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1.
Haemophilia ; 27(2): 245-252, 2021 Mar.
Artículo en Inglés | MEDLINE | ID: mdl-33595148

RESUMEN

INTRODUCTION: People with inherited and long-term conditions such as haemophilia have been shown to adapt to their levels of disability, often reporting better quality of life (QoL) than expected from the general population (the disability paradox). AIM: To investigate the disability paradox in people with haemophilia in the United States by examining preference differences in health state valuations versus the general population. METHODS: We conducted a discrete choice experiment including duration to capture valuations of health states based on patient-reported preferences. Participants indicated their preferences for hypothetical health states using the EQ-5D-5L, where each participant completed 15 of the 120 choice tasks. Response inconsistencies were evaluated with dominated and repeated scenarios. Conditional-logit regressions with random sampling of the general population responses were used to match the sample of patients with haemophilia. We compared model estimates and derived preferences associated with EQ-5D-5L health states. RESULTS: After removing respondents with response inconsistencies, 1327/2138 (62%) participants remained (177/283 haemophilia; 1150/1900 general population). Patients with haemophilia indicated higher preference value for 99% of EQ-5D-5L health states compared to the general population (when matched on age and gender). The mean health state valuation difference of 0.17 indicated a meaningful difference compared to a minimal clinically important difference threshold of 0.07. Results were consistent by haemophilia type and severity. CONCLUSION: Our findings indicated the presence of a disability paradox among patients with haemophilia, who reported higher health states than the general population, suggesting the impact of haemophilia may be underestimated if general population value sets are used.


Asunto(s)
Hemofilia A , Calidad de Vida , Estado de Salud , Humanos , Medición de Resultados Informados por el Paciente , Encuestas y Cuestionarios
2.
Haemophilia ; 26(4): 711-717, 2020 Jul.
Artículo en Inglés | MEDLINE | ID: mdl-32268007

RESUMEN

INTRODUCTION: The benefits of physical activity (PA) for people with haemophilia (PWH) may include improvements in joint, bone and muscle health. However, the factor VIII activity level required to avoid a bleeding episode associated with PA is unknown. AIM: To elicit the opinion of clinical experts on the minimum level and ideal factor VIII activity ('level') required to avoid a bleeding episode during participation in different types of PA for PWH. METHODS: Based on the 2017 National Hemophilia Foundation PA descriptions, clinical experts estimated a minimally acceptable and an ideal factor level at which a bleed could be avoided. The uncertainty around estimates was quantified using an approach to construct a probability distribution to represent expert opinion. RESULTS: Minimum and ideal factor level increased with higher risk PA, whether or not joint morbidity was present, as did the experts' uncertainty in their estimates (ie the range between lowest and highest estimates for minimum and ideal levels). Mean minimum levels ranged from 4% to 48% for low to high risk for people without joint morbidity, and from 7% to 47% for those with joint morbidity. For ideal factor levels, corresponding figures were 9%-52% and 12%-64%, respectively. CONCLUSION: To support a patient-centric outcome, expert opinion indicates that the clinical norm of 0.01 IU/mL (1%) trough level is insufficient. It is anticipated that introducing a more targeted approach to meet the needs of patients who are increasingly physically active will benefit patients further in addition to recent treatment advances.


Asunto(s)
Ejercicio Físico/fisiología , Hemartrosis/prevención & control , Hemofilia A/terapia , Hemorragia/prevención & control , Adolescente , Adulto , Anciano , Niño , Preescolar , Conferencias de Consenso como Asunto , Factor VIII/análisis , Hemartrosis/diagnóstico , Hemartrosis/etiología , Hemofilia A/sangre , Hemofilia A/complicaciones , Hemorragia/etiología , Humanos , Lactante , Artropatías/sangre , Artropatías/diagnóstico , Artropatías/patología , Persona de Mediana Edad , Medición de Riesgo , Adulto Joven
4.
J Clin Apher ; 31(5): 434-42, 2016 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-26415895

