scholarly journals Investigating modifications to participant information materials to improve recruitment into a large randomized trial

Trials ◽  
2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Richard Haynes ◽  
◽  
Fang Chen ◽  
Elizabeth Wincott ◽  
Rejive Dayanandan ◽  
...  
Keyword(s):  
Focus Groups ◽  
Randomized Trial ◽  
Randomized Trials ◽  
Trial Participation ◽  
Efficacy And Safety ◽  
Information Leaflet ◽  
Potential Participant ◽  
Information Materials ◽  
Large Randomized Trial ◽  
Participant Information

Abstract Background Large randomized trials are the best method to test the efficacy and safety of treatments expected to have moderate effects. We observed a significant decline in potential participants’ response to mailed invitations to participate in such trials over a 10-year period and investigated possible reasons behind this and potential modifications to the invitation process to mitigate it. Methods Participants who declined to participate in the HPS2-THRIVE trial were asked to give a reason. Formal focus groups were conducted to explore the reasons that potential participants might have for not participating. In addition, two embedded randomized comparisons around the timing of provision of the full participant information leaflet (PIL) and its style were conducted during recruitment into this large randomized trial. HPS2-THRIVE is registered at ClinicalTrials.gov (NCT00461630). Results The commonest reason given for declining invitations related to mobility and transportation (despite the offer of travel expenses). Both the focus groups and potential participants who declined their invitation indicated concern about side-effects of the treatment (as presented in the PIL) as a reason for declining the invitation. Neither delaying provision of the full PIL until the potential participant attended the trial clinic, nor modifying the style of the PIL improved the proportion of potential participants entering the trial: odds ratio (OR) 1.05 (95% confidence interval (CI) 0.94–1.17) and 1.10 (95% CI 0.94–1.28), respectively. However, modifying the style of the PIL did increase the proportion of participants attending screening appointments (OR 1.17, 95% CI 1.03–1.33). Conclusions Many reasons given for not participating in trials are not tractable to individual trials. However, modification of the PIL does show potential to modestly improve participation. If further trials could identify similar simple interventions that were beneficial, their net effects could substantially improve trial participation and facilitate recruitment into large trials.

scholarly journals Biochemical efficacy and safety trial of vitamin D (BEST-D): finding an appropriate dose to test in a large randomized trial

Trials ◽  
2013 ◽  
Vol 14 (S1) ◽  
Author(s):  
Jane Armitage ◽  
Harold Hin ◽  
Joseph Tomson ◽  
Mike Lay ◽  
Mike Hill ◽  
...  
Keyword(s):  
Vitamin D ◽  
Randomized Trial ◽  
Efficacy And Safety ◽  
Large Randomized Trial

scholarly journals Patient Partner Perspectives Regarding Ethically and Clinically Important Aspects of Trial Design in Pragmatic Cluster Randomized Trials for Hemodialysis

2021 ◽  
Vol 8 ◽  
pp. 205435812110328
Author(s):  
Stuart G. Nicholls ◽  
Kelly Carroll ◽  
Cory E. Goldstein ◽  
Jamie C. Brehaut ◽  
Charles Weijer ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Focus Groups ◽  
Family Member ◽  
Trial Design ◽  
Randomized Trials ◽  
Patient Choice ◽  
Trial Participation ◽  
Cluster Randomized Trials ◽  
Waiver Of Consent ◽  
Cluster Randomized

Background: Cluster randomized trials (CRTs) are trials in which intact groups such as hemodialysis centers or shifts are randomized to treatment or control arms. Pragmatic CRTs have been promoted as a promising trial design for nephrology research yet may also pose ethical challenges. While randomization occurs at the cluster level, the intervention and data collection may vary in a CRT, challenging the identification of research participants. Moreover, when a waiver of patient consent is granted by a research ethics committee, there is an open question as to whether and to what degree patients should be notified about ongoing research or be provided with a debrief regarding the nature and results of the trial upon completion. While empirical and conceptual research exploring ethical issues in pragmatic CRTs has begun to emerge, there has been limited discussion with patients, families, or caregivers of patients undergoing hemodialysis. Objective: To explore with patients and families with experience of hemodialysis research the challenges raised by different approaches to designing pragmatic CRTs in hemodialysis. Specifically, their perceptions of (1) the use of a waiver of consent, (2) notification processes and information provided to participants, and (3) any other concerns about cluster randomized designs in hemodialysis. Design: Focus group and interview discussions of hypothetical clinical trial designs. Setting: Focus groups and interviews were conducted in-person or via videoconference or telephone. Participants: Patient partners in hemodialysis research, defined as patients with personal experience of dialysis or a family member who had experience supporting a patient receiving hemodialysis, who have been actively involved in discussions to advise a research team on the design, conduct, or implementation of a hemodialysis trial. Methods: Participants were invited to participate in focus groups or individual discussions that were audio recorded with consent. Recorded interviews were transcribed verbatim prior to analysis. Transcripts were analyzed using a thematic analysis approach. Results: Two focus groups, three individual interviews, and one interview involving a patient and family member were conducted with 17 individuals between February 2019 and May 2020. Participants expressed support for approaches that emphasized patient choice. Disclosure of patient-relevant risks and information were key themes. Both consent and notification processes served to generate trust, but bypassing patient choice was perceived as undermining this trust. Participants did not dismiss the option of a waiver of consent. They were, however, more restrictive in their views about when a waiver of consent may be acceptable. Patient partners were skeptical of claims to impracticability based on costs or the time commitments for staff. Limitations: All participants were from Canada and had been involved in the design or conduct of a trial, limiting the degree to which results may be extrapolated. Conclusions: Given the preferences of participants to be afforded the opportunity to decide about trial participation, we argue that investigators should thoroughly investigate approaches that allow participants to make an informed choice regarding trial participation. In keeping with the preference for autonomous choice, there remains a need to further explore how consent approaches can be designed to facilitate clinical trial conduct while meeting their ethical requirements. Finally, further work is needed to define the limited circumstances in which waivers of consent are appropriate.

