Oblikue Consulting (Spain)

Generalized myasthenia gravis (gMG) is a rare, chronic autoimmune neuromuscular disorder that currently lacks a curative treatment. Efgartigimod alfa is the first human IgG1 Fc fragment approved in Spain for the management of this condition. This study aims to evaluate the value of efgartigimod for treating gMG with anti-acetylcholine receptor (gMG AChR+) antibodies compared with ravulizumab, zilucoplan, and rozanolixizumab using a multi-criteria decision analysis (MCDA) framework. A multidisciplinary group of eight experts evaluated the value of efgartigimod against ravulizumab, zilucoplan, and rozanolixizumab. The MCDA framework adapted for evaluating orphan drugs (ODs) in Spain, comprising nine quantitative and three qualitative criteria, was used. gMG AChR+ has been recognised as a severe and debilitating condition with considerable unmet needs. Efgartigimod achieved favourable average scores compared with ravulizumab, zilucoplan, and rozanolixizumab across all comparative parameters, including efficacy, safety, and patient-reported outcomes (PROs), potentially resulting in cost savings. Efgartigimod was deemed to have a significant therapeutic impact, with supporting data considered high quality. Efgartigimod alfa showed a higher overall value contribution than the three comparators, with the difference being most notable for ravulizumab. Furthermore, it was concluded that efgartigimod aligns well with the specific priorities, objectives, and capacities of the National Healthcare System (NHS). Efgartigimod has been recognised as a valuable option for gMG AChR+ treatment in Spain by a multidisciplinary panel of experts through the application of MCDA, receiving higher scores compared with ravulizumab, zilucoplan, and rozanolixizumab.

El cáncer infantil es una enfermedad que puede ser vista desde diferentes áreas del conocimiento. Los principales afectados son los sujetos que la padecen, por lo cual, se considera que su experiencia es de suma importancia; sin embargo, actualmente existe poca literatura acerca de este tema en Colombia. Por esta razón, se desarrolló este estudio con el objetivo de interpretar y comprender la experiencia del cáncer infantil por medio de la fenomenología hermenéutica. Entre septiembre y noviembre de 2022 se realizaron 18 entrevistas semiestructuradas a población pediátrica oncológica en una fundación en Bogotá. En estas se indagó sobre la experiencia vivida con el impacto del diagnóstico, las redes de apoyo, los cambios en hábitos y rutinas, la religión/espiritualidad, las creencias sobre la enfermedad, entre otros aspectos. Como resultado, la población infantil sufre el tratamiento no solo por el dolor físico secundario, sino por la pérdida de la identidad y la alteración en la relación con la familia y los amigos; no obstante, ellos mismos han desarrollado mecanismos de afrontamiento y aprendizaje ante la enfermedad. En conclusión, el cáncer es un proceso que cambia la percepción de la vida de la población pediátrica oncológica que lo padece, y la fenomenología es una herramienta que permite entender de mejor manera la vivencia del cáncer desde la perspectiva personal, esto contribuye a tener un trato más humanizado e integral de la enfermedad.

Abstract. The Land Use/Cover Area frame Survey (LUCAS) of the European Union (EU) presents a rich resource for detailed understanding of land cover and use, making it invaluable for Earth Observation (EO) applications. This manuscript discusses the recent advancements and improvements in the LUCAS Copernicus module, particularly the data collection process of 2022, its protocol simplifications, and geometry definitions compared to the 2018 survey and data. With approximately 150,000 polygons collected in 2022, an increase from 60,000 in 2018, the LUCAS Copernicus 2022 data provides a unique and comprehensive in-situ dataset for EO applications. The protocol simplification also facilitates a faster and more efficient data collection process. In 2022, there are 137,966 polygons generated, out of the original 149,408 LUCAS Copernicus points, which means 92.3 % of the points were actually surveyed. The data holds 82 land cover classes for the Copernicus module LUCAS level 3 legend (88 classes). For land use the data holds 40 classes, along with 18 classes of land use types. The dataset is available here for download (PID: http://data.europa.eu/89h/e3fe3cd0-44db-470e-8769-172a8b9e8874). The paper further elaborates on the implications of these enhancements and the need for continuous harmonisation to ensure semantic consistency and temporal usability of data across different periods. Moreover, it calls for additional studies exploring the potential of the collected data, especially in the context of remote sensing and computer vision. The manuscript ends with a discussion on future data usage and dissemination strategies.

