Real-World Evidence
40 videos · last 6 months shuffled daily, then older videos newest first
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1:22:57 Engaging Patients and Communities in Clinical Research
Tracy Yu-Ping Wang, MD, MHS, MSc, Chief Officer for Comparative Effectiveness Research at PCORI, gives the 2026 UB CTSI Annual Forum keynote on engaging patients and communities in clinical research, with Q&A.
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40:31 Simons Searchlight: A Natural History Registry for 15q13.3
Cora Taylor, PhD, principal investigator of Simons Searchlight at Geisinger, explains the natural history study and registry for rare genetic conditions including 15q13.3, what enrolling involves and how researchers use the data, for Reed's Reach for 15q13.3.
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1:01:07 Beyond Patient Knowledge to Inform Decision-Making - Lessons from HTA
Dr Karen Facey and Dr Anke-Peggy Holtorf discuss how patient involvement in health technology assessment has evolved and how patient-based evidence can inform medicines evaluation, reimbursement and access, in a Consilium Scientific seminar.
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32:38 Getting 15q13.3 Ready for Clinical Trials
Ingo Helbig, MD, of Children's Hospital of Philadelphia and the University of Pennsylvania describes seizures in 15q13.3 and what it takes to make the condition trial-ready, from natural history data to EEG biomarkers, for Reed's Reach for 15q13.3.
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57:07 How Genomic Data Improves Drug Development Timelines and Success
A webinar from GeneDx explores how integrating clinical-grade genomic data with longitudinal insights can fuel drug discovery, accelerate precise clinical trials, advance HEOR research, and generate evidence to deliver value across the drug development lifecycle.
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36:36 Oracle's Chris Boone on AI in Drug Development
Chris Boone, Group Vice President of Research and Services for Life Sciences at Oracle and formerly of Pfizer, discusses generative AI in drug development, real-world evidence researchers, AI governance and digital twins, on Massive Bio's Massively Insightful.
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7:44 CheckRare on MAVORIC Trial Results in Cutaneous T-Cell Lymphoma
H. Miles Prince, MD, of Peter MacCallum Cancer Centre compares results from the MAVORIC trial of mogamulizumab against vorinostat with outcomes recorded in an Australian cancer registry, in cutaneous T-cell lymphoma.
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10:15 BfArM and EMA: Data, AI and Medicines Regulation in Europe
Prof. Dr. Karl Broich, President of BfArM, and Dr. Peter Arlett, Head of the Data Analytics and Methods Task Force at the European Medicines Agency, discuss AI, real-world data and the European Health Data Space in medicines regulation.
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18:40 What Is Real-World Evidence Revealing About CAR T Therapy?
Prof Benoit Tessoulin of CHU Nantes discusses what the French DESCAR-T registry shows about CAR T-cell therapy in routine practice, including patient selection, product comparisons and toxicities, on a European Hematology Association podcast.
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13:27 Prader-Willi Syndrome: Diazoxide Choline Extended Release Improves Hyperphagia
Evelien Gevers, PhD, MD, of Barts Health NHS Trust on the effect of diazoxide choline extended release on hyperphagia in patients with Prader-Willi syndrome.
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46:46 How Patient Data Drives FSHD Research
Kayleigh Worek, MS, of the FSHD Society explains how patient registries, surveys and natural history studies contribute to FSHD research, inform drug development and shape healthcare policy.
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1:01:27 Evidence Beyond FEV1 for Respiratory Biologics
A Lindus Health panel discusses designing Phase 3b, Phase 4 and prospective real-world evidence studies for asthma and COPD biologics that meet payor and HTA needs, including remote spirometry and digital biomarkers.
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52:43 From Pathology to Biotech: Unlocking Impact Beyond Patient Care - Beyond Outcomes E4
On GCP-Mindset's Beyond Outcomes, Michelle Mudge-Riley describes moving from pathology into biotech leadership and discusses development pathways, patient-reported outcomes, bridging strategies and real-world evidence.
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2:19:14 GCP-Mindset on Evidence Standards in European Medical Device Regulation
Andreas Beust talks to Prof. Tom Melvin about whether European medical device regulation gives innovators enough clarity, covering early regulatory advice, randomized trials, registries, real-world evidence, orphan and paediatric devices, and what Europe might take from the US, Japan and the UK.
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8:17 Mogamulizumab in Danish Patients With CTCL
Lena Specht, MD, DMSc, of the University of Copenhagen on a Danish study of mogamulizumab in cutaneous T-cell lymphoma, approved in Europe in 2018 on the MAVORIC results.
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28:41 Inside EMA: Data Trust and the Future of Medicines
EMA's Peter Arlett and Denise Umuhire explain how real-world evidence from health records complements clinical trials in EU medicines regulation, including the DARWIN EU network.
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35:29 Elli Brimble on Natural History Data for ZTTK Research
Elli Brimble, Head of Clinical Strategy at Citizen Health, updates the ZTTK Citizen natural history study, now at 65 participants, and explains how FOXG1 data became a control for a placebo-free gene therapy trial, for the ZTTK SON-Shine Foundation.
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9:28 Obesity With Mortality and Bleeding in Cancer-Associated VT
Álvaro Dubois-Silva, MD, and Marijan Bosevski, MD, PhD, discuss their Mayo Clinic Proceedings cohort study from the RIETE registry linking obesity to lower mortality and higher bleeding risk in cancer-associated venous thromboembolism.
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4:42:24 Duke-Margolis on the State of Real-World Evidence Policy
The Duke-Margolis Real-World Evidence Collaborative convenes its annual meeting on improving how real-world data is developed and used in health care decisions.
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58:14 U.S. Food and Drug Administration Webinar on the RCT-DUPLICATE Initiative
An FDA webinar on RCT-DUPLICATE, one of the agency's demonstration projects, which tests when non-randomized studies of real-world data reach the same conclusions as randomized trials.
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58:55 Using External Data in RCTs Without Introducing Bias
Unlearn founder Charles Fisher describes using existing data and predicted placebo outcomes in randomised controlled trials to run smaller trials while keeping control of bias.
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1:15:05 Designing and Conducting Observational Studies with Thomas Newman, MD, MPH
Dr. Thomas Newman (UCSF Professor & co-author of Designing Clinical Research) breaks down how to design, analyze, and interpret observational studies without falling into common traps like bias and confounding.
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59:30 Kaftrio and the CFTR Modulator Trials in Cystic Fibrosis
Dr Ian Balfour-Lynn, consultant in paediatric respiratory medicine at the Royal Brompton Hospital, reviews the clinical trials that led to licensing of CFTR modulators such as Kaftrio and real-world experience with them, for the Genomics Education Programme.
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1:00:00 Arterial Health Biomarkers in Drug Development
A Cardiex webinar explains how a wearable device measuring central blood pressure and pulse wave analysis can provide arterial-stiffness biomarkers and remote real-world evidence for drug development and cardiovascular trials.