply with Dutch willingness-to-pay reference values, the price of AVXS-101 must decrease to 680 000. CONCLUSIONS: Based on this model, treatment with AVXS-101 is unlikely to be cost-effective under Dutch willingness-to-pay reference values. Uncertainty regarding the long-term curative properties of AVXS-101 can result in multiplication of the ICER. Decision-makers are advised to appropriately balance these uncertainties against the price they are willing to pay now.
Sujet(s)
Produits biologiques/économie , Produits biologiques/usage thérapeutique , Coûts des médicaments , Thérapie génétique/économie , Oligonucléotides/économie , Oligonucléotides/usage thérapeutique , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/usage thérapeutique , Amyotrophies spinales infantiles/économie , Amyotrophies spinales infantiles/thérapie , Produits biologiques/effets indésirables , Essais cliniques comme sujet , Recherche comparative sur l'efficacité , Analyse coût-bénéfice , Femelle , Thérapie génétique/effets indésirables , État de santé , Humains , Nourrisson , Mâle , Modèles économiques , Pays-Bas , Oligonucléotides/effets indésirables , Années de vie ajustées sur la qualité , Protéines de fusion recombinantes/effets indésirables , Récidive , Amyotrophies spinales infantiles/diagnostic , Amyotrophies spinales infantiles/génétique , Évaluation de la technologie biomédicale , Facteurs temps , Résultat thérapeutiqueRÉSUMÉ
OBJECTIVES: To evaluate alternative methods to calculate and/or attribute economic surplus in the cost-effectiveness analysis of single or short-term therapies. METHODS: We performed a systematic literature review of articles describing alternative methods for cost-effectiveness analysis of potentially curative therapies whose assessment using traditional methods may suggest unaffordable valuations owing to the magnitude of estimated long-term quality-adjusted life-year (QALY) gains or cost offsets. Through internal deliberation and discussion with staff at the Health Technology Assessment bodies in England and Canada, we developed the following 3 alternative methods for further evaluation: (1) capping annual costs in the comparator arm at $150 000 per year; (2) "sharing" the economic surplus with the health sector by apportioning only 50% of cost offsets or 50% of cost offsets and QALY gains to the value of the therapy; and (3) crediting the therapy with only 12 years of the average annual cost offsets or cost offsets and QALY gains over the lifetime horizon. The impact of each alternative method was evaluated by applying it in an economic model of 3 hypothetical condition-treatment scenarios meant to reflect a diversity of chronicity and background healthcare costs. RESULTS: The alternative with greatest impact on threshold price for the fatal pediatric condition spinal muscular atrophy type 1 was the 12-year cutoff scenario. For a hypothetical one-time treatment for hemophilia A, capping cost offsets at $150 000 per year had the greatest impact. For chimeric antigen receptor T-cell treatment of non-Hodgkin's lymphoma, capping cost offsets or using 12-year threshold had little impact, whereas 50% sharing of surplus including QALY gains and cost offsets greatly reduced threshold pricing. CONCLUSIONS: Health Technology Assessment bodies and policy makers will wrestle with how to evaluate single or short-term potentially curative therapies and establish pricing and payment mechanisms to ensure sustainability. Scenario analyses using alternative methods for calculating and apportioning economic surplus can provide starkly different assessment results. These methods may stimulate important societal dialogue on fair pricing for these novel treatments.
Sujet(s)
Traitement médicamenteux/économie , Thérapie génétique/économie , Coûts des soins de santé , Immunothérapie adoptive/économie , Évaluation de la technologie biomédicale/économie , Anticorps bispécifiques/économie , Anticorps bispécifiques/usage thérapeutique , Anticorps monoclonaux humanisés/économie , Anticorps monoclonaux humanisés/usage thérapeutique , Produits biologiques/économie , Produits biologiques/usage thérapeutique , Économies , Analyse coût-bénéfice , Coûts des médicaments , Thérapie génétique/effets indésirables , Hémophilie A/traitement médicamenteux , Hémophilie A/économie , Humains , Immunothérapie adoptive/effets indésirables , Lymphome malin non hodgkinien/économie , Lymphome malin non hodgkinien/thérapie , Modèles économiques , Années de vie ajustées sur la qualité , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/usage thérapeutique , Induction de rémission , Amyotrophies spinales infantiles/économie , Amyotrophies spinales infantiles/génétique , Amyotrophies spinales infantiles/thérapie , Facteurs temps , Résultat thérapeutiqueRÉSUMÉ
BACKGROUND: Age-related macular degeneration (AMD) is a leading cause of blindness worldwide and is the most common cause of blindness in developed countries. Despite antivascular endothelial growth factor (anti-VEGF) therapy demonstrating improvements in visual and anatomical outcomes, unmet needs remain. Brolucizumab-dbll (ie, brolucizumab), a VEGF inhibitor for treatment of neovascular (wet) AMD and recently approved by the FDA for its treatment of wet AMD, attempts to mitigate treatment burden through less frequent injections. OBJECTIVE: To assess the incremental cost-effectiveness of brolucizumab compared with aflibercept and ranibizumab, given similar costs per injection and the potential for longer dosing intervals based on phase 3 clinical trial data. METHODS: A Markov model was developed to model the treatment of wet AMD patients with brolucizumab vs aflibercept and vs ranibizumab over a lifetime time horizon (base case) and 5-year time horizon (scenario analysis). The Markov model consisted of 3 primary health states: on treatment, off treatment, and death. Markov substates (5 total) described visual acuity (VA) ranging from no vision impairment to blindness. These VA-based substates were defined by best-corrected visual acuity (BCVA) values measured using Early Treatment Diabetic Retinopathy Study letters. Fixed-dosing regimens for each therapy were included in the model: dosing every 4 weeks (q4w) for the first 3 months followed by dosing q8w/q12w for brolucizumab, dosing q4w for the first 3 months followed by dosing q8w for aflibercept, and q4w for ranibizumab. RESULTS: In the base