To improve the outcomes of research and medicine, government-based international research funding agencies have implemented various types of policies and mechanisms with respect to sex as a biological variable and gender as a sociocultural factor. After the 1990s, the US National Institutes of Health (NIH), the Canadian Institutes of Health Research (CIHR), and the European Commission (EC) began requesting that applicants address sex and gender considerations in grant proposals, and offering resources to help the scientific community integrate sex and gender into biomedical research. Although it is too early to analyze data on the success of all of the policies and mechanisms implemented, here we review the use both of carrots (incentives) and sticks (requirements) developed to motivate researchers and the entire scientific research enterprise to consider sex and gender influences on health and in science. The NIH focused on sex as a biological variable (SABV) aligned with an initiative to enhance reproducibility through rigor and transparency; CIHR instituted a sex- and gender-based analysis (SGBA) policy; and the EC required the integration of the "gender dimension," which incorporates sex, gender, and intersectional analysis into research and innovation. Other global efforts are briefly summarized. Although we are still learning what works, we share lessons learned to improve the integration of sex and gender considerations into research. In conjunction with refining and expanding the policies of funding agencies and mechanisms, private funders/philanthropic groups, editors of peer-reviewed journals, academic institutions, professional organizations, ethics boards, health care systems, and industry also need to make concerted efforts to integrate sex and gender into research, and we all must bridge across silos to promote systemwide solutions throughout the biomedical enterprise. For example, policies that encourage researchers to disaggregate data by sex and gender, the development of tools to better measure gender effects, or policies similar to SABV and/or SGBA adopted by private funders would accelerate progress. Uptake, accountability for, and a critical appraisal of sex and gender throughout the biomedical enterprise will be crucial to achieving the goal of relevant, reproducible, replicable, and responsible science that will lead to better evidence-based, personalized care for all, but especially for women.

The evaluation of child healthcare is not yet widely explored, especially from a cross-country comparison perspective. The routine adoption of measures by national assessment agencies is under-investigated. Though the guiding principles developed at international level call for a child-centric multi-dimensional evaluation of child care, its feasibility is hampered by the availability of robust and harmonized data.

This article describes information resources of toxicological or chemical interest that are available electronically from US federal government agencies. Topics covered include hazard assessment, risk assessment, chemical testing, food safety, hazardous waste, and occupational safety and health.

There are calls for policymakers to make greater use of research when formulating policies. Therefore, it is important that policy organisations have a range of tools and systems to support their staff in using research in their work. The aim of the present study was to measure the extent to which a range of tools and systems to support research use were available within six Australian agencies with a role in health policy, and examine whether this was related to the extent of engagement with, and use of research in policymaking by their staff. The presence of relevant systems and tools was assessed via a structured interview called ORACLe which is conducted with a senior executive from the agency. To measure research use, four policymakers from each agency undertook a structured interview called SAGE, which assesses and scores the extent to which policymakers engaged with (i.e., searched for, appraised, and generated) research, and used research in the development of a specific policy document. The results showed that all agencies had at least a moderate range of tools and systems in place, in particular policy development processes; resources to access and use research (such as journals, databases, libraries, and access to research experts); processes to generate new research; and mechanisms to establish relationships with researchers. Agencies were less likely, however, to provide research training for staff and leaders, or to have evidence-based processes for evaluating existing policies. For the majority of agencies, the availability of tools and systems was related to the extent to which policymakers engaged with, and used research when developing policy documents. However, some agencies did not display this relationship, suggesting that other factors, namely the organisation's culture towards research use, must also be considered.

The use of technology such as computers, tablets, and smartphones to improve access to and the delivery of mental health care (eMental Health care) is growing worldwide. However, despite the rapidly expanding evidence base demonstrating the efficacy of eMental Health care, its implementation in clinical practice and health care systems remains fragmented. To date, no peer-reviewed, key-informant studies have reported on the perspectives of decision-makers concerned with whether and how to implement eMental Health care.