Objetivo: El objetivo del estudio fue estimar la carga socioeconómica, en relación con los costes sanitarios directos, indirectos e intangibles de los pacientes con mielofibrosis (MF) con y sin anemia en España. Material y métodos: Se realizó un estudio de la carga de la MF desde la perspectiva del Sistema Nacional de Salud (SNS) español con un enfoque prevalente, que estima la carga de la enfermedad teniendo en cuenta el período de 1 año, reflejando la carga socioeconómica de la MF en 2023. Para ello se realizó una revisión de la literatura de estudios relacionados con el uso de recursos y costes de la MF publicados entre 2012 y mayo 2023. Los datos obtenidos fueron validados por un grupo multidisciplinar de expertos españoles. Se siguieron las recomendaciones de manejo y tratamiento de la MF descritos en la Guía de Neoplasias Mieloproliferativas Crónicas Filadelfia Negativas elaborada por GEMFIN (2020)2, las guías ICOPraxis (2020)3 y el manual GAMFIN4 para el manejo y tratamiento de la MF. El modelo se desarrolló en Excel y se estructuró en cuatro secciones principales: carga epidemiológica, costes directos médicos, costes indirectos y costes intangibles. Resultados: Se estima que 3.992 personas padecen MF en España, de los cuales 7,5% (299) son candidatos al trasplante alogénico de progenitores hematopoyéticos (alo-TPH) y el 77,1% (3.078) están en tratamiento activo. De estos, el 46,75% (1.439) presentan anemia. El coste anual por paciente asociado al diagnóstico de MF fue de 1.614 €/paciente. Los costes asociados a la atención hospitalaria se estimaron en 2.283 €/paciente con MF sin anemia y en 5.815 €/paciente con anemia. El coste relacionado con el tratamiento específico de las manifestaciones hiperproliferativas de la MF (esplenomegalia y síntomas constitucionales) se estimó en 22.296 y 23.295 €/paciente sin y con anemia, respectivamente. Por último, el coste del tratamiento y manejo específico de la anemia se estimó en 7.765€/paciente. Los costes indirectos, considerando las incapacidades temporales y permanentes, y los cuidados informales supondrían unos costes totales en los pacientes con MF de aproximadamente 20 M€. Conclusiones: La MF representa una carga económica importante para el SNS español, con un coste total de unos 140 millones de euros en 2023. La anemia asociada a la MF implica un coste adicional de aproximadamente 36 millones de euros en costes médicos directos al SNS. Palabras Clave: Mielofibrosis, anemia asociada a mielofibrosis, epidemiología, carga económica, costes directos, costes indirectos, uso de recursos, transfusiones.

IntroductionEpilepsy is a serious neurological disease, ranking high in the top causes of disability. The main goal of its treatment is to achieve seizure freedom without intolerable adverse effects. However, approximately 40% of patients suffer from Drug-Resistant Epilepsy (DRE) despite the availability of the latest options called third-generation Anti-Seizure Medications(ASMs). Cenobamate is the first ASM approved in Spain for the adjunctive treatment of Focal-Onset Seizures (FOS) in adult patients with DRE. The introduction of a new drug increases the number of therapeutic options available, making it important to compare it with existing alternatives in terms of clinical benefit and efficiency. PurposeThis study aimed to compare the clinical benefit, in terms of the Number Needed to Treat (NNT), and the efficiency, in terms of Cost per NNT (CNT), associated with cenobamate versus third-generation ASMs used in Spain for the adjunctive treatment of FOS in patients with DRE. MethodsThe Number Needed to Treat data was calculated based on the ≥50% responder rate and seizure freedom endpoints (defined as the percentage of patients achieving 50% and 100% reduction in seizure frequency, respectively), obtained from pivotal clinical trials performed with cenobamate, brivaracetam, perampanel, lacosamide, and eslicarbazepine acetate. The NNT was established as the inverse of the treatment responder rate minus the placebo responder rate and was calculated based on the minimum, mid-range Daily Defined Dose (DDD), and maximum doses studied in the pivotal clinical trials of each ASM. CNT was calculated by multiplying the annual treatment cost by NNT values for each treatment option. ResultsIn terms of NNT, cenobamate was the ASM associated with the lowest values at all doses for both ≥50% responder rate and seizure freedom compared with the alternatives. In terms of CNT, for ≥50% responder rate, cenobamate was the ASM associated with the lowest CNT values at DDD and lacosamide and eslicarbazepine acetate at the minimum and maximum dose, respectively. For seizure freedom, cenobamate was associated with the lowest CNT value at DDD and the maximum dose and lacosamide at the minimum dose. ConclusionsCenobamate could represent the most effective ASM in all doses studied compared to the third-generation ASMs and the most efficient option at DDD for both ≥50% responder rate and seizure freedom. This study could represent an important contribution towards informed decision-making regarding the selection of the most appropriate therapy for FOS in adult patients with DRE from a clinical and economical perspective in Spain.

