scholarly journals Think Stop before Going 'Off-Label'

2004 ◽  
Vol 18 (5) ◽  
pp. 287-288
Author(s):  
Gerald Y Minuk
Keyword(s):  
Clinical Trials ◽  
Review Process ◽  
Medical Condition ◽  
Regulatory Body ◽  
Extensive Review ◽  
Efficacy Data ◽  
Safety And Efficacy ◽  
Off Label ◽  
Specific Medical Condition

Only after careful and extensive review of all available safety and efficacy data derived from preclinical and clinical trials will a regulatory body approve the licensing of a drug for a specific medical condition ('indication'). Experience with thalidomide, fialuridine and troglitazone, to name a few, reveal that despite these efforts, the process is not infallible. What then can we expect when a drug is used for a medical condition in which no such review process has been undertaken ('offlabel' use of the drug)?

scholarly journals Technology for Measuring and Monitoring Treatment Compliance Remotely

2021 ◽  
pp. 1-5
Author(s):  
Richard H. Christie ◽  
Anzar Abbas ◽  
Vidya Koesmahargyo
Keyword(s):  
Clinical Trials ◽  
Clinical Study ◽  
Neurological Disorders ◽  
Treatment Compliance ◽  
Quality Of Data ◽  
Efficacy Data ◽  
Safety And Efficacy ◽  
Study Sites ◽  
Monitoring Treatment

Medication non-adherence during clinical trials is an ongoing challenge that can result in insufficient safety and efficacy data. For patients with Parkinson’s disease and other neurological disorders, symptomatology such as forgetfulness compounds traditional obstacles to adherence. Today, sponsors and clinical study sites can call upon various technology tools that improve adherence by monitoring and confirming dosage in near real-time. These tools have the potential to improve the quality of data gleaned from these studies.

Safety and Efficacy of Sub-Antimicrobial-Dose Doxycycline Therapy in Patients with Adult Periodontitis

1998 ◽  
Vol 12 (1) ◽  
pp. 27-31 ◽  
Author(s):  
Sebastian Ciancio ◽  
Robert Ashley
Keyword(s):  
Clinical Trials ◽  
Randomized Clinical Trials ◽  
Dosing Regimen ◽  
Periodontal Health ◽  
Efficacy Data ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Double Blind Placebo ◽  
Optimal Dosing

The objectives of the studies presented here were to assess the safety and efficacy of the adjunctive administration of sub-antimicrobial-dose doxycycline (SDD) for the treatment of adult periodontitis and to confirm the optimal dosing regimen. The studies summarized included four double-blind, placebo-controlled, randomized clinical trials, conducted over a period of 9 to 12 months. Analysis of efficacy data demonstrated that adjunctive SDD treatment resulted in: (1) increases in clinical attachment levels; (2) decreases in probing pocket depths; and (3) reductions in bleeding on probing in patients with adult periodontitis. There were no significant adverse events or unwanted long-term antimicrobial effects associated with orally administered SDD. The results of these clinical trials indicate that the adjunctive use of SDD 20 mg BID is an effective and well-tolerated regimen which can significantly improve several indices of periodontal health.

scholarly journals Treating patients with compounds or biological agents that pass the safety-test

2016 ◽  
Author(s):  
Jianyi Zhang
Keyword(s):  
Clinical Trials ◽  
Clinical Setting ◽  
Patient Population ◽  
Review Process ◽  
Biological Agents ◽  
Controlled Clinical Trials ◽  
Safety And Efficacy ◽  
Medical Disorders ◽  
Safety Test ◽  
The U.S

There are numerous medical disorders without effective treatments. It is possible that many of un-treatable conditions need more than one compound co-operatively for improvement or cure. Approval of drugs in the U.S. is governed by a rigorous review process by the Food and Drug Administration (FDA) regulation. A sponsor must demonstrate the drug’s safety and efficacy in well controlled clinical trials in the proper patient population for which it is indicated. Development of a new compound entity (NCE) is a very costly and time-consuming process; it takes roughly 15 years and 1.5 billion dollars on average. The high cost of the process makes the development of a new drug with multiple NCEs almost impossible. There are many compounds or biological agents which pass the safety test, but fail in the efficacy test. Those compounds might be critical for treatment of these medical conditions. The author suggests that compounds or biological agents, which pass the safety test, but the fail in efficacy test, should be allowed to use in clinical setting. The safety of those compounds is comparable with any other drugs approved by FDA. Benefits would heavily outweigh risks for patients treated by those compounds. The same principle should also be applied to biological agents, such as vaccines against HIV infection, cancers, etc.

