Can patient decision aids help people make good decisions about participating in clinical trials? A study protocol

<p>Abstract</p> <p>Background</p> <p>Evidence shows that the standard process for obtaining informed consent in clinical trials can be inadequate, with study participants frequently not understanding even basic information fundamental to giving informed consent. Patient decision aids are effective decision support tools originally designed to help patients make difficult treatment or screening decisions. We propose that incorporating decision aids into the informed consent process will improve the extent to which participants make decisions that are informed and consistent with their preferences. A mixed methods study will test this proposal.</p> <p>Methods</p> <p>Phase one of this project will involve assessment of a stratified random sample of 50 consent documents from recently completed investigator-initiated clinical trials, according to existing standards for supporting good decision making. Phase two will involve interviews of a purposive sample of 50 trial participants (10 participants from each of five different clinical areas) about their experience of the informed consent process, and how it could be improved. In phase three, we will convert consent forms for two completed clinical trials into decision aids and pilot test these new tools using a user-centered design approach, an iterative development process commonly employed in computer usability literature. In phase four, we will conduct a pilot observational study comparing the new tools to standard consent forms, with potential recruits to two hypothetical clinical trials. Outcomes will include knowledge of key aspects of the decision, knowledge of the probabilities of different outcomes, decisional conflict, the hypothetical participation decision, and qualitative impressions of the experience.</p> <p>Discussion</p> <p>This work will provide initial evidence about whether a patient decision aid can improve the informed consent process. The larger goal of this work is to examine whether study recruitment can be improved from (barely) informed consent based on disclosure-oriented documents, towards a process of high-quality participant decision-making.</p

Forecasts for the Attainment of Major Research Milestones in Parkinson's Disease.

BACKGROUND: Projections about when research milestones will be attained are often of interest to patients and can help inform decisions about research funding and health system planning. OBJECTIVE: To collect aggregated expert forecasts on the attainment of 11 major research milestones in Parkinson's disease (PD). METHODS: Experts were asked to provide predictions about the attainment of 11 milestones in PD research in an online survey. PD experts were identified from: 1) The Michael J. Fox Foundation for Parkinson's Research data base, 2) doctors specializing in PD at top ranked neurology centers in the US and Canada, and 3) corresponding authors of articles on PD in top medical journals. Judgments were aggregated using coherence weighting. We tested the relationship between demographic variables and individual judgments using a linear regression. RESULTS: 249 PD experts completed the survey. In the aggregate, experts believed that new treatments like gene therapy for monogenic PD, immunotherapy and cell therapy had 56.1%, 59.7%, and 66.6% probability, respectively of progressing in the clinical approval process within the next 10 years. Milestones involving existing management approaches, like the approval of a deep brain stimulation device or a body worn sensor had 78.4% and 82.2% probability of occurring within the next 10 years. Demographic factors were unable to explain deviations from the aggregate forecast (R2 = 0.029). CONCLUSIONS: Aggregated expert opinion suggests that milestones for the advancement of new treatment options for PD are still many years away. However, other improvements in PD diagnosis and management are believed to be near at hand

Comparison of Patient and Expert Perceptions of the Attainment of Research Milestones in Parkinson's Disease.

BACKGROUND: Commentators suggest that patients have unrealistic expectations about the pace of research advances and that such expectations interfere with patient decision-making. OBJECTIVE: The objective of this study was to compare expert expectations about the timing of research milestone attainment with those of patients who follow Parkinson's disease (PD) research. METHODS: Patients with PD and experts were asked to provide forecasts about 11 milestones in PD research in an online survey. PD experts were identified from a Michael J. Fox Foundation database, highly ranked neurology centers in the United States and Canada, and corresponding authors of articles on PD in top medical journals. Patients with PD were recruited through the Michael J. Fox Foundation. We tested whether patient forecasts differed on average from expert forecasts. We also tested whether differences between patient forecasts and the average expert forecasts were associated with any demographic factors. RESULTS: A total of 256 patients and 249 PD experts completed the survey. For 9 of the 11 milestones, patients' forecasts were on average higher than those of experts. Only exercise therapy met our 10% difference threshold for practical significance. Education was the only demographic that predicted patient deviations from expert forecasts on milestone forecasts. Patients offered significantly higher forecasts than experts that the clinical trials used in milestone queries would report positive primary outcomes. CONCLUSIONS: Differences between patient and expert expectations about research milestones were generally minor, suggesting that there is little cause for concern that patients who follow PD research are unduly swayed by inaccurate representations of research advancement in the media or elsewhere. © 2020 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society

Identifying and understanding factors that affect the translation of therapies from the laboratory to patients: a study protocol

Background: The process of translating preclinical findings into a clinical setting takes decades. Previous studies have suggested that only 5-10% of the most promising preclinical studies are successfully translated into viable clinical applications. The underlying determinants of this low success rate (e.g. poor experimental design, suboptimal animal models, poor reporting) have not been examined in an empirical manner. Our study aims to determine the contemporary success rate of preclinical-to-clinical translation, and subsequently determine if an association between preclinical study design and translational success/failure exists. Methods: Established systematic review methodology will be used with regards to the literature search, article screening and study selection process. Preclinical, basic science studies published in high impact basic science journals between 1995 and 2015 will be included. Included studies will focus on publicly available interventions with potential clinical promise. The primary outcome will be successful clinical translation of promising therapies - defined as the conduct of at least one Phase II trial (or greater) with a positive finding. A case-control study will then be performed to evaluate the association between elements of preclinical study design and reporting and the likelihood of successful translation. Discussion: This study will provide a comprehensive analysis of the therapeutic translation from the laboratory bench to the bedside. Importantly, any association between factors of study design and the success of translation will be identified. These findings may inform future research teams attempting preclinical-to-clinical translation. Results will be disseminated to identified knowledge users that fund/support preclinical research

Assessing the Completeness of Reporting in Preclinical Oncolytic Virus Therapy Studies

Irreproducibility of preclinical findings could be a significant barrier to the “bench-to-bedside” development of oncolytic viruses (OVs). A contributing factor is the incomplete and non-transparent reporting of study methodology and design. Using the NIH Principles and Guidelines for Reporting Preclinical Research, a core set of seven recommendations, we evaluated the completeness of reporting of preclinical OV studies. We also developed an evidence map identifying the current trends in OV research. A systematic search of MEDLINE and Embase identified all relevant articles published over an 18 month period. We screened 1,554 articles, and 236 met our a priori-defined inclusion criteria. Adenovirus (43%) was the most commonly used viral platform. Frequently investigated cancers included colorectal (14%), skin (12%), and breast (11%). Xenograft implantation (61%) in mice (96%) was the most common animal model. The use of preclinical reporting guidelines was listed in 0.4% of articles. Biological and technical replicates were completely reported in 1% of studies, statistics in 49%, randomization in 1%, blinding in 2%, sample size estimation in 0%, and inclusion/exclusion criteria in 0%. Overall, completeness of reporting in the preclinical OV therapy literature is poor. This may hinder efforts to interpret, replicate, and ultimately translate promising preclinical OV findings