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Clinical Trial News

Fox Chase Trial Explores Active Surveillance for Muscle-Invasive Bladder Cancer

• The RETAIN Bladder study investigates active surveillance as an alternative to total bladder removal for muscle-invasive bladder cancer (MIBC) patients responding to chemotherapy. • The trial employs a risk-adapted approach, utilizing clinical features and genetic mutations to predict chemotherapy effectiveness and tailor treatment recommendations. • Researchers aim to identify biomarkers that predict response to chemotherapy, potentially allowing some patients to avoid cystectomy and preserve bladder function. • The study assesses metastasis-free survival, bladder preservation rates, quality of life, and genomic correlates to refine bladder cancer treatment strategies.

UK Unveils New Five-Year Drug Pricing Agreement to Accelerate NHS Medicine Access

• The UK government and pharmaceutical industry have agreed to a new Voluntary Scheme for Branded Medicines Pricing and Access, replacing the 60-year-old PPRS with a 2% annual cap on branded medicine sales growth.
• The agreement promises to accelerate NICE appraisals by up to six months and includes £930 million in projected NHS cost savings for 2019 through pharmaceutical company rebates.
• New active substances will receive 36-month pricing freedom, while smaller companies will benefit from improved exemptions, aiming to maintain the UK's position as an attractive hub for life sciences research.

FDA Approves First Treatment for Primary Hemophagocytic Lymphohistiocytosis, Marking 24-Year Breakthrough

Monoclonal AntibodyDrug ApprovalRare DiseaseOrphan Drug
  • The FDA has approved emapalumab-lzsg (Gamifant) as the first treatment specifically indicated for primary hemophagocytic lymphohistiocytosis (HLH), an ultra-rare and life-threatening immune disorder.
  • In a pivotal phase 2/3 study of 27 patients with refractory disease, 63% demonstrated overall response and 70% proceeded to hematopoietic stem cell transplant.
  • The monoclonal antibody targets interferon gamma, representing the first significant improvement in primary HLH induction therapy in 24 years.
  • Primary HLH typically affects children within the first year of life and has a median survival of less than two months without treatment.

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

NCT01818492CompletedPhase 2
Swedish Orphan Biovitrum
Posted 7/1/2013

VR-CAP Regimen Demonstrates Superior Overall Survival in Mantle Cell Lymphoma

  • The VR-CAP regimen (bortezomib, rituximab, cyclophosphamide, doxorubicin, and prednisone) significantly extended overall survival compared to R-CHOP in transplant-ineligible MCL patients.
  • After a median follow-up of 82 months, VR-CAP showed a median overall survival of 90.7 months versus 55.7 months for R-CHOP (HR 0.66, p=0.001).
  • The VR-CAP regimen presented a manageable safety profile, supporting its consideration as a frontline treatment option for mantle cell lymphoma.

Study of the Combination of Rituximab, Cyclophosphamide, Doxorubicin, VELCADE, and Prednisone or Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone in Patients With Newly Diagnosed Mantle Cell Lymphoma

NCT00722137CompletedPhase 3
Millennium Pharmaceuticals, Inc.
Posted 5/1/2008

FDA Approves First Direct-to-Consumer Pharmacogenetic Test from 23andMe

Drug Approval
  • The FDA approved 23andMe's Personal Genome Service Pharmacogenetic Reports, marking the first direct-to-consumer genetic test to help patients understand how their genetics may affect responses to over 50 common prescription and over-the-counter drugs.
  • The test analyzes 33 genetic variants associated with drug metabolism, including responses to blood thinners like clopidogrel and mental health treatments, but comes with strict limitations prohibiting its use for treatment decisions or medical advice.
  • Medical experts express caution about the clinical utility of pharmacogenetic testing for general consumers, noting that while genetic variants affect drug metabolism, the clinical interpretation and prescription implications remain unclear for most patients.

Novartis Launches Biome Innovation Lab Network to Accelerate Digital Health Solutions

  • Novartis establishes the Novartis Biome, a digital health innovation lab network headquartered in San Francisco, to foster collaboration with healthcare technology startups and accelerate digital transformation.
  • The initiative includes the HealthX World Series challenges, with recent competitions focusing on heart failure solutions, where startups Veta Health and Wavy emerged as winners.
  • Participating startups will receive workspace, mentorship, access to Novartis datasets, and opportunities for proof-of-concept studies, without mandatory company ownership requirements.

Pfizer Strengthens Digital Strategy with New Chief Digital Officer Appointment

• Pfizer has appointed Lidia Fonseca as its new Chief Digital and Technology Officer, joining the executive leadership team in January to accelerate the company's digital capabilities.
• The appointment comes as part of a broader executive restructuring following Albert Bourla's succession as CEO, reflecting the growing importance of digital technology in pharmaceutical R&D and operations.
• The leadership reorganization includes new roles for key executives, with Mikael Dolsten overseeing medical operations, Angela Hwang leading innovative medicines, and Freda Lewis-Hall becoming Chief Patient Officer.

FDA Launches Cybersecurity Playbook to Protect Medical Devices from Digital Threats

  • The FDA has released a comprehensive cybersecurity playbook in collaboration with Mitre Corporation to help healthcare organizations protect medical devices and prepare for potential security breaches.
  • FDA Commissioner Scott Gottlieb warns that cyber threats to medical devices are no longer theoretical, with potential impacts on network-connected equipment like radiologic imaging devices.
  • The agency is establishing new partnerships through memoranda of understanding and creating a Centre of Excellence for Digital Health to strengthen medical device cybersecurity measures.

J&J's Continuous Manufacturing Journey: Pioneering Success with HIV Drug Prezista

  • Johnson & Johnson achieved a historic FDA approval in 2016 to transition HIV medication Prezista from batch to continuous manufacturing, marking a significant advancement in pharmaceutical production methods.
  • The continuous manufacturing system demonstrates superior efficiency through real-time quality monitoring, reduced waste, and flexible production capabilities, while maintaining the highest quality standards.
  • J&J's strategic decision to implement continuous manufacturing with an established product like Prezista, rather than a new drug, has provided valuable insights and lessons for future pharmaceutical manufacturing innovation.

Survey Reveals Gap Between Patient-Centric Aspirations and Implementation in Pharma Industry

  • New industry survey shows 91% of pharma professionals value patient-focused missions, yet only 22% understand how to effectively engage with patients.
  • Significant trust gap emerges as only 36% of patients express strong trust in the pharmaceutical industry, with notable disparities between employee and patient perceptions of patient-centric initiatives.
  • Industry leaders emphasize that connecting employees to their sense of purpose and patient impact is key to achieving true patient centricity and sustainable business outcomes.

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