A Risk-Based Assessment for Determining the Pharmacokinetic Comparability Requirements of Biologic-Device Combination Products Administered by Subcutaneous Injection.

The development of new large molecule drug therapies along with the innovation of biologic-device combination products such as prefilled syringes, autoinjectors and pen injectors have significantly impacted the treatment of new diseases and has improved the process of administering parenteral medicines. To support the regulatory approval of a new biologic-device combination products or subsequent chemistry, manufacturing and control changes impacting a combination product, sponsor companies must thoroughly assess the potential impact to product quality, safety and efficacy. In this report, a risk-based process to determine the potential impact to product quality, safety, and efficacy as well as corresponding regulatory actions supporting a chemistry, manufacturing and control change is presented.

Nonacog beta pegol prophylaxis in children with hemophilia B: safety, efficacy, and neurodevelopmental outcomes for up to 8 years.

Background: Nonacog beta pegol (N9-GP) is an extended half-life PEGylated factor (F)IX product with established efficacy and short-term safety in persons with hemophilia B (HB). Long-term safety has been evaluated for polyethylene glycol exposure but not N9-GP.

Objectives: To assess safety, neurodevelopmental, and efficacy outcomes of children with HB receiving N9-GP prophylaxis across 2 open-label, single-arm, phase 3 studies: paradigm5 (previously treated patients [PTPs]) and paradigm6 (previously untreated patients [PUPs]) in this interim analysis.

Life situation of older people living with severe mental illness - A scoping review.

An increasing number of older people with severe mental illness (SMI) poses additional challenges to healthcare and social services. This scoping review aimed to investigate the life situation of older people living with severe mental illness and identify the research gaps in the existing literature. The current review followed the methodological framework for conducting scoping reviews by Arksey and O'Malley, consisting of five main stages: (1) identifying the research question (2) identifying relevant studies, (3) selecting the studies, (4) charting the data, and (5) collating, summarising, and reporting the results.

Acetaminophen treatment in children and adults with spinal muscular atrophy: a lower tolerance and higher risk of hepatotoxicity.

Acute liver failure has been reported sporadically in patients with spinal muscular atrophy (SMA) and other neuromuscular disorders with low skeletal muscle mass receiving recommended dosages of acetaminophen. It is suggested that low skeletal muscle mass may add to the risk of toxicity. We aimed to describe the pharmacokinetics and safety of acetaminophen in patients with SMA.

Pharmacokinetics and safety of prolonged paracetamol treatment in neonates: An interventional cohort study.

Aims: To investigate the pharmacokinetics and safety of prolonged paracetamol use (>72 h) for neonatal pain.

Methods: Neonates were included if they received paracetamol orally or intravenously for pain treatment. A total of 126 samples were collected.