Feasibility of disease terminology systems for mapping orphan conditions and therapeutic indications of designated orphan medicines in the European Union.

Background: In the European Union, rare diseases are defined as diseases that affect maximum 5 in 10,000 citizens. These diseases are typically associated with a high unmet medical need. To stimulate development and authorisation of medicines for rare diseases ('orphan conditions'), the European Commission (EC) can grant orphan designations.

Uptake of orphan drugs in the WHO essential medicines lists.

Objective: We evaluated the uptake of medicines licensed as orphan drugs by the United States Food and Drug Administration (FDA) or European Medicines Agency (EMA) into the and the from 1977 to 2021.

Methods: We collated and analysed data on drug characteristics, reasons for adding or rejecting medicines, and time between regulatory approval and inclusion in the lists. We compared trends in listing orphan drugs before and after revisions to the inclusion criteria of the essential medicines lists in 2001, as well as differences in trends for listing orphan and non-orphan drugs, respectively.

Mapping the risk of infections in patients with multiple sclerosis: A multi-database study in the United Kingdom Clinical Practice Research Datalink GOLD and Aurum.

Background: People with multiple sclerosis (pwMS) have an increased risk of infections; risk factors include underlying disease, physical impairment and use of some disease-modifying treatments.

Objective: To quantify changes in population-level infection rates among pwMS and compare these to the general population and people with rheumatoid arthritis (pwRA), and identify patient characteristics predictive of infections after MS diagnosis.

Methods: We conducted a multi-database study using data on 23,226 people with MS diagnosis from the UK Clinical Practice Research Datalink Aurum and GOLD (January 2000-December 2020).

Access to oxytocin and misoprostol for management of postpartum haemorrhage in Kenya, Uganda and Zambia: a cross-sectional assessment of availability, prices and affordability.

Objective: To assess access (availability and affordability) to oxytocin and misoprostol at health facilities in Kenya, Uganda and Zambia to improve prevention and management of postpartum haemorrhage (PPH).

Design: The assessment was undertaken using data from Health Action International (HAI) research on sexual and reproductive health commodities based on a cross-sectional design adapted from the standardised WHO/HAI methodology.

Setting: Data were collected from 376 health facilities in in Kenya, Uganda and Zambia in July and August 2017.

Application of Managed Entry Agreements for Innovative Therapies in Different Settings and Combinations: A Feasibility Analysis.

The reimbursement of expensive, innovative therapies poses a challenge to healthcare systems. This study investigated the feasibility of managed entry agreements (MEAs) for innovative therapies in different settings and combinations. First, a systematic literature review included studies describing used or conceptual agreements between payers and manufacturers (i.

Regulating advanced therapy medicinal products through the Hospital Exemption: an analysis of regulatory approaches in nine EU countries.

To study regulatory approaches for the implementation and utilization of the Hospital Exemption (HE) in nine EU countries. Using public regulatory documentation and interviews with authorities we characterized the national implementation process of the HE, including national implementation characteristics and two outcomes: national licensing provisions and the amount of license holders. National licensing provisions vary substantially among selected countries as a result of different regulatory considerations that relate to unmet medical needs, benefit/risk balance, and innovation.

The Role of Regulator-Imposed Post-Approval Studies in Health Technology Assessments for Conditionally Approved Drugs.

Background: The European Medicines Agency (EMA) aims to resolve uncertainties associated with conditionally approved drugs by imposing post-approval studies. Results from these studies may be relevant for health technology assessment (HTA) organizations. This study investigated the role of regulator-imposed post-approval studies within HTA.

Are Further Interventions Needed to Prevent and Manage Hospital-Acquired Hyponatraemia? A Nationwide Cross-Sectional Survey of IV Fluid Prescribing Practices.

Background: Hyponatraemia is associated with increased morbidity, increased mortality and is frequently hospital-acquired due to inappropriate administration of hypotonic fluids. Despite several attempts to minimise the risk, knowledge is lacking as to whether inappropriate prescribing practice continues to be a concern.

Methods: A cross-sectional survey was performed in Danish emergency department physicians in spring 2019.

Publication rates and reported results in a cohort of gene- and cell-based therapy trials.

