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Clinical Trial News

Medidata CEO Outlines Vision for Clinical Trial Evolution: Balancing Innovation with Regulatory Compliance

  • Medidata's leadership emphasizes the need for measured innovation in clinical trials, highlighting the delicate balance between rapid advancement and patient safety in pharmaceutical development.
  • The company is actively engaging with regulatory bodies like FDA and EMA to implement patient-centric approaches, including e-consent solutions and synthetic control arms to improve trial efficiency.
  • Industry transformation is being driven by regulatory demands for comprehensive data, payer focus on value-based outcomes, and the emergence of potentially curative compounds requiring nimbler development processes.

Alder BioPharmaceuticals Presents New Data on Eptinezumab's Clinical Profile for Migraine Prevention

Alder BioPharmaceuticals has unveiled new data from post-hoc analyses of its PROMISE-1 and PROMISE-2 Phase 3 clinical trials for eptinezumab, highlighting its potential in preventing migraines. The data, focusing on migraine-free months, severity, and quality of life improvements, supports eptinezumab's clinical profile. The findings were presented at the American Headache Society’s 61st Annual Scientific Meeting.

Feasibility of Monitoring Compliance with Intermittent Occlusion Therapy Glasses for Amblyopia Treatment

A study investigated the feasibility of using a microsensor to monitor objective compliance with intermittent occlusion (IO) therapy glasses in children with unilateral amblyopia. The study found that compliance varied among individuals and slightly declined over time, with an average compliance of 51.6%. Despite this, visual acuity improved in patients, indicating the potential effectiveness of IO therapy glasses. The study highlights the importance of monitoring compliance in amblyopia treatment and suggests that IO therapy glasses could be a viable alternative to traditional patching methods.

Efficacy of Intermittent Occlusion Therapy Glasses for Amblyopia

NCT02687581TerminatedNot Applicable
Salus University
Posted 12/19/2016

Regimens of Intermittent Occlusion Therapy for Amblyopia in Children

NCT02767856TerminatedNot Applicable
Salus University
Posted 9/1/2016

Real-World Evidence Transforms Drug Development: FDA Embraces New Paradigm in Clinical Research

  • Real-world evidence (RWE) is revolutionizing drug development by providing crucial data from patient experiences outside traditional clinical trials, helping bring safer and more effective treatments to market.
  • The FDA has demonstrated strong commitment to RWE adoption, exemplified by Pfizer's Ibrance receiving expanded indication for male breast cancer based solely on real-world data.
  • RWE is being utilized across pharmaceutical functions, from informing clinical trial design to supporting market access decisions and value-based care initiatives worldwide.

SMi's 9th Annual Orphan Drugs Conference to Address Key Challenges in Rare Disease Treatment Access

• The 9th Annual Orphan Drugs & Rare Diseases Conference, scheduled for October 15-16 in London, will bring together experts to accelerate orphan drug development and improve patient access.
• Industry leaders, including NICE and Genetic Alliance UK representatives, will discuss critical issues such as early access programs, treatment value assessment, and manufacturer-payer dialogue.
• Conference co-chair Rick Thompson of Findacure emphasizes the event's focus on patient centricity and collaborative approaches in rare disease drug development.

Sequana Medical Receives FDA Approval for Alfapump Device Trial

Sequana Medical has obtained FDA approval for a pivotal study of its Alfapump device, aimed at treating patients with recurrent or refractory ascites due to liver cirrhosis. The study will enroll 60 patients in the U.S. and Canada, focusing on the device's effectiveness and safety.

Immunotherapy Drug Shows Potential to Cure Advanced Lung Cancer

A study involving Yale Cancer Center and Smilow Cancer Hospital researchers has shown that the immunotherapy drug pembrolizumab (Keytruda) significantly increases survival rates for patients with advanced non-small cell lung cancer (NSCLC), marking a significant advancement in lung cancer treatment.

Novartis Receives Patent for Novel NLRP3 Inflammasome Inhibitors with Potential for Multiple Inflammatory Diseases

  • Novartis AG has been granted US Patent 11208399B2 for a new class of NLRP3 inflammasome inhibitors featuring pyridazin-phenol compounds with potential therapeutic applications across multiple inflammatory conditions.
  • The patent covers specific chemical compounds designed to inhibit NLRP3 inflammasome signaling, a key pathway involved in inflammatory diseases including multiple sclerosis, cardiovascular disease, and various autoinflammatory disorders.
  • The compounds demonstrate activity against NLRP3-mediated inflammatory processes and may offer new treatment options for conditions where current therapies are inadequate or unavailable.
  • The patent includes detailed synthetic methods and pharmaceutical compositions, positioning Novartis to develop these compounds for clinical applications in inflammatory disease management.

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