RESUMEN
OBJECTIVE: Evaluate the cost-effectiveness of primary prophylaxis (PP) or secondary prophylaxis (SP) with pegfilgrastim, filgrastim (6-day and 11-day), or no prophylaxis to reduce the risk of febrile neutropenia (FN) in patients with recurrent ovarian cancer receiving docetaxel or topotecan. METHODS: A Markov model was used to evaluate the cost-effectiveness of PP vs SP from a US payer perspective. Model inputs, including the efficacy of each strategy (relative risk of FN with prophylaxis compared to no prophylaxis) and mortality, costs, and utility values were estimated from public sources and peer-reviewed publications. Incremental cost-effectiveness was evaluated in terms of net cost per FN event avoided, incremental cost per life-year saved (LYS), and incremental cost per quality-adjusted life-year (QALY) gained over a lifetime horizon. Deterministic and probabilistic sensitivity analyses (DSA and PSA) were conducted. RESULTS: For patients receiving docetaxel, the incremental cost-effectiveness ratio (ICER) for PP vs SP with pegfilgrastim was $7900 per QALY gained, and PP with pegfilgrastim dominated all other comparators. For patients receiving topotecan, PP with pegfilgrastim dominated all comparators. Model results were most sensitive to baseline FN risk. PP vs SP with pegfilgrastim was cost effective in 68% and 83% of simulations for docetaxel and in >99% of simulations for topotecan at willingness-to-pay thresholds of $50,000 and $100,000 per QALY. CONCLUSIONS: PP with pegfilgrastim should be considered cost effective compared to other prophylaxis strategies in patients with recurrent ovarian cancer receiving docetaxel or topotecan with a high risk of FN.
Asunto(s)
Antineoplásicos/efectos adversos , Neutropenia Febril Inducida por Quimioterapia/prevención & control , Factor Estimulante de Colonias de Granulocitos/uso terapéutico , Recurrencia Local de Neoplasia/tratamiento farmacológico , Neoplasias Glandulares y Epiteliales/tratamiento farmacológico , Neoplasias Ováricas/tratamiento farmacológico , Carcinoma Epitelial de Ovario , Neutropenia Febril Inducida por Quimioterapia/etiología , Análisis Costo-Beneficio , Docetaxel , Costos de los Medicamentos , Femenino , Filgrastim , Factor Estimulante de Colonias de Granulocitos/economía , Humanos , Cadenas de Markov , Persona de Mediana Edad , Polietilenglicoles , Años de Vida Ajustados por Calidad de Vida , Proteínas Recombinantes/economía , Proteínas Recombinantes/uso terapéutico , Taxoides/efectos adversos , Topotecan/efectos adversosRESUMEN
BACKGROUND/OBJECTIVES: The phase 3 REGAIN study and its open-label extension demonstrated the efficacy of the complement C5 inhibitor eculizumab in patients with treatment-refractory, acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG). The aim of the ELEVATE study was to assess the effectiveness of eculizumab in clinical practice in adults with MG in the United States. METHODS: A retrospective chart review was conducted in adults with MG who initiated eculizumab treatment between October 23, 2017 and December 31, 2019. Outcomes assessed before and during eculizumab treatment using a pre- versus post-treatment study design included Myasthenia Gravis-Activities of Daily Living (MG-ADL) total scores; minimal symptom expression (MSE); physician impression of clinical change; minimal manifestation status (MMS); and concomitant medication use. RESULTS: In total, 119 patients were included in the study. A significant reduction was observed in mean MG-ADL total score, from 8.0 before eculizumab initiation to 5.4 at 3 months and to 4.7 at 24 months after eculizumab initiation (both p < 0.001). At 24 months after eculizumab initiation, MSE was achieved by 19% of patients. MMS or better was achieved by 30% of patients at 24 months. Additionally, 64% of patients receiving prednisone at eculizumab initiation had their prednisone dosage reduced during eculizumab treatment and 13% discontinued prednisone; 32% were able to discontinue nonsteroidal immunosuppressant therapy. DISCUSSION: Eculizumab treatment was associated with sustained improvements in MG-ADL total scores through 24 months in adults with MG. Prednisone dosage was reduced in approximately two-thirds of patients, suggesting a steroid-sparing effect for eculizumab.
RESUMEN
INTRODUCTION: Patients with generalized myasthenia gravis (gMG) experience functional impairment due to MG symptoms. This study aimed to assess, from the patient perspective, the symptoms, impacts, and treatment goals of individuals diagnosed with gMG. METHODS: Semi-structured, in-depth concept-elicitation interviews were conducted with 28 individuals diagnosed with gMG in the United States. RESULTS: Participants reported gMG symptoms that affected many body regions and functions, with an average of 16 symptoms per participant. The most frequently reported symptoms were eyelid drooping (93%), physical fatigue (89%), symptoms affecting the legs (82%), difficulty breathing (82%), and difficulty holding head up (82%). Nearly all participants (96%) reported fluctuations in symptoms and severity. Participants' most bothersome symptoms were blurry/double vision (43%), breathing difficulties (36%), all-over fatigue (36%), and swallowing problems (29%). Impacts on physical functioning included an inability to participate in hobbies/sports, need for increased planning, and difficulties performing activities of daily living. All participants reported emotional impacts and impacts on their work and finances. Their treatment goals included reduced fatigue and weakness, improved symptom stability, and minimization of the impact of symptoms, in particular the emotional impact. CONCLUSIONS: The fluctuating and unpredictable nature of gMG symptoms was found to have a substantial impact on patients' emotional, social, and economic well-being. Participants' goals for symptom management suggest that greater focus is needed to help them quickly resume a normal lifestyle by achieving symptom stability. Impacts of fluctuating and unpredictable symptoms are difficult to measure, but it is important to consider symptom fluctuation as well as ongoing symptomatology when making treatment decisions, and to recognize the impact of uncontrolled symptoms on patients, their partners/caregivers, and family/friends. These factors are often not reflected in burden/cost-of-illness studies.
