scholarly journals Lessons Learned from Conducting Internet-Based Randomized Clinical Trials During a Global Pandemic

2020 ◽  
Author(s):  
Matthew F Pullen ◽  
Katelyn A Pastick ◽  
Darlisha A Williams ◽  
Alanna A Nascene ◽  
Ananta S Bangdiwala ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Randomized Clinical Trials ◽  
Clinical Trial Design ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Lessons Learned ◽  
Online Data ◽  
Global Pandemic ◽  
Patient Reported

Abstract As the SARS-CoV-2 pandemic evolved, it was apparent that well designed and rapidly conducted randomized clinical trials were urgently needed. However, traditional clinical trial design presented several challenges. Notably, disease prevalence initially varied by time and region, and the pockets of outbreaks evolved geographically over time. Coupled with an occupational hazard from in-person study visits, timely recruitment would prove difficult in a traditional in-person clinical trial. Thus, our team opted to launch nationwide internet-based clinical trials using patient-reported outcome measures. In total, 2795 participants were recruited using traditional and social media, with screening and enrollment performed via an online data capture system. Follow-up surveys and survey reminders were similarly managed through this online system with manual participant outreach in the event of missing data. Here, we present a narrative of our experience running internet-based clinical trials and provide recommendations for the design of future clinical trials during a world pandemic.

Gaps in patient-reported outcome measures in randomized clinical trials of cardiac catheter ablation: a systematic review

2020 ◽  
Vol 6 (4) ◽  
pp. 234-242
Author(s):  
Yang Chen ◽  
Myura Nagendran ◽  
Manuel Gomes ◽  
Peter V Wharton ◽  
Rosalind Raine ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Catheter Ablation ◽  
Outcome Measures ◽  
Comparative Effectiveness ◽  
Randomized Clinical Trials ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Cardiac Catheter ◽  
Patient Reported

Abstract The aim of this systematic review was to evaluate randomized clinical trials (RCTs) of cardiac catheter ablation (CCA) and to assess the prevalence, characteristics and reporting standards of clinically relevant patient-reported outcome measures (PROMs). Electronic database searches of Medline, Embase, CENTRAL, and the WHO Trial Registry were conducted in March 2019. The study protocol was registered on PROSPERO (CRD42019133086). Of 7125 records identified, 237 RCTs were included for analysis, representing 35 427 patients with a mean age of 59 years. Only 43 RCTs (18%) reported PROMs of which 27 included a generic PROM that measured health-related quality of life (HRQL) necessary to conduct comparative effectiveness research. There was notable under-representation of certain patient groups—only 31% were women and only 8% were of non-Caucasian ethnicity, in trials which reported such data. The reporting standard of PROMs was highly variable with 8–62% adherence against CONSORT PRO-specific items. In summary, PROMs play a crucial role in determining the clinical and cost-effectiveness of treatments which primarily offer symptomatic improvement, such as CCA. Their underuse significantly limits evaluation of the comparative effectiveness of treatments. Using CCA as an exemplar, there are additional issues of infrequent assessment, poor reporting and under-representation of many population groups. Greater use of PROMs, and specifically validated HRQL questionnaires, is paramount in giving patients a voice in studies, generating more meaningful comparisons between treatments and driving better patient-centred clinical and policy-level decision-making.

scholarly journals Looking Ahead: Visual and Anatomical Endpoints in Future Trials of Diabetic Macular Ischemia

2021 ◽  
Author(s):  
Chui Ming Gemmy Cheung ◽  
Elizabeth Pearce ◽  
Beau Fenner ◽  
Piyali Sen ◽  
Victor Chong ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Foveal Avascular Zone ◽  
Consensus Definition ◽  
Clinical Trial Endpoints ◽  
Patient Reported ◽  
Trial Endpoints ◽  
Macular Ischemia

