Implementation of telemedicine in cancer clinical trials: Connectpatienttodoctor study. (Preprint)

2021 ◽  
Author(s):  
Yasmine MEGHIREF ◽  
Charles Parnot ◽  
Claire DUVERGER ◽  
Françoise DIFOUM ◽  
Audrey Gourden ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Remote Monitoring ◽  
Routine Care ◽  
Health Care Team ◽  
Digital Tool ◽  
Early Management ◽  
Median Response ◽  
Patient Reported ◽  
High Level

BACKGROUND Telemedicine is currently being adopted for the management of patients in routine care. However, its use remains limited in the context of clinical trials. OBJECTIVE The Connectpatienttodoctor study aims to evaluate a connected telemonitoring platform, Haya by Cureety, in cancer patients included in clinical trials. METHODS Patients included in an interventional oncology clinical trial were eligible. Patients were registered with a digital tool to respond to a patient reported outcomes (ePRO) questionnaire based on CTCAE v.5.0, personalized to their pathology and treatment. An algorithm evaluates the health status of the patient based on the reported adverse events (AEs), with a classification in four different states (Correct, Compromise, State to be monitored or Critical State). The main objective was to evaluate the feasibility of remote monitoring via a connected platform of patients included in a clinical trial. RESULTS From July 1, 2020, to March 31, 2021, 39 patients were included. The median age was 71 years (range, 41-94), 74% were male, and 59% had metastatic disease. Out of 969 ePRO questionnaires completed over the course of the study, 77% were classified as "correct", 11% as "compromised" and 12% as "to be monitored" or “critical”. The median response time was 7 days (IQR 7-15.5) and 76% of patients (n=25/33) were compliant. Out of the 35 patients who answered a satisfaction questionnaire, 95% (n=33) were satisfied or very satisfied with the tool, 85% (n=30) were satisfied with their relationship with the health care team. There were 5 unscheduled hospitalizations during the study period. CONCLUSIONS Remote monitoring in clinical trials is feasible, with a high level of patient participation and satisfaction. It benefits them but also ensures the quality of the trial, through early management of AEs and better knowledge of the tolerance profile of experimental treatments. This e-technology will likely be deployed more widely in our clinical trials.

scholarly journals Longitudinal Analysis of Patient-Reported Outcomes in Clinical Trials: Applications of Multilevel and Multidimensional Item Response Theory

Psychometrika ◽  
2021 ◽  
Author(s):  
Li Cai ◽  
Carrie R. Houts
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Latent Variable ◽  
Patient Reported Outcomes ◽  
Multilevel Models ◽  
Latent Variable Models ◽  
Trial Data ◽  
Regulatory Science ◽  
Measurement Information ◽  
Patient Reported

AbstractWith decades of advance research and recent developments in the drug and medical device regulatory approval process, patient-reported outcomes (PROs) are becoming increasingly important in clinical trials. While clinical trial analyses typically treat scores from PROs as observed variables, the potential to use latent variable models when analyzing patient responses in clinical trial data presents novel opportunities for both psychometrics and regulatory science. An accessible overview of analyses commonly used to analyze longitudinal trial data and statistical models familiar in both psychometrics and biometrics, such as growth models, multilevel models, and latent variable models, is provided to call attention to connections and common themes among these models that have found use across many research areas. Additionally, examples using empirical data from a randomized clinical trial provide concrete demonstrations of the implementation of these models. The increasing availability of high-quality, psychometrically rigorous assessment instruments in clinical trials, of which the Patient-Reported Outcomes Measurement Information System (PROMIS®) is a prominent example, provides rare possibilities for psychometrics to help improve the statistical tools used in regulatory science.

scholarly journals End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain

2019 ◽  
Vol 3 (23) ◽  
pp. 3982-4001 ◽  
Author(s):  
Ann T. Farrell ◽  
Julie Panepinto ◽  
C. Patrick Carroll ◽  
Deepika S. Darbari ◽  
Ankit A. Desai ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Cell Disease ◽  
End Points ◽  
Additional Clinical Trial ◽  
Patient Reported ◽  
The Brain

Abstract To address the global burden of sickle cell disease (SCD) and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to: patient-reported outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.

scholarly journals The impact of patient-reported outcome (PRO) data from clinical trials: a systematic review and critical analysis

2019 ◽  
Vol 17 (1) ◽  
Author(s):  
Samantha Cruz Rivera ◽  
Derek G. Kyte ◽  
Olalekan Lee Aiyegbusi ◽  
Anita L. Slade ◽  
Christel McMullan ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Case Studies ◽  
Real World ◽  
Clinical Trial Data ◽  
Research Impact ◽  
Trial Data ◽  
Patient Reported ◽  
The Impact

