Reform of economic evaluation of medicines in Spain: proposals from the Advisory Committee for Pharmaceutical Financing.

This paper describes the reforms recommended by the Advisory Committee on the Financing of Pharmaceuticals (CAPF) for the National Health System (NHS) of Spain from 2019 to 2024 for the drug pricing and reimbursement process, to integrate economic evaluations and improve efficiency and sustainability. The CAPF has proposed a three-phase reform of the economic evaluation (EE) and budget impact analysis (BIA) processes. The first phase involves the mandatory submission of EE and BIA by applicants for new drugs.

Everolimus Through Plasmatic Concentrations in Cancer Patients: Prospective Longitudinal Observational Multicentric Study (DIANA-1 Project).

Everolimus, an oral inhibitor of the mammalian target of rapamycin (mTOR), is actually used to prevent organ transplant rejection and treat metastatic breast, renal, and neuroendocrine cancers. Despite significant pharmacokinetic variability among patients, routine therapeutic drug monitoring (TDM) is not commonly used in oncology. The aim of this multicenter, prospective observational cohort study is to assess the prevalence of everolimus minimum concentration at a steady state (Cminss) falling outside the therapeutic range (10-26.

Real-world treatment outcomes of immune checkpoint inhibitors used off-label in oncology: A comprehensive cancer institution experience.

Off-label use (OLU) is quite common in oncology due to the complexity of cancer and the time-consuming regulatory process. However, outcomes of OLU in cancer treatment remain unclear. This study aimed to evaluate the overall survival (OS), event-free survival (EFS), duration of treatment (DOT), and reason for treatment discontinuation in patients receiving immune checkpoint inhibitors (ICI) as OLU for solid tumors from 2011 to 2020.

Real-world treatment outcomes of medicines used in special situations (off-label and compassionate use) in oncology and hematology: A retrospective study from a comprehensive cancer institution.

Purpose: Medicines in special situations (MSS) refer to off-label or to unlicensed drugs under investigation (compassionate use). Our objectives were to evaluate characteristics and to estimate overall survival (OS), event-free survival (EFS), and the duration of treatment (DT) of MSS used for cancer treatment at a multicentre comprehensive cancer institution.

Methods: Retrospective cohort study on adult cancer patients for whom an MSS treatment was requested (January 2011-December 2020).

Clinical practice guidelines for the optimization of hemato-oncological care: the ICOPraxis.

Introduction: The Catalan Institute of Oncology (ICO) is a Comprehensive Cancer Center (CCC) responsible for the oncological care of 46% of the Catalan population.

Objective: Given the increasing and ongoing approval of onco-hematological treatments, the professionals at the ICO decided to have clinical practice guidelines (called ICOPraxis) based on available evidence. In this report, we intend to share how the ICOPraxis has developed and what its impact has been in the 14 years it has been running.

Implementing Risk-Sharing Arrangements for Innovative Medicines: The Experience in Catalonia (Spain).

Objectives: Publications assessing health and economic outcomes of risk-sharing arrangements (RSAs) are limited. Better knowledge of these outcomes would shed light on the pertinence of such arrangements, informing design improvements for the future. The aim of the study is to describe the different types of RSAs implemented in Catalonia and their health and economic outcomes.

COVID-19 among workers of a comprehensive cancer centre between first and second epidemic waves (2020): a seroprevalence study in Catalonia, Spain.

Objectives: Patients with cancer are at higher risk for severe COVID-19 infection. COVID-19 surveillance of workers in oncological centres is crucial to assess infection burden and prevent transmission. We estimate the SARS-CoV-2 seroprevalence among healthcare workers (HCWs) of a comprehensive cancer centre in Catalonia, Spain, and analyse its association with sociodemographic characteristics, exposure factors and behaviours.

Clinical Presentations and Outcomes Related to Tuberculosis in Children Younger Than 2 Years of Age in Catalonia.

Children younger than 2 years have an increased risk of complications associated with tuberculosis (TB) due to the immaturity of the innate and adaptive immune response. We aimed to identify TB clinical presentations and outcomes as well as risk factors for complications in this age group. Multicenter, retrospective, cross-sectional study of TB cases in children aged <2 years in Catalonia (2005-2013).

Financial consequences of a payment-by-results scheme in Catalonia: gefitinib in advanced EGFR-mutation positive non-small-cell lung cancer.

Background: In 2011 the first payment-by-results (PbR) scheme in Catalonia was signed between the Catalan Institute of Oncology (ICO), the Catalan Health Service, and AstraZeneca (AZ) for the introduction of gefitinib in the treatment of advanced EGFR-mutation positive non-small-cell lung cancer. The PbR scheme includes two evaluation points: at week 8, responses, stabilization and progression were evaluated, and at week 16 stabilization was confirmed. AZ was to reimburse the total treatment cost of patients that failed treatment, defined as progression at weeks 8 or 16.

Colorectal Cancer Screening Programme in Spain: Results of Key Performance Indicators After Five Rounds (2000-2012).

Effective quality assurance is essential in any screening programme. This article provides a unique insight into key quality indicators of five rounds of the first population-based colorectal cancer screening programme implemented in Spain (2000-2012), providing the results according to the type of screening (prevalent or first screen and incident or subsequent screen) and test (guaiac or immunochemical). The total crude participation rate increased from 17.

Cost-effectiveness of primary antifungal prophylaxis with posaconazole versus itraconazole in allogeneic hematopoietic stem cell transplantation.

