scholarly journals Current challenges and opportunities for pharmacogenomics: perspective of the Industry Pharmacogenomics Working Group (I-PWG)

2021 ◽  
Author(s):  
Karina Bienfait ◽  
Aparna Chhibber ◽  
Jean-Claude Marshall ◽  
Martin Armstrong ◽  
Charles Cox ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Working Group ◽  
Direct Sequencing ◽  
Risk Scores ◽  
Pharmaceutical Companies ◽  
Patient Privacy ◽  
Clinical Implementation ◽  
Genomic Databases ◽  
Group I ◽  
Trial Participants

AbstractPharmaceutical companies have increasingly utilized genomic data for the selection of drug targets and the development of precision medicine approaches. Most major pharmaceutical companies routinely collect DNA from clinical trial participants and conduct pharmacogenomic (PGx) studies. However, the implementation of PGx studies during clinical development presents a number of challenges. These challenges include adapting to a constantly changing global regulatory environment, challenges in study design and clinical implementation, and the increasing concerns over patient privacy. Advances in the field of genomics are also providing new opportunities for pharmaceutical companies, including the availability of large genomic databases linked to patient health information, the growing use of polygenic risk scores, and the direct sequencing of clinical trial participants. The Industry Pharmacogenomics Working Group (I-PWG) is an association of pharmaceutical companies actively working in the field of pharmacogenomics. This I-PWG perspective will provide an overview of the steps pharmaceutical companies are taking to address each of these challenges, and the approaches being taken to capitalize on emerging scientific opportunities.

scholarly journals Assessing an Electronic Health Record research platform for identification of clinical trial participants

2021 ◽  
Vol 21 ◽  
pp. 100692
Author(s):  
Niina Laaksonen ◽  
Juha-Matti Varjonen ◽  
Minna Blomster ◽  
Antti Palomäki ◽  
Tuija Vasankari ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Electronic Health Record ◽  
Health Record ◽  
Research Platform ◽  
Electronic Health ◽  
Trial Participants

Achieving Consensus on Total Joint Replacement Trial Outcome Reporting Using the OMERACT Filter: Endorsement of the Final Core Domain Set for Total Hip and Total Knee Replacement Trials for Endstage Arthritis

2017 ◽  
Vol 44 (11) ◽  
pp. 1723-1726 ◽  
Author(s):  
Jasvinder A. Singh ◽  
Michelle M. Dowsey ◽  
Michael Dohm ◽  
Susan M. Goodman ◽  
Amye L. Leong ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Total Knee Replacement ◽  
Knee Replacement ◽  
Joint Replacement ◽  
Working Group ◽  
Total Joint Replacement ◽  
Core Domain ◽  
Total Knee ◽  
Core Domain Set

Objective.Discussion and endorsement of the OMERACT total joint replacement (TJR) core domain set for total hip replacement (THR) and total knee replacement (TKR) for endstage arthritis; and next steps for selection of instruments.Methods.The OMERACT TJR working group met at the 2016 meeting at Whistler, British Columbia, Canada. We summarized the previous systematic reviews, the preliminary OMERACT TJR core domain set and results from previous surveys. We discussed preliminary core domains for TJR clinical trials, made modifications, and identified challenges with domain measurement.Results.Working group participants (n = 26) reviewed, clarified, and endorsed each of the inner and middle circle domains and added a range of motion domain to the research agenda. TJR were limited to THR and TKR but included all endstage hip and knee arthritis refractory to medical treatment. Participants overwhelmingly endorsed identification and evaluation of top instruments mapping to the core domains (100%) and use of subscales of validated multidimensional instruments to measure core domains for the TJR clinical trial core measurement set (92%).Conclusion.An OMERACT core domain set for hip/knee TJR trials has been defined and we are selecting instruments to develop the TJR clinical trial core measurement set to serve as a common foundation for harmonizing measures in TJR clinical trials.

Baseline characteristics and follow-up outcomes in routine clinical practice patients categorised by renal function in the ETNA-AF-Europe registry

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
R De Caterina ◽  
M Gwechenberger ◽  
A Bakhai ◽  
P Monteiro ◽  
P Kelly ◽  
...  
Keyword(s):  
Renal Function ◽  
Total Mortality ◽  
Haemorrhagic Stroke ◽  
Risk Scores ◽  
Funding Source ◽  
Private Company ◽  
Group I ◽  
Baseline Characteristics ◽  
Event Rates

