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Advanced medicinal products; Financing government; Health technology assessment Medicamentos avanzados; Financiación del gobierno; Evaluación de tecnologías sanitarias Medicaments avançats; Finançament del govern; Avaluació de tecnologies sanitàries Objectives The uncertainty in the cost-benefit of advanced therapy medicinal products (ATMPs) is a current challenge for their reimbursement in health systems. This study aimed to provide a comparative analysis of the National Health Authorities (NHAs) reimbursement recommendations issued in different European countries. Methods The NHA reimbursement recommendations for the approved ATMPs were compared among 8 European Union (EU) Countries (EU8: Ireland, England/Wales, Scotland, The Netherlands, France, Germany, Spain, and Italy). The search was carried out until December 31, 2021. Results A total of 19 approved ATMPs and 76 appraisal reports were analyzed. The majority of the ATMPs were reimbursed, although with uncertainty in added therapeutic value. No relationship between the type of the European Medicines Agency approval and reimbursement was found. Managed entry agreements, such as payment by results, were necessary to ensure market access. The main issue during the evaluation was to base the cost-effectiveness analyses on assumptions because of the limited long-term data. The estimated incremental cost-effectiveness ratio among countries reveals high variability. Overall, the median time to NHA recommendation for the EU8 is in the range of 9 to 17 months. Conclusions Transparent, harmonized, and systematic assessments across the EU NHAs in terms of cost-effectiveness, added therapeutic value, and grade of innovativeness are needed. This could lead to a more aligned access, increasing the EU market attractiveness and raising public fairness in terms of patient access and pricing.

A new category of products, i.e. regenerative medicine products (RPs), has been defined for advanced therapies medicinal products in Japan, as well as a legislative and regulatory framework to promote their clinical development. This review analyses the most relevant features of the regulatory strategies and clinical development that led RPs to their approval in Japan. As of 31st September 2021, a total of 14 RPs were approved for 16 indications. From a regulatory standpoint, the available designations allow attractive benefit packages that promote the development of innovative products in Japan and is one of the key points to consider when the global regulatory strategy for the product is being developed. RPs regulations in Japan allow adaptive licensing and constitute shortcut through the clinical development to the approval. RPs have been mainly approved so far based on small studies with inconclusive and limited evidence of efficacy and safety, prioritizing the unmet medical needs of the target diseases, and therefore, the early access for patients. This review also compares the regulatory and clinical development for the current approved RPs in Japan with the development trends in the European Union and United States of America.

Incontinència urinària; Tractament; Algorisme Incontinencia urinaria; Tratamiento; Algoritmo Urinary incontinence; Treatment; Algorithm Aquesta pauta té l’objectiu d’harmonitzar el tractament farmacològic dels pacients amb incontinència urinària i es dirigeix, principalment, als professionals de l’àmbit de l’atenció primària, comunitària i especialitzada. Es prioritzen les guies de pràctica clínica de referència vigents i s’identifiquen les revisions sistemàtiques i els assaigs clínics de rellevància que plantegen preguntes clíniques concretes. La pauta també incorpora la revisió de l’evidència científica generada després de la publicació de les esmentades guies i la utilització de criteris d’eficiència per a la realització de recomanacions de selecció i priorització de medicaments i/o grups terapèutics, d’acord amb la política farmacèutica del CatSalut. Esta pauta tiene el objetivo de armonizar el tratamiento farmacológico de los pacientes con incontinencia urinaria y se dirige, principalmente, a los profesionales del ámbito de la atención primaria, comunitaria y especializada. Se priorizan las guías de práctica clínica de referencia vigentes y se identifican las revisiones sistemáticas y los ensayos clínicos de relevancia que plantean preguntas clínicas concretas. La pauta también incorpora la revisión de la evidencia científica generada tras la publicación de dichas guías y la utilización de criterios de eficiencia para la realización de recomendaciones de selección y priorización de medicamentos y / o grupos terapéuticos, de acuerdo con la política farmacéutica del CatSalut. This guideline aims to harmonize the pharmacological treatment of patients with urinary incontinence and is aimed primarily at professionals in the field of primary, community and specialized care. Current clinical practice guidelines are prioritized and systematic reviews and relevant clinical trials that raise specific clinical questions are identified. The guideline also includes the review of the scientific evidence generated after the publication of the aforementioned guidelines and the use of efficiency criteria for making recommendations for the selection and prioritization of drugs and / or therapeutic groups, in accordance with the pharmaceutical policy of CatSalut.