scholarly journals Treating patients with compounds or biological agents that pass the safety-test

2016 ◽  
Author(s):  
Jianyi Zhang
Keyword(s):  
Clinical Trials ◽  
Clinical Setting ◽  
Patient Population ◽  
Review Process ◽  
Biological Agents ◽  
Controlled Clinical Trials ◽  
Safety And Efficacy ◽  
Medical Disorders ◽  
Safety Test ◽  
The U.S

There are numerous medical disorders without effective treatments. It is possible that many of un-treatable conditions need more than one compound co-operatively for improvement or cure. Approval of drugs in the U.S. is governed by a rigorous review process by the Food and Drug Administration (FDA) regulation. A sponsor must demonstrate the drug’s safety and efficacy in well controlled clinical trials in the proper patient population for which it is indicated. Development of a new compound entity (NCE) is a very costly and time-consuming process; it takes roughly 15 years and 1.5 billion dollars on average. The high cost of the process makes the development of a new drug with multiple NCEs almost impossible. There are many compounds or biological agents which pass the safety test, but fail in the efficacy test. Those compounds might be critical for treatment of these medical conditions. The author suggests that compounds or biological agents, which pass the safety test, but the fail in efficacy test, should be allowed to use in clinical setting. The safety of those compounds is comparable with any other drugs approved by FDA. Benefits would heavily outweigh risks for patients treated by those compounds. The same principle should also be applied to biological agents, such as vaccines against HIV infection, cancers, etc.

scholarly journals Vitamin C in the Treatment of COVID-19

Nutrients ◽  
2021 ◽  
Vol 13 (4) ◽  
pp. 1172
Author(s):  
Gregorio Paolo Milani ◽  
Marina Macchi ◽  
Anat Guz-Mark
Keyword(s):  
Clinical Trials ◽  
Vitamin C ◽  
Respiratory Infections ◽  
Medical Condition ◽  
Case Reports ◽  
Case Series ◽  
Theoretical Background ◽  
Current Evidence ◽  
Vitamin C Supplementation ◽  
Essential Nutrient

Vitamin C is an essential nutrient that serves as antioxidant and plays a major role as co-factor and modulator of various pathways of the immune system. Its therapeutic effect during infections has been a matter of debate, with conflicting results in studies of respiratory infections and in critically ill patients. This comprehensive review aimed to summarize the current evidence regarding the use of vitamin C in the prevention or treatment of patients with SARS-CoV2 infection, based on available publications between January 2020 and February 2021. Overall, 21 publications were included in this review, consisting of case-reports and case-series, observational studies, and some clinical trials. In many of the publications, data were incomplete, and in most clinical trials the results are still pending. No studies regarding prevention of COVID-19 with vitamin C supplementation were found. Although some clinical observations reported improved medical condition of patients with COVID-19 treated with vitamin C, available data from controlled studies are scarce and inconclusive. Based on the theoretical background presented in this article, and some preliminary encouraging studies, the role of vitamin C in the treatment of patients with SARS-CoV-2 infection should be further investigated.

scholarly journals Effects of open-label placebos in clinical trials: a systematic review and meta-analysis

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Melina von Wernsdorff ◽  
Martin Loef ◽  
Brunna Tuschen-Caffier ◽  
Stefan Schmidt
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Medical Condition ◽  
Data Extraction ◽  
Meta Analysis ◽  
Risk Of Bias ◽  
Open Label ◽  
Cancer Related Fatigue ◽  
Promising Treatment ◽  
Irritable Bowel

AbstractOpen-label placebos (OLPs) are placebos without deception in the sense that patients know that they are receiving a placebo. The objective of our study is to systematically review and analyze the effect of OLPs in comparison to no treatment in clinical trials. A systematic literature search was carried out in February 2020. Randomized controlled trials of any medical condition or mental disorder comparing OLPs to no treatment were included. Data extraction and risk of bias rating were independently assessed. 1246 records were screened and thirteen studies were included into the systematic review. Eleven trials were eligible for meta-analysis. These trials assessed effects of OLPs on back pain, cancer-related fatigue, attention deficit hyperactivity disorder, allergic rhinitis, major depression, irritable bowel syndrome and menopausal hot flushes. Risk of bias was moderate among all studies. We found a significant overall effect (standardized mean difference = 0.72, 95% Cl 0.39–1.05, p < 0.0001, I2 = 76%) of OLP. Thus, OLPs appear to be a promising treatment in different conditions but the respective research is in its infancy. More research is needed, especially with respect to different medical and mental disorders and instructions accompanying the OLP administration as well as the role of expectations and mindsets.