The aim of this study was to understandfrom the patient's point of viewthe range of generalized myasthenia gravis (gMG) symptoms that they experience, which symptoms bother them most, and which symptoms have the greatest impact on everyday life, as well as patients' treatment goals. Researchers asked these questions in one-on-one interviews with 28 people in the US who have gMG. Study participants reported living with symptoms that come and go, and are sometimes severe, making it difficult to lead a normal life. The most frequently reported symptoms were eyelid drooping (reported by 93% of study participants), physical fatigue (89%), symptoms affecting the legs (82%), difficulty breathing (82%), and difficulty holding head up (82%). The symptoms that bothered patients most were difficulties with vision (43%), problems breathing (36%), all-over fatigue (36%), and trouble swallowing (29%). Participants reported that gMG symptoms affected physical functioning, making it hard to participate in hobbies/sports, increasing the amount of planning needed to conduct their daily lives, and hindering their ability to do day-to-day activities (like driving, eating, and bathing). All participants reported that they were affected emotionally, and that the symptoms of gMG impacted their ability to work and their financial well-being. Participants' treatment goals included reducing fatigue and weakness, making symptoms more stable, and reducing the impact of symptoms, particularly emotional impacts. These responses reveal the extensive effects of gMG symptoms on patients' daily lives and highlight that symptom stability is especially important to people with gMG.
RESUMEN
OBJECTIVE: To summarise the relationship between joint damage and functional disability in rheumatoid arthritis (RA) patients. METHODS: A systematic review of the literature from 1990 to 2008 was conducted using MEDLINE and EMBASE databases. The search strategy focused on RA, joint damage and disability. Only longitudinal studies or randomised clinical trials with 1 year or more of follow-up containing data correlating joint damage and disability were included. The comparisons were categorised in four ways: baseline damage versus disability at end of follow-up (correlation A); damage versus disability measured cross-sectionally at each of several time points (correlation B); changes in damage versus final disability (correlation C) and changes in damage versus changes in disability (correlation D). RESULTS: From a total of 1902 abstracts, 42 studies met the inclusion/exclusion criteria. More than 50% of the studies that measured baseline damage to later disability (A) reported a statistically significant association. Correlation was significant when measured at multiple time points over time (B; 16/19 studies). Statistically significant associations between changes in damage and either disability at end of follow-up or changes in disability were also found (C and D; 11/13 studies). CONCLUSIONS: While many of the studies did not include multivariate analysis with confounder adjustment, the published evidence indicates a link between joint damage and functional disability and that an increase in joint damage is associated with an increase in disability over time. Treatments to limit progressive joint damage may lead to better joint function and improved patient outcome with less disability.
Asunto(s)
Artritis Reumatoide/patología , Artritis Reumatoide/fisiopatología , Evaluación de la Discapacidad , Articulaciones/patología , Articulaciones/fisiopatología , Actividades Cotidianas , Artritis Reumatoide/terapia , Humanos , Ensayos Clínicos Controlados Aleatorios como AsuntoRESUMEN
OBJECTIVES: This analysis estimated the cost per response and the incremental cost per additional responder of romplostim, eltrombopag, and the "watch-and-rescue" (monitoring until rescue therapies are required) strategy in adults with chronic immune thrombocytopenia (ITP). STUDY DESIGN: The decision tree is designed to estimate the total cost per response for romiplostim, eltrombopag, and watch and rescue over a 24-week time horizon; cost-effectiveness was evaluated in terms of incremental cost per additional responder. METHODS: Model inputs including response rates, bleeding-related episode (BRE) rates, and costs were estimated from registrational trial data, an independent Bayesian indirect comparison, database analyses, and peer-reviewed publications. Costs were applied to the proportions of patients with treatment response and nonresponse (based on platelet count). The total cost per response and the incremental cost per additional responder for each treatment were calculated. Sensitivity analyses and alternative analyses were performed. RESULTS: With higher total costs and greater treatment efficacy, romiplostim and eltrombopag had a lower 24-week cost per response and a lower average number of BREs than watch and rescue. Eltrombopag was weakly dominated by romiplostim. The incremental cost-effectiveness ratio of romiplostim versus watch and rescue was $46,000 per additional responder. The model results are most sensitive to response rates of romiplostim and watch and rescue and the BRE rate for splenectomized nonresponders. Alternative analyses results were similar to the base case. CONCLUSIONS: In adults with chronic ITP, romiplostim represents an efficient way to achieve response, with lower costs per response than eltrombopag; both romiplostim and eltrombopag had lower costs per response than watch and rescue.
