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BACKGROUND: Patients with alpha-1 antitrypsin deficiency (AATD), commonly categorized as a rare disease, have been affected by the changes in healthcare management brought about by COVID-19. This study's aim was to identify the changes that have taken place in AATD patient care as a result of the COVID-19 pandemic in Spain and to propose experts' recommendations aimed at ensuring humanized and quality care for people with AATD in the post-pandemic situation. METHODS: A qualitative descriptive case study with a holistic single-case design was conducted, using focus groups with experts in AATD clinical management, including 15 health professionals with ties to the Spanish health system (12 pneumologists and 2 hospital pharmacists from 11 different hospitals in Spain) and 1 patient representative. RESULTS: COVID-19 has had a major impact on numerous aspects of AATD clinical patient management in Spain, including diagnostic, treatment, and follow-up phases. The experts concluded that there is a need to strengthen coordination between Primary Care and Hospital Care and improve the coordination processes across all the organizations and actors involved in the healthcare system. Regarding telemedicine and telecare, experts have concluded that it is necessary to promote this methodology and to develop protocols and training programs. Experts have recommended developing personalized and precision medicine, and patient participation in decision-making, promoting self-care and patient autonomy to optimize their healthcare and improve their quality of life. The possibility of monitoring and treating AATD patients from home has also been proposed by experts. Another result of the study was the recommendation of the need to ensure that plasma donations are made on a regular basis by a sufficient number of healthy individuals. CONCLUSION: The study advances knowledge by highlighting the challenges faced by health professionals and changes in AATD patient management in the context of the COVID-19 pandemic. It also proposes experts' recommendations aimed at ensuring humanized and quality care for people with AATD in the post-pandemic situation. This work could serve as a reference study for physicians on their daily clinical practice with AATD patients and may also provide guidance on the changes to be put in place for the post-pandemic situation.
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COVID-19 , Enfermedad Pulmonar Obstructiva Crónica , Deficiencia de alfa 1-Antitripsina , Humanos , Pandemias , Calidad de Vida , COVID-19/epidemiología , Deficiencia de alfa 1-Antitripsina/diagnóstico , Deficiencia de alfa 1-Antitripsina/tratamiento farmacológico , Atención a la Salud , Enfermedad Pulmonar Obstructiva Crónica/terapiaRESUMEN
OBJECTIVE: This article describes a study protocol for evaluating adherence to oral chemotherapy (OCT) in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) in Spain. METHODS: This multicenter, observational, prospective study will be conducted by 6 hospital pharmacists from 6 Spanish hospitals. The study will include men and women aged 18â¯years or older with a diagnosis of locally advanced or metastatic NSCLC who are being treated or have been prescribed OCT. Once included, the patient will be active and prospectively followed up for 3â¯months, including 4 study visits to record information on sociodemographic variables, antineoplastic treatment and adherence, pharmaceutical care, clinical variables, and patient-reported outcomes (PRO) (the 3-level version of EQ-5D, the EORTC Core Quality of Life Questionnaire, the Brief Illness Perception Questionnaire, the Treatment Satisfaction with Medicines Questionnaire, and the PRO version of Common Terminology Criteria for Adverse Events). Twelve months after patient inclusion, we will record information on the disease progression status and dispensed prescriptions. The primary outcome is the percentage of treatment adherence that will be calculated based on the pill count as follows: the difference between the number of pills dispensed minus the number of unused pills will be divided by the number of days of treatment multiplied by the number of pills/day prescribed by the oncologist; this quotient will be multiplied by 100 to obtain the percentage of adherence. Based on the that pill count reconciliation, those with a percentage adherence >80% will be primarily categorized as adherent. Secondarily, treatment adherence will be also calculated based on the proportion of days covered and the 4-items Morisky Green Levine Medication Adherence Scale. To analyze the impact of patients' and treatment characteristics on adherence, bivariate analyses will be performed using different adherence cut-off points. To evaluate the impact of adherence on treatment efficacy as evaluated by progression-free survival, we will be using the Kaplan-Meier method and compare it with the log-rank test and univariate Cox regression analysis. CONCLUSIONS: We expect that our study will provide initial information on key aspects of adherence to OCT (i.e., measurement, facilitators, and barriers) and its relationship with patients' and clinically relevant outcomes in the setting of NSCLC, and that this information will help in designing pharmaceutical interventions to improve adherence.
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Artificial intelligence (AI) is a broad concept that includes the study of the ability of computers to perform tasks that would normally require the intervention of human intelligence. By exploiting large volumes of healthcare data, artificial intelligence algorithms can identify patterns and predict outcomes, which can help healthcare organizations and their professionals make better decisions and achieve better results. Machine learning, deep learning, neural networks or natural language processing are among the most important methods, allowing systems to learn and improve from data without the need for explicit programming. AI has been introduced in biomedicine, accelerating processes, improving safety and efficiency, and improving patient care. By using AI algorithms and Machine Learning, hospital pharmacists can analyze a large volume of patient data, including medical records, laboratory results, and medication profiles, aiding them in identifying potential drug-drug interactions, assessing the safety and efficacy of medicines, and making informed recommendations. AI integration will improve the quality of pharmaceutical care, optimize processes, promote research, deploy open innovation, and facilitate education. Hospital pharmacists who master AI will play a crucial role in this transformation.
