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.

Abstract WP333: Clinical Research Coordinators' Barriers and Best Practices to Minority Recruitment in Stroke Clinical Trials

Stroke ◽  
2016 ◽  
Vol 47 (suppl_1) ◽  
Author(s):  
Jazmin Rivera ◽  
Lauren Southwick ◽  
Nina S Parikh ◽  
Sean Haley ◽  
Bernadette Boden-Albala
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Research ◽  
Focus Groups ◽  
Team Building ◽  
Patient Centered ◽  
Organizational Barriers ◽  
Internet Resources ◽  
Clinical Research Coordinators ◽  
Recruitment Challenges

Background: Poor recruitment and retention of clinical trial participants continues to be a major contributor to the early termination of many clinical trials. Specifically, low racial-ethnic minority and female participation rates can limit scientific, economic, and ethical value of a clinical trial. Previous literature has explored patient-centered enrollment barriers, while research has yet to investigate recruitment challenges facing clinical research coordinators (CRCs). Aim: To gain insight of the barriers facings CRCs with particular attention to minority and female recruitment efforts. Methods: Two semi-structured focus groups were conducted with a purposive sample of stroke CRCs in 2013 (N=17) and 2014 (N=23). Discussion topics included: 1) integrating NIH Inclusion Policy into trial design and recruitment strategies; 2) experiences recruiting minorities and women in stroke/neurological trials; and 3) strategies to enhance minority and female involvement. Two reviewers independently developed an initial set of base codes (n=6). Reviewers met and reconciled their independent coding using the 2013 transcript before applying the same process to the 2014 transcript. Codes and sub-codes were used to identify thematic areas. Results: Both focus groups included majority female participants with nearly half holding nursing degree. Codes informed three thematic areas: administration, enrollment and “other.” Administrative findings highlight organizational barriers. Enrollment findings identified challenges related to the severity of condition and time limitations. The “other” theme cut across administration and enrollment, often informed by coordinators’ personal experiences. Personal barriers include biases and uncertainty approaching patients from diverse backgrounds. Participants identified coordinator conferences, trainings, Internet resources, direct support from PIs and team building between hospital and research staff as key strategies. Conclusion: Our findings identified multi-dimensional organizational, administrative and resource barriers and the need for more focused CRC support to enhance participant recruitment efforts in stroke clinical research.

scholarly journals The Investigational Clinical Center: a clinical-supportive and patient-centered trial unit model. Ten years of experience through normal and pandemic times of a large pediatric Trial Center in Italy.

2021 ◽  
Author(s):  
Giuseppe Pontrelli ◽  
Marco Ciabattini ◽  
Franco De Crescenzo ◽  
Isabella Biondi ◽  
Rossana Cocchiola ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Research ◽  
Good Clinical Practice ◽  
Legal Framework ◽  
Patient Centered ◽  
Unit Model ◽  
Clinical Center ◽  
High Workload ◽  
Pediatric Trial

Abstract Evidence-based medicine relies on appropriately designed, conducted and reported clinical trials (CTs) to provide the best proofs of efficacy and safety for pharmacological and non-pharmacological treatments. Modern clinical research features high complexity and requires a high workload for the management of trials-related activities, often hampering physicians’ participation to clinical trials. Dealing with children in clinical research adds complexity: rare diseases, parents or legal guardian reluctance to engage and recruitment difficulties are major reasons of pediatric trials failure. However, because in pediatrics many treatments are prescribed off-label or are lacking, well-designed clinical trials are particularly needed. Clinical Trial Units (CTUs) are indeed an important asset in the implementation of clinical trials, but their support to investigators is limited to administrative and non-clinical tasks. In this paper we present the model of the Investigational Clinical Center (ICC) of the Bambino Gesù Children’s Hospital in Rome. The ICC includes clinicians supporting the Principal Investigators for clinical management of enrolled patients in compliance of Good Clinical Practice, the legal framework of Clinical Trials. Furthermore, we present ten years’ experience in pediatric clinical trials and how it has been affected in 2020 by the COVID-19 pandemic. The activity of the ICC has been evaluated according to specific metrics of performance. The ICC model offers a complete support, helping investigators, patients and their families to overcome majority of barriers linked to clinical research, even in time of pandemic. We propose this organization as an innovative model for total-supportive and patient-centered clinical trial implementation.

scholarly journals Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 196-205
Author(s):  
Sherif M. Badawy
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stakeholder Engagement ◽  
Patient Reported Outcomes ◽  
Outcomes Assessment ◽  
Cell Disease ◽  
Patient Centered ◽  
Patient Reported

Abstract Patients with sickle cell disease (SCD) have significant impairment in their quality of life across the life span as a consequence of serious disease burden with several SCD-related complications. A number of disease-modifying therapies are currently available, yet long-term clinical benefits in real-world settings remain unclear. Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including: (1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (consensus measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient-reported outcomes assessment is highly recommended, and patient-reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations for Interventional Trials guidelines. Patient-centered outcomes research (PCOR) approaches and meaningful stakeholder engagement throughout the process have the potential to optimize the execution and success of clinical trials in SCD with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient-centered research approaches and stakeholder engagement. A proposed hematology stakeholder-engagement framework for clinical trials is also discussed.

scholarly journals The Investigational Clinical Center: a clinical-supportive and patient-centered trial unit model. Ten years of experience through normal and pandemic times of a large pediatric Trial Center in Italy.

