scholarly journals Navigating off‐label and unlicensed medicines use in obstetric and paediatric clinical practice

2019 ◽  
Vol 49 (4) ◽  
pp. 389-395
Author(s):  
Susan G. Gray ◽  
Treasure M. McGuire
Keyword(s):  
Clinical Practice ◽  
Off Label ◽  
Unlicensed Medicines ◽  
Medicines Use

scholarly journals A study of off-label and unlicensed medicines use in neonatal units

2016 ◽  
Vol 62 (2) ◽  
pp. 65-72
Author(s):  
Elizabeta Zisovska ◽  
Blerina Koshi ◽  
Renata Slaveska-Raichki
Keyword(s):  
Off Label Use ◽  
Medicine Use ◽  
Off Label ◽  
Unlicensed Medicines ◽  
Gynecology And Obstetrics ◽  
Life Saving ◽  
Medicines Use ◽  
High Level ◽  
Evidence Based Guidelines ◽  
Label Use

Despite all efforts to improve the on-label use of licensed medicines for neonates, there is still high percentage and demands of offlabel and unlicensed medicines use in neonatal therapy. Therefore, the general objective of this survey is to provide a description of off-label and unlicensed medicines use within the neonatal therapeutic areas in a Department of Neonatology, University Clinic for Gynecology and Obstetrics, in Skopje, Republic of Macedonia. All analyzed prescriptions were given in accordance with the Evidence Based Guidelines applied in the Department. During the three months’ period in 2015, there were given 1595 prescriptions, comprising 3.99 prescriptions per newborn during the hospital stay. Out of them, 532 (33.3%) were on-label medicine uses, 1052 (66%) were off-label medicines, and 11 prescriptions (0.7%) were unlicensed medicines. The results from this study show the high level of off-label medicine use in neonatal therapy. These results present only the “top of the iceberg” and require more comprehensive analysis that will gradually evolve in a National Guideline on off-label use areas in pediatric medicine, especially for critically ill newborns who are prescribed much more life-saving medicines.

scholarly journals Apixaban for Stroke Prevention in Atrial Fibrillation: Why are Event Rates Higher in Clinical Practice than in Randomized Trials?—A Systematic Review

2020 ◽  
Vol 120 (09) ◽  
pp. 1323-1329 ◽  
Author(s):  
Tim A. C. de Vries ◽  
Jack Hirsh ◽  
Ke Xu ◽  
Imaad Mallick ◽  
Vinai C. Bhagirath ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Practice ◽  
Thromboembolic Event ◽  
Meta Analysis ◽  
Patient Characteristics ◽  
Thromboembolic Events ◽  
Off Label Use ◽  
Off Label ◽  
Event Rates ◽  
Label Use

Abstract Background Recent reports suggest an important contribution from frequent off-label use of apixaban 2.5 mg twice daily to the higher rates of thromboembolic events observed in observational studies (OSs) relative to in randomized controlled trials (RCTs), and consequently, advocate against such use in all patients. Objectives To examine factors contributing to the higher thromboembolic event rates, we estimated the prevalence of off-label use in contemporary practice, and compared patient characteristics and rates of stroke/systemic embolism, major bleeding, and mortality by apixaban dose and by study design in a systematic review and meta-analysis. Results and Discussion We identified 18 OSs and 2 RCTs that included 155,228 and 11,928 patients, respectively. Patients in OSs more often received apixaban 2.5 mg twice daily (31.3% vs. 5.1%), were older (mean age 73.8 vs. 69.8 years), and had higher CHA2DS2-VASc scores (mean 3.6 vs. 2.9) versus those in RCTs. We observed a consistent pattern of higher rates of thromboembolic events, bleeding, and mortality in patients treated with 2.5 versus 5 mg twice daily apixaban in both OSs and RCTs. Conclusion The higher risk profiles of patients in OSs versus RCTs, and higher rates of both bleeding and mortality not attributable to thromboembolism in patients treated with apixaban 2.5 versus 5 mg twice daily suggest that differences in patient characteristics are additional important contributors to the higher than expected thromboembolic event rates in clinical practice.

scholarly journals Esketamine for treatment resistant depression: a trick of smoke and mirrors?