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Inteligencia Artificial , Servicio de Farmacia en Hospital , Servicio de Farmacia en Hospital/organización & administración , Humanos , Farmacéuticos , Algoritmos , Aprendizaje Automático , Redes Neurales de la ComputaciónRESUMEN
Artificial intelligence is a broad concept that includes the study of the ability of computers to perform tasks that would normally require the intervention of human intelligence. By exploiting large volumes of healthcare data, Artificial intelligence algorithms can identify patterns and predict outcomes, which can help healthcare organizations and their professionals make better decisions and achieve better results. Machine learning, deep learning, neural networks, or natural language processing are among the most important methods, allowing systems to learn and improve from data without the need for explicit programming. Artificial intelligence has been introduced in biomedicine, accelerating processes, improving accuracy and efficiency, and improving patient care. By using Artificial intelligence algorithms and machine learning, hospital pharmacists can analyze a large volume of patient data, including medical records, laboratory results, and medication profiles, aiding them in identifying potential drug-drug interactions, assessing the safety and efficacy of medicines, and making informed recommendations. Artificial intelligence integration will improve the quality of pharmaceutical care, optimize processes, promote research, deploy open innovation, and facilitate education. Hospital pharmacists who master Artificial intelligence will play a crucial role in this transformation.
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Inteligencia Artificial , Servicio de Farmacia en Hospital , Servicio de Farmacia en Hospital/organización & administración , Humanos , Farmacéuticos , Algoritmos , Aprendizaje AutomáticoRESUMEN
OBJECTIVE: To identify and promote hospital pharmacy initiatives to improve the management of patients with hereditary angioedema (HAE) within the Spanish healthcare system. METHOD: A panel of experts comprising hospital pharmacists, an allergist and a nurse/member of the Spanish Hereditary Angioedema Association (Asociación Española de Angioedema Familiar) highlighted initiatives to improve care for patients with HAE after identifying, evaluating and prioritising them. Prioritisation was assessed based on the impact on patient care and the feasibility of their implementation on a scale of 1-5. RESULTS: Seven key areas of activity for the role of hospital pharmacists in the management of patients with HAE were identified: evaluation and selection of medicines; hospital pharmacy dispensation and telepharmacy; pharmacotherapy follow-up and telemedicine; coordination with other healthcare teams involved in the care of patients with HAE; patient health education and training; research on HAE; and continuous education and training of hospital pharmacy service personnel. Ten initiatives with a mean impact score of 5 and a mean feasibility score of ≥4.1 were considered as high-priority initiatives. Half of the initiatives belong to the area concerning patient education and training (50%), followed by care coordination initiatives (30%) and continuous education and training (20%). CONCLUSIONS: Ten high-priority initiatives for the management of patients with HAE were identified by a panel of experts. The implementation of such initiatives by the hospital pharmacy service should enhance the management of patients with HAE in the Spanish healthcare system.
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OBJECTIVE: To prioritize the initiatives to be developed for the development of the Strategic Map of Outpatient Care (MAPEX) project to improve the quality of care and Pharmaceutical Care for patients seen in Hospital Pharmacy outpatient clinics in the period 2024-2027 in Spain. METHOD: The study was carried out in 4 phases between January and December 2023. For phase 1, a literature review of the evolution of the project was carried out by the coordinating committee with the aim of establishing a basis on which to define a new proposal for initiatives. In addition, an analysis was made of the health trends that will have an impact in the coming years. In phase 2, a working group of 19 specialists from all the autonomous communities was created, who were called regional ambassadors. They all made a preliminary proposal of initiatives and established revisions for their adjustment and final version both online and in telematic meetings. In phase 3, a consensus was established based on the Delphi-Rand/UCLA methodology with two rounds of online voting to select the initiatives classified as: priority and key or breakthrough. Between the first and second round of voting, a face-to-face "Consensus Conference" was held, where the results of the first round were presented. In phase 4, a public presentation was made in scientific forums and through the web. RESULTS: Ten trends in the health sector were identified. A list of 34 initiatives grouped into five lines of work was established. A total of 103 panelists participated in the first round and 76 in the second. Finally, five initiatives were established as priority and 29 as key. Among those prioritized were external visibility, adaptations to the CMO methodology, strengthening certification and improving training. CONCLUSIONS: The initiatives agreed upon as priorities were aimed at improving professional visibility, broadening the methodology of care work, expanding the quality of care, enhancing the training of professionals and the voice of patients.
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INTRODUCTION: Pre-exposure prophylaxis (PrEP) against the human immunodeficiency virus (HIV) is an effective and safe preventive measure. However, it has not reached all target users who could benefit from it. The study aimed to understand the sociodemographic, clinical and behavioral baseline characteristics of PrEP users. As a secondary objective, the use of concomitant medication and drug consumption were described. METHODOLOGY: Observational, retrospective and descriptive study of the sociodemographic, clinical and behavioral characteristics of the users who were included in the PrEP program of the Community of Madrid during the first two years of experience. RESULTS: Two thousand two hundred fifty-six PrEP users were included, 99.0% men, with a mean age of 36.9 years (SD 8.68). 33.1% presented a sexually transmitted infection (STI) on the first visit, highlighting chlamydiasis and rectal gonococci. 70.4% reported using drugs associated with sex, and 42.4% participated in chemsex sessions in the last 3 months. A high percentage of users with concomitant medication was observed (37.6%), highlighting drugs related to mental health and alopecia. CONCLUSIONS: A multidisciplinary approach is required to cover all the needs of PrEP users, including mental health evaluation measures and addiction treatment with the clinical approach.