Abstract Background Edoxaban is an oral factor Xa inhibitor anticoagulant with 50% renal clearance, and proven efficacy and safety in patients (pts) with atrial fibrillation (AF). The post-authorisation, observational, ETNA-AF-Europe registry (NCT02944019) assessed the risks and benefits of edoxaban in pts with AF from 10 European countries. Purpose Evaluate baseline characteristics and event rates in pts categorised by creatinine clearance (CrCl) at 1-year follow-up of the ETNA-AF-Europe registry. Methods In this analysis, pts were divided into three groups according to CrCl: ≤50 ml/min (I), 50–80 mL/min (II) and ≥80 mL/min (III) (calculated using Cockcroft-Gault). Outcomes were descriptively analysed. Results Pts with the lowest CrCl (Group I) were mostly females, and had a higher mean age, lower body weight, higher stroke and bleeding risk scores and were considered more frail than those with higher CrCl (Groups II and III) (Table). Group I experienced higher rates of stroke or SEE, major or CRNM bleeding, cardiovascular death, and had a higher total mortality (Figure). Rates of intracranial haemorrhage (ICH) and haemorrhagic stroke (intracerebral and subarachnoid haemorrhage) were low and similar in pts across the range of CrCl. Conclusions Those with lower CrCl had more comorbidities and higher event rates than those with higher CrCl, with the exception of ICH and haemorrhagic stroke. A steep rise in the proportion of pts perceived as frail and in overall mortality in the lowest renal function tertile, raises the question whether low renal function is a determinant or a correlate of mortality. Funding Acknowledgement Type of funding source: Private company. Main funding source(s): Daiichi Sankyo Europe GmbH, Munich, Germany

Report of Working Group I: Gases

1986 ◽  
pp. 1-10
Author(s):  
J. D. Burton ◽  
P. G. Brewer ◽  
R. Chesselet
Keyword(s):  
Working Group ◽  
Group I

scholarly journals Excision with primary midline closure compared with Limberg flap in the treatment of sacrococcygeal pilonidal disease: a randomised clinical trial

2019 ◽  
Vol 101 (1) ◽  
pp. 21-29 ◽  
Author(s):  
M Arnous ◽  
H Elgendy ◽  
W Thabet ◽  
SH Emile ◽  
SA Elbaz ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Hospital Stay ◽  
Recurrence Rate ◽  
Primary Closure ◽  
Randomised Clinical Trial ◽  
Limberg Flap ◽  
Complication Rates ◽  
Cosmetic Results ◽  
Group I ◽  
Group Ii

BackgroundAlthough several surgical techniques for treatment of sacrococcygeal pilonidal sinus (SPND) have been described, there is no consensus on the optimal surgical procedure. In this study we compared excision with primary closure and Limberg flap in the treatment of SPND.MethodsThis was a prospective randomised clinical trial in patients with SPND who were randomly allocated to one of two groups: group I (excision and primary closure) and group II (Limberg flap technique). The primary outcome of the trial was recurrence of SPND whereas postoperative complications, return to work and cosmetic results were the secondary outcomes.ResultsSixty patients were included, with a mean age of 24.1 years and mean body mass index (BMI) of 26.8 kg/m2. Group 1 had significantly shorter operation time than group II. Both groups had similar hospital stay and comparable complication rates (43.3% vs 30%; P = 0.4). Group I had significantly higher recurrence rate (20% vs 0; P < 0.02) and significantly better cosmetic satisfaction score than group II. Being hairy (P = 0.04), positive family history (P = 0.03), diabetes mellitus (P = 0.005) and history of previous surgery for SPND (P = 0.01) were the significant predictors for recurrence.ConclusionsThe Limberg flap is an effective technique for the treatment of SPND with very low recurrence rate and comparable complication rate and hospital stay to excision and primary closure. Excision and primary closure offered the advantages of quicker healing time, earlier resumption of daily activities, better cosmetic results, which may render it more suitable for patients with low risk for recurrence.

scholarly journals Balance rehabilitation with a virtual reality protocol for patients with hereditary spastic paraplegia: Protocol for a clinical trial

PLoS ONE ◽  
2021 ◽  
Vol 16 (4) ◽  
pp. e0249095
Author(s):  
Bianca Simone Zeigelboim ◽  
Maria Renata José ◽  
Geslaine Janaina Bueno dos Santos ◽  
Maria Izabel Rodrigues Severiano ◽  
Hélio Afonso Ghizoni Teive ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Virtual Reality ◽  
Hereditary Spastic Paraplegia ◽  
Vestibular Rehabilitation ◽  
Rehabilitation Program ◽  
Controlled Clinical Trial ◽  
Spastic Paraplegia ◽  
Post Intervention ◽  
Group I ◽  
Alternative Treatment Option