Asunto(s)
Benzoatos/economía , Análisis Costo-Beneficio , Árboles de Decisión , Costos de los Medicamentos , Hidrazinas/economía , Púrpura Trombocitopénica Idiopática/tratamiento farmacológico , Pirazoles/economía , Proteínas Recombinantes de Fusión/economía , Trombopoyetina/economía , Adulto , Teorema de Bayes , Benzoatos/uso terapéutico , Relación Dosis-Respuesta a Droga , Esquema de Medicación , Femenino , Humanos , Hidrazinas/uso terapéutico , Masculino , Púrpura Trombocitopénica Idiopática/diagnóstico , Púrpura Trombocitopénica Idiopática/economía , Pirazoles/uso terapéutico , Receptores Fc/uso terapéutico , Proteínas Recombinantes de Fusión/uso terapéutico , Trombopoyetina/uso terapéutico , Insuficiencia del Tratamiento , Resultado del TratamientoRESUMEN
INTRODUCTION: We reviewed the evolution of the methods used in cost-effectiveness analyses of granulocyte colony-stimulating factors (G-CSFs) in the primary and secondary prevention of febrile neutropenia (FN) in patients receiving myelosuppressive cancer chemotherapy. Areas covered: FN is a side effect of myelosuppressive chemotherapy associated with significant morbidity, mortality, and costs. The risk of FN may depend on the drugs used within a chemotherapy regimen, and an FN event may cause chemotherapy dose reductions or delays in subsequent cycles. Expert commentary: More recent pharmacoeconomic models have reflected these clinical observations by modeling sequential chemotherapy regimens to account for FN risk on a per-cycle basis, and by accounting for chemotherapy dose reductions and consequent survival losses.
Asunto(s)
Neutropenia Febril/prevención & control , Factor Estimulante de Colonias de Granulocitos/uso terapéutico , Modelos Económicos , Antineoplásicos/efectos adversos , Análisis Costo-Beneficio , Neutropenia Febril/inducido químicamente , Neutropenia Febril/economía , Factor Estimulante de Colonias de Granulocitos/economía , Humanos , Neoplasias/tratamiento farmacológico , Prevención Primaria/economía , Prevención Primaria/métodos , Prevención Secundaria/economía , Prevención Secundaria/métodosRESUMEN
OBJECTIVE: The objective of this study was to evaluate the cost effectiveness of no prophylaxis, primary prophylaxis (PP), or secondary prophylaxis (SP) with granulocyte colony-stimulating factors (G-CSFs), i.e., pegfilgrastim, lipegfilgrastim, filgrastim (6- and 11-day), or lenograstim (6- and 11-day), to reduce the incidence of febrile neutropenia (FN) in patients with stage II breast cancer receiving TC (docetaxel, cyclophosphamide) and in patients with non-Hodgkin lymphoma (NHL) receiving R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone) over a lifetime horizon from a Belgian payer perspective. METHODS: A Markov cycle tree tracked FN events during chemotherapy (3-week cycles) and long-term survival (1-year cycles). Model inputs, including the efficacy of each strategy, risk of reduced relative dose intensity (RDI), and the impact of RDI on mortality, utilities, and costs (in ; 2014 values) were estimated from public sources and the published literature. Incremental cost-effectiveness ratios (ICERs) were assessed for each strategy for costs per FN event avoided, life-year (LY) saved, and quality-adjusted LY (QALY) saved. LYs and QALYs saved were discounted at 1.5% annually. Deterministic and probabilistic sensitivity analyses (DSAs and PSAs) were conducted. RESULTS: Base-case ICERs for PP with pegfilgrastim relative to SP with pegfilgrastim were 15,500 per QALY and 14,800 per LY saved for stage II breast cancer and 7800 per QALY and 6900 per LY saved for NHL; other comparators were either more expensive and less effective than PP or SP with pegfilgrastim or had lower costs but higher ICERs (relative to SP with pegfilgrastim) than PP with pegfilgrastim. Results of the DSA for breast cancer and NHL comparing PP and SP with pegfilgrastim indicate that the model results were most sensitive to the cycle 1 risk of FN, the proportion of FN events requiring hospitalization, the relative risk of FN in cycles ≥2 versus cycle 1, no history of FN, and the mortality hazard ratio for RDI (<90% vs ≥90% [for NHL]). In the PSAs for stage II breast cancer and NHL, the probabilities that PP with pegfilgrastim was cost effective or dominant versus all other prophylaxis strategies at a 30,000/QALY willingness-to-pay threshold were 52% (other strategies ≤24%) and 58% (other strategies ≤24%), respectively. CONCLUSION: From a Belgian payer perspective, PP with pegfilgrastim appears cost effective compared to other prophylaxis strategies in patients with stage II breast cancer or NHL at a 30,000/QALY threshold.