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OBJECTIVE: We simulated the impact of implementing different health interventions to improve the HIV continuum of care for people diagnosed, on treatment, and virologically suppressed in Spain for the 2020-2030 period. METHODS: The model was carried out in four phases involving a multidisciplinary expert panel: (1) literature review; (2) selection/definition of the interventions and their effectiveness; (3) consensus meeting; and (4) development of an analytical decision model to project the impact of implementing/strengthening these interventions to improve the HIV continuum of care, corresponding to 2017-2019 (87% diagnosed, 97% on treatment, 90% with viral suppression), through the creation of different scenarios for 2020-2030. A total of 19 interventions were selected based on expanding the offer of HIV rapid tests and implementing training/peer programmes, electronic alerts, multidisciplinary care, and mHealth, among others. The effectiveness of the interventions was defined by the percentage increases in diagnosis, treatment, and viral suppression after their implementation, targeting the entire population and specific groups at high-risk (men who have sex with men, migrants, female sex workers, transgender people, and people who inject drugs). RESULTS: Implementing eight interventions for diagnosis, three for treatment, and eight for viral suppression for the target populations during 2020-2030 would increase the continuum of care to approximately 100% diagnosed (remaining residual undetectable cases), 98% treated, and 96% virologically suppressed. CONCLUSIONS: Planification, prioritization, and implementation of selected interventions based on the current HIV continuum of care could allow achievement of the 95-95-95 UNAIDS goals in Spain by 2030.
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Infecciones por VIH , Trabajadores Sexuales , Minorías Sexuales y de Género , Masculino , Humanos , Femenino , Infecciones por VIH/tratamiento farmacológico , Homosexualidad Masculina , Objetivos , Atención a la SaludRESUMEN
Introduction: Autophagy is a very active process that plays an important role in cell and organ differentiation and remodelling, being a crucial system to guarantee health. This physiological process is activated in starvation and inhibited in the presence of nutrients. This short review comments on the three types of autophagy: macroautophagy, microautophagy, and chaperone-mediated autophagy, as well as different aspects that control autophagy and its relationship with health and degenerative diseases. As autophagy is highly dependent on functional autophagy (ATG) proteins integrating the phagophore, the role of some key ATG genes and epigenes are briefly commented on. The manuscript deepens discussing some central aspects of type-2 diabetes mellitus and their relationship with the cell cleaning process and mitochondria homeostasis maintenance, as well as the mechanisms through which antidiabetic drugs affect autophagy. Well-designed studies are needed to elucidate whether autophagy plays a casual or causal role in T2DM.
Introducción: La autofagia es un proceso muy activo que juega un papel importante en la diferenciación y remodelación de células y órganos, siendo un sistema crucial para garantizar la salud. Este proceso fisiológico se activa en la inanición y se inhibe en presencia de nutrientes. En esta breve revisión se definen los tres tipos de autofagia: macroautofagia, microautofagia y autofagia mediada por chaperonas, y los diferentes aspectos que controlan la autofagia y su relación con la salud y las enfermedades degenerativas. Como la autofagia depende en gran medida de las proteínas de autofagia funcional (ATG) que integran el fagóforo, se comenta brevemente el papel clave de algunos genes y epigenes de las ATG. El manuscrito profundiza discutiendo algunos aspectos centrales de la diabetes mellitus de tipo 2 (DMT2) y su relación con el proceso de limpieza celular y el mantenimiento de la homeostasis mitocondrial, así como los mecanismos a través de cuales los fármacos antidiabéticos afectan a la autofagia. Se necesitan estudios bien diseñados para dilucidar si la autofagia juega un papel de casualidad o causalidad en el desarrollo de la DMT2.
