Recommendations for Self-Report Outcome Measures in Vulvodynia Clinical Trials

2017 ◽  
Vol 33 (8) ◽  
pp. 756-765 ◽  
Author(s):  
Caroline F. Pukall ◽  
Sophie Bergeron ◽  
Candace Brown ◽  
Gloria Bachmann ◽  
Ursula Wesselmann
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Self Report

scholarly journals Music Therapy Clinical Trials in Cross-Cultural Settings

2020 ◽  
Vol 4 (Supplement_1) ◽  
pp. 930-930
Author(s):  
Tara Rose
Keyword(s):  
Clinical Trials ◽  
Music Therapy ◽  
Outcome Measures ◽  
Cross Cultural ◽  
Self Report ◽  
Medical Conditions ◽  
Cultural Perspective ◽  
People With Dementia ◽  
Cultural Research ◽  
Nonpharmacological Intervention

Abstract Music therapy in clinical trials has shown efficacy as a nonpharmacological intervention for multiple medical conditions and procedures. Every culture has music and virtually everyone on this globe enjoys music suggesting the universality of music therapy. However, in the US, most music therapy clinical trials participants are English-speaking Caucasians. That narrow pool limits our understanding of the benefits of music in an ethnically and culturally heterogeneous nation. This study looks to the international clinical trials for lessons and information that can advance U.S. studies by expanding the methodology and clinical reach to benefit a more extensive population of patients. A review of 449 studies in 48 countries from clinical trials registries supports an effort to expand music therapy studies and interventions by incorporating a cross-cultural perspective. Researchers and clinicians using international resources can increase their understanding and capacity. Globally, many standardized measures have been translated, including self-report measures of behavioral and mental health, pain, sleep, medical conditions, and symptom severity used for outcome measures, as well as music therapy measures and intervention checklists. Scientifically accepted physiological outcome measures have shown the benefits of music interventions for older adults regardless of cultural or ethnic differences. For example, neuroimaging research supports the clinically derived notion that music can address needs of people with dementia. The future will require new standards for multi-cultural research. To expand studies and methodologies, we need to include more diverse populations. This paper proposes that to do that, we must look to the global scientific community.

scholarly journals Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

2021 ◽  
Vol 30 (160) ◽  
pp. 210026
Author(s):  
Meena Kalluri ◽  
Fabrizio Luppi ◽  
Ada Vancheri ◽  
Carlo Vancheri ◽  
Elisabetta Balestro ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Interstitial Lung Diseases ◽  
Self Report ◽  
Patient Reported ◽  
Probability Of Success ◽  
History Of ◽  
Disease Specific

Patient-reported outcome measures (PROMs), tools to assess patient self-report of health status, are now increasingly used in research, care and policymaking. While there are two well-developed disease-specific PROMs for interstitial lung diseases (ILD) and idiopathic pulmonary fibrosis (IPF), many unmet and urgent needs remain. In December 2019, 64 international ILD experts convened in Erice, Italy to deliberate on many topics, including PROMs in ILD. This review summarises the history of PROMs in ILD, shortcomings of the existing tools, challenges of development, validation and implementation of their use in clinical trials, and the discussion held during the meeting. Development of disease-specific PROMs for ILD including IPF with robust methodology and validation in concordance with guidance from regulatory authorities have increased user confidence in PROMs. Minimal clinically important difference for bidirectional changes may need to be developed. Cross-cultural validation and linguistic adaptations are necessary in addition to robust psychometric properties for effective PROM use in multinational clinical trials. PROM burden of use should be reduced through appropriate use of digital technologies and computerised adaptive testing. Active patient engagement in all stages from development, testing, choosing and implementation of PROMs can help improve probability of success and further growth.

scholarly journals When All Else Fails, Listen to the Patient E-technology and Patient Self-Report Can Improve Precision in Mental Health Clinical Trials (Preprint)

2018 ◽  
Author(s):  
Aaron M Mofsen ◽  
Thomas L Rodebaugh ◽  
Ginger E Nicol ◽  
Colin A Depp ◽  
Philip J Miller ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Late Phase ◽  
Smart Phone ◽  
Self Report ◽  
Recall Bias ◽  
Development Programs ◽  
Assay Sensitivity ◽  
Nuanced Understanding ◽  
Ecological Momentary