case, brolucizumab was less costly than aflibercept ($63,614 vs $72,189), and brolucizumab generated 0.0079 more quality-adjusted life-years (QALYs) than aflibercept (4.580 vs 4.572). Lower total costs with brolucizumab were driven by reduced drug costs ($56,432 vs $64,057), reduced administration costs ($6,013 vs $6,825), and reduced monitoring costs ($1,168 vs $1,306). When evaluating the cost-effectiveness of brolucizumab over a 5-year time horizon, brolucizumab was less costly than aflibercept ($44,644 vs $50,772) and generated an additional 0.0049 QALYs (2.953 vs 2.948). Additionally, brolucizumab was less costly than ranibizumab ($63,614 vs $128,163) and generated 0.0078 more QALYs than ranibizumab (4.580 vs 4.572) in the base case. Lower total costs with brolucizumab were driven by reduced drug costs ($56,432 vs $114,516), reduced administration costs ($6,013 vs $11,541), and reduced monitoring costs ($1,168 vs $2,107). When evaluating the cost-effectiveness of brolucizumab over a 5-year time horizon, brolucizumab was less costly than ranibizumab ($44,644 vs $89,665), and brolucizumab generated an additional 0.0046 QALYs (2.953 vs 2.948). CONCLUSIONS: Brolucizumab can be cost saving and cost-effective compared with aflibercept and ranibizumab in the treatment of wet AMD. DISCLOSURES: Novartis Pharmaceuticals Corporation provided funding to Xcenda for the cost-effectiveness analysis and preparation of this manuscript. Carlton is an employee of Xcenda. Agashivala is employed by Novartis Pharmaceuticals Corporation; Yu was an employee of Novartis Pharmaceutical Corporation at the time of this study. Hassan reports personal fees from iOPEN, BVI/Visitrec, ArcticDx, Bayer, F. Hoffmann-La Roche Ltd, Broadspot, BMC, Katalyst Surgical, Alcon, Vitreq, Surgicube, personal Ocugenix, Regeneron, Allergan, Oculus Surgical, Novartis, Genentech, and Eyepoint, unrelated to this work. Wykoff reports personal fees from Corcept Therapeutics, DORC, EyePoint, Gyroscope, IVERIC Bio, Merck, Notal Vision, ONL Therapeutics, Oxurion, Palatin, PolyPhotonix, Takeda, Thea Open Innovation; grants from Aerie Pharmaceuticals, Aldeyra, Gemini Therapeutics, Graybug Vision, IONIS Pharmaceutical, LMRI, Mylan, Neurotech Pharmaceuticals, Outlook Pharmaceuticals, Samsung Bioepis, Senju, Taiwan Liposome Company, Xbrane BioPharma, Santen; and grants and personal fees from Adverum, Allergan, Apellis, Chengdu Kanghong Biotechnologies (KHB), Clearside Biomedical, Genentech, Kodiak Sciences, NGM Biopharmaceuticals, Novartis, Opthea, Recens Medical, Regenxbio, Roche, and Regeneron, unrelated to this work. This research was presented as a virtual poster at the AMCP 2020 Annual Meeting, April 2020.
Sujet(s)
Inhibiteurs de l'angiogenèse/économie , Anticorps monoclonaux humanisés/économie , Ranibizumab/économie , Protéines de fusion recombinantes/économie , Dégénérescence maculaire humide/traitement médicamenteux , Adolescent , Adulte , Inhibiteurs de l'angiogenèse/usage thérapeutique , Anticorps monoclonaux humanisés/usage thérapeutique , Analyse coût-bénéfice , Études transversales , Femelle , Humains , Mâle , Adulte d'âge moyen , Ranibizumab/usage thérapeutique , Récepteurs aux facteurs de croissance endothéliale vasculaire/usage thérapeutique , Protéines de fusion recombinantes/usage thérapeutique , Acuité visuelle , Jeune adulteRÉSUMÉ
Importance: Ten percent of the Medicare Part B budget is spent on aflibercept, used to treat a myriad of ocular neovascular diseases. A substantial portion of these costs can be attributed to a few hundred ophthalmologists, raising concerns regarding the influence of pharmaceutical companies on the choice of medication by a relatively small group of clinicians. One approach to protect patients' health care interests is to include them in deliberations on the choice of therapy for their eye disease. Objective: To examine factors associated with patients' choice between an effective and less expensive off-label drug or a more effective, but also more expensive, US Food and Drug Administration (FDA)-approved drug. Design, Setting, and Participants: This retrospective cohort analysis used data from the satellite office of a tertiary referral center from August 2, 2013, to April 9, 2018. Insured patients initiating treatment with anti-vascular endothelial growth factor were included in the analysis. Data were analyzed from March 26, 2018, to June 10, 2020. Interventions: Patients were asked to choose between bevacizumab (approximately $100 per dose), a chemotherapy that is effective, but not FDA approved, for the treatment of ocular vascular disease, or aflibercept (approximately $2000 per dose), an FDA-approved drug for ocular vascular disease that may be more effective than bevacizumab in some patients. Independent of this choice, patients were separately asked by a study coordinator to participate in an invasive clinical study for which they would not be compensated, there was a small risk for an adverse event, and they would not personally benefit from participating (a surrogate marker for altruism). Main Outcomes and Measures: Factors associated with patients' choice of medication, including age, sex, ocular disease, race, and participation in an invasive clinical study. Results: A total of 189 patients were included in the analysis (106 women [56%]; mean [SEM] age, 74.6 [0.8] years). Despite being told that it may not be as effective as aflibercept, 100 patients (53%) selected bevacizumab for their own eye care. An act of altruism (ie, participation in an invasive clinical study) when the patient was making a choice between the 2 drugs was associated with a patient's choice of bevacizumab (odds ratio [OR], 7.03; 95% CI, 2.27-21.80; P < .001); the OR for selecting bevacizumab for patients who never agreed to participate in the clinical study was 0.45 (95% CI, 0.25-0.83; P = .001). Age (OR, 1.00; 95% CI, 0.97-1.03; P = .86), race (OR, 0.70; 95% CI, 0.41-1.22; P = .21), sex (OR, 0.72; 95% CI, 0.39-1.35; P = .31), presence of diabetes (OR, 1.52; 95% CI, 0.59-3.93; P = .39), and type of eye disease (OR, 0.56; 95% CI, 0.30-1.04; P = .07) were not associated with choice of therapy. Conclusions and Relevance: These findings suggest that clinicians must consider the ethical implications of the influence of altruism when patients participate in the decision between cost-effective vs the most effective medicines for their own health care.