Incontinència urinària; Tractament no farmacològic; Medicaments Incontinencia urinaria; Tratamiento no farmacológico; Medicamentos Urinary incontinence; Non-pharmacological treatment; Medications Les Pautes d’harmonització farmacoterapèutica del CatSalut són una eina adreçada a tots els professionals assistencials per establir unes bones pràctiques en el maneig farmacològic de diferents patologies prioritzades. Les pautes de la incontinència urinària incorporen les darreres novetats terapèutiques i científiques, i consideren, en termes poblacionals, els criteris d’eficiència i sostenibilitat de la prestació farmacèutica per garantir l’accessibilitat universal i l’equitat. Tot i que l’evidència d’aquest estudi és molt limitada, el fet que sigui un estudi amb un bon disseny i realitzat en el nostre entorn ha fet que s’inclogui una recomanació basada en els seus resultats i que es recomani vigilar el consum d’algunes infusions. Las Pautas de armonización farmacoterapéutica del CatSalut son una herramienta dirigida a todos los profesionales asistenciales para establecer unas buenas prácticas en el manejo farmacológico de diferentes patologías priorizadas. Las pautas de la incontinencia urinaria incorporan las últimas novedades terapéuticas y científicas, y consideran, en términos poblacionales, los criterios de eficiencia y sostenibilidad de la prestación farmacéutica para garantizar la accesibilidad universal y la equidad. Aunque la evidencia de este estudio es muy limitada, el hecho de que sea un estudio con un buen diseño y realizado en nuestro entorno ha hecho que se incluya una recomendación basada en sus resultados y que se recomiende vigilar el consumo de algunas infusiones. The CatSalut Pharmacotherapeutic Harmonization Guidelines are a tool aimed at all healthcare professionals to establish good practices in the pharmacological management of different priority pathologies. Urinary incontinence guidelines incorporate the latest therapeutic and scientific developments, and consider, in population terms, the criteria for efficiency and sustainability of pharmaceutical delivery to ensure universal accessibility and equity. Although the evidence in this study is very limited, the fact that it is a study with a good design and carried out in our environment has led to the inclusion of a recommendation based on its results and that it is recommended to monitor consumption of some infusions.

BACKGROUND: Consensus Conferences and Guidelines for deep vein thrombosis prophylaxis have been published, which recommend the use of prophylactic heparins in patients with risk of venous thromboembolism (VTE). The aim of this study was the assessment of the prophylaxis of VTE and the adherence to accepted guideline recommendations throughout the hospital. METHODS: A cross-sectional study was carried out in a teaching hospital after guidelines were implemented. Patients' risk factors of deep vein thrombosis, risk categories of patients, and prophylaxis used in different wards were recorded. Appropriate adherence to the guidelines was analysed. RESULTS: Of 397 patients, prophylaxis was used in 231 patients (58%), and low-molecular-weight heparins (LMWH) were used in 224 of them (97%). Patients with prophylaxis had a higher mean number of risk factors (SD) than those without prophylaxis [3.1 (1.4) vs 1.9 (1.4); p0.05)]. Prophylaxis was used in 72% and 90% of moderate and high-risk patients respectively. Appropriate adherence to all guideline recommendations was observed in 42% of patients. Adherence to guidelines was high as regards the use of prophylaxis according to patients' risk factors (78%) and the use of appropriate types of prophylaxis (99%), but was low regarding appropriate heparin dosage (47%) and preoperative dosage (37%). Appropriate prophylaxis use was higher in critical care and surgical wards than in medical wards. CONCLUSION: Prophylaxis of VTE is generally used in risk patients, but appropriate adherence to guidelines is less frequent and variable among different wards. Continuing medical education, discussion and dissemination of guidelines, and regular clinical audit are necessary to improve prophylaxis of VTE in clinical practice.