RESUMEN

Plerixafor is an effective haematopoietic stem cell mobilising agent in candidates for autologous transplantation, including patients with myeloma and lymphoma. Here we compare 98 plerixafor recipients in the PHANTASTIC trial with 151 historic controls mobilised by conventional chemotherapy (each with granulocyte colony-stimulating factor, G-CSF). Seventy (71.4%) plerixafor-mobilised patients achieved the composite primary endpoint of ≥4 × 10(6) CD34+ cells kg(-1) in ≤2 aphereses and no clinically significant neutropenia, compared to 48 (31.8%) historic controls (P < 0.001), and this significant advantage was maintained in scenario analyses testing components of this composite endpoint. A patient-level cost analysis was undertaken for 249 patients, which included the cost of remobilising patients where initial mobilisation had failed. Combined mean treatment cost for plerixafor mobilised patients was £12,679 compared with £11,694 for historical controls. However, plerixafor produces an average saving of £3,828 per lymphoma patient but average cost increase by £5,245 per myeloma patient. The present data demonstrate cost-effectiveness for plerixafor as a first line mobilisation agent, certainly for lymphoma patients, where substantial resource savings and achievement of the primary endpoint are likely. J. Clin. Apheresis 31:434-442, 2016. © 2015 Wiley Periodicals, Inc.


Asunto(s)
Movilización de Célula Madre Hematopoyética/métodos , Compuestos Heterocíclicos/uso terapéutico , Bencilaminas , Análisis Costo-Beneficio , Costos y Análisis de Costo , Ciclamas , Factor Estimulante de Colonias de Granulocitos/economía , Factor Estimulante de Colonias de Granulocitos/uso terapéutico , Movilización de Célula Madre Hematopoyética/economía , Compuestos Heterocíclicos/economía , Estudio Históricamente Controlado , Humanos , Linfoma/economía , Linfoma/terapia , Mieloma Múltiple/economía , Mieloma Múltiple/terapia
5.
Patient ; 17(4): 421-439, 2024 Jul.
Artículo en Inglés | MEDLINE | ID: mdl-38530509

RESUMEN

BACKGROUND: Individuals living with transfusion-dependent ß-thalassemia (TDT) experience reduced health-related quality of life due to fatigue and chronic pain, which cause disruptions to daily life. Currently, limited qualitative data exist that describe these impacts. OBJECTIVE: This study aimed to examine the ways in which symptoms and current treatments of TDT impact health-related quality of life, to holistically describe the humanistic burden of TDT, and to identify the unmet needs of individuals living with TDT. METHODS: Adults (aged ≥ 18 years) with TDT and caregivers of adolescents (aged 12‒17 years) with TDT participated in semi-structured one-on-one virtual interviews and focus group discussions. Interviews were conducted in the USA and UK and lasted approximately 60 minutes. After transcription, the interviews were analyzed thematically using a framework approach. RESULTS: A total of ten interviews/focus group discussions (six interviews and four focus group discussions) were conducted with 14 adults with TDT and two caregivers of adolescents with TDT. A framework analysis revealed five themes describing health-related quality of life (negative impacts on daily activities, social life, family life, work and education, and psychological well-being) and three themes describing the lived experience of TDT (impact of red blood cell transfusions and iron chelation therapy, treatment, and stigma). Physical, psychological, and treatment-related factors contributed to negative impacts on daily activities, social and family life, and work and education. Concerns about reduced lifespan, relationships and family planning, and financial independence were detrimental to participants' mental well-being. Participants reported having high resilience to the many physical and psychological challenges of living with TDT. A lack of TDT-specific knowledge among healthcare professionals, particularly regarding chronic pain associated with the disease, left some participants feeling ignored or undermined. Additionally, many participants experienced stigma and were reluctant to disclose their disease to others. CONCLUSIONS: Individuals living with TDT experience substantial negative impacts on health-related quality of life that disrupt their daily lives, disruptions that are intensified by inadequate healthcare interactions, demanding treatment schedules, and stigma. Our study highlights the unmet needs of individuals living with TDT, especially for alternative treatments that reduce or eliminate the need for red blood cell transfusions and iron chelation therapy.