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Autofagia , Diabetes Mellitus Tipo 2 , Humanos , Autofagia/fisiología , Homeostasis , MitocondriasRESUMEN
OBJECTIVE: To determine the value contribution of cabotegravir + rilpivirine, the first injectable every two months long-acting antiretroviral regimen, using multi-criteria decision analysis. METHOD: The study was developed in two phases. After a small pilot, a field ork study with a larger number of multidisciplinary experts was carried out. Seven single-tablet regimens, currently recommended by the GeSIDA guidelines, were selected as comparators. EVIDEM methodology was followed, with a framework composed by 12 quantitative and 5 contextual criteria. Mean and standard deviations were calculated for quantitative criteria (1 to 5 scale; comparative criteria -5 to +5), whereas qualitative criteria were analyzed as percentages of experts that considered a positive, neutral or negative impact for the National Health System. RESULTS: 35 experts participated in the study. Human immunodeficiency virus- 1 infection was considered severe (mean ± standard deviation: 3.0 ± 1.0), with moderate size of affected population (2.7 ± 1.2) and unmet needs (2.8 ± 1.0). Minimal differences were found in comparative efficacy/effectiveness (0.1 ± 0.5), safety/tolerability (-0.5 ± 0.7), and cost criteria: cost of the intervention (0.5 ± 2.0), other medical costs (0.2 ± 1.8) and non- medical/indirect costs (0.5 ± 1.6). Experts perceived an improvement with cabotegravir + rilpivirine long-acting, compared to current daily oral single-tablet regimens, in patient-reported outcomes (2.7 ± 1.4). Therapeutic benefit of the long-acting regimen was considered moderate-to-high (3.5 ± 1.2). Experts considered the evidence provided by cabotegravir + rilpivirine long- actingrobust (4.3 ± 0.8), with elevated consensus on its future recommendation in guidelines (3.2 ± 1.0). In contextual criteria, most experts considered positive the impact on population priorities and access (91%), common goal and specific interests (63%) and political, historical, and cultural context criteria (60%). Impact was neutral in system capacity and appropriate use (40%), and opportunity costs and affordability criteria (51%). Result of the weighted global value contribution of cabotegravir + rilpivirine long-acting was 0.34 (-1 to +1 scale), with Patient Reported Outcomes comparative criterion bringing the highest added value. CONCLUSIONS: Cabotegravir + rilpivirine long-acting provides added value contribution to human immunodeficiency virus-1 management in Spain compared to daily oral single-tablet regimens. Patient Reported Outcomes and therapeutic benefit of cabotegravir + rilpivirine long-acting were highly valued by experts, as the expected benefit in adherence and stigma-related issues would improve overall quality of life for people living with human immunodeficiency virus-1.
OBJETIVO: Determinar la contribución de valor de cabotegravir + rilpivirina, el primer tratamiento antirretroviral inyectable de acción prolongada, utilizando metodología de análisis de decisión multicriterio.Método: El estudio se desarrolló en dos fases: una prueba piloto y una fase de extensión, con un grupo multidisciplinar más grande. Se seleccionaron siete regímenes de comprimido único orales diarios recomendados en las guías GeSIDA como comparadores. Se utilizó el marco EVIDEM, compuesto por 12 criterios cuantitativos y 5 contextuales. Los criterios cuantitativos se analizaron calculando la media y desviación estándar, y los cualitativos se analizaron mediante el porcentaje de expertos que consideraron el impacto positivo, neutro o negativo para el Sistema Nacional de Salud. RESULTADOS: Un total de 35 expertos participaron en el estudio. La infección por virus de la inmunodeficiencia humana 1 se consideró grave (media ± desviación estándar: 3,0 ± 1,0), con un tamaño de población afectada (2,7 ± 1,2) y unas necesidades no cubiertas (2,8 ± 1,0) moderadas. Las diferencias fueron mínimas en los criterios comparativos de eficacia/efectividad (0,1 ± 0,5), seguridad/tolerabilidad (0,5 ± 0,7) y coste: coste del tratamiento (0,5 ± 2,0), otros costes médicos (0,2 ± 1,8) y costes no-médicos/indirectos (0,5 ± 1,6). Los expertos observaron una emtrimejora con cabotegravir + rilpivirina de acción prolongada en los resultados reportados por los pacientes (2,7 ± 1,4). El beneficio terapéutico (3,5 ± 1,2) se consideró moderado-alto. La evidencia de cabotegravir + rilpivirina de acción prolongada fue considerada robusta (4,3 ± 0,8), con elevado consenso sobre su futura recomendación en las guías (3,2 ± 1,0). En los criterios contextuales, el impacto fue positivo en los criterios de prioridades de acceso (91%), objetivo común (63%) y contexto político (60%). El impacto fue neutro en la capacidad del sistema (40%) y los costes de oportunidad (51%). El resultado promedio de la contribución del valor global de cabotegravir + rilpivirina de acción prolongada fue de 0,34 (escala de 1 a +1), siendo el criterio de resultados reportados por el paciente el que proporcionó la mayor contribución de valor (0,04). CONCLUSIONES: Cabotegravir + rilpivirina de acción prolongada aporta un valor añadido en el manejo del virus de la inmunodeficiencia humana 1 en España en comparación con los regímenes de comprimido único utilizados actualmente. Los expertos valoraron positivamente los resultados reportados por los pacientes y el beneficio terapéutico de cabotegravir + rilpivirina de acción prolongada, considerando que el beneficio esperado en la adherencia y los problemas relacionados con el estigma produciría una mejora en la calidad de vida de las personas con virus de la inmunodeficiencia humana 1.