We investigated publication rates and reported results for gene- and cell-based therapy trials. In a cohort of Institutional Review Board (IRB)-authorized trials during 2007-2017 in the Netherlands (n = 105), we examine publication rates and reported results in scientific papers and conference abstracts as well as associations with the occurrence of trial characteristics. The publication rate for scientific papers was 27% and 17% for conference abstracts (median survival time: 1050 days).

Identification of potentially inappropriate medications with risk of major adverse cardiac and cerebrovascular events among elderly patients in ambulatory setting and long-term care facilities.

Purpose: Cardiovascular diseases (CVDs) are extremely common among the elderly, but information on the use of potentially inappropriate medications (PIMs) with cardiovascular risk is scarce. We aimed to determine the prevalence of PIMs with risk of cardiac and cerebrovascular adverse events (CCVAEs), including major adverse cardiac and cerebrovascular events (MACCE).

Patients And Methods: A cross-sectional study was performed using a convenience sample from four long-term care facilities and one community pharmacy in Portugal.

Learning from patients: Identifying design features of medicines that cause medication use problems.

Usability is a key factor in ensuring safe and efficacious use of medicines. However, several studies showed that people experience a variety of problems using their medicines. The purpose of this study was to identify design features of oral medicines that cause use problems among older patients in daily practice.

Adverse events and patients' perceived health-related quality of life at the end of multidrug-resistant tuberculosis treatment in Namibia.

Purpose: The health-related quality of life (HRQoL) of patients completing multidrug-resistant tuberculosis (MDR-TB) treatment in Namibia and whether the occurrence of adverse events influenced patients' rating of their HRQoL was evaluated.

Patients And Methods: A cross-sectional analytic survey of patients completing or who recently completed MDR-TB treatment was conducted. The patients rated their HRQoL using the simplified Short Form-™ (SF-8) questionnaire consisting of eight Likert-type questions.

National medicines policies - a review of the evolution and development processes.

Objectives: Continuous provision of appropriate medicines of assured quality, in adequate quantities, and at reasonable prices is a concern for all national governments. A national medicines policy (NMP) developed in a collaborative fashion identifies strategies needed to meet these objectives and provides a comprehensive framework to develop all components of a national pharmaceutical sector. To meet the health needs of the population, there is a general need for medicine policies based on universal principles, but nevertheless adapted to the national situation.

Testing bias in clinical databases: methodological considerations.

Background: Laboratory testing in clinical practice is never a random process. In this study we evaluated testing bias for neutrophil counts in clinical practice by using results from requested and non-requested hematological blood tests.

Methods: This study was conducted using data from the Utrecht Patient Oriented Database.

Medication changes prior to hospitalization for obstructive lung disease: a case-crossover study.

Background: Hospitalizations have always been seen as a solid outcome parameter in pharmacoepidemiology. However, the period leading to hospitalization and prehospital management of the patient are equally important.

Objective: To evaluate medication changes in the period prior to hospitalization for obstructive lung disease and to quantify the association between medication use and the risk of hospitalization.

Towards equitable access to medicines for the rural poor: analyses of insurance claims reveal rural pharmacy initiative triggers price competition in Kyrgyzstan.

Background: A rural pharmacy initiative (RPI) designed to increase access to medicines in rural Kyrgyzstan created a network of 12 pharmacies using a revolving drug fund mechanism in 12 villages where no pharmacies previously existed. The objective of this study was to determine if the establishment of the RPI resulted in the unforeseen benefit of triggering medicine price competition in pre-existing (non-RPI) private pharmacies located in the region.

Methods: We conducted descriptive and multivariate analyses on medicine insurance claims data from Kyrgyzstan's Mandatory Health Insurance Fund for the Jumgal District of Naryn Province from October 2003 to December 2007.

Difference in risks of allergic reaction to sulfonamide drugs based on chemical structure.

Background: The chemical structure of sulfonamide antibiotics and sulfonamide nonantibiotics can affect the potential for adverse reactions.

Objective: To assess whether differences in chemical structure of the various sulfonamide drugs influence the risk of allergic events.

Methods: A case-control study was conducted among patients with diabetes mellitus (DM) using data from the General Practice Research Database.