ObjectiveThe objective of this study was to assess the non-adherence level of Spanish clinical practice to guideline recommendations for the treatment of chronic obstructive pulmonary disease (COPD) and to estimate the potential impact on pharmaceutical expenditure resulting from transitioning current treatment patterns according to guidelines.MethodsA model was developed to compare current prescribing patterns with two alternative scenarios: the first aligned with the Global Initiative for Chronic Obstructive Lung Disease (GOLD 2020) recommendations, and the second with the Spanish Guidelines for COPD (GesEPOC 2017). Current treatment practice was obtained from publications that describe treatment patterns by pulmonology departments in Spain. The economic impact between patterns was calculated from the perspective of the Spanish National Health System (NHS), considering the annual pharmacological costs of COPD inhaled maintenance therapy. Two additional analyses were performed: one that included current prescribing patterns of patients managed by pulmonology and primary care centers in Spain (published aggregated data); and another that only considered the appropriate use of inhaled corticosteroids (ICS) treatment according to guidelines.ResultsIt was estimated that 54% and 38% of patients were not treated in line with GOLD and GesEPOC recommendations, respectively, mainly due to a broader use of ICS-based therapies. Adapting treatment to recommendations could provide a potential annual cost-saving of €17,792,022 (according to GOLD) and €5,881,785 (according to GesEPOC). In scenario analysis 1, a 26% of non-adherence to GesEPOC guideline was observed with a potential annual pharmacological cost-saving of €2,707,554. In scenario analysis 2, considering only inappropriate use of ICS treatment, an annual cost-saving of €17,863,750 (according to GOLD) and €9,904,409 (according to GesEPOC) was calculated.ConclusionMore than a third of treatments for COPD patients in Spain are not prescribed in accordance with guideline recommendations. The adaptation of clinical practice to guideline recommendations could provide important cost-savings for the Spanish NHS.

Storm Gloria was the 10th named storm in Europe for the 2019–2020 winter season, and it severely affected Spain and France. This powerful storm represents an excellent study case to analyze the capabilities of the different ocean model systems available in the Spanish Mediterranean coasts to simulate extreme events, as well as to assess their suitability to enhance preparedness in maritime disasters with high impacts on coastal areas. Five different operational ocean forecasting services able to predict the storm-induced ocean circulation are evaluated. Three of the systems are delivered by the Copernicus Marine Service (hereafter CMEMS): the CMEMS global scale solution (GLO-1/12°), the specific Mediterranean basin scale one (MED-1/24°), and the regional solution for the Atlantic façade (IBI-1/36°), which includes also part of the western Mediterranean. These CMEMS core products are complemented with two higher resolution models focused on more limited areas, which provide operational forecasts for coastal applications: the WMOP system developed at the Balearic Islands Coastal Observing and Forecasting System (SOCIB) with a horizontal resolution of roughly 2 km and the Puertos del Estado (PdE) SAMOA systems with a 350-m resolution that cover the coastal domains of the Spanish Port Authorities of Barcelona, Tarragona, Castellón and Almeria. Both the WMOP and SAMOA models are nested in CMEMS regional systems (MED and IBI, respectively) and constitute good examples of coastal-scale-oriented CMEMS downstream services. The skill of these five ocean models in reproducing the surface dynamics in the area during Gloria is evaluated using met-ocean in situ measurements from numerous buoys (moored in coastal and open waters) and coastal meteorological stations as a reference to track the effects of the storm in essential ocean variables such as surface current, water temperature, and salinity throughout January 2020. Furthermore, modeled surface dynamics are validated against hourly surface current fields from the two high-frequency radar systems available in the zone (the SOCIB HF-Radar system covering the eastern part of the Ibiza Channel and the PdE one at Tarragona, which covers the Ebro Delta, one of the coastal areas most impacted by Gloria). The results assess the performance of the dynamical downscaling at two different levels: first, within the own CMEMS service (with their regional products, as enhanced solutions with respect to the global one) and second in the coastal down-streaming service side (with very high-resolution models reaching coastal scales). This multi-model study case focused on Storm Gloria has allowed to identify some strengths and limitations of the systems currently in operations, and it can help outlining future model service upgrades aimed at better forecasting extreme coastal events.