Sujet(s)
Altruisme , Inhibiteurs de l'angiogenèse/économie , Bévacizumab/économie , Comportement de choix , Prise de décision , Maladies de l'oeil/traitement médicamenteux , Participation des patients , Protéines de fusion recombinantes/économie , , Sujet âgé , Inhibiteurs de l'angiogenèse/usage thérapeutique , , Bévacizumab/usage thérapeutique , Études de cohortes , Analyse coût-bénéfice , Rétinopathie diabétique/traitement médicamenteux , Coûts des médicaments , Femelle , Humains , Dégénérescence maculaire/traitement médicamenteux , Mâle , Adulte d'âge moyen , Néovascularisation pathologique/traitement médicamenteux , Odds ratio , Utilisation hors indication , Récepteurs aux facteurs de croissance endothéliale vasculaire/usage thérapeutique , Protéines de fusion recombinantes/usage thérapeutique , Occlusion veineuse rétinienne/traitement médicamenteux , Études rétrospectives , Résultat thérapeutique , Acuité visuelle ,RÉSUMÉ
Extended half-life of factor IX (FIX) demonstrated clinical benefit and lower treatment burden than standard half-life FIX products in clinical trials. We analysed the impact in efficacy, pharmacokinetics (PKs) and costs of the switch from nonacog alfa (rFIX) to albutrepenonacog alfa (rFIX-FP) in the first patient with haemophilia B (HB) treated in Spain outside clinical trials. A 7-year-old boy presented with HB with poor venous access and repetition infections using rFIX, which was switched to rFIX-FP. Prophylaxis was adjusted by PKs using WAPPS-Hemo tailoring from 100 IU/kg/week of rFIX to 80 IU/kg/3 weeks of rFIX-FP. Comparing 6 months before, rFIX-FP reduced 68.5% FIX consumption/kg and 58.3% infusion frequency, but total costs/weight showed a slight increase. Ratio of half-life between rFIX and rFIX-FP was 3.4-3.7. This case report revealed that switch to rFIX-FP decreased frequency and FIX consumption, without adverse events and bleeds.
Sujet(s)
Facteur IX/administration et posologie , Hémophilie B/traitement médicamenteux , Hémorragie/prévention et contrôle , Protéines de fusion recombinantes/administration et posologie , Sérumalbumine/administration et posologie , Tests de coagulation sanguine , Enfant , Coûts des médicaments , Substitution de médicament/économie , Facteur IX/économie , Facteur IX/pharmacocinétique , Période , Hémophilie B/complications , Hémophilie B/diagnostic , Hémophilie B/économie , Hémorragie/économie , Hémorragie/étiologie , Humains , Mâle , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/pharmacocinétique , Sérumalbumine/économie , Sérumalbumine/pharmacocinétique , Indice de gravité de la maladieRÉSUMÉ
PURPOSE: The clinic efficiency and cost savings achieved by eliminating formal visual acuity (VA) and dilated fundus examinations (DFEs) were assessed for established patients receiving optical coherence tomography (OCT)-guided intravitreal injections. DESIGN: Comparative cost analysis. METHODS: Two different treatment models were evaluated. The first model included patients undergoing routine VA assessment, DFEs, OCT imaging, and intravitreal injections. The second model eliminated the routine VA assessment and DFE while using OCT imaging through an undilated pupil followed by the intravitreal injection. The 2 models incorporated both bevacizumab and aflibercept. The number of patients per clinic day, the cost per visit, and the daily revenues were compared between the 2 models. RESULTS: Optimized schedules with and without VA assessments and DFEs allowed for 48 and 96 patients to be injected per day, respectively. Excluding drug costs, the cost per encounter for the visits with and without a DFE were $39.33 and $22.63, respectively. Including the drug costs, the costs per encounter for the visits with and without a DFE were $85.55 and $68.85 for bevacizumab and $1787.58 and $17770.88 for aflibercept, respectively. Once the reimbursements for each visit type were included, the clinics that eliminated the VA and DFEs were more cost efficient. CONCLUSION: Eliminating both VA assessments and DFEs for patients undergoing OCT-guided retreatment with intravitreal injections resulted in decreased exposure times between patients and clinic staff, decreased cost per encounter, and increased patient volumes per clinic day, resulting in improved clinic efficiency and safety while seeing more patients in a clinic day.