2020 ◽  
Author(s):  
Giuseppe Pontrelli ◽  
Marco Ciabattini ◽  
Franco De Crescenzo ◽  
Isabella Biondi ◽  
Rossana Cocchiola ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Research ◽  
Good Clinical Practice ◽  
Legal Framework ◽  
Patient Centered ◽  
Unit Model ◽  
Clinical Center ◽  
High Workload ◽  
Pediatric Trial

Abstract Evidence-based medicine relies on appropriately designed, conducted and reported clinical trials (CTs) to provide the best proofs of efficacy and safety for pharmacological and non-pharmacological treatments. Modern clinical research features high complexity and requires a high workload for the management of trials-related activities, often hampering physicians’participation to clinical trials. Dealing with children in clinical research adds complexity: rare diseases, parents or legal guardian reluctance to engage and recruitment difficulties are major reasons of pediatric trials failure. However, because in paediatrics many treatments are prescribed off-label or are lacking, well-designed clinical trials are particularly needed. Clinical Trial Units (CTUs) are indeed an important asset in the implementation of clinical trials, but their support to investigators is limited to administrative and non-clinical tasks. In this paper we present the model of the Investigational Clinical Center (ICC) of the Bambino Gesù Children’s Hospital in Rome. The ICC includes clinicians supporting the Principal Investigators (PIs) for clinical management of enrolled patients in compliance of Good Clinical Practice, the legal framework of Clinical Trials. Furthermore, we present ten years’ experience in paediatric clinical trials and how it has been disrupted during the COVID-19 pandemic. The activity of the ICC has been evaluated according to six metrics of performance. The ICC model offers a complete support, helping investigators, patients and their families to overcome majority of barriers linked to clinical research, even in time of pandemic. We propose this organization as an innovative model for total-supportive and patient-centered clinical trial implementation.

scholarly journals The Investigational Clinical Center: a clinical-supportive and patient-centered trial unit model. Ten years of experience through normal and pandemic times of a large pediatric trial center in Italy

2021 ◽  
Vol 47 (1) ◽  
Author(s):  
Giuseppe Pontrelli ◽  
Marco Ciabattini ◽  
Franco De Crescenzo ◽  
Isabella Biondi ◽  
Rossana Cocchiola ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Research ◽  
Good Clinical Practice ◽  
Legal Framework ◽  
Patient Centered ◽  
Unit Model ◽  
Clinical Center ◽  
High Workload ◽  
Pediatric Trial

AbstractEvidence-based medicine relies on appropriately designed, conducted and reported clinical trials (CTs) to provide the best proofs of efficacy and safety for pharmacological and non-pharmacological treatments. Modern clinical research features high complexity and requires a high workload for the management of trials-related activities, often hampering physicians’ participation to clinical trials. Dealing with children in clinical research adds complexity: rare diseases, parents or legal guardian reluctance to engage and recruitment difficulties are major reasons of pediatric trials failure.However, because in pediatrics many treatments are prescribed off-label or are lacking, well-designed clinical trials are particularly needed. Clinical Trial Units (CTUs) are indeed an important asset in the implementation of clinical trials, but their support to investigators is limited to administrative and non-clinical tasks. In this paper we present the model of the Investigational Clinical Center (ICC) of the Bambino Gesù Children’s Hospital in Rome. The ICC includes clinicians supporting the Principal Investigators for clinical management of enrolled patients in compliance of Good Clinical Practice, the legal framework of Clinical Trials. Furthermore, we present 10 years’ experience in pediatric clinical trials and how it has been affected in 2020 by the COVID-19 pandemic. The activity of the ICC has been evaluated according to specific metrics of performance. The ICC model offers a complete support, helping investigators, patients and their families to overcome majority of barriers linked to clinical research, even in time of pandemic. We propose this organization as an innovative model for total-supportive and patient-centered clinical trial implementation.