Public health agencies (PHAs) have increasingly incorporated social media into their communication mix during successive pandemics in the 21st century. However, the quality, timing, and accuracy of their health messages have varied significantly, resulting in mixed outcomes for communication, audience engagement, and pandemic management. This study aimed to identify factors influencing the effectiveness of pandemic-related health messages shared by PHAs on social media and to report their impact on public engagement as documented in the literature. A scoping literature review was conducted following a predefined protocol. An electronic search of 7 relevant databases and 5 grey literature repositories yielded 9,714 papers published between January 2003 and November 2022. Seventy-three papers were deemed eligible and selected for review. The results underscored the insufficiency of social media guidance policies for PHAs. Six themes were identified: message source, message topic, message style, message timing, content credibility and reliability, and message recipient profile. These themes encompassed 20 variables that could inform PHAs' social media public health communication during pandemics. Additionally, the findings revealed potential interconnectedness among the variables, and this study concluded by proposing a conceptual model that expands upon existing theoretical foundations for developing and evaluating pandemic-related health messaging.

Information on rare adverse effects is often limited when a medication is initially approved for marketing. Medicines regulators use safety advisories to warn health professionals and consumers about emerging harms. This study aimed to identify characteristics and advice provided in cardiac safety advisories released by regulators in Australia, Canada, the United Kingdom, and the United States. This was a retrospective study of safety advisories about cardiac-related adverse events issued by these four international medicines regulators between 2010 and 2016. A descriptive overview was followed by a more detailed content analysis, focusing on recommended actions for health professionals, including monitoring advice. For the latter, we applied the systematic information for monitoring (SIM) scale to assess adequacy. Over this period, 164 safety advisories about cardiac harms were issued by the four regulators. There were 61 drugs with advisories of cardiac risk, only 9 (14.7%) of which had advisories from all regulators in countries where the drug was approved. The most common adverse events were cardiac arrhythmias (n = 97, 59.1%) and coronary artery disorders (n = 39, 23.8%). The most frequent advice to prescribers was to monitor patients (n = 74, 45.1%), although only 41.2% of these advisories provided detailed advice on how monitoring should occur. We found many differences in the decision to warn and the advice provided. Patient monitoring was most often recommended, but key information such as frequency or thresholds for action was often lacking. Healthcare professionals and consumers need consistent information about rare serious harms so that they can make informed decisions.

 To investigate the practice of post-marketing studies in Germany during a three year period and to evaluate whether these trials meet the aims specified in the German Medicinal Products Act.

A commonly reported problem with the conduct of multicentre randomised controlled trials (RCTs) is that recruitment is often slower or more difficult than expected, with many trials failing to reach their planned sample size within the timescale and funding originally envisaged. The aim of this study was to explore factors that may have been associated with good and poor recruitment in a cohort of multicentre trials funded by two public bodies: the UK Medical Research Council (MRC) and the Health Technology Assessment (HTA) Programme.

Soccer injuries are a recognized problem worldwide. Several injury prevention programs have been confirmed to reduce the number of injuries in female and male players. Unfortunately, there is a lack of data about their cost, burden, and benefit for the health care system. In this paper we aim to systematically review the literature and critically evaluate the economic quality of injury prevention interventions implemented across different populations of soccer players. Web of Science, Medline, SPORTDiscus, Ovid, and other databases were searched from January 2011 through July 2021. Research articles were only selected for analysis if they focused on the cost-effectiveness of injury prevention, were experimental papers written in English, and were published following the peer-review process. Three cluster RCT and one retrospective study met the criteria. Cost data on incremental cost-effectiveness ratios (ICERs) were extracted. The included studies had a good/average quality of economic evaluation. Based on ICERs, injury prevention interventions were cost-effective in three out of the three comparisons. One study did not report the ICER value. However, since economic analyses were reported with varying methodological approaches and results, more data are required to recognize the cost-effectiveness of soccer-specific injury prevention interventions and their benefit for the health care system.