Asunto(s)
Cuidadores , Grupos Focales , Investigación Cualitativa , Calidad de Vida , Talasemia beta , Humanos , Masculino , Talasemia beta/psicología , Talasemia beta/terapia , Femenino , Adolescente , Estados Unidos , Adulto , Reino Unido , Persona de Mediana Edad , Cuidadores/psicología , Transfusión Sanguínea/psicología , Entrevistas como Asunto , Niño , Adulto Joven , Actividades Cotidianas , Fatiga/psicología , Dolor Crónico/psicología
6.
Value Health Reg Issues ; 34: 31-39, 2023 Mar.
Artículo en Inglés | MEDLINE | ID: mdl-36463835

RESUMEN

OBJECTIVE: This study aimed to measure the cost-effectiveness of prophylaxis with emicizumab in PsHAhri on ITI in Brazil. METHODS: A cost-effectiveness modeling analysis was used to estimate the costs per PsHAhri on ITI and the number of prevented bleedings from undertaking one intervention (prophylaxis with BpA) over another (prophylaxis with emicizumab), based on the Brazilian Ministry of Health perspective. Costs of ITI with recombinant FVIII, prophylaxis with BpA or emicizumab, and treated bleeding episodes with BpA costs were evaluated for PsHAhri who had ITI success or failure. This study was conducted with the perspective of the Brazilian Ministry of Health (payer). RESULTS: During ITI, prophylaxis with BpA cost US $924 666/PsHAhri/ITI, whereas prophylaxis with emicizumab cost US $488 785/PsHAhri/ITI. During ITI, there was an average of 9.32 bleeding episodes/PsHAhri/ITI when BpA were used as prophylaxis and 0.67 bleeding/PsHAhri/ITI when emicizumab was used. By univariate deterministic sensitivity analysis, emicizumab remained dominant whichever variable was modified. CONCLUSION: In this study, prophylaxis with emicizumab during ITI is a dominant option compared with prophylaxis with BpA during ITI.


Asunto(s)
Hemofilia A , Humanos , Niño , Factor VIII/uso terapéutico , Análisis Costo-Beneficio , Análisis de Costo-Efectividad , Hemorragia/prevención & control , Tolerancia Inmunológica
7.
Eur J Health Econ ; 24(1): 27-38, 2023 Feb.
Artículo en Inglés | MEDLINE | ID: mdl-35347553

RESUMEN

BACKGROUND: Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. METHODS: Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. RESULTS: The mean age of participants was 41.50 years (SD 16.01, range 18-81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. CONCLUSIONS: This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies.


Asunto(s)
Talasemia beta , Humanos , Femenino , Masculino , Talasemia beta/terapia , Terapia por Quelación , Transfusión Sanguínea/métodos , Análisis Costo-Beneficio , Análisis de Costo-Efectividad
8.
J Exp Bot ; 63(3): 1155-66, 2012 Feb.
Artículo en Inglés | MEDLINE | ID: mdl-22105847

RESUMEN

Heat stress can cause severe crop yield losses by impairing reproductive development. However, the underlying mechanisms are poorly understood. We examined patterns of carbon allocation and activities of sucrose cleavage enzymes in heat-tolerant (HT) and -sensitive (HS) tomato (Solanum lycopersicum L.) lines subjected to normal (control) and heat stress temperatures. At the control temperature of 25/20 °C (day/night) the HT line exhibited higher cell wall invertase (CWIN) activity in flowers and young fruits and partitioned more sucrose to fruits but less to vegetative tissues as compared to the HS line, independent of leaf photosynthetic capacity. Upon 2-, 4-, or 24-h exposure to day or night temperatures of 5 °C or more above 25/20 °C, cell wall (CWIN) and vacuolar invertases (VIN), but not sucrose synthase (SuSy), activities in young fruit of the HT line were significantly higher than those of the HS line. The HT line had a higher level of transcript of a CWIN gene, Lin7, in 5-day fruit than the HS line under control and heat stress temperatures. Interestingly, heat induced transcription of an invertase inhibitor gene, INVINH1, but reduced its protein abundance. Transcript levels of LePLDa1, encoding phospholipase D, which degrades cell membranes, was less in the HT line than in the HS line after exposure to heat stress. The data indicate that high invertase activity of, and increased sucrose import into, young tomato fruit could contribute to their heat tolerance through increasing sink strength and sugar signalling activities, possibly regulating a programmed cell death pathway.


