Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries.

Objectives: The aim of the study was to assess the reimbursement policy for orphan drugs (ODs) in selected European countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedures and reimbursement decision-making.

Materials And Methods: A list of authorized ODs was extracted from a web-based registry of the European Medicines Agency, including information on active substance, Anatomical Therapeutic Chemical (ATC) classification code, and therapeutic area. A country-based questionnaire survey was conducted between September 2022 and September 2023 among selected experts from 12 European countries.

Predictors of Dietary Supplement Use Among Children Attending Care and Educational Institutions in Krakow, Poland.

Background/objectives: Socioeconomic status and parental lifestyle influence dietary behaviors, including the administration of oral dietary supplements in children. The aim of this study was to evaluate the effects of selected health, dietary, and sociodemographic factors on the use of dietary supplements by children.

Methods: In this cross-sectional observational study, a diagnostic survey based on the computer-assisted web interview method was conducted in November 2022 among 2826 parents whose children attend public educational institutions in Krakow, Poland.

Network meta-analysis of novel targeted therapies for relapsed/refractory chronic lymphocytic leukemia.

Background: The recent development of new antileukemic therapies (anti-CD20 monoclonal antibodies, Bruton tyrosine kinase inhbitors, phosphoinositide 3-kinase inhibitors, and B-cell lymyphoma-2 antagonists) improved the progression-free survival (PFS) compared with selected standard regimens in clinical trials for patients with relapsed/refractory chronic lymphocytic leukemia (CLL). Unfortunately, the relative efficacy of all possible therapeutic options remains unknown because there is no direct evidence for all possible comparisons.

Objectives: We aimed to compare the efficacy and safety of novel agents, chemotherapy, and immunotherapy using a Bayesian network meta-analysis (NMA).

Evidence for external beam radiotherapy in mediastinal Hodgkin and non-Hodgkin lymphoma - systematic review.

Introduction And Objective: Proton beam therapy (PBT) provides the opportunity for a more localized delivery of high energy protons and may reduce the damage to healthy tissues and vital organs. The aim of this review was to assess the effects of proton therapy for patients diagnosed with Hodgkin or non-Hodgkin lymphoma treated with mediastinal irradiation.

Review Methods: A systematic search of EMBASE, MEDLINE via OVID and Cochrane Library was conducted in May 2022 according to PRISMA guidelines to identify relevant data on the efficacy and toxicity of proton beam therapy for patients diagnosed with Hodgkin or non-Hodgkin lymphoma.

Provision of special diets to children in public nurseries and kindergartens in Kraków (Poland).

Background: A specialized diet could be due to an allergy or other medical needs and also religious or cultural reasons. This study aimed to assess the availability and provision of special diets in kindergartens and nurseries financed by the Municipality of Kraków.

Methods: This observational cross-sectional study was based on a diagnostic survey carried out using the Computer-Assisted Web Interview method and addressed to the managers of nurseries ( = 21) and kindergartens ( = 71) and, separately, to the parents of children attending these facilities ( = 1,096).

Clinical aspects of reimbursement policies for orphan drugs in Central and Eastern European countries.

The aim of this study was to characterize the reimbursement policy for orphan drugs (ODs) in Central and Eastern European (CEE) countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedure, selected economic indicators, and the drug type according to indications. A list of authorized medicines with orphan designation and information about active substance, Anatomical Therapeutic Chemical (ATC) classification, and therapeutic area was extracted from the web-based register of the European Medicines Agency (EMA). A country-based questionnaire survey was performed between September 2021 and January 2022 in a group of selected experts from nine CEE countries (an invitation was sent to 11 countries).

Comparative safety of tyrosine kinase inhibitors in the treatment of metastatic renal cell carcinoma: a systematic review and network meta-analysis.

This study aimed to compare the safety profile of tyrosine kinase inhibitors (TKIs) approved for use as monotherapy or combination therapy for the first-line treatment of adult patients with metastatic clear cell renal cell carcinoma (RCC). A systematic review with frequentist network meta-analysis (NMA) was performed according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. We included randomized controlled trials (RCTs) investigating the use of: cabozantinib, pazopanib, sorafenib, sunitinib, tivozanib, cabozantinib + nivolumab, lenvatinib + pembrolizumab, axitinib + avelumab, and axitinib + pembrolizumab in previously untreated adult patients with metastatic clear cell RCC.

Proposition of a New POLA Index to Assess the Immunomodulatory Properties of the Diet and Its Relationship with the Gut Microbiota, Using the Example of the Incidence of COVID-19 in a Group of People without Comorbidities.

A rise in the incidence of infections with severe acute respiratory syndrome coronavirus 2 has sparked the search for protective strategies against the new pathogen. It is known that individual food components can interact with different immune cells, modulating the immune response of the body. The aim of this study was to develop an index assessing the immunomodulatory potential of diet (POLA index) and to test its utility for the prediction of coronavirus disease 2019 (COVID-19) in a group of healthy young people following a traditional or vegetarian diet.

Out-of-pocket expenses of patients with inflammatory bowel disease: a comparison of patient-reported outcomes across 12 European countries.

Background: There is a high variability of out-of-packet patient costs of inflammatory bowel diseases (IBDs), but the issue is not widely recognised. Therefore, we compared patient costs of IBDs between 12 European countries.

Methods: A questionnaire-based study was conducted among adult patients with IBD.