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Fármacos Anti-VIH , Infecciones por VIH , VIH-1 , Fármacos Anti-VIH/uso terapéutico , Antirretrovirales/uso terapéutico , Dicetopiperazinas , Infecciones por VIH/tratamiento farmacológico , Humanos , Piridonas/uso terapéutico , Calidad de Vida , Rilpivirina/uso terapéuticoRESUMEN
OBJECTIVE: The consolidation of Telepharmacy during the COVID-19 pandemic has raised the need for managing large volumes of real-time activity data through data analysis. The aim of this project was to design a dynamic, user- friendly, customizable scorecard in a hospital pharmacy service for the visualization and analysis of Telepharmacy activity indicators through the use of advanced business intelligence technology. METHOD: The software tool was developed by a multidisciplinary team between April and May 2021, driven from the hospital pharmacy service. Once the Telepharmacy indicators of interest were established, datasets were extracted from raw databases (administrative databases, Telepharmacy database, outpatient dispensing software, drug catalogues) through data analysis. The different data sources were integrated in a scorecard using PowerBI®. The criteria for processing missing and duplicated data were defined, and data pre-processing, normalization and transformation were performed. Once the pilot scorecard was validated by different profiles of users, the structure was designed for the panels to automatically update as databases were updated. RESULTS: Design and implementation of a scorecard of Telepharmacy activity: general descriptive panel (demographic profile of patients, count and delivery conditions, program and medical service); geolocation of destination; pharmacological profile; relative analysis of patients involved in the Telepharmacy program with respect to the total of outpatients. In the last updating as of January 2022, data from 16,000 dispensations to more than 4,000 patients had been collected. This means that 21.93% of outpatients had benefited at some time point from the Telepharmacy service. Filters enable the visualization of timeline progress and patient characterization, and measure Telepharmacy activity by program. CONCLUSIONS: The processing of large Telemedicine datasets from various sources through Business Intelligence in a hospital pharmacy service makes it possible to synthesize information, generate customized reports, and visualize information in a dynamic and attractive format. The application of this new technology will help us improve strategic clinical and management decision making.
OBJETIVO: La consolidación de la Telefarmacia en el contexto de la pandemia por la COVID-19 exige manejar a tiempo real un gran volumen de datos de actividad mediante análisis de datos. El objetivo de este trabajo fue diseñar un cuadro de mando ágil, personalizable y dinámico para la visualización y análisis de indicadores de actividad en Telefarmacia en un servicio de farmacia de hospital, mediante el empleo de herramientas avanzadas de inteligencia empresarial (business intelligence).Método: Un equipo de trabajo multidisciplinar desarrolló una herramienta de software entre abril y mayo de 2021 impulsado desde el servicio de farmacia de hospital. Una vez consensuados los indicadores de interés en Telefarmacia, se extrajeron los datos a partir de bases de datos brutas (base de datos de Telefarmacia, programa de dispensación de pacientes externos, bases de datos administrativas, catálogos de fármacos) mediante análisis de datos. La integración de las diferentes fuentes de datos en el cuadro de mando se realizó mediante PowerBI®. Se definió el manejo de los datos perdidos y duplicados y se aplicó preprocesamiento, normalización y transformación de los datos. Una vez validado el piloto por diferentes tipos de usuarios, se diseñó la estructura para actualización automática de los paneles con las sucesivas actualizaciones de las fuentes de datos. RESULTADOS: Diseño e implementación de un cuadro de mando de la actividad en Telefarmacia: panel descriptivo general (perfil demográfico de pacientes, recuento y condiciones de envíos, programa y servicio médico); geolocalización de destino; perfil farmacológico; análisis relativo de los pacientes beneficiarios de Telefarmacia respecto del total de pacientes externos. En el último corte, a enero de 2022, se habían incluido datos de 16.000 dispensaciones con entrega informada a más de 4.000 pacientes, lo que supone que el 21,93% de los pacientes externos han estado en algún momento en el programa de Telefarmacia. La aplicación de filtros permite visualizar la evolución temporal, caracterizar grupos de pacientes y dimensionar la actividad por programas. CONCLUSIONES: El procesamiento de paquetes de datos de Telemedicina, de gran volumen, difícil manejo y procedentes de diversas fuentes relativas a Telefarmacia mediante inteligencia empresarial, en un servicio de farmacia de hospital, permite sintetizar la información y proporcionar informes personalizados y visualizaciones dinámicas y atractivas. La aplicación de estas nuevas tecnologías puede ayudarnos a mejorar la toma de decisiones estratégicas, tanto clínicas como de gestión.
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COVID-19 , Servicio de Farmacia en Hospital , Telemedicina , Humanos , Pandemias , Análisis de Datos , InteligenciaRESUMEN
Objective: To compare patient experience in a real-life population of people living with HIV (PLWH) who received pharmaceutical care (PC) based on the Capacity-Motivation-Opportunity (CMO) model versus the traditional model. Methods: Prospective cohort study in PLWH receiving either CMO-based PC or traditional PC in Spain between October 2019 and June 2021 (24 weeks), performed by the pharmacy department of 14 Spanish hospitals. Participants were adult patients with a clinical diagnosis of HIV treated with antiretrovirals who had been monitored in the participating hospital pharmacies for >1 year. Patient experience (IEXPAC questionnaire), clinical outcomes (cholesterol, triglycerides, HDL, glycated haemoglobin, and blood pressure), adherence to treatment, virologic control and patient satisfaction were determined. Results: Patient experience in the CMO group at week 24 was significantly better (7.6 vs 6.9) than in the traditional group, with a higher mean improvement. Adherence was better in the CMO group, particularly with regard to concomitant medications (53.2% to 91.7%, p<0.001); no changes were observed in the traditional group. Patient satisfaction improved in the CMO group vs the traditional group (48 vs 44, p<0.001). Conclusion: To our knowledge, this is the first study to compare CMO vs traditional methodology. The CMO model showed an overall improvement in real-life patient experience, satisfaction, and adherence to treatment compared to the traditional methodology.