UNSTRUCTURED The global burden of psychiatric illness continues to increase during this troubling period of industry disinvestment in novel therapies. Reduced assay sensitivity in primary outcome measures has contributed to the exodus of the pharmaceutical industry from the CNS space insofar as it has contributed to late phase failures in major CNS development programs. There are a number of reasons for this reduced assay sensitivity in psychiatry outcome measures including inappropriately broad measures, recall bias, and poor inter-rater reliability. A lack of a more nuanced understanding of how disorders like depression behave also contributes to measurement error in psychiatry clinical trials. We believe that Ecological Momentary Assessment (EMA) or frequent, real time assessment delivered via smart phone will help us overcome these psychometric challenges and prevent late phase failures by increasing the sensitivity of measurement, eliminating recall bias, eliminated bias added by the rater, and finally by producing data that will give researchers a better understanding of how the illnesses we treat behave.

Digital Technology in Clinical Trials for Multiple Sclerosis: a systematic review. (Preprint)

2020 ◽  
Author(s):  
Marcello De Angelis ◽  
Luigi Lavorgna ◽  
Antonio Carotenuto ◽  
Martina Petruzzo ◽  
Roberta Lanzillo ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Digital Technology ◽  
Clinical Trial Design ◽  
Motor Rehabilitation ◽  
Robot Assisted ◽  
Future Directions ◽  
Treatment Side Effects

BACKGROUND Clinical trials in multiple sclerosis (MS) have leveraged the use of digital technology to overcome limitations in treatment and disease monitoring. OBJECTIVE To review the use of digital technology in concluded and ongoing MS clinical trials. METHODS In March 2020, we searched for “multiple sclerosis” and “trial” on pubmed.gov and clinicaltrials.gov using “app”, “digital”, “electronic”, “internet” and “mobile” as additional search words, separately. Overall, we included thirty-five studies. RESULTS Digital technology is part of clinical trial interventions to deliver psychotherapy and motor rehabilitation, with exergames, e-training, and robot-assisted exercises. Also, digital technology has become increasingly used to standardise previously existing outcome measures, with automatic acquisitions, reduced inconsistencies, and improved detection of symptoms. Some trials have been developing new patient-centred outcome measures for the detection of symptoms and of treatment side effects and adherence. CONCLUSIONS We will discuss how digital technology has been changing MS clinical trial design, and possible future directions for MS and neurology research.

scholarly journals Use of connected digital products in clinical research following the COVID-19 pandemic: a comprehensive analysis of clinical trials

BMJ Open ◽  
2021 ◽  
Vol 11 (6) ◽  
pp. e047341
Author(s):  
Caroline Marra ◽  
William J Gordon ◽  
Ariel Dora Stern
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Outcome Measures ◽  
Remote Monitoring ◽  
Search Algorithm ◽  
Primary Outcome Measure ◽  
Secondary Outcome ◽  
Digital Products ◽  
Clinical Trial Registry ◽  
Before And After

ObjectivesIn an effort to mitigate COVID-19 related challenges for clinical research, the US Food and Drug Administration (FDA) issued new guidance for the conduct of ‘virtual’ clinical trials in late March 2020. This study documents trends in the use of connected digital products (CDPs), tools that enable remote patient monitoring and telehealth consultation, in clinical trials both before and after the onset of the pandemic.DesignWe applied a comprehensive text search algorithm to clinical trial registry data to identify trials that use CDPs for remote monitoring or telehealth. We compared CDP use in the months before and after the issuance of FDA guidance facilitating virtual clinical trials.SettingAll trials registered on ClinicalTrials.gov with start dates from May 2019 through February 2021.Outcome measuresThe primary outcome measure was the overall percentage of CDP use in clinical trials started in the 10 months prior to the pandemic onset (May 2019–February 2020) compared with the 10 months following (May 2020–February 2021). Secondary outcome measures included CDP usage by trial type (interventional, observational), funder type (industry, non-industry) and diagnoses (COVID-19 or non-COVID-19 participants).ResultsCDP usage in clinical trials increased by only 1.65 percentage points, from 14.19% (n=23 473) of all trials initiated in the 10 months prior to the pandemic onset to 15.84% (n=26 009) of those started in the 10 months following (p<0.01). The increase occurred primarily in observational studies and non-industry funded trials and was driven entirely by CDP usage in trials for COVID-19.ConclusionsThese findings suggest that in the short-term, new options created by regulatory guidance to stimulate telehealth and remote monitoring were not widely incorporated into clinical research. In the months immediately following the pandemic onset, CDP adoption increased primarily in observational and non-industry funded studies where virtual protocols are likely medically necessary due to the participants’ COVID-19 diagnosis.

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.

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