Abstract Background Patient-reported outcomes (PROs) are commonly collected in clinical trials and should provide impactful evidence on the effect of interventions on patient symptoms and quality of life. However, it is unclear how PRO impact is currently realised in practice. In addition, the different types of impact associated with PRO trial results, their barriers and facilitators, and appropriate impact metrics are not well defined. Therefore, our objectives were: i) to determine the range of potential impacts from PRO clinical trial data, ii) identify potential PRO impact metrics and iii) identify barriers/facilitators to maximising PRO impact; and iv) to examine real-world evidence of PRO trial data impact based on Research Excellence Framework (REF) impact case studies. Methods Two independent investigators searched MEDLINE, EMBASE, CINAHL+, HMIC databases from inception until December 2018. Articles were eligible if they discussed research impact in the context of PRO clinical trial data. In addition, the REF 2014 database was systematically searched. REF impact case studies were included if they incorporated PRO data in a clinical trial. Results Thirty-nine publications of eleven thousand four hundred eighty screened met the inclusion criteria. Nine types of PRO trial impact were identified; the most frequent of which centred around PRO data informing clinical decision-making. The included publications identified several barriers and facilitators around PRO trial design, conduct, analysis and report that can hinder or promote the impact of PRO trial data. Sixty-nine out of two hundred nine screened REF 2014 case studies were included. 12 (17%) REF case studies led to demonstrable impact including changes to international guidelines; national guidelines; influencing cost-effectiveness analysis; and influencing drug approvals. Conclusions PRO trial data may potentially lead to a range of benefits for patients and society, which can be measured through appropriate impact metrics. However, in practice there is relatively limited evidence demonstrating directly attributable and indirect real world PRO-related research impact. In part, this is due to the wider challenges of measuring the impact of research and PRO-specific issues around design, conduct, analysis and reporting. Adherence to guidelines and multi-stakeholder collaboration is essential to maximise the use of PRO trial data, facilitate impact and minimise research waste. Trial registration Systematic Review registration PROSPERO CRD42017067799.

Accelerating the clinical trial start-up process for early-phase cancer studies: A test of process change to support rapid activation in an academic medical center.

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. e17507-e17507 ◽  
Author(s):  
Sheilah K Hurley ◽  
Therica M Miller ◽  
Rebecca Flores Stella ◽  
Keren Dunn ◽  
Ryan Schroeder ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Medical Center ◽  
Academic Medical Center ◽  
Cancer Center ◽  
Small Series ◽  
Academic Medical ◽  
One Year ◽  
Activation Times ◽  
High Level

e17507 Background: Clinical trial sponsors have strong scientific, financial, and regulatory interests in rapidly activating studies at participating sites. Academic medical centers have difficulty activating trials within a few weeks of sponsor agreement because, among other inefficiencies, they engage the necessary committee reviews, regulatory approvals, contracting, and budgeting in serial fashion. Incremental revisions in such workflows do not result in strong improvements. Methods: We redesigned our institutional workflow to complete clinical trial activation tasks within six weeks. Historical procedures were replaced rather than scrutinized. A high level leadership committee was required to change and integrate procedures across the medical center, and engage sponsors to improve their turnaround times. A web-based collaborative workflow tracking tool was created to help coordinate the necessary tasks and measure performance. Six clinical trials from the Cancer Center portfolio were used to test and improve the new workflow. Results: Clinical trial activation redesign took one year. For the six studies used as tests of change, the activation times were 49, 54, 78, 58, 62, and 32 days. Times in excess of 6 weeks were largely due to sponsor delays. Conclusions: Considerable effort is required to significantly alter a complex workflow like clinical trial activation. Appropriate priorities, leadership, staffing, and tools are required. Markedly shortened study activation for a small series of cancer trials taught our academic medical center lessons that will be useful for improving the process for all clinical trials, and will make us a better partner for pharmaceutical and academic sponsors as well as for investigator initiated research. [Table: see text]

Patients Driving the Clinical Trial Designs – Democracy in Clinical Research

2019 ◽  
Vol 14 (4) ◽  
pp. 237-246
Author(s):  
Payal Bhardwaj ◽  
Jeba Kumar ◽  
Raj Kumar Yadav
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Research ◽  
Retention Rate ◽  
Patient Centered ◽  
Regulatory Review ◽  
Patient Organizations ◽  
Patient Reported ◽  
Clinical Trial Designs ◽  
The Right