Insulin U-500, the Practical Solution for the treatment of Patients with High Insulin Requirements

2020 ◽  
Vol 17 (1) ◽  
pp. 26-29
Author(s):  
Nasser Mikhail
Keyword(s):  
Type 2 Diabetes ◽  
Randomized Trials ◽  
Regular Insulin ◽  
Injection Pain ◽  
Regular Human Insulin ◽  
Efficacy And Safety ◽  
Insulin Requirements ◽  
High Insulin

Background: Human regular insulin 500 (U-500) is 5 five times more concentrated than the traditional regular human insulin (U-100). Thus, every 1 ml of U-500 contains 500 units of insulin as opposed to 100 units/ml with most types of insulin. Methods: Review of all the relevant clinical studies related to insulin U-500 until February 12, 2020. Results: Insulin U-500 is indicated in patients with type 2 diabetes who require more than 200 units of insulin per day. Insulin U-500 has both prandial and basal actions, and can be injected as monotherapy in a convenient twice-daily regimen. Available data suggest that insulin U-500 is effective, associated with better compliance, and decreased injection pain compared with non-concentrated insulins. Its main limitations are hypoglycemia and weight gain, and the possibility of dosing errors. Conclusions: Overall, insulin U-500 is an effective and safe treatment for patients with type 2 diabetes and insulin resistance. Randomized trials are needed to compare the long-term efficacy and safety of insulin U-500 with other forms of insulin regimens.

scholarly journals Safety of switching between rituximab biosimilars in onco-hematology

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Silvana A. M. Urru ◽  
Stefania Spila Alegiani ◽  
Anna Guella ◽  
Giuseppe Traversa ◽  
Annalisa Campomori
Keyword(s):  
Chronic Lymphocytic Leukemia ◽  
Adverse Events ◽  
Prospective Observational Study ◽  
Randomized Trials ◽  
Lymphocytic Leukemia ◽  
Safety Signal ◽  
Efficacy And Safety ◽  
Clinical Safety ◽  
Study Population ◽  
Grade 3

AbstractComparable clinical efficacy and safety of the reference rituximab (MABTHERA) and its biosimilars has been established in randomized trials. However, safety concerns are often raised when switching from reference to biosimilar products and between different biosimilars. In this prospective observational study we aimed at evaluating the safety of switching between reference and biosimilar rituximab (TRUXIMA and RIXATHON) at Trento General Hospital (Italy). All patients (n = 83) with Non Hodgkin’s Lymphoma (NHL, n = 72) and Chronic Lymphocytic Leukemia (CLL, n = 11) who received rituximab between March 2018 and March 2019 were asked to take part in the study. In 2017 and 2018 two tenders were carried out and two different biosimilars became available in the hospital, these were used sequentially. Thus, patients with or without previous treatments with the originator rituximab either received a biosimilar or were switched between different biosimilars. The incidence of adverse events in these groups of patients is described. The study population received 465 rituximab infusions and all received biosimilars. Fifty patients (60%) experienced at least one switch between different biosimilars or between rituximab originator and biosimilar, whereas 33 (40%) received one of the two biosimilars and one patient received reference rituximab. Adverse events (n = 146) were reported in 71 patients (84.5%). Treatment-related grade 3–4 events were reported in 5 patients (5.9%), whereas grade 1 rituximab related infusion events were observed in 6 patients (7.1%). No safety signal emerged in association with the use of a specific biosimilar nor with the practice of switching. Adverse events were similar, in terms of seriousness and frequency, to those described in the literature, providing further support to the clinical safety of rituximab biosimilars.

scholarly journals Generalizing Treatment Effect Estimates From Sample to Population: A Case Study in the Difficulties of Finding Sufficient Data

2016 ◽  
Vol 41 (4) ◽  
pp. 357-388 ◽  
Author(s):  
Elizabeth A. Stuart ◽  
Anna Rhodes
Keyword(s):  
Head Start ◽  
Randomized Trial ◽  
Treatment Effect ◽  
External Validity ◽  
Randomized Trials ◽  
Target Population ◽  
Data Sets ◽  
Common Data Elements ◽  
Ex Post

Background: Given increasing concerns about the relevance of research to policy and practice, there is growing interest in assessing and enhancing the external validity of randomized trials: determining how useful a given randomized trial is for informing a policy question for a specific target population. Objectives: This article highlights recent advances in assessing and enhancing external validity, with a focus on the data needed to make ex post statistical adjustments to enhance the applicability of experimental findings to populations potentially different from their study sample. Research design: We use a case study to illustrate how to generalize treatment effect estimates from a randomized trial sample to a target population, in particular comparing the sample of children in a randomized trial of a supplemental program for Head Start centers (the Research-Based, Developmentally Informed study) to the national population of children eligible for Head Start, as represented in the Head Start Impact Study. Results: For this case study, common data elements between the trial sample and population were limited, making reliable generalization from the trial sample to the population challenging. Conclusions: To answer important questions about external validity, more publicly available data are needed. In addition, future studies should make an effort to collect measures similar to those in other data sets. Measure comparability between population data sets and randomized trials that use samples of convenience will greatly enhance the range of research and policy relevant questions that can be answered.

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