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OBJECTIVE: Matching-Adjusted Indirect Comparison is a methodology that has been developed to assess new treatments vs alternatives when a direct comparison is not available through a randomized controlled trial. These comparisons are of particular interest in the areas of oncology and hematology where uncertainty in decision-making on the inclusion of new drugs is frequently accentuated by both the severity of the disease and the high cost of treatment. The objective of this study was to describe how Matching-Adjusted Indirect Comparison methodology has been used to date in the assessment of hematological cancer drugs by international agencies. METHOD: Between January 2015 and October 2019, an exhaustive search was conducted of the websites of European National Agencies that provided public information on the assessment process. The assessments provided by these agencies were reviewed to obtain a list of hematological cancer drugs for which the presentation of a Matched-Adjusted Indirect Comparison was recorded. For this list of drugs, the role of the comparison in the assessment process was analyzed for each selected agency. RESULTS: Thirteen hematological and oncological treatments were found in which the pharmaceutical marketing authorization holder had presented Matching-Adjusted Indirect Comparisons: most of this information referred to the first half of 2018. Acceptance of this methodology diverges among agencies, ranging from 50% in the case of the British National Institute for Health and Clinical Excellence, to 40% in the case of French National Authority for Health, to not having been taken into account in any of the 3 cases assessed by the German Institute for Quality and Efficiency in Health Care. The main cause of non-acceptance was matching-related problems. CONCLUSIONS: Matching- Adjusted Indirect Comparison methodology is a tool that is being utilized in the decision-making process for assessing new hematological cancer treatments.
Objetivo: La comparación indirecta ajustada con emparejamiento es una metodología desarrollada para la evaluación de nuevos tratamientos frente a sus alternativas cuando no se dispone de comparación directa mediante un ensayo clínico aleatorizado y controlado. Estas comparaciones son de especial interés en el área de la hematooncología, en la que la incertidumbre en la toma de decisiones sobre la inclusión de nuevos fármacos se ve frecuentemente acentuada tanto por la gravedad de la enfermedad como por el elevado coste del tratamiento. El objetivo de este artículo es describir cómo la metodología de comparación indirecta ajustada con emparejamiento ha sido empleada hasta la fecha en la evaluación de fármacos hematooncológicos por parte de agencias internacionales.Método: Para la obtención de los datos del análisis se ha realizado una búsqueda exhaustiva en las páginas web de las agencias nacionales europeas entre enero de 2015 y octubre de 2019 que mostraran información pública del proceso evaluativo. Se revisaron las evaluaciones de estas agencias para obtener un listado de fármacos oncohematológicos para los que constara la presentación de documentación de una comparación indirecta ajustada con emparejamiento. Para este listado de fármacos se analizó para cada agencia seleccionada el papel que dicha comparación tuvo en la evaluación.Resultados: Se han encontrado 13 tratamientos para patologías hematooncológicas en las que el laboratorio había presentado comparaciones con metodología de comparación indirecta ajustada con emparejamiento en su documentación, principalmente a partir del primer semestre de 2018. La aceptación de la metodología diverge entre agencias, pasando de un 50% en el caso del Instituto Nacional para la Salud y la Excelencia Clínica británico, a un 40% en el Alto Comisionado de Salud francés, a no haberse tenido en cuenta en ninguno de los tres casos evaluados por el Instituto para la Calidad y Eficiencia en los cuidados de salud alemán. La principal causa de no aceptación fue la existencia de problemas relacionados con el emparejamiento.Conclusiones: La metodología de comparación indirecta ajustada con emparejamiento es una herramienta de comparación indirecta que está siendo considerada por las agencias analizadas en el proceso de toma de decisiones de evaluación de nuevos medicamentos.
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Atención a la Salud , Preparaciones Farmacéuticas , HumanosRESUMEN
Background: Transthyretin amyloid polyneuropathy (ATTR-PN) is a fatal disease associated with substantial burden of illness. Three therapies are approved by the European Medicines Agency for the management of this rare disease. The aim of this study was to compare the total annual treatment specific cost per-patient associated with ATTR-PN in Spain.Methods: An Excel-based patient burden and cost estimator tool was developed to itemize direct and indirect costs related to treatment with inotersen, patisiran, and tafamidis in the context of ATTR-PN. The product labels and feedback from five Spanish ATTR-PN experts were used to inform resource use and cost inputs.Results: Marked differences in costs were observed between the three therapies. The need for patisiran- and inotersen-treated patients to visit hospitals for pre-treatment, administration, and monitoring was associated with increased patient burden and costs compared to those treated with tafamidis. Drug acquisition costs per-patient per-year were 291,076 (inotersen), 427,250 (patisiran) and 129,737 (tafamidis) and accounted for the majority of total costs. Overall, the total annual per-patient costs were lowest for patients treated with tafamidis (137,954), followed by inotersen (308,358), and patisiran (458,771).Conclusions: Treating patients with tafamidis leads to substantially lower costs and patient burden than with inotersen or patisiran.