Background: Many of the clinical trials remain inefficient owing to the low retention rate, and an impact on the power of the study. In addition, regulatory bodies recommend including the patients’ experience, especially, patient-reported outcomes, while making clinical decisions, and approvals. Introduction: Patient centricity has reached the stage where patients are both willing and required to participate in clinical trial designs, regulatory review and experts on other panels. Efforts are being made in the right direction and there are multiple aspects that have been or are being addressed. Objective: The current article focuses on how to include patients in clinical trial designs, the benefits, challenges, and solutions. This means patients who were merely the participants until now, they will be the drivers of trials now, and hence the clinical trials will be more efficient and productive. Key Findings: There is a drive to enhance patients’ participation in clinical trial designs, especially, visits, efficacy outcomes and their expectations with the treatment. Patients want to remain informed, right from before participation to the completion of the trial. Patients are now an important part of regulatory review, as apparent from recent initiatives by the FDA and EMA. This will enhance patients’ awareness, and bring ownership and transparency. Various patient organizations, advocacy groups have made some great suggestions and taken initiatives in this direction. Clinical Trials Transformation Initiative, European Patient’s Academy on Therapeutic Innovation, and Patient- Centered Outcomes Research Institute are a few key initiatives. However, there is a set of challenges emanating from the complexity of trials, associated with unique mechanism of action of drugs, their efficacy and safety profiles, which has to be dealt with properly. Conclusion: Overall, the pharma domain is at the verge of putting the patient in the spotlight, to achieve a near-real democracy, where the clinical research is the by the patient, for the patient, and, of the patient.

A 5-year review of an educational program focused on meeting the needs of patients with advanced breast cancer.

2012 ◽  
Vol 30 (27_suppl) ◽  
pp. 87-87
Author(s):  
Ivy A. Ahmed ◽  
Allison Harvey ◽  
Marni Amsellem
Keyword(s):  
Breast Cancer ◽  
Clinical Trials ◽  
Advanced Breast Cancer ◽  
Metastatic Breast ◽  
Breast Cancer Diagnosis ◽  
Health Care Team ◽  
Advanced Breast ◽  
Healthcare Team ◽  
Cancer Support ◽  
High Level

87 Background: Women living with advanced breast cancer have distinct often unmet needs, even compared to other women with breast cancer. For the past five years, the Cancer Support Community has been committed to delivering Frankly Speaking About Cancer: Advanced Breast Cancer, a comprehensive psychosocial education program created for women with metastatic breast cancer and their families. The program’s clinically facilitated evidence-based education workshops have reached 2,690 attendees since its inception in 2006. The workshop provides valuable information about current treatments, clinical trials, side-effect management, and social and emotional challenges of an advanced breast cancer diagnosis. Workshop outcomes are analyzed annually, informing program content and ensuring the program is meeting the needs of those served. As the program enters its sixth year, data were consolidated across years to investigate how the program has been meeting the needs of these women and their loved ones. Methods: 1,827 workshop attendees since the program’s beginning have completed evaluations (68% response rate) which included assessing levels of pre- and post-workshop knowledge about advanced breast cancer, patient-provider communication, and general workshop feedback. Results: 64.9% of respondents were survivors, 78.5% were Caucasian, and the average age was 56. Most workshop respondents (89.6%) reported gaining a high or very high level of knowledge about advanced breast cancer, with significant increases compared with pre-workshop levels (p <.01). Most reported intent to increase communication with their clinical team following the workshop. A majority reported feeling better equipped to ask questions to their health care team (92.1%), and discuss the possibility of clinical trials with their healthcare team (77.7%). Furthermore, 90% of attendees felt confident that after the workshop they could speak knowledgably about side effects of cancer treatment with their doctor, a key issue in regards to quality of life. Conclusions: Taken together, the data indicate the continued relevance of this program for those affected by advanced breast cancer.

A pilot study of the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE) in a phase I clinical trial.

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 6587-6587 ◽  
Author(s):  
Rui Qin ◽  
Amylou C. Dueck ◽  
Daniel Satele ◽  
Julian R. Molina ◽  
Charles Erlichman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Pilot Study ◽  
Adverse Events ◽  
Phase I ◽  
Patient Reported Outcomes ◽  
Point Of View ◽  
Phase I Clinical Trial ◽  
Phase I Clinical Trials ◽  
Patient Reported

