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CVAC System Demonstrates Superior Stone Clearance in Multiple Real-World Studies at AUA 2025

Real World Evidence
• Real-world data presented at AUA 2025 shows the CVAC System achieves significant kidney stone volume reduction of up to 99% in most patients, with one study reporting a median clearance rate of 96.4%.
• The CVAC System demonstrated superior performance compared to flexible and navigable ureteric access sheath (FANS), removing significantly more absolute stone volume while maintaining consistently low intrarenal pressure.
• Multiple studies using Quantitative Stone Analysis Software (QSAS) validate stone volume as a more precise and clinically meaningful measure for assessing kidney stone treatment outcomes.

Fremanezumab Demonstrates Long-Term Efficacy in Migraine Prevention Across Multiple Real-World Studies

Monoclonal AntibodyReal World Evidence
  • The phase 4 PEARL study involving 1,140 patients confirmed fremanezumab's long-term effectiveness, with 66% of episodic migraine patients and 51.6% of chronic migraine patients achieving at least 50% reduction in monthly migraine days over 24 months.
  • A phase 2b/3 trial in Japan and Korea showed fremanezumab significantly reduced acute medication use by approximately 2.8 days per month and decreased migraine-associated symptoms including nausea, vomiting, and light sensitivity.
  • Real-world evidence demonstrates high treatment adherence with over 75% of patients completing the 2-year study duration, while 25.9% of patients were able to discontinue treatment due to sustained improvement.
  • Teva Pharmaceuticals is seeking FDA approval to expand fremanezumab's indication to pediatric patients aged 6-17 years, supported by positive results from the phase 3 SPACE study.

Fremanezumab Compassionate Use Program for Pediatric Patients

NCT03539393available
Teva Branded Pharmaceutical Products R&D, Inc.

UroLift System Demonstrates Superior Patient Outcomes in Head-to-Head BPH Treatment Trials

Real World Evidence
  • New clinical data presented at the 2025 AUA Annual Meeting shows the UroLift System provides better early patient satisfaction and faster symptom relief compared to Rezūm and tamsulosin for BPH treatment.
  • Two randomized controlled trials revealed UroLift patients experienced shorter catheterization times, better sexual function outcomes, and higher overall satisfaction during early recovery periods.
  • Real-world data from the American Urological Association Quality Registry confirms UroLift provides rapid symptom improvement at three months, reinforcing its position as a leading minimally invasive BPH treatment option.

EHR Data Emerges as Critical Source for Real-World Evidence Generation in Drug Development

Real World EvidenceAI
  • Electronic health record (EHR) data is becoming increasingly valuable for generating real-world evidence (RWE) in therapy development, offering clinical context that claims data cannot provide.
  • Artificial intelligence and machine learning are essential for processing large volumes of EHR data, with rigorous quality control processes needed to prevent biases and ensure accuracy.
  • A recent ophthalmology study using faricimab for neovascular age-related macular degeneration demonstrated how curated RWD can reveal treatment patterns and reduce injection frequency in real-world settings.
  • The combination of structured and unstructured EHR data with advanced AI tools is positioning real-world evidence as a complement to traditional clinical trial data in regulatory decision-making.

Real-World Evidence Shows Apixaban Reduces Major Bleeding Risk Compared to Rivaroxaban in Atrial Fibrillation Patients

Real World Evidence
  • Multiple large-scale real-world studies consistently demonstrate that apixaban is associated with 31-45% lower risk of major bleeding events compared to rivaroxaban in patients with non-valvular atrial fibrillation.
  • The COBRA-AF study, analyzing 177,866 propensity-matched patient pairs, found apixaban reduced the composite risk of major and clinically relevant non-major bleeding by 31% versus rivaroxaban.
  • Apixaban showed superior safety profiles across diverse patient populations, including those with chronic liver disease and bleeding history, while maintaining comparable stroke prevention efficacy.

Ruxolitinib Maintains Clinical Benefit in Myelofibrosis Patients Despite Treatment-Related Anemia

Real World Evidence
  • Post-hoc analysis of the phase 3b JUMP trial demonstrates that ruxolitinib maintains clinical benefit in myelofibrosis patients who develop new or worsening anemia during treatment.
  • The study found no significant difference in overall survival between patients with and without new or worsening anemia, with median OS of 58.3 months versus not evaluable in baseline anemic patients.
  • Results from the largest ruxolitinib trial in myelofibrosis support continued use of the drug regardless of baseline anemia status or development of treatment-related anemia.
  • Findings validate previous COMFORT trial results and provide real-world evidence for ruxolitinib's sustained efficacy across diverse patient populations including those with lower platelet counts.

Controlled Myelofibrosis Study With Oral Janus-associated Kinase (JAK) Inhibitor Treatment-II: The COMFORT-II Trial

NCT00934544CompletedPhase 3
Novartis Pharmaceuticals
Posted 7/1/2009

INC424 for Patients With Primary Myelofibrosis, Post Polycythemia Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis.