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Neuropatías Amiloides Familiares/terapia , Benzoxazoles/administración & dosificación , Costo de Enfermedad , Oligonucleótidos/administración & dosificación , ARN Interferente Pequeño/administración & dosificación , Neuropatías Amiloides Familiares/economía , Benzoxazoles/economía , Costos de los Medicamentos/estadística & datos numéricos , Costos de la Atención en Salud/estadística & datos numéricos , Hospitalización/economía , Humanos , Oligonucleótidos/economía , ARN Interferente Pequeño/economía , EspañaRESUMEN
We conducted research to assess hospital pharmacists' familiarity with/interpretation of data requirements for the different regulatory approval frameworks and the impact of this on their approach to substitution in the formulary. The online questionnaire included a small molecule (acetylsalicylic acid-follow-ons approved via the generic pathway), two biologic drugs (insulin glargine and etanercept-follow-ons approved via the biosimilar pathway), a non-biologic complex drug (NBCD; glatiramer acetate-follow-ons approved via the hybrid pathway) and a nanomedicine, ferric carboxymaltose (no follow-ons approved as yet). The study was conducted in two phases: an initial qualitative pilot study with 30 participants, followed by a quantitative stage involving 201 pharmacists from five European countries. Most expected negligible safety/efficacy differences between reference and follow-on products. Head-to-head clinical data showing therapeutic equivalence as a prerequisite for reference product/follow-on substitution was perceived to be needed most for biologics (47%), followed by NBCDs (44%)/nanomedicines (39%) and small molecules (23%). Overall, 28% did not know the data requirements for follow-on approval via the hybrid pathway; 16% were familiar with this pathway, compared with 50% and 55% for the generic and biosimilar pathways, respectively. Overall, 19% of respondents thought the European Medicines Agency (EMA) was responsible for defining the substitutability of follow-ons. Education is required to increase hospital pharmacist's knowledge of regulatory approval frameworks and their relevance to substitution practices.
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The approval pathway for biosimilars of monoclonal antibodies in the European Union is aimed at ruling out the presence of significant differences with the original biological in quality attributes, efficacy, immunogenicity and safety. It also provides the rationale for extrapolating the evidence obtained with a biosimilar in at least one indication to the rest of the approved indications of its original biological, thus simplifying the development programme of biosimilars. Biosimilars of monoclonal antibodies available in the European Union for the treatment of inflammatory diseases and cancer have fulfilled all the requirements for approval, and many of them have additional evidence available. Moreover, real world data confirms the safety and efficacy of these drugs in the indications they are being used for. In Spain, many scientific societies endorse the regulatory pathway of biosimilars and acknowledge their role in the efficiency of the healthcare system. Even so, some barriers remain that limit their use. The implementation of different measures at the patient, prescriber, institutional, and national levels might increase the penetration of biosimilars, freeing up resources that may be invested in other therapies and, potentially, boost innovation.
El proceso de aprobación de los biosimilares de anticuerpos monoclonales en la Unión Europea está dirigido a descartar la presencia de diferencias significativas con el biológico original en los atributos de calidad, eficacia, inmunogenicidad y seguridad. Proporciona además la justificación para extrapolar la evidencia obtenida con un biosimilar en al menos una indicación al resto de indicaciones aprobadas para su biológico original, simplificando el programa de desarrollo de los biosimilares. Los biosimilares de anticuerpos monoclonales disponibles en la Unión Europea para el tratamiento de enfermedades inflamatorias y del cáncer han cumplido todos los requerimientos establecidos para la aprobación, y en muchos casos disponen de evidencia adicional. Además, los datos de uso en la vida real están confirmando la seguridad y eficacia de estos fármacos en las distintas patologías en las que se están utilizando. En España, varias sociedades médicas avalan el proceso regulatorio de los biosimilares y reconocen su papel en la eficiencia del sistema sanitario. No obstante, todavía existen algunas barreras que limitan su uso. La aplicación de diferentes medidas a nivel de paciente, prescriptor, institucional y nacional podría aumentar la penetración de los biosimilares, liberando recursos que podrían invertirse en otras terapias y, potencialmente, favorecer la innovación.
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Antiinflamatorios/uso terapéutico , Anticuerpos Monoclonales/uso terapéutico , Antineoplásicos Inmunológicos/uso terapéutico , Biosimilares Farmacéuticos , Inflamación/tratamiento farmacológico , Neoplasias/tratamiento farmacológico , Aprobación de Drogas , HumanosRESUMEN
BACKGROUND: The clinical and economic management of retinal diseases has become more complex following the introduction of new intravitreal treatments. Multicriteria decision analysis (MCDA) offers the potential to overcome the challenges associated with traditional decision-making tools. OBJECTIVES: A MCDA to determine the most relevant criteria to decision-making in the management of diabetic macular edema (DME) based on the perspectives of multiple stakeholders in Spain was developed. This MCDA was termed the MULTIDEX-EMD study. METHODS: Nineteen stakeholders (7 physicians, 4 pharmacists, 5 health authorities and health management experts, 1 psychologist, and 2 patient representatives) participated in this three-phase project. In phase A, an advisory board defined all of the criteria that could influence DME treatment decision-making. These criteria were then screened using a discrete choice experiment (DCE) (phase B). Next, a multinomial logit model was fitted by applying the backward elimination algorithm (relevant criteria: p value < 0.05). Finally, the results were discussed in a deliberative process (phase C). RESULTS: Thirty-one criteria were initially defined (phase A) and grouped into 5 categories: efficacy/effectiveness, safety, organizational and economic impact, patient-reported outcomes, and other therapeutic features. The DCE results (phase B) showed that 10 criteria were relevant to the decision-making process for a 50- to 65-year-old DME patient: mean change in best corrected visual acuity (p value < 0.001), percentage of patients with an improvement of ≥ 15 letters (p value < 0.001), effect duration per administration (p value = 0.008), retinal detachment (p value < 0.001), endophthalmitis (p value = 0.012), myocardial infarction (p value < 0.001), intravitreal hemorrhage (p value = 0.021), annual treatment cost per patient (p value = 0.001), health-related quality of life (HRQoL) (p value = 0.004), and disability level (p value = 0.021). CONCLUSIONS: From a multi-stakeholder perspective, the selection of an appropriate treatment for DME patients should guarantee patient safety and maximize the visual acuity improvement and treatment effect duration. It should also contribute to system sustainability by being affordable, it should have a positive impact on HRQoL, and it should prevent disability.