The WHO Model List of Essential Medicines (MLEM) has since 1977 helped prioritize and ensure availability of medicines especially in low- and middle-income countries. The MLEM consists mainly of generic medicines, though recent trends point towards listing expensive on-patent medicines and increasing global support for medicines against non-communicable diseases. However, the implications of such changes for national essential medicines list (NEML) updates for access to essential medicines has received relatively little attention. This study examined how government agencies and other actors in Kenya, Uganda and Tanzania participate in and influence the NEML update process and subsequent availability of prioritized medicines; and the alignment of these processes to WHO guidance. A mixed study design was used, with qualitative documentary review, key informant interviews and thematic data analysis. Results show that NEML updating processes were similar amongst the three countries and aligned to WHO guidelines, albeit conducted irregularly, with tendency to reprioritization during procurement stages, and were not always accompanied by revision of clinical guidelines. Variations were noted in the inclusion of medicines against cancer and hepatitis C, and the utilization of health technology assessment (HTA). For medicines against diseases with high global engagement, such as HIV/AIDS and TB, national stakeholders had more limited inputs in prioritization and funding. Furthermore, national actors were not influenced by the pharmaceutical industry during the NEML update process, nor were any conflicting agendas identified between health, trade and industrial policies. Hence, the study suggests that more attention should be paid to the combination of HTAs and NEMLs, particularly as countries work towards universal health coverage, in addition to heightened awareness of how global disease-specific initiatives may confound national implementation of the NEML. The study concludes with a call to strengthen country-level policy and procedural coherence around the process of prioritizing and ensuring availability of essential medicines.

Competency profiles for occupational clusters within Canada's substance abuse workforce (SAW) define the need for skill and knowledge in evidence-based practice (EBP) across all its members. Members of the Senior Management occupational cluster hold ultimate responsibility for decisions made within addiction services agencies and therefore must possess the highest level of proficiency in EBP. The objective of this study was to assess the knowledge of the principles of EBP, and use of the components of the evidence-based decision making (EBDM) process in members of this occupational cluster from selected addiction services agencies in Nova Scotia.

Randomised controlled trials (RCTs) are the gold standard assessment for health technologies. A key aspect of the design of any clinical trial is the target sample size. However, many publicly-funded trials fail to reach their target sample size. This study seeks to assess the current state of recruitment success and grant extensions in trials funded by the Health Technology Assessment (HTA) program and the UK Medical Research Council (MRC).

The need to optimize drug development and facilitate faster access for patients has ignited discussions around the importance of improving interactions between health technology assessment (HTA) bodies and regulatory agencies. In this study, we conducted a systematic review to examine processes, progress, outcomes, and challenges of harmonization/interaction initiatives between HTA bodies and regulatory agencies. MEDLINE, EMBASE, and the International Pharmaceutical Abstracts database were searched up to 21 October 2019. Searches for gray literature (working papers, commissioned reports, policy documents, etc.) were performed via Google scholar and several institutional websites. An online cross-sectional survey was also conducted among HTA (n = 22) and regulatory agencies (n = 6) across Europe to supplement the systematic review. Overall, we found that while there are areas of divergence, there has been progress over time in narrowing the gap in evidentiary requirements for HTA bodies and regulatory agencies. Most regulatory agencies (4/6; 67%) and half (11/22, 50%) of the HTA bodies reported having a formal link for "collaborating" with the other. Several mechanisms such as early tripartite dialogues, parallel submissions (reviews), adaptive licensing pathways, and postauthorization data generation have been explored as avenues for improving collaboration. A number of pilot initiatives have shown positive effects of these models to reduce the time between regulatory and HTA decisions, which may translate into faster access for patients to life-saving therapies. Thus, future approaches aimed at improving harmonization/interaction between HTA bodies and regulatory agencies should build on these existing models/mechanisms while examining their long-term impacts. Several barriers including legal, organizational, and resource-related factors were also identified, and these need to be addressed to achieve greater alignment in the current regulatory and reimbursement landscape.