Where do we go next in antidepressant drug discovery? A new generation of antidepressants: a pivotal role of AMPA receptor potentiation and mGlu2/3 receptor antagonism.

Introduction: Major depressive disorder remains a prevalent world-wide health problem. Currently available antidepressant medications take weeks of dosing, do not produce antidepressant response in all patients, and have undesirable ancillary effects.

Areas Covered: The present opinion piece focuses on the major inroads to the creation of new antidepressants.

How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries.

The expansion of orphan drug treatment at increasing prices, together with uncertainties regarding their (cost-)effectiveness raises difficulties for decision-makers to assess these drugs for reimbursement. The present qualitative study aims to gain better insight into current value assessment and appraisal frameworks for orphan drugs, and provides guidance for improvement. 22 European experts from 19 different countries were included in a qualitative survey, followed by in-depth semi-structured interviews.

Socio-Economic Factors Influencing the Use of Dietary Supplements by Schoolchildren from Małopolska Voivodship (Southern Poland).

The use of regular supplementation may be important in alleviating the potential effects of specific nutrient deficiencies. The aim of this cross-sectional study was to assess the socio-economic and lifestyle factors influencing the administration of dietary supplements to schoolchildren from the Małopolskie voivodship. The study was conducted in March-June 2018 on 332 healthy children and adolescents (187 boys, 145 girls) aged 7-14 from the city and municipality of Niepołomice and the city of Kraków.

Indirect Costs of Inflammatory Bowel Diseases: A Comparison of Patient-Reported Outcomes Across 12 European Countries.

Background: National studies report a high variability of indirect costs of inflammatory bowel disease (IBD). In this study, selected aspects of the societal burden of IBDs were compared between 12 European countries.

Methods: A questionnaire-based study among adult patients with IBD was performed.

Opicapone as an add-on to levodopa for reducing end-of-dose motor fluctuations in Parkinson's disease: a systematic review and meta-analysis.

To assess the clinical efficacy and safety profile of opicapone (25 and 50 mg once daily) versus placebo. Levodopa-treated adults with Parkinson's disease. A systematic review and meta-analysis were conducted.

Comparative safety of high-efficacy disease-modifying therapies in relapsing-remitting multiple sclerosis: a systematic review and network meta-analysis.

Objective: This study aimed to compare the safety profile of high-efficacy disease-modifying therapies (DMTs) natalizumab, fingolimod, alemtuzumab, cladribine, ocrelizumab, ofatumumab, ozanimod, as well as a potentially high-efficacy DMT, ponesimod, in adult patients with relapsing-remitting multiple sclerosis (RRMS).

Methods: A systematic review with frequentist network meta-analysis (NMA) was performed according to the Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) guidelines. We included randomized controlled trials (RCTs) with at least 48-week follow-up investigating the use of natalizumab, fingolimod, alemtuzumab, cladribine, ocrelizumab, ofatumumab, ozanimod, and ponesimod, as well as other DMTs, in adult patients with RRMS.

Associations of Nutritional Behavior and Gut Microbiota with the Risk of COVID-19 in Healthy Young Adults in Poland.

The numerous consequences of the coronavirus disease 2019 (COVID-19) pandemic in healthy young people and the lack of clarity as to the long-term disease outcomes have spurred the search for risk factors for SARS-CoV-2 infection. We aimed to evaluate the associations of nutritional behaviors, gut microbiota, and physical activity with the risk of COVID-19 in healthy young nonobese people. Data on body composition, anthropometric measurements, physical activity, dietary intake, and gut microbiota were obtained from 95 adults (mean age, 34.

The Impact of Reimbursement Practices on the Pharmaceutical Market for Off-Patent Medicines in Slovakia.

The aim of this study was to investigate the impact of selected legislative initiatives and their implementation for off-patent medicinal products in Slovakia compared with the rest of the Visegrád Group (V4 countries). We analyzed the development of applications for the reimbursement of generic and biosimilar drugs. Particular emphasis was placed on a) the availability and penetration of biosimilars from 2006 to 2020 in Slovakia, b) a comparative analysis of biosimilars in V4 countries based on the national reimbursement lists of medicinal products for August 2021.

Chest computed tomography versus RT-PCR in early diagnostics of COVID-19 - a systematic review with meta-analysis.

The purpose of this study was to compare the sensitivity and specificity of computed tomography (CT) scans of the chests of patients with the reference reverse-transcription real-time polymerase chain reaction (RT-PCR) in early diagnosis of COVID-19. A systematic review with meta-analysis for numerical outcomes was performed, including 10 studies (6528 patients). High risk of systematic bias (spectrum bias) was demonstrated in all studies, while in several studies research information bias was found to be possible.

Association between health risk factors and dietary flavonoid intake in cohort studies.

The aim of this study was to identify the health risk factors associated with flavonoid intake in cohort studies investigating the association between dietary polyphenols and the risk of cardiovascular disease (CVD). A systematic search of the PubMed and EMBASE databases was performed. Prospective studies with the background characteristics given for categories of flavonoid intake were eligible to inclusion.

Cultural adaptation of the Safety Attitudes Questionnaire - Short Form (SAQ-SF) in Poland.

Background: It is essential to provide safe healthcare in complex, difficult, and quickly changing conditions. The quality of healthcare services directly influences the safety of both the patients and staff. Understanding healthcare staff attitudes toward safety in the healthcare delivery context is foundational for building a culture of safety.