Clinical research is a key factor in healthcare progress, as it contributes toward improving our knowledge on the prevention, etiology, and treatment of different conditions. Healthcare professionals and researchers should be familiar with this specific terminology and procedures of clinical research to understand and be able to evaluate clinical trial results and make decisions using up-to-date recommendations. To do so, they must be familiar with different methodological aspects: from establishing the type of design, the study population, and the groups to be studied, to understanding the randomization and blinding processes. Additionally, when it comes to communicating the results and publishing them, it is also necessary to know how to do it adequately to ensure transparency. This work includes a description of different concepts commonly used in clinical research, particularly in the clinical trials field, in an attempt to compile different topics by providing a brief and accessible overview.

OBJECTIVE: Health-system pharmacists play a crucial role in monitoring the pharmaceutical pipeline to manage formularies, allocate resources, and optimize clinical programs for new therapies. This article aims to support pharmacists by providing periodic updates on new and anticipated novel drug approvals.
CONCLUSIONS: Selected drug approvals anticipated in the 12-month period covering the second quarter of 2024 through the first quarter of 2025 are reviewed. The analysis emphasizes drugs expected to have significant clinical and financial impact in hospitals and clinics, as selected from 52 novel drugs awaiting US Food and Drug Administration approval. New cellular and gene therapies for cancers continued to strengthen the pipeline, in addition to new drugs targeting previously untreatable conditions. Several novel drugs are being developed for rare and ultra-rare diseases such as hemophilia, Niemann-Pick disease type C, hereditary angioedema, and aromatic l-amino acid decarboxylase deficiency.
CONCLUSIONS: The current drug pipeline includes new drugs with various indications for cancers and rare diseases as well as diabetes, acute coronary syndrome, chronic skin disorder, and chronic obstructive pulmonary disease.

For the first time after many decades, many new antidepressants have been approved and many more are under various stages of development and will soon be available in the market. The new drugs present a range of new mechanisms of action with benefits in terms of speed of action, tolerability and range of treatable disorders. Neurosteroids have been recently approved and their rapid benefit may extend from postpartum depression to anxious depression and bipolar depression, dextromethorphan and bupropion combination may prove useful in major depression but also in treatment resistant depression, dextromethadone is a possible augmentation in partial antidepressant response, psychedelic drugs have the potential of long lasting benefits after a single administration, though are still experimental treatments. Botulinum has the same advantage of psychedelics of a single administration and its antidepressant effects may last for weeks or more. Further potentially interesting new antidepressant mechanisms include new drug targets, drug repurposing and genetic or epigenetic manipulations. It is therefore important that clinicians are kept up to date with new evidence so that new evidence can be rapidly translated into clinical practice.

OBJECTIVE: This project aimed to characterize the resources necessary for pharmacists to support the required steps for obtaining and handling investigational drugs outside of a study protocol in the individual patient and intermediate-size population Expanded Access Program (EAP) processes. The second aim was to characterize the types of EAP requests received.
CONCLUSIONS: This retrospective, single-center, observational study was performed by reviewing EAP requests initiated at Duke University Hospital (DUH) between August 1, 2017, and February 11, 2023. The annualized cost of unreimbursed EAP study services was projected to be approximately $196,500 at DUH for 2023. Of the 168 EAP requests submitted after the institutional policy requiring pharmacy and therapeutics (P&T) committee approval was established, 162 (96.4%) were approved by the P&T committee.
CONCLUSIONS: Given the lack of published information on a pharmacist-led workflow related to EAP services, this study sought to share DUH\'s process for managing EAP requests. As there is no mechanism for reimbursement of EAP services, they can be difficult to manage given the labor resources required. Further work is needed to recoup unreimbursed investigational drug service labor costs to ensure compassionate use programs can be implemented in a manner that is financially sustainable for a health system.

UNASSIGNED: Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease associated with inflammation, fibrosis, and destruction of intra- and extrahepatic bile ducts. Despite substantial recent advances in our understanding of PSC, the only proven treatment of PSC is liver transplantation. There is an urgent unmet need to find medical therapies for this disorder.
UNASSIGNED: Multiple drugs are currently under evaluation as therapeutic options for this disease. This article summarizes the literature on the various novel therapeutic options that have been investigated and are currently under development for the treatment of PSC.
UNASSIGNED: In the next decade, more than one drug will likely be approved for the treatment of the disease, and we will be looking at combination therapies for the optimal management of the disease.