Sujet(s)
Inhibiteurs de l'angiogenèse/usage thérapeutique , Néovascularisation choroïdienne/traitement médicamenteux , Analyse coût-bénéfice , Examen physique/économie , Tomographie par cohérence optique/économie , Acuité visuelle , Dégénérescence maculaire humide/traitement médicamenteux , Sujet âgé , Inhibiteurs de l'angiogenèse/économie , Bévacizumab/économie , Bévacizumab/usage thérapeutique , Néovascularisation choroïdienne/économie , Économies/économie , Femelle , Humains , Injections intravitréennes , Mâle , Adulte d'âge moyen , Mydriatiques/administration et posologie , Pupille/effets des médicaments et des substances chimiques , Récepteurs aux facteurs de croissance endothéliale vasculaire/usage thérapeutique , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/usage thérapeutique , Reprise du traitement , Résultat thérapeutique , Facteur de croissance endothéliale vasculaire de type A/antagonistes et inhibiteurs , Dégénérescence maculaire humide/économieRÉSUMÉ
PURPOSE: The addition of aflibercept (AFL) or ramucirumab (RAM) to folinic acid, fluorouracil, and irinotecan (FOLFIRI) prolongs overall survival and progression-free survival compared with FOLFIRI alone in patients with metastatic colorectal cancer (mCRC) as second-line therapy. Although these combination regimens are recommended among the standard therapies, significant additional cost is a concern. The comparative cost-effectiveness of AFL and RAM was examined from the perspective of the Japanese health care payer. METHODS: A partitioned survival analysis was constructed. The data sources were the VELOUR (Aflibercept Versus Placebo in Combination With Irinotecan and 5-FU in the Treatment of Patients With Metastatic Colorectal Cancer After Failure of an Oxaliplatin Based Regimen) and RAISE (Ramucirumab Versus Placebo in Combination With Second-Line FOLFIRI in Patients With Metastatic Colorectal Carcinoma That Progressed During or After First-Line Therapy With Bevacizumab, Oxaliplatin, and a Fluoropyrimidine) trials, which compared FOLFIRI alone with AFL or RAM in second-line treatment for mCRC. The cost and effectiveness of the combination of AFL or RAM with FOLFIRI were compared with those of FOLFIRI alone and examined between both agents in a 10-year time horizon. The health outcomes were life-years (LYs) and quality-adjusted life-years (QALYs). The costs were 2019 revisions to the drug prices and medical fees. The robustness of the model was verified by 1-way sensitivity analyses and a probability sensitivity analysis. A 2% annual discount was applied to the expenses and QALYs. A willingness-to-pay threshold of ¥7.5 million was used. FINDINGS: Compared with FOLFIRI alone, combination AFL or RAM with FOLFIRI had incremental effects of 0.173 QALYs (0.253 LYs) and 0.137 QALYs (0.197 LYs), incremental costs of ¥3,423,481 (US $31,010) and ¥5,766,106 (US $52,229), and incremental cost-effectiveness ratios of ¥19, 836, 504 (US $179,678) and ¥41, 947, 989 (US $379,964) per QALY, respectively. Results of 1-way sensitivity analyses and probability sensitivity analysis all exceeded a willingness-to-pay threshold of ¥7.5 million. In the comparison of the 2 agents, AFL was a dominant over RAM. IMPLICATIONS: Adding AFL or RAM to FOLFIRI in the second line of mCRC treatment was not cost-effective in the Japanese health care system. On the basis of the results of this study, in the treatment of mCRC, it will be necessary to adjust the prices of AFL and RAM with the improvement of clinical parameters, such as survival time and adverse events. Of the 2 agents, AFL was more cost-effective than RAM.
Sujet(s)
Anticorps monoclonaux humanisés/économie , Protocoles de polychimiothérapie antinéoplasique/économie , Camptothécine/analogues et dérivés , Tumeurs colorectales/économie , Protéines de fusion recombinantes/économie , Adulte , Anticorps monoclonaux humanisés/usage thérapeutique , Protocoles de polychimiothérapie antinéoplasique/usage thérapeutique , Camptothécine/économie , Camptothécine/usage thérapeutique , Tumeurs colorectales/traitement médicamenteux , Tumeurs colorectales/anatomopathologie , Analyse coût-bénéfice , Fluorouracil/économie , Fluorouracil/usage thérapeutique , Humains , Japon , Leucovorine/économie , Leucovorine/usage thérapeutique , Récepteurs aux facteurs de croissance endothéliale vasculaire/usage thérapeutique , Protéines de fusion recombinantes/usage thérapeutique ,RÉSUMÉ
PURPOSE: To model Medicare Part B and patient savings associated with increased bevacizumab payment and use for intravitreal anti-vascular endothelial growth factor (VEGF) therapy. DESIGN: Cost analysis. PARTICIPANTS: Intelligent Research in Sight (IRIS®) Registry data. METHODS: Medicare claims and IRIS® Registry data were used to calculate Medicare Part B expenditures and patient copayments for anti-VEGF agents with increasing reimbursement and use of bevacizumab relative to ranibizumab and aflibercept. MAIN OUTCOME MEASURES: Medicare Part B costs and patient copayments for anti-VEGF agents in the Medicare fee-for-service population. RESULTS: Increasing bevacizumab reimbursement to $125.78, equalizing the dollar margin with aflibercept, would result in Medicare Part B savings of $468 million and patient savings of $119 million with a 10% increase in bevacizumab market share. CONCLUSIONS: Increased use of bevacizumab achievable with increased reimbursement to eliminate the financial disincentive to its use would result in substantial savings for the Medicare Part B program and for patients receiving anti-VEGF intravitreal injections.
Sujet(s)
Inhibiteurs de l'angiogenèse/économie , Économies/économie , Régimes de rémunération à l'acte/économie , Medicare part B (USA)/économie , Bévacizumab/économie , Dépenses de santé , Injections intravitréennes , Ranibizumab/économie , Récepteurs aux facteurs de croissance endothéliale vasculaire , Protéines de fusion recombinantes/économie , Enregistrements , États-Unis , Facteur de croissance endothéliale vasculaire de type A/antagonistes et inhibiteursRÉSUMÉ
PURPOSE: To perform 11- and 2-year health care sector (ophthalmic) and societal cost perspective reference case, cost-utility analyses comparing bevacizumab, ranibizumab, and aflibercept monotherapies for neovascular age-related macular degeneration (NVAMD). DESIGN: Cost-utility analysis. METHODS: The authors performed 11-year and 2-year ophthalmic and societal cost perspective, cost-utility analyses comparing bevacizumab, ranibizumab, and aflibercept monotherapies for neovascular age-related macular degeneration (NVAMD). We employed patient utilities, bilateral outcomes, 2018 U.S. dollars, vision-related mortality, a Medicare fee schedule, and CATT (Comparison of Age-Related Macular Degeneration Treatments) study and VIEW (VEGF Trap-Eye: Investigation of Efficacy and Safety in Wet AMD) trial. Cochrane data were also used. SETTING: Center for Value-Based Medicine. Patient/study population: patients with NVAMD. INTERVENTION: Cost-utility analyses using published data. Data-modeled 10-year vision outcomes were modeled forward to year 11. MAIN OUTCOME MEASUREMENT: These included cost-utility ratios (CURs), costs, and quality-adjusted life-years (QALYs) gained. $100,00/QALY was considered the US cost-effectiveness upper limit. RESULTS: Bevacizumab and ranibizumab each conferred an 11-year, 1.339 QALY gain versus observation. Aflibercept conferred a 1.380 QALY gain. Aflibercept conferred greater QALY gain for less cost than ranibizumab but was not cost-effective compared to bevacizumab ($1,151,451/QALY incremental CUR). The average ophthalmic cost perspective CUR for bevacizumab was $11,033/QALY, $79,600/QALY for ranibizumab, and $44,801/QALY for aflibercept. Eleven-year therapies saved a 1.0 year-of-life loss without treatment from the 11.0-year life expectancy. Early treatment was 138%-149% more cost-effective than late treatment. Two-year therapy prevented a 1-month-of-life loss, and revealed bevacizumab, ranibizumab, and aflibercept conferred 0.141, 0.141, and 0.164 QALY gains, respectively, with corresponding average CURs of $40,371/QALY, $335,726/QALY, and $168,006/QALY, respectively. CONCLUSIONS: From an ophthalmic (medical) cost perspective, bevacizumab, ranibizumab, and aflibercept NVAMD monotherapies were all cost-effective over 11 years, with bevacizumab 6.21× more cost-effective than ranibizumab and 3.06× more cost-effective than aflibercept. Two-year modeling revealed bevacizumab was cost-effective, whereas ranibizumab and aflibercept were not. Early treatment was critical for obtaining optimal vision and cost-effectiveness, as is long-term follow-up and adherence to treatment.