Background: Pathogenic variants in the Leucine-rich repeat kinase 2 (LRRK2) gene are the most common known monogenic cause of Parkinson's disease (PD). LRRK2-linked PD is clinically indistinguishable from idiopathic PD and inherited in an autosomal dominant fashion with reduced penetrance and variable expressivity that differ across ethnicities and geographic regions. Objective: To systematically assess clinical signs and symptoms including non-motor features, comorbidities, medication and environmental factors in PD patients, unaffected LRRK2 pathogenic variant carriers, and controls. A further focus is to enable the investigation of modifiers of penetrance and expressivity of LRRK2 pathogenic variants using genetic and environmental data. Methods: Eligible participants are invited for a personal or online examination which comprises completion of a detailed eCRF and collection of blood samples (to obtain DNA, RNA, serum/plasma, immune cells), urine as well as household dust. We plan to enroll 1,000 participants internationally: 300 with LRRK2-linked PD, 200 with LRRK2 pathogenic variants but without PD, 100 PD patients with pathogenic variants in the GBA or PRKN genes, 200 patients with idiopathic PD, and 200 healthy persons without pathogenic variants. Results: The eCRF consists of an investigator-rated (1 h) and a self-rated (1.5 h) part. The first part includes the Movement Disorder Society Unified Parkinson's Disease Rating, Hoehn &Yahr, and Schwab & England Scales, the Brief Smell Identification Test, and Montreal Cognitive Assessment. The self-rating part consists of a PD risk factor, food frequency, autonomic dysfunction, and quality of life questionnaires, the Pittsburgh Sleep Quality Inventory, and the Epworth Sleepiness as well as the Hospital Anxiety and Depression Scales. The first 15 centers have been initiated and the first 150 participants enrolled (as of March 25th, 2021). Conclusions: LIPAD is a large-scale international scientific effort focusing on deep phenotyping of LRRK2-linked PD and healthy pathogenic variant carriers, including the comparison with additional relatively frequent genetic forms of PD, with a future perspective to identify genetic and environmental modifiers of penetrance and expressivity Clinical Trial Registration:ClinicalTrials.gov, NCT04214509.

X-linked hypophosphataemia (XLH) is a rare, hereditary, progressive and lifelong phosphate wasting disorder characterised by pathological elevations in fibroblast growth factor (FGF) 23 concentration and activity; XLH has an incidence of approximately 1 in 20-25,000 individuals. Excess FGF23 activity leads to increased phosphate excretion in the kidneys - mediated by downregulation of renal tubular phosphate transporters - and reduced phosphate absorption in the intestines - due to impaired vitamin D activation. This results in impaired bone growth and mineralisation, short and disproportionate stature, leg bowing, musculoskeletal pain, spontaneous dental abscesses, rickets, and osteomalacia. The spectrum of manifestations differs between paediatric and adult patients. Those involved in the treatment of this condition face many challenges, including a lack of robust natural history and demographic data. This multicentre, international, rare-disease patient registry (XLH Registry) was established to address the paucity of data in XLH and to help inform future clinical practice.

Effective coordination between organizations, agencies and bodies providing or financing health services in humanitarian crises is required to ensure efficiency of services, avoid duplication, and improve equity. The objective of this review was to assess how, during and after humanitarian crises, different mechanisms and models of coordination between organizations, agencies and bodies providing or financing health services compare in terms of access to health services and health outcomes.

The study of international collaborations can help in understanding the benefits of such relationships and aid in developing national financing policies. In this paper, the international collaboration of Brazilian scientists was studied using SciVal® and Incites® database, looking at its effect on the universities, financing agencies and different areas of knowledge and research topic clusters. Cluster and principal component analyses of scientometric data were carried out. While the results confirmed known knowledge that international collaboration increases impact, this study shows that Brazilian researchers are contributing to prominent research topics worldwide, in all areas of knowledge. This finding is contrary to several points of view that identify Brazil as a regional and not an international partner in science. Important also to note the impact of Brazilian authors in international collaboration that is well above the world mean. The collaboration of Brazil with foreign partners brings benefits for both sides, creating the opportunity of Brazilian research access to financing from international agencies. Increases in measures of impact are also seen for both sides. Foreign partners likewise benefit from higher impact factors in the same topic cluster, when collaborating with Brazilian partners. Publishing open access in high impact journals is fundamental for maintaining Brazilian science at the forefront.

Omics technologies, enabling the measurements of genes (genomics), mRNA (transcriptomics), proteins (proteomics) and metabolites (metabolomics), are valuable tools for personalized decision-making. We aimed to identify the existing value assessment frameworks used by health technology assessment (HTA) doers for the evaluation of omics technologies through a systematic review.

No abstract available