Asunto(s)
Flores/metabolismo , Frutas/metabolismo , Proteínas de Plantas/metabolismo , Solanum lycopersicum/enzimología , Solanum lycopersicum/metabolismo , Sacarosa/metabolismo , beta-Fructofuranosidasa/metabolismo , Flores/enzimología , Frutas/enzimología , Calor
9.
Plant J ; 63(4): 651-61, 2010 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-20545890

RESUMEN

Transfer cells are specialised transport cells containing invaginated wall ingrowths that generate an amplified plasma membrane surface area with high densities of transporter proteins. They trans-differentiate from differentiated cells at sites at which enhanced rates of nutrient transport occur across apo/symplasmic boundaries. Despite their physiological importance, little is known of the molecular mechanisms regulating construction of their intricate wall ingrowths. We investigated the genetic control of wall ingrowth formation in phloem parenchyma transfer cells of leaf minor veins in Arabidopsis thaliana. Wall ingrowth development in these cells is substantially enhanced upon exposing plants to high-light or cold treatments. A hierarchical bioinformatic analysis of public microarray datasets derived from the leaves of plants subjected to these treatments identified GIGANTEA (GI) as one of 46 genes that are commonly up-regulated twofold or more under both high-light and cold conditions. Histological analysis of the GI mutants gi-2 and gi-3 showed that the amount of phloem parenchyma containing wall ingrowths was reduced 15-fold compared with wild-type. Discrete papillate wall ingrowths were formed in gi-2 plants but failed to develop into branched networks. Wall ingrowth development in gi-2 was not rescued by exposing these plants to high-light or cold conditions. In contrast, over-expression of GI in the gi-2 background restored wall ingrowth deposition to wild-type levels. These results indicate that GI regulates the ongoing development of wall ingrowth networks at a point downstream of inputs from environmental signals.


Asunto(s)
Proteínas de Arabidopsis/fisiología , Arabidopsis/fisiología , Pared Celular/fisiología , Floema/metabolismo , Arabidopsis/genética , Arabidopsis/metabolismo , Proteínas de Arabidopsis/genética , Proteínas de Arabidopsis/metabolismo , Bencenosulfonatos/química , Bencenosulfonatos/metabolismo , Pared Celular/metabolismo , Pared Celular/ultraestructura , Frío , Perfilación de la Expresión Génica , Regulación del Desarrollo de la Expresión Génica/efectos de la radiación , Regulación de la Expresión Génica de las Plantas/efectos de la radiación , Prueba de Complementación Genética , Luz , Microscopía Electrónica de Rastreo , Microscopía Electrónica de Transmisión , Microscopía Fluorescente , Mutación , Análisis de Secuencia por Matrices de Oligonucleótidos , Floema/citología , Floema/ultraestructura , Hojas de la Planta/genética , Hojas de la Planta/metabolismo , Hojas de la Planta/fisiología
10.
Eur J Hum Genet ; 29(4): 699-708, 2021 04.
Artículo en Inglés | MEDLINE | ID: mdl-33328582

RESUMEN

Predictive BRCA testing is offered to asymptomatic individuals to predict future risk where a variant has been identified in a relative. It is uncertain whether all eligible relatives access testing, and whether this is related to health care inequalities. Our aim was to analyse trends and inequalities in uptake of testing, and identify predictors of testing and time-to-receipt of testing. A database from April 2010 to March 2017 was collated. Multivariate analysis explored individual associations with testing. Predictor variables included gender, BRCA test type, cancer history, Index of Multiple Deprivation (IMD) and education status. To evaluate factors associated with time-to-testing, a Cox proportional-hazards (CP) model was used. Of 779 tests undertaken, 336 (43.1%) were identified with a BRCA variant. A total of 537 (68.9%) were female and in 83.4% (387/464) of probands, predictive testing was received by relatives. Analysis identified inequalities since decreased testing was found when the proband was unaffected by cancer (OR 0.14, 95% CI 0.06-0.33). Median time-to-testing was 390 days (range, 0-7090 days) and the CP model also identified inequalities in the hazard ratio (HR) for testing for people aged >40 was higher than for aged <40 (HR 1.41, 95% CI 1.20-1.67) and BRCA2 testing was higher than for BRCA1 testing (HR 1.39, 95% CI 1.18-1.64). Reduced testing was found when probands were unaffected by cancer and time-to-testing was found to vary by age and BRCA1/2 test. Given limited study sample size, further research is recommended to examine inequalities in predictive BRCA testing.