6587 Background: Recently the Patient-Reported Outcomes version of the CTCAE was developed to augment clinically graded adverse events with information reported directly by patients on clinical trials (Basch, 2009). The validation and potential application of PRO-CTCAE in phase I clinical trials are of great interest as toxicity is the primary endpoint. Methods: Selected PRO-CTCAE items (21 items measuring 12 symptomatic adverse events) corresponding to the major adverse events required to be graded clinically were collected in an ongoing phase I clinical trial of weekly cilengitide and paclitaxel in patients with advanced solid malignancies (NCT01276496). PRO-CTCAE was administered in a paper booklet by a clinical research associate prior to treatment on days 1, 8 and 15 of their regular visits. These PRO-CTCAE items were summarized descriptively in comparison to clinician-assessed CTCAE ver 4.0 (NCI, 2009) during the first cycle. As a pilot study to assess feasibility of PRO-CTCAE in phase I trials, PRO-CTCAE was not intended for determination of dose-limiting toxicity. Results: Twelve patients were accrued to two separate doses of cilengitide and paclitaxel. The median age was 56 (range 36—67) and half of patients were female. All patients had an ECOG performance score <= 1. Over 90% of patients had received prior surgery and chemotherapy. All but one patient completed weekly PRO-CTCAE during the first cycle, the only patient refused to complete weeks 2 and 3 did not give a reason. PRO-CTCAE captured most of the symptomatic adverse events reflected in clinician-assessed CTCAE. Some symptomatic adverse events were not reported clinically by CTCAE but were reported by patients by PRO-CTCAE. Overall, PRO-CTCAE items indicated slightly more severe degree of symptoms experienced by patients than those reported in CTCAE. Conclusions: This is the first study that PRO-CTCAE items were integrated within regular study visits in a phase I trial. The administration of PRO-CTCAE has been proved feasible and fruitful, providing consistent and enhanced symptomatic toxicity from the patient point of view. The addition of PRO-CTCAE did not significantly increase patient burden. Clinical trial information: NCT01276496.

Elucidation of qualitative patient-reported outcome (PRO) data using latent dirichlet allocation (LDA) in patients (pts) undergoing radical cystectomy.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e18245-e18245
Author(s):  
Thomas Michael Atkinson ◽  
Bruce D. Rapkin ◽  
Elizabeth Schofield ◽  
Bernard H. Bochner ◽  
Yuelin Li
Keyword(s):  
Decision Making ◽  
Clinical Trials ◽  
Radical Cystectomy ◽  
Clinical Decision Making ◽  
Routine Care ◽  
Free Text ◽  
Post Surgery ◽  
Patient Reported ◽  
Latent Topics ◽  
Prospective Longitudinal

e18245 Background: As PROs become commonplace in clinical trials and routine care, we must identify efficient analytic methods to better understand and ultimately integrate this information into clinical decision-making. While PROs are often captured via qualitative methods to provide context and pt voice to traditional objective and/or quantitative assessments, the current analytic standard is resource intensive and impractical for timely delivery of this information to clinicians and trialists. To address this problem, we propose the use of LDA, a natural language processing technique that automates the distillation of vast amounts of free-text data into underlying topics. Methods: As part of a prospective longitudinal trial of pts ( N = 200; M age = 63.8; 20% female; 46%) undergoing radical cystectomy (52% stage T2-T4) and urinary diversion (49% continent) for bladder cancer (ClinicalTrials.gov identifier NCT00745355), pts completed 30 minute interviews at baseline and 6-months post-surgery where they were asked to characterize their quality of life with respect to goals or milestones they would like to reach, problems they would like to solve, situations they would like to prevent, things they would like to remain the same, and commitments they would be willing to let go. LDA was used to extract latent topics and themes, stratified by time. Results: LDA extracted 7 latent topics and showed shifting patient priorities. At baseline, pt primary concerns were related to surgery and recovery. Six months post-surgery, pt goals transitioned toward themes related to concerns about cancer recurrence, regaining a sense of normalcy, return to work, enjoyment of life and increased appreciation of friends and family. Conclusions: LDA was used as an automated tool to illustrate a shift in pt goals and concerns as they transition into survivorship. This novel data analytic technique offers the possibility of significantly reducing the resources required to summarize qualitative PRO data. As such, this information may now be more readily available for immediate inclusion in the decision-making process as part of routine care and clinical trials. Clinical trial information: NCT00745355.

Designing clinical trials in 3L+ pancreatic cancer.