Asunto(s)
Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Neutropenia Febril/prevención & control , Factor Estimulante de Colonias de Granulocitos/administración & dosificación , Años de Vida Ajustados por Calidad de Vida , Protocolos de Quimioterapia Combinada Antineoplásica/administración & dosificación , Protocolos de Quimioterapia Combinada Antineoplásica/economía , Bélgica , Neoplasias de la Mama/tratamiento farmacológico , Neoplasias de la Mama/patología , Análisis Costo-Beneficio , Neutropenia Febril/economía , Neutropenia Febril/epidemiología , Femenino , Factor Estimulante de Colonias de Granulocitos/economía , Hospitalización/economía , Hospitalización/estadística & datos numéricos , Humanos , Incidencia , Linfoma no Hodgkin/tratamiento farmacológico , Linfoma no Hodgkin/patología , Masculino , Cadenas de Markov , Estadificación de Neoplasias , Tasa de SupervivenciaRESUMEN
PURPOSE: The goal of this study was to assess and compare the potential clinical and economic value of emerging bone-forming agents using the only currently available agent, teriparatide, as a reference case in patients at high, near-term (imminent, 1- to 2-year) risk of osteoporotic fractures, extending to a lifetime horizon with sequenced antiresorptive agents for maintenance treatment. METHODS: Analyses were performed by using a Markov cohort model accounting for time-specific fracture protection effects of bone-forming agents followed by antiresorptive treatment with denosumab. The alternative bone-forming agent profiles were defined by using assumptions regarding the onset and total magnitude of protection against fractures with teriparatide. The model cohort comprised 70-year-old female patients with T scores below -2.5 and a previous vertebral fracture. Outcomes included clinical fractures, direct costs, and quality-adjusted life years. The simulated treatment strategies were compared by calculating their incremental "value" (net monetary benefit). FINDINGS: Improvements in the onset and magnitude of fracture protection (vs the teriparatide reference case) produced a net monetary benefit of $17,000,000 per 10,000 treated patients during the (1.5-year) bone-forming agent treatment period and $80,000,000 over a lifetime horizon that included 3.5 years of maintenance treatment with denosumab. IMPLICATIONS: Incorporating time-specific fracture effects in the Markov cohort model allowed for estimation of a range of cost savings, quality-adjusted life years gained, and clinical fractures avoided at different levels of fracture protection onset and magnitude. Results provide a first estimate of the potential "value" new bone-forming agents (romosozumab and abaloparatide) may confer relative to teriparatide.
Asunto(s)
Conservadores de la Densidad Ósea/uso terapéutico , Osteoporosis Posmenopáusica/tratamiento farmacológico , Fracturas Osteoporóticas/prevención & control , Anciano , Anticuerpos Monoclonales/economía , Anticuerpos Monoclonales/uso terapéutico , Conservadores de la Densidad Ósea/economía , Análisis Costo-Beneficio , Denosumab/economía , Denosumab/uso terapéutico , Femenino , Humanos , Modelos Teóricos , Osteoporosis Posmenopáusica/economía , Fracturas Osteoporóticas/economía , Proteína Relacionada con la Hormona Paratiroidea/economía , Proteína Relacionada con la Hormona Paratiroidea/uso terapéutico , Posmenopausia , Años de Vida Ajustados por Calidad de Vida , Riesgo , Teriparatido/economía , Teriparatido/uso terapéuticoRESUMEN
Purpose. To evaluate the cost-effectiveness of denosumab versus other osteoporotic treatments in older men with osteoporosis from a US payer perspective. Methods. A lifetime cohort Markov model previously developed for postmenopausal osteoporosis (PMO) was used. Men in the model were 78 years old, with a BMD T-score of -2.12 and a vertebral fracture prevalence of 23%. During each 6-month Markov cycle, patients could have experienced a hip, vertebral or nonhip, nonvertebral (NHNV) osteoporotic fracture, remained in a nonfracture state, remained in a postfracture state, or died. Background fracture risks, mortality rates, persistence rates, health utilities, and medical and drug costs were derived from published sources. Previous PMO studies were used for drug efficacy in reducing fracture risk. Lifetime expected costs and quality-adjusted life-years (QALYs) were estimated for denosumab, generic alendronate, risedronate, ibandronate, teriparatide, and zoledronate. Results. Denosumab had an incremental cost-effectiveness ratio (ICER) of $16,888 compared to generic alendronate and dominated all other treatments. Results were most sensitive to changes in costs of denosumab and the relative risk of hip fracture. Conclusion. Despite a higher annual treatment cost compared to other medications, denosumab is cost-effective compared to other osteoporotic treatments in older osteoporotic US men.