Asunto(s)
Tamización de Portadores Genéticos/estadística & datos numéricos , Conocimientos, Actitudes y Práctica en Salud , Síndrome de Cáncer de Mama y Ovario Hereditario/genética , Adulto , Factores de Edad , Proteína BRCA1/genética , Proteína BRCA2/genética , Escolaridad , Femenino , Asesoramiento Genético/psicología , Asesoramiento Genético/normas , Asesoramiento Genético/estadística & datos numéricos , Síndrome de Cáncer de Mama y Ovario Hereditario/diagnóstico , Síndrome de Cáncer de Mama y Ovario Hereditario/psicología , Humanos , Masculino , Reino Unido
11.
J Med Econ ; 24(1): 363-372, 2021.
Artículo en Inglés | MEDLINE | ID: mdl-33591884

RESUMEN

AIMS: Hemophilia B (HB) is a rare congenital disorder characterized by bleeding-related complications which are managed by prophylactic or post-bleeding event ("on-demand") replacement of clotting factor IX (FIX). The standard of care for severe HB is life-long prophylaxis with standard half-life (SHL) or extended half-life (EHL) products given every 2-3 or 7-14 days, respectively. FIX treatment costs in the US have been investigated, but the lifetime costs of HB treatment have not been well characterized, particularly related to the impact of joint health deterioration and associated health resource utilization. We developed a decision-analytic model to explore outcomes, costs and underlying cost drivers associated with FIX treatment options over the lifetime of an adult with severe or moderately severe HB. MATERIALS AND METHODS: With participation from clinicians, health technology assessment specialists and patient advocates, a Markov model was constructed to estimate bleeding events and costs associated with health states including "bleed into joint", "bleed not into joint", "no bleed" and "death". Sub-models of joint health were based on 0, 1, or ≥2 areas of chronic joint damage. US third-party payer and societal perspectives were considered with a lifetime horizon; sensitivity analyses tested the robustness of primary findings. RESULTS: Total adult lifetime costs per patient with severe and moderately severe HB were $21,086,607 for SHL FIX prophylaxis, $22,987,483 for EHL FIX prophylaxis, and $20,971,826 for on-demand FIX treatment. For FIX prophylaxis, the cost of FIX treatment accounts for >90% of the total HB treatment costs. CONCLUSIONS: This decision analytic model demonstrated significant economic burden associated with the current HB treatment paradigm.


Asunto(s)
Hemofilia A , Hemofilia B , Adulto , Factor IX/uso terapéutico , Semivida , Costos de la Atención en Salud , Hemofilia A/tratamiento farmacológico , Hemofilia B/tratamiento farmacológico , Humanos
12.
J Comp Eff Res ; 10(12): 1019-1052, 2021 08.
Artículo en Inglés | MEDLINE | ID: mdl-34241546

RESUMEN

Aim: Global expenditure on medicines is rising up to 6% per year driven by increasing prevalence of non-communicable diseases (NCDs) and new premium priced medicines for cancer, orphan diseases and other complex areas. This is difficult to sustain without reforms. Methods: Extensive narrative review of published papers and contextualizing the findings to provide future guidance. Results: New models are being introduced to improve the managed entry of new medicines including managed entry agreements, fair pricing approaches and monitoring prescribing against agreed guidance. Multiple measures have also successfully been introduced to improve the prescribing of established medicines. This includes encouraging greater prescribing of generics and biosimilars versus originators and patented medicines in a class to conserve resources without compromising care. In addition, reducing inappropriate antibiotic utilization. Typically, multiple measures are the most effective. Conclusion: Multiple measures will be needed to attain and retain universal healthcare.


