2 results
The evolving role of data & safety monitoring boards for real-world clinical trials
- Bryan J. Bunning, Haley Hedlin, Jonathan H. Chen, Jody D. Ciolino, Johannes Opsahl Ferstad, Emily Fox, Ariadna Garcia, Alan Go, Ramesh Johari, Justin Lee, David M. Maahs, Kenneth W. Mahaffey, Krista Opsahl-Ong, Marco Perez, Kaylin Rochford, David Scheinker, Heidi Spratt, Mintu P. Turakhia, Manisha Desai
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- Journal:
- Journal of Clinical and Translational Science / Volume 7 / Issue 1 / 2023
- Published online by Cambridge University Press:
- 02 August 2023, e179
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Introduction:
Clinical trials provide the “gold standard” evidence for advancing the practice of medicine, even as they evolve to integrate real-world data sources. Modern clinical trials are increasingly incorporating real-world data sources – data not intended for research and often collected in free-living contexts. We refer to trials that incorporate real-world data sources as real-world trials. Such trials may have the potential to enhance the generalizability of findings, facilitate pragmatic study designs, and evaluate real-world effectiveness. However, key differences in the design, conduct, and implementation of real-world vs traditional trials have ramifications in data management that can threaten their desired rigor.
Methods:Three examples of real-world trials that leverage different types of data sources – wearables, medical devices, and electronic health records are described. Key insights applicable to all three trials in their relationship to Data and Safety Monitoring Boards (DSMBs) are derived.
Results:Insight and recommendations are given on four topic areas: A. Charge of the DSMB; B. Composition of the DSMB; C. Pre-launch Activities; and D. Post-launch Activities. We recommend stronger and additional focus on data integrity.
Conclusions:Clinical trials can benefit from incorporating real-world data sources, potentially increasing the generalizability of findings and overall trial scale and efficiency. The data, however, present a level of informatic complexity that relies heavily on a robust data science infrastructure. The nature of monitoring the data and safety must evolve to adapt to new trial scenarios to protect the rigor of clinical trials.
2135 Impact of primary care physician gatekeeping on medication prescriptions for atrial fibrillation
- Andrew Y. Chang, Mariam Askari, Jun Fan, Paul A. Heidenreich, P. Michael Ho, Kenneth W. Mahaffey, Alexander C. Perino, Mintu P. Turakhia
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- Journal:
- Journal of Clinical and Translational Science / Volume 2 / Issue S1 / June 2018
- Published online by Cambridge University Press:
- 21 November 2018, pp. 82-83
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OBJECTIVES/SPECIFIC AIMS: Atrial fibrillation (AF) is the most commonly encountered arrhythmia in clinical practice, and has widely varying treatments for stroke prevention and rhythm management. Some of these therapies are increasingly being prescribed by primary care physicians (PCPs). We therefore sought to investigate if healthcare plans with PCP gatekeeping for access to specialists are associated with different pharmacologic treatment strategies for the disease. In particular, we focused on oral anticoagulants (OACs), non-vitamin K-dependent oral anticoagulants (NOACs), rate control, and rhythm control medications. METHODS/STUDY POPULATION: We examined a commercial pharmaceutical claims database (Truven Marketscan™) to compare the prescription frequency of OAC, rate control, and rhythm control medications used to treat AF between patients with PCP-gated health plans (where the PCP is the gatekeeper to specialist referral—i.e., HMO, EPO, POS) and patients with non-PCP-gatekeeper health plans (i.e., comprehensive, PPO, CHDP, HDHP). To control for potential confounders, we also used multivariable logistic regression models to calculate adjusted odds ratios which accounted for age, sex, region, Charlson comorbidity index, CHADS2Vasc score, hypertension, diabetes, stroke/transient ischemic attack, prior myocardial infarction, peripheral artery disease, and antiplatelet medication use. We also calculated median time to therapy to determine if there was a difference in time to new prescription of these medications. RESULTS/ANTICIPATED RESULTS: We found only small differences between patients in PCP-gated and non-PCP-gated plans regarding prescription proportion of anticoagulants at 90 days following new AF diagnosis (OAC 44.2% vs. 42%, p<0.01; warfarin 39.1% vs. 37.1%, p<0.01; NOAC 5.9% vs. 6.0%, p=0.64). We observed similar trends for rate control agents (76.4% vs. 73.4%, p<0.01) and rhythm control agents (24.4% vs. 24.6%, p=0.83). We found similar odds of OAC prescription at 90 days following new AF diagnosis between patients in PCP-gated and non-PCP-gated plans (adjusted OR for PCP-gated plans relative to non-gated plans: OAC 1.006, p=0.84; warfarin 1.054, p=0.08; NOAC 0.815, p=0.001; dabigatran 0.833, p=0.004; and rivaroxaban 0.181, p=0.02). We observed similar trends for rate control agents (1.166, p<0.0001) and rhythm control agents (0.927, p=0.03). Elapsed time until receipt of medication was similar between PCP-gated and non-gated groups [OAC 4±14 days (interquartile range) vs. 5±16 days, p<0.0001; warfarin 4±14 vs. 5±14, p<0.0001; NOAC 7±26 vs. 6±23, p=0.2937; rhythm control 13±35 vs. 13±34, p=0.8661; rate control 10±25 vs. 11±30, p<0.0001]. DISCUSSION/SIGNIFICANCE OF IMPACT: We found that plans with PCP gatekeeping to specialist referrals were not associated with clinically meaningful differences in prescription rates or delays in time to prescription of oral anticoagulation, rate control, and rhythm control drug therapy. In some cases, PCP gatekeeping plans had very small but statistically significant lower odds of being prescribed NOACs. These findings suggest that PCP gatekeeping does not appear to be a major structural barrier in receipt of medications for AF, although non-PCP-gated plans may vary slightly favor facilitating the prescription of NOACs. Our findings that overall OAC prescriptions did not differ by PCP gating status may suggest completion of the rapid dissemination and uptake phase for most AF treatments. The small but statistically significant odds ratios favoring the non-PCP-gated populations in NOACs further suggests that in this newer drug group, the process is ongoing, with more specialists representing early adopters. Interestingly, the low primary care odds ratio of rivaroxaban use, relative to dabigatran, may be indicative of a gradient of uptake of later-generation NOACs, although interpretability is limited by the small number of patients in the rivaroxaban group.