New research and findings from open-access journals and the U.S. research agencies.
FDA has a critical role in ensuring the safety of patients in clinical trials studying drugs for the prevention or treatment of COVID-19 that require an investigational new drug (IND) application.
by Jin Ding, Michael M. Hopkins, Paul A. Martin Background The United States (US) and European Union (EU) have long-established orphan drug regulations to incentivise the development of medicines for rare diseases. While the numbers of orphan approvals have risen rapidly, there is increasing discordance in regulatory outcomes between the US and EU. This discordance primarily stems from two sets of cases: US Food and Drug Administration (FDA) orphan approvals not authorised by the European Medicines Agency (EMA), and FDA orphan approvals with EMA authorisation but without orphan designation. We…
Objectives To estimate the cost-effectiveness of immediate septoplasty compared with 6 months of medical management with the option for delayed septoplasty in individuals with nasal obstruction associated with septal deviation. Design Economic evaluation alongside a multicentre, open label, randomised controlled trial. Setting 17 otolaryngology clinics in the UK, recruiting from January 2018 to December 2019. Participants Adults aged≥18 years with symptoms of nasal obstruction associated with septal deviation with at least moderate symptoms of nasal obstruction (score>30 on the Nasal…
Background Preparedness for emerging pathogens can be strengthened by leveraging lessons from COVID-19 therapeutic trials. The COVERAGE Africa trial (NCT04920838, ANRS033, 2021–22), one of the few COVID-19 therapeutics trials conducted in resource-limited settings, sought to identify early treatment strategies for ambulatory patients at-risk for severe disease in Guinea and Burkina Faso. This implementation research, based on secondary data from the Burkina Faso sites, aimed to document the trial’s implementation and to identify the factors that influence it. Methods The PEARLES framework was…
Introduction Bronchopulmonary dysplasia (BPD) remains a major complication among extremely low gestational age (ELGA) infants, with long-term respiratory and neurodevelopmental consequences. Despite advances in neonatal care, effective therapies to prevent BPD are lacking. Mesenchymal stromal cells (MSCs), particularly those derived from umbilical cord (UC-MSC), offer promise due to their pleiotropic effects. Preclinical and early-phase clinical studies have demonstrated safety and potential efficacy of MSC in neonatal lung injury. The Helping Underdeveloped Lungs with Cells (HULC)-2 trial…
Introduction Type 2 diabetes mellitus (T2DM) is a well-established independent risk factor for dementia and is associated with dementia pathophysiology; however, the role of income level remains uninvestigated in this patient population. The aim of this study was to examine the association between income level and incident dementia risk among adults aged ≥50 years with newly diagnosed T2DM. Research design and methods We conducted a retrospective population-based assessment of Korean National Health Insurance Service–Senior Cohort (2002–2019) data. Individuals aged ≥50 years with a new…
Objectives Elective non-emergent surgical wait times have increased across countries such as Canada, straining operating room (OR) resources and affecting patient outcomes and healthcare spending. Manual scheduling systems in Ontario orthopaedic centres create wide variations in wait times, with recent declines in meeting benchmark targets despite increased procedure volumes. Challenges stem from fragmented referral processes, outdated scheduling methods and resource constraints. Artificial intelligence and machine learning (ML) offer potential solutions for optimising scheduling; however…
Introduction The treatment of comorbid post-traumatic stress disorder (PTSD) and alcohol use disorder (AUD) is significantly more challenging than the treatment of either disorder alone. While gold standard evidence-based treatments exist for this comorbidity, clinically significant improvements are only observed in approximately half of clinical trial participants. The use of adjunctive pharmacotherapies, such as 3,4-methylenedioxymethamphetamine (MDMA), may serve to optimise gold standard interventions. The primary aim of the MDMA-assisted PTSD and Alcohol Therapy Trial study is to examine…
Objective To inform the adaptation of an existing tobacco use treatment (TUT) programme into a contextually appropriate service for Lebanon’s safety-net primary care system. Design In-person focus group discussions with an in-depth thematic analysis. Setting Two primary healthcare centres in Beirut, Lebanon Participants 61 individuals who smoked cigarettes (n=36) and waterpipe (n=25). Results The analysis revealed four primary themes: 1) smoking as a coping mechanism for national crisis, 2) lack of perceived fit of the programme with the Lebanese context, 3) differing perceptions of harm and…
Introduction Postoperative nausea and vomiting (PONV) remains one of the most common complications after laparoscopic cholecystectomy, especially in female patients faced with high baseline risk. Although multimodal antiemetic strategies are routinely used in perioperative care, opioid exposure remains an important contributor to PONV. Tegileridine, a novel G protein-biased μ-opioid receptor agonist, may provide effective analgesia with fewer gastrointestinal adverse effects than conventional opioids such as sufentanil. However, evidence on its role in preventing PONV in female patients…