OBJECTIVE: Health-system pharmacists play a crucial role in monitoring the pharmaceutical pipeline to manage formularies, allocate resources, and optimize clinical programs for new therapies. This article aims to support pharmacists by providing periodic updates on new and anticipated novel drug approvals.
CONCLUSIONS: Selected drug approvals anticipated in the 12-month period covering the first quarter of 2024 through the fourth quarter of 2024 are reviewed. The analysis emphasizes drugs expected to have significant clinical and financial impact in hospitals and clinics, as selected from 59 novel drugs awaiting US Food and Drug Administration approval. This year\'s pipeline includes recently added drugs with various indications including oncology, infectious diseases, genetic disorders, and rare diseases. New cellular and gene therapies are rapidly evolving and being studied for several rare diseases and cancers.
CONCLUSIONS: More oncology agents, including gene therapies, oral agents, and monoclonal antibodies, are in the pipeline this year. Additional diseases targeted by new novel drugs, including cellular and gene therapies, are hemophilia, nonalcoholic steatohepatitis, Alzheimer\'s disease, and rare diseases such as galactosemia and epidermolysis bullosa.

UNASSIGNED: Irritable bowel syndrome (IBS) has a significant impact on society and quality of life. Current treatments are ineffective, and new investigational drugs are necessary.
UNASSIGNED: Numerous potential therapies are developing, targeting different areas such as cannabinoid signaling, opioid receptors, tachykinin (NK2) receptors, β3-adrenergic receptors, intestinal microbiota, inflammation, and 5HT receptors. Clinical trial evidence has shown that loperamide, eluxadoline, alosetron, ramosetron, bile acid sequestrants, and rifaximin can modulate GI alterations and benefit patients with IBS-D. Among the potential therapies, ibodutant, ibudilast, blautix, BOS-589, solabegron, vibegron, olorinab, ebastine, and ORP-101 have demonstrated possible effects but remain confirmed.
UNASSIGNED: Individuals with IBS-D require cost-effective treatment options that do not impede their productivity or that of their caregivers. This is necessary for consistent healthcare and improved quality of life. Therefore, we should focus on developing new, efficient, and affordable medications for IBS-D. The government, insurers, and society must recognize this need and collaborate to ensure its fulfillment.

UNASSIGNED: Severe asthma patients often remain uncontrolled despite high-intensity therapies. Biological therapies targeting thymic stromal lymphopoietin (TSLP), a key player in asthma pathogenesis, have emerged as potential options. Currently, the only TSLP inhibitor approved for the treatment of severe asthma is the immunoglobulin G (IgG) 2λ anti-TSLP monoclonal antibody (mAb) tezepelumab.
UNASSIGNED: This systematic review assesses the efficacy and safety of investigational TSLP inhibitors across different stages of development for asthma treatment.
UNASSIGNED: TSLP contributes to airway inflammation, making it a pivotal therapeutic target. Ecleralimab, an inhaled antibody fragment antigen binding, shows promising evidence in enhancing efficacy and reducing systemic adverse events. SAR443765, with its NANOBODY® formulation and bispecific inhibition of TSLP and IL-13, offers improved tissue penetration and efficacy. The mAB TQC2731 exhibits high in vitro bioactivity, and the strength of the mAb UPB-101 is to act against the TSLP receptor. Some studies include mild and moderate asthma patients, suggesting the potential for extending biological therapy to non-severe patients. This systematic review highlights the potential of TSLP inhibitors as valuable additions to asthma treatment, even in milder forms of the disease. Future research and cost-reduction efforts are needed to expanding access to these promising therapies.

OBJECTIVE: The clinical trials pharmacists have an essential role in managing the pharmaceutical part of interventional studies. The primary objective of this article was to provide a template for improving trials management for the growing number of studies without increasing personnel resources.
METHODS: A retrospective study was conducted between 2016 and 2020 at the service of pharmacy at Lausanne University Hospital in Switzerland.
RESULTS: The number of clinical trials (in progress) managed at the pharmacy increased from 77 to 115 (+49%) between 2016 and 2020. The majority of these studies were in oncology and were sponsored by industry. Therefore, different changes in routine tasks were decided during the 5 years term to meet the above challenge. These modifications allowed to improve pharmaceutical and administrative management of clinical trials, without increasing personnel resources. The management template was accepted by the sponsors, and no issues were mentioned by national and international audit authorities.
CONCLUSIONS: Changes could be made in the routine practice of the clinical trials pharmacists to improve the management of studies, while the number of trials is increasing every year.

OBJECTIVE: Health-system pharmacists play a crucial role in monitoring the pharmaceutical pipeline to manage formularies, allocate resources, and optimize clinical programs for new therapies. This article aims to support pharmacists by providing updates on new and anticipated novel drug approvals.
CONCLUSIONS: Selected drug approvals anticipated in the 12-month period covering the fourth quarter of 2023 through the third quarter of 2024 are reviewed. The analysis emphasizes drugs selected from 58 novel drugs awaiting FDA approval that are expected to have significant clinical and financial impact in hospitals and clinics. The pipeline includes recently added drugs with various indications, including oncology, infectious diseases such as complicated urinary tract infection and pneumonia, and rare diseases.
CONCLUSIONS: Cellular and gene therapies continue to strengthen the pipeline as potential new treatment options for genetic disorders, rare diseases, and cancer. Additional diseases treated by new agents include pulmonary arterial hypertension, chronic obstructive pulmonary disease, diabetes, and obesity.