Background Neurodegenerative diseases are sporadic hereditary conditions characterized by progressive dysfunction of the nervous system. Among the symptoms, vestibulopathy is one of the causes of discomfort and a decrease in quality of life. Hereditary spastic paraplegia is a heterogeneous group of hereditary degenerative diseases involving the disorder of a single gene and is characterized by the progressive retrograde degeneration of fibers in the spinal cord. Objective To determine the benefits of vestibular rehabilitation involving virtual reality by comparing pre intervention and post intervention assessments in individuals with hereditary spastic paraplegia. Methods In this randomized controlled clinical trial from the Rebec platform RBR-3jmx67 in which allocation concealment was performed and the evaluators be blinded will be included. The participants will include 40 patients diagnosed with hereditary spastic paraplegia. The interventions will include vestibular rehabilitation with virtual reality using the Wii® console, Wii-Remote and Wii Balance Board (Nintendo), and the studies will include pre- and post intervention assessments. Group I will include twenty volunteers who performed balance games. Group II will include twenty volunteers who performed balance games and muscle strength games. The games lasted from 30 minutes to an hour, and the sessions were performed twice a week for 10 weeks (total: 20 sessions). Results This study provides a definitive assessment of the effectiveness of a virtual reality vestibular rehabilitation program in halting the progression of hereditary spastic paraplegia, and this treatment can be personalized and affordable. Conclusion The study will determine whether a vestibular rehabilitation program with the Nintendo Wii® involving virtual reality can reduce the progressive effect of hereditary spastic paraplegia and serve as an alternative treatment option that is accessible and inexpensive. Rebec platform trial: RBR-3JMX67.

scholarly journals Guidelines on how to assess the validity of results presented in subgroup analysis of clinical trials

2002 ◽  
Vol 57 (2) ◽  
pp. 83-88 ◽  
Author(s):  
Edson Duarte Moreira ◽  
Ezra Susser
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Randomized Clinical Trial ◽  
Subgroup Analysis ◽  
Observational Studies ◽  
Randomized Clinical Trials ◽  
Usual Method ◽  
Validity Of Results ◽  
Results Of Treatment ◽  
Trial Participants

In observational studies, identification of associations within particular subgroups is the usual method of investigation. As an exploratory method, it is the bread and butter of epidemiological research. Nearly everything that has been learned in epidemiology has been derived from the analysis of subgroups. In a randomized clinical trial, the entire purpose is the comparison of the test subjects and the controls, and when there is particular interest in the results of treatment in a certain section of trial participants, a subgroup analysis is performed. These subgroups are examined to see if they are liable to a greater benefit or risk from treatment. Thus, analyzing patient subsets is a natural part of the process of improving therapeutic knowledge through clinical trials. Nevertheless, the reliability of subgroup analysis can often be poor because of problems of multiplicity and limitations in the numbers of patients studied. The naive interpretation of the results of such examinations is a cause of great confusion in the therapeutic literature. We emphasize the need for readers to be aware that inferences based on comparisons between subgroups in randomized clinical trials should be approached more cautiously than those based on the main comparison. That is, subgroup analysis results derived from a sound clinical trial are not necessarily valid; one must not jump to conclusions and accept the validity of subgroup analysis results without an appropriate judgment.

scholarly journals Acceptability of Pulse-Fortified Foods by Two Groups: Participants in a Clinical Trial and Participants in a Consumer Acceptability Panel

Foods ◽  
2018 ◽  
Vol 7 (8) ◽  
pp. 129 ◽  
Author(s):  
Donna Ryland ◽  
Peter Zahradka ◽  
Carla Taylor ◽  
Rhonda Bell ◽  
Michel Aliani
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Research Question ◽  
Health Benefit ◽  
Influential Factors ◽  
Consumer Acceptability ◽  
Fortified Foods ◽  
Overall Acceptability ◽  
Regular Consumption ◽  
Trial Participants

Pulses are nutrient-rich ingredients used as interventions in clinical trials to determine their effect on lowering blood lipids, which are risk factors for cardiovascular disease. Acceptability of these foods is critical for compliance by participants in clinical trials as well as regular consumption by those eating them for their health benefit. Commercialisation of foods that prove positive for health is required to make them available to the general population. Since the target for commercialisation would be products that will be procured by as many people as possible, the research question becomes whether or not testing is required by the clinical trial participants, by consumer acceptability testing in a sensory unit, or by both to ensure acceptability. The objective of this study was to determine the acceptability of pulse-based soups and casseroles destined for a clinical trial by both the participants in the clinical trial and by consumer participants not in the clinical trial. Neither group received any training regarding sensory analysis. Acceptability of aroma, appearance, flavor, texture, overall acceptability, and the frequency of eating the samples of five formulations fortified with either peas or beans was measured. Groups differed in their acceptability of foods for different attributes with the clinical trial participants providing less discrimination among the sensory attributes for their acceptability. Influential factors could include motivation for healthy eating, age, number of times the product was consumed, amount of the product consumed, and where it was consumed. In conclusion, acceptance measures from both groups are required in order to gain as much information as possible regarding acceptability of attributes for commercialisation of pulse-fortified foods that provide a health benefit.

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