2019 ◽  
Vol 37 (4_suppl) ◽  
pp. 226-226
Author(s):  
Victoria G. Manax ◽  
Valery Chatikhine ◽  
Saundra Kirven ◽  
Ben R Taylor
Keyword(s):  
Pancreatic Cancer ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Patient Outcomes ◽  
Drug Effects ◽  
Study Drug ◽  
Sample Trial ◽  
High Level ◽  
Ecog Ps ◽  
Clinical Trial Information

226 Background: An increasing percentage of pancreatic cancer (PC) patients are looking for 3L+ options. As patient outcomes are improving with advances in treatment for PC, more patients need 3L+ treatments. Few trials focus on this patient segment as evaluation of drug effects can be complicated by comorbidity rates and disease-related SAEs. Having exhausted SOC chemotherapy backbones, patients increasingly search for novel therapies and clinical trial options. This assessment was to evaluate 3L+ PC trials for 1) sufficient patient interest, 2) a clinically evaluable patient population, and 3) guidelines/restrictions that could improve clinical assessment. Methods: Initiated as a retrospective evaluation of consenting patients screened for a prospective 2L+ pancreatic cancer Phase II trial with SM-88. A comparison of recurrent PC trials was performed by Novella to assess industry wide enrollment characteristics. Results: 32% of patients searching for clinical trials YTD 2018 in PanCAN’s database were looking for 3L+ treatments, up from 25% in 2016. In the sample trial, rate of consents (n=72) increased hyperbolically, reaching ~1/pt/day by end of analysis (24 sites). Inc/exc criteria were broadly standard for sponsor-initiated trials, however ECOG PS 2 were eligible. 38% screen failed, with hepatic labs (esp Alb & ALP) as most common reason. Industry analysis indicated expected 0.35 p/s/m for refractory PC trials and 20% screen fail. 93% (37/40) of SAEs were unrelated to study drug, including 11 G4 or G5 prior to receiving drug. Preliminary safety and efficacy results for the trial are reported elsewhere. Conclusions: Rapid enrollment from 3L+ patients demonstrated significant interest among this patient segment. The screen failure rate was within reasonable expectations, implying a majority of the patients were within standard inc/exc criteria. The high level of unrelated SAEs during the consent period and initial enrollment period indicate this may not be a suitable population to properly assess drug safety. Subgroup statistical analyses may be more relevant than ITT medians since several comorbidities, such as biliary obstruction, have elsewhere been demonstrated to have a dramatic impact on OS. Clinical trial information: NCT03512756.

Impact of clinical trial enrollment on episode costs in the Oncology Care Model (OCM).

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 6513-6513
Author(s):  
Garrett Young ◽  
Larry Edward Bilbrey ◽  
Edward Arrowsmith ◽  
L. Johnetta Blakely ◽  
Davey B. Daniel ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Routine Care ◽  
Cost Of Care ◽  
Trial Participation ◽  
Clinical Trial Participation ◽  
Total Cost ◽  
Comparator Group ◽  
Hospice Use ◽  
The Impact

6513 Background: Clinical trials are critical for improving outcomes for patients with cancer. However, there is some concern from health insurers that clinical trial participation can increase total cost of care for cancer patients. We investigated the impact of clinical trial participation on total costs paid by Medicare during the OCM program in a large community-based practice. Methods: Tennessee Oncology (TO) is a community oncology practice comprising over 90 oncologists across 30 sites of care. We linked TO trial data and electronic medical record data with OCM data for episodes of care from 2016-2018. To assess the impact of trial participation on total cost relative to routine care, we created matched comparator groups for each OCM episode based on cancer type, metastatic status, number of comorbidities, performance status, and age. Patients with breast cancer receiving hormone therapy only were excluded. Absolute and percent cost differences between groups were calculated for episodes that had a comparator group size of five or greater. Differences in total cost for trial episodes were compared to non-trial episodes, and significance was assessed using the Mann–Whitney U test. We also studied the impact of trial participation on receipt of active treatment in the last 14 days of life (TxEOL), hospice use, and hospitalizations. Results: During the study period, 8,026 completed OCM episodes met study criteria. Patients were enrolled in a clinical trial for 459 of these episodes. On average, episodes during which patients were on trial cost $5,973 less than matched non-trial episodes (Table), independent of early versus late-phase trial. Most savings resulted from decreased drug costs. There were no differences in rates of TxEOL (15% vs. 14% p=1.0), rates of hospitalizations (31% vs. 30% p=0.54), or hospice use (52% vs. 62% p=0.08) between trial and non-trial episodes. Median difference from comparator group average cost was significantly lower for clinical trial episodes (-18% vs. -6%, p<0.01). Conclusions: In the community setting, total costs paid by Medicare for patients participating in clinical trials during OCM episodes were lower than costs for similar patients receiving routine care. Clinical trial participation did not adversely impact end-of-life care or likelihood of hospitalization. These findings suggest that patient participation in clinical trials does not increase total cost of care nor enhance financial risk to payers.[Table: see text]

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