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Real-World Evidence Validates Proenkephalin A 119-159 as Superior Kidney Function Biomarker in Critical Care

Real World Evidence
  • A comprehensive real-world study of over 4,000 ICU patients demonstrates that Proenkephalin A 119-159 (penKid) significantly outperforms traditional creatinine markers in detecting acute kidney injury and assessing kidney function.
  • PenKid successfully identifies high-risk patients at ICU admission, particularly those with normal serum creatinine levels, and predicts AKI development within 24-48 hours more accurately than conventional methods.
  • The biomarker maintains its diagnostic effectiveness during renal replacement therapy, providing dynamic insights into kidney recovery that could optimize treatment duration and reduce unnecessary interventions.
  • International recognition from Mayo Clinic researchers confirms penKid's clinical value, highlighting its ability to detect rapid kidney function changes and predict major adverse kidney events in critically ill patients.

Real-World Evidence Oncology Market Projected to Reach $3.51 Billion by 2035 with 14.7% CAGR

Real World EvidenceOncologyAIPrecision Medicine
  • The real-world evidence oncology solutions market is projected to grow from $893 million in 2025 to $3.51 billion by 2035, representing a compound annual growth rate of 14.7%.
  • Market growth is driven by increasing demand for real-world evidence in drug development, rising focus on value-based healthcare, growing global cancer incidence, and advancements in data analytics and AI technologies.
  • North America is expected to hold the largest market share in 2025, while the Asia-Pacific region is projected to experience the fastest growth during the forecast period.
  • The datasets segment is expected to account for the largest market share, while pharmaceutical, biotechnology, and medical device companies represent the primary end-user segment.

Decade-Long UK Registry Reveals Key Insights into Idiopathic Pulmonary Fibrosis Management and Future Directions

Real World Evidence
  • The British Thoracic Society's analysis of the UK Idiopathic Pulmonary Fibrosis Registry provides the first comprehensive long-term data on over 5,000 IPF cases across 64 UK hospitals.
  • Key findings include significant diagnostic delays with 60% of patients experiencing symptoms for over a year before their first clinic visit, and a shift in antifibrotic treatment preferences from pirfenidone to nintedanib after 2017.
  • Experts are now advocating for a shift from consensus-based criteria toward personalized, data-driven approaches for early diagnosis and treatment, with initiatives like the global PRIME-PPF study aiming to refine risk stratification.

Real-World Studies Confirm Bevacizumab Biosimilars Deliver Cost Savings and Equivalent Clinical Outcomes in Metastatic Colorectal Cancer

Monoclonal AntibodyReal World EvidenceOncology
  • A Canadian population-based study found bevacizumab biosimilars Mvasi and Zirabev provided $6,379 CAD in annual cost savings per patient compared to originator Avastin while maintaining equivalent survival outcomes in metastatic colorectal cancer.
  • An Indian real-world analysis of 944 patients demonstrated comparable progression-free survival and overall survival between bevacizumab biosimilars and the reference product in first-line and second-line treatment settings.
  • Both studies support the cost-effectiveness and clinical equivalence of bevacizumab biosimilars, providing evidence for healthcare policy decisions and building confidence among oncologists treating metastatic colorectal cancer.

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.

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

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

NCT00934544CompletedPhase 3
Novartis Pharmaceuticals
Posted 7/1/2009

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