Value contribution of etranacogene dezaparvovec gene therapy in moderately severe and severe haemophilia B through multi-criteria decision analysis.

Introduction: The value of gene therapies for haemophilia needs to be assessed holistically.

Aim: To determine the value of etranacogene dezaparvovec (ED) compared to current extended half-life (EHL) recombinant factors (rFIX), using multi-criteria decision analysis (MCDA).

Method: MCDA EVIDEM methodology adapted to orphan drugs was used, with nine quantitative criteria and four contextual criteria.

A Review of Current Approaches to Evaluating and Reimbursing New Medicines in a Subset of OECD Countries.

Objectives: The aim of this study was to review the current evaluation and funding processes for new drugs in different developed countries, to provide a comparative framework with detailed, homogeneous, and up-to-date information.

Methods: Scientific publications, reports and websites were reviewed between July and December 2021 using PubMed, Google Scholar, and grey literature sources. The main items searched were actors and processes, including timelines, characteristics of clinical and economic evaluations, participation of stakeholders, elements of price and reimbursement decisions, cost-effectiveness thresholds and specific funds.

Comparison of GLP-1 receptor agonists and other Glucose-Lowering agents on cardiovascular outcomes in individuals with type 2 diabetes and Obesity: A Spanish Real-World Population-Based study.

Aims: Assess the impact of glucagon-like peptide receptor agonists (GLP-1RA) compared to other glucose-lowering agents on cardiovascular outcomes in individuals with type 2 diabetes and obesity in a Spanish metropolitan area.

Methods: A retrospective population-based type 2 diabetes cohort was identified from the Valencia Clinic-Malvarrosa Department electronic databases (2014-2019). Study groups included GLP-1RA, sodium-glucose co-transporter-2 inhibitors (SGLT2i), Insulin, and Miscellany (other glucose-lowering agents).

Real-World Evaluation of GLP-1 Receptor Agonist Therapy Persistence, Adherence and Therapeutic Inertia Among Obese Adults with Type 2 Diabetes.

Introduction: In type 2 diabetes (T2D), key barriers to optimal glycaemic control include lack of persistence with treatment, reduced medication adherence and therapeutic inertia. This study aimed to assess the impact of these barriers in obese adults with type 2 diabetes treated with a GLP-1 receptor agonist (GLP-1RA) and compare them against other glucose-lowering agents in a real-world setting.

Methods: A retrospective study was conducted using electronic medical records from 2014 to 2019 for adults with T2D at the Valencia Clínico-Malvarrosa Department of Health (Valencia, Spain).

Incidence and impact of atrial fibrillation in heart failure patients: real-world data in a large community.

Aims: The objective of the present study is to assess the bidirectional association between heart failure (HF) and atrial fibrillation (AF) using real-world data.

Methods And Results: From an electronic health recording with a population of 3 799 885 adult subjects, those with prevalent or incident HF were selected and followed throughout a study period of 5 years. Prevalence and incidence of AF, and their impact in the risk for acute HF hospitalization, worsening renal function, ischaemic and haemorrhagic stroke, and all-cause mortality were identified.

Real world data of anticoagulant treatment in non-valvular atrial fibrillation across renal function status.

The objective is to assess the impact of anticoagulant treatment in non-valvular atrial fibrillation (AF) and different categories of renal dysfunction in real world. Electronic Health recordings of patients with diagnosis of AF and renal function collected throughout 5 years and classified according to KDIGO categories. Stroke, transitory ischemic attack (TIA), intracranial hemorrhage and all-cause mortality were identified.

Real-World Data of Anticoagulant Treatment in Non-valvular Atrial Fibrillation.

Aims: To assess the impact of anticoagulant treatment on risk for stroke and all-cause mortality of patients with atrial fibrillation using real-world data (RWD).

Methods: Patients with prevalent or incident atrial fibrillation were selected throughout a study period of 5 years. Stroke, transitory ischemic attack, hemorrhagic stroke, and all-cause mortality were identified in the claims of the electronic health records (EHRs).

Therapeutic inertia in patients with type 2 diabetes treated with non-insulin agents.

Aims: To analyze therapeutic inertia in type 2 diabetes (T2D) subjects with suboptimal glycemic control and treated with ≥2 non-insulin antidiabetic agents in a primary care setting.

Methods: A retrospective study was conducted using electronic medical records from subjects with HbA1c ≥7.0% (≥53 mmol/mol).

Impact of Acute Hemoglobin Falls in Heart Failure Patients: A Population Study.

This study assessed the impact of acute hemoglobin (Hb) falls in heart failure (HF) patients. HF patients with repeated Hb values over time were included. Falls in Hb greater than 30% were considered to represent an acute episode of anemia and the risk of hospitalization and all-cause mortality after the first episode was assessed.

A Multiple Stakeholder Multicriteria Decision Analysis in Diabetic Macular Edema Management: The MULTIDEX-EMD Study.

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.

Acute kidney injury in heart failure: a population study.

Aims: The objective of the present study is to assess the prognostic value of acute kidney injury (AKI) in the evolution of patients with heart failure (HF) using real-world data.

Methods And Results: Patients with a diagnosis of HF and with serial measurements of renal function collected throughout the study period were included. Estimated glomerular filtration rate (GFR) was calculated with the CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration).

Number of drugs used in secondary cardiovascular prevention and late survival in the population of Valencia Community, Spain.

Background: Drug treatment for secondary prevention of cardiovascular disease is recommended by guidelines, but it is not always followed in real life. This study wanted to assess the size of this gap and its impact on mortality in subjects after a cardiovascular event (MACE).

Methods: Patients with any of MACE in the period from January 1st 2011 to December 31st 2013, and more than one year of follow-up were selected from population of the Valencian Community.

Explaining primary healthcare pharmacy expenditure using classification of medications for chronic conditions.

Background: The Valencian Autonomous Community (Spain) has implemented a scheme of purchasing services with the participation of public and private providers. Five districts are managed using public-private partnership. The financing model is capitation and inter-center invoice.

Adherence to and appropriateness of anti-osteoporotic treatments in patients aged 50 and over in the Valencia Region (Spain). The ESOSVAL-AD study.

Background: A study to evaluate the adherence to and appropriateness of anti-osteoporotic treatments in a cohort of men and women aged 50 and over participating in the ESOSVAL-R study.

Design: An observational, longitudinal, prospective cohort study;

Study Subjects: Men and women aged 50 and over living in the Valencia Region (Spain) who initiated treatment between June 15, 2009, and June 15, 2011, in primary healthcare centers with electronic medical records;

Data Sources: The main data source will be electronic medical records. Measurement of results: Degree of compliance with and persistence of anti-osteoporotic treatments, and the proportion of patients with appropriate anti-osteoporotic treatment in accordance with the most relevant and high impact recommendations with clearly defined treatment algorithms in Spain (the Spanish National Health System guide (2010), the General Practitioners' Society (2007) and the General Directorate for Pharmacy and Medical Products of Madrid (2007)), and with the National Osteoporosis Foundation (NOF, 2010), and the International Osteoporosis Foundation guidelines (IOF, 2008);

Analysis: 1.

Antidepressant utilization in primary care in a Spanish region: impact of generic and reference-based pricing policy (2000-2004).

Objective: To describe the evolution of antidepressant use in primary care in the Valencian region (Spain) from 2000 to 2004 and to analyze the effects of reference-based price and generic drugs introduction on drug utilization and cost saving.

Methods: Retrospective observational study in primary care using sales data collected from antidepressant group (N06A), corresponding to the period 2000-2004. Defined daily dose (DDD)/1000 inhabitants per day were obtained as consumption data.