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OBJECTIVE: To evaluate the burden of diabetes mellitus (DM) in adult patients with acromegaly treated with second-line pharmacotherapy, from the perspective of the Spanish National Health System (NHS). METHODS: A Markov model was developed including three states: normal glucose metabolism, DM and death. The evolution of a hypothetical cohort of acromegaly patients requiring second-line pharmacological treatment (pegvisomant or pasireotide) after first generation somatostatin analogues therapy was analyzed. Direct healthcare costs regarding acromegaly management, diabetes management and drugs costs were obtained from Spanish sources. Transition probabilities between health states were obtained from published studies. Deterministic and probabilistic sensitivity analyses were undertaken. RESULTS: Compared to pasireotide, pegvisomant increased the likelihood of glucose normalization and reduced the likelihood of DM. Consequently, in a cohort of 1,000 patients with acromegaly, treatment with pegvisomant compared to pasireotide would prevent 243, 413 and 453 cases of DM after 1, 2 and 5 years, respectively, and would reduce mortality by 0.1% after 5 years of treatment. This would result in 1 million euros savings for the NHS in 5 years. These health benefits would be obtained with savings of 1,512, 3,422 and 10,162 per patient treated with pegvisomant, after 1, 2 and 5 years, respectively. After 5 years of treatment, the probability that pegvisomant generated savings versus pasireotide would be 65.3%. CONCLUSION: The favorable effects of pegvisomant on glucose metabolism would allow a considerable number of cases of DM to be avoided compared to pasireotide, resulting in savings for the NHS in Spain.
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INTRODUCTION: The objective of this study is to estimate the economic impact associated with the optimisation of triple antiretroviral treatment (ART) in patients with undetectable viral load according to the recommendations from the GeSIDA/PNS (2015) Consensus and their applicability in the Spanish clinical practice. METHODS: A pharmacoeconomic model was developed based on data from a National Hospital Prescription Survey on ART (2014) and the A-I evidence recommendations for the optimisation of ART from the GeSIDA/PNS (2015) consensus. The optimisation model took into account the willingness to optimise a particular regimen and other assumptions, and the results were validated by an expert panel in HIV infection (Infectious Disease Specialists and Hospital Pharmacists). The analysis was conducted from the NHS perspective, considering the annual wholesale price and accounting for deductions stated in the RD-Law 8/2010 and the VAT. RESULTS: The expert panel selected six optimisation strategies, and estimated that 10,863 (13.4%) of the 80,859 patients in Spain currently on triple ART, would be candidates to optimise their ART, leading to savings of 15.9M/year (2.4% of total triple ART drug cost). The most feasible strategies (>40% of patients candidates for optimisation, n=4,556) would be optimisations to ATV/r+3TC therapy. These would produce savings between 653 and 4,797 per patient per year depending on baseline triple ART. CONCLUSION: Implementation of the main optimisation strategies recommended in the GeSIDA/PNS (2015) Consensus into Spanish clinical practice would lead to considerable savings, especially those based in dual therapy with ATV/r+3TC, thus contributing to the control of pharmaceutical expenditure and NHS sustainability.
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Antirretrovirales/economía , Antirretrovirales/uso terapéutico , Costos y Análisis de Costo , Adhesión a Directriz/economía , Infecciones por VIH/tratamiento farmacológico , Guías de Práctica Clínica como Asunto , Infecciones por VIH/virología , Humanos , España , Carga ViralRESUMEN
This chapter reviews the current model of HIV patient care and its economic impact. There are clinical and geographical differences in the care HIV patients receive and a need to develop new models of comprehen-sive HIV care has been identified. HIV infection in Spain is an important and expensive public health problem. The main costs are due to an-tiretroviral therapy, indirect costs and the cost of admission, consultation and diagnostic testing. A strong correlation between severity and cost has been identified. Patients diagnosed late have a poorer clinical course, increased mortality and require more medical and therapeutic resources. Early detection of the disease significantly improves the clinical outcomes of patients. The increased number of patients receiving treatment and their progressive ageing will increase costs of HIV healthcare in the coming years.