RESUMEN
BACKGROUND: Adjuvant trastuzumab treatment is administered to early stage breast cancer patients in physician office (POV) or hospital outpatient (HOP) settings. OBJECTIVE: To identify treatment patterns, utilization, and costs by site of care (POV vs. HOP) of patients with adjuvant treatment of breast cancer with trastuzumab. METHODS: This retrospective analysis identified adult, female breast cancer patients who initiated trastuzumab treatment (index date) from a large U.S. claims database. Inclusion criteria also required ≥ 2 claims for both trastuzumab (from July 1, 2006, to July 31, 2012) and breast cancer (during 6-month pre-index baseline period), no evidence of metastatic breast cancer or other cancers in the baseline period, and continuous enrollment with commercial or Medicare Advantage coverage 6 months pre- and post-index, except that patients who died during follow-up were retained. Patients with evidence of trastuzumab receipt during the baseline period or more than 1 site of care during follow-up were excluded. Patients were stratified by site of care and were followed from index date to 30 days after the last trastuzumab infusion prior to a gap ≥ 90 days, death, disenrollment, or end-of-study period. Differences in treatment patterns between the POV and HOP cohorts were assessed by t-test and chi-square test. The relationship between site of care and health care costs was modeled with a generalized linear model with gamma distribution and log link, and the number of trastuzumab infusions was modeled with negative binomial regression controlling for log follow-up time. All models were adjusted for age, baseline comorbidity score, and insurance type. RESULTS: Of the 3,439 breast cancer patients identified, 77.6% (2,669) received adjuvant trastuzumab in the POV setting. Mean age (53.7 years) and baseline comorbidity score (3.91) were similar among cohorts; a higher percentage of POV versus HOP patients had commercial insurance (91.1% vs. 86.4%, P < 0.001). Compared with the POV cohort, HOP patients had a shorter mean duration of trastuzumab treatment (324.8 vs. 343.0 days, P < 0.001); more treatment gaps (30-59 day gap: 67.4% vs. 24.1%, P < 0.001); and fewer trastuzumab infusions per month (1.37 vs. 1.98, P < 0.001) during follow-up. In multivariate analysis, the monthly count of trastuzumab infusions in the HOP cohort was lower than the POV cohort (incidence rate ratio = 0.693; 95% CI = 0.672-0.715). Adjusted per patient per month total health care costs were 53.6 % higher in the HOP setting (cost ratio = 1.536, 95% CI = 1.472-1.604). CONCLUSIONS: Breast cancer patients treated with adjuvant trastuzumab in the HOP setting had a shorter duration of trastuzumab treatment and fewer trastuzumab infusions but incurred higher monthly total costs than patients treated in the POV setting.
Asunto(s)
Atención Ambulatoria/economía , Anticuerpos Monoclonales Humanizados/administración & dosificación , Anticuerpos Monoclonales Humanizados/economía , Antineoplásicos/administración & dosificación , Antineoplásicos/economía , Neoplasias de la Mama/tratamiento farmacológico , Neoplasias de la Mama/economía , Costos de los Medicamentos , Recursos en Salud/economía , Visita a Consultorio Médico/economía , Servicio Ambulatorio en Hospital/economía , Neoplasias de la Mama/patología , Quimioterapia Adyuvante , Distribución de Chi-Cuadrado , Esquema de Medicación , Revisión de la Utilización de Medicamentos , Femenino , Recursos en Salud/estadística & datos numéricos , Humanos , Modelos Lineales , Programas Controlados de Atención en Salud , Persona de Mediana Edad , Modelos Económicos , Análisis Multivariante , Estadificación de Neoplasias , Pautas de la Práctica en Medicina/economía , Estudios Retrospectivos , Factores de Tiempo , Trastuzumab , Estados UnidosRESUMEN
PURPOSE: The objective of this study was to compare the clinical benefit across adjuvant therapies for cancer treatment, including adjuvant imatinib, and to quantify the results using the number-needed-to-treat (NNT) approach. METHOD: We reviewed studies meeting the following criteria: 1) US and European randomized clinical trial populations consisting of patients with cancer who underwent surgical resection of the primary tumor and were considered cancer free; 2) comparators were either placebo or no treatment; and 3) recurrence-free survival (RFS) and overall survival (OS) rates were reported and showed benefit with the experimental treatment. The NNT was calculated as the inverse of the difference in event rate between the study groups in each trial. RESULTS: We identified 26 adjuvant treatment trials in 9 cancer types. With longer follow-up (3 years vs 1 year), 62.5% of treatments compared with placebo showed a decreased RFS NNT, including imatinib (7 vs 4). The largest relative decrease in RFS NNT over time was 91% (with trastuzumab or cyclophosphamide therapy). Approximately 25% of the treatments resulted in an increase in RFS NNT over time. The RFS NNT for imatinib was lower than that for all other treatments at 3 years of follow-up and lower than that for all but 2 treatments at 1 year. At both year 1 and year 3, the NNT for OS ranged from 6 to 100. Imatinib had an OS NNT of 31 at 3 years. CONCLUSION: With longer follow-up duration, most adjuvant cancer treatments showed a decreased NNT. Imatinib had one of the lowest NNTs among the adjuvant treatments at 1 and 3 years of follow-up using the RFS data.