2019 ◽  
Vol 29 ◽  
Author(s):  
C. Gastaldon ◽  
D. Papola ◽  
G. Ostuzzi ◽  
C. Barbui
Keyword(s):  
Clinical Practice ◽  
Severe Depression ◽  
Practice Research ◽  
Treatment Resistant Depression ◽  
Treatment Resistant ◽  
Off Label ◽  
The Us ◽  
The Usa ◽  
Resistant Depression ◽  
Spray Formulation

Abstract In March 2019, the US Food and Drug Administration (FDA) approved a nasal spray formulation of esketamine for the treatment of resistant depression in adults. Esketamine is the S-enantiomer of ketamine, an FDA-approved anaesthetic, known to cause dissociation and, occasionally, hallucinations. While ketamine has not been approved for depression in the USA or in any other country, it has been used off-label in cases of severe depression. This commentary critically reviewed the evidence on esketamine submitted to the FDA, aiming to draw implications for clinical practice, research and regulatory science.

Off-Label High-Dose Secukinumab for the Treatment of Moderate-to-Severe Psoriasis

2019 ◽  
Vol 23 (4) ◽  
pp. 391-393 ◽  
Author(s):  
Michelle Phung ◽  
Arvin Ighani ◽  
Jorge R Georgakopoulos ◽  
Ron Vender ◽  
Lyne Giroux ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Dose Escalation ◽  
Case Series ◽  
Severe Psoriasis ◽  
Adult Patients ◽  
High Dose ◽  
Physician Global Assessment ◽  
Upper Respiratory Tract ◽  
Safety And Efficacy ◽  
Off Label

Background: Secukinumab is an anti-IL-17A monoclonal antibody approved for the treatment of moderate-to-severe psoriasis in adult patients. Despite its favourable safety and efficacy profile in clinical trials, some patients in clinical practice fail to respond adequately to the approved maintenance regimen of 300 mg subcutaneous monthly. Some clinicians manage these patients by using off-label high-dose secukinumab regimens, which include shortening the dosing interval to 300 mg every 2 or 3 weeks instead of monthly, or increasing the monthly dose to 450 mg. Objective: This study aims to investigate the safety and efficacy of high-dose secukinumab regimens for the treatment of psoriasis to inform real-world clinical practice. Methods: We performed a retrospective chart review at 5 dermatology clinics for adult patients diagnosed with moderate-to-severe psoriasis treated with an off-label high-dose secukinumab regimen. Efficacy was measured using the Psoriasis Area and Severity Index or a Physician Global Assessment score of 0 or 1 after dose escalation. Adverse events were recorded to assess safety outcomes. Results: Twenty-five patients were included in this case series, and 14 of them achieved efficacy from dose escalation with secukinumab based on our study endpoints. There was 1 case of the common cold and 1 upper respiratory tract infection reported after dose escalation. Conclusion: Our study provides evidence that dose escalation with secukinumab results in clinical benefit and is well tolerated among patients with moderate-to-severe psoriasis who failed to respond adequately to the approved regimen. This work necessitates larger studies to fully characterize the efficacy and long-term safety profile of secukinumab dose escalation.

Efficacy and Safety of SGLT2 Inhibitors in Type 1 Diabetes After the Introduction of an Off-Label Use Protocol for Clinical Practice

2020 ◽  
Vol 22 (3) ◽  
pp. 208-215 ◽  
Author(s):  
Ane Bayona Cebada ◽  
Lía Nattero-Chávez ◽  
Sara Alonso Díaz ◽  
Héctor F. Escobar-Morreale ◽  
Manuel Luque-Ramírez
Keyword(s):  
Type 1 Diabetes ◽  
Clinical Practice ◽  
Sglt2 Inhibitors ◽  
Efficacy And Safety ◽  
Off Label Use ◽  
Off Label ◽  
Label Use

scholarly journals Propranolol in Use for Treatment of Complex Infant Hemangiomas: Literature Review Regarding Current Guidelines for Preassessment and Standards of Care before Initiation of Therapy

2013 ◽  
Vol 2013 ◽  
pp. 1-9 ◽  
Author(s):  
Andreas Fette
Keyword(s):  
Clinical Practice ◽  
Literature Review ◽  
Paradigm Shift ◽  
Standards Of Care ◽  
Current Evidence ◽  
Off Label Use ◽  
Off Label ◽  
Positive Effects ◽  
Initiation Of Therapy ◽  
Current Guidelines

