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Rituximab Outperforms Cladribine in Real-World Multiple Sclerosis Study

Monoclonal AntibodyReal World Evidence
  • A Norwegian observational study of 285 RRMS patients found rituximab demonstrated superior disease control compared to cladribine, with only 17% showing new MRI activity versus 57% for cladribine over 4 years.
  • Rituximab-treated patients experienced significantly lower relapse rates (6% vs 17%) and treatment discontinuation rates (7% vs 21%), with patients remaining free from new MRI activity for an average of 16.8 months longer.
  • The study showed rituximab patients had better disability improvement outcomes (21% vs 4%) and were 15.9 times more likely to achieve NEDA-3 status, though hospitalization rates for adverse effects were comparable between treatments.
  • Both treatments demonstrated similar safety profiles overall, with no deaths reported and comparable infection rates, though more rituximab patients were hospitalized for COVID-19 during the pandemic period.

Merit Medical Enrolls First Patient in WRAPSODY Registry for Hemodialysis Vascular Access

FDA ApprovalReal World Evidence
  • Merit Medical Systems has enrolled the first patient in its WRAP North America registry, a prospective multicenter study designed to collect real-world safety and effectiveness data on the WRAPSODY Cell-Impermeable Endoprosthesis for hemodialysis patients.
  • The registry will enroll up to 250 US and Canadian hemodialysis patients experiencing vascular access obstructions such as stenosis or occlusion, with clinical outcomes evaluated over three years.
  • The WRAPSODY CIE received FDA premarket approval in December 2024 and Health Canada approval in April 2025, following superior performance demonstrated in the WAVE pivotal trial.
  • If completed as designed, this registry would represent the largest cohort of patients treated with an implantable device to restore vascular access for hemodialysis.

Merit WRAPSODY™ Endoprosthesis for Treatment of Stenosis or Occlusion (WRAP)

NCT05062291Recruiting
Merit Medical Systems, Inc.
Posted 6/23/2022

WRAP North America

NCT06807099Recruiting
Merit Medical Systems, Inc.
Posted 7/31/2025

TransCelerate and FDA Release Framework to Scale Pragmatic Clinical Trials in Routine Care Settings

Real World Evidence
  • TransCelerate BioPharma and FDA's CDER Center for Clinical Trial Innovation released a summary report from a December 2024 tabletop exercise involving over 30 pharmaceutical R&D leaders and eight FDA offices to advance pragmatic trial implementation.
  • The collaboration identified key opportunities to integrate clinical research into routine care settings through simplified designs, broader patient populations, and electronic health record integration to address unmet clinical needs.
  • A new resource guide, "Getting Started with Embedded Pragmatic Trials," provides practical implementation guidance for stakeholders seeking to incorporate pragmatic elements into clinical trial designs.
  • The initiative supports FDA's Streamlined Trials Embedded in clinical Practice (STEP) program and aims to improve patient access while generating reliable real-world evidence from care settings.

Atropos Health and Novartis Partner to Deploy AI for Faster Rare Disease Diagnosis in PNH Patients

AIRare DiseaseReal World EvidencePrecision Medicine
  • Atropos Health has partnered with Novartis to develop AI models that identify undiagnosed patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder affecting 10-20 people per million worldwide.
  • The collaboration addresses critical diagnostic delays in PNH, where many patients wait over a year for diagnosis and some wait more than five years due to the disease's rarity and varied symptoms.
  • The AI model, trained on real-world data from the Atropos Evidence Network, is now available for integration into health systems to accelerate appropriate diagnosis at the point of care.
  • The partnership aims to reduce time from initial symptoms to testing, diagnosis and treatment by implementing patient-finding models across health system members of the Atropos Evidence Network.

PSMA PET/CT Before Salvage Radiotherapy Improves Survival in Recurrent Prostate Cancer

Real World Evidence
  • A Danish nationwide study of 844 patients demonstrates that PSMA PET/CT imaging before salvage radiotherapy significantly improves overall survival rates in men with biochemically recurrent prostate cancer.
  • Patients who underwent PSMA PET/CT showed superior five-year survival rates of 98.1% compared to 93.8% for those who did not receive the imaging.
  • The study provides the first real-world evidence that PSMA PET/CT-guided treatment selection leads to improved clinical outcomes in salvage radiotherapy patients.
  • These findings support broader adoption of PSMA PET/CT as a standard imaging modality for treatment planning in recurrent prostate cancer cases.