Diabetic macular ischemia (DMI) is a common complication of diabetic retinopathy that can lead to progressive and irreversible visual loss. Despite substantial clinical burden, there are no treatments for DMI, no validated clinical trial endpoints, and few clinical trials focusing on DMI. Therefore, generating consensus on validated endpoints that can be used in DMI for the development of effective interventions is vital. In this review, we discuss potential endpoints appropriate for use in clinical trials of DMI, and consider the data required to establish acceptable and meaningful endpoints. A combination of anatomical, functional, and patient-reported outcome measures will provide the most complete picture of changes that occur during the progression of DMI. Potential endpoint measures include change in size of the foveal avascular zone measured by optical coherence tomography angiography and change over time in best-corrected visual acuity. However, these endpoints must be supported by further research. We also recommend studies to investigate the natural history and progression of DMI. In addition to improving understanding of how patient demographics and comorbidities such as diabetic macular edema affect clinical trial endpoints, these studies would help to build the consensus definition of DMI that is currently missing from clinical practice and research.

scholarly journals Clinical trial design for cutaneous neurofibromas

Neurology ◽  
2018 ◽  
Vol 91 (2 Supplement 1) ◽  
pp. S31-S37 ◽  
Author(s):  
Ashley Cannon ◽  
Kurt Jarnagin ◽  
Bruce Korf ◽  
Brigitte C. Widemann ◽  
Denise Casey ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Trial Design ◽  
Skin Diseases ◽  
Clinical Trial Design ◽  
Patient Reported Outcome ◽  
Clinical Development ◽  
Patient Reported ◽  
Intervention Patient ◽  
Cutaneous Neurofibromas

ObjectiveSeveral clinical trials targeting cutaneous neurofibromas (cNF) have been conducted; however, none has resulted in meaningful changes to care. The Clinical Trial Design and Development subgroup's goals were to (1) define key considerations in the design of clinical trials for cNF, (2) summarize existing data in relation to these considerations, and (3) provide consensus recommendations about key elements of trial design to accelerate the clinical development of therapies for cNF.MethodsThe subgroup, with experts from genetics, dermatology, neurology, oncology, and basic science, spanning academia, government research, and regulatory programs, and industry, reviewed published and unpublished data on clinical trials for cNF and other diseases in the skin. Discussions of these data resulted in formulation of a list of priority issues to address in order to develop efficient and effective clinical trials for cNF.ResultsThe subgroup identified 2 natural history studies of cNF, 4 priority outcome measures, and 6 patient-reported outcome tools for potential use in efficacy trials of cNF. Time to initiate intervention, patient eligibility, mechanism of action, route of administration, safety monitoring, and regulatory agency interactions were identified as key factors to consider when designing clinical trials for cNF.ConclusionsAlignment on endpoints and methods for the measurement and quantification of cNF represent a priority for therapeutic development for cNF. Advances in technological methods and outcome tools utilized in other skin diseases may be applicable to cNF studies. Patient age is an important factor guiding trial design and clinical development path.

Optimizing electronic capture of patient-reported outcome measures in oncology clinical trials: lessons learned from a qualitative study

2020 ◽  
Vol 9 (17) ◽  
pp. 1195-1204
Author(s):  
Florence D Mowlem ◽  
Brad Sanderson ◽  
Jill V Platko ◽  
Bill Byrom
Keyword(s):  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Lessons Learned ◽  
Structured Interviews ◽  
Patient Reported ◽  
Oncology Clinical Trials ◽  
Anticancer Treatment ◽  
Fit For Purpose ◽  
The Impact ◽  
Electronic Implementation

Aim: To understand the impact of anticancer treatment on oncology patients’ ability to use electronic solutions for completing patient-reported outcomes (ePRO). Materials & methods: Semi-structured interviews were conducted with seven individuals who had experienced a cancer diagnosis and treatment. Results: Participants reported that the following would impact the ability to interact with an ePRO solution: peripheral neuropathy of the hands (4/7), fatigue and/or concentration and memory issues (6/7), where they are in a treatment cycle (5/7). Approaches to improve usability included: larger, well-spaced buttons to deal with finger numbness, the ability to pause a survey and complete at a later point and presenting the recall period with every question to reduce reliance on memory. Conclusion: Symptoms associated with cancers and anticancer treatments can impact the use of technologies. The recommendations for optimizing the electronic implementation of patient-reported outcome instruments in this population provides the potential to improve data quality in oncology trials and places patient needs at the forefront to ensure ‘fit-for-purpose’ solutions.