Asunto(s)
Biosimilares Farmacéuticos , Medicamentos Genéricos , Gastos en Salud , Humanos , Formulación de Políticas
13.
Expert Rev Pharmacoecon Outcomes Res ; 21(4): 527-540, 2021 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-33535841

RESUMEN

Introduction: There are growing concerns among European health authorities regarding increasing prices for new cancer medicines, prices not necessarily linked to health gain and the implications for the sustainability of their healthcare systems.Areas covered: Narrative discussion principally among payers and their advisers regarding potential approaches to the pricing of new cancer medicines.Expert opinion: A number of potential pricing approaches are discussed including minimum effectiveness levels for new cancer medicines, managed entry agreements, multicriteria decision analyses (MCDAs), differential/tiered pricing, fair pricing models, amortization models as well as de-linkage models. We are likely to see a growth in alternative pricing deliberations in view of ongoing challenges. These include the considerable number of new oncology medicines in development including new gene therapies, new oncology medicines being launched with uncertainty regarding their value, and continued high prices coupled with the extent of confidential discounts for reimbursement. However, balanced against the need for new cancer medicines. This will lead to greater scrutiny over the prices of patent oncology medicines as more standard medicines lose their patent, calls for greater transparency as well as new models including amortization models. We will be monitoring these developments.


Asunto(s)
Antineoplásicos/economía , Atención a la Salud/economía , Costos de los Medicamentos/tendencias , Neoplasias/tratamiento farmacológico , Costos y Análisis de Costo , Desarrollo de Medicamentos , Europa (Continente) , Humanos , Modelos Económicos , Neoplasias/economía , Patentes como Asunto , Mecanismo de Reembolso/economía
14.
BMJ Open ; 10(9): e036222, 2020 09 16.
Artículo en Inglés | MEDLINE | ID: mdl-32938592

RESUMEN

OBJECTIVE: To examine surgical outcomes and trends in the implementation of minimally invasive surgery (MIS) use for endometrial cancer (EC). DESIGN: Retrospective cohort study. SETTING: English National Health Service hospitals 2011-2017/2018. POPULATION: 35 304 patients having a hysterectomy for EC identified from Hospital Episode Statistics. METHODS: Univariate and multivariate analyses compared MIS to open hysterectomy (OH) by assessing the association between demographic, clinical and hospital characteristics by using logistic regression. A propensity score was created, to control for confounding factors including demographics, clinical and hospital characteristics, from a logistic regression which enabled the inverse probability weighting of treatment to be applied in order to compare outcomes of treatment. MAIN OUTCOME MEASURES: The association between route of surgery on perioperative morbidity and mortality. RESULTS: The MIS rate rose from 40.3% in 2011 to 68.7% in 2017/2018, however, there was significant geographical variation (p<0.001). The overall 90-day mortality was significantly higher with OH versus MIS (OR 0.34, 95% CI 0.18 to 0.62, p=0.0002). MIS rates were significantly lower in patients from the lowest socioeconomic group (LSEG) compared with patients from the highest group (HSEG) (55.4% vs 59.9%, p<0.01), and in the black population as compared with white and Asian populations (40.4% vs 58.6% and 56.0%, p<0.0001). When patients from LSEG and black patients were treated in hospitals with high MIS rates, the MIS rate increased close to that of the HSEG and white patients (81.0% and 74.1% vs 83.2% and 82.6%). CONCLUSIONS: Further investigation is needed to understand the barriers to MIS and improve access so that as many patients as possible can benefit from the reduced morbidity/mortality associated with MIS.


Asunto(s)
Neoplasias Endometriales , Medicina Estatal , Neoplasias Endometriales/cirugía , Inglaterra/epidemiología , Femenino , Humanos , Procedimientos Quirúrgicos Mínimamente Invasivos , Estudios Retrospectivos
15.
Pharmacoecon Open ; 4(2): 331-342, 2020 Jun.
Artículo en Inglés | MEDLINE | ID: mdl-31368087