Asunto(s)
Antineoplásicos/uso terapéutico , Neoplasias/tratamiento farmacológico , Benzamidas/uso terapéutico , Quimioterapia Adyuvante , Supervivencia sin Enfermedad , Humanos , Mesilato de Imatinib , Neoplasias/mortalidad , Neoplasias/cirugía , Números Necesarios a Tratar , Piperazinas/uso terapéutico , Pirimidinas/uso terapéutico , Ensayos Clínicos Controlados Aleatorios como Asunto , Tasa de Supervivencia , Resultado del TratamientoRESUMEN
BACKGROUND: Recent clinical trial data have demonstrated that 3 years vs 1 year of adjuvant imatinib therapy for patients with surgically resected Kit+ Gastrointestinal Stromal Tumors (GIST) leads to a significant improvement in recurrence-free survival and overall survival. This study assesses the cost-effectiveness of treating patients with 3 years vs 1 year of imatinib from a US payer's perspective. METHODS: A Markov model was developed to predict GIST recurrence and treatment costs. Patients enter the model after surgery and transition among three health states: free of recurrence, recurrence, and death. Recurrence, mortality, costs, and utilities were derived from clinical trial and published literature. Expected costs and quality-adjusted life years (QALYs) were estimated and discounted at 3%/year. Deterministic and probabilistic sensitivity analyses were conducted. RESULTS: Patients receiving 3 years of imatinib had higher QALYs (8.53 vs 7.18) than those receiving 1 year of imatinib. Total lifetime per-patient cost was $302,100 for 3 years vs $217,800 for 1 year of imatinib. Incremental cost effectiveness ratio of 3 years vs 1 year of imatinib was $62,600/QALY. Model results were sensitive to long-term rate of GIST recurrence (beyond 5 years) and cost of imatinib. At a threshold of $100,000/QALY, 3 years vs 1 year of imatinib was cost-effective in 100% of simulations. LIMITATIONS: The model is a simplified representation of disease natural history and may not account for all possible health states and complications associated with disease. Resource utilization on treatment was estimated using the resource use data from previous trials, therefore calculated medical costs might be over-estimated compared to the real-world setting. CONCLUSIONS: Model results suggest that treatment with 3 years vs 1 year of imatinib is cost-effective at a $100,000/QALY threshold. Clinical and economic results suggest treating surgically resected Kit+ GIST patients with 3 years of imatinib would result in improved quality-adjusted survival.
Asunto(s)
Antineoplásicos/economía , Antineoplásicos/uso terapéutico , Benzamidas/economía , Benzamidas/uso terapéutico , Tumores del Estroma Gastrointestinal/tratamiento farmacológico , Piperazinas/economía , Piperazinas/uso terapéutico , Pirimidinas/economía , Pirimidinas/uso terapéutico , Quimioterapia Adyuvante , Análisis Costo-Beneficio , Supervivencia sin Enfermedad , Relación Dosis-Respuesta a Droga , Tumores del Estroma Gastrointestinal/epidemiología , Tumores del Estroma Gastrointestinal/cirugía , Humanos , Mesilato de Imatinib , Cadenas de Markov , Modelos Econométricos , Años de Vida Ajustados por Calidad de Vida , Factores de Tiempo , Estados Unidos/epidemiologíaRESUMEN
BACKGROUND: In the US, 26 % of women aged ≥65 years, and over 50 % of women aged ≥85 years are affected with postmenopausal osteoporosis (PMO). Each year, the total direct health care costs are estimated to be $US12-18 billion. OBJECTIVE: The cost effectiveness of denosumab versus oral bisphosphonates in postmenopausal osteoporotic women from a US third-party payer perspective was evaluated. METHODS: A lifetime cohort Markov model was developed with seven health states: 'well', hip fracture, vertebral fracture, 'other' osteoporotic fracture, post-hip fracture, post-vertebral fracture, and dead. During each cycle, patients could have a fracture, remain healthy, remain in a post-fracture state or die. Relative fracture risk reductions, background fracture risks, mortality rates, treatment-specific persistence rate, utilities, and medical and drug costs were derived using published sources. Expected costs and quality-adjusted life years (QALYs) were estimated for generic alendronate, denosumab, branded risedronate, and branded ibandronate in the overall PMO population and high-risk subgroups: (a) ≥2 of the following risks: >70 years of age, bone mineral density (BMD) T score less than or equal to -3.0, and prevalent vertebral fracture; and (b) ≥75 years of age. Costs and QALYs were discounted at 3 % annually, and all costs were inflated to 2012 US dollars. Sensitivity analyses were conducted by varying parameters e.g., efficacies of interventions, costs, utilities, and the medication persistence ratio. RESULTS: In the overall PMO population, total lifetime costs for alendronate, denosumab, risedronate, and ibandronate were $US64,400, $US67,400, $US67,600 and $US69,200, respectively. Total QALYs were 8.2804, 8.3155, 8.2735 and 8.2691, respectively. The incremental cost-effectiveness ratio (ICER) for denosumab versus generic alendronate was $US85,100/QALY. Risedronate and ibandronate were dominated by denosumab. In the high-risk subgroup (a), total costs for alendronate, denosumab, risedronate and ibandronate were $US70,400, $US70,800, $US74,000 and $US76,900, respectively. Total QALYs were 7.2006, 7.2497, 7.1969 and 7.1841, respectively. Denosumab had an ICER of $US7,900/QALY versus generic alendronate and dominated all other strategies. Denosumab dominated all strategies in women aged ≥75 years. Base-case results between denosumab and generic alendronate were most sensitive to the relative risk of hip fracture for both drugs and the cost of denosumab. CONCLUSION: In each PMO population examined, denosumab represented good value for money compared with branded bisphosphonates. Furthermore, denosumab was either cost effective or dominant compared with generic alendronate in the high-risk subgroups.