The Use of Digital Clinical Trial Methods in the Evaluation of Digital Therapeutics: A Scoping Review (Preprint)

2019 ◽  
Author(s):  
Allison Hirsch ◽  
Mahip Grewal ◽  
Anthony James Martorell ◽  
Brian Michael Iacoviello
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Research ◽  
Scoping Review ◽  
Digital Technologies ◽  
Good Clinical Practice ◽  
The United States ◽  
Clinical Trial Research ◽  
Digital Methods ◽  
Clinical Trial Methods

BACKGROUND Digital Therapeutics (DTx) provide evidence based therapeutic health interventions that have been clinically validated to deliver therapeutic outcomes, such that the software is the treatment. Digital methodologies are increasingly adopted to conduct clinical trials due to advantages they provide including increases in efficiency and decreases in trial costs. Digital therapeutics are digital by design and can leverage the potential of digital and remote clinical trial methods. OBJECTIVE The principal purpose of this scoping review is to review the literature to determine whether digital technologies are being used in DTx clinical research, which type are being used and whether publications are noting any advantages to their use. As DTx development is an emerging field there are likely gaps in the knowledge base regarding DTx and clinical trials, and the purpose of this review is to illuminate those gaps. A secondary purpose is to consider questions which emerged during the review process including whether fully remote digital clinical research is appropriate for all health conditions and whether digital clinical trial methods are inline with the principles of Good Clinical Practice. METHODS 1,326 records were identified by searching research databases and 1,227 reviewed at the full-article level in order to determine if they were appropriate for inclusion. Confirmation of clinical trial status, use of digital clinical research methods and digital therapeutic status as well as inclusion and exclusion criteria were applied in order to determine relevant articles. Digital methods employed in DTx research were extracted from each article and these data were synthesized in order to determine which digital methods are currently used in clinical trial research. RESULTS After applying our criteria for scoping review inclusion, 11 articles were identified. All articles used at least one form of digital clinical research methodology enabling an element of remote research. The most commonly used digital methods are those related to recruitment, enrollment and the assessment of outcomes. A small number of articles reported using other methods such as online compensation (n = 3), or digital reminders for participants (n = 5). The majority of digital therapeutics clinical research using digital methods is conducted in the United States and increasing number of articles using digital methods are published each year. CONCLUSIONS Digital methods are used in clinical trial research evaluating DTx, though not frequently as evidenced by the low proportion of articles included in this review. Fully remote clinical trial research is not yet the standard, more frequently authors are using partially remote methods. Additionally, there is tremendous variability in the level of detail describing digital methods within the literature. As digital technologies continue to advance and the clinical research DTx literature matures, digital methods which facilitate remote research may be used more frequently.

Shared-Task Worklists Improve Clinical Trial Recruitment Workflow in an Academic Emergency Department

2021 ◽  
Vol 12 (02) ◽  
pp. 293-300
Author(s):  
Kevin S. Naceanceno ◽  
Stacey L. House ◽  
Phillip V. Asaro
Keyword(s):  
Clinical Trial ◽  
Emergency Department ◽  
Clinical Trials ◽  
Clinical Research ◽  
Substantial Reduction ◽  
Text Message ◽  
High Volume ◽  
Text Messages ◽  
Shared Task ◽  
Clinical Research Coordinators

Abstract Background Clinical trials performed in our emergency department at Barnes-Jewish Hospital utilize a centralized infrastructure for alerting, screening, and enrollment with rule-based alerts sent to clinical research coordinators. Previously, all alerts were delivered as text messages via dedicated cellular phones. As the number of ongoing clinical trials increased, the volume of alerts grew to an unmanageable level. Therefore, we have changed our primary notification delivery method to study-specific, shared-task worklists integrated with our pre-existing web-based screening documentation system. Objective To evaluate the effects on screening and recruitment workflow of replacing text-message delivery of clinical trial alerts with study-specific shared-task worklists in a high-volume academic emergency department supporting multiple concurrent clinical trials. Methods We analyzed retrospective data on alerting, screening, and enrollment for 10 active clinical trials pre- and postimplementation of shared-task worklists. Results Notifications signaling the presence of potentially eligible subjects for clinical trials were more likely to result in a screen (p < 0.001) with the implementation of shared-task worklists compared with notifications delivered as text messages for 8/10 clinical trials. The change in workflow did not alter the likelihood of a notification resulting in an enrollment (p = 0.473). The Director of Research reported a substantial reduction in the amount of time spent redirecting clinical research coordinator screening activities. Conclusion Shared-task worklists, with the functionalities we have described, offer a viable alternative to delivery of clinical trial alerts via text message directly to clinical research coordinators recruiting for multiple concurrent clinical trials in a high-volume academic emergency department.

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.

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

2021 ◽  
Vol 16 ◽  
Author(s):  
Erica Winter ◽  
Scott Schliebner
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
Statistical Analyses ◽  
Clinical Trial Designs ◽  
Novel Approaches

: Characterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the patient.

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