scholarly journals Measurement properties of patient-reported outcome measures (PROMs) in hyperhidrosis: a systematic review

2021 ◽  
Author(s):  
Michaela Gabes ◽  
Helge Knüttel ◽  
Gesina Kann ◽  
Christina Tischer ◽  
Christian J. Apfelbacher
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Measurement Properties ◽  
High Quality ◽  
High Quality Evidence ◽  
Patient Reported ◽  
Quality Evidence

Abstract Purpose To critically appraise, compare and summarize the quality of all existing PROMs that have been validated in hyperhidrosis to at least some extend by applying the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology. Thereby, we aim to give a recommendation for the use of PROMs in future clinical trials in hyperhidrosis. Methods We considered studies evaluating, describing or comparing measurement properties of PROMs as eligible. A systematic literature search in three big databases (MEDLINE, EMBASE and Web of Science) was performed. We assessed the methodological quality of each included study using the COSMIN Risk of Bias checklist. Furthermore, we applied predefined quality criteria for good measurement properties and finally, graded the quality of the evidence. Results Twenty-four articles reporting on 13 patient-reported outcome measures were included. Three instruments can be further recommended for use. They showed evidence for sufficient content validity and moderate- to high-quality evidence for sufficient internal consistency. The methodological assessment showed existing evidence gaps for eight other PROMs, which therefore require further validation studies to make an adequate decision on their recommendation. The Hyperhidrosis Disease Severity Measure-Axillary (HDSM-Ax) and the short-form health survey with 36 items (SF-36) were the only questionnaires not recommended for use in patients with hyperhidrosis due to moderate- to high-quality evidence for insufficient measurement properties. Conclusion Three PROMs, the Hyperhidrosis Quality of Life Index (HidroQoL), the Hyperhidrosis Questionnaire (HQ) and the Sweating Cognitions Inventory (SCI), can be recommended for use in future clinical trials in hyperhidrosis. Results obtained with these three instruments can be seen as trustworthy. Nevertheless, further validation of all three PROMs is desirable. Systematic review registration PROSPERO CRD42020170247

scholarly journals Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Theresa M. Coles ◽  
Adrian F. Hernandez ◽  
Bryce B. Reeve ◽  
Karon Cook ◽  
Michael C. Edwards ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Contextual Factors ◽  
The United States ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Think Tank ◽  
Level Of Evidence ◽  
Patient Reported ◽  
Evidentiary Standards

Abstract Objectives There has been limited success in achieving integration of patient-reported outcomes (PROs) in clinical trials. We describe how stakeholders envision a solution to this challenge. Methods Stakeholders from academia, industry, non-profits, insurers, clinicians, and the Food and Drug Administration convened at a Think Tank meeting funded by the Duke Clinical Research Institute to discuss the challenges of incorporating PROs into clinical trials and how to address those challenges. Using examples from cardiovascular trials, this article describes a potential path forward with a focus on applications in the United States. Results Think Tank members identified one key challenge: a common understanding of the level of evidence that is necessary to support patient-reported outcome measures (PROMs) in trials. Think Tank participants discussed the possibility of creating general evidentiary standards depending upon contextual factors, but such guidelines could not be feasibly developed because many contextual factors are at play. The attendees posited that a more informative approach to PROM evidentiary standards would be to develop validity arguments akin to courtroom briefs, which would emphasize a compelling rationale (interpretation/use argument) to support a PROM within a specific context. Participants envisioned a future in which validity arguments would be publicly available via a repository, which would be indexed by contextual factors, clinical populations, and types of claims. Conclusions A publicly available repository would help stakeholders better understand what a community believes constitutes compelling support for a specific PROM in a trial. Our proposed strategy is expected to facilitate the incorporation of PROMs into cardiovascular clinical trials and trials in general.

scholarly journals International guidance on the selection of patient-reported outcome measures in clinical trials: a review

2020 ◽  
Author(s):  
Norah L. Crossnohere ◽  
Michael Brundage ◽  
Melanie J. Calvert ◽  
Madeleine King ◽  
Bryce B. Reeve ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Selection Of
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