John Rogers’ Post

Erik van Zwet, PhD, et al. have examined the primary efficacy results of 23,551 randomized clinical trials from the Cochrane Database of Systematic Reviews.      The authors estimate that the great majority of trials have much lower statistical power for actual effects than the 80 or 90% for the stated effect sizes. Consequently, “statistically significant” estimates tend to seriously overestimate actual treatment effects, “nonsignificant” results often correspond to important effects, and efforts to replicate often fail to achieve “significance” and may even appear to contradict initial results. To address these issues, the authors reinterpret the P value in terms of a reference population of studies that are, or could have been, in the Cochrane Database.      Read the Original Article “A New Look at P Values for Randomized Clinical Trials” by Erik van Zwet, PhD, et al., from Leiden University Medical Center and elsewhere: https://eviden.cc/3NrObRI     #ClinicalTrials #MedicalResearch 

Randomized controlled trials are considered the gold standard in regulatory decision making, as observational studies are known to have important methodological limitations. However, real-world evidence (#RWE) may be helpful in specific situations.   Recently, PhD student Stefan Verweij and his colleagues, observed that for multiple sclerosis, real-world studies yielded treatment effect #estimates similar to those obtained from randomized controlled trials (#RCT’s). They compared evidence obtained from tens of published RCTs with evidence obtained from observational studies in a comprehensive systematic review and network meta analysis (#NMA), using posterior predictive distributions. The authors argue that, in regulatory decision making, observational studies may supplement pivotal randomized controlled trials in relapsing–remitting multiple sclerosis. For instance, by facilitating the extrapolation of trial results to the broader patient population. ➡ Do you want to learn more about this study? Read the open access article here: https://lnkd.in/ek9ZWsft #RegulatoryScience #MS Wouter Ahmed, Guiling Zhou, Dimitris Mavridis, Stavros Nikolakopoulos, Katrien Oude Rengerink, Maarten J. Bijlsma, Peter Mol, Prof. dr. Eelko Hak.

In case you were wondering what the PMD Foundation Natural History Study is… The Natural History Study, in partnership with Children’s Hospital of Philadelphia (CHOP) and other leukodystrophy-focused clinicians and researchers, is a research project actively working to collect robust data on individuals with PMD. Why is this Study so important? This all goes back to the FDA and clinical trial readiness. New therapeutic approaches for PMD will need to be tested in clinical trials and be approved by the FDA. Researchers need to understand the natural history of PMD (how it progresses over time) before they can design clinical trials to test whether these therapies are safe and effective for use in the patient population. We are looking for more families to complete this survey. Visit our website to learn more. https://lnkd.in/gbKVcgRa

New #URochesterResearch led by Zahira Quiñones Tavarez, MD, PhD, MPH, MS, a recent graduate of UR CTSI's Translational Biomedical PhD Program (https://lnkd.in/eaP33rQR), shows that people who use fruity-flavored e-cigarettes have a higher risk of developing nighttime dry cough, an early indicator of lung disease.   Quiñones and colleagues Steve Gill, Dongmei Li, Andrew Wojtovich, Irfan Rahman, and Deborah Ossip, analyzed data from 19,000 participants in the U.S. to uncover the link. They hope this work can inform future e-cig regulations.   "We are studying an early marker of disease, so we don't have to wait years to study the effects of these electronic cigarette flavors," Quiñones said.   Read the full study in @PLOS > https://lnkd.in/eMYFYqPe Ver en español > https://lnkd.in/ebXGH2_5

Meet Dr. Howard Fillit, a leader in Alzheimer's research and a member of our Scientific Advisory Board. Dr. Fillit is the Co-Founder and Chief Science Officer of the Alzheimer’s Drug Discovery Foundation, a Clinical Professor of Geriatric Medicine and Palliative Care at The Icahn School of Medicine, and has authored or co-authored over 300 publications. When discussing the future of Alzheimer's, Dr. Fillit shared, “This is a defining time for Alzheimer’s research with better diagnostics, a solid scientific understanding, and 120 drugs in clinical trials looking at novel treatments for Alzheimer’s disease.” Learn more about our Scientific Advisory Board: https://lnkd.in/g5hSexqd #AlzheimersCare #AlzheimersResearch #DementiaCare

Research Paper of the Week: Improving Access to General Practice for People with Severe and Multiple Disadvantage 🔍 Study Insights: This qualitative study, led by Lucy Potter at Centre for Academic Primary Care, reveals the power of co-production in creating improvements in patients with severe and multiple disadvantage (SMD). 🛠️ Key Innovations: - Inclusion Patient List: Prioritising patients with SMD for more flexible access and continuity through a care coordinator and a dedicated micro-team. - Information Sharing: Developing tools to ensure sensitive and efficient communication about patients' backgrounds and needs. - Trauma-Informed Care Training: For all practice staff, enhancing understanding and improving interactions with SMD patients. 📈 Looking Forward: These principles should be implemented across the country. The study also shows that meaningful engagement with patients and integration of their perspectives into service design improves care. Full article here https://lnkd.in/e3rRJKrT Tracey Stone Helen Cramer Helen McGeown Florrie C. Jeremy Horwood Gene Feder One25 #HealthcareInnovation #GeneralPractice #HealthEquity

Thanks Americas Committee for Treatment & Research in Multiple Sclerosis (ACTRIMS) and European Charcot Foundation, for fostering further global collaboration on Global PROMS Initiative, maximizing science with and for patient input. Together we are stronger! The time is right to: * Enable deep phenotyping of pwMS experiential knowledge for the use of appropriate Patient-Reported Outcome Measures (PROMs) towards personalized medicine. * Develop targeted approaches with relevant PROMs to ensure that clinical trials and clinical practice are aligned with patient values. * Enable digital technologies to capture PROMs changes over time. * Standardize the use of valid, reliable, and responsive PRO measures, via MS Registers data sharing, for their targeted – personalized – use. Special thanks to @Professor Mark Freedman, Americas Committee for Treatment & Research in Multiple Sclerosis (ACTRIMS) President, and Conference Chair Professor Daniel Ontaneda for his valuable expert partnership in the Global PROMS Initiative mission.

Very interesting tool to bridge #patients and #researchers towards patient engagement in I&I. #Impact European Commission #research #investigacao #inovacao #saude #medicina #hospitais #associacoesdoentes #cidadao #investigacaoclinica EATRIS European Patients' Forum #H2020 #EUproject #HEU

Want to know more about AI for breast cancer risk prediction? Register for the webinar Clinical Talks, on February 16, where Fredrik Strand, Docent and Radiologist at Karolinska Institutet tells more. Clinical talks is an educational seminar series open to everyone, organized by SciLifeLab. #lifescience #Hagastaden #cancer

Join our ECTRIMS Webinar today: Ageing and MS: Underlying Science and Considerations for Clinical Research and Care30 APRIL, 2024 | 16:00-17:00 CEST FREE REGISTRATION https://bit.ly/3Ukr1j5