scholarly journals Rivaroxaban versus warfarin for the management of left ventricle thrombus

2021 ◽  
Vol 73 (1) ◽  
Author(s):  
Monirah A. Albabtain ◽  
Yahya Alhebaishi ◽  
Ola Al-Yafi ◽  
Hatim Kheirallah ◽  
Adel Othman ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Retrospective Study ◽  
Left Ventricle ◽  
Surface Area ◽  
Median Time ◽  
Randomized Clinical Trials ◽  
Off Label Use ◽  
Duration Of Treatment ◽  
Off Label ◽  
Warfarin Group

Abstract Background Rivaroxaban has been recently introduced for the management of non-valvular intra-cardiac thrombosis with variable results. We aimed to compare the results of the off-label use of rivaroxaban versus warfarin in the management of patients with left ventricle (LV) thrombus. This research is a retrospective study conducted on 63 patients who had LV thrombus from January to December 2016. We compared patients treated with warfarin (n=35) to patients who had rivaroxaban (n=28), and study outcomes were time to thrombus resolution, bleeding, stroke, and mortality. Results The median duration of treatment was 9.5 (25th-75th percentiles: 6-32.5) months for rivaroxaban and 14 (3-41) months for warfarin. Thrombus resolution occurred in 24 patients in the warfarin group (68.6%) and 20 patients in the rivaroxaban group (71.4%). The median time to resolution in the warfarin group was 9 (4-20) months and 3 (2-11.5) months in the rivaroxaban group. Thrombus resolution was significantly faster in patients on rivaroxaban (p= 0.019). Predictors of thrombus resolution were thrombus surface area (HR: 1.21; CI 95% (1.0-1.46); p= .048) and the use of rivaroxaban (HR: 1.92; CI 95% (1.01-3.65); p= 0.048). There was no difference in stroke, bleeding, and mortality between both groups. Conclusion Rivaroxaban was as effective and safe as warfarin in managing patients with left ventricle thrombus. Larger randomized clinical trials are recommended to confirm our findings.

scholarly journals “Nanomaterials of curcumin-hyaluronic acid”: their various methods of formulations, clinical and therapeutic applications, present gap, and future directions

2021 ◽  
Vol 7 (1) ◽  
Author(s):  
Tanzeel Rehman Charan ◽  
Muhammad Aqeel Bhutto ◽  
Mihr Ali Bhutto ◽  
Azhar Ali Tunio ◽  
Ghulam Murtaza Khuhro ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Hyaluronic Acid ◽  
Future Research ◽  
Main Body ◽  
Future Directions ◽  
Research Directions ◽  
Safety And Efficacy ◽  
Therapeutic Applications ◽  
Future Research Directions ◽  
Neural Disorders

Abstract Background Nanomaterials of curcumin with hyaluronic acid have gained a lot of attention for potential therapeutic applications of curcumin and hyaluronic acid with or without other additional drugs. Overall studies of curcumin and hyaluronic acid show that nanomaterials of curcumin with hyaluronic acid accelerate the efficacy of curcumin in the treatment of various disorders like arthritis, cancer, hepatic fibrosis, neural disorders, wound healing, and skin regeneration, it is largely due to the combined effect of hyaluronic acid and curcumin. However, due to limited clinical trials and experiments on humans and animals, there is a substantial gap in research for the safety and efficacy of nanomaterials of curcumin-hyaluronic acid in the treatment of curcumin and hyaluronic acid targeted diseases and disorders. Main body of the abstract In this current review, we have first described various reported synthetic nanomaterials of curcumin-hyaluronic acid, then in the next section, we have described various fields, disorders, and diseases where these are being applied and in the final section of this review, we discussed the research gap, and future research directions needed to propose the fabricated nanocurcumin-hyaluronic acid biomaterials. Short conclusion There are substantial gaps in research for the safety and efficacy of nanomaterials of curcumin with hyaluronic acid due to limited available data of clinical trials and experiments of nanocurcumin-hyaluronic acid biomaterials on humans and animals. So, it entirely requires serious and committed efforts through the well-organized system of practical and clinical trials which provide results, data, and detections that lead to the formulation of the best drug from curcumin with hyaluronic acid for the treatment of curcumin and hyaluronic acid targeted diseases and disorders.

Sign in / Sign up

Export Citation Format

Share Document