RESUMEN

BACKGROUND: Chronic obstructive pulmonary disease (COPD) is a major cause of morbidity and mortality, especially in low- and middle-income countries (LMICs) such as India. Medicine costs are a key issue in LMICs, with typically high patient co-payments. In addition, pharmacists are underutilised in LMICs, including India. However, pharmacist-led educational interventions may improve the care of patients with COPD, as well as reduce medicine costs. Consequently, the objective of this study was to assess the effectiveness of a pharmacist-led intervention in reducing medicine costs. METHODOLOGY: We assessed the impact of a pharmacist intervention on direct medicine costs in COPD patients (medicine costs and pharmacist time) in a randomised controlled study involving an intervention and control group, conducted at a tertiary care teaching hospital in India. RESULTS: The 6-monthly cost of medicines at baseline increased with disease severity, from a maximum of US$29.46 for those with mild COPD to US$63.28 for those with very severe COPD. Substantial savings in medical costs were achieved with the pharmacist-led programme, to a maximum of US$20.49 over 6 months for very severe patients. This equates to a reduction of 30.6% in medicine costs (p < 0.001), reduced to 26.1% when pharmacists' time (US$3.00/patient) was included. CONCLUSION: There could be a key role for pharmacists as educators for COPD patients in LMICs, to improve care and reduce costs, including patient co-payments.

16.
Appl Health Econ Health Policy ; 18(1): 5-16, 2020 02.
Artículo en Inglés | MEDLINE | ID: mdl-31696433

RESUMEN

The potential benefits of early patient access to new medicines in areas of high unmet medical need are recognised, but uncertainties concerning effectiveness, safety and added value when new medicines are authorised, and subsequently funded based on initial preliminary data only, have important implications. In 2016 olaratumab received accelerated conditional approval from both the European Medicines Agency and the US Food and Drug Administration for the treatment of soft-tissue sarcoma, based on the claims of a substantial reduction in the risk of death with an 11.8-month improvement in median overall survival in a phase II trial in combination with doxorubicin vs. doxorubicin alone. The failure to confirm these benefits in the post-authorisation pivotal trial has highlighted key concerns regarding early access and conditional approvals for new medicines. Concerns include potentially considerable clinical and economic costs, so that patients may have received suboptimal treatment and any money spent has foregone the opportunity to improve access to effective treatments. As a result, it seems reasonable to reconsider current marketing authorisation models and approaches. Potential pathways forward include closer collaboration between regulators, pharmaceutical companies and payers to enhance the generation of rapid and comparative confirmatory trials in a safe and fair manner, with minimal patient exposure as required to achieve robust evidence. Additionally, it may be time to review early access systems, and to explore new avenues regarding who should pay or part pay for new treatments whilst information is being collected as part of any obligations for conditional marketing authorisation. Greater co-operation between countries regarding the collection of data in routine clinical care, and further research on post-marketing data analysis and interpretation, may also contribute to improved appraisal and continued access to new innovative cancer treatments.


Asunto(s)
Antibióticos Antineoplásicos/uso terapéutico , Anticuerpos Monoclonales/uso terapéutico , Antineoplásicos/normas , Antineoplásicos/uso terapéutico , Doxorrubicina/uso terapéutico , Aprobación de Drogas , Neoplasias/tratamiento farmacológico , Adulto , Anciano , Anciano de 80 o más Años , Europa (Continente) , Femenino , Humanos , Masculino , Persona de Mediana Edad
17.
Front Pharmacol ; 11: 588106, 2020.
Artículo en Inglés | MEDLINE | ID: mdl-33628173

RESUMEN

Background: Countries across Africa and Asia have introduced a variety of measures to prevent and treat COVID-19 with medicines and personal protective equipment (PPE). However, there has been considerable controversy surrounding some treatments including hydroxychloroquine where the initial hype and misinformation led to shortages, price rises and suicides. Price rises and shortages were also seen for PPE. Such activities can have catastrophic consequences especially in countries with high co-payment levels. Consequently, there is a need to investigate this further. Objective: Assess changes in utilisation, prices, and shortages of pertinent medicines and PPE among African and Asian countries since the start of pandemic. Our approach: Data gathering among community pharmacists to assess changes in patterns from the beginning of March until principally the end of May 2020. In addition, suggestions on ways to reduce misinformation. Results: One hundred and thirty one pharmacists took part building on the earlier studies across Asia. There were increases in the utilisation of principally antimalarials (hydroxychloroquine) and antibiotics (azithromycin) especially in Nigeria and Ghana. There were limited changes in Namibia and Vietnam reflecting current initiatives to reduce inappropriate prescribing and dispensing of antimicrobials. Encouragingly, there was increased use of vitamins/immune boosters and PPE across the countries where documented. In addition, generally limited change in the utilisation of herbal medicines. However, shortages have resulted in appreciable price increases in some countries although moderated in others through government initiatives. Suggestions in Namibia going forward included better planning and educating patients. Conclusion: Encouraging to see increases in the utilisation of vitamins/immune boosters and PPE. However, concerns with increased utilisation of antimicrobials needs addressing alongside misinformation, unintended consequences from the pandemic and any appreciable price rises. Community pharmacists and patient organisations can play key roles in providing evidence-based advice, helping moderate prices through improved stock management, and helping address unintended consequences of the pandemic.