Sujet(s)
Inhibiteurs de l'angiogenèse/économie , Néovascularisation choroïdienne/économie , Analyse coût-bénéfice , Facteur de croissance endothéliale vasculaire de type A/antagonistes et inhibiteurs , Facteur de croissance endothéliale vasculaire de type A/économie , Dégénérescence maculaire humide/économie , Sujet âgé , Inhibiteurs de l'angiogenèse/usage thérapeutique , Bévacizumab/économie , Bévacizumab/usage thérapeutique , Néovascularisation choroïdienne/traitement médicamenteux , Coûts des médicaments , Femelle , Coûts des soins de santé , Humains , Injections intravitréennes , Mâle , Medicare (USA) , Années de vie ajustées sur la qualité , Ranibizumab/économie , Ranibizumab/usage thérapeutique , Récepteurs aux facteurs de croissance endothéliale vasculaire/usage thérapeutique , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/usage thérapeutique , États-Unis , Acuité visuelle , Dégénérescence maculaire humide/traitement médicamenteuxRÉSUMÉ
BACKGROUND: Age-related macular degeneration (AMD) is one of the leading causes of permanent blindness worldwide. The current mainstay of treatment for neovascular AMD (nAMD) is intravitreal injection of anti-vascular endothelial growth factor (anti-VEGF) agents: aflibercept, ranibizumab, and off-label bevacizumab. Injections can be given monthly, every two or three months ('extended-fixed'), or as needed (pro re nata (PRN)). A variant of PRN is 'treat-and-extend' whereby injections are resumed if recurrence is detected and then delivered with increasing intervals. Currently, injection frequency varies among practitioners, which underscores the need to characterize an optimized approach to nAMD management. OBJECTIVES: To investigate the effects of monthly versus non-monthly intravitreous injection of an anti-VEGF agent in people with newly diagnosed nAMD. SEARCH METHODS: We searched CENTRAL, MEDLINE, Embase, LILACS, and three trials registers from 2004 to October 2019; checked references; handsearched conference abstracts; and contacted pharmaceutical companies to identify additional studies. SELECTION CRITERIA: We included randomized controlled trials (RCTs) that compared different treatment regimens for anti-VEGF agents in people with newly diagnosed nAMD. We considered standard doses only (ranibizumab 0.5 mg, bevacizumab 1.25 mg, aflibercept 2.0 mg, or a combination of these). DATA COLLECTION AND ANALYSIS: We used standard Cochrane methods for trial selection, data extraction, and analysis. MAIN RESULTS: We included 15 RCTs. The total number of participants was 7732, ranging from 37 to 2457 in each trial. The trials were conducted worldwide. Of these, six trials exclusively took place in the US, and three included centers from more than one country. Eight trials were at high risk of bias for at least one domain and all trials had at least one domain at unclear risk of bias. Seven trials (3525 participants) compared a PRN regimen with a monthly injection regimen, of which five trials delivered four to eight injections using standard PRN and three delivered nine or 10 injections using a treat-and-extend regimen in the first year. The overall mean change in best-corrected visual acuity (BCVA) at one year was +8.8 letters in the monthly injection group. Compared to the monthly injection, there was moderate-certainty evidence that the mean difference (MD) in BCVA change at one year for the standard PRN subgroup was -1.7 letters (95% confidence interval (CI) -2.8 to -0.6; 4 trials, 2299 participants), favoring monthly injections. There was low-certainty evidence of a similar BCVA change with the treat-and-extend subgroup (0.5 letters, 95% CI -3.1 to 4.2; 3 trials, 1226 participants). Compared to monthly injection, there was low-certainty evidence that fewer participants gained 15 or more lines of vision with standard PRN treatment at one year (risk ratio (RR) 0.87, 95% CI 0.76 to 0.99; 4 trials, 2299 participants) and low-certainty evidence of a similar gain with treat-and-extend versus monthly regimens (RR 1.11, 95% CI 0.91 to 1.36; 3 trials, 1169 participants). The mean change in central retinal thickness was a decrease of -166 µm in the monthly injection group; the MD compared with standard PRN was 21 µm (95% CI 6 to 32; 4 trials, 2215 participants; moderate-certainty evidence) and with treat-and extend was 22 µm (95% CI 37 to -81 µm; 2 trials, 635 participants; low-certainty evidence), in favor of monthly injection. Only one trial (498 participants) measured quality of life and reported no evidence of a difference between regimens, but data could not be extracted (low-certainty evidence). Both PRN regimens (standard and 'treat-and-extend') used fewer injections than monthly regimens (standard PRN: MD -4.6 injections, 95% CI -5.4 to -3.8; 4 trials, 2336 participants; treat-and-extend: -2.4 injections, 95% CI -2.7 to -2.1 injections; moderate-certainty evidence for both comparisons). Two trials provided cost data (1105 participants, trials conducted in the US and the UK). They found that cost differences between regimens were reduced if bevacizumab rather than aflibercept or ranibizumab were used, since bevacizumab was less costly (low-certainty evidence). PRN regimens were associated with a reduced risk of endophthalmitis compared with monthly injections (Peto odds ratio (OR) 0.13, 95% CI 0.04 to 0.46; 6 RCTs, 3175 participants; moderate-certainty evidence). Using data from all trials included in this review, we estimated the risk of endophthalmitis with monthly injections to be 8 in every 1000 people per year. The corresponding risk for people receiving PRN regimens was 1 in every 1000 people per year (95% CI 0 to 4). Three trials (1439 participants) compared an extended-fixed regimen (number of injections reported in only one large trial: 7.5 in one year) with monthly injections. There was moderate-certainty evidence that BCVA at one year was similar for extended-fixed and monthly injections (MD in BCVA change compared to extended-fixed group: -1.3 letters, 95% CI -3.9 to 1.3; RR of gaining 15 letters or more: 0.94, 95% CI 0.80 to 1.10). The change in central retinal thickness was a decrease of 137 µm in the monthly group; the MD with the extended-fixed group was 8 µm (95% CI -11 to 27; low-certainty evidence). The frequency of endophthalmitis was lower