Introduction Pancreaticoduodenectomy (PD) is the preferred surgical approach for resectable periampullary carcinomas. Non-pancreatic periampullary carcinomas (NPPC) demonstrate distinct biological behaviours and are generally less aggressive than pancreatic cancer. Despite over three decades of advancements, the role of laparoscopic PD (LPD) remains debated. Few studies have directly compared LPD and open PD (OPD) while accounting for tumour invasiveness, and the data specifically evaluating LPD vs OPD for NPPC are limited. Randomised controlled trials are necessary to determine whether…
Introduction To preserve the validity and reliability of health instruments like patient-reported outcome measures across linguistic and cultural contexts, translation, cross-cultural adaptation and psychometric validation must be conducted using standardised, rigorous methodologies. Although international standards proposed by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) are available, adherence to these guidelines in Bangla-language instrument studies has not been…
Introduction Gynaecological cancers increasingly affect women of reproductive age, necessitating fertility-sparing treatments that balance oncological safety with reproductive potential. However, the long-term impact of these treatments on survivorship—specifically regarding sexual function, quality of life (QoL) and reproductive outcomes—remains understudied. LIFE-Repro is a prospective multicentre cohort study designed to evaluate these impacts in young female cancer survivors. Methods and analysis Between 2023 and 2027, eleven tertiary referral hospitals in Korea will recruit 200 patients…
Background Fibromyalgia syndrome (FMS) is a chronic condition characterised by the presence of complex multiple symptoms, often accompanied by psychiatric comorbidities and sleep problems. The wide spectrum of symptoms and comorbid problems complicates treatment and management of the ensuing disability and symptoms. Physiotherapy approaches and mind-body practices including yoga and mindfulness are among the non-pharmacological treatment methods commonly used in FMS treatment. While there is initial evidence for these interventions, definitive conclusions about their effectiveness are still…
Introduction Preventive parenting interventions are a cornerstone of child mental health promotion and prevention efforts, with growing evidence supporting their overall effectiveness. However, less is known about which specific intervention formats and core components are essential and most strongly associated with improved outcomes for children and parents. This protocol describes an umbrella review aimed at identifying core components of existing preventive parenting interventions that meet rigorous criteria for methodological quality and demonstrate effectiveness. Methods and analysis We…
Background Obstructive sleep apnoea (OSA) is a common and modifiable risk factor for atrial fibrillation (AF), yet it remains frequently unrecognised and undertreated. Despite guideline recommendations to consider OSA screening, no consensus exists on optimal strategies. The VIR-SAAF (VIRtual national study with home Sleep Apnea investigation in patients with Atrial Fibrillation) trial investigates whether home-based OSA screening and referral for potential treatment improve quality of life, physical activity and AF-related outcomes compared with standard care. Methods and analysis VIR-SAAF is…
Introduction Ultra-processed food (UPF) reduction has become an increasingly important target in chronic disease prevention and management. Although contemporary dietary guidance has moved towards food-based and skill-oriented approaches, it remains unclear how nurse-delivered education explicitly targeting UPF reduction has been conceptualised, implemented and evaluated in community and outpatient settings. This scoping review aims to map the extent, characteristics and knowledge gaps of evidence on real food-based, nurse-delivered education designed to reduce UPF consumption among adults in…
Objectives To translate, cross-culturally adapt and validate the Korean version of the revised Patients’ Attitudes Towards Deprescribing (rPATD) questionnaire for caregivers. Design Cross-sectional validation study. Setting Long-term care facilities (LTCFs) in South Korea. Participants 1000 caregivers of older adult residents in LTCFs. Primary and secondary outcome measures Internal consistency was assessed using Cronbach’s alpha and corrected item–total correlations. An alpha-if-item-deleted analysis was conducted to evaluate individual item contributions. Construct validity was evaluated…
Statistics and Research Methods
Oncology
This cohort study compares hepatitis C care cascade outcome measures using laboratory and pharmacy data to document viral clearance and treatment initiation, respectively, among insured US adults.
This nonrandomized clinical trial evaluates image quality and patient experience associated with at-home transvaginal ultrasonography among premenopausal women.
This secondary analysis of a randomized clinical trial evaluates associations between aerobic exercise and reductions in subthreshold depressive symptoms in adolescents and explores potential neural mechanisms underpinning symptom improvement.
This randomized clinical trial of patients with major depressive disorder evaluates the antidepressant efficacy of left dorsolateral prefrontal cortex transcranial pulse stimulation and its neuromodulatory effects.