NCT01493414CompletedPhase 3
Novartis Pharmaceuticals
Posted 8/16/2011

COntrolled MyeloFibrosis Study With ORal JAK Inhibitor Treatment: The COMFORT-I Trial

NCT00952289CompletedPhase 3
Incyte Corporation
Posted 8/1/2009

Kite's Yescarta Shows Superior Manufacturing Success and T-Cell Performance in Second-Line Treatment of Large B-Cell Lymphoma

CAR-TReal World Evidence
  • Analysis of 4,175 patients demonstrates that Yescarta achieves significantly higher first-pass manufacturing success rates when used as second-line therapy (95.08%) versus third-line treatment (92.48%) for relapsed/refractory large B-cell lymphoma.
  • Patients receiving second-line treatment showed approximately twice as many naïve-like T-cells during leukapheresis collection compared to third-line patients, potentially supporting improved therapeutic outcomes.
  • Preliminary data from ZUMA-24 and real-world studies suggest outpatient administration of Yescarta and Tecartus is feasible without added toxicity when appropriate monitoring is in place.

Real-World Teclistamab Data Shows Comparable Efficacy to Clinical Trials Despite Higher Toxicity Rates in Multiple Myeloma

CAR-TReal World Evidence
  • A multicenter real-world study of 110 patients treated with teclistamab demonstrated a 62% overall response rate and 51% very good partial response rate, comparable to the pivotal MajesTEC-1 trial despite treating a more heavily pretreated population.
  • The incidence of severe cytokine release syndrome and neurotoxicity was substantially higher in real-world practice (3.6% and 4.5% respectively) compared to clinical trials (0.6% each), likely due to higher disease burden in routine practice.
  • Primary intravenous immunoglobulin prophylaxis significantly reduced infection rates by approximately 70%, with patients receiving IVIG showing lower cumulative incidence of grade 3 or higher infections compared to those without prophylaxis.
  • Analysis of FDA adverse event data across BCMA-targeted therapies revealed teclistamab had the highest rates of life-threatening events and death, with infections being the predominant cause of non-relapse mortality.

A Study of Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT04557098Active, Not RecruitingPhase 2
Janssen Research & Development, LLC
Posted 9/17/2020

Dose Escalation Study of Teclistamab, a Humanized BCMA*CD3 Bispecific Antibody, in Participants With Relapsed or Refractory Multiple Myeloma

NCT03145181Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 5/16/2017

Combination Therapy Shows Promise for Chronic GVHD as Real-World Data Confirms Ruxolitinib's Sustained Activity

Real World Evidence
  • Real-world data from 471 patients demonstrates that ruxolitinib maintains sustained activity in chronic graft-versus-host disease, with 33.1% of patients remaining on treatment for a median duration of 389 days.
  • A small study of 20 patients revealed that combination therapy with ruxolitinib plus belumosudil achieved a 55% overall response rate, including responses in patients who had previously failed monotherapy with either agent.
  • The combination therapy showed potential synergistic effects by targeting different inflammatory pathways, with all responding patients able to reduce or discontinue other immunosuppressive agents.
  • Dose adjustments were common in real-world practice, with 58.4% of patients requiring modifications, primarily dose increases, at a median time of 82 days after initiation.

A Study of Ruxolitinib vs Best Available Therapy (BAT) in Patients With Steroid-refractory Chronic Graft vs. Host Disease (GvHD) After Bone Marrow Transplantation (REACH3)

NCT03112603CompletedPhase 3
Incyte Corporation
Posted 6/29/2017

Treatment Of Steroid-Refractory Acute Graft-versus-host Disease With Mesenchymal Stromal Cells Versus Best Available Therapy

NCT04629833RecruitingPhase 3
medac GmbH
Posted 8/16/2021

Activity, Safety and Pharmacokinetics in Pediatric Subjects With Moderate and Severe Chronic Graft vs. Host Disease After Allogeneic Stem Cell Transplant

NCT03774082CompletedPhase 2
Novartis Pharmaceuticals
Posted 5/20/2020

Trial of Efficacy and Safety of MC0518 Versus Best Available Therapy in Participants With Steroid-Refractory Acute Graft Versus Host Disease

NCT06075706RecruitingPhase 2
medac GmbH
Posted 11/13/2023

Study of Pharmacokinetics, Activity and Safety of Ruxolitinib in Pediatric Patients With Grade II-IV Acute Graft vs. Host Disease

NCT03491215CompletedPhase 1
Novartis Pharmaceuticals
Posted 2/21/2019

South Korea Advances Real-World Evidence Integration in Drug Development and Regulatory Decision-Making

Real World Evidence
  • South Korea's Ministry of Food and Drug Safety has issued new guidelines for using real-world evidence in regulatory decision-making, focusing on clinical trial design and post-market surveillance.
  • The MFDS published the Guideline for Medical Information Database Research in June 2021, emphasizing the use of national health insurance data and electronic medical records for post-marketing safety studies.
  • The Korea Comprehensive Plan for Drug Safety Management (2020-2024) outlines plans to build a local ecosystem leveraging innovative technology, including real-world evidence, to support new drug development.
  • Healthcare claims databases are increasingly being utilized to generate real-world evidence for effectiveness and safety assessments of clinical therapeutics in South Korea.

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