Individual Patient-Level Data Sharing for Continuous Learning: A Strategy for Trial Data Sharing

Kuntz, Richard E.; Antman, Elliott M.; Califf, Robert M.; Ingelfinger, Julie R.; Krumholz, Harlan M.; Ommaya, Ayub K.; Peterson, Eric D.; Ross, Joseph S.; Waldstreicher, Joanne; Wang, Shirley; Zarin, Deborah A.; Whicher, Danielle; Siddiqi, Sameer; Lopez, Marianne Hamilton

doi:10.31478/201906b

Cited by 10 publications

(10 citation statements)

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“…23 Including no treatment arms in the RCTs to understand the role of natural history and regression to the mean more clearly would be beneficial for future trials, and similar comparisons between objective and subjective outcomes in other diseases may corroborate or challenge our findings. For new drug development, efforts to understand how baseline covariates and confounding factors in the placebo responses via data sharing initiatives 24,25 can be helpful to study design considerations, expedite clinical drug development, and ultimately bring effective treatments to patients in need. Role of the Funder/Sponsor: Neither TransCelerate Biopharma nor the data providers contributed to design and conduct of the study; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication.…”

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confidence: 99%

Assessment of Placebo Response in Objective and Subjective Outcome Measures in Rheumatoid Arthritis Clinical Trials

et al. 2020

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Key Points Question Are subjective patient-reported outcomes vs objective biomarkers associated with higher placebo responses in clinical trials? Findings In this cross-sectional study examining the placebo arms of 5 randomized clinical trials of rheumatoid arthritis including 788 patients, objective markers of inflammation and subjective pain ratings improved in a comparable clinically meaningful magnitude. Baseline values were associated with placebo response, suggesting that regression to the mean might dominate response to randomized placebo treatment. Meaning The findings of this study suggest that investigators may need to improve their understanding of natural history and baseline levels of outcomes because these factors can be important contributors to the response in placebo arms.

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confidence: 99%

Assessment of Placebo Response in Objective and Subjective Outcome Measures in Rheumatoid Arthritis Clinical Trials

et al. 2020

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“…The current data set consisted of patient-level AE data from patients with schizophrenia, bipolar disorder, autism, or Alzheimer’s disease who received risperidone in 17 controlled clinical trials. These data were obtained through the YODA project [ 22 ].…”

Section: Methodsmentioning

confidence: 99%

Depicting Risperidone Safety Profiles in Clinical Trials Across Different Diagnoses Using a Dopamine D2-Based Pharmacological Class Effect Query Defined by FAERS

et al. 2022

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Background and Objective Current methods are not designed to relate the incidence of individual adverse events reported in clinical trials to the plurality of adverse events accumulated in spontaneous reporting databases during real-world use. We have previously reported on a pharmacological class-effect query of clinical trial data defined by a disproportionality analysis of the US Food and Drug Administration Adverse Event Reporting System (FAERS) post-marketing data. The aim of the current analysis was to apply a dopamine D 2 -based pharmacological class-effect query to clinical trial safety data of an atypical antipsychotic tested across different patient populations. Methods Patient-level adverse event data (n = 4400) from controlled clinical trials of the antipsychotic risperidone in schizophrenia, bipolar disorder, Alzheimer's disease psychosis, and autism were obtained through the Yale University Open Data Access (YODA) project. An Empirical Bayes Geometric Mean analysis was performed, and a three-fold threshold incidence level was applied to determine if a preferred term met criteria for being an antipsychotic class-related adverse event.Results In pooled data from seven trials of adult schizophrenia, class-specific adverse events were identified in 49% of patients treated with risperidone; in 49% of risperidone-treated patients in two trials in adolescent schizophrenia; in 65% of risperidone-treated patients in four trials in adult bipolar disorder; in 50% of risperidone-treated patients in two trials in adolescent schizophrenia; in 36% of risperidone-treated patients in one trial in Alzheimer's disease; and in 94% of risperidonetreated patients in one trial in autism. Conclusions The cumulative curves of class-specific adverse events in risperidone clinical trials of schizophrenia were similar to those first reported for other atypical antipsychotic drugs. However, the class-specific adverse event curves were notably lower for Alzheimer's disease and higher for autism, suggesting that the diagnostic indication may have an important effect on the cumulative class-specific side-effect burden.

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“…Longitudinal data sharing over many years may also be necessary, although effort will be needed to overcome the many barriers to full implementation of data sharing. 78…”

Section: More Funding For Researchmentioning

confidence: 99%

The American Heart Association at 100: A Century of Scientific Progress and the Future of Cardiovascular Science: A Presidential Advisory From the American Heart Association

Elkind,

Arnett,

Benjamin

et al. 2024

Circulation

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In 1924, the founders of the American Heart Association (AHA) envisioned an international society focused on the heart and aimed at facilitating research, disseminating information, increasing public awareness, and developing public health policy related to heart disease. This presidential advisory provides a comprehensive review of the past century of cardiovascular and stroke science, with a focus on the AHA’s contributions, as well as informed speculation about the future of cardiovascular science into the next century of the organization’s history. The AHA is a leader in fundamental, translational, clinical, and population science, and it promotes the concept of the “learning health system,” in which a continuous cycle of evidence-based practice leads to practice-based evidence, permitting an iterative refinement in clinical evidence and care. This advisory presents the AHA’s journey over the past century from instituting professional membership to establishing extraordinary research funding programs; translating evidence to practice through clinical practice guidelines; affecting systems of care through quality programs, certification, and implementation; leading important advocacy efforts at the federal, state and local levels; and building global coalitions around cardiovascular and stroke science and public health. Recognizing an exciting potential future for science and medicine, the advisory offers a vision for even greater impact for the AHA’s second century in its continued mission to be a relentless force for longer, healthier lives.

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Individual Patient-Level Data Sharing for Continuous Learning: A Strategy for Trial Data Sharing

Cited by 10 publications

References 47 publications

Assessment of Placebo Response in Objective and Subjective Outcome Measures in Rheumatoid Arthritis Clinical Trials

Assessment of Placebo Response in Objective and Subjective Outcome Measures in Rheumatoid Arthritis Clinical Trials

Depicting Risperidone Safety Profiles in Clinical Trials Across Different Diagnoses Using a Dopamine D2-Based Pharmacological Class Effect Query Defined by FAERS

The American Heart Association at 100: A Century of Scientific Progress and the Future of Cardiovascular Science: A Presidential Advisory From the American Heart Association

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