Caris Life Sciences Study Reveals Optimal Sequencing Strategy for Antibody-Drug Conjugates in HER2-Negative Breast Cancer

Precision MedicineReal World Evidence
  • Caris Life Sciences published a real-world evidence study in Breast Cancer Research comparing trastuzumab deruxtecan and sacituzumab govitecan in over 4,000 HER2-negative breast cancer patients.
  • The study found that trastuzumab deruxtecan demonstrated superior treatment duration for hormone receptor-positive patients, while sacituzumab govitecan showed better first-line results in HR-negative and HER2-null tumors.
  • Both antibody-drug conjugates provided comparable benefits in triple-negative breast cancer patients, highlighting the importance of personalized treatment strategies based on tumor subtype.
  • The research addresses the absence of head-to-head clinical trials between the two most commonly used ADCs in breast cancer treatment.

Real-World Study Confirms High-Volume Intravitreal Injections Do Not Increase Glaucoma Risk

Real World Evidence
  • A large retrospective multicenter study of 2,405 eyes found no increased risk of glaucoma or ocular hypertension with high-volume intravitreal agents avacincaptad pegol, pegcetacoplan, and aflibercept 8mg compared to standard 50 µL injections.
  • The study analyzed treatment-naïve patients with neovascular age-related macular degeneration over 18-24 months, showing stable intraocular pressure across all treatment groups with differences within 1 mmHg.
  • These real-world findings corroborate clinical trial data and provide reassurance for clinicians using the three FDA-approved high-volume therapies that entered the market in 2023.

Large-Scale Study Confirms Tamiflu Safety in Children, Debunks Neuropsychiatric Risk Concerns

Real World Evidence
  • A comprehensive study of over 692,000 children found that Tamiflu reduces neuropsychiatric events by 50% compared to untreated influenza, contradicting long-standing safety concerns.
  • The research analyzed Tennessee Medicaid data from 2016-2020, including 129,000 children treated for influenza, with 67% receiving oseltamivir treatment.
  • Findings published in JAMA Neurology demonstrate that influenza itself, not Tamiflu treatment, is associated with increased risk of seizures, mood disorders, and self-harm behaviors.
  • The study provides reassurance to healthcare providers and parents about Tamiflu's safety profile when used early in pediatric influenza treatment.

SOPHiA GENETICS and AstraZeneca Expand AI Partnership to Optimize Breast Cancer Treatment Outcomes

AIReal World Evidence
  • SOPHiA GENETICS and AstraZeneca announced a multi-year collaboration expansion to leverage multimodal AI Factories for generating evidence on breast cancer therapy efficacy and real-world impact.
  • The partnership will utilize AI to analyze genomics, imaging, and clinical data to develop predictive models aimed at optimizing treatment outcomes for breast cancer patients.
  • The collaboration will focus on real-world evidence generation across Europe and North America to identify key drivers of treatment efficacy and enhance clinical decision-making.
  • AstraZeneca will integrate SOPHiA GENETICS's AI platform to augment their frontier AI solutions across oncology clinical development and improve understanding of disease biology.

Large Real-World Study Questions Effectiveness of Immunoglobulin Replacement Therapy in CLL Patients

Real World Evidence
  • A retrospective analysis of 6,217 CLL patients in Australia found that regular immunoglobulin replacement therapy was not associated with reduced risk of serious infections requiring hospitalization.
  • Despite increasing use of immunoglobulin therapy over the 14-year study period, serious infection rates doubled from 1.9% to 3.9%, with higher infection frequency during treatment periods compared to off-treatment intervals.
  • The study revealed that 45.9% of patients who started immunoglobulin therapy died during follow-up, with median survival of approximately six years from first treatment.
  • Researchers call for better clinical guidelines given the therapy's high cost, patient burden, and limited evidence of effectiveness in preventing serious infections.

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