In 2008, the positive effects of propranolol on infantile hemangiomas (IH) have been discovered serendipitously by Léauté-Labrèze and her coworkers. Since then, propranolol has been in use in allday clinical practice worldwide for treatment of IH. It even caused some kind of paradigm shift in the overall management of these lesions, though propranolol is still not FDA approved, respectively, in “off-label” use for this indication in the majority of institutions. Thus, the aim of this communication is to evaluate the literature for current evidence regarding guidelines for preassessment and standards of care before initiation of therapy.

scholarly journals Trastuzumab and Metastatic Breast Cancer: Trastuzumab Use in Australia—Monitoring the Effect of an Expensive Medicine Access Program

2007 ◽  
Vol 25 (24) ◽  
pp. 3688-3693 ◽  
Author(s):  
Sallie-Anne Pearson ◽  
Clare L. Ringland ◽  
Robyn L. Ward
Keyword(s):  
Breast Cancer ◽  
Clinical Trials ◽  
Metastatic Breast Cancer ◽  
Clinical Practice ◽  
Metastatic Breast ◽  
Cardiac Monitoring ◽  
Off Label Use ◽  
Off Label ◽  
Trastuzumab Therapy ◽  
Drug Wastage

Purpose Data from clinical trials are used for drug registration; however, many cancer medicines are ultimately used off-label. This study examines the extent to which the clinical practice use of trastuzumab for the treatment of metastatic breast cancer differs from its use under trial conditions. Methods This study involved all women (N = 1,469) with metastatic breast cancer who received trastuzumab in Australia between December 2001 and March 2005. Given that Australia operates a universal health care system, administrative databases could be examined to determine the duration of therapy, rate of off-label use, compliance with cardiac monitoring, and the extent of drug wastage (volume and cost). Results A total of 433 enrollees (29.5%) received trastuzumab as monotherapy and 1,036 enrollees (70.5%) received the drug in combination with chemotherapy. A total of 321 women (22%) received off-label trastuzumab. The median duration of trastuzumab therapy was longer than that on trial: 5.6 v 3.1 months for enrollees receiving monotherapy and 12.5 v 6.9 months for concomitant chemotherapy. Only 47 (3%) of enrollees received cardiac monitoring before and during trastuzumab therapy. We estimated 24% of trastuzumab dispensed was discarded, at a cost of $21.1 million Australian. Alternative administration schedules and the addition of another vial size potentially reduce wastage to 6% of volume dispensed. Conclusion Debates about the use of expensive cancer medicines should consider postmarketing assessments as well as trial experience. The longer duration of trastuzumab use in clinical practice and the high rates of off-label use provide incentive for new clinical trials. Strategies to improve cardiac monitoring and to minimize drug wastage are issues that require immediate attention.

A perspective on the contribution of animal models to the pharmacological treatment of posttraumatic stress disorder

2017 ◽  
Vol 25 (4) ◽  
pp. 342-347 ◽  
Author(s):  
Valerie Bertaina-Anglade ◽  
Susan M O’Connor ◽  
Emile Andriambeloson
Keyword(s):  
Posttraumatic Stress Disorder ◽  
Clinical Practice ◽  
Animal Models ◽  
Posttraumatic Stress ◽  
Potential Role ◽  
Stress Disorder ◽  
Traumatic Event ◽  
Ptsd Symptoms ◽  
Off Label ◽  
Dsm 5

Objectives: Posttraumatic stress disorder (PTSD) is a prevalent, chronic, disabling disorder that may develop following exposure to a traumatic event. This review summarizes currently used animal models of PTSD and their potential role in the development of better therapeutics. Heterogeneity is one of the main characteristics of PTSD with the consequence that many pharmacological approaches are used to relieve symptoms of PTSD. To address the translational properties of the animal models, we discuss the types of stressors used, the rodent correlates of human PTSD (DSM-5) symptoms, and the efficacy of approved, recommended and off-label drugs used to treat PTSD in ‘PTSD-animals’. Conclusions: Currently available animal models reproduce most PTSD symptoms and are validated by existing therapeutics. However, novel therapeutics are needed for this disorder as not one drug alleviates all symptoms and many have side effects that lead to non-compliance among PTSD patients. The true translational power of animal models of PTSD will only be demonstrated when new therapeutics acting through novel mechanisms become available for clinical practice.

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