in the extended-fixed regimen compared to the monthly group, but this estimate was imprecise (RR 0.19, 95% CI 0.03 to 1.11; low-certainty evidence). If we assumed a risk of 8 cases of endophthalmitis in 1000 people receiving monthly injections over one year, then the corresponding risk with extended-fixed regimen was 2 in 1000 people (95% CI 0 to 9). Other evidence comparing different extended-fixed or PRN regimens yielded inconclusive results. AUTHORS' CONCLUSIONS: We found that, at one year, monthly regimens are probably more effective than PRN regimens using seven or eight injections in the first year, but the difference is small and clinically insignificant. Endophthalmitis is probably more common with monthly injections and differences in costs between regimens are higher if aflibercept or ranibizumab are used compared to bevacizumab. This evidence only applies to settings in which regimens are implemented as described in the trials, whereas undertreatment is likely to be common in real-world settings. There are no data from RCTs on long-term effects of different treatment regimens.
Sujet(s)
Inhibiteurs de l'angiogenèse/administration et posologie , Dégénérescence maculaire/traitement médicamenteux , Facteur de croissance endothéliale vasculaire de type A/antagonistes et inhibiteurs , Acuité visuelle/effets des médicaments et des substances chimiques , Sujet âgé , Sujet âgé de 80 ans ou plus , Inhibiteurs de l'angiogenèse/économie , Bévacizumab/administration et posologie , Bévacizumab/économie , Biais (épidémiologie) , Calendrier d'administration des médicaments , Endophtalmie/épidémiologie , Endophtalmie/étiologie , Humains , Injections intravitréennes/effets indésirables , Dégénérescence maculaire/anatomopathologie , Qualité de vie , Essais contrôlés randomisés comme sujet , Ranibizumab/administration et posologie , Ranibizumab/économie , Récepteurs aux facteurs de croissance endothéliale vasculaire/administration et posologie , Protéines de fusion recombinantes/administration et posologie , Protéines de fusion recombinantes/économie , Rétine/effets des médicaments et des substances chimiquesRÉSUMÉ
OBJECTIVES: Outcomes-based contracts tie rebates and discounts for expensive drugs to outcomes. The objective was to estimate the utility of outcomes-based contracts for diabetes medications using real-world data and to identify methodologic limitations of this approach. METHODS: A population-based cohort study of adults newly prescribed a medication for diabetes with a publicly announced outcomes-based contract (ie, exenatide microspheres ["exenatide"], dulaglutide, or sitagliptin) was conducted. The comparison group included patients receiving canagliflozin or glipizide. The primary outcome was announced in the outcomes-based contract: the percentage of adults with a follow-up hemoglobin A1C <8% up to 1 year later. Secondary outcomes included the percentage of patients diagnosed with hypoglycemia and the cost of a 1-month supply. RESULTS: Thousands of adults newly filled prescriptions for exenatide (n = 5079), dulaglutide (n = 6966), sitagliptin (n = 40 752), canagliflozin (n = 16 404), or glipizide (n = 59 985). The percentage of adults subsequently achieving a hemoglobin A1C below 8% ranged from 83% (dulaglutide, sitagliptin) to 71% (canagliflozin). The rate of hypoglycemia was 25 per 1000 person-years for exenatide, 37 per 1000 person-years for dulaglutide, 28 per 1000 person-years for sitagliptin, 18 per 1000 person-years for canagliflozin, and 34 per 1000 person-years for glipizide. The cash price for a 1-month supply was $847 for exenatide, $859 for dulaglutide, $550 for sitagliptin, $608 for canagliflozin, and $14 for glipizide. CONCLUSION: Outcomes-based pricing of diabetes medications has the potential to lower the cost of medications, but using outcomes such as hemoglobin A1C may not be clinically meaningful because similar changes in A1C can be achieved with generic medications at a far lower cost.
Sujet(s)
Contrats/économie , Diabète de type 2/traitement médicamenteux , Hypoglycémiants/administration et posologie , /méthodes , Sujet âgé , Canagliflozine/administration et posologie , Canagliflozine/économie , Études de cohortes , Diabète de type 2/économie , Exénatide/administration et posologie , Exénatide/économie , Femelle , Études de suivi , Glipizide/administration et posologie , Glipizide/économie , Peptides glucagon-like/administration et posologie , Peptides glucagon-like/analogues et dérivés , Peptides glucagon-like/économie , Humains , Hypoglycémiants/économie , Fragments Fc des immunoglobulines/administration et posologie , Fragments Fc des immunoglobulines/économie , Mâle , Adulte d'âge moyen , Protéines de fusion recombinantes/administration et posologie , Protéines de fusion recombinantes/économie , Phosphate de sitagliptine/administration et posologie , Phosphate de sitagliptine/économieRÉSUMÉ
OBJECTIVE: The standard-of-care treatment for age-related macular degeneration (AMD) and diabetic macular edema (DME) includes inhibiting blood vessel proliferation and reducing macular edema or swelling using anti-vascular endothelial growth factor therapies, such as ranibizumab and aflibercept. To conduct a cost-minimization analysis of ranibizumab and aflibercept for treating Saudi patients with visual impairment owing to AMD or DME. METHODS: Cost minimization was analyzed assuming that ranibizumab and aflibercept have equivalent clinical effectiveness. The third-party payer's perspective was used in several clinical scenarios. The base-case scenario was DME cases followed monthly using a protocol-specific follow-up. In scenario 1, AMD cases followed a treat-and-extend protocol over 2 years. In scenario 2, AMD cases followed the PRN (pro re nata) regimen over 2 years. In scenario 3, DME cases followed the PRN regimen for 1 year only. RESULTS: Aflibercept yielded cost savings of 25.75%, 31.54%, 51.30%, and 9.28% compared with ranibizumab for the base case, scenario 1, scenario 2, and scenario 3, respectively, which supports the premise that aflibercept is more cost saving than ranibizumab. CONCLUSIONS: From the third-party payer perspective, aflibercept is a cost-containment option that provides substantial savings over ranibizumab for treating Saudi patients with AMD or DME.