18.
Curr Med Res Opin ; 36(2): 301-327, 2020 02.
Artículo en Inglés | MEDLINE | ID: mdl-31794332

RESUMEN

Introduction: Antibiotics are indispensable to maintaining human health; however, their overuse has resulted in resistant organisms, increasing morbidity, mortality and costs. Increasing antimicrobial resistance (AMR) is a major public health threat, resulting in multiple campaigns across countries to improve appropriate antimicrobial use. This includes addressing the overuse of antimicrobials for self-limiting infections, such as upper respiratory tract infections (URTIs), particularly in lower- and middle-income countries (LMICs) where there is the greatest inappropriate use and where antibiotic utilization has increased the most in recent years. Consequently, there is a need to document current practices and successful initiatives in LMICs to improve future antimicrobial use.Methodology: Documentation of current epidemiology and management of URTIs, particularly in LMICs, as well as campaigns to improve future antimicrobial use and their influence where known.Results: Much concern remains regarding the prescribing and dispensing of antibiotics for URTIs among LMICs. This includes considerable self-purchasing, up to 100% of pharmacies in some LMICs. However, multiple activities are now ongoing to improve future use. These incorporate educational initiatives among all key stakeholder groups, as well as legislation and other activities to reduce self-purchasing as part of National Action Plans (NAPs). Further activities are still needed however. These include increased physician and pharmacist education, starting in medical and pharmacy schools; greater monitoring of prescribing and dispensing practices, including the development of pertinent quality indicators; and targeted patient information and health education campaigns. It is recognized that such activities are more challenging in LMICs given more limited resources and a lack of healthcare professionals.Conclusion: Initiatives will grow across LMICs to reduce inappropriate prescribing and dispensing of antimicrobials for URTIs as part of NAPs and other activities, and these will be monitored.


Asunto(s)
Antibacterianos/uso terapéutico , Prescripción Inadecuada/prevención & control , Infecciones del Sistema Respiratorio/tratamiento farmacológico , Países en Desarrollo , Educación en Salud , Humanos , Renta
19.
Pharmacoecon Open ; 3(4): 631-641, 2019 Dec.
Artículo en Inglés | MEDLINE | ID: mdl-30868410

RESUMEN

BACKGROUND: Haemodialysis (HD) and peritoneal dialysis (PD) are commonly used treatments for the management of patients with end-stage renal disease (ESRD). The costs of managing these patients have grown in recent years with increasing rates of non-communicable diseases, which will adversely impact on national health budgets unless addressed. Currently, there is limited knowledge of the costs of ESRD within the public healthcare system in South Africa. OBJECTIVE: The aim of this study was to examine the direct costs of HD and PD in South Africa from a healthcare provider's perspective. METHODS: A prospective, observational study was undertaken at a leading public hospital in South Africa. A micro-costing approach was applied to estimate healthcare costs using 46 adult patients with ESRD who had been receiving HD and PD for at least 3 months. RESULTS: The highest proportion of patients (35%) were aged 40-50 years. Patients aged 29-39 years were mostly on HD (28% vs. 21% on PD) while those aged 51-59 years mostly used PD (29% vs. 16% on HD). The average age of patients on HD and PD were 41 and 42 years, respectively. Fixed costs were the principal cost driver for HD ($16,231.45) while variable costs were the principal cost driver for PD (US$20,488.79). The annual cost of HD per patient (US$31,993.12) was higher than PD (US$25,282.00 per patient), even though the difference was not statistically significant (p = 0.816). CONCLUSION: HD costs more than PD from the provider's perspective. These cost estimates may be useful for carrying out future cost-effectiveness and cost-utility analyses in South Africa.

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