Asunto(s)
Anticuerpos Monoclonales Humanizados/economía , Conservadores de la Densidad Ósea/economía , Difosfonatos/economía , Osteoporosis Posmenopáusica/prevención & control , Anciano , Anciano de 80 o más Años , Alendronato/economía , Alendronato/uso terapéutico , Anticuerpos Monoclonales Humanizados/uso terapéutico , Conservadores de la Densidad Ósea/uso terapéutico , Análisis Costo-Beneficio , Denosumab , Difosfonatos/uso terapéutico , Costos de los Medicamentos , Ácido Etidrónico/análogos & derivados , Ácido Etidrónico/economía , Ácido Etidrónico/uso terapéutico , Femenino , Costos de la Atención en Salud/estadística & datos numéricos , Humanos , Reembolso de Seguro de Salud/economía , Reembolso de Seguro de Salud/estadística & datos numéricos , Cadenas de Markov , Osteoporosis Posmenopáusica/economía , Ácido Risedrónico , Suecia , Tiofenos/economía , Tiofenos/uso terapéutico , Estados UnidosRESUMEN
BACKGROUND: Results from the PROVE IT trial suggest that patients with acute coronary syndrome (ACS) treated with atorvastatin 80 mg/day (A80) have significantly lower rates of cardiovascular events compared with patients treated with pravastatin 40 mg/day (P40). In a genetic post hoc substudy of the PROVE IT trial, the rate of event reduction was greater in carriers of the Trp719Arg variant in kinesin family member 6 protein (KIF6) than in noncarriers. We assessed the cost effectiveness of testing for the KIF6 variant followed by targeted statin therapy (KIF6 Testing) versus not testing patients (No Test) and treating them with P40 or A80 in the USA from a payer perspective. METHODS: A Markov model was developed in which 2-year event rates from PROVE IT were extrapolated over a lifetime horizon. Costs and utilities were derived from published literature. All costs were in 2010 US dollars except the cost of A80, which was in 2012 US dollars because the generic formulation was available in 2012. Expected costs and quality-adjusted life-years (QALYs) were estimated for each strategy over a lifetime horizon. RESULTS: Lifetime costs were US$31,700; US$37,100 and US$41,300 for No Test P40, KIF6 Testing and No Test A80 strategies, respectively. The No Test A80 strategy was associated with more QALYs (9.71) than the KIF6 Testing (9.69) and No Test P40 (9.57) strategies. No Test A80 had an incremental cost-effectiveness ratio (ICER) of US$232,100 per QALY gained compared with KIF6 Testing. KIF6 Testing had an ICER of US$45,300 per QALY compared with No Test P40. CONCLUSIONS: Testing ACS patients for KIF6 carrier status may be a cost-effective strategy at commonly accepted thresholds. Treating all patients with A80 is more expensive than treating patients on the basis of KIF6 results, but the modest gain in QALYs is achieved at a cost/QALY that is generally considered unacceptable compared with the KIF6 Testing strategy. Compared with treating all patients with P40, the KIF6 Testing strategy had an ICER below US$50,000 per QALY. The conclusions from this study are sensitive to the price of generic A80 and the effect on adherence of knowing KIF6 carrier status. The results were based on a post hoc substudy of the PROVE IT trial, which was not designed to test the effectiveness of KIF6 testing.
Asunto(s)
Síndrome Coronario Agudo/tratamiento farmacológico , Ácidos Heptanoicos/uso terapéutico , Inhibidores de Hidroximetilglutaril-CoA Reductasas/uso terapéutico , Cinesinas/genética , Pirroles/uso terapéutico , Síndrome Coronario Agudo/economía , Síndrome Coronario Agudo/genética , Atorvastatina , Análisis Costo-Beneficio , Relación Dosis-Respuesta a Droga , Femenino , Pruebas Genéticas/economía , Pruebas Genéticas/métodos , Genotipo , Ácidos Heptanoicos/economía , Ácidos Heptanoicos/farmacología , Humanos , Inhibidores de Hidroximetilglutaril-CoA Reductasas/economía , Inhibidores de Hidroximetilglutaril-CoA Reductasas/farmacología , Masculino , Cadenas de Markov , Persona de Mediana Edad , Terapia Molecular Dirigida , Pravastatina/administración & dosificación , Pravastatina/economía , Pravastatina/uso terapéutico , Pirroles/economía , Pirroles/farmacología , Años de Vida Ajustados por Calidad de VidaRESUMEN
OBJECTIVE: To determine the cost-effectiveness of several external beam radiation treatment modalities for the treatment of patients with localized prostate cancer. METHODS: A lifetime Markov model incorporated the probabilities of experiencing treatment-related long-term toxicity or death. Toxicity probabilities were derived from published sources using meta-analytical techniques. Utilities and costs in the model were obtained from publicly available secondary sources. The model calculated quality-adjusted life expectancy and expected lifetime cost per patient, and derived ratios of incremental cost per quality-adjusted life year (QALY) gained between treatments. Analyses were conducted from both payer and societal perspectives. One-way and probabilistic sensitivity analyses were performed. RESULTS: Compared to intensity-modulated radiation therapy (IMRT) and proton beam therapy (PT), stereotactic body radiation therapy (SBRT) was less costly and resulted in more QALYs. Sensitivity analyses showed that the conclusions in the base-case scenario were robust with respect to variations in toxicity and cost parameters consistent with available evidence. At a threshold of $50,000/QALY, SBRT was cost-effective in 75% and 94% of probabilistic simulations compared to IMRT and PT, respectively, from a payer perspective. From a societal perspective, SBRT was cost-effective in 75% and 96% of simulations compared to IMRT and PT, respectively, at a threshold of $50,000/QALY. In threshold analyses, SBRT was less expensive with better outcomes compared to IMRT at toxicity rates 23% greater than the SBRT base-case rates. CONCLUSION: Based on the assumption that each treatment modality results in equivalent long-term efficacy, SBRT is a cost-effective strategy resulting in improved quality-adjusted survival compared to IMRT and PT for the treatment of localized prostate cancer.