Sujet(s)
Dégénérescence maculaire/traitement médicamenteux , Ranibizumab/économie , Dépistage visuel/économie , Inhibiteurs de l'angiogenèse/économie , Inhibiteurs de l'angiogenèse/usage thérapeutique , Coûts et analyse des coûts , Dépenses de santé/normes , Dépenses de santé/statistiques et données numériques , Humains , Dégénérescence maculaire/économie , Ranibizumab/usage thérapeutique , Récepteurs aux facteurs de croissance endothéliale vasculaire/usage thérapeutique , Protéines de fusion recombinantes/économie , Protéines de fusion recombinantes/usage thérapeutique , Arabie saoudite , Facteurs temps , Dépistage visuel/méthodesRÉSUMÉ
PURPOSE: Eltrombopag was evaluated as a second-line treatment for adult chronic immune thrombocytopenia (ITP) in the 2006 Phase III RAISE (Eltrombopag for Management of Chronic Immune Thrombocytopenia) randomized, placebo-controlled trial. More than 80% of patients reached satisfactory platelet counts within 2 weeks. However, the economic value of eltrombopag as a second-line treatment for ITP remains to be formally assessed. This study aimed to estimate the cost-effectiveness of treating ITP with a comparable thrombopoietin receptor agonist (eltrombopag vs romiplostim). METHODS: A Markov model was implemented over a lifetime time horizon to estimate the benefits and costs of each treatment. The model featured 3 health states based on current guidelines: (1) on treatment; (2) treatment failure/discontinuation; and (3) mortality. In line with therapeutic goals in ITP, model patients could experience 3 events: no bleeding, mild/moderate bleeding, or severe bleeding. Data on eltrombopag use were obtained from an open-label extension of previous Phase II/III trials, including RAISE. Romiplostim data were obtained from Phase III trials and an extension study. Lifetime overall survival was extrapolated by using treatment-specific mortality rates derived from severe bleeding and natural mortality rates. The costs of drugs, routine care, bleeding episodes, adverse events, and mortality were represented in the model. FINDINGS: Eltrombopag-treated patients gained 17.58 life years and 14.68 quality-adjusted life years, whereas romiplostim-treated patients gained 17.52 life years and 14.67 quality-adjusted life years. The total lifetime cost of eltrombopag treatment was estimated at $1.58 million versus $2.13 million for romiplostim. Sensitivity analyses supported base case findings. Deterministic sensitivity analysis predicted the greatest sensitivity to the rates of severe bleeding, discontinuation, and natural mortality. Probabilistic sensitivity analysis showed that eltrombopag would be an efficient use of resources at a $50,000 threshold in 52.8% of cases. In all probabilistic iterations, the total cost of eltrombopag treatment was lower than with romiplostim, primarily because of lower drug costs. IMPLICATIONS: Clinical data were applied in an economic analysis, and eltrombopag exhibited economic dominance compared with romiplostim, driven largely by the reduced costs of primary therapy. This model was limited by a lack of specific patient-level data and robust data on the duration of secondary therapy, as well as by the fact that utilization values are likely conservative estimates for routine care use.
Sujet(s)
Benzoates/économie , Hydrazines/économie , Purpura thrombopénique idiopathique/économie , Pyrazoles/économie , Récepteurs à la thrombopoïétine/agonistes , Protéines de fusion recombinantes/économie , Thrombopoïétine/économie , Benzoates/effets indésirables , Benzoates/usage thérapeutique , Essais cliniques de phase II comme sujet , Essais cliniques de phase III comme sujet , Analyse coût-bénéfice , Hémorragie/induit chimiquement , Humains , Hydrazines/effets indésirables , Hydrazines/usage thérapeutique , Numération des plaquettes , Purpura thrombopénique idiopathique/traitement médicamenteux , Pyrazoles/effets indésirables , Pyrazoles/usage thérapeutique , Années de vie ajustées sur la qualité , Essais contrôlés randomisés comme sujet , Récepteur Fc/usage thérapeutique , Protéines de fusion recombinantes/effets indésirables , Protéines de fusion recombinantes/usage thérapeutique , Thrombopoïétine/effets indésirables , Thrombopoïétine/usage thérapeutique , États-UnisRÉSUMÉ
BACKGROUND: Ranibizumab and aflibercept are FDA-approved treatments for patients with neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME). Although these agents differ in cost and labeled dosing, it is unclear whether these differences are reflected in clinical practice. OBJECTIVE: To compare the real-world frequency and cost of ranibizumab and aflibercept injections among treatment-naive and previously treated patients with nAMD and DME. METHODS: Claims data from MarketScan Research Databases were retrospectively reviewed to identify treatment-naive patients with nAMD who initiated intravitreal ranibizumab or aflibercept between January 1, 2014, and January 1, 2016, and treatment-naive patients with DME who initiated intravitreal ranibizumab or aflibercept between July 29, 2014, and July 1, 2016. Patients who switched to subsequent-line aflibercept or ranibizumab during the study period were eligible to enter previously treated subgroups. Multivariable regression models were derived to compare the per-patient frequency and cost of injections between ranibizumab- and aflibercept-treated patients with nAMD over 12 months (treatment-naive: n = 1,087 and n = 1,578; previously treated: n = 221 and n = 751) and 24 months (treatment-naive: n = 454 and n = 568; previously treated: n = 93 and n = 284) and in patients with DME over 6 months (treatment-naive: n = 507 and n = 681; previously treated: n = 53 and n = 223) and 12 months (treatment-naive: n = 326 and n = 382; previously treated: n = 24 and n = 122). RESULTS: After adjusting for patient demographics and clinical characteristics, per-patient injection frequency and cost were not significantly different between treatment-naive patients with nAMD who received ranibizumab versus aflibercept over 12 months (5.62 vs. 5.54; P = 0.52, and $11,351 vs. $10,702; P = 0.06, respectively) and 24 months (7.86 vs. 8.37; P = 0.16, and $16,286 vs. $16,666; P = 0.69, respectively). In previously treated patients with nAMD, injection frequency was significantly lower among ranibizumab- versus aflibercept-treated patients over 24 months (7.98 vs. 9.63; P = 0.03), whereas treatment costs were comparable over 12 months ($11,512 vs. $12,050; P = 0.44) and 24 months ($16,303 vs. $19,361; P = 0.13). In treatment-naive patients with DME, ranibizumab was associated with significantly fewer injections and lower costs than aflibercept over 6 months (2.60 vs. 2.92 and $3,379 vs. $5,925, respectively; both P < 0.001) and 12 months (3.33 vs. 3.87 and $4,136 vs. $7,656, respectively; both P < 0.001). Similar cost savings were observed among previously treated patients with DME who received ranibizumab over 6 months ($3,834 vs. $6,775 for aflibercept; P = 0.0001) and 12 months ($4,606 vs. $9,190; P = 0.02), despite nonsignificant differences in injection frequency during follow-up. CONCLUSIONS: Although the frequency and cost of ranibizumab and aflibercept injections were generally comparable among patients treated for nAMD, ranibizumab was associated with estimated per-patient-per-year cost savings of $3,500-$4,500 in those treated for DME. Most patients received fewer injections than any FDA-indicated dosing schedule, suggesting potential undertreatment that may result in suboptimal vision outcomes. DISCLOSURES: Study funding was provided by Genentech, a member of the Roche Group. The sponsor participated in the design of the study; collection, analysis, and interpretation of the data; preparation of the manuscript; and the decision to submit the article for publication. Kiss has been a consultant for and received honoraria from Alcon, Alimera, Allergan, BioMarin, Novartis, and Spark; has been on the advisory board for, a consultant for, received honoraria from, and held stock options in Adverum and Regenxbio; has been a consultant for, received honoraria from, and held stock/stock options in Fortress; has been on the advisory board for, a consultant and investigator for, and received grants and honoraria from Genentech and Regeneron; and has been on the advisory board for, a consultant for, and received grants and honoraria from Optos. Malangone-Monaco, Wilson, Varker, Stetsovsky, and Smith are employees of IBM Watson Health, which received funding from Genentech to undertake this study. Garmo is an employee of Genentech. Data reported in this manuscript were presented in part at the Academy of Managed Care Pharmacy (AMCP) Managed Care and Specialty Pharmacy Annual Meeting; April 23-26, 2018; Boston, MA.
Sujet(s)
Rétinopathie diabétique/traitement médicamenteux , Oedème maculaire/traitement médicamenteux , Ranibizumab/administration et posologie , Récepteurs aux facteurs de croissance endothéliale vasculaire/administration et posologie , Protéines de fusion recombinantes/administration et posologie , Sujet âgé , Sujet âgé de 80 ans ou plus , Inhibiteurs de l'angiogenèse/administration et posologie , Inhibiteurs de l'angiogenèse/économie , Rétinopathie diabétique/économie , Calendrier d'administration des médicaments , Coûts des médicaments , Femelle , Humains , Injections intravitréennes , Dégénérescence maculaire/traitement médicamenteux , Dégénérescence maculaire/économie , Oedème maculaire/économie , Mâle , Adulte d'âge moyen , Ranibizumab/économie , Protéines de fusion recombinantes/économie , Études rétrospectivesRÉSUMÉ
INTRODUCTION: Once-weekly semaglutide 1 mg is a novel glucagon-like peptide 1 receptor agonist (GLP-1 RA) that, in the SUSTAIN clinical trials, has demonstrated greater reductions in glycated haemoglobin (HbA1c) and body weight than the other GLP-1 RAs exenatide extended-release (ER) 2 mg, dulaglutide 1.5 mg and liraglutide 1.2 mg. The aim of this analysis was to evaluate the relative cost of control of achieving treatment goals in people with type 2 diabetes (T2D) treated with once-weekly semaglutide versus exenatide ER, dulaglutide and liraglutide from a UK perspective. METHODS: Proportions of patients reaching HbA1c targets (< 7.0% and < 7.5%), weight loss targets (≥ 5% reduction in body weight) and composite endpoints (HbA1c < 7.0% without weight gain or hypoglycaemia; reduction in HbA1c of ≥ 1% and weight loss of ≥ 5%) were obtained from the SUSTAIN clinical trials. Annual per patient treatment costs were based on wholesale acquisition costs from July 2019 in the UK. Cost of control was calculated by plotting relative treatment costs against relative efficacy. RESULTS: The annual per patient cost was similar for all GLP-1 RAs. Once-weekly semaglutide was superior to exenatide ER, dulaglutide and liraglutide in bringing patients to HbA1c and weight loss targets, and to composite endpoints. When looking at the composite endpoint of HbA1c < 7.0% without weight gain or hypoglycaemia, exenatide ER, dulaglutide and liraglutide were 50.0%, 21.6% and 51.3% less efficacious in achieving this, respectively, than once-weekly semaglutide. Consequently, the efficacy-to-cost ratios for once-weekly semaglutide were superior to all comparators in bringing patients to all endpoints. CONCLUSIONS: The present study showed that once-weekly semaglutide offers superior cost of control versus exenatide ER, dulaglutide and liraglutide in terms of achieving clinically relevant, single and composite endpoints. Once-weekly semaglutide 1 mg would therefore represent good value for money in the UK setting.