Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain)

Early access to medicines allows the prescription of a medicine before it is available in the public formulary to patients with severe or rare diseases with high unmet needs who have no authorised therapeutic alternatives available. In this context, consistent decision making is difficult, and a systematic assessment procedure could be useful to tackle complex situations and guarantee the equity of medicines' access. A multidisciplinary panel (MP) conducted four workshops to develop an early access framework based on a reflective multiple criteria decision analysis (MCDA). A set of 12 criteria was agreed: eight quantitative (severity of disease, urgency, efficacy, safety, internal and external validity, therapeutic benefit and plausibility) and four qualitative (therapeutic alternative, existence of precedents, management impact and costs). Quantitative criteria were weighted using a five-point scale. The relative importance of quantitative criteria had mean weights from 4.7 to 3.6, showing its relevance in the decisions. The framework was tested using two case studies, and reliability was assessed by re-test. The re-test revealed no statistical differences, indicating the consistency and replicability of the framework developed. MCDA may help to structure discussions for heterogeneous treatment requests, providing predictability and robustness in decision making involving sensitive and complex situations

Outpatient multimodal intravenous analgesia in patients undergoing day-case surgery : description of a three year experience

The use of elastomeric devices for ambulatory intravenous pain treatment in Major Ambulatory Surgery (MAS) has been described to improve postoperative pain management. The objective of the study was to describe the first 3 years experience of the use of elastomeric devices for ambulatory intravenous pain treatment in MAS implemented at our site since 2010. Data were retrieved from the medical records for all patients who, between January 2010 and March 2014, underwent surgical procedures at the ambulatory surgical centre at our hospital and were prescribed a home-based continuous intravenous analgesia. Data were retrieved from the medical records of 1128 patients. The most frequent surgical interventions included orthopedic and proctology surgeries. 80 % of patients were discharged home without pain; during the first 48 h after discharge roughly 40 % of subjects were completely free of pain, 50 % reported mild pain (VAS 1 to 3) and 9 % reported higher pain scores (4 and above). Peripheral nerve block was associated to better pain control in the immediate postoperative period. Vomiting in the first 24 h was 4.6 % before introducing haloperidol into the drug schemes, and 2.6 % thereafter. Complications related with the intravenous route required treatment withdrawal in 1.1 % cases. Only 3.5 % of patients returned to the hospital in the first 72 h, mainly for non-pain related reasons. Overall, 99.5 % of patients were satisfied with the treatment received at home. Our initial experience suggest that outpatient multimodal intravenous analgesia in patients undergoing day-case surgery is a feasible alternative in our setting, that allows an effective management of postoperative pain with a small rate of adverse events and complications requiring readmission

Changes in treatment patterns and costs for lung cancer have not resulted in relevant improvements in survival: a population-based observational study in Catalonia

Objective: Few published studies have described multidisciplinary therapeutic strategies for lung cancer. This study aims to describe the different approaches used for treating lung cancer in Catalonia in 2014 and 2018 and to assess the associated cost and impact on patient survival. Methods: A retrospective observational cohort study using data of patients with lung cancer from health care registries in Catalonia was carried out. We analyzed change in treatment patterns, costs and survival according to the year of treatment initiation (2014 vs. 2018). The Kaplan-Meier method was used to estimate survival, with the follow-up until 2021. Results: From 2014 to 2018, the proportion of patients undergoing surgery increased and treatments for unresectable tumors decreased, mainly in younger patients. Immunotherapy increased by up to 9% by 2018. No differences in patient survival were observed within treatment patterns. The mean cost per patient in the first year of treatment increased from EUR 14,123 (standard deviation [SD] 4327) to EUR 14,550 (SD 3880) in surgical patients, from EUR 4655 (SD 3540) to EUR 5873 (SD 6455) in patients receiving curative radiotherapy and from EUR 4723 (SD 7003) to EUR 6458 (SD 10,116) in those treated for unresectable disease. Conclusions: From 2014 to 2018, surgical approaches increased in younger patients. The mean cost of treating patients increased, especially in pharmaceutical expenditure, mainly related to the use of several biomarker-targeted treatments. While no differences in overall patient survival were observed, it seems reasonable to expect improvements in this outcome in upcoming years as more patients receive innovative treatments

Registry of patients and treatments of hospital medicines in Catalonia: a tool to improve quality and efficiency of hospital drug use

Medicació hospitalària; Registre de pacients; ConsumMedicación hospitalaria; Registro de pacientes; ConsumoHospital medication; Patient registration; ConsumptionEines i polítiques de col·laboració innovadores per millorar la qualitat del servei i l'atenció al registre de pacients i tractaments de medicaments hospitalaris a Catalunya, una eina per millorar la qualitat i l'eficiència del consum de fàrmacs hospitalari

Pautes per a l’harmonització dels tractaments farmacològics

Pautes del tractament farmacològic; Harmonització farmacoterapèutica; CronicitatPautas del tratamiento farmacológico; Armonización farmacoterapéutica; CronicidadGuidelines for pharmacological treatment; Pharmacotherapeutic Harmonization; ChronicityL’augment de l’esperança de vida i el consegüent envelliment de la població, juntament amb els avenços mèdics que transformen malalties agudes en patologies de llarga evolució (p. ex. VIH o càncer, entre d’altres), enllacen de forma ineludible amb la cronificació. L’existència de diferents visions terapèutiques en un entorn multidisciplinari, la falta de comunicació entre professionals i les diferents perspectives assistencials fan que en ocasions es prenguin decisions terapèutiques heterogènies, que afecten tant els resultats en salut com l’equitat en la utilització de recursos. L’objectiu de les pautes d’harmonització és l'’optimització de la prestació farmacoterapèutica en termes d’ús racional, de manera que permeti maximitzar el benefici i minimitzar els riscs de l’exposició individual i poblacional als medicaments, d’una manera sostenible pel sistema.El aumento de la esperanza de vida y el consiguiente envejecimiento de la población, junto con los avances médicos que transforman enfermedades agudas en patologías de larga evolución (p. Ej. VIH o cáncer, entre otros), enlazan de forma ineludible con la cronificación. La existencia de diferentes visiones terapéuticas en un entorno multidisciplinar, la falta de comunicación entre profesionales y las diferentes perspectivas asistenciales hacen que en ocasiones se tomen decisiones terapéuticas heterogéneas, que afectan tanto a los resultados en salud como la equidad en la utilización de recursos. El objetivo de las pautas de armonización es la optimización de la prestación farmacoterapéutica en términos de uso racional, de forma que permita maximizar el beneficio y minimizar los riesgos de la exposición individual y poblacional a los medicamentos, de una manera sostenible por el sistema.The increase in life expectancy and the consequent aging of the population, together with the medical advances that transform acute illnesses into long-standing pathologies (eg HIV or cancer, among others), are inextricably linked with the chronification The existence of different therapeutic visions in a multidisciplinary environment, the lack of communication between professionals and the different healthcare perspectives, sometimes lead to heterogeneous therapeutic decisions, which affect both health outcomes and equity in the use of resources. The goal of the harmonization guidelines is to optimize the pharmacotherapeutic benefit in terms of rational use, in order to maximize the benefit and minimize the risks of individual and population exposure to medication in a way sustainable by the system

Changes in Treatment Patterns and Costs for Lung Cancer Have Not Resulted in Relevant Improvements in Survival : A Population-Based Observational Study in Catalonia

Real-world data collect clinical and economic information from daily clinical practice and can support decisions in the context of health evaluation and management. The aim of our retrospective cohort study was to describe the different approaches used for treating lung cancer in Catalonia in 2014 and 2018 and to assess the associated cost and impact on patient survival until December 2021. Treatment patterns for lung cancer changed in younger patients, and all costs of treatment increased significantly. These changes, mainly related to the use of several novel drugs, such as immunotherapy and targeted therapy, were not associated with an increase in the overall patient survival in the period of time under evaluation. Objective: Few published studies have described multidisciplinary therapeutic strategies for lung cancer. This study aims to describe the different approaches used for treating lung cancer in Catalonia in 2014 and 2018 and to assess the associated cost and impact on patient survival. A retrospective observational cohort study using data of patients with lung cancer from health care registries in Catalonia was carried out. We analyzed change in treatment patterns, costs and survival according to the year of treatment initiation (2014 vs. 2018). The Kaplan-Meier method was used to estimate survival, with the follow-up until 2021. From 2014 to 2018, the proportion of patients undergoing surgery increased and treatments for unresectable tumors decreased, mainly in younger patients. Immunotherapy increased by up to 9% by 2018. No differences in patient survival were observed within treatment patterns. The mean cost per patient in the first year of treatment increased from EUR 14,123 (standard deviation [SD] 4327) to EUR 14,550 (SD 3880) in surgical patients, from EUR 4655 (SD 3540) to EUR 5873 (SD 6455) in patients receiving curative radiotherapy and from EUR 4723 (SD 7003) to EUR 6458 (SD 10,116) in those treated for unresectable disease. From 2014 to 2018, surgical approaches increased in younger patients. The mean cost of treating patients increased, especially in pharmaceutical expenditure, mainly related to the use of several biomarker-targeted treatments. While no differences in overall patient survival were observed, it seems reasonable to expect improvements in this outcome in upcoming years as more patients receive innovative treatments

Dealing With Immunoglobulin Shortages : A Rationalization Plan From Evidence-Based and Data Collection

The demand and consumption of immunoglobulins (IgGs) are growing, and there are many difficulties in obtaining supplies. The aim of the study was to analyze the evolution of IgG consumption and cost over a decade, describe the measures implemented for clinical management in the context of regional public health system, and evaluate the initial impact of these measures. We performed a retrospective longitudinal study including patients of all public health systems in Catalonia. First, we analyzed data on consumption and cost of IgGs during a period between 1 January, 2010 and 31 December 2021. Second, we analyzed the impact of a set of regional measures in terms of annual consumption and cost of IgGs. Regional measures were based on rational evidence-based measures and computer registries. We compared the data of year before applying intervention measures (1 January and 31 December 2020) with data of year after applying clinical management interventions (1 January and 31 December 2021). In addition, detailed information on clinical indications of IgG use between 1 January and 31 December 2021 was collected. Overall, in terms of population, the consumption of IgGs (g/1,000 inhabitants) increased from 40.4 in 2010 to 94.6 in 2021. The mean cost per patient increased from €10,930 in 2010 to €15,595 in 2021. After implementing the measures, the mean annual estimated consumption per patient in 2021 was statistically lower than the mean annual estimated consumption per patient in 2020 (mean difference −47 g, 95% CI −62.28 g, −31.72 g, p = 0.03). The mean annual estimated cost per patient in 2021 was also lower than the mean annual estimated cost per patient in 2020 (the mean difference was -€1,492, 95% CI -€2,132.12, -€851.88; p = 0.027). In 2021, according to evidence-based classification, 75.66% treatments were prescribed for a demonstrated therapeutic evidence-based indication, 12.17% for a developed therapeutic evidence-based indication, 4.66% for non-evidence-based therapeutic role indication, and 8.1% could not be classified because of lack of information. The annual consumption and cost of IgGs have grown steadily over the last decade in our regional public health system. After implementing a set of regional measures, the annual consumption of IgGs per patient and annual cost per patient decreased. However, the decrease has occurred in the context of the coronavirus disease 2019 (COVID-19) pandemic, which may have influenced their clinical use. Managing the use of IgGs through a rational plan with strategies including evidence-based and data collection may be useful in a shortage situation with growing demand. Registries play a key role in collection of systematic data to analyze, synthesize, and obtain valuable information for decision support. The action developed needs close monitoring in order to verify its effectiveness

Registry of patients and treatments of hospital medicines in Catalonia (Spain): 10 years of clinical data

Registre de pacients; Medicaments hospitalaris; Dades clíniquesPatient registration; Hospital medicines; Clinical dataRegistro de pacientes; Medicamentos hospitalarios; Datos clínicosDurante el año 2011 se diseñó el actual Registro de Pacientes y Tratamientos MHDA (RPT-MHDA), un único registro específico y centralizado para todos los hospitales del SISCAT, con el objetivo de recoger, de forma sistemática, información sobre utilización, efectividad y seguridad de los MHDA en condiciones de práctica clínica habitual, así como el grado de adherencia a los criterios definidos por el PHF

Polymorphisms in prostaglandin synthase 2/cyclooxygenase 2 (PTGS2/COX2) and risk of colorectal cancer

Inflammation plays a key role in the development of colorectal cancers. We have investigated the relationship between PTGS2 (COX2) polymorphisms and colorectal cancer risk in a hospital based case-control study. We recruited 292 patients with colorectal cancer and 274 controls from new patients admitted to Bellvitge Hospital, Barcelona, Spain, from 1996 to 1998. Subjects responded to a questionnaire on risk factors. Genotypes of the eight more frequent polymorphisms of PTGS2 were determined. Two polymorphisms are located in the promoter sequence, one in the untranslated region of exon 1, one in exon 3, one in intron 5, two in the untranslated region of exon 10, and one downstream of the last polyadenylation (poly-A) signal. Associations were analysed with logistic regression models assuming a dominant effect for rare variants to increase statistical power. An association was detected between colorectal cancer and a polymorphism in the untranslated region of exon 10 of PTGS2, with an odds ratio (OR) of 2.49, 95% confidence interval (CI) of 1.17-5.32, P=0.01. A nearby polymorphism downstream of the last poly-A signal also showed a nonsignificant increase in risk (OR 2.17, 95% CI 0.99-4.78, P=0.05). Analysis of haplotypes confirmed that individuals with these variants were at increased risk of colorectal cancer (OR compared to the most frequent haplotype: 2.17, 95% CI 0.97-4.84, P=0.06) Interactions between PTGS2 genotype and use of nonsteroidal anti-inflammatory drugs and risk of colorectal cancer were also explored

Heterogeneity in pharmacological treatment and outcomes in Crohn's disease patients in Catalonia : a population-based observational study

Heterogeneity in the treatment of a disease is a marker of suboptimal quality of care. The aim of this study is to evaluate the heterogeneity in the treatment used and the outcomes for Crohn's disease (CD) in Catalonia. All patients with CD included in the Catalan Health Surveillance System (data on more than seven million individuals from 2011 to 2017) were identified. The different Catalonian health areas were grouped into 19 district groups (DG). Treatments used rates (systemic corticosteroids, non-biological and biological immunosuppressant) and outcomes rates (hospitalization and surgery) were calculated. The use of systemic corticosteroids presented a decreasing trend over the study period, with an average rate of use in the different territories between 11% and 17%. The use of non-biological immunosuppressant treatment has remained stable, with an average rate of use ranging from 22% to 40% per year depending on the DG. The use of biological immunosuppressant treatment increased with an average rate of use in the different territories ranging from 10 to 23%. Hospitalizations for any reason showed an increasing trend between 2011 and 2017 with an average rate of between 23% and 32% per year depending on the area. Hospitalizations for CD presented a decreasing trend, with an average rate of between 5% and 11% per year. Surgical treatment remained stable over time, rates per year were between 0.5% and 2%. A remarkable geographical heterogeneity in the use of different treatments and in outcomes of CD was observed between different geographical areas of Catalonia