RESUMEN
BACKGROUND: Trial TAX 324 showed that induction chemotherapy with docetaxel plus cisplatin and 5-fluorouracil (TPF) compared with cisplatin and 5-fluorouracil (PF) followed by chemoradiation increases survival and time to progression in squamous cell carcinoma of head and neck (SCCHN). METHODS: A Markov model was developed to estimate the cost-effectiveness of induction chemotherapy with docetaxel in the United Kingdom from the payer perspective. Health states were based on the WHO criteria for objective response to cancer treatments. Efficacy and safety data were obtained from the trial. Resource utilization, costs, and utility data were primarily derived from the literature. RESULTS: A patient on TPF gained 4.1 quality adjusted life years (QALYs) versus 2.0 QALYs for PF patient. Corresponding lifetime costs were pound 32,440 and pound 28,718. The cost per QALY gained for TPF versus PF was pound 1782. CONCLUSIONS: TPF is cost-effective as induction chemotherapy in locally advanced SCCHN compared with PF. Local treatment patterns may impact findings for other countries.
Asunto(s)
Antimetabolitos Antineoplásicos/uso terapéutico , Protocolos de Quimioterapia Combinada Antineoplásica/economía , Carcinoma de Células Escamosas/tratamiento farmacológico , Neoplasias de Cabeza y Cuello/tratamiento farmacológico , Fármacos Sensibilizantes a Radiaciones/uso terapéutico , Taxoides/economía , Antimetabolitos Antineoplásicos/economía , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Carcinoma de Células Escamosas/economía , Carcinoma de Células Escamosas/radioterapia , Cisplatino/administración & dosificación , Costo de Enfermedad , Análisis Costo-Beneficio , Progresión de la Enfermedad , Docetaxel , Fluorouracilo/administración & dosificación , Fluorouracilo/economía , Neoplasias de Cabeza y Cuello/economía , Neoplasias de Cabeza y Cuello/radioterapia , Humanos , Cadenas de Markov , Terapia Neoadyuvante , Calidad de Vida , Años de Vida Ajustados por Calidad de Vida , Taxoides/uso terapéutico , Reino UnidoRESUMEN
Fibromyalgia is a complex, chronic condition involving persistent and widespread pain of unknown origin. In the USA the syndrome affects approximately 2% of the population, with prevalence increasing with age, and has a greater predominance in females. The results of this review have found that there is a considerable economic burden associated with this disease, and in particular, the effect of the syndrome on economic losses associated with work activities is at least as important as direct medical costs. The humanistic burden is more difficult to quantify due to the numerous symptoms associated with this disease: widespread pain, persistent fatigue, feeling of weakness, sleep disturbances, morning stiffness, bowel and bladder irritability, mood disturbances, cognitive difficulties, dyesthesia/parethesia, chronic rhinitis, palpitations, auditory/vestibular/ocular complaints, regional pain and joint swelling. Overall, additional research is needed to quantify the impact of fibromyalgia on sufferers in the USA and the cost-effectiveness of alternative treatment strategies.
RESUMEN
Chronic low back pain is identified by the length of time a patient suffers from low back pain, the location of the pain and the etiology of the symptoms. Approximately 5-10% of patients with low back pain develop chronic low back pain that lasts longer than 3 months. There has been no consensus regarding the definition of low back pain; therefore, there is a wide variation in the prevalence estimates reported in the literature. Commonly used drugs for chronic low back pain include antidepressants, analgesics, antiepileptic drugs and muscle relaxants. In the USA, back pain is one of the most frequent reasons for hospitalization and physician visits, resulting in high medical care costs.
RESUMEN
Given the burden of illness related to diabetes, hypertension and dyslipidemia, it is very important to achieve glycemic control, optimal blood pressure and low-density lipoprotein cholesterol (LDL-C) in order to avoid severe long-term complications. Patients' adherence with, and persistence to, the treatment regimen is a critical factor in achieving this goal. Medication taking behavior in these chronic, nonsymptomatic ('silent') diseases is generally low, although a wide range of results have been reported. The literature has shown that nonadherence to medications is a multidimensional phenomenon; relating factors can be grouped into the following categories: health system related, social/economic, condition-related, therapy-related and patient-related factors. Although several interventions exist to improve patients' medication-taking behavior, none appear to be clearly superior to others. The key steps to improve adherence are to identify individual barriers and to develop patient-specific self-management plans to overcome them (called 'patient-centric' approach). When developing intervention strategies one should always remember that 'one size does not fit all'. Well designed (but not randomized), observational studies (for example, patient registries) may be required with sufficient follow-up periods and multiple adherence measurements in order to advance the field.