Purpose: To evaluate the cost-effectiveness of posaconazole vs itraconazole in the prevention of invasive fungal infections (IFIs) in recipients of allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Methods: Total hospital-based costs from initial admission for allo-HSCT until day 100 after transplantation were evaluated for 49 patients in whom the clinical efficacy of antifungal prophylaxis with posaconazole vs itraconazole had been previously analyzed and reported. Clinical and economic data were used to determine the incremental costs per IFI avoided and per life-year gained for posaconazole compared with itraconazole.

A phase I clinical and pharmacokinetic study (LIPOTEC - GP PHARM/DOXO 01) of a new liposomal doxorubicin given as 3-week schedule in patients with solid tumors.

As a dose-finding phase I study of a new liposomal formulation of doxorubicin (LipD), patients (n = 39; median age: 60 years; range, 41-75; median ECOG performance status, 1; range, 0-2) with refractory cancer had a starting dose of LipD administered at 30 mg/m(2) as a 1-hour intravenous infusion. Cycle duration was 21 days. At the recommended dose (RD), patients received a first cycle of nonliposomal doxorubicin (non-LipD) to evaluate intrapatient pharmacokinetic differences between non-LipD and LipD.

Critical appraisal of temozolomide formulations in the treatment of primary brain tumors: patient considerations.

Chemotherapy is assuming an increasingly important role in the treatment of malignant gliomas, of which temozolomide (TMZ) is a key part. TMZ belongs to a class of second-generation imidazotetrazinone prodrugs that exhibit linear pharmacokinetics and do not require hepatic metabolism for activation to the active metabolite. New intravenous (iv) TMZ formulations have recently been approved based on studies of bioequivalence between iv and oral TMZ.

[A bibliometric analysis of the Farmacia Hospitalaria journal (2001-2006)].

Objective: To carry out a bibliometric analysis of the Farmacia Hospitalaria journal from 2001 to 2006.

Method: A retrospective analysis of all of the articles published in Farmacia Hospitalaria from 2001-2006 was performed and the main bibliometric indicators for production, circulation, distribution and sales were calculated.

Results: 416 articles by 1,515 authors were analysed.

[Impact on budget of new drugs for colorectal cancer treatment].

Background And Objective: To assess the impact on the medicines budget of the introduction of new treatments in colorectal cancer, as monoclonal antibodies cetuximab and bevacizumab and oxaliplatin in the adjuvant setting, for the Catalan health public system in 2006.

Method: In advanced stages of the disease, the medicines budget impact of the introduction of cetuximab and bevacizumab in relation to the standard treatment (FOLFIRI and FOLFOX regimes) was evaluated. In adjuvant treatment stage II-III, the medicines budget impact of the utilization of FOLFOX regime compared to the combination of fluorouracil and folinic acid was evaluated.

Description of controlled trials published in Methods and Findings, 1979-2002.

It is thought that the controlled trial (CT) is the most adequate research method to assess a therapeutic intervention in terms of efficacy, and it also constitutes the basis for the development of systematic reviews on health interventions. To identify and obtain the majority of published CTs is not an easy task, mainly because of limitations concerning the currently available electronic sources. The aim of the present work was to identify, describe, and assess the quality of CTs published in the journal Methods and Findings in Experimental and Clinical Pharmacology (M&F).

Absence of veno-occlussive disease in a cohort of multiple myeloma patients undergoing autologous stem cell transplantation with targeted busulfan dosage.

Background: Plasma concentrations of oral busulfan (BU) were measured in multiple myeloma (MM) patients undergoing autologous peripheral blood stem cell transplantation (ASCT) with a double alkylating conditioning protocol in order to individualise doses of BU based on individual pharmacokinetic parameters and to reduce toxicities related to BU exposure.

Patients And Methods: Forty-four consecutive patients with MM participating in the co-operative Spanish protocol were prospectively evaluated. Conditioning regimen prior to autologous infusion consisted of BU followed by melphalan.

Reduced-intensity conditioning allogeneic blood stem cell transplantation with fludarabine and oral busulfan with or without pharmacokinetically targeted busulfan dosing in patients with myeloid leukemia ineligible for conventional conditioning.

We prospectively compared outcomes after a fludarabine (Flu) plus oral busulfan (Bu)-containing reduced-intensity conditioning regimen (150 mg/m2 Flu and 10 mg/kg oral Bu), with (n = 32; Flu- T Bu group) or without (n = 30; Flu-Bu group) therapeutic dose monitoring and dose adjustment of Bu. All patients received peripheral blood stem cells from a genoidentical sibling, and study cohorts had similar patient characteristics. Dose adjustments of Bu were required in 20 (63%) patients in the Flu- T Bu group (median final dose, 8.

Mouth-washings with recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF) do not improve grade III-IV oropharyngeal mucositis (OM) in patients with hematological malignancies undergoing stem cell transplantation. Results of a randomized double-blind placebo-controlled study.

We investigated whether daily oral washings with recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) solution improved grade III-IV oropharyngeal mucositis (OM) in patients with hematological malignancies undergoing stem cell transplantation. Forty-one consecutive patients (21 males and 20 females, median age (range) 44 (16-69) years) were prospectively randomized to perform daily mouth-washes with either a 400 microg rhGM- CSF (Molgramostin, Schering-Plough) solution (group A, n = 18) or with a saline solution (group B, n = 23). Primary end-points were the intensity of OM, night rest quality and characteristics of food intake.

[Hematopoietic allogeneic transplantation with attenuated conditioning regimen on an outpatient basis. First experience in Spain].

Background: Allogeneic hematopoietic transplantation using attenuated conditioning regimens seems promising. This procedure associates to relatively low morbidity and mortality. In consequence, an outpatient management